Outcomes after Haploidentical Stem Cell Transplantation with Post-Transplantation Cyclophosphamide in Patients with Primary Immunodeficiency Diseases.
Allogeneic hematopoietic stem cell transplantation (HCT) can cure primary immunodeficiency diseases (PID). When a HLA-matched donor is not available, a haploidentical family donor may be considered. The use of T cell-replete haploidentical HCT with post-transplantation cyclophosphamide (haplo-PTCy) in children with PID has been reported in few case series. A donor is usually readily available, and haplo-PTCy can be used in urgent cases. We studied the outcomes of 73 patients with PID who underwent haplo-PTCy, including 55 patients who did so as a first transplantation and 18 who did so as a salvage transplantation after graft failure of previous HCT. The median patient age was 1.6 years. Most of the children were male (n = 54) and had active infection at the time of transplantation (n = 50); 10 children had severe organ damage. The diagnosis was severe combined immunodeficiency (SCID) in 34 patients and non-SCID in 39 (Wiskott-Aldrich syndrome; n = 14; chronic granulomatous disease, n = 10; other PID, n = 15). The median duration of follow-up of survivors was 2 years. The cumulative incidence of neutrophil recovery was 88% in the SCID group and 84% in non-SCID group and was 81% for first transplantations and 83% after a salvage graft. At 100 days, the cumulative incidence of acute GVHD grade II-IV and III-IV was 33% and 14%, respectively. The majority of patients reached 200/μL CD4+ and 1000/μL CD3+ cell counts between 3 and 6 months. The estimated 2-year overall survival was 66%; it was 64% for SCID patients and 65% for non-SCID patients and 63% for first HCT and 77% for salvage transplantations. Twenty-five patients died, most of them due to infection early after transplantation (before 100 days). In conclusion, haplo-PTCy is a feasible procedure, can cure two-thirds of children with PID, and can be used as rescue treatment for previous graft failure. © 2020 American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc.
['Fernandes|Juliana Folloni|JF|', 'Nichele|Samantha|S|', 'Arcuri|Leonardo Javier|LJ|', 'Ribeiro|Lisandro|L|', 'Zamperlini-Netto|Gabriele|G|', 'Loth|Gisele|G|', 'Rodrigues|Ana Luiza Melo|ALM|', 'Kuwahara|Cilmara|C|', 'Koliski|Adriana|A|', 'Trennepohl|Joanna|J|', 'Garcia|Julia Lopes|JL|', 'Daudt|Liane Esteves|LE|', 'Seber|Adriana|A|', 'Gomes|Alessandra Araujo|AA|', 'Fasth|Anders|A|', 'Pasquini|Ricardo|R|', 'Hamerschlak|Nelson|N|', 'Rocha|Vanderson|V|', 'Bonfim|Carmem|C|']
D002648:Child D003520:Cyclophosphamide D005260:Female D006086:Graft vs Host Disease D018380:Hematopoietic Stem Cell Transplantation D006801:Humans D007223:Infant D008297:Male D000081207:Primary Immunodeficiency Diseases D019172:Transplantation Conditioning
Outcomes Post-transplantation cyclophosphamide Primary immunodeficiency disease Haploidentical transplantation