Transplant for non-malignant disorders: an International Society for Cell & Gene Therapy Stem Cell Engineering Committee report on the role of alternative donors, stem cell sources and graft engineering
Orly R. Klein, Carmem Bonfim, Allistair Abraham, Annalisa Ruggeri, Duncan Purtill, Sandra Cohen, Robert Wynn, Athena Russell, Akshay Sharma, Rachele Ciccocioppo, Susan E. Prockop, Jaap Jan Boelens, Alice Bertaina
Abstract
Hematopoietic stem cell transplantation (HSCT) is curative for many non-malignant disorders. As HSCT and supportive care technologies improve, this life-saving treatment may be offered to more and more patients. With the development of new preparative regimens, expanded alternative donor availability, and graft manipulation techniques, there are many options when choosing the best regimen for patients. Herein the authors review transplant considerations, transplant goals, conditioning regimens, donor choice, and graft manipulation strategies for patients with non-malignant disorders undergoing HSCT.