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Posttransplant cyclophosphamide for haploidentical stem cell transplantation in children with Wiskott–Aldrich syndrome
Author(s) -
Yue Yan,
Shi Xiaodong,
Song Zeliang,
Qin Jiayue,
Li Junhui,
Feng Shunqiao,
Liu Rong
Publication year - 2018
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.27092
Subject(s) - medicine , wiskott–aldrich syndrome , cyclophosphamide , stem cell , transplantation , immunology , oncology , chemotherapy , genetics , gene , biology
Abstract Background Hematopoietic stem cell transplantation (HSCT) is the curative treatment for Wiskott–Aldrich syndrome (WAS). However, it is difficult to find a matched donor for patients. Therefore, haploidentical donors should be considered for patients lacking a suitable donor. Our pilot study evaluated whether HSCT with posttransplantation cyclophosphamide (PTCy) is an effective treatment for WAS. Methods Haploidentical family donors were selected as donor sources for a total of five patients without a suitable donor between March 2015 and March 2017. A modified transplant protocol using PTCy (50 mg/kg/day on days +3 and +4) was performed, including busulfan (16 mg/kg), fludarabine (150 mg/m 2 ), and rabbit antihuman thymocyte globulin (7.5 mg/kg). Results The median time for neutrophil recovery over 1,000 × 10 3 /mm 3 was 15 days (range, 12–18 days), and that for keeping platelets counts over 50,000/mm 3 was 27.5 days (range, 20–35 days). The median follow‐up was 2.1 years (range, 1.4–2.5 years). Two patients developed grade I acute graft‐versus‐host disease (GVHD), and one patient had limited chronic GVHD. All five patients are alive and independent of platelet infusion with 100% donor chimerism. Conclusion Our pilot study suggests that HSCT with modified PTCy is a safe and effective treatment for WAS, which needs further clinical practice and research.