Sistema FMUSP-HC: Faculdade de Medicina da Universidade de São Paulo (FMUSP) e Hospital das Clínicas da FMUSPFERNANDES, Juliana FolloniBONFIM, CarmemKERBAUY, Fabio RodriguesRODRIGUES, MorganiESTEVES, IracemaSILVA, Nathalia HalleyAZAMBUJA, Alessandra PrandiniMANTOVANI, Luiz FernandoKUTNER, Jose MauroLOTH, GiseleKUWAHARA, Cilmara CristinaBUENO, ClarissaKONDO, Andrea TiemiRIBEIRO, Andreza Alice FeitosaKOK, FernandoHAMERSCHLAK, Nelson2018-07-052018-07-052018BONE MARROW TRANSPLANTATION, v.53, n.4, p.392-399, 20180268-3369https://observatorio.fm.usp.br/handle/OPI/26976Allogeneic hematopoietic stem cell transplantation (HSCT) is the only treatment that enhances survival and stabilizes neurologic symptoms in X-linked adrenoleukodystrophy (X-ALD) with cerebral involvement, a severe demyelinating disease of childhood. Patients with X-ALD who lack a well-matched HLA donor need a rapid alternative. Haploidentical HSCT using post transplant cyclophosphamide (PT/Cy) has been performed in patients with malignant and nonmalignant diseases showing similar outcomes compared to other alternative sources. We describe the outcomes of transplants performed for nine X-ALD patients using haploidentical donors and PT/Cy. Patients received conditioning regimen with fludarabine 150 mg/m(2) , cyclophosphamide 29 mg/kg and 2 Gy total body irradiation (TBI) with or without antithymocyte globulin. Graft-vs.-host disease prophylaxis consisted of cyclophosphamide 50 mg/kg/day on days +3 and +4, tacrolimus or cyclosporine A and mycophenolate mofetil. One patient had a primary graft failure and was not eligible for a second transplant. Three patients had secondary graft failure and were successfully rescued with second haploidentical transplants. Trying to improve engraftment, conditioning regimen was changed, substituting 2 Gy TBI for 4 Gy total lymphoid irradiation. Eight patients are alive and engrafted (17-37 months after transplant). Haploidentical HSCT with PT/Cy is a feasible alternative for X-ALD patients lacking a suitable matched donor. Graft failure has to be addressed in further studies.engrestrictedAccesshematopoietic-cell transplantationversus-host-diseaseposttransplantation cyclophosphamideaplastic-anemiairradiationengraftmentoutcomestherapybmtarticleCopyright NATURE PUBLISHING GROUP10.1038/s41409-017-0015-2BiophysicsOncologyHematologyImmunologyTransplantation1476-5365