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Detection of Residual Donor Erythroid Progenitor Cells after Hematopoietic Stem Cell Transplantation for Patients with Hemoglobinopathies
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Hematopoietic Stem Cell Transplantation in Thalassemia

Mattia Algeri1, Mariachiara Lodi1, Franco Locatelli2

  • 1Department of Hematology/Oncology, Cell and Gene Therapy - IRCCS, Bambino Gesù Children's Hospital, Rome, Italy.

Hematology/Oncology Clinics of North America
|March 12, 2023
PubMed

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only consolidated, potentially curative treatment for patients with transfusion-dependent thalassemia major. In the past few decades, several new approaches have reduced the toxicity of conditioning regimens and decreased the incidence of graft-versus-host disease, improving patients' outcomes and quality of life. In addition, the progressive availability of alternative stem cell sources from unrelated or haploidentical donors or umbilical cord blood has made HSCT a feasible option for an increasing number of subjects lacking an human leukocyte antigen (HLA)-identical sibling. This review provides an overview of allogeneic hematopoietic stem cell transplantation in thalassemia, reassesses current clinical results, and discusses future perspectives.

Keywords:
Cord blood transplantationHaploidentical transplantationHematopoietic stem cell transplantationHemoglobinopathiesSibling donor transplantationThalassemiaUnrelated donor transplantation

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