Phase 2
Completed N=384
Allo vs Hypomethylating/Best Supportive Care in MDS (BMTCTN1102)
Source: ClinicalTrials.gov NCT02016781 ↗Enrolled (actual)
384
Serious AEs
0.0%
Results posted
Jul 2022
Primary outcomePrimary: Percentage of Participants With Overall Survival (OS) — 47.9; 26.6 percentage of participants — p=0.0001
Summary
This study is designed as a multicenter trial, with biological assignment to one of two study arms; Arm 1: Reduced intensity conditioning allogeneic hematopoietic cell transplantation (RIC-alloHCT), Arm 2: Non-Transplant Therapy/Best Supportive Care.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Overall Survival (OS) |
47.9; 26.6 | 0.0001 sig |
| SECONDARY Percentage of Participants With Leukemia-free Survival (LFS) |
35.8; 20.6 | 0.0030 sig |
| SECONDARY Quality of Life (QOL) - Functional Assessment of Cancer Therapy-General (FACT-G) |
81.5; 79.3; 81.4; 78.6; 84.0; 80.8 | 0.2777 |
| SECONDARY Quality of Life (QOL) - Medical Outcomes Study Short Form (MOS SF-36) |
38.8; 37.9; 38.1; 37.8; 39.9; 37.7 | 0.5583 |
| SECONDARY Quality of Life (QOL) - EQ-5D |
0.800; 0.823; 0.779; 0.792; 0.792; 0.772 | 0.1768 |
| SECONDARY Percentage of Participants With Overall Survival (OS) in As-treated Population |
49.4; 17.4 | < 0.0001 sig |
| SECONDARY Percentage of Participants With Leukemia-free Survival (LFS) in As-treated Population |
39.5; 11.2 | < 0.0001 sig |
| SECONDARY Percentage of Participants on HCT Arm With Overall Survival (OS) |
55.7 | — |
| SECONDARY Percentage of Participants on HCT Arm With Disease Relapse |
29.6 | — |
| SECONDARY Percentage of Participants on HCT Arm With Disease-free Survival (DFS) |
49.7 | — |
| SECONDARY Percentage of Participants on HCT Arm With Treatment-related Mortality |
20.6 | — |
| SECONDARY Percentage of Participants on HCT Arm With Grade II-IV Acute GVHD (aGVHD) |
43.1 | — |
| SECONDARY Percentage of Participants on HCT Arm With Grade III-IV Acute GVHD |
17.1 | — |
| SECONDARY Percentage of Participants on HCT Arm With Chronic GVHD |
55.5 | — |
Eligibility Criteria
Inclusion Criteria
- Patients fulfilling the following criteria will be eligible for entry into this study:
- Patients with de novo MDS who have, or have previously had, Intermediate-2 or High risk disease as determined by the International Prognostic Scoring System (IPSS). Current Intermediate-2 or High risk disease is NOT a requirement.
- Patients must have an acceptable MDS subtype:
- Refractory cytopenia with unilineage dysplasia (RCUD) (includes refractory anemia (RA))
- Refractory anemia with ringed sideroblasts (RARS)
- Refractory anemia with excess blasts (RAEB-1)
- Refractory anemia with excess blasts (RAEB-2)
- Refractory cytopenia with multilineage dysplasia (RCMD)
- Myelodysplastic syndrome with isolated del(5q) (5q-syndrome)
- Myelodysplastic syndrome (MDS), unclassifiable
- Patients must have fewer than 20% marrow blasts within 60 days of consent.
- Patients may have received prior therapy for the treatment of MDS, including but not limited to: growth factor, transfusion support, immunomodulatory (IMID) therapy, DNA hypomethylating therapy, or cytotoxic chemotherapy prior to enrollment.
- Age 50.0-75.0 years.
- Karnofsky performance status > 70 or Eastern Cooperative Oncology Group (ECOG) ≤ 1.
- Patients are eligible if no formal unrelated donor search has been activated prior to date of consent. A formal unrelated donor search begins at the time at which samples are requested from potential National Marrow Donor Program (NMDP) donors. Patients who have started a sibling donor search or who have found a matched sibling donor are eligible.
- Patients and physicians must be willing to comply with treatment assignment:
- No intent to proceed with alloHCT using donor sources not specified in this protocol, including human leukocyte antigen (HLA)-mismatched related or unrelated donors ( 5 years previously will be allowed. Cancer treated with curative surgery < 5 years previously will not be allowed unless approved by the Protocol Officer or one of the Protocol Chairs.
- Prior autologous or allogeneic HCT
- Human Immunodeficiency Virus (HIV) infection
- Patients of childbearing potential unwilling to use contraceptive techniques
- Patients with psychosocial conditions that would prevent study compliance
Data sourced from ClinicalTrials.gov (NCT02016781). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.