Clinical trial · Interventional
Peripheral Blood Stem Cell Transplant vs Bone Marrow Transplant in Individuals With Hematologic Cancers (BMT CTN 0201)
A Phase III Randomized, Multicenter Trial Comparing G-CSF Mobilized Peripheral Blood Stem Cell With Marrow Transplantation From Human Leukocyte Antigen (HLA) Compatible Unrelated Donors (BMT CTN #0201)
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (3)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
| Myelodysplastic-Myeloproliferative Diseases | Myelodysplastic/Myeloproliferative Neoplasm | ALIAS | 0.90 |
| Myeloproliferative Disorders | Myeloproliferative Neoplasm | ALIAS | 0.90 |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Allogeneic bone marrow transplantation | Biological | — | UNRESOLVED |
| Peripheral blood stem cell transplantation | Biological | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- type
- ACTIVE_COMPARATOR
- label
- Bone Marrow Transplant
- description
- Allogeneic bone marrow transplantation
- interventionNames
- Biological: Allogeneic bone marrow transplantation
- type
- ACTIVE_COMPARATOR
- label
- Blood Stem Cell Transplant
- description
- Peripheral blood stem cell transplantation
- interventionNames
- Biological: Peripheral blood stem cell transplantation
Primary outcomes (1)
- measure
- Two-year Overall Survival
- timeFrame
- Measured at 2 years
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 66 Years
Show eligibility criteria text
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 1. One antigen mismatch at HLA-A, B, or DRB1 is acceptable with or without mismatch at HLA-C 2. 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
References
Publications (8)
- RESULTAnasetti C, Logan BR, Lee SJ, Waller EK, Weisdorf DJ, Wingard JR, Cutler CS, Westervelt P, Woolfrey A, Couban S, Ehninger G, Johnston L, Maziarz RT, Pulsipher MA, Porter DL, Mineishi S, McCarty JM, Khan SP, Anderlini P, Bensinger WI, Leitman SF, Rowley SD, Bredeson C, Carter SL, Horowitz MM, Confer DL; Blood and Marrow Transplant Clinical Trials Network. Peripheral-blood stem cells versus bone marrow from unrelated donors. N Engl J Med. 2012 Oct 18;367(16):1487-96. doi: 10.1056/NEJMoa1203517. PMID 23075175
- RESULTFoley B, Cooley S, Verneris MR, Curtsinger J, Luo X, Waller EK, Anasetti C, Weisdorf D, Miller JS. Human cytomegalovirus (CMV)-induced memory-like NKG2C(+) NK cells are transplantable and expand in vivo in response to recipient CMV antigen. J Immunol. 2012 Nov 15;189(10):5082-8. doi: 10.4049/jimmunol.1201964. Epub 2012 Oct 17. PMID 23077239
- RESULTSwitzer GE, Bruce JG, Harrington D, Haagenson M, Drexler R, Foley A, Confer D, Bishop M, Anderlini P, Rowley S, Leitman SF, Anasetti C, Wingard JR. Health-related quality of life of bone marrow versus peripheral blood stem cell donors: a prespecified subgroup analysis from a phase III RCT-BMTCTN protocol 0201. Biol Blood Marrow Transplant. 2014 Jan;20(1):118-27. doi: 10.1016/j.bbmt.2013.10.024. Epub 2013 Nov 1. PMID 24184336
- RESULTWaller EK, Logan BR, Harris WA, Devine SM, Porter DL, Mineishi S, McCarty JM, Gonzalez CE, Spitzer TR, Krijanovski OI, Linenberger ML, Woolfrey A, Howard A, Wu J, Confer DL, Anasetti C. Improved survival after transplantation of more donor plasmacytoid dendritic or naive T cells from unrelated-donor marrow grafts: results from BMTCTN 0201. J Clin Oncol. 2014 Aug 1;32(22):2365-72. doi: 10.1200/JCO.2013.54.4577. Epub 2014 Jun 30. PMID 24982459
- RESULTKhera N, Majhail NS, Brazauskas R, Wang Z, He N, Aljurf MD, Akpek G, Atsuta Y, Beattie S, Bredeson CN, Burns LJ, Dalal JD, Freytes CO, Gupta V, Inamoto Y, Lazarus HM, LeMaistre CF, Steinberg A, Szwajcer D, Wingard JR, Wirk B, Wood WA, Joffe S, Hahn TE, Loberiza FR, Anasetti C, Horowitz MM, Lee SJ. Comparison of Characteristics and Outcomes of Trial Participants and Nonparticipants: Example of Blood and Marrow Transplant Clinical Trials Network 0201 Trial. Biol Blood Marrow Transplant. 2015 Oct;21(10):1815-22. doi: 10.1016/j.bbmt.2015.06.004. Epub 2015 Jun 11.