Assessment Of Calcineurin Inhibitor-Associated Neurotoxicity In Patients With Sickle Cell Disease Receiving A Matched Sibling Donor Or T-Cell Depleted Haploidentical Hematopoietic Stem Cell Transplantation

BLOOD(2018)

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摘要
Introduction: Sickle cell disease (SCD) is one of the most prevalent monogenic hematological disorders leading to progressive multi-organ damage and reduced life expectancy despite a significant improvement of conventional and supportive care. Allogeneic hematopoietic stem cell transplantation (HSCT) with a matched sibling donor (MSD) represents currently the only curative treatment option, limited by a donor availability <20%. In a pilot study, we compared α/β/CD19 and CD3/CD19, respectively T-cell depleted haploidentical HSCT (T-haplo-HSCT) with MSD HSCT in patients (pts) with advanced stage SCD, using almost identical HSCT regimens for both donor groups.
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