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International Cooperation for the Cure and Prevention of Severe Hemoglobinopathies

Faulkner, Lawrence B. MD; Uderzo, Cornelio MD; Masera, Giuseppe MD

Journal of Pediatric Hematology/Oncology: August 2013 - Volume 35 - Issue 6 - p 419–423
doi: 10.1097/MPH.0b013e31829cd920
Invited Review Article

Thalassemia major (TM) is the most frequent life-threatening noninfectious disease of childhood in the Middle East, South Asia, and Pacific Islands where it accounts for a significant proportion of childhood mortality, morbidity, and related health care expenses. In spite of major advances in supportive care during the last decade, many patients in low-income and middle-income countries still fare poorly because of high treatment costs and lack of accessible multidisciplinary teams, not to consider the risk of blood-borne infections, primarily hepatitis C. In selected low-risk patients with a compatible sibling, TM is highly curable by bone marrow transplantation (BMT), which also improves the quality of life and is cost-effective. Starting in 2008, the Cure2Children Foundation (C2C), an Italian Non-Governmental Organization, has supported a BMT network in Pakistan, which during 2012 was extended to India. The primary aim of this project was to assess feasibility, outcomes, and costs of matched-related BMT for thalassemia in young low-risk children using a well-established and tolerable strategy. A total of 100 matched-related BMTs have been performed to date by partner institutions within this C2C-supported network; in the 50 low-risk cases with TM, over 90% disease-free survival was obtained with procedure expenses within 10,000 USD/BMT, that is, an outcome comparable to that obtained in affluent countries but with a fraction of the expenses. This cure rate was also obtained in start-up BMT centers (1 in Pakistan and 1 in India) within a structured and intensive cooperation program. Twinning and other international cooperation strategies based on shared principles and a common vision may substantially facilitate access to BMT.

*The Cure2Children Foundation, Florence

Department of Pediatrics, University of Milano-Bicocca, San Gerardo Hospital, Monza, Italy

The authors declare no conflict of interest.

Reprints: Lawrence B. Faulkner, MD, The Cure2Children Foundation, Via Marconi 30, Florence 50131, Italy (e-mail:

Received April 26, 2013

Accepted May 1, 2013

Copyright © 2013 Wolters Kluwer Health, Inc. All rights reserved.