Hematopoietic stem cell transplantation for hemoglobinopathies: current practice and emerging trends

Pediatr Clin North Am. 2010 Feb;57(1):181-205. doi: 10.1016/j.pcl.2010.01.003.


Despite improvements in the management of thalassemia major and sickle cell disease, treatment complications are frequent and life expectancy remains diminished for these patients. Hematopoietic stem cell transplantation (HSCT) is the only curative option currently available. Existing results for HSCT in patients with hemoglobinopathy are excellent and still improving. New conditioning regimens are being used to reduce treatment-related toxicity and new donor pools accessed to increase the number of patients who can undergo HSCT.

Publication types

  • Review

MeSH terms

  • Hematopoietic Stem Cell Transplantation / methods*
  • Hematopoietic Stem Cell Transplantation / trends*
  • Hemoglobinopathies / blood
  • Hemoglobinopathies / surgery*
  • Hemoglobins / biosynthesis*
  • Humans
  • Public Health*
  • Quality of Life


  • Hemoglobins