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CLINICAL TRIAL
JOURNAL ARTICLE
RESEARCH SUPPORT, NON-U.S. GOV'T
HLA-mismatched hematopoietic SCT without in vitro T-cell depletion for myelodysplastic syndrome.
Bone Marrow Transplantation 2010 August
Allogeneic hematopoietic SCT (HSCT) is currently the only curative treatment for myelodysplastic syndrome (MDS). However, many patients cannot find an HLA-matched donor. We have developed a new protocol for HLA-mismatched (including haploidentical) HSCT using G-CSF-primed BM plus G-CSF-mobilized PBSCs without in vitro T-cell depletion. A total of 36 patients diagnosed with high-risk MDS (RAEB (refractory anemia with excess blasts) or RAEBt (RAEB in transformation)) underwent transplantation from HLA-mismatched family donors. All patients achieved sustained myeloid engraftment. The cumulative incidence of grades II-IV acute GVHD (aGVHD) was 60% and that of grades III and IV aGVHD was 15%. The 2-year cumulative incidence of chronic GVHD was 56%. After a median follow-up of 17 months, 4 patients had relapsed and died and 25 patients were still alive. The 2-year probability of leukemia-free survival (LFS) was 65%. Patients transplanted within 7 months of diagnosis had better LFS (89 vs 43% ). Severe aGVHD decreased the LFS significantly by increasing non-relapse mortality (NRM). This study confirms that HLA-mismatched HSCT is a treatment option for MDS. Patients with high-risk MDS benefit from receiving HSCT early in the course of the disease.
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