Peripheral Blood Stem Cell Transplant vs Bone Marrow Transplant in Individuals With Hematologic Cancers (BMT CTN 0201)
Study Details
Study Description
Brief Summary
The study is designed as a Phase III, randomized, open label, multicenter, prospective, comparative trial of granulocyte colony stimulating factor (G-CSF)-mobilized peripheral blood stem cells (PBSC) versus marrow from unrelated donors for transplantation in patients with hematologic malignancies. Recipients will be stratified by transplant center and disease risk and will be randomized to either the PBSC or marrow arm in a 1:1 ratio.
Condition or Disease | Intervention/Treatment | Phase |
---|---|---|
|
Phase 3 |
Detailed Description
BACKGROUND:
Many studies of allogeneic marrow transplantation have shown that a higher dose of marrow cells correlates with more robust hematopoietic engraftment and lower mortality from infectious complications. Peripheral blood stem cells (PBSC) collected after mobilization with granulocyte colony stimulating factor (G-CSF) contain a larger number of CD34-positive (CD34) progenitors and total cells than bone marrow. These observations led to the hypothesis that transplantation of PBSC would lead to lower mortality compared to transplantation of marrow. In addition, PBSC grafts have a higher T cell content, predicting a possibly more powerful anti-leukemia effect. However, the higher T cell content of PBSC may also lead to increased incidence and severity of acute and chronic graft-versus-host disease (GVHD). This concern is especially serious when the donor is unrelated to the recipient. This prospective, randomized, multicenter clinical trial of unrelated donor transplantation will test the hypothesis that transplantation of PBSC leads to similar patient survival compared to transplantation of marrow.
DESIGN NARRATIVE:
This is a Phase III randomized, open label, multicenter clinical trial sponsored by the National Marrow Donor Program (NMDP) and the National Institutes of Health (NIH). The objective of the trial is to test the null hypothesis that there is no difference in overall survival after PBSC versus marrow transplants from HLA compatible unrelated donors. The study will compare G-CSF-mobilized PBSC transplantation with bone marrow transplantation from HLA-compatible unrelated donors for patients with leukemia, myelodysplastic or myeloproliferative syndromes. Conditioning and GVHD prophylaxis regimens will vary by center and within centers, however, the center must declare before randomization what regimens will be used for each patient. The primary endpoint of this trial is 2-year survival following randomization. Secondary analyses will consider neutrophil and platelet recovery, acute and chronic GVHD, time off all immunosuppressive therapy, relapse, infections, adverse events and immune reconstitution. The trial will include evaluation of patient and donor quality of life, composition of the graft, and immune reconstitution. Accrual is anticipated for 3 years with a follow-up period of 3 years.
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
---|---|
Active Comparator: Bone Marrow Transplant Allogeneic bone marrow transplantation |
Biological: Allogeneic bone marrow transplantation
Bone marrow transplant from HLA compatible unrelated donors.
|
Active Comparator: Blood Stem Cell Transplant Peripheral blood stem cell transplantation |
Biological: Peripheral blood stem cell transplantation
Peripheral blood transplant from HLA compatible unrelated donors.
|
Outcome Measures
Primary Outcome Measures
- Two-year Overall Survival [Measured at 2 years]
Overall survival rate at 2 years according to an intention-to-treat analysis.
Secondary Outcome Measures
- Neutrophil Engraftment [Measured at Day 28]
- Platelet Engraftment [Measured at Day 180]
- Graft Failure [Measured at 28 and 100 days]
- Extensive Chronic Graft-versus-host Disease (GVHD) [Measured at 730 days]
- Chronic GVHD [Measured at 2 years]
- Relapse [Measured at 2 years]
Analysis restricted to patients who received the transplant.
- Infections [Measured at 1 and 2 years]
Number of infection reports per patient.
- Grades III-V Unexpected Adverse Events [Measured by 2 years]
- Acute GVHD Grade II-IV [100 days, 180 days]
- Acute GVHD Grade III-IV [100 days, 180 days]
- Current Immunosuppressive (IS) Free Survival [Measured at 2 years]
This outcome measure takes into account subsequent immunosuppressive therapy that may occur following discontinuation of initial immunosuppressive therapy.
- Immune Reconstitution [Measured at 100 days, 6 months, and 1 and 2 years]
- Donor Recovery of Baseline Complete Blood Count (CBC) and White Blood Cell Count (WBC) Differential [Measured at 1, 6, and 12 months]
- Donor Recovery to Baseline Toxicity Scores [Measured at 1, 6, and 12 months]
- Donor Quality of Life [Measured at 1, 6, and 12 months]
- Patient Quality of Life [Measured at baseline, 6 months, and 1, 2, and 5 years]
Eligibility Criteria
Criteria
Patient Inclusion Criteria:
One of the following diagnoses:
-
Acute myelogenous leukemia at the following stages: first remission, second remission, third or subsequent remission, or not in remission
-
Acute lymphoblastic leukemia at the following stages: first remission, second remission, third or subsequent remission, or not in remission
-
Chronic myelogenous leukemia at the following stages: chronic phase, accelerated phase, or blast phase
-
Myelodysplastic syndromes (MDS) at the following stages: refractory anemia; refractory anemia with ringed sideroblasts; refractory cytopenia with multilineage dysplasia; refractory cytopenia with multilineage dysplasia and ringed sideroblasts; refractory anemia with excess blasts-1 (5-10% blasts); refractory anemia with excess blasts-2 (10-20% blasts); myelodysplastic syndrome, unclassified; or MDS associated with isolated del (5q)
-
Myeloproliferative diseases: chronic myelomonocytic leukemia; agnogenic myeloid metaplasia with myelofibrosis (idiopathic myelofibrosis); juvenile myelomonocytic leukemia
-
Therapy-related acute myelogenous leukemia (AML) or MDS with prior malignancy that has been in remission for at least 12 months. If the remission is less than 12 months, Medical Monitor or Protocol Chair approval is required for eligibility
Patient Exclusion Criteria:
-
Prior allogeneic or autologous transplants using any hematopoietic stem cell source; patients with secondary malignancies who have had a prior autologous transplant will be eligible; the prior autologous transplant must have been performed for the primary malignancy (such as lymphoma) and must have occurred 12 or more months prior to enrollment
-
Lymphoma (11% of 2001 NMDP transplants), other malignant disorders (6%), and non-malignant disorders (9%)
Donor Inclusion Criteria:
- Matched for HLA-A, B, and DRB1 antigens
-
One antigen mismatch at HLA-A, B, or DRB1 is acceptable with or without mismatch at HLA-C
-
Typing is by DNA techniques: intermediate resolution for A, B, and C, and high resolution for DRB1. HLA-C typing is mandatory but will not count in the match.
-
Willing to undergo both bone marrow harvest and G-CSF administration with apheresis
-
Willing to be randomly assigned to either marrow or PBSC collection
-
Adequate peripheral venous access for leukapheresis or willing to undergo placement of a central catheter
-
Donor center affiliation with NMDP
-
Additional donor inclusion criteria can be found in the Donor Companion Manual
Donor Exclusion Criteria:
-
Pregnant (positive serum β-HCG) or uninterruptible breastfeeding
-
Known allergy to G-CSF or to E. Coli-derived recombinant protein products
-
History of autoimmune disorders
-
History of deep vein thrombosis or venous thromboembolism
-
History of iritis or episcleritis
-
History of serious adverse reaction to anesthesia
-
Thrombocytopenia (platelets less than 150,000 per mcL) at baseline evaluation
-
Current treatment with lithium
-
Presence of sickle hemoglobin as demonstrated by appropriate testing such as hemoglobin electrophoresis
-
Receiving experimental therapy or investigational agents
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
---|---|---|---|---|---|
1 | University of Alabama at Birmingham | Birmingham | Alabama | United States | 35294 |
2 | City of Hope National Medical Center | Duarte | California | United States | 91010 |
3 | UCSD Cancer Center | La Jolla | California | United States | 92093-0960 |
4 | University of California, San Francisco | San Francisco | California | United States | 94143 |
5 | Stanford Hospital and Clinics | Stanford | California | United States | 94305 |
6 | University of Florida College of Medicine (Shands) | Gainesville | Florida | United States | 32610-100277 |
7 | Emory University | Atlanta | Georgia | United States | 30322 |
8 | Loyola University | Maywood | Illinois | United States | 60153 |
9 | IBMT (Indiana Blood and Marrow Transplant) at St Francis Franciscan Health | Indianapolis | Indiana | United States | 46237 |
10 | University of Iowa Hospitals and Clinics | Iowa City | Iowa | United States | 52242 |
11 | University of Kansas Hospital | Kansas City | Kansas | United States | 66160 |
12 | University of Maryland | Baltimore | Maryland | United States | 21228 |
13 | DFCI/Brigham & Women's | Boston | Massachusetts | United States | 02114 |
14 | University of Michigan Medical Center | Ann Arbor | Michigan | United States | 48109-0942 |
15 | University of Minnesota | Minneapolis | Minnesota | United States | 55455 |
16 | Mayo Clinic Cancer Center | Rochester | Minnesota | United States | 55905 |
17 | Washington University/Barnes Jewish Hospital | Saint Louis | Missouri | United States | 63110 |
18 | Washington University/St. Louis Children's Hospital | Saint Louis | Missouri | United States | 63110 |
19 | University of Nebraska Medical Center | Omaha | Nebraska | United States | 68198-3330 |
20 | Hackensack University Medical Center Cancer Center | Hackensack | New Jersey | United States | 07601 |
21 | Roswell Park Cancer Institute | Buffalo | New York | United States | 14263-0001 |
22 | Cohen Children's Hospital | New Hyde Park | New York | United States | 11040 |
23 | Duke University Medical Center | Durham | North Carolina | United States | 27705 |
24 | Wake Forest University Health Sciences | Winston-Salem | North Carolina | United States | 27157-1082 |
25 | Ohio State/Arthur G. James Cancer Hospital | Columbus | Ohio | United States | 43210 |
26 | University of Oklahoma Medical Center | Oklahoma City | Oklahoma | United States | 73104 |
27 | Oregon Health & Science University (Peds) | Portland | Oregon | United States | 97239-3098 |
28 | Oregon Health Sciences University | Portland | Oregon | United States | 97239 |
29 | University of Pennsylvania Cancer Center | Philadelphia | Pennsylvania | United States | 19104 |
30 | University of Pittsburgh Cancer Institute | Pittsburgh | Pennsylvania | United States | 15232 |
31 | Vanderbilt University | Nashville | Tennessee | United States | 37232-6838 |
32 | Baylor University Medical Center | Dallas | Texas | United States | 77030 |
33 | Baylor College of Medicine/The Methodist Hospital | Houston | Texas | United States | 77030-2399 |
34 | University of Texas/MD Anderson CRC | Houston | Texas | United States | 77030-4009 |
35 | Texas Transplant Institute | San Antonio | Texas | United States | 78229 |
36 | Utah BMT/Primary Children's Medical Center | Salt Lake City | Utah | United States | 84112 |
37 | Utah BMT/University of Utah Medical School | Salt Lake City | Utah | United States | 84132 |
38 | Virginia Commonwealth University MCV Hospitals | Richmond | Virginia | United States | 23298-0037 |
39 | Fred Hutchinson Cancer Research Center | Seattle | Washington | United States | 98104 |
40 | Tom Baker Cancer Centre, Calgary | Calgary | Alberta | Canada | T2N 4N2 |
41 | Vancouver General Hospital | Vancouver | British Columbia | Canada | V5Z1M9 |
42 | Hamilton Health Sciences - McMaster Site | Hamilton | Ontario | Canada | |
43 | Ottawa Hospital | Ottawa | Ontario | Canada | |
44 | University of Toronto, Princess Margaret Hospital | Toronto | Ontario | Canada | M5G 2M9 |
45 | Queen Elizabeth II Health Sciences Centre - Halifax | Halifax | Canada |
Sponsors and Collaborators
- Medical College of Wisconsin
- National Heart, Lung, and Blood Institute (NHLBI)
- Blood and Marrow Transplant Clinical Trials Network
- National Cancer Institute (NCI)
- National Marrow Donor Program
Investigators
- Study Director: Mary Horowitz, MD, Center for International Blood and Marrow Transplant Research
Study Documents (Full-Text)
None provided.More Information
Additional Information:
Publications
None provided.- BMTCTN0201
- U01HL069294-05
- BMT CTN 0201
- 5U24CA076518
- NCT00321776
- NCT00473395
Study Results
Participant Flow
Recruitment Details | |
---|---|
Pre-assignment Detail |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Patients who underwent transplantation of bone marrow from unrelated donors; all randomized patients were included in the primary, intention-to-treat analysis. | Patients who underwent transplantation of peripheral-blood stem cells from unrelated donors; all randomized patients were included in the primary, intention-to-treat analysis. |
Period Title: Overall Study | ||
STARTED | 278 | 273 |
COMPLETED | 264 | 262 |
NOT COMPLETED | 14 | 11 |
Baseline Characteristics
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells | Total |
---|---|---|---|
Arm/Group Description | Bone marrow transplant from HLA compatible unrelated donors | Peripheral blood stem cell transplant from HLA compatible unrelated donors | Total of all reporting groups |
Overall Participants | 278 | 273 | 551 |
Age, Customized (participants) [Number] | |||
0-10 years |
9
3.2%
|
9
3.3%
|
18
3.3%
|
11-20 years |
26
9.4%
|
11
4%
|
37
6.7%
|
21-30 years |
42
15.1%
|
45
16.5%
|
87
15.8%
|
31-40 years |
42
15.1%
|
49
17.9%
|
91
16.5%
|
41-50 years |
52
18.7%
|
55
20.1%
|
107
19.4%
|
51-60 years |
69
24.8%
|
75
27.5%
|
144
26.1%
|
>=61 years |
38
13.7%
|
29
10.6%
|
67
12.2%
|
Sex: Female, Male (Count of Participants) | |||
Female |
110
39.6%
|
127
46.5%
|
237
43%
|
Male |
168
60.4%
|
146
53.5%
|
314
57%
|
Ethnicity (NIH/OMB) (Count of Participants) | |||
Hispanic or Latino |
10
3.6%
|
13
4.8%
|
23
4.2%
|
Not Hispanic or Latino |
258
92.8%
|
244
89.4%
|
502
91.1%
|
Unknown or Not Reported |
10
3.6%
|
16
5.9%
|
26
4.7%
|
Race (NIH/OMB) (Count of Participants) | |||
American Indian or Alaska Native |
1
0.4%
|
0
0%
|
1
0.2%
|
Asian |
4
1.4%
|
4
1.5%
|
8
1.5%
|
Native Hawaiian or Other Pacific Islander |
0
0%
|
0
0%
|
0
0%
|
Black or African American |
12
4.3%
|
10
3.7%
|
22
4%
|
White |
250
89.9%
|
248
90.8%
|
498
90.4%
|
More than one race |
5
1.8%
|
0
0%
|
5
0.9%
|
Unknown or Not Reported |
6
2.2%
|
11
4%
|
17
3.1%
|
Diagnosis (participants) [Number] | |||
Acute myeloid leukemia |
130
46.8%
|
131
48%
|
261
47.4%
|
Acute lymphoblastic leukemia |
61
21.9%
|
56
20.5%
|
117
21.2%
|
Chronic myeloid leukemia |
29
10.4%
|
37
13.6%
|
66
12%
|
Myelodysplastic syndrome |
52
18.7%
|
41
15%
|
93
16.9%
|
Chronic myelomonocytic leukemia |
4
1.4%
|
4
1.5%
|
8
1.5%
|
Myelofibrosis |
2
0.7%
|
4
1.5%
|
6
1.1%
|
Karnofsky performance-status score (participants) [Number] | |||
90-100% |
172
61.9%
|
154
56.4%
|
326
59.2%
|
<90% |
68
24.5%
|
74
27.1%
|
142
25.8%
|
Unknown |
38
13.7%
|
45
16.5%
|
83
15.1%
|
Cytomegalovirus serologic testing (participants) [Number] | |||
Seropositivity for CMV |
142
51.1%
|
123
45.1%
|
265
48.1%
|
Seronegativity for CMV |
121
43.5%
|
138
50.5%
|
259
47%
|
Unknown |
15
5.4%
|
12
4.4%
|
27
4.9%
|
Conditioning regimen (participants) [Number] | |||
Cyclophosphamide and total-body irradiation |
133
47.8%
|
133
48.7%
|
266
48.3%
|
Cyclophosphamide and busulfan |
90
32.4%
|
75
27.5%
|
165
29.9%
|
Fludarabine, busulfan, and antithymocyte globulin |
39
14%
|
40
14.7%
|
79
14.3%
|
Fludarabine and melphalan |
16
5.8%
|
25
9.2%
|
41
7.4%
|
GVHD prophylaxis (participants) [Number] | |||
Cyclosporine and methotrexate |
67
24.1%
|
59
21.6%
|
126
22.9%
|
Tacrolimus and methotrexate |
183
65.8%
|
196
71.8%
|
379
68.8%
|
Other |
28
10.1%
|
18
6.6%
|
46
8.3%
|
Transplantation patients (participants) [Number] | |||
Underwent transplantation |
264
95%
|
262
96%
|
526
95.5%
|
Did not undergo transplantation |
14
5%
|
11
4%
|
25
4.5%
|
Antithymocyte globulin treatment (participants) [Number] | |||
Antithymocyte globulin treatment |
65
23.4%
|
72
26.4%
|
137
24.9%
|
No Antithymocyte globulin treatment |
193
69.4%
|
183
67%
|
376
68.2%
|
Unknown |
20
7.2%
|
18
6.6%
|
38
6.9%
|
Number of donor mismatches at HLA-A, B, C, and DRB1 (participants) [Number] | |||
0 |
200
71.9%
|
209
76.6%
|
409
74.2%
|
1 |
55
19.8%
|
50
18.3%
|
105
19.1%
|
2 |
7
2.5%
|
3
1.1%
|
10
1.8%
|
3 |
2
0.7%
|
0
0%
|
2
0.4%
|
Missing |
14
5%
|
11
4%
|
25
4.5%
|
CD34+ cell dose per kilogram (x10^-6) (cell dose per kilogram (x10^-6)) [Median (Inter-Quartile Range) ] | |||
Median (Inter-Quartile Range) [cell dose per kilogram (x10^-6)] |
2.75
|
7.70
|
5.23
|
Outcome Measures
Title | Two-year Overall Survival |
---|---|
Description | Overall survival rate at 2 years according to an intention-to-treat analysis. |
Time Frame | Measured at 2 years |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Bone marrow transplant from HLA compatible unrelated donors | Peripheral blood stem cell transplant from HLA compatible unrelated donors |
Measure Participants | 278 | 273 |
Number (95% Confidence Interval) [percentage of patients] |
46
|
51
|
Title | Neutrophil Engraftment |
---|---|
Description | |
Time Frame | Measured at Day 28 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Bone marrow transplant from HLA compatible unrelated donors | Peripheral blood stem cell transplant from HLA compatible unrelated donors |
Measure Participants | 278 | 273 |
Number [percentage of patients] |
87
|
97
|
Title | Platelet Engraftment |
---|---|
Description | |
Time Frame | Measured at Day 180 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Bone marrow transplant from HLA compatible unrelated donors | Peripheral blood stem cell transplant from HLA compatible unrelated donors |
Measure Participants | 278 | 273 |
Platelet engraftment >20K from time of transplant |
85.8
|
91.6
|
Platelet engraftment >50K from time of transplant |
80.3
|
86.7
|
Title | Graft Failure |
---|---|
Description | |
Time Frame | Measured at 28 and 100 days |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Bone marrow transplant from HLA compatible unrelated donors | Peripheral blood stem cell transplant from HLA compatible unrelated donors |
Measure Participants | 278 | 274 |
Number (95% Confidence Interval) [percentage of patients] |
9
|
3
|
Title | Extensive Chronic Graft-versus-host Disease (GVHD) |
---|---|
Description | |
Time Frame | Measured at 730 days |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Patients who received transplantation of bone marrow from unrelated donors. | Patients who received transplantation of peripheral-blood stem cells from unrelated donors. |
Measure Participants | 278 | 273 |
Number (95% Confidence Interval) [percentage of patients] |
32
|
48
|
Title | Chronic GVHD |
---|---|
Description | |
Time Frame | Measured at 2 years |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Bone marrow transplant from HLA compatible unrelated donors | Peripheral blood stem cell transplant from HLA compatible unrelated donors |
Measure Participants | 278 | 273 |
Number (95% Confidence Interval) [percentage of participants] |
41
14.7%
|
53
19.4%
|
Title | Relapse |
---|---|
Description | Analysis restricted to patients who received the transplant. |
Time Frame | Measured at 2 years |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Bone marrow transplant from HLA compatible unrelated donors | Peripheral blood stem cell transplant from HLA compatible unrelated donors |
Measure Participants | 264 | 262 |
Number (95% Confidence Interval) [percentage of patients] |
28.9
|
27.5
|
Title | Infections |
---|---|
Description | Number of infection reports per patient. |
Time Frame | Measured at 1 and 2 years |
Outcome Measure Data
Analysis Population Description |
---|
Analysis restricted to patients who received the transplant. |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Bone marrow transplant from HLA compatible unrelated donors | Peripheral blood stem cell transplant from HLA compatible unrelated donors |
Measure Participants | 264 | 262 |
0 infections |
49
17.6%
|
68
24.9%
|
1 infection |
52
18.7%
|
50
18.3%
|
2 infections |
46
16.5%
|
48
17.6%
|
3 infections |
30
10.8%
|
25
9.2%
|
4 infections |
26
9.4%
|
17
6.2%
|
5 infections |
18
6.5%
|
16
5.9%
|
6 infections |
11
4%
|
8
2.9%
|
>6 infections |
32
11.5%
|
30
11%
|
Title | Grades III-V Unexpected Adverse Events |
---|---|
Description | |
Time Frame | Measured by 2 years |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Bone marrow transplant from HLA compatible unrelated donors | Peripheral blood stem cell transplant from HLA compatible unrelated donors |
Measure Participants | 278 | 273 |
Pulmonary/dyspnea |
1
0.4%
|
0
0%
|
Pericarditis with pericardial effusion |
1
0.4%
|
0
0%
|
Myocardial infarction |
1
0.4%
|
1
0.4%
|
Malignancy: skin squamous cell CA |
1
0.4%
|
0
0%
|
Acute renal failure |
1
0.4%
|
0
0%
|
R portal vein obstruction |
1
0.4%
|
0
0%
|
Right side pulmonary embolus |
1
0.4%
|
1
0.4%
|
Facial trauma - fractured L lamina |
1
0.4%
|
0
0%
|
Pulmonary embolism |
1
0.4%
|
1
0.4%
|
Blindness |
1
0.4%
|
0
0%
|
Confusion |
1
0.4%
|
0
0%
|
Intra-abdominal hemorrhage |
1
0.4%
|
0
0%
|
Death |
1
0.4%
|
0
0%
|
Altered mental status |
1
0.4%
|
0
0%
|
Pneumonia |
1
0.4%
|
0
0%
|
Vascular: pulmonary embolus |
0
0%
|
1
0.4%
|
Sudden death |
0
0%
|
1
0.4%
|
Hypertensive crisis |
0
0%
|
1
0.4%
|
Elevated LFT S |
0
0%
|
1
0.4%
|
Hemolytic transfusion reaction |
0
0%
|
1
0.4%
|
Confusion, dehydration, hygroma |
0
0%
|
1
0.4%
|
Respiratory decompensation |
0
0%
|
1
0.4%
|
Retinal tear |
0
0%
|
1
0.4%
|
Retinopathy both eyes |
0
0%
|
1
0.4%
|
Title | Acute GVHD Grade II-IV |
---|---|
Description | |
Time Frame | 100 days, 180 days |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Bone marrow transplant from HLA compatible unrelated donors | Peripheral blood stem cell transplant from HLA compatible unrelated donors |
Measure Participants | 278 | 273 |
100 days from transplantation |
45.6
|
46.7
|
180 days from transplantation |
50
|
51
|
Title | Acute GVHD Grade III-IV |
---|---|
Description | |
Time Frame | 100 days, 180 days |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Bone marrow transplant from HLA compatible unrelated donors | Peripheral blood stem cell transplant from HLA compatible unrelated donors |
Measure Participants | 278 | 273 |
100 days from transplantation |
13.8
|
16.4
|
180 days from transplantation |
14.9
|
18.8
|
Title | Current Immunosuppressive (IS) Free Survival |
---|---|
Description | This outcome measure takes into account subsequent immunosuppressive therapy that may occur following discontinuation of initial immunosuppressive therapy. |
Time Frame | Measured at 2 years |
Outcome Measure Data
Analysis Population Description |
---|
No data collected. |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Patients who received transplantation of bone marrow from unrelated donors. | Patients who received transplantation of peripheral-blood stem cells from unrelated donors. |
Measure Participants | 0 | 0 |
Title | Immune Reconstitution |
---|---|
Description | |
Time Frame | Measured at 100 days, 6 months, and 1 and 2 years |
Outcome Measure Data
Analysis Population Description |
---|
No data collected |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Patients who received transplantation of bone marrow from unrelated donors. | Patients who received transplantation of peripheral-blood stem cells from unrelated donors. |
Measure Participants | 0 | 0 |
Title | Donor Recovery of Baseline Complete Blood Count (CBC) and White Blood Cell Count (WBC) Differential |
---|---|
Description | |
Time Frame | Measured at 1, 6, and 12 months |
Outcome Measure Data
Analysis Population Description |
---|
No data collected |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Bone marrow transplant from HLA compatible unrelated donors | Peripheral blood stem cell transplant from HLA compatible unrelated donors |
Measure Participants | 0 | 0 |
Title | Donor Recovery to Baseline Toxicity Scores |
---|---|
Description | |
Time Frame | Measured at 1, 6, and 12 months |
Outcome Measure Data
Analysis Population Description |
---|
No data collected |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Bone marrow transplant from HLA compatible unrelated donors | Peripheral blood stem cell transplant from HLA compatible unrelated donors |
Measure Participants | 0 | 0 |
Title | Donor Quality of Life |
---|---|
Description | |
Time Frame | Measured at 1, 6, and 12 months |
Outcome Measure Data
Analysis Population Description |
---|
No data collected |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Bone marrow transplant from HLA compatible unrelated donors | Peripheral blood stem cell transplant from HLA compatible unrelated donors |
Measure Participants | 0 | 0 |
Title | Patient Quality of Life |
---|---|
Description | |
Time Frame | Measured at baseline, 6 months, and 1, 2, and 5 years |
Outcome Measure Data
Analysis Population Description |
---|
No data collected |
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells |
---|---|---|
Arm/Group Description | Patients who received transplantation of bone marrow from unrelated donors. | Patients who received transplantation of peripheral-blood stem cells from unrelated donors. |
Measure Participants | 0 | 0 |
Adverse Events
Time Frame | 3-years post-transplant | |||
---|---|---|---|---|
Adverse Event Reporting Description | Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected adverse events are reported here. | |||
Arm/Group Title | Bone Marrow | Peripheral-Blood Stem Cells | ||
Arm/Group Description | Bone marrow transplant from HLA compatible unrelated donors | Peripheral blood stem cell transplant from HLA compatible unrelated donors | ||
All Cause Mortality |
||||
Bone Marrow | Peripheral-Blood Stem Cells | |||
Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
Total | / (NaN) | / (NaN) | ||
Serious Adverse Events |
||||
Bone Marrow | Peripheral-Blood Stem Cells | |||
Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
Total | 13/260 (5%) | 10/261 (3.8%) | ||
Cardiac disorders | ||||
Myocardial infarction | 0/260 (0%) | 1/261 (0.4%) | 1 | |
Pericarditis | 1/260 (0.4%) | 1 | 0/261 (0%) | 1 |
Eye disorders | ||||
Blindness | 1/260 (0.4%) | 1 | 0/261 (0%) | 1 |
Retinal tear | 0/260 (0%) | 1/261 (0.4%) | 1 | |
Retinopathy | 0/260 (0%) | 1/261 (0.4%) | 1 | |
Gastrointestinal disorders | ||||
Intra-abdominal haemorrhage | 1/260 (0.4%) | 1 | 0/261 (0%) | 1 |
General disorders | ||||
Death | 1/260 (0.4%) | 1 | 0/261 (0%) | 1 |
Sudden death | 0/260 (0%) | 1/261 (0.4%) | 1 | |
Hepatobiliary disorders | ||||
Portal vein occlusion | 1/260 (0.4%) | 1 | 0/261 (0%) | 1 |
Infections and infestations | ||||
Pneumonia | 1/260 (0.4%) | 1 | 0/261 (0%) | 1 |
Injury, poisoning and procedural complications | ||||
Facial bones fracture | 1/260 (0.4%) | 1 | 0/261 (0%) | 1 |
Investigations | ||||
Hepatic enzyme increased | 0/260 (0%) | 1/261 (0.4%) | 1 | |
Neoplasms benign, malignant and unspecified (incl cysts and polyps) | ||||
Squamous cell carcinoma of skin | 1/260 (0.4%) | 1 | 0/261 (0%) | 1 |
Psychiatric disorders | ||||
Confusional state | 1/260 (0.4%) | 1 | 0/261 (0%) | 1 |
Renal and urinary disorders | ||||
Renal failure acute | 1/260 (0.4%) | 1 | 0/261 (0%) | 1 |
Respiratory, thoracic and mediastinal disorders | ||||
Acute respiratory failure | 0/260 (0%) | 1/261 (0.4%) | 1 | |
Dyspnoea | 1/260 (0.4%) | 1 | 0/261 (0%) | 1 |
Pulmonary embolism | 2/260 (0.8%) | 2 | 3/261 (1.1%) | 3 |
Vascular disorders | ||||
Hypertensive crisis | 0/260 (0%) | 1/261 (0.4%) | 1 | |
Other (Not Including Serious) Adverse Events |
||||
Bone Marrow | Peripheral-Blood Stem Cells | |||
Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
Total | 0/260 (0%) | 0/261 (0%) |
Limitations/Caveats
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.
Results Point of Contact
Name/Title | Adam Mendizabal |
---|---|
Organization | The EMMES Corporation |
Phone | 301-251-1161 |
amedizabal@EMMES.com |
- BMTCTN0201
- U01HL069294-05
- BMT CTN 0201
- 5U24CA076518
- NCT00321776
- NCT00473395