Posttransplant cyclophosphamide for haploidentical stem cell transplantation in children with Wiskott-Aldrich syndrome.

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. 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/m2 ), and rabbit antihuman thymocyte globulin (7.5 mg/kg). The median time for neutrophil recovery over 1,000 × 103 /mm3 was 15 days (range, 12-18 days), and that for keeping platelets counts over 50,000/mm3 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. Our pilot study suggests that HSCT with modified PTCy is a safe and effective treatment for WAS, which needs further clinical practice and research.

Yue Y ，Shi X ，Song Z ，Qin J ，Li J ，Feng S ，Liu R ... -  《-》

Successful Haploidentical Stem Cell Transplant With Posttransplant Cyclophosphamide in Wiskott-Aldrich Syndrome With Myeloablative Conditioning.

Hematopoietic stem cell transplant (HSCT) is the only curative treatment modality for Wiskott-Aldrich syndrome. Haploidentical HSCT with posttransplant cyclophosphamide (PTCy) is an upcoming option in children with nonmalignant conditions. However, only few cases have been reported for Wiskott-Aldrich syndrome HSCT with PTCy approach. Here we report a 4-year-old boy, treated successfully by haploidentical HSCT with myeloablative conditioning (busulfan, fludarabine, and thiotepa) and PTCy. Posttransplant chimerism was fully donor. Of 13 cases (current case and other 12 published cases) 10 are alive and disease free after haploidentical HSCT with PTCy. Haploidentical HSCT with PTCy using myeloablative conditioning is feasible and safe.

Sharma A ，Rastogi N ，Kapoor R ，Chatterjee G ，Yadav SP ... -  《-》

Successful Reduced Intensity Conditioning Alternate Donor Stem Cell Transplant for Wiskott-Aldrich Syndrome.

Thakkar D ，Katewa S ，Rastogi N ，Kohli S ，Nivargi S ，Yadav SP ... -  《-》

Allogeneic hemopoietic stem cell transplantation (HSCT) for Wiskott-Aldrich syndrome: a report of the Spanish Working Party for Blood and Marrow Transplantation in Children (GETMON).

Muñoz A ，Olivé T ，Martinez A ，Bureo E ，Maldonado MS ，Diaz de Heredia C ，Sastre A ，Gonzalez-Vicent M ，Spanish Working Party for Blood and Marrow Transplantation in Children (GETMON) ... -  《-》

Successful Haploidentical Stem Cell Transplant With Posttransplant Cyclophosphamide for Hemophagocytic Lymphohistiocytosis.

Kohli S ，Rastogi N ，Nivargi S ，Thakkar D ，Katewa S ，Yadav SP ... -  《-》