Transplantation in low resource countries L. Faulkner, N. Yaqub, 2 S. Khalid, T. Zhara, S. Ansari, 1 T. Farzana, 1 T. Shamsi 1 Cure2Children Foundation, Florence, Italy, 1 Pakistan Institute of Medical Sciences Children’s Hospital, Islamabad, 2 National Institute of Blood Diseases, Karachi, Pakistan Abstract Thalassemia major (TM) is the most common deadly genetic disor- der, a major cause of chronic non-infectious morbidity and financial burden in many low and middle-income regions. In these settings few children reach adulthood because proper long-term supportive care is seldom available. Bone marrow transplantation (BMT) is the only available curative modality and it can be very successful and cost-effec- tive for young children with low-risk features and a compatible related donor. However, in countries where TM is most prevalent, there is a dire shortage of BMT centers. The Cure2Children Foundation has sup- ported a feasibility study evaluating safety, efficacy and costs of devel- oping a new BMT center in an underserved lower-middle-income coun- try with relatively untrained professionals within a structured collabo- ration and knowledge-transfer program. A total of 24 consecutive patients who underwent BMT in Pakistan between September 2008 and August 2010 are included in this prospective analysis, 17 from an established bone marrow transplant center, the National Institute for Blood Diseases in Karachi, Pakistan and the initial 7 BMTs from a start up unit in a government civil hospital, the Pakistan Institute of Medical Sciences Children’s Hospital in Islamabad. Patients were matched for age, nutritional status, growth, disease, disease status and post-BMT follow-up time. All patients had a matched-related sib- ling donor, were younger than 10 years of age at the time of transplan- tation, received the same conditioning regimen. All needy families could rely on a support program throughout the 8-month post-trans- plant period. The Cure2Children Foundation provided professional and financial support as well as a structured web-based data management and cooperation platform. At a median follow up of 19.6 months (range 8.7 to 31.5) actuarial thalassemia-free survival is 85.6% and 85.7% and overall survival 94.1% and 85.7% in the established and start-up center respectively with no statistically significant differences. Other out- come indices like infectious complications, engraftment parameters, transplant-related complications, and post-BMT performance scores also did not differ. The median cost of matched-related transplants in the start-up center, including pre-BMT evaluation, was 11,513 USD (range 7,518 to 21,176). Within structured cooperation strategies bone marrow transplantation for thalassemia major can be performed safe- ly, effectively, and affordably even in start-up centers in lower-middle- income countries, like Pakistan, were most thalassemia patients live. This observation may have important implications to increase access to cure for thalassemia worldwide. Introduction Aslam’s story Aslam is a 2 year and 3 months old boy with thalassemia major. His father is a mill worker and his mother a housewife, they live in interi- or Sindh, Pakistan. The father can read and write Sindhi (the local lan- guage). They need a 5-hour bus ride to the nearest thalassemia care center in Karachi were Aslam gets a transfusion once a month, the parents are aware they should go more often but it is very hard for them, the whole family income is less than 1,000 USD/year and a sub- stantial part of it goes for the care of Aslam. Nine children have died of thalassemia in their extended family. Aslam’s doctor explained in Sindhi that an international NGO was supporting access to cure of thalassemia by bone marrow transplanta- tion for needy families in Pakistan and presents this option to the par- ents because he has a 4 year-old healthy sister who could be the poten- tial donor, even if she had thalassemia minor. The parents know that it will be very hard for them to provide Aslam with the long-term care he will need to be able to survive, they know and trust their doctors and decide to go for a cure even if they are aware that there is chance he may die from transplantation. The NGO will also provide accommoda- tion and family support in Karachi. Aslam’s sister turns out to be compatible by HLA testing and three months later he is transplanted her bone marrow, he stays in hospital with his mom for a little over a month and then another 6 months with his family in the Karachi apartment provided by the NGO which also regularly sends a nice lady (herself cured by transplantation), to make sure they have the basic necessities and are able to keep the apart- ment clean. Almost 8 months later they go back to their village, Aslam has not needed transfusions for over 7 months, his parents are very happy and grateful for their new life even though the doctor explained that he might have problems having children when he grows up. Prenatal diagnosis, should the mother expect another baby, was also discussed. Correspondence: L. Faulkner, Cure2Children Foundation, via Marconi 30, 50131 Florence, Italy. Key words: transplantation developing countries. Acknowledgements: This work was possible thanks to the support of Fondazione Monte dei Paschi di Siena, Italy. ©Copyright L. Faulkner et al., 2011 Licensee PAGEPress, Italy Thalassemia Reports 2011; 1(s2):e9 doi:10.4081/thal.2011.s2.e9 This article is distributed under the terms of the Creative Commons Attribution Noncommercial License (by-nc 3.0) which permits any noncom- mercial use, distribution, and reproduction in any medium, provided the orig- inal author(s) and source are credited. Parts of this work were presented at the “12th International Conference on Thalassemia and Hemoglobinopathies”, Antalya (Turkey), 11-14 May 2011. [page 30] [Thalassemia Reports 2011; 1(s2):e9] Thalassemia Reports 2011; volume 1(s2):e9 Non-commercial use only