Stem Cell Transplantation in Patients With High-Risk and Recurrent Pediatric Sarcomas

• Sponsor: National Cancer Institute (NCI) (NIH)
• Overall Status: Completed
• CT.gov ID: NCT00043979
• Collaborator: (none)
• Enrollment: 60
• Location: 1
• Arms: 2
• Actual Duration (Months): 110.8
• Patients Per Site Per Month: 0.5

Study Details

Study Description

Brief Summary

This study will examine the safety and effectiveness of stem cell transplantation for treating patients with sarcomas (tumors of the bone, nerves, or soft tissue). Stem cells are immature cells in the bone marrow and blood stream that develop into blood cells. Stem cells transplanted from a healthy donor travel to the patient's bone marrow and begin producing normal cells. In patients with certain cancers, such as leukemia and lymphoma, the donor's immune cells attack the patient's cancer cells in what is called a "graft-versus-tumor" effect, contributing to cure of the disease. This study will determine whether this treatment can be used successfully to treat patients with sarcomas.

Patients between 4 and 35 years of age with a sarcoma that has spread from the primary site or cannot be removed surgically, and for whom effective treatment is not available, may be eligible for this study. Candidates must have been diagnosed by the age of 30 at the time of enrollment. They must have a matched donor (usually a sibling). Participants undergo the following procedures:

Donors: Stem cells are collected from the donor. To do this, the hormone granulocyte colony stimulating factor (G-CSF) is injected under the skin for several days to move stem cells out of the bone marrow into the bloodstream. Then, the cells are collected by apheresis. In this procedure the blood is drawn through a needle placed in one arm and pumped into a machine where the stem cells are separated out and removed. The rest of the blood is returned to the donor through a needle in the other arm.

Patients: For patients who do not already have a central venous catheter (plastic tube), one is placed into a major vein. This tube can stay in the body the entire treatment period for giving medications, transfusing blood, , withdrawing blood samples, and delivering the donated stem cells. Before the transplant procedure, patients receive from one to three cycles of "induction" chemotherapy, with each cycle consisting of 5 days of fludarabine, cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone followed by at least a 17-day rest period. All the drugs are infused through the catheter except prednisone, which is taken by mouth. After the induction therapy, the patient is admitted to the hospital for 5 days of chemotherapy with high doses of cyclophosphamide, melphalan, and fludarabine. Two days later, the stem cells are infused. The anticipated hospital stay is about 3 weeks, but may be longer if complications arise. Patients are discharged when their white cell count is near normal, they have no fever or infection, they can take sufficient food and fluids by mouth, and they have no signs of serious graft-versus-host disease (GVHD)-a condition in which the donor's cells "see" the patient's cells as foreign and mount an immune response against them.

After hospital discharge, patients are followed in the clinic at least once or twice weekly for a medical history, physical exam, and blood tests for 100 days. They receive medications to prevent infection and GVHD and, if needed, blood transfusions. If GVHD has not developed by about 120 days post transplant, patients receive additional white cells to boost the immune response. After 100 days, follow-up visits may be less frequent. Follow-up continues for at least 5 years. During the course of the study, patients undergo repeated medical evaluations, including blood tests and radiology studies, to check on the cancer and on any treatment side effects. On four occasions, white blood cells may be collected through apheresis to see if immune responses can be generated against the sarcomas treated in this study. Positron emission tomography (PET) scans may be done on five occasions. This test uses a radioactive material to produce images useful in detecting primary tumors and cancer that has spread.

Condition or Disease	Intervention/Treatment	Phase
Sarcoma	Drug: F-18 Fluorodeoxyglucose; Biological: therapeutic allogeneic lymphocytes; Drug: cyclophosphamide; Drug: cyclosporine; Drug: doxorubicin hydrochloride; Drug: etoposide; Drug: fludarabine phosphate; Drug: melphalan; Drug: prednisone; Drug: sirolimus; Drug: tacrolimus; Drug: vincristine sulfate; Procedure: peripheral blood stem cell transplantation; Drug: Filgrastim; Procedure: Peripheral Blood Stem Cell donation	Phase 2

Detailed Description

Background:

• Treatment of pediatric sarcomas has enjoyed progress in the past 25 years for patients with localized, chemosensitive disease and prognostic factors are now available to identify subsets of patients who have very dismal prognoses; patients with primary metastatic disease, especially those with bone and bone marrow metastases.

• Patients with primary chemoresistant disease and early recurrence also have very poor prognoses and lack suitable treatment options. For these patients, it is critical that alternative approaches to cytotoxic chemotherapy be identified.

• Basic laboratory studies have shown that Ewing's sarcoma is susceptible to immune mediated mechanisms of cytolysis in vitro. Interestingly, for Ewing's sarcoma this appears to be true for both chemosensitive and chemoresistant cell lines.

• Recent progress in the field of bone marrow transplantation has identified approaches that can reproducibly induce allogeneic peripheral blood stem cell engraftment in adults with hematologic malignancies. In some cases, this same approach has shown beneficial effects for patients with solid tumors as a result of the development of allogeneic, immune-mediated graft versus tumor effects.

Objectives:

• To determine if the transplantation of human leukocyte antigen (HLA) matched, peripheral blood stem cells can result in full donor engraftment (greater than 95 percent by day

1. in patients with high risk-pediatric sarcomas.

• To identify and characterize the toxicities of HLA-matched peripheral blood stem cell transplant (PBSCT) in patients with high-risk pediatric sarcomas. In particular we will identify the incidence of graft versus host disease (GVHD) and the pace of immune reconstitution in this population.

• To determine if allogeneic graft-versus-tumor responses following allogeneic PBSCT can induce clinically significant anti-tumor effects as measured by radiographic evidence of antitumor responses following PBSCT in patients with measurable disease and improved clinical outcome compared to historical controls in this patient population with a universally poor outcome.

Eligibility:

• Patients, age of greater than 4 years at enrollment to less than 30 years at diagnosis and age less than 35 at enrollment, with ultra-high risk Ewing's sarcoma family of tumors, desmoplastic small round cell tumor or alveolar rhabdomyosarcoma.

• Patients must have completed standard front-line therapy and salvage therapy.

• Patient must have the availability of a 5 or 6 antigen HLA-matched first-degree relative donor or a genotypically identical twin.

• Patients must have adequate cardiac, pulmonary, renal, liver, and marrow function.

Design:

-Donor will be prepared for peripheral blood stem cell harvest with Filgrastim mobilization, 10 microg/kg per day subcutaneous (SQ) for 5-7 days until they have stem cell collected by apheresis. The stem cells will then be cryopreserved.

Patients will receive 1 to 3 21 day cycles of Fludarabine-EPOCH induction chemotherapy. The preparative regimen will consist of cyclophosphamide, fludarabine and melphalan followed by stem cell infusion. GVHD prophylaxis will consist of sirolimus and tacrolimus.

Study Design

• Study Type: Interventional
• Actual Enrollment: 60 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Pilot Study of Allogeneic/Syngeneic Blood Stem Cell Transplantation in Patients With High-Risk and Recurrent Pediatric Sarcomas
• Actual Study Start Date: Sep 19, 2002
• Actual Primary Completion Date: May 1, 2009
• Actual Study Completion Date: Dec 14, 2011

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Arm 1- Sibling Donors Donors (n = 30) were matched first degree relatives who were eligible to donate peripheral blood stem cells.	Drug: Filgrastim; Procedure: Peripheral Blood Stem Cell donation
Experimental: Arm 2 - Recipients Recipients (n=30) were enrolled to receive peripheral blood stem cells (PBSC) and receive either cyclosporine or tacrolimus and sirolimus for graft versus host disease (GVHD) prophylaxis.	Drug: F-18 Fluorodeoxyglucose; Other Names: FDG ; Biological: therapeutic allogeneic lymphocytes; Lymphocyte cells are collected from a healthy donor by apheresis and infused into the patient with a central venous catheter.; Drug: cyclophosphamide; Induction - 750 mg/m^2 intravenous (IV) infusion over 30 minutes x 1 dose. Day 5. Transplant - 1200 mg/m^2 per day IV infusion over 2 hours daily for 4 days; days -6, -5, -4, -3.; Other Names: Cytoxan ; Drug: cyclosporine; 6 mg/kg per dose orally every other day (no day 9 dose).; Other Names: Sandimmune ; Drug: doxorubicin hydrochloride; Induction - 10 mg/m^2 per day continuous intravenous (IV) infusion over 24 hours daily for 4 days. Days 1, 2, 3, 4.; Other Names: Adriamycin ; Drug: etoposide; 50 mg/m^2 per day continuous intravenous (IV) infusion over 24 hours daily for 4 days. Days 1, 2, 3, 4.; Other Names: Vepesid ; Drug: fludarabine phosphate; Induction - 25 mg/m^2 per day intravenous (IV) infusion over 30 minutes daily for 3 days. Days 1, 2, 3. Transplant - 30 mg/m^2 per day IV infusion over 30 minutes daily for 4 days; days -6, -5, -4, -3.; Other Names: Fludara ; Drug: melphalan; Transplant - 100 mg/m^2 per day intravenous (IV) infusion over 15 minutes for 1 day; day -2.; Other Names: Alkeran ; Drug: prednisone; Induction - 60 mg/m^2 per day in 2-4 divided doses by mouth daily for 5 days; days 1, 2, 3, 4, 5.; Other Names: Deltasone ; Drug: sirolimus; Initiated on day +3. Patients >40kg, the initial dose will be 2 mg every 24 hours orally. Patients <40 kg, the initial dose will be 1 mg/m^2.; Other Names: Rapamune ; Drug: tacrolimus; Day -1 at least 24 hours before the stem cell infusion at a dose of 0.03 mg/kg/day as a continuous infusion. Twelve hours later oral dose initiated at a dose of 0.1-0.15 mg/kg/day in two divided doses every 12 hours.; Other Names: Prograf ; Drug: vincristine sulfate; Induction - 0.4 mg/m^2 per day continuous intravenous (IV) infusion over 24 hours daily for 4 days; 1, 2, 3, 4.; Other Names: Oncovin ; Procedure: peripheral blood stem cell transplantation; Stem cells from a healthy donor are collected and transplanted into the patient using a central venous catheter.; Other Names: PBSCT ; Drug: Filgrastim

Outcome Measures

Primary Outcome Measures

1. Number of Participants With Engraftment [100 days]

   Engraftment is defined as rapid conversion to complete donor chimerism and is assessed by blood counts and chimerism, >95% donor engraftment at day 100 in >75% of patients.

2. Toxicity [16.5 months]

   Here is the number of participants with adverse events. For a detailed list of adverse events see the adverse event module.

Secondary Outcome Measures

1. Number of Participants With Acute and Chronic GVHD [up to 5 years or death]

   Acute GVHD as by Modified Glucksberg Criteria occurring before day 100. Chronic GVHD as per Seattle criteria occurring after day 100.

2. Median Time to Reach Absolute Neutrophil Count of 500/mm(3) [up to 12 days]

   Days for participants to achieve a neutrophil count of 500/mm(3).

3. Median Time to Reach a Platelet Count of 50,000/mm(3) [up to 43 days]

   Days for participants to achieve a platelet count of 50,000/mm(3).

4. Early Post Transplantation Relapse [up to 300 days]

   Participants who experienced recurrence or progression of disease following transplant.

5. Median Progression Free Survival [up to 77 months]

   Progression free survival was based on the time from on-study date until progression or last follow-up.

6. Two Year Survival Rate for Patients Undergoing Allo-Hematopoietic Stem Cell Transplant [2 years]

   Participants who are alive at two years following Allo-Hematopoietic Stem Cell Transplant.

7. Number of Participants to Complete Conversion to >95% Donor Chimerism [up to 30 days]

   Participants who tolerated the transplantation regimen and accepted >95% of the donors blood, marrow, and/or tissue.

8. Cluster of Differentiation 4 (CD4) Reconstitution [Day +28-42]

   The median CD4 count with a range of 85-1565 (absolute count) was used to determine recovery and were considered recovered if in this range. The CD4 count was established by flow cytometry testing.

9. Best Response Post-Hematopoietic Stem Cell Transplant EOCH (Etoposide, Vincristine, Cyclophosphamide, and Doxorubicin) [up to 10 cycles of therapy or 280 days]

   Response is defined by the Response Evaluation Criteria in Solid Tumors (RECIST). RECIST criteria offer a simplified, conservative, extraction of imaging data for wide application in clinical trials. They presume that linear measures are an adequate substitute for 2-D (dimensional) methods and registers four response categories: Complete response (CR) is disappearance of all target lesions. Partial response (PR) is 30% increase in the sum of the longest diameter of target lesions. Progressive disease (PD) is 20% increase in the sum of the longest diameter of target lesions. Stable disease (SD) is small changes that do not meet above criteria. For the purposes of this study very good partial response ((VGPR) is >75% reduction in disease) was also employed.

10. Median Survival From Date of Progression [up to 77 months]

    Median survival from date of progression is based on the time from on-study date until progression or last follow-up.

Other Outcome Measures

1. Number of Participants Who Experienced Graft Versus Tumor Effect (GVT) [up to day 100]

   GVT is defined as tumor response after day 42 post-transplantation without cytotoxic therapy.

2. Post-Hematopoietic Stem Cell Transplant (HSCT) Radiotherapy [up to 6 cycles or 168 days]

   Site of radiotherapy (high energy radiation) and/or toxicity experienced by the participants post HSCT radiotherapy. Grading was preformed using the Modified Glucksberg Criteria.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 5 Years to 35 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

• INCLUSION CRITERIA: PATIENT

The following diagnoses will be considered:

1. Patients with Ewing's sarcoma family of tumors, or alveolar

rhabdomyosarcoma in one of the following categories:

• Patients who present at the time of initial diagnosis with bone or bone marrow metastases may be enrolled after completion of standard front-line therapy. Standard front line therapy for alveolar rhabdomyosarcoma should include vincristine and cyclophosphamide, plus actinomycin D and/or adriamycin. For patients with Ewing's sarcoma, standard front line therapy should include vincristine, cyclophosphamide, adriamycin, ifosfamide and etoposide.

• Patients with recurrence of tumor at any site less than one year after completing standard front-line therapy or with a second or subsequent recurrence at any time after completing standard front-line therapy.

• Patients with progression or persistence of disease while receiving standard front-line chemotherapy who cannot achieve a complete response (CR) with local treatment modalities.

1. The following patients with desmoplastic small round cell tumor are eligible after receiving front line standard therapy, which is defined as a regimen containing at least vincristine, cyclophosphamide, and adriamycin:

• unresectable disease

• metastatic tumor (abdominal and extra-abdominal disease)

• progressive or persistent while receiving standard therapy

• recurrence within one year of completing therapy

• Patients without evaluable tumor at the time of enrollment are eligible

• Patients who have previously received high-dose chemotherapy with autologous stem cell rescue are eligible for this trial.

• Patient age 5-35 at enrollment.

• Availability of a 5 or 6 antigen human leukocyte antigen (HLA)-matched first-degree relative donor (single HLA-A or B mismatch allowed). Genotypically identical twins may serve as stem cell donors. Genotypic identity must be confirmed by restrictive fragment length polymorphism (RFLP) analysis.

• Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 or, for children less than or equal 10 years of age, Lansky greater than or equal 60

• Cardiac function: Left ventricular ejection fraction greater than or equal to 45% by multi-gated acquisition scan (MUGA), fractional shortening greater than or equal 28% by echocardiogram (ECHO) or left ventricular ejection fraction greater than or equal 55% by ECHO.

• Pulmonary function: carbon monoxide diffusing capacity (DLCO) greater than or equal to 50% of the expected value corrected for alveolar volume.

• Renal function: Age-adjusted normal serum creatinine according to the following table or a creatinine clearance greater than or equal to 60 ml/min/1.73 m^2. Age (years) Maximum serum creatinine (mg/dl) less than or equal to 5 0.8 greater than 5, less than or equal to 10 1.0 greater than 10, less than or equal to15 1.2, greater than 15 1.5

• Liver function: Serum total bilirubin less than 2 mg/dl, serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) less than or equal to 2.5 times upper limit of normal.

• Marrow function: absolute neutrophil count (ANC) must be greater than 750/mm^{3 (unless due to underlying disease in which case there is no grade restriction), platelet count must be greater than or equal to 75,000/mm}3 (not achieved by transfusion) unless due to underlying disease in which case there is no grade restriction). Lymphopenia, cluster of differentiation 4 (CD4) lymphopenia, leukopenia, and anemia will not render patients ineligible.

• Ability to give informed consent. For patients less than18 years of age their legal guardian must give informed consent. Pediatric patients will be included in age appropriate discussion in order to obtain verbal assent.

• Durable power of attorney form completed (patients greater than or equal to18 years of age only).

INCLUSION CRITERIA: DONOR

• Weight greater than or equal 15 kilograms.

• First degree relative with genotypic identity at 5 or 6 HLA loci (single HLAA or B locus mismatch allowed). Genotypically identical twins may serve as stem cell donors. Genotypic identity must be confirmed by RFLP analysis.

• For donors less than 18 years of age, he/she must be the oldest suitable donor, their legal guardian must give informed consent, the donor must give verbal assent, and he/she must be cleared by social work and a mental health specialist to participate.

• For donors greater than or equal to 18 years of age, ability to give informed consent.

• Adequate peripheral venous access for apheresis or consent to use a temporary central venous catheter for apheresis.

• Donor selection criteria will be in accordance with National Institutes of Health (NIH)/Clinical Center (CC) Department of Transfusion Medicine Standards.

EXCLUSION CRITERIA: PATIENT

• Uncontrolled fungal infection.

• History of untreated CNS tumor involvement. Extradural masses which have not invaded the brain parenchyma (as is commonly observed in Ewing's sarcoma family of tumors) or parameningeal tumors (as is commonly observed in rhabdomyosarcoma) without evidence for leptomeningeal spread will not render the patient ineligible. Patients with previous central nervous system (CNS) tumor involvement that has been treated and has been stable for at least 6 weeks are eligible.

• Lactating or pregnant females.

• Human immunodeficiency virus (HIV) positive (due to unacceptable risk following allogeneic transplantation).

• Hepatitis B surface antigen (HBsAg) positive or hepatitis C antibody positive with elevated liver transaminases. All patients with chronic active hepatitis (including those on treatment) are ineligible.

• High risk of inability to comply with transplant protocol, or inability to give appropriate informed consent in the estimation of the principal investigator (PI), social work, or the stem cell transplant team.

• Fanconi Anemia

EXCLUSION CRITERIA: DONOR

• History of medical illness which poses a risk to donation in the estimation of the PI or the Department of Transfusion Medicine physician including, but not limited to stroke, hypertension that is not controlled with medication, or heart disease. Individuals with symptomatic angina or a history of coronary bypass grafting or angioplasty will not be eligible.

• History of congenital hematologic, immunologic, oncologic or metabolic disorder, which poses a prohibitive risk to the recipient in the estimation of the PI.

• Anemia (Hb less than 11 gm/dl) or thrombocytopenia (platelets less than 100,000/micro l).

• Lactating or pregnant females. Donors of childbearing potential must use an effective method of contraception during the time they are receiving growth colony stimulating factor (G-CSF). The effects of cytokine administration on a fetus are unknown and may be potentially harmful. The effects upon breast milk are also unknown and may potentially be harmful to the infant.

• Human immunodeficiency virus (HIV)-positive, hepatitis B surface antigen (HBsAg) positive or hepatitis C antibody positive. Donors are providing an allogeneic blood product and there is the potential risk of transmitting these viral illnesses to the recipient.

• High risk of inability to comply with transplant protocol.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	National Institutes of Health Clinical Center, 9000 Rockville Pike	Bethesda	Maryland	United States	20892

Sponsors and Collaborators

• National Cancer Institute (NCI)

Investigators

• Principal Investigator: Terry Fry, M.D., National Cancer Institute (NCI)

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• NIH Clinical Center Detailed Web Page

Publications

• Burdach S, Jürgens H, Peters C, Nürnberger W, Mauz-Körholz C, Körholz D, Paulussen M, Pape H, Dilloo D, Koscielniak E, et al. Myeloablative radiochemotherapy and hematopoietic stem-cell rescue in poor-prognosis Ewing's sarcoma. J Clin Oncol. 1993 Aug;11(8):1482-8.
• Deméocq F, Oberlin O, Benz-Lemoine E, Boilletot A, Gentet JC, Zucker JM, Behar C, Poutard P, Olive D, Brunat-Mentigny M, et al. Initial chemotherapy including ifosfamide in the management of Ewing's sarcoma: preliminary results. A protocol of the French Pediatric Oncology Society (SFOP). Cancer Chemother Pharmacol. 1989;24 Suppl 1:S45-7.
• Dunst J, Sauer R, Burgers JM, Hawliczek R, Kürten R, Winkelmann W, Salzer-Kuntschik M, Müschenich M, Jürgens H. Radiation therapy as local treatment in Ewing's sarcoma. Results of the Cooperative Ewing's Sarcoma Studies CESS 81 and CESS 86. Cancer. 1991 Jun 1;67(11):2818-25.

• Responsible Party: Terry Fry, M.D., Principal Investigator, National Institutes of Health Clinical Center (CC)
• ClinicalTrials.gov Identifier: NCT00043979
• Other Study ID Numbers: 020259; 02-C-0259; NCT00047372
• First Posted: Aug 16, 2002
• Last Update Posted: May 31, 2017
• Last Verified: Apr 1, 2017

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Terry Fry, M.D., Principal Investigator, National Institutes of Health Clinical Center (CC): Ewing's Sarcoma; Rhabdomyosarcoma; Desmoplastic Small Round Cell Tumor; Sarcoma; Ewing Sarcoma
• Additional relevant MeSH terms: Sarcoma; Cyclosporine; Sirolimus; Prednisone; Cyclophosphamide; Melphalan; Doxorubicin; Liposomal doxorubicin; Fludarabine; Fludarabine phosphate; Etoposide; Vincristine; Tacrolimus; Cyclosporins; Fluorodeoxyglucose F18

Study Results

Participant Flow

Arm/Group Title	Sibling Donors	Recipients Cyclosporine GVHD Prophylaxis	Recipients Tacrolimus /Sirolimus Prophylaxis
Arm/Group Description	Donors (n = 30) were matched first degree relatives who were eligible to donate peripheral blood stem cells.In period 1 they donated cells.	In period 1 recipients received EPOCH-F/chemotherapy. In period 2 recipients received peripheral blood stem transplant. Post transplant recipients received cyclosporine for GVHD prophylaxis.	In period 1 recipients received EPOCH-F/chemotherapy. In period 2 recipients received peripheral blood stem transplant. Post transplant recipients received tacrolimus & sirolimus for GVHD prophylaxis.
Period Title: Period 1
STARTED	30	17	13
COMPLETED	23	13	10
NOT COMPLETED	7	4	3
Period Title: Period 1
STARTED	0	13	10
COMPLETED	0	13	10
NOT COMPLETED	0	0	0

Baseline Characteristics

Arm/Group Title	Arm 1-Sibling Donors	Arm 2-Recipients	Total
Arm/Group Description	Donors (n=30) were matched first degree relatives who were eligible to donate peripheral blood stem cells.	Recipients (n=30) were enrolled to receive peripheral blood stem cells (PBSC) and receive either cyclosporine or tacrolimus and sirolimus for graft versus host disease (GVHD) prophylaxis.	Total of all reporting groups
Overall Participants	30	30	60
Age (Count of Participants)
<=18 years	17 56.7%	14 46.7%	31 51.7%
Between 18 and 65 years	13 43.3%	16 53.3%	29 48.3%
>=65 years	0 0%	0 0%	0 0%
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	21.12 (10.9)	19.98 (5.8)	20.55 (8.35)
Sex: Female, Male (Count of Participants)
Female	19 63.3%	10 33.3%	29 48.3%
Male	11 36.7%	20 66.7%	31 51.7%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	1 3.3%	1 3.3%	2 3.3%
Not Hispanic or Latino	29 96.7%	29 96.7%	58 96.7%
Unknown or Not Reported	0 0%	0 0%	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	0 0%	0 0%
Asian	0 0%	0 0%	0 0%
Native Hawaiian or Other Pacific Islander	0 0%	0 0%	0 0%
Black or African American	0 0%	0 0%	0 0%
White	30 100%	30 100%	60 100%
More than one race	0 0%	0 0%	0 0%
Unknown or Not Reported	0 0%	0 0%	0 0%
Region of Enrollment (Count of Participants)
United States	30 100%	30 100%	60 100%
Number of Prior Regimens of Participants that Received Stem Cell Transplant (regimens) [Number]
Patient # 1	5	5
Patient # 2	3	3
Patient # 3	2	2
Patient # 4	3	3
Patient # 6	2	2
Patient # 8	1	1
Patient # 9	2	2
Patient # 10	2	2
Patient # 12	2	2
Patient # 13	3	3
Patient # 14	1	1
Patient # 15	2	2
Patient # 17	2	2
Patient # 18	2	2
Patient # 20	3	3
Patient # 21	1	1
Patient # 22	1	1
Patient # 24	2	2
Patient # 25	2	2
Patient # 27	1	1
Patient # 28 (c)	1	1
Patient # 29	2	2
Patient # 30	1	1
Number of Prior Regimens of Participants that Did Not Receive Stem Cell Transplant (regimens) [Number]
Patient # 5	2	2
Patient # 7	1	1
Patient # 11	3	3
Patient # 16	2	2
Patient # 19	2	2
Patient # 23	4	4
Patient # 26	3	3
Participants who Received Stem Cell Transplant Disease Status at Enrollment (Count of Participants)
Progressive Disease (PD)	18 60%	18 60%
No Evidence of Disease (NED)	4 13.3%	4 13.3%
Stable Disease (SD)	1 3.3%	1 3.3%
Participants who Did Not Receive Stem Cell Transplant Disease Status(Progressive Disease)/Enrollment (Count of Participants)
Count of Participants [Participants]	7 23.3%	7 23.3%
Disease Status at Hematopoietic Stem Cell Transplant (Count of Participants)
Partial Response (PR)	4 13.3%	4 13.3%
Stable Disease (SD)	9 30%	9 30%
No Evidence of Disease (NED)	4 13.3%	4 13.3%
Progressive Disease (PD)	3 10%	3 10%
Complete Response (CR)/(NED)	3 10%	3 10%
Disease Status (Progressive Disease) at Hematopoietic Stem Cell Transplant (Count of Participants)
Count of Participants [Participants]	7 23.3%	7 23.3%
Diagnosis (Participants that Received Stem Cell Transplant) (Count of Participants)
aRMS	7 23.3%	7 23.3%
ESFT	5 16.7%	5 16.7%
DSRCT	11 36.7%	11 36.7%
Diagnosis (Participants that Did Not Receive Stem Cell Transplant) (Count of Participants)
ESFT	6 20%	6 20%
DSRCT	1 3.3%	1 3.3%
High-Risk Features (Participants that Received Stem Cell Transplant) (Count of Participants)
MR	6 20%	6 20%
ER	2 6.7%	2 6.7%
ER, B/BM, MR	1 3.3%	1 3.3%
PP	1 3.3%	1 3.3%
B/BM	7 23.3%	7 23.3%
Metastatic	4 13.3%	4 13.3%
PP/Metastatic	1 3.3%	1 3.3%
PP, B/BM	1 3.3%	1 3.3%
High-Risk Features (Participants that Did Not Receive Stem Cell Transplant) (Count of Participants)
ER, B/BM	1 3.3%	1 3.3%
PP, B/BM	1 3.3%	1 3.3%
ER	3 10%	3 10%
PP	2 6.7%	2 6.7%

Outcome Measures

1. Primary Outcome

Title	Number of Participants With Engraftment
Description	Engraftment is defined as rapid conversion to complete donor chimerism and is assessed by blood counts and chimerism, >95% donor engraftment at day 100 in >75% of patients.
Time Frame	100 days

Outcome Measure Data

Analysis Population Description

E.g ...in >75% of patients, shown above is not a conclusion but refers to a hypothesis, the objective of the protocol. 23 were analyzed because 7 donors were taken off study and stem cells not collected due to progressive disease of sibling.

Arm/Group Title	Arm 2-Recipients
Arm/Group Description	Recipients (n=30) were enrolled to receive peripheral blood stem cells (PBSC) and receive either cyclosporine or tacrolimus and sirolimus for graft versus host disease (GVHD) prophylaxis.
Measure Participants	23
Number [Participants]	23 76.7%

2. Primary Outcome

Title	Toxicity
Description	Here is the number of participants with adverse events. For a detailed list of adverse events see the adverse event module.
Time Frame	16.5 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Arm 2-Recipients
Arm/Group Description	Recipients (n=30) were enrolled to receive peripheral blood stem cells (PBSC) and receive either cyclosporine or tacrolimus and sirolimus for graft versus host disease (GVHD) prophylaxis.
Measure Participants	30
Number [Participants]	30 100%

3. Secondary Outcome

Title	Number of Participants With Acute and Chronic GVHD
Description	Acute GVHD as by Modified Glucksberg Criteria occurring before day 100. Chronic GVHD as per Seattle criteria occurring after day 100.
Time Frame	up to 5 years or death

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Recipients -Cyclosporine GVHD Prophylaxis	Recipients -Tacrolimus/Sirolimus GVHD Prophylaxis
Arm/Group Description
Measure Participants	13	10
acute GVHD	12 40%	5 16.7%
chronic GVHD	12 40%	5 16.7%

4. Secondary Outcome

Title	Median Time to Reach Absolute Neutrophil Count of 500/mm(3)
Description	Days for participants to achieve a neutrophil count of 500/mm(3).
Time Frame	up to 12 days

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Arm 2-Recipients
Arm/Group Description	Recipients (n=30) were enrolled to receive peripheral blood stem cells (PBSC) and receive either cyclosporine or tacrolimus and sirolimus for graft versus host disease (GVHD) prophylaxis.
Measure Participants	30
Median (Full Range) [Days]	9

5. Secondary Outcome

Title	Median Time to Reach a Platelet Count of 50,000/mm(3)
Description	Days for participants to achieve a platelet count of 50,000/mm(3).
Time Frame	up to 43 days

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Arm 2-Recipients
Arm/Group Description	Recipients (n=30) were enrolled to receive peripheral blood stem cells (PBSC) and receive either cyclosporine or tacrolimus and sirolimus for graft versus host disease (GVHD) prophylaxis.
Measure Participants	30
Median (Full Range) [Days]	15

6. Secondary Outcome

Title	Early Post Transplantation Relapse
Description	Participants who experienced recurrence or progression of disease following transplant.
Time Frame	up to 300 days

Outcome Measure Data

Analysis Population Description

One out of 23 participants did not have a recurrence, thus was excluded from analysis.

Arm/Group Title	Arm 2-Recipients
Arm/Group Description	Recipients (n=30) were enrolled to receive peripheral blood stem cells (PBSC) and receive either cyclosporine or tacrolimus and sirolimus for graft versus host disease (GVHD) prophylaxis.
Measure Participants	22
Median (Full Range) [Days]	100

7. Secondary Outcome

Title	Median Progression Free Survival
Description	Progression free survival was based on the time from on-study date until progression or last follow-up.
Time Frame	up to 77 months

Outcome Measure Data

Analysis Population Description

23 were analyzed because 7 donors were taken off study and stem cells not collected due to progressive disease of sibling.

Arm/Group Title	Arm 2-Recipients
Arm/Group Description	Recipients (n=30) were enrolled to receive peripheral blood stem cells (PBSC) and receive either cyclosporine or tacrolimus and sirolimus for graft versus host disease (GVHD) prophylaxis.
Measure Participants	23
Median (Full Range) [Months]	15.9

8. Secondary Outcome

Title	Two Year Survival Rate for Patients Undergoing Allo-Hematopoietic Stem Cell Transplant
Description	Participants who are alive at two years following Allo-Hematopoietic Stem Cell Transplant.
Time Frame	2 years

Outcome Measure Data

Analysis Population Description

23 were analyzed because 7 donors were taken off study and stem cells not collected due to progressive disease of sibling.

Arm/Group Title	Arm 2-Recipients
Arm/Group Description	Recipients (n=30) were enrolled to receive peripheral blood stem cells (PBSC) and receive either cyclosporine or tacrolimus and sirolimus for graft versus host disease (GVHD) prophylaxis.
Measure Participants	23
From date of enrollment	39.1 130.3%
From date of transplantation	34.8 116%

9. Secondary Outcome

Title	Number of Participants to Complete Conversion to >95% Donor Chimerism
Description	Participants who tolerated the transplantation regimen and accepted >95% of the donors blood, marrow, and/or tissue.
Time Frame	up to 30 days

Outcome Measure Data

Analysis Population Description

23 were analyzed because 7 donors were taken off study and stem cells not collected due to progressive disease of sibling.

Arm/Group Title	Arm 2-Recipients
Arm/Group Description	Recipients (n=30) were enrolled to receive peripheral blood stem cells (PBSC) and receive either cyclosporine or tacrolimus and sirolimus for graft versus host disease (GVHD) prophylaxis.
Measure Participants	23
Day +14	23 76.7%
Day +28	23 76.7%

10. Secondary Outcome

Title	Cluster of Differentiation 4 (CD4) Reconstitution
Description	The median CD4 count with a range of 85-1565 (absolute count) was used to determine recovery and were considered recovered if in this range. The CD4 count was established by flow cytometry testing.
Time Frame	Day +28-42

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Arm 2-Recipients
Arm/Group Description	Recipients (n=30) were enrolled to receive peripheral blood stem cells (PBSC) and receive either cyclosporine or tacrolimus and sirolimus for graft versus host disease (GVHD) prophylaxis.
Measure Participants	30
Median (Full Range) [mm(3)]	284

11. Secondary Outcome

Title	Best Response Post-Hematopoietic Stem Cell Transplant EOCH (Etoposide, Vincristine, Cyclophosphamide, and Doxorubicin)
Description	Response is defined by the Response Evaluation Criteria in Solid Tumors (RECIST). RECIST criteria offer a simplified, conservative, extraction of imaging data for wide application in clinical trials. They presume that linear measures are an adequate substitute for 2-D (dimensional) methods and registers four response categories: Complete response (CR) is disappearance of all target lesions. Partial response (PR) is 30% increase in the sum of the longest diameter of target lesions. Progressive disease (PD) is 20% increase in the sum of the longest diameter of target lesions. Stable disease (SD) is small changes that do not meet above criteria. For the purposes of this study very good partial response ((VGPR) is >75% reduction in disease) was also employed.
Time Frame	up to 10 cycles of therapy or 280 days

Outcome Measure Data

Analysis Population Description

EPOCH-F (Etoposide, Vincristine, Prednisone, Cyclophosphamide, Doxorubicin, and Fludarabine) was modified to EOCH and administered to 12 patients for post-transplantation disease progression.

Arm/Group Title	Arm 2-Recipients
Arm/Group Description	Recipients (n=30) were enrolled to receive peripheral blood stem cells (PBSC) and receive either cyclosporine or tacrolimus and sirolimus for graft versus host disease (GVHD) prophylaxis.
Measure Participants	12
Complete Response (CR)	2 6.7%
Progressive Disease (PD)	4 13.3%
Partial Response (PR)	4 13.3%
Very Good Partial Response (VGPR)	2 6.7%

12. Secondary Outcome

Title	Median Survival From Date of Progression
Description	Median survival from date of progression is based on the time from on-study date until progression or last follow-up.
Time Frame	up to 77 months

Outcome Measure Data

Analysis Population Description

23 were analyzed because 7 donors were taken off study and stem cells not collected due to progressive disease of sibling.

Arm/Group Title	Arm 2-Recipients
Arm/Group Description	Recipients (n=30) were enrolled to receive peripheral blood stem cells (PBSC) and receive either cyclosporine or tacrolimus and sirolimus for graft versus host disease (GVHD) prophylaxis.
Measure Participants	23
Participants who did not receive a transplant(n=7)	3.3
Participants who received a transplant (n=23)	19.1

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Arm 2-Recipients
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	.0003
	Comments	7 participants who did not receive a transplant compared with 21 participants transplanted.
	Method	Kaplan-Meier
	Comments

13. Other Pre-specified Outcome

Title	Number of Participants Who Experienced Graft Versus Tumor Effect (GVT)
Description	GVT is defined as tumor response after day 42 post-transplantation without cytotoxic therapy.
Time Frame	up to day 100

Outcome Measure Data

Analysis Population Description

23 were analyzed because 7 donors were taken off study and stem cells not collected due to progressive disease of sibling.

Arm/Group Title	Arm 2-Recipients
Arm/Group Description	Recipients (n=30) were enrolled to receive peripheral blood stem cells (PBSC) and receive either cyclosporine or tacrolimus and sirolimus for graft versus host disease (GVHD) prophylaxis.
Measure Participants	23
Count of Participants [Participants]	0 0%

14. Other Pre-specified Outcome

Title	Post-Hematopoietic Stem Cell Transplant (HSCT) Radiotherapy
Description	Site of radiotherapy (high energy radiation) and/or toxicity experienced by the participants post HSCT radiotherapy. Grading was preformed using the Modified Glucksberg Criteria.
Time Frame	up to 6 cycles or 168 days

Outcome Measure Data

Analysis Population Description

G1, grade 1; G2, grade 2; G3, grade 3; G4, grade 4; G5, grade 5. 23 were analyzed because 7 donors were taken off study and stem cells not collected due to progressive disease of sibling.

Arm/Group Title	Arm 2-Recipients
Arm/Group Description	Recipients (n=30) were enrolled to receive peripheral blood stem cells (PBSC) and receive either cyclosporine or tacrolimus and sirolimus for graft versus host disease (GVHD) prophylaxis.
Measure Participants	23
Chest wall; G2 skin	1 3.3%
Abdomen; G4 GI	1 3.3%
Pancreas; G4 LFTs, G4 pancreatitis	1 3.3%
Pleura, mediastinum; G4 LFTs, G2 mucositis	1 3.3%
Chest wall; G4 skin, G3 mucositis	1 3.3%
Spine, skull; G2 nausea+vomiting, G2 fatigue	1 3.3%
Pelvis; G4 enteritis	1 3.3%
Pulmonary (cyberknife)	1 3.3%
Brain; B3 mucositis	1 3.3%
Whole lung; G3 mucositis, G3 skin, G5 lung	1 3.3%
L arm, R shoulder, B/L femur	1 3.3%

Adverse Events

Time Frame	16.5 months
Arm/Group Title	Arm 2-Recipients
Arm/Group Description	Recipients (n=30) were enrolled to receive peripheral blood stem cells (PBSC) and receive either cyclosporine or tacrolimus and sirolimus for graft versus host disease (GVHD) prophylaxis.
All Cause Mortality
	Arm 2-Recipients
	Affected / at Risk (%)	# Events
Total	29/30 (96.7%)
Serious Adverse Events
	Arm 2-Recipients
	Affected / at Risk (%)	# Events
Total	29/30 (96.7%)
Cardiac disorders
Cardiac General: Hypotension	1/30 (3.3%)	1
Pericardial effusion	1/30 (3.3%)	1
Thrombosis	2/30 (6.7%)	2
Supraventricular arrhythmias (SVT/atrial fibrillation/flutter)	1/30 (3.3%)	3
Gastrointestinal disorders
Gastrointestinal: Diarrhea	2/30 (6.7%)	2
Gastrointestinal:Mucositis/stomatitis (clinical exam)::oral cavity	1/30 (3.3%)	1
GI, other	1/30 (3.3%)	1
Intussception	1/30 (3.3%)	1
Stomatitis	1/30 (3.3%)	1
General disorders
Constitutional Symptoms: Fever	1/30 (3.3%)	1
Death: Death not associated with CTCAE term: Disease progression NOS	29/30 (96.7%)	29
Pain - right chest wall	1/30 (3.3%)	1
Infections and infestations
Infection with neutropenia	1/30 (3.3%)	1
Infection without neutropenia	1/30 (3.3%)	1
Infection: Febrile neutropenia	1/30 (3.3%)	1
Metabolism and nutrition disorders
Metabolic/Laboratory: Bilirubin, serum-high (hyperbilirubinemia)	1/30 (3.3%)	1
Metabolic/Laboratory: ALT, SGPT (serum glutamic pyruvic transaminase)	1/30 (3.3%)	2
Metabolic/Laboratory: AST, SGOT (serum glutamic oxaloacetic transaminase)	1/30 (3.3%)	1
AST, high	1/30 (3.3%)	1
Nervous system disorders
Mood alteration; suicide gesture	1/30 (3.3%)	1
Neurology: Mood alteration: anxiety	1/30 (3.3%)	1
Neurology: Seizure	2/30 (6.7%)	2
Renal and urinary disorders
Creatinine	1/30 (3.3%)	1
Vaginitis	1/30 (3.3%)	1
Respiratory, thoracic and mediastinal disorders
Pulmonary/Upper Respiratory: Pneumonitis/pulmonary infiltrates	1/30 (3.3%)	1
Pulmonary - Other (pulmonary edema w/normal 02 sat)	1/30 (3.3%)	1
Pulmonary/Upper Respiratory: Dyspnea (shortness of breath)	2/30 (6.7%)	2
Pulmonary/Upper Respiratory: Hypoxia	2/30 (6.7%)	2
Respiratory failure	1/30 (3.3%)	1
Pneumonitits/Pulmonary infiltrates	1/30 (3.3%)	1
Skin and subcutaneous tissue disorders
Radiation Recall Reaction (anterior chest)	1/30 (3.3%)	1
Other (Not Including Serious) Adverse Events
	Arm 2-Recipients
	Affected / at Risk (%)	# Events
Total	30/30 (100%)
Blood and lymphatic system disorders
Adenopathy	1/30 (3.3%)	1
Bacteremia	2/30 (6.7%)	2
Blood/Bone Marrow:Platelets	26/30 (86.7%)	375
Blood/Bone Marrow:Hemoglobin	30/30 (100%)	393
Blood/Bone Marrow:Leukocytes (total WBC)	27/30 (90%)	241
Blood/Bone Marrow:Neutrophils/granulocytes (ANC/AGC)	26/30 (86.7%)	235
Edema, extremities	1/30 (3.3%)	1
Epistaxis	2/30 (6.7%)	2
Hematoma	1/30 (3.3%)	1
Lymphatics	1/30 (3.3%)	1
Lymphatics: edema: limb	5/30 (16.7%)	8
Lymphocytes	4/30 (13.3%)	4
Neutropenia	1/30 (3.3%)	1
Other, edema	1/30 (3.3%)	1
Other, fluid overload	1/30 (3.3%)	1
Coagulation: PTT (partial thromboplastin time)	28/30 (93.3%)	159
PT, prolonged	8/30 (26.7%)	8
RBC transfusion	1/30 (3.3%)	1
Transfusion, platelet	2/30 (6.7%)	2
Transfusion, RBC	2/30 (6.7%)	2
Ankle edema B/L	1/30 (3.3%)	1
Ankle swelling, bilaterally	1/30 (3.3%)	1
Blood/Bone Marrow:CD4 Count	1/30 (3.3%)	4
Blood/Bone Marrow:Haptoglobin	1/30 (3.3%)	5
Coagulation: Other (Specify, bruising to lower extremities)	1/30 (3.3%)	1
Coagulation: INR (International Normalized Ration of prothrombin time)	7/30 (23.3%)	29
Coagulation: Other (Specify, positive lupus anticoagulant)	1/30 (3.3%)	1
Coagulation: thrombotic microangiopathy	1/30 (3.3%)	3
Hemolytic uremic syndrome	1/30 (3.3%)	1
Hemorrhage/Bleeding: Hematoma	1/30 (3.3%)	5
Hemorrhage::Rectal bleeding/hematochezia	2/30 (6.7%)	5
Lymphatics::Edema:head and neck	1/30 (3.3%)	5
Lymphatics::Lymphatics - Other (Specify, palpable inguinal adenopathy)	1/30 (3.3%)	2
Lymphedema, left LE	1/30 (3.3%)	1
Thrombocytopenia	2/30 (6.7%)	2
Cardiac disorders
Cardiac Arrhythmia: Supraventricular and nodal arrhythmia - Sinus tachycardia	7/30 (23.3%)	13
Cardiac General: Hypertension	7/30 (23.3%)	16
Cardiac General:Hypotension	9/30 (30%)	20
Compression of vena cava by tumor	1/30 (3.3%)	1
Pericardial fluid	1/30 (3.3%)	1
Tachycardia	3/30 (10%)	3
Tachypnea	1/30 (3.3%)	1
Cardiac Arrhythmia - Prolonged QTc interval	2/30 (6.7%)	7
Cardiac Arrhythmia: Supraventricular and nodal arrhythmia - Sinus bradycardia	1/30 (3.3%)	1
Cardiac Arrhythmia: Supraventricular arrhythmias (SVT/atrial fibrillation/flutter)	2/30 (6.7%)	4
Cardiac General: Other, capillary leak syndrome	1/30 (3.3%)	5
Cardiac General: Pericardial effusion (non-malignant)	1/30 (3.3%)	1
Cardiovascular (Arrhythmia): Sinus bradycardia	1/30 (3.3%)	1
Cardiovascular General: Cardiac left ventricular function	1/30 (3.3%)	2
Cardiovascular/General: Other (Specify, venous valve)	1/30 (3.3%)	3
Ear and labyrinth disorders
Bullous myringitis	1/30 (3.3%)	1
Ears-dry	1/30 (3.3%)	1
Pain, ear	1/30 (3.3%)	1
pain/auditory/ear: external	1/30 (3.3%)	1
Pain: Pain: Auditory/Ear	1/30 (3.3%)	1
Auditory/Ear: Hearing	1/30 (3.3%)	3
Auditory/Hearing: External auditory canal	1/30 (3.3%)	1
Pain:Earache	1/30 (3.3%)	1
Endocrine disorders
Endocrine: Cushingoid appearance	9/30 (30%)	29
Endocrine: Hot flashes/Flushes	1/30 (3.3%)	1
Endocrine: Other (Specify, secondary ovarian failure)	1/30 (3.3%)	1
Eye disorders
Blurred vision	3/30 (10%)	3
Diplopia	1/30 (3.3%)	1
Eye discharge	1/30 (3.3%)	1
Eyes - dry bilateral	1/30 (3.3%)	1
Ocular/Visual: dry eye syndrome	2/30 (6.7%)	9
Ocular/Visual: cataract	1/30 (3.3%)	2
Ocular/Visual: Ocular/Visual - Other (specify, erythema)	1/30 (3.3%)	5
Ocular/Visual: Vision-blurred vision	2/30 (6.7%)	2
Pain/Ocular: eye	1/30 (3.3%)	2
Uveitis, OU	1/30 (3.3%)	1
Xeropthalmia	1/30 (3.3%)	1
Pain - left eye	1/30 (3.3%)	1
Pain - right eye	1/30 (3.3%)	1
Tearing	2/30 (6.7%)	2
Visual changes	1/30 (3.3%)	1
Ocular/Visual:Dry eyes	1/30 (3.3%)	2
Gastrointestinal disorders
Abdominal cramping	1/30 (3.3%)	2
Abdominal discomfort	1/30 (3.3%)	1
Abdominal pain	1/30 (3.3%)	1
Abscesses (crypt)	1/30 (3.3%)	1
Appetite decreased	1/30 (3.3%)	1
Bilious emesis	1/30 (3.3%)	1
Bilious vomiting	1/30 (3.3%)	1
Dehydration	4/30 (13.3%)	4
Dry mouth	1/30 (3.3%)	1
Dysphagia	4/30 (13.3%)	4
Emesis	1/30 (3.3%)	1
Esophagitis, chronic	1/30 (3.3%)	1
Gastritis	1/30 (3.3%)	1
Gastroesophageal reflux	1/30 (3.3%)	1
Gastrointestinal: Anorexia	10/30 (33.3%)	16
Gastrointestinal:Constipation	13/30 (43.3%)	24
Gastrointestinal:Dental: periodontal disease	1/30 (3.3%)	5
Gastrointestinal:Diarrhea	22/30 (73.3%)	74
Gastrointestinal:Distention, bloating, abdominal	2/30 (6.7%)	2
Gastrointestinal:Flatulence	2/30 (6.7%)	3
Gastrointestinal:Heartburn/Dyspepsia	5/30 (16.7%)	10
Gastrointestinal:Hemorrhoids	2/30 (6.7%)	7
Gastrointestinal:Mucositis/stomatitis (clinical exam)::oral cavity	9/30 (30%)	14
Gastrointestinal:Mucositis/stomatitis (functional/symptomatic)::pharynx	2/30 (6.7%)	3
Gastrointestinal:Nausea	21/30 (70%)	44
GVHD	4/30 (13.3%)	12
GVHD, GI	2/30 (6.7%)	2
GVHD - cutaneous	1/30 (3.3%)	1
GVHD, GI & skin & liver	2/30 (6.7%)	2
GVHD, liver	1/30 (3.3%)	1
GVHD: skin, liver, oral and vaginal mucosa	1/30 (3.3%)	1
GVHD - skin, liver, oral buccal mucosa	1/30 (3.3%)	1
Hematochezia	1/30 (3.3%)	1
Itching (anal warts)	1/30 (3.3%)	1
Mucositis	1/30 (3.3%)	1
Mild constipation	1/30 (3.3%)	1
Oral candidiasis	1/30 (3.3%)	3
Oral pain, esophageal pain	1/30 (3.3%)	1
Pain mouth sore	1/30 (3.3%)	1
Pain perianal	1/30 (3.3%)	1
Pain, abdominal	8/30 (26.7%)	9
Pain, oral mucosa	1/30 (3.3%)	1
Colitis	1/30 (3.3%)	1
Pain/Gastrointestinal:dental/teeth/periodontal	1/30 (3.3%)	2
Pain:Gastrointestinal: oral cavity	3/30 (10%)	8
Poor appetite	3/30 (10%)	3
Proctitis	1/30 (3.3%)	1
Rectal pain	1/30 (3.3%)	1
Ulcer, duodenal	1/30 (3.3%)	1
Ulcers near molars	1/30 (3.3%)	1
Vomiting	9/30 (30%)	9
Perianal pain	1/30 (3.3%)	1
Sensitive teeth	1/30 (3.3%)	1
Small bowel obstruction	1/30 (3.3%)	1
Stomatitis	6/30 (20%)	6
Stomatitis, candida	1/30 (3.3%)	1
Ulceration on right buccal mucosa	1/30 (3.3%)	1
C. difficile colitis	1/30 (3.3%)	1
Dental caries	1/30 (3.3%)	1
Dyspepsia	1/30 (3.3%)	1
Gastrointestinal: Other (Specify, early satiety)	1/30 (3.3%)	2
Gastrointestinal: Distention/bloating	1/30 (3.3%)	2
Gastrointestinal: Mucositis/stomatitis - Oral cavity	1/30 (3.3%)	1
Gastrointestinal: Ascites (non-malignant)	1/30 (3.3%)	2
Gastrointestinal: Dehydration	3/30 (10%)	3
Gastrointestinal: Colitis	3/30 (10%)	4
Gastrointestinal:	2/30 (6.7%)	4
Gastrointestinal: Dysphagia (difficulty swallowing)	3/30 (10%)	5
Gastrointestinal: Esophagitis	1/30 (3.3%)	1
Gastrointestinal: Gastritis (including bile reflux gastritis)	2/30 (6.7%)	4
Gastrointestinal: Mucositis/stomatitis (functional/symptomatic)::Oral cavity	2/30 (6.7%)	9
Gastrointestinal: Other	1/30 (3.3%)	1
Gastrointestinal:other, specify::Cholestasis	1/30 (3.3%)	3
Gastrointestinal: Proctitis	2/30 (6.7%)	3
Gastrointestinal: Vomiting	5/30 (16.7%)	8
Gastrointestinal: Abdominal pain	1/30 (3.3%)	1
Gastrointestinal: Anorexia	1/30 (3.3%)	1
Gastrointestinal: Constipation	1/30 (3.3%)	1
Gastrointestinal: Dehydration	1/30 (3.3%)	1
Gastrointestinal: Dental:Teeth	1/30 (3.3%)	4
Gastrointestinal: Nausea	2/30 (6.7%)	2
Hematochezia/rectal bleeding	1/30 (3.3%)	1
Increased perirectal pain	1/30 (3.3%)	1
Mucositis of throat and rectal area	1/30 (3.3%)	1
Pain with defecation	1/30 (3.3%)	1
Pain/Gastrointestinal::Abdomen NOS	9/30 (30%)	39
Pain: Gastrointestinal - Anus	2/30 (6.7%)	3
Pain::Pain:: Abdomen NOS	1/30 (3.3%)	3
Pain::Pain::Oral cavity	2/30 (6.7%)	5
Pain::Pain::Throat/pharynx/larynx	1/30 (3.3%)	3
Pain::Rectal or perirectal pain (Proctalgia)	1/30 (3.3%)	1
Perirectal pain	1/30 (3.3%)	1
Proctitis/perirectal skin breakdown	1/30 (3.3%)	1
Ulcerations - soft palate	1/30 (3.3%)	1
General disorders
Chills	3/30 (10%)	3
Constitutional Symptoms - Other (pallor)	1/30 (3.3%)	1
Constitutional Symptoms: Fatigue (asthenia, lethargy, malaise)	20/30 (66.7%)	43
Constitutional Symptoms: Fever	20/30 (66.7%)	55
Constitutional Symptoms: Insomnia	6/30 (20%)	11
Constitutional Symptoms: Weight loss	6/30 (20%)	11
Diaphoresis	1/30 (3.3%)	1
Dyspareunia	1/30 (3.3%)	1
Fissure	1/30 (3.3%)	1
Flat effect	1/30 (3.3%)	1
Headache	5/30 (16.7%)	6
Headaches-intermittent	1/30 (3.3%)	1
Intermittent headache	1/30 (3.3%)	1
Low energy level	1/30 (3.3%)	1
Low grade fever	2/30 (6.7%)	2
Pain	3/30 (10%)	3
Pain, head	1/30 (3.3%)	1
Pain thoracic	1/30 (3.3%)	1
Sweating	1/30 (3.3%)	1
Swelling	1/30 (3.3%)	1
Pain: NOS	1/30 (3.3%)	2
Rigors	1/30 (3.3%)	1
Severe fatigue	1/30 (3.3%)	1
Sleepiness	1/30 (3.3%)	1
BMT Complex/Multicomponent Events: Graft versus host disease	1/30 (3.3%)	2
Constitutional Symptoms: Rigors/Chills	4/30 (13.3%)	5
Constitutional Symptoms: Sweating (diaphoresis)	3/30 (10%)	6
Constitutional Symptoms: Weight gain	2/30 (6.7%)	8
Death: Death not associated with CTCAE term: Disease progression NOS	4/30 (13.3%)	4
Mild fatigue	1/30 (3.3%)	2
Pain - Other (Specify, pain at site of biopsy)	1/30 (3.3%)	1
Pain - right lateral	1/30 (3.3%)	1
Pain - Other	1/30 (3.3%)	1
Pain/General::Tumor pain	1/30 (3.3%)	4
Pain:General	1/30 (3.3%)	1
Pain::General - Tumor pain	1/30 (3.3%)	1
Low grade fevers	1/30 (3.3%)	1
Death	1/30 (3.3%)	1
Hepatobiliary disorders
Albumin low	1/30 (3.3%)	1
Alk. phosph.	1/30 (3.3%)	1
Hepatic: Bilirubin associated with graft versus host disease (GVHD) for BMT studies,	4/30 (13.3%)	8
Transaminitis	1/30 (3.3%)	1
Hepatic: Albumin, serum-low (hypoalbuminemia)	2/30 (6.7%)	4
Hepatic:Bilirubin (hyperbilirubinemia)	1/30 (3.3%)	4
Hepatobiliary/Pancreas:: Cholecystitis	1/30 (3.3%)	1
Hepatobiliary/Pancreas:: Pancreatitis	1/30 (3.3%)	3
Immune system disorders
Allergic rhinitis-rhinorrhea	1/30 (3.3%)	1
Allergy/Immunology: Allergic reaction/hypersensitivity (including drug fever)	2/30 (6.7%)	2
Allergy/Immunology: Allergic rhinitis (including sneezing, nasal stuffiness, postnasal drip)	18/30 (60%)	60
Allergy/Immunology: Autoimmune reaction	1/30 (3.3%)	5
Congestion	3/30 (10%)	3
Mild nasal congestion	1/30 (3.3%)	1
Allergy/Immunology: Allergic rhinitis	2/30 (6.7%)	8
Rhinorrhea	1/30 (3.3%)	1
Infections and infestations
Infection	1/30 (3.3%)	1
Infection-Other	3/30 (10%)	5
Infection-Other (specify) tinea pedis, left foot	1/30 (3.3%)	1
Infection - sexual/reproductive function - vulva	1/30 (3.3%)	1
Infection w/neutropenia	1/30 (3.3%)	1
Infection without neutropenia	4/30 (13.3%)	4
Infection, C. diff & rota virus	1/30 (3.3%)	1
Infection, CVL	1/30 (3.3%)	1
Infection, influenza (w/o neutropenia)	1/30 (3.3%)	1
Infection, musculo-skeletal, soft tissue	1/30 (3.3%)	1
Infection: Febrile neutropenia	5/30 (16.7%)	5
Infection: Infection	5/30 (16.7%)	5
Infection: Infection (documented clinically or microbiologically)	4/30 (13.3%)	5
Infection: Infection with normal ANC or Grade 1 or 2 neutrophils:	1/30 (3.3%)	1
Infection: Infection with normal ANC or Grade 1 or 2 neutrophils: select - conjunctiva	4/30 (13.3%)	8
Infection: Infection with normal ANC or Grade 1 or 2 neutrophils: skin	1/30 (3.3%)	1
Infection: Infection with normal ANC or Grade 1 or 2 neutrophils: skin (cellulitis)	1/30 (3.3%)	1
Infection: Infection with normal ANC or Grade 1 or 2 neutrophils: upper airway NOS	1/30 (3.3%)	1
Infection-upper respiratory symptoms	1/30 (3.3%)	1
Paronychia, hallux, B/L	1/30 (3.3%)	1
Positive viral culture	1/30 (3.3%)	1
Sepsis	1/30 (3.3%)	1
Infection: Infection with normal ANC or Grade 1 or 2 neutrophils: Dermatology-Skin	1/30 (3.3%)	1
Infection: Other	2/30 (6.7%)	4
Infection - Other (Specify, abrasion right great toe)	1/30 (3.3%)	12
Infection - Select - Renal/Genitourinary-kidney	1/30 (3.3%)	1
Infection General::Wound	1/30 (3.3%)	1
Infection/Febrile neutropenia::Infection	1/30 (3.3%)	1
Infection::Infection (documented clincally or micro) with grade 3 or 4 neutrophils	3/30 (10%)	3
Infection::Colitis, infectious (e.g., Clostridium difficile)	2/30 (6.7%)	3
Infection::Infection	1/30 (3.3%)	1
Infection::Infection with normal ANC or Grade 1 or 2 neutrophils::Gluteal abscess left side	1/30 (3.3%)	1
Infection::Infection with normal ANC or Grade 1 or 2 neutrophils::Sinusitis	1/30 (3.3%)	1
Infection::Infection with unknown ANC::Lung (pneumonia)	1/30 (3.3%)	1
Infection::Infection with unknown ANC::Urinary tract NOS	1/30 (3.3%)	3
Infection::Opportunistic infection associated with > grade 2 lymphopenia	1/30 (3.3%)	6
Infection::Infection - Other (Specify, clostridium difficile)	1/30 (3.3%)	1
Infection: General, catheter related	1/30 (3.3%)	1
Injury, poisoning and procedural complications
Burn	1/30 (3.3%)	1
Burns, radiation	1/30 (3.3%)	1
Pain::Pain - Other (Specify, surgical incision pain)	1/30 (3.3%)	3
Metabolism and nutrition disorders
Metabolic/Laboratory: Bilirubin, serum-high (hyperbilirubinemia)	21/30 (70%)	134
Metabolic/Laboratory: Other (Urea nitrogen, low???)	8/30 (26.7%)	27
Metabolic/Laboratory: Cholesterol, serum high (hypercholestremia)	6/30 (20%)	23
Metabolic/Laboratory: AST, SGOT (serum glutamic oxaloacetic)	24/30 (80%)	253
Metabolic/Laboratory: Calcium, serum-low (hypocalcemia)	24/30 (80%)	163
Metabolic/Laboratory: Glucose, serum-high (hyperglycemia)	27/30 (90%)	408
Metabolic/Laboratory: Glucose, serum-low (hypoglycemia)	8/30 (26.7%)	14
Metabolic/Laboratory: Magnesium, serum-high (hypermagnesemia)	15/30 (50%)	43
Metabolic/Laboratory: Magnesium, serum-low (hypomagnesemia)	22/30 (73.3%)	153
Metabolic/Laboratory: Potassium, serum-high (hyperkalemia)	9/30 (30%)	14
Metabolic/Laboratory: Sodium, serum-low (hyponatremia)	25/30 (83.3%)	165
Metabolic/Laboratory: Triglyceride, serum-high (hypertriglyceridemia)	8/30 (26.7%)	36
Mg, low	3/30 (10%)	3
Metabolic/Laboratory: Potassium, serum-low (hypokalemia)	10/30 (33.3%)	26
Phosphatemia	2/30 (6.7%)	2
Protein low	1/30 (3.3%)	1
Cholesterol	1/30 (3.3%)	1
Metabolic/Laboratory: Albumin, serum-low (hypoalbuminemia)	13/30 (43.3%)	195
Metabolic/Laboratory: Other, (Specify, BUN, high)	3/30 (10%)	6
Metabolic/Laboratory: Alkaline phosphatase	1/30 (3.3%)	3
Metabolic/Laboratory: ALT, SGPT (serum glutamic pyruvic transaminase)	11/30 (36.7%)	209
Metabolic/Laboratory: Calcium, serum-high (hypercalcemia)	4/30 (13.3%)	7
Metabolic/Laboratory: Creatinine	5/30 (16.7%)	17
Metabolic/Laboratory: Hemogloginuria	1/30 (3.3%)	1
Metabolic/Laboratory: Metabolic/Laboratory: Other, (Specify, LDH)	20/30 (66.7%)	163
Metabolic/Laboratory: Phosphate, serum-low (hypophosphatemia)	6/30 (20%)	32
Metabolic/Laboratory:Potassium, serum-low (hypokalemia)	11/30 (36.7%)	102
Metabolic/Laboratory: Proteinuria	7/30 (23.3%)	42
Metabolic/Laboratory: Sodium, serum-high (hypernatremia)	6/30 (20%)	12
Metabolic/Laboratory: Other, urine protein	3/30 (10%)	12
Musculoskeletal and connective tissue disorders
Arthralgia	4/30 (13.3%)	5
Avascular necrosis	1/30 (3.3%)	1
Joint soreness, intermittent	1/30 (3.3%)	1
Joint stiffness	1/30 (3.3%)	1
Joint stiffness & pain	1/30 (3.3%)	1
Low back pain	1/30 (3.3%)	1
Mild tenderness in lumbosacral area	1/30 (3.3%)	1
Muscle aches-back, hip, left neck, right lateral chest wall, right scapular	1/30 (3.3%)	1
Musculoskeletal/other-joint stiffness	1/30 (3.3%)	1
Musculoskeletal/Soft tissue: joint-effusion	1/30 (3.3%)	2
Musculoskeletal/Soft tissue: Muscle weakness, genralized or specific area	1/30 (3.3%)	1
Musculoskeletal/Soft tissue: Fracture	2/30 (6.7%)	7
Musculoskeletal/Soft tissue: Muscle weakness, generalized or specific area (not due to neuropathy):	2/30 (6.7%)	7
Musculoskeletal/Soft tissue: Musculoskeletal/Soft tissue - Other	1/30 (3.3%)	5
Musculoskeletal/Soft tissue: Osteonecrosis (avascular necrosis)	2/30 (6.7%)	6
Myalgia-Muscle cramps	1/30 (3.3%)	1
Myalgias	1/30 (3.3%)	1
Pain, chest	1/30 (3.3%)	1
Pain, extremities (throbbing)	1/30 (3.3%)	1
Pain, knee	1/30 (3.3%)	1
Pain, left trapezius and shoulder area	1/30 (3.3%)	1
Pain, myalgia	3/30 (10%)	3
Pain, musculoskeletal	1/30 (3.3%)	1
Pain, tenderness (chest)	1/30 (3.3%)	1
Pain/Musculoskeletal:Back	8/30 (26.7%)	20
Pain/Musculoskeletal:Extremity-limb	9/30 (30%)	26
Pain/Musculoskeletal:Bone	3/30 (10%)	5
Pain/Musculoskeletal:Buttock	1/30 (3.3%)	2
Pain/Musculoskeletal:Neck	3/30 (10%)	4
Pain:Chest wall	1/30 (3.3%)	1
Pain: Musculoskeletal - Joint	7/30 (23.3%)	11
Pain-ankles & knees	1/30 (3.3%)	1
Pain - back	4/30 (13.3%)	4
Pain - chest	3/30 (10%)	3
Pain - scapular	1/30 (3.3%)	1
Pain - skull	1/30 (3.3%)	1
Right hip - slightly limited range of motion	1/30 (3.3%)	1
Sacro-iliac pain, right side	1/30 (3.3%)	1
Tenderness	1/30 (3.3%)	1
Weakness	1/30 (3.3%)	1
Body aches	1/30 (3.3%)	1
Chest pain	1/30 (3.3%)	1
Flexion contracture, left knee (musculoskeletal, other)	1/30 (3.3%)	1
Left hip pain/left groin pain (musculoskeletal)	1/30 (3.3%)	1
Musculoskeletal/Soft tissue: Cervical spine-range of motion	1/30 (3.3%)	1
Musculoskeletal/Soft tissue: Extremity-lower	1/30 (3.3%)	3
Musculoskeletal/Soft tissue: Extremity-lower (gait/walking)	1/30 (3.3%)	1
Musculoskeletal/Soft tissue: Muscle weakness	1/30 (3.3%)	1
Musculoskeletal/Soft tissue: Muscular/Skeletal hypoplasia	1/30 (3.3%)	6
Pain/Musculoskeletal::Extremity-limb, upper	1/30 (3.3%)	3
Musculoskeletal::Muscle	4/30 (13.3%)	22
Pain/Musculoskeletal::Oral cavity	1/30 (3.3%)	1
Pain/Myalgia (Muscle pain)	1/30 (3.3%)	3
Pain:Arthralgia (joint pain)	1/30 (3.3%)	1
Pain:Musculoskeletal - Joint	3/30 (10%)	5
Pain:Other back	1/30 (3.3%)	1
Pain: Other hip	1/30 (3.3%)	1
Pain: Other leg	1/30 (3.3%)	1
Pain: Other neck	1/30 (3.3%)	1
Pain: Other upper right quadrant	1/30 (3.3%)	1
Pain::Pain::Back	5/30 (16.7%)	12
Pain::Pain::Bone	5/30 (16.7%)	17
Pain::Pain::Chest wall	1/30 (3.3%)	2
Pain::Pain::Chest/thorax NOS	2/30 (6.7%)	3
Pain::Pain::Extremity-limb	4/30 (13.3%)	5
Pain::Pain::Muscle	5/30 (16.7%)	15
Pain::Pain::Paraspinal	1/30 (3.3%)	3
Pain::Joint	1/30 (3.3%)	1
Sore rib cage on left side	1/30 (3.3%)	1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Pain:Tumor	1/30 (3.3%)	1
Nervous system disorders
Agitated mental status	1/30 (3.3%)	1
Dystonia	1/30 (3.3%)	1
Hallucination(s)	2/30 (6.7%)	2
Mild neuropathy of right foot	1/30 (3.3%)	1
Mood alteration - anxiety	3/30 (10%)	3
Mood alteration-depression	2/30 (6.7%)	2
Neurology: Mood alteration: agitation	2/30 (6.7%)	3
Neurology: mood alteration: anxiety	9/30 (30%)	21
Neurology: mood alteration: depression	12/30 (40%)	27
Neurology: neuropathy: cranial - select neuropathy: CN XII motor- tongue	1/30 (3.3%)	1
Neurology: neuropathy: sensory	7/30 (23.3%)	20
Neurology: seizure	2/30 (6.7%)	3
Neurology: tremor	9/30 (30%)	19
Neuropathy	1/30 (3.3%)	1
Other, sixth nerve palsy	1/30 (3.3%)	1
Pain, Headache	3/30 (10%)	3
Pain/Neurology:Head/Headache	6/30 (20%)	12
Somnolence	1/30 (3.3%)	1
Vertigo (sensation)	1/30 (3.3%)	1
Visual hallucination	1/30 (3.3%)	1
Anxiety	1/30 (3.3%)	1
Neurology: Extrapyramidal/involuntary movement/restlessness	1/30 (3.3%)	1
Neurology:Ataxia (incoordination)	1/30 (3.3%)	5
Neurology: Confusion	1/30 (3.3%)	1
Neurology: Insomnia	1/30 (3.3%)	3
Neurology: neuropathy: motor	1/30 (3.3%)	5
Neurology: other	1/30 (3.3%)	4
Pain/Neurology::Neuralgia/peripheral nerve	1/30 (3.3%)	2
Pain::Pain::Head/headache	3/30 (10%)	8
Pain::Head/headache	1/30 (3.3%)	1
Psychiatric disorders
Psychotic episode	1/30 (3.3%)	1
Renal and urinary disorders
Creatinine	4/30 (13.3%)	4
Dysuria	3/30 (10%)	3
Hematuria	1/30 (3.3%)	1
Proteinuria	1/30 (3.3%)	1
Urinary tract infection	1/30 (3.3%)	1
Cystitis	1/30 (3.3%)	1
Hemorrhage/Bleeding: Hemorrhage, GU::Urinary NOS	2/30 (6.7%)	8
Interstitial nephritis	1/30 (3.3%)	1
Pain - Select: Renal/Genitourinary - Kidney	1/30 (3.3%)	2
Renal/Genitourinary::Dysuria	2/30 (6.7%)	2
Renal/Genitourinary::Obstruction, GU::Ureter	1/30 (3.3%)	3
Renal/Genitourinary::Stricture/Stenosis, GU - Select, left hydronephrosis	1/30 (3.3%)	1
Renal/Genitourinary::Urinary frequency/urgency	1/30 (3.3%)	2
Renal/Genitourinary::Urinary retention	1/30 (3.3%)	1
URI/Sinusitis	1/30 (3.3%)	1
Reproductive system and breast disorders
Hemorrhage/Bleeding: Vagina	1/30 (3.3%)	3
Pain:vaginally	1/30 (3.3%)	1
Vaginal bleeding	1/30 (3.3%)	1
Vaginal irritation	1/30 (3.3%)	1
Pain/Sexual/Reproductive Function::Urethra	1/30 (3.3%)	2
Pain: Pelvis	1/30 (3.3%)	1
Pain::Pain - Select: Sexual/Reproductive Function - Scrotum	1/30 (3.3%)	8
Sexual/Reproductive Function - Erectile dysfunction	2/30 (6.7%)	9
Sexual/Reproductive::Other	1/30 (3.3%)	3
Respiratory, thoracic and mediastinal disorders
Asthma	1/30 (3.3%)	1
Fibrosis lung	1/30 (3.3%)	1
Hemorrhage, epistaxis	4/30 (13.3%)	4
Hemorrhage/Bleeding: Hemorrhage, pulmonary/upper respiratory-select	1/30 (3.3%)	2
Hypoxia-Hypoxemic	1/30 (3.3%)	1
Increased cavitary nodular infiltrate in left lower lobe	1/30 (3.3%)	1
Infiltrate (lung)	1/30 (3.3%)	1
Mild cough	1/30 (3.3%)	1
Pain/Pulmonary/Upper Respiratory: Chest/thorax NOS	1/30 (3.3%)	2
Pain/Pulmonary/Upper Respiratory:Chest wall	5/30 (16.7%)	6
Pain/Pulmonary/Upper Respiratory:Pleura	1/30 (3.3%)	1
Pain/Pulmonary/Upper Respiratory:Sinus	1/30 (3.3%)	3
Pain: pleuritic	1/30 (3.3%)	1
Respiratory tract infection	1/30 (3.3%)	1
Respiratory, other, tachypnea	1/30 (3.3%)	1
Pansinusitis	2/30 (6.7%)	2
Pneumonia	3/30 (10%)	3
Pleural effusion	1/30 (3.3%)	1
Pneumonitis/pulmonary infiltrates	1/30 (3.3%)	1
Post-nasal drainage	1/30 (3.3%)	1
Pulmonary effusion	2/30 (6.7%)	2
Pulmonary emboli	1/30 (3.3%)	1
Pulmonary/Upper Respiratory: Other	1/30 (3.3%)	1
Pulmonary/Upper Respiratory: Atelectasis	1/30 (3.3%)	1
Pulmonary/Upper Respiratory: Hypoxemia	1/30 (3.3%)	1
Pulmonary/Upper Respiratory: Pneumothorax	1/30 (3.3%)	2
Skin lesions	1/30 (3.3%)	1
Hemorrhage/Bleeding: Hemorrhage, pulmonary/upper respiratory::Nose	2/30 (6.7%)	2
Pain - Other: Breathing	1/30 (3.3%)	3
Pain/Pulmonary/Upper Respiratory::Throat/pharynx/Larynx	5/30 (16.7%)	18
Pulmonary/Upper Respiratory::Bronchospasm, wheezing	5/30 (16.7%)	8
Pulmonary/Upper Respiratory: Cough	18/30 (60%)	40
Pulmonary/Upper Respiratory:Dyspnea (shortness of breath)	10/30 (33.3%)	14
Pulmonary/Upper Respiratory: Fev1	1/30 (3.3%)	3
Pulmonary/Upper Respiratory: Hypoxia	2/30 (6.7%)	2
Pulmonary/Upper Respiratory: Nasal cavity/paranasal sinus reactions	1/30 (3.3%)	3
Pulmonary/Upper Respiratory:pleural effusion (non-malignant)	1/30 (3.3%)	3
Pulmonary/Upper Respiratory: Pneumonitis/pulmonary infiltrates	3/30 (10%)	5
Pulmonary/Upper Respiratory: Pulmonary/Upper Respiratory: Other (Specify, crackles)	3/30 (10%)	5
Pulmonary: Pulmonary Other	1/30 (3.3%)	5
Pulmonary:Dyspnea	2/30 (6.7%)	2
Pulmonary emboli, B/L	1/30 (3.3%)	1
Skin and subcutaneous tissue disorders
Acne breakout	1/30 (3.3%)	1
Dermatology/Skin: Pigmentation changes	2/30 (6.7%)	3
Dermatology/Skin: Dermatology/Skin-Other (lip abrasion)	1/30 (3.3%)	1
Dermatology/Skin: Dermatology/Skin-Other (specify: verrucae)	1/30 (3.3%)	2
Dermatology/Skin: Dry skin	9/30 (30%)	29
Dermatology/Skin: Rash/dermatitis associated with high dose chemotherapy or BMT studies	1/30 (3.3%)	11
Dermatology/Skin: Rash: acne	1/30 (3.3%)	2
Dermatology/Skin: Bruising (in absence of Grade 3 or 4 thrombocytopenia)	6/30 (20%)	14
Dermatology/Skin: Dermatology/Skin-Other (specify, keratosis pilaris)	2/30 (6.7%)	2
Dermatology/Skin: Hypopigmentation	3/30 (10%)	10
Dermatology/Skin: Nail changes	2/30 (6.7%)	5
Dermatology/Skin: Photosensitivity	1/30 (3.3%)	4
Dermatology/Skin: Rash/desquamation	15/30 (50%)	130
Dermatology/Skin: Rash/desquamation associated with graft versus host disease (GVHD)	5/30 (16.7%)	28
Dermatology/Skin: Rash: dermatitis associated with radiation-select: radiation	1/30 (3.3%)	1
Dermatology/Skin: Striae	1/30 (3.3%)	5
Desquamation of palmar surface of hands, B/L	1/30 (3.3%)	1
Desquamation, vaginal	1/30 (3.3%)	1
Epidermal abrasions	1/30 (3.3%)	1
Erythema	2/30 (6.7%)	2
Erythema, palms of hands	2/30 (6.7%)	2
Erythema - abdominal incision	1/30 (3.3%)	1
Erythema - buccal mucosa	1/30 (3.3%)	1
Erythema-face	1/30 (3.3%)	1
Erythema - right great toe pus	1/30 (3.3%)	1
Erythemateous patchy rash - mild, primarily involving trunk but listed (back, chest, arms)	1/30 (3.3%)	1
Hemorrhage/Bleeding: Petechiae/purpura (hemorrhage/bleeding into skin or mucosa)	4/30 (13.3%)	7
Hives	1/30 (3.3%)	1
Macular rash	1/30 (3.3%)	1
Macular rash on anterior and posterior chest	1/30 (3.3%)	1
Molluscum rash to upper thighs B/L	1/30 (3.3%)	1
Papular rash hands	1/30 (3.3%)	1
Peeling-bilateral palms	1/30 (3.3%)	1
Urticaria	2/30 (6.7%)	2
Palmar erythema	1/30 (3.3%)	1
Patchy erythema, upper and lower extremities, palms and soles, B/L	1/30 (3.3%)	1
Petechiae	1/30 (3.3%)	1
Pigmentation changes	1/30 (3.3%)	1
Pruritis	4/30 (13.3%)	4
Radiation Dermatits	2/30 (6.7%)	2
Rash - erythematous, mild, lacy	1/30 (3.3%)	1
Rash - trunk, arms, face, legs, sparing feet	1/30 (3.3%)	1
Rash (Acne)	1/30 (3.3%)	1
Rash (maculopapular) GVHD	1/30 (3.3%)	1
Rash (port erythema)	1/30 (3.3%)	1
Rash, B/L LEs and UEs	1/30 (3.3%)	1
Rash, erythematous (GVHD)	1/30 (3.3%)	1
Rash, palmar surface	1/30 (3.3%)	1
Rash, erythema	1/30 (3.3%)	1
Rash - chest & palms of hands	1/30 (3.3%)	1
Rash - soles	1/30 (3.3%)	1
Skin fibrosis	1/30 (3.3%)	1
Dermatology/Skin: Bruising	2/30 (6.7%)	12
Dermatology/Skin: Hand-foot skin reaction	1/30 (3.3%)	3
Dermatology/Skin: Chelitis	2/30 (6.7%)	3
Dermatology/Skin: Hair loss/alopecia (scalp or body)	11/30 (36.7%)	47
Dermatology/Skin: Injection site reaction/extravasation changes	1/30 (3.3%)	1
Dermatology/Skin: Other	1/30 (3.3%)	3
Dermatology/Skin: Pruritis	1/30 (3.3%)	1
Dermatology/Skin: Pruritis/itching	4/30 (13.3%)	9
Dermatology/Skin: Radiation dermatitis	1/30 (3.3%)	7
Dermatology/Skin: Rash: acne/acneiform	6/30 (20%)	24
Dermatology/Skin: Rash: dermatitis	1/30 (3.3%)	2
Dermatology/Skin: Skin breakdown/decubitus ulcer	3/30 (10%)	6
Dermatology/Skin: Ulceration	1/30 (3.3%)	1
Dermatology/Skin: Urticaria (hives, welts, wheals)	3/30 (10%)	3
Dermatology/Skin: Bruising (in absence of Grade 3	1/30 (3.3%)	3
Dermatology: Hypopigmentation	1/30 (3.3%)	1
Hemorrhage/Bleeding: Petechiae/purpura (hemorrhage/bleeding into skin or mucosa)	1/30 (3.3%)	2
Hemorrhage: Petechiae	1/30 (3.3%)	1
Mild erythema (IV access site)	1/30 (3.3%)	1
Mult. abdominal ecchymosis	1/30 (3.3%)	1
Pain/Dermatology/Skin::Face	1/30 (3.3%)	1
Pain/Dermatology/Skin::Oral	1/30 (3.3%)	1
Pain:Dermatology/Skin - Oral gums	1/30 (3.3%)	1
Radiodermatitis, right lateral leg	1/30 (3.3%)	3
Verrucae	1/30 (3.3%)	1
Vascular disorders
Thrombophlebitis, superficial, B/L	1/30 (3.3%)	1
Vascular: thrombosis/thrombus/embolism	1/30 (3.3%)	1
Vascular::Vessel injury - vein-Select:Other NOS	1/30 (3.3%)	4

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.