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Successfull Engraftment and Clearance of Donor Specific Antibodies after Haploidentical Related Stem Cell Transplantation for an Adult Patient with Sickle Cell Disease

Blood(2014)

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摘要
Introduction: Sickle cell disease (SCD) is one of the most common genetic disorders in the world and despite advances in best supportive care it remains a disease with high risk of morbidity and mortality. Allogeneic hematopoietic stem cell transplantation (alloHSCT) is the only curative treatment modality. Reports of adults are limited because of the lack of suitable matched donors and the high treatment related toxicity (TRT) after conventional myeloablative conditioning regimen resulting from the accumulated disease specific end organ damage. Initial reports of non myeloablative conditioning regimen showed disappointing results because of the high risk of graft failure (GF) and disease recurrence. Recently, the development of reduced toxicity conditioning regimen (RTC) with lower TRT and the use of haploidentical family donors has widened the applicability of alloHSCT. Since SCD patients (pt) are highly alloimmunosized, donor HLA-specific antibodies (DSA) are often detectable and might compromise engraftment in HLA mismatched transplants.
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