Abstract

Hematopoietic cell transplantation is the only curative therapy available for patients with the common and morbid inherited hemoglobin disorders, -thalassemia major and sickle cell anemia. As will be discussed, a number of complex independent factors need to be carefully weighed in making a decision as to whether the patient is an appropriate candidate for hematopoietic cell transplantation (HCT). In this review, we will consider patients with -thalassemia major and -thalassemia intermedia, along with those with sickle cell anemia, and discuss options and limitations of current and future approaches to treat these hemoglobinopathies.

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