Unrelated donor stem cell transplantation for transfusion-dependent thalassemia

Shalini Shenoy*, Alexis A Thompson

*Corresponding author for this work

Research output: Contribution to journalArticle

4 Citations (Scopus)

Abstract

Thalassemia major is characterized by severe anemia dependent on red cell transfusions from infancy. Conservative management requires a safe source of compatible blood throughout life, strategies to combat iron overload, monitoring and treatment of transfusion-related complications, and management of cardiac and/or hepatic dysfunction from iron accumulation. Complications can result in premature morbidity and mortality. Stem cell transplantation is curative, but outcomes depend on the availability of a histocompatible donor, recipient age, and disease-related complications. Successful transplantation requires stable donor engraftment and donor-derived erythropoiesis and a low incidence of graft-versus-host disease, organ toxicities, and mortality. This translates to a cure with good quality of life and life span. Since recipients are at a high risk for graft rejection (prior transfusions, immunocompetency), myeloablative transplants have been the norm. Recent modifications to standard preparative regimens have significantly reduced transplant toxicities, resulting in >80% disease-free survival in children. Aiming to further reduce regimen-related toxicities, such as veno-occlusive liver disease and sterility, a recent trial explored reduced-intensity conditioning in unrelated donor (URD) transplants utilizing marrow or umbilical cord blood in patients without suitable familial donors. This report summarizes advances in URD transplantation for thalassemia, focusing on conditioning regimen nuances.

Original languageEnglish (US)
Pages (from-to)122-126
Number of pages5
JournalAnnals of the New York Academy of Sciences
Volume1368
Issue number1
DOIs
StatePublished - Mar 1 2016

Fingerprint

Unrelated Donors
Thalassemia
Stem Cell Transplantation
Stem cells
Transplants
Tissue Donors
Toxicity
Grafts
Blood
Iron
Transplantation
Premature Mortality
Iron Overload
Erythropoiesis
beta-Thalassemia
Graft Rejection
Graft vs Host Disease
Fetal Blood
Liver
Infertility

Keywords

  • Bone marrow
  • Stem cell transplantation
  • Thalassemia
  • Umbilical cord blood
  • Unrelated donors

ASJC Scopus subject areas

  • Neuroscience(all)
  • Biochemistry, Genetics and Molecular Biology(all)
  • History and Philosophy of Science

Cite this

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title = "Unrelated donor stem cell transplantation for transfusion-dependent thalassemia",
abstract = "Thalassemia major is characterized by severe anemia dependent on red cell transfusions from infancy. Conservative management requires a safe source of compatible blood throughout life, strategies to combat iron overload, monitoring and treatment of transfusion-related complications, and management of cardiac and/or hepatic dysfunction from iron accumulation. Complications can result in premature morbidity and mortality. Stem cell transplantation is curative, but outcomes depend on the availability of a histocompatible donor, recipient age, and disease-related complications. Successful transplantation requires stable donor engraftment and donor-derived erythropoiesis and a low incidence of graft-versus-host disease, organ toxicities, and mortality. This translates to a cure with good quality of life and life span. Since recipients are at a high risk for graft rejection (prior transfusions, immunocompetency), myeloablative transplants have been the norm. Recent modifications to standard preparative regimens have significantly reduced transplant toxicities, resulting in >80{\%} disease-free survival in children. Aiming to further reduce regimen-related toxicities, such as veno-occlusive liver disease and sterility, a recent trial explored reduced-intensity conditioning in unrelated donor (URD) transplants utilizing marrow or umbilical cord blood in patients without suitable familial donors. This report summarizes advances in URD transplantation for thalassemia, focusing on conditioning regimen nuances.",
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Unrelated donor stem cell transplantation for transfusion-dependent thalassemia. / Shenoy, Shalini; Thompson, Alexis A.

In: Annals of the New York Academy of Sciences, Vol. 1368, No. 1, 01.03.2016, p. 122-126.

Research output: Contribution to journalArticle

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AU - Shenoy, Shalini

AU - Thompson, Alexis A

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AB - Thalassemia major is characterized by severe anemia dependent on red cell transfusions from infancy. Conservative management requires a safe source of compatible blood throughout life, strategies to combat iron overload, monitoring and treatment of transfusion-related complications, and management of cardiac and/or hepatic dysfunction from iron accumulation. Complications can result in premature morbidity and mortality. Stem cell transplantation is curative, but outcomes depend on the availability of a histocompatible donor, recipient age, and disease-related complications. Successful transplantation requires stable donor engraftment and donor-derived erythropoiesis and a low incidence of graft-versus-host disease, organ toxicities, and mortality. This translates to a cure with good quality of life and life span. Since recipients are at a high risk for graft rejection (prior transfusions, immunocompetency), myeloablative transplants have been the norm. Recent modifications to standard preparative regimens have significantly reduced transplant toxicities, resulting in >80% disease-free survival in children. Aiming to further reduce regimen-related toxicities, such as veno-occlusive liver disease and sterility, a recent trial explored reduced-intensity conditioning in unrelated donor (URD) transplants utilizing marrow or umbilical cord blood in patients without suitable familial donors. This report summarizes advances in URD transplantation for thalassemia, focusing on conditioning regimen nuances.

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