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 Prockop; Jaap Jan Boelens; Alice Bertaina

doi:10.1016/j.jcyt.2022.12.005

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

Cytotherapy. 2023 May;25(5):463-471. doi: 10.1016/j.jcyt.2022.12.005. Epub 2023 Jan 27.

Authors

Affiliations

¹ Division of Hematology, Oncology and Stem Cell Transplant and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Palo Alto, California, USA. Electronic address: [email protected].
² Pediatric Blood and Marrow Transplantation Division and Pele Pequeno Principe Research Institute, Hospital Pequeno Principe, Curitiba, Brazil.
³ Center for Cancer and Immunology Research, Cell Enhancement and Technologies for Immunotherapy, Children's National Hospital, Washington, DC, USA.
⁴ Istituto di Ricovero e Cura a Carattere Scientifico Ospedale San Raffaele, Milan, Italy.
⁵ Department of Hematology, Fiona Stanley Hospital, Perth, Australia.
⁶ Université de Montréal and Maisonneuve Rosemont Hospital, Montréal, Canada.
⁷ Faculty of Biology, Medicine and Health, University of Manchester, Manchester, UK.
⁸ Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
⁹ Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
¹⁰ Gastroenterology Unit, Department of Medicine, Azienda Ospedaliera Universitaria Integrata Policlinico G.B. Rossi and University of Verona, Verona, Italy.
¹¹ Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Massachusetts, USA.
¹² Stem Cell Transplantation and Cellular Therapies, Memorial Sloan Kettering Cancer Center, New York, New York, USA; Department of Pediatrics, Weill Cornell Medical College of Cornell University, New York, New York, USA.
¹³ Division of Hematology, Oncology and Stem Cell Transplant and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Palo Alto, California, USA.

PMID: 36710227
DOI: 10.1016/j.jcyt.2022.12.005

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.

Keywords: Bone marrow failure disorders; Hemoglobinopathies; Metabolic disorders; Primary immune deficiency disorders; Primary immune regulatory disorders; Severe aplastic anemia.

MeSH terms

Graft vs Host Disease* / etiology
Hematopoietic Stem Cell Transplantation* / adverse effects
Humans
Tissue Donors
Transplantation Conditioning
Transplantation, Homologous