title: Alternative donor allogeneic hematopoietic cell transplantation for hemoglobinopathies.
abstract: Allogeneic hematopoietic stem cell transplantation (HSCT) offers a curative therapy for patients with hemoglobinopathies, mainly severe sickle cell disease (SCD) and thalassemia (TM). However, the applicability of HSCT has been limited mainly by donor availability, with a less than 25%-30% of eligible patients having human leukocyte antigen (HLA)-matched sibling donors. Previous outcomes using alternate donor options have been markedly inferior due to increased regimen-related toxicity, transplant-related mortality, graft failure, and graft-versus-host disease (GVHD). Advances in transplant technology, including high-resolution HLA typing, improved GVHD prophylactic approaches with tolerance induction, and better supportive care over the last decade, are addressing these historical challenges, resulting in increasing donor options. Herein, we review alternate donor HSCT approaches for severe SCD and TM using unrelated donors, umbilical cord blood units, or related haploidentical donors. Though this is an emerging field, early results are promising and in selected patients, this may be the preferred option to mitigate against the age-related morbidity and early mortality associated with these disorders.
authors.affiliation: Adult Hematology and Hematopoietic Stem Cell Transplantation, King Faisal Hospital and Research Centre, Riyadh, Saudi Arabia. Electronic address: Falfraih@kfshrc.edu.sa.
authors.forename: Feras
authors.initials: F
authors.lastname: Alfraih
authors.affiliation: Molecular and Clinical Hematology Branch, NHLBI, NIH, Bethesda, MD, USA.
authors.forename: Mahmoud
authors.initials: M
authors.lastname: Aljurf
authors.affiliation: Division of Hematology and Oncology, Department of Medicine and Vanderbilt- Meharry Center for Excellence in Sickle Cell Disease, Vanderbilt University Medical Center, Nashville, TN, USA.
authors.forename: Courtney D
authors.initials: CD
authors.lastname: Fitzhugh
authors.affiliation: Division of Hematology and Oncology, Department of Medicine and Vanderbilt- Meharry Center for Excellence in Sickle Cell Disease, Vanderbilt University Medical Center, Nashville, TN, USA.
authors.forename: Adetola A
authors.initials: AA
authors.lastname: Kassim
mesh_terms: D006086:Graft vs Host Disease / Q000209:etiology; D006238:Haploidy; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006453:Hemoglobinopathies / Q000628:therapy*; D006650:Histocompatibility Testing; D006801:Humans; D014184:Transplantation, Homologous
keywords: Allogeneic hematopoietic stem cell transplantation; Alternative donor; Hemoglobinopathies; Sickle cell disease; Thalassemia