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

Fingerprint

Thalassemia
treosulfan
beta-Thalassemia
Stem cells
Toxicity
Transplantation
Hematopoietic Stem Cell Transplantation
Grafts
Allografts
Incidence
Tissue Donors
Morbidity
Transplants
Mortality
Therapeutics

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)

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

The role of reduced intensity preparative regimens in patients with thalassemia given hematopoietic transplantation. / Bertaina, Alice; Bernardo, Maria Ester; Mastronuzzi, Angela; Nasa, Giorgio L.; Locatelli, Franco.

Annals of the New York Academy of Sciences. Vol. 1202 2010. p. 141-148 (Annals of the New York Academy of Sciences; Vol. 1202).

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

Bertaina, A, Bernardo, ME, Mastronuzzi, A, Nasa, GL & 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, Annals of the New York Academy of Sciences, vol. 1202, pp. 141-148. https://doi.org/10.1111/j.1749-6632.2010.05590.x
Bertaina A, Bernardo ME, Mastronuzzi A, Nasa GL, Locatelli F. 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. 2010. p. 141-148. (Annals of the New York Academy of Sciences). https://doi.org/10.1111/j.1749-6632.2010.05590.x
Bertaina, Alice ; Bernardo, Maria Ester ; Mastronuzzi, Angela ; Nasa, Giorgio L. ; Locatelli, Franco. / The role of reduced intensity preparative regimens in patients with thalassemia given hematopoietic transplantation. Annals of the New York Academy of Sciences. Vol. 1202 2010. pp. 141-148 (Annals of the New York Academy of Sciences).
@inproceedings{a3222d41a6374901a896567fdb0bb779,
title = "The role of reduced intensity preparative regimens in patients with thalassemia given hematopoietic transplantation",
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.",
keywords = "allogeneic hematopoietic stem cell transplantation, cord blood, reduced-intensity preparative regimens, thalassemia, unrelated donors",
author = "Alice Bertaina and Bernardo, {Maria Ester} and Angela Mastronuzzi and Nasa, {Giorgio L.} and Franco Locatelli",
year = "2010",
month = "8",
doi = "10.1111/j.1749-6632.2010.05590.x",
language = "English",
isbn = "9781573317825",
volume = "1202",
series = "Annals of the New York Academy of Sciences",
pages = "141--148",
booktitle = "Annals of the New York Academy of Sciences",

}

TY - GEN

T1 - The role of reduced intensity preparative regimens in patients with thalassemia given hematopoietic transplantation

AU - Bertaina, Alice

AU - Bernardo, Maria Ester

AU - Mastronuzzi, Angela

AU - Nasa, Giorgio L.

AU - Locatelli, Franco

PY - 2010/8

Y1 - 2010/8

N2 - 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.

AB - 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.

KW - allogeneic hematopoietic stem cell transplantation

KW - cord blood

KW - reduced-intensity preparative regimens

KW - thalassemia

KW - unrelated donors

UR - http://www.scopus.com/inward/record.url?scp=77955859649&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=77955859649&partnerID=8YFLogxK

U2 - 10.1111/j.1749-6632.2010.05590.x

DO - 10.1111/j.1749-6632.2010.05590.x

M3 - Conference contribution

C2 - 20712785

AN - SCOPUS:77955859649

SN - 9781573317825

VL - 1202

T3 - Annals of the New York Academy of Sciences

SP - 141

EP - 148

BT - Annals of the New York Academy of Sciences

ER -