The role of reduced intensity preparative regimens in patients with thalassemia given hematopoietic transplantation

Alice Bertaina, Maria Ester Bernardo, Angela Mastronuzzi, Giorgio L. Nasa, Franco Locatelli

Research output: Chapter in Book/Report/Conference proceedingConference contribution

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) still remains the only curative treatment for patients with thalassemia major (TM). However, HSCT is associated with a non-negligible risk of both transplantation-related mortality (TRM) and morbidity. Great interest and relevant expectations have been raised by the introduction in the clinical practice of reduced-intensity preparative regimens, which may represent an effective strategy to reduce the toxicity of transplantation and may also help reduce the incidence of late effects. Although some reports have documented the feasibility of using reduced-intensity preparative regimens for successfully treating patients with TM, a high incidence of graft failure has been frequently reported. Recently, treosulfan-based myeloablation has been demonstrated to be associated with limited extra-medullary toxicity and a high rate of sustained donor engraftment. This novel approach is a promising alternative for reducing the risk of life-threatening complications and increasing the number of TM patients successfully cured with an allograft.

Original languageEnglish
Title of host publicationAnnals of the New York Academy of Sciences
Pages141-148
Number of pages8
Volume1202
DOIs
Publication statusPublished - Aug 2010

Publication series

NameAnnals of the New York Academy of Sciences
Volume1202
ISSN (Print)00778923
ISSN (Electronic)17496632

Keywords

  • allogeneic hematopoietic stem cell transplantation
  • cord blood
  • reduced-intensity preparative regimens
  • thalassemia
  • unrelated donors

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)

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  • Cite this

    Bertaina, A., Bernardo, M. E., Mastronuzzi, A., Nasa, G. L., & Locatelli, F. (2010). The role of reduced intensity preparative regimens in patients with thalassemia given hematopoietic transplantation. In Annals of the New York Academy of Sciences (Vol. 1202, pp. 141-148). (Annals of the New York Academy of Sciences; Vol. 1202). https://doi.org/10.1111/j.1749-6632.2010.05590.x