Haploidentical stem cell transplantation with post-transplant cyclophosphamide for osteopetrosis and other nonmalignant diseases

被引:16
作者
Even-Or, Ehud [1 ]
NaserEddin, Adeeb [1 ]
Dinur Schejter, Yael [1 ]
Shadur, Bella [1 ,2 ,3 ]
Zaidman, Irina [1 ]
Stepensky, Polina [1 ]
机构
[1] Hadassah Hebrew Univ, Med Ctr, Dept Bone Marrow Transplantat & Canc Immunotherap, Jerusalem, Israel
[2] Garvan Inst Med Res, Grad Res Sch, Dept Immunol, Sydney, NSW, Australia
[3] Univ New South Wales, Sydney, NSW, Australia
关键词
OUTCOMES; CHILDREN;
D O I
10.1038/s41409-020-01040-9
中图分类号
Q6 [生物物理学];
学科分类号
071011 ;
摘要
Allogeneic hematopoietic stem cell transplantation (HSCT) is curative for a variety of nonmalignant disorders including osteopetrosis, bone marrow failures, and immune deficiencies. Haploidentical HSCT is a readily available option in the absence of a matched donor, but engraftment failure and other post-transplant complications are a concern. Post-transplant cyclophosphamide (PT-Cy) regimens are gaining popularity and recent reports show promising results. We report our experience with nine pediatric patients with nonmalignant diseases who were transplanted from a haploidentical donor with PT-Cy. From 2015 to 2019, nine children with nonmalignant diseases underwent haploidentical HSCT with PT-Cy, two as a second transplant and seven as primary grafts after upfront serotherapy and busulfan-based myeloablative conditioning. Patient's diseases included osteopetrosis (n = 5), congenital amegakaryocytic thrombocytopenia (n = 2), hemophagocytic lymphohistiocytosis (n = 1), and Wiskott Aldrich syndrome (n = 1). Two patients failed to engraft following upfront PT-Cy transplants, one was salvaged with a second PT-Cy transplant, and the other with a CD34+ selected graft. None of the patients suffered from graft-versus-host disease. Three patients died from early posttransplant infectious complications and six patients are alive and well. In conclusion, haploidentical HSCT with PT-Cy is a feasible option for pediatric patients with nonmalignant diseases lacking a matched donor.
引用
收藏
页码:434 / 441
页数:8
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