Haploidentical bone marrow transplant with post-transplant cyclophosphamide plus thiotepa improves donor engraftment in patients with sickle cell anemia: results of an international learning collaborative.

Biology of Blood and Marrow Transplantation(2019)

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摘要
•Multicenter learning collaborative model of haploidentical bone marrow transplantation (BMT) for sickle cell disease (SCD).•Using haploidentical BMT as curative therapy for SCD increases the donor pool.•Haploidentical BMT with post-transplantation cyclophosphamide (PTCy) plus thiotepa increases donor engraftment in SCD.•Event-free survival and overall survival are improved following haplo-BMT with PTCy plus thiotepa.
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关键词
Haploidentical,Bone marrow transplantation,Sickle cell disease,Thiotepa,Post-transplantation cyclophosphamide
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