Emanuele Angelucci, Federica Pilo, Clara Targhetta, Martina Pettinau, Cristina Depau, Claudia Cogoni, Sara Usai, Mario Pani, Laura Dessì, Donatella Baronciani
  • Emanuele Angelucci
    Ospedale Oncologico di Riferimento Regionale “Armando Businco” Cagliari, Italy | emnang@tin.it
  • Federica Pilo
    Affiliation not present
  • Clara Targhetta
    Affiliation not present
  • Martina Pettinau
    Affiliation not present
  • Cristina Depau
    Affiliation not present
  • Claudia Cogoni
    Affiliation not present
  • Sara Usai
    Affiliation not present
  • Mario Pani
    2Servizio Immmunoematologia e Trasfusionale, azienda Ospedaliera “Brotzu”, Cagliari,
  • Laura Dessì
    Affiliation not present
  • Donatella Baronciani
    Affiliation not present


The   basis of allogeneic hemopoietic stem cell (HSC) transplantation in thalassemia consists in substituting the ineffective thalassemic erythropoiesis with and allogeneic effective one. This approach is an efficient way to obtain a long lasting, probably permanent, clinical effective correction of the anaemia avoiding transfusion requirement and subsequent complications like iron overload.   The first HSC transplant for thalassemia was performed in Seattle on Dec 2, 1981. In the early eighties transplantation procedure was limited to very few centres worldwide. Subsequently between 17 December 1981 and 31 January 2003, over  1000 consecutive patients, aged from 1 to 35 years, underwent transplantation in Pesaro. After the  pioneering work by the Seattle  and Peasaro groups,  this therapeutic approach is now widely applied worldwide.

Medical therapy of thalassemia is one of the most spectacular successes of the medical practice in the last decades. In recent years advances in knowledge of iron overload patho-physiopathology, improvement and diffusion of diagnostic capability together with the development of new effective and safe oral chelators  promise to further increase success of medical therapy. Nevertheless situation is dramatically different in non-industrialized countries were the very large majority of patients live today . Transplantation technologies have improved substantially during the last years and transplantation outcome is likely to be much better today than in the ‘80s. Recent data indicated a probability of overall survival and thalassemia free survival of 97% and 89% for patients with no advanced disease and of 87% and 80% for patients with advanced disease.  Thus the central role of HSC in thalassemia has now been fully established. Thalassemia remains the only definitive curative therapy for thalassemia and other hemoblobinopathies. The development of oral chelators has not changed this position. However this has not settled the controversy on how this curative but potentially lethal treatment stands in front of medical therapy for adults and advanced disease patients. In  sickle cell disease  HSC transplantation currently is reserved almost exclusively for patients with clinical features that indicate a poor outcome or significant sickle-related morbidity.


Anemia, Thalassemic syndromes, Hemopoietic Stem Cell Transplantation

Full Text:

Submitted: 2014-06-11 11:31:32
Search for citations in Google Scholar
Related articles: Google Scholar
Abstract views:


Article Metrics

Metrics Loading ...

Metrics powered by PLOS ALM

Copyright (c) 2016 Mediterranean Journal of Hematology and Infectious Diseases

Creative Commons License
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.
© PAGEPress 2008-2018     -     PAGEPress is a registered trademark property of PAGEPress srl, Italy.     -     VAT: IT02125780185     •     Privacy