Autologous Transplant for Multiple Myeloma

• Sponsor: Masonic Cancer Center, University of Minnesota (Other)
• Overall Status: Completed
• CT.gov ID: NCT00177047
• Collaborator: (none)
• Enrollment: 363
• Location: 1
• Arm: 1
• Actual Duration (Months): 195.4
• Patients Per Site Per Month: 1.9

Study Details

Study Description

Brief Summary

This is a study of a regimen of melphalan and autologous stem cells for patients with multiple myeloma. We hypothesize that this particular regimen will improve the survival of these patients.

Condition or Disease	Intervention/Treatment	Phase
Multiple Myeloma	Procedure: Stem Cell Transplant; Drug: Cyclophosphamide + Mesna; Drug: Melphalan; Biological: Granulocyte-colony stimulating factor	Phase 2/Phase 3

Detailed Description

Before starting treatment in this study, the bone marrow transplant (BMT) doctor will check the subject's general health. Subjects will have the following tests and evaluations to find out if they can participate:--Medical history and physical examination, including height and weight.--Blood tests (approximately 4 - 5 tablespoons) --Urine tests--Chest x-ray--Electrocardiogram (ECG or EKG)--Heart Scan (MUGA)--Pulmonary Function Test (PFT)--Bone marrow biopsies and aspirates. --If Female subjects of child-bearing age will have a serum pregnancy test performed. After eligible patients have been completely staged and exercised consent, they may undergo one cycle of chemotherapy (cyclophosphamide and Mesna) and growth factor (G-CSF) to effect cytoreduction and mobilization of PBSC for collection. All patients will receive high-dose melphalan followed by an autologous stem cell transplant (SCT). Blood tests will be performed frequently to evaluate the subject's response to treatment and possible side effects of treatment. If necessary, platelet and red cell transfusions will be given to maintain adequate levels and antibiotics will be given to treat or prevent infection. Subjects may also require intravenous nutritional support and pain medications during or after transplantation. The study coordinators will collect health information over three years. They will collect information every week for 100 days, then at 6 months, 1 year, 2 years, and 3 years.

Study Design

• Study Type: Interventional
• Actual Enrollment: 363 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Autologous Transplantation for Multiple Myeloma
• Actual Study Start Date: Apr 20, 2004
• Actual Primary Completion Date: Aug 1, 2020
• Actual Study Completion Date: Aug 1, 2020

Arms and Interventions

Arm

Intervention/Treatment

Experimental: Chemotherapy and Transplant Treatment Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.

Procedure: Stem Cell Transplant; As part of the stem cell transplant process, patients receive high doses of chemotherapy and/or radiation to treat their underlying disease, such as cancer. As one of its effects, this treatment also kills the healthy stem cells that are already in the marrow. The transplant provides new stem cells for the patient from a healthy donor; that replace the bone marrow and allow the blood counts to recover.; Other Names: Bone Marrow Transplant ; Drug: Cyclophosphamide + Mesna; Cyclophosphamide: 4mg/m^2 + Mesna. Mesna is used to reduce the undesired side effects of certain chemotherapy drugs.; Other Names: Cytoxan ; Drug: Melphalan; Administered intravenously 200 mg/m^2; Other Names: Alkeran ; Biological: Granulocyte-colony stimulating factor; Administered intravenously 10 ug/kg/day pretransplant then 5 ug/kg/day post-transplant.; Other Names: G-CSF

Outcome Measures

Primary Outcome Measures

1. Number of Participants Achieving a Complete Response [100 Days post transplant]

   Myeloma Response Definitions - Using International Uniform Response Criteria: Stringent Complete Response (sCR)requires, plus CR: Normal free light chain ratio Absence of clonal cells in bone marrow Complete Response (CR): Absence of the original monoclonal paraprotein <5% plasma cells in a bone marrow aspirate and also on trephine bone biopsy No increase in size or number of lytic bone lesions Disappearance of soft tissue plasmacytomas.

2. Number of Participants Achieving a Complete Response [6 months post transplant]

   Myeloma Response Definitions - Using International Uniform Response Criteria: Stringent Complete Response (sCR)requires, plus CR: Normal free light chain ratio Absence of clonal cells in bone marrow Complete Response (CR): Absence of the original monoclonal paraprotein <5% plasma cells in a bone marrow aspirate and also on trephine bone biopsy No increase in size or number of lytic bone lesions Disappearance of soft tissue plasmacytomas.

3. Number of Participants Achieving a Complete Response [12 months post transplant]

   Myeloma Response Definitions - Using International Uniform Response Criteria: Stringent Complete Response (sCR)requires, plus CR: Normal free light chain ratio Absence of clonal cells in bone marrow Complete Response (CR): Absence of the original monoclonal paraprotein <5% plasma cells in a bone marrow aspirate and also on trephine bone biopsy No increase in size or number of lytic bone lesions Disappearance of soft tissue plasmacytomas.

Secondary Outcome Measures

1. Number of Patients With Extended Disease-free Survival [36 Months]

   Extended disease free survival will be defined as percentage of patients surviving more than 36 months without relapse or disease progression.

2. Number of Participants With Overall Survival [1 year]

   The percentage of people in a study or treatment group who are alive for a certain period of time after they were diagnosed with or treated for a disease, such as cancer. Also called survival rate.

3. Number of Participants With Overall Survival [2 years]

   The percentage of people in a study or treatment group who are alive for a certain period of time after they were diagnosed with or treated for a disease, such as cancer. Also called survival rate.

4. Number of Participants With Overall Survival [3 years]

   The percentage of people in a study or treatment group who are alive for a certain period of time after they were diagnosed with or treated for a disease, such as cancer. Also called survival rate.

5. Count of Participants Experiencing Transplant Related Mortality [1 year]

   In the field of transplantation, toxicity is high and all deaths without previous relapse or progression are usually considered as related to transplantation.

6. Number of Participants Experiencing Incidence of Relapse [1 year]

   The return of disease after its apparent recovery/cessation.

7. Number of Participants With Disease Progression [1 year]

   Myeloma Response Definitions - Using International Uniform Response Criteria: Progressive Disease (PD) For patients not in CR or sCR, progressive disease requires one or more of the following: >25% increase in the level of the serum monoclonal paraprotein, which must also be an absolute increase of at least 0.5 g/dL. >25% increase in 24-hour urine protein electrophoresis, which must also be an absolute increase of at least 200 mg/24 hours. Absolute increase in the difference between involved and uninvolved FLC levels (absolute increase must be >10 mg/dl), only in patients without measurable paraprotein in the serum and urine. >25% increase in plasma cells in a bone marrow aspirate or on trephine biopsy, which must also be an absolute increase of at least 10%. Definite increase in the size of existing bone lesions or soft tissue plasmacytomas.

8. Time to Progression [1 year]

   Mean number of days among patients progressing

9. Time to Relapse [1 year]

   Mean number of days among patients relapsing

10. Number of Participants With Absolute Neutrophil Recovery [Day 42]

    Hematologic recovery is defined by absolute neutrophil count (ANC) >2500/μl and platelets > 100,000/μl

11. Time to Attainment of CR [12 months post transplant]

    Mean (STD) among patients achieving complete remission (CR) Myeloma Response Definitions - Using International Uniform Response Criteria: Complete Response (CR): Absence of the original monoclonal paraprotein <5% plasma cells in a bone marrow aspirate and also on trephine bone biopsy No increase in size or number of lytic bone lesions Disappearance of soft tissue plasmacytomas

12. Time to Attainment of CR+PR [12 months post transplant]

    Mean (STD) among patients achieving complete remission (CR) and partial remission (PR) Myeloma Response Definitions - Using International Uniform Response Criteria: Complete Response (CR): Absence of the original monoclonal paraprotein <5% plasma cells in a bone marrow aspirate and also on trephine bone biopsy No increase in size or number of lytic bone lesions Disappearance of soft tissue plasmacytomas. Partial Response (PR): Greater than or equal to 50% reduction in the level of the serum monoclonal paraprotein and/or reduction in 24 hour urinary monoclonal paraprotein either by greater than or equal to 90% or to <200 mg/24 hours in light chain disease. If the only measurable non-bone marrow parameter is FLC, greater than or equal to 50% reduction in the difference between involved and uninvolved FLC levels or a 50% decrease in level

13. Duration of Maintenance Treatment [During study]

14. Dropout Rate From Maintenance Therapy [Post transplant phase]

15. Number of Participants With Toxicities [By first 100 days]

    Occurrence of toxicities by first 100 days of transplant

16. Number of Participants With Infections [By first 100 days]

    Occurrence of infections in the patients by the first 100 days of transplant

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 75 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Patients meeting the Durie and Salmon criteria for initial diagnosis of multiple myeloma, requiring therapy and meeting one of the following:

• After initial therapy in either first complete or partial remission or no objective response

• After achieving initial response and later disease progression, patient will be eligible after subsequent therapy upon achievement of either complete or partial response

• Is not eligible or has refused any protocols of higher priority

• 18 - 75 years of age

• Adequate organ function defined as:

• Hematologic: hemoglobin ≥ 8 gm/dl (untransfused), white blood cells (WBC) ≥ 3000/μl, absolute neutrophil count (ANC) ≥ 1500/μl, platelets ≥ 100,000/μl (untransfused)

• Cardiac: no active ischemia, left ventricular ejection fraction > 45% by MUGA scan

• Hepatic: bilirubin < 2.0 mg/dl, ALT < 3x the upper limit of normal

• Pulmonary: FEV1-Forced Expiratory Volume in One Second AND Forced vital capacity (FVC) >50% predicted and Carbon Monoxide Diffusing Capacity (DLCO) (corrected) > 50% predicted

• Performance status: Karnofsky performance of > 80%.

• Free of active uncontrolled infection at the time of study entry.

• At time of study enrollment > 4 weeks from prior myelosuppressive chemotherapy; and > 6 weeks from prior nitrosoureas.

• Patients must exercise informed voluntary consent and sign a consent form approved by the University of Minnesota IRB: Human Subjects Committee.

Exclusion Criteria:

• Patients will be ineligible if they have advanced myeloma refractory and unresponsive to salvage chemotherapy regimens.

• Female patients who are pregnant (positive b-HCG) or breastfeeding will be excluded from study entry. In addition fertile men or women unwilling to use contraceptive techniques during and for 12 months following treatment, particularly after thalidomide will also be excluded from study entry.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Masonic Cancer Center, University of Minnesota	Minneapolis	Minnesota	United States	55455

Sponsors and Collaborators

• Masonic Cancer Center, University of Minnesota

Investigators

• Principal Investigator: Claudio Brunstein, MD, PhD, Masonic Cancer Center, University of Minnesota

Study Documents (Full-Text)

• Study Protocol and Statistical Analysis Plan - Mar 23, 2017

More Information

Publications

None provided.

• Responsible Party: Masonic Cancer Center, University of Minnesota
• ClinicalTrials.gov Identifier: NCT00177047
• Other Study ID Numbers: 2004LS001; MT2003-13; 0312M54569; NCT00293306
• First Posted: Sep 15, 2005
• Last Update Posted: Nov 9, 2021
• Last Verified: Nov 1, 2021

• Keywords provided by Masonic Cancer Center, University of Minnesota: stem cell transplantation; chemotherapy; multiple myeloma; autologous
• Additional relevant MeSH terms: Multiple Myeloma; Neoplasms, Plasma Cell; Cyclophosphamide; Melphalan; Lenograstim

Study Results

Participant Flow

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Period Title: Overall Study
STARTED	363
COMPLETED	363
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Overall Participants	363
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	257 70.8%
>=65 years	106 29.2%
Sex: Female, Male (Count of Participants)
Female	166 45.7%
Male	197 54.3%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	8 2.2%
Not Hispanic or Latino	313 86.2%
Unknown or Not Reported	42 11.6%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%
Asian	1 0.3%
Native Hawaiian or Other Pacific Islander	2 0.6%
Black or African American	35 9.6%
White	304 83.7%
More than one race	1 0.3%
Unknown or Not Reported	20 5.5%
Region of Enrollment (participants) [Number]
United States	363 100%

Outcome Measures

1. Primary Outcome

Title	Number of Participants Achieving a Complete Response
Description	Myeloma Response Definitions - Using International Uniform Response Criteria: Stringent Complete Response (sCR)requires, plus CR: Normal free light chain ratio Absence of clonal cells in bone marrow Complete Response (CR): Absence of the original monoclonal paraprotein <5% plasma cells in a bone marrow aspirate and also on trephine bone biopsy No increase in size or number of lytic bone lesions Disappearance of soft tissue plasmacytomas.
Time Frame	100 Days post transplant

Outcome Measure Data

Analysis Population Description

173 participants were not evaluable

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	190
Count of Participants [Participants]	51 14%

2. Primary Outcome

Title	Number of Participants Achieving a Complete Response
Description	Myeloma Response Definitions - Using International Uniform Response Criteria: Stringent Complete Response (sCR)requires, plus CR: Normal free light chain ratio Absence of clonal cells in bone marrow Complete Response (CR): Absence of the original monoclonal paraprotein <5% plasma cells in a bone marrow aspirate and also on trephine bone biopsy No increase in size or number of lytic bone lesions Disappearance of soft tissue plasmacytomas.
Time Frame	6 months post transplant

Outcome Measure Data

Analysis Population Description

54 participants were not evaluable

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	309
Count of Participants [Participants]	99 27.3%

3. Primary Outcome

Title	Number of Participants Achieving a Complete Response
Description	Myeloma Response Definitions - Using International Uniform Response Criteria: Stringent Complete Response (sCR)requires, plus CR: Normal free light chain ratio Absence of clonal cells in bone marrow Complete Response (CR): Absence of the original monoclonal paraprotein <5% plasma cells in a bone marrow aspirate and also on trephine bone biopsy No increase in size or number of lytic bone lesions Disappearance of soft tissue plasmacytomas.
Time Frame	12 months post transplant

Outcome Measure Data

Analysis Population Description

55 participants were not evaluable

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	308
Count of Participants [Participants]	123 33.9%

4. Secondary Outcome

Title	Number of Patients With Extended Disease-free Survival
Description	Extended disease free survival will be defined as percentage of patients surviving more than 36 months without relapse or disease progression.
Time Frame	36 Months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	363
Count of Participants [Participants]	164 45.2%

5. Secondary Outcome

Title	Number of Participants With Overall Survival
Description	The percentage of people in a study or treatment group who are alive for a certain period of time after they were diagnosed with or treated for a disease, such as cancer. Also called survival rate.
Time Frame	1 year

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	363
Count of Participants [Participants]	349 96.1%

6. Secondary Outcome

Title	Number of Participants With Overall Survival
Description	The percentage of people in a study or treatment group who are alive for a certain period of time after they were diagnosed with or treated for a disease, such as cancer. Also called survival rate.
Time Frame	2 years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	363
Count of Participants [Participants]	328 90.4%

7. Secondary Outcome

Title	Number of Participants With Overall Survival
Description	The percentage of people in a study or treatment group who are alive for a certain period of time after they were diagnosed with or treated for a disease, such as cancer. Also called survival rate.
Time Frame	3 years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	363
Count of Participants [Participants]	301 82.9%

8. Secondary Outcome

Title	Count of Participants Experiencing Transplant Related Mortality
Description	In the field of transplantation, toxicity is high and all deaths without previous relapse or progression are usually considered as related to transplantation.
Time Frame	1 year

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	363
Count of Participants [Participants]	3 0.8%

9. Secondary Outcome

Title	Number of Participants Experiencing Incidence of Relapse
Description	The return of disease after its apparent recovery/cessation.
Time Frame	1 year

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	363
Count of Participants [Participants]	69 19%

10. Secondary Outcome

Title	Number of Participants With Disease Progression
Description	Myeloma Response Definitions - Using International Uniform Response Criteria: Progressive Disease (PD) For patients not in CR or sCR, progressive disease requires one or more of the following: >25% increase in the level of the serum monoclonal paraprotein, which must also be an absolute increase of at least 0.5 g/dL. >25% increase in 24-hour urine protein electrophoresis, which must also be an absolute increase of at least 200 mg/24 hours. Absolute increase in the difference between involved and uninvolved FLC levels (absolute increase must be >10 mg/dl), only in patients without measurable paraprotein in the serum and urine. >25% increase in plasma cells in a bone marrow aspirate or on trephine biopsy, which must also be an absolute increase of at least 10%. Definite increase in the size of existing bone lesions or soft tissue plasmacytomas.
Time Frame	1 year

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	363
Count of Participants [Participants]	34 9.4%

11. Secondary Outcome

Title	Time to Progression
Description	Mean number of days among patients progressing
Time Frame	1 year

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	34
Mean (Standard Deviation) [Days]	159.4 (109.8)

12. Secondary Outcome

Title	Time to Relapse
Description	Mean number of days among patients relapsing
Time Frame	1 year

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	69
Mean (Standard Deviation) [Days]	182.9 (113.5)

13. Secondary Outcome

Title	Number of Participants With Absolute Neutrophil Recovery
Description	Hematologic recovery is defined by absolute neutrophil count (ANC) >2500/μl and platelets > 100,000/μl
Time Frame	Day 42

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	363
Count of Participants [Participants]	363 100%

14. Secondary Outcome

Title	Time to Attainment of CR
Description	Mean (STD) among patients achieving complete remission (CR) Myeloma Response Definitions - Using International Uniform Response Criteria: Complete Response (CR): Absence of the original monoclonal paraprotein <5% plasma cells in a bone marrow aspirate and also on trephine bone biopsy No increase in size or number of lytic bone lesions Disappearance of soft tissue plasmacytomas
Time Frame	12 months post transplant

Outcome Measure Data

Analysis Population Description

Data was not available on everyone for this endpoint so could only evaluable for 308 out of 363 patients due to data for participants is not available as the EPIC system was not in place when this study was conducted and participants moved to different clinics we do not have follow-up details.

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	308
Mean (Standard Deviation) [months]	4.6 (1.5)

15. Secondary Outcome

Title	Time to Attainment of CR+PR
Description	Mean (STD) among patients achieving complete remission (CR) and partial remission (PR) Myeloma Response Definitions - Using International Uniform Response Criteria: Complete Response (CR): Absence of the original monoclonal paraprotein <5% plasma cells in a bone marrow aspirate and also on trephine bone biopsy No increase in size or number of lytic bone lesions Disappearance of soft tissue plasmacytomas. Partial Response (PR): Greater than or equal to 50% reduction in the level of the serum monoclonal paraprotein and/or reduction in 24 hour urinary monoclonal paraprotein either by greater than or equal to 90% or to <200 mg/24 hours in light chain disease. If the only measurable non-bone marrow parameter is FLC, greater than or equal to 50% reduction in the difference between involved and uninvolved FLC levels or a 50% decrease in level
Time Frame	12 months post transplant

Outcome Measure Data

Analysis Population Description

55 patients were not evaluable Data was not available on everyone for this endpoint so could not evaluate all 363 patients

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	308
Mean (Standard Deviation) [months]	4.3 (1.5)

16. Secondary Outcome

Title	Duration of Maintenance Treatment
Time Frame	During study

Outcome Measure Data

Analysis Population Description

Data not available for this outcome at our center, maintenance treatment data for participants is not available as the EPIC system was not in place when this study was conducted and participants moved to different clinics so follow-up details were no available.

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	0

17. Secondary Outcome

Title	Dropout Rate From Maintenance Therapy
Time Frame	Post transplant phase

Outcome Measure Data

Analysis Population Description

Data not available for this outcome at our center, maintenance treatment data for participants is not available as the EPIC system was not in place when this study was conducted and participants moved to different clinics so follow-up details were no available.

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	0

18. Secondary Outcome

Title	Number of Participants With Toxicities
Description	Occurrence of toxicities by first 100 days of transplant
Time Frame	By first 100 days

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	363
Count of Participants [Participants]	68 18.7%

19. Secondary Outcome

Title	Number of Participants With Infections
Description	Occurrence of infections in the patients by the first 100 days of transplant
Time Frame	By first 100 days

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate.
Measure Participants	363
Count of Participants [Participants]	68 18.7%

Adverse Events

Time Frame	3 years
Arm/Group Title	Chemotherapy and Transplant Treatment
Arm/Group Description	Patients receiving peripheral blood stem cell mobilization, chemotherapy (cyclophosphamide + Mesna, growth factor (Granulocyte-colony stimulating factor) and autologous Peripheral Blood Stem Cell transplant with high dose melphalan (200 mg/m^2). Post-transplant maintenance therapy is then prescribed if appropriate. Stem Cell Transplant: As part of the stem cell transplant process, patients receive high doses of chemotherapy and/or radiation to treat their underlying disease, such as cancer. As one of its effects, this treatment also kills the healthy stem cells that are already in the marrow. The transplant provides new stem cells for the patient from a healthy donor; that replace the bone marrow and allow the blood counts to recover. Cyclophosphamide + Mesna: Cyclophosphamide: 4mg/m^2 + Mesna. Mesna is used to reduce the undesired side effects of certain chemotherapy drugs. Melphalan: Administered intravenously 200 mg/m^2 Granulocyte-colony stimulating factor: Administered intravenously 10 ug/kg/day pretransplant then 5 ug/kg/day post-transplant.
All Cause Mortality
	Chemotherapy and Transplant Treatment
	Affected / at Risk (%)	# Events
Total	62/363 (17.1%)
Serious Adverse Events
	Chemotherapy and Transplant Treatment
	Affected / at Risk (%)	# Events
Total	2/363 (0.6%)
Gastrointestinal disorders
GI bleeding	1/363 (0.3%)	1
Investigations
M-spike increase	1/363 (0.3%)	1
Other (Not Including Serious) Adverse Events
	Chemotherapy and Transplant Treatment
	Affected / at Risk (%)	# Events
Total	194/363 (53.4%)
Blood and lymphatic system disorders
Cytopenia	1/363 (0.3%)	1
Thrombocytopenia	2/363 (0.6%)	2
Thrombosis	12/363 (3.3%)	12
Cardiac disorders
Artrial fibrillation	8/363 (2.2%)	8
Cardiomyopathy	2/363 (0.6%)	2
Congestive heart failure	1/363 (0.3%)	1
Heart abnormality	1/363 (0.3%)	2
Left vertricular hypertrophy	1/363 (0.3%)	1
Low Ejection fraction	2/363 (0.6%)	2
Pericardial effusion	4/363 (1.1%)	4
Plueral effusions	1/363 (0.3%)	1
stent placement for occlusion	1/363 (0.3%)	1
Tachycardia	9/363 (2.5%)	9
Ear and labyrinth disorders
Hearing loss	2/363 (0.6%)	2
Eye disorders
Cataract	4/363 (1.1%)	4
Eye hemorrhage	2/363 (0.6%)	2
Macular degeneration	1/363 (0.3%)	1
Ocular muscle fatigue	1/363 (0.3%)	1
Gastrointestinal disorders
bowl obstruction	5/363 (1.4%)	5
Diarrhea	2/363 (0.6%)	2
Epigastric pain	1/363 (0.3%)	1
Gastritis	1/363 (0.3%)	1
GI bleed	8/363 (2.2%)	8
GI toxicity	1/363 (0.3%)	1
General disorders
Engraftment syndrome	5/363 (1.4%)	5
Hepatobiliary disorders
Encephalopathy	1/363 (0.3%)	1
Liver parenchymal disease	1/363 (0.3%)	1
Immune system disorders
Gout	3/363 (0.8%)	3
Infections and infestations
Infection	140/363 (38.6%)	246
Pneumonia	54/363 (14.9%)	77
Sepsis	2/363 (0.6%)	2
Injury, poisoning and procedural complications
Hernia	2/363 (0.6%)	2
Investigations
Elevated troponin	1/363 (0.3%)	1
Hypercalcemia	1/363 (0.3%)	1
Hypothyroidism	1/363 (0.3%)	1
Transminitis	1/363 (0.3%)	1
Metabolism and nutrition disorders
Anorexia	1/363 (0.3%)	2
Hyperglycemia	1/363 (0.3%)	1
Malnutrition	7/363 (1.9%)	8
Musculoskeletal and connective tissue disorders
Muscle tear	1/363 (0.3%)	1
Myalgia	1/363 (0.3%)	1
Physical Deconditioning	3/363 (0.8%)	3
Nervous system disorders
Akinesis	1/363 (0.3%)	1
Neuropathy	15/363 (4.1%)	15
Neurotoxicity	3/363 (0.8%)	3
Psychiatric disorders
Hallucinations	2/363 (0.6%)	2
Renal and urinary disorders
Acute kidney injury	4/363 (1.1%)	4
Edema	2/363 (0.6%)	2
Renal calculus	1/363 (0.3%)	1
Renal insufficiency	2/363 (0.6%)	2
Respiratory, thoracic and mediastinal disorders
Atelectasis	1/363 (0.3%)	1
Chest opacities	1/363 (0.3%)	3
Intubation	2/363 (0.6%)	2
Pulmonary edema	2/363 (0.6%)	2
Pulmonary embolism	3/363 (0.8%)	3
Skin and subcutaneous tissue disorders
Emphysema	1/363 (0.3%)	1
Rashes	1/363 (0.3%)	1
skin; stevens Johnson syndrome	1/363 (0.3%)	1
Surgical and medical procedures
Cholecystectomy	1/363 (0.3%)	1
Left hio hemiarhroplasty	1/363 (0.3%)	1
Vascular disorders
Blood clot	3/363 (0.8%)	3
Hemorrhage	1/363 (0.3%)	1
Hypertension	5/363 (1.4%)	5
Hypotension	2/363 (0.6%)	2

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE 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.