Salvage HLA-haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide for graft failure in non-malignant disorders

Albert, Michael H.; Sirin, Mehtap; Hoenig, Manfred; Hauck, Fabian; Schuetz, Catharina; Bhattacharyya, Rajat; Stepensky, Polina; Jacoby, Elad; Gungor, Tayfun; Beier, Rita and Schulz, Ansgar (2021): Salvage HLA-haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide for graft failure in non-malignant disorders. In: Bone Marrow Transplantation, Vol. 56, No. 9: pp. 2248-2258

Abstract

Graft failure requires urgent salvage HSCT, but there is no universally accepted approach for this situation. We investigated T-cell replete haploidentical HSCT with post-transplantation cyclophosphamide following serotherapy-based, radiation-free, reduced intensity conditioning in children with non-malignant disorders who had rejected their primary graft. Twelve patients with primary or secondary graft failure received T-cell replete bone marrow grafts from haploidentical donors and post-transplantation cyclophosphamide. The recommended conditioning regimen comprised rituximab 375 mg/m(2), alemtuzumab 0.4 mg/kg, fludarabine 150 mg/m(2), treosulfan 20-24 g/m(2) and cyclophosphamide 29 mg/kg. After a median follow-up of 26 months (7-95), eleven of twelve patients (92%) are alive and well with complete donor chimerism in ten. Neutrophil and platelet engraftment were observed in all patients after a median of 18 days (15-61) and 39 days (15-191), respectively. Acute GVHD grade I was observed in 1/12 patients (8%) and mild chronic GVHD in 1/12 patients (8%). Viral reactivations and disease were frequent complications at 75% and 42%, respectively, but no death from infectious causes occurred. In summary, this retrospective analysis demonstrates that a post-transplantation cyclophosphamide-based HLA-haploidentical salvage HSCT after irradiation-free conditioning results in excellent engraftment and overall survival in children with non-malignant diseases.

• Item Type: Journal article
• Faculties: Medicine
• Subjects: 600 Technology > 610 Medicine and health
• ISSN: 0268-3369
• Language: English