Transplantation in low resource countries

Thalassemia major (TM) is the most common deadly genetic disorder, 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-effective 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 supported a feasibility study evaluating safety, efficacy and costs of developing a new BMT center in an underserved lower-middle-income country with relatively untrained professionals within a structured collaboration 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 sibling donor, were younger than 10 years of age at the time of transplantation, received the same conditioning regimen. All needy families could rely on a support program throughout the 8-month post-transplant 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 outcome 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 safely, effectively, and affordably even in start-up centers in lower-middleincome countries, like Pakistan, were most thalassemia patients live. This observation may have important implications to increase access to cure for thalassemia worldwide. 重型地中海贫血（TM）是最常见的也是最致命的基因错乱，在许多中低收入地区，基因错乱是造成非传染性慢性疾病和经济负担的重大原因。 在这种情况下，许多儿童因接受不到长期支持治疗而活不到成年。 骨髓移植（BMT）是目前唯一能治愈该疾病的方法。 若病患儿童有低风险的特点并且具有匹配的捐献物， BMT 将是治疗幼童成功且低成本的疗法。 然而，在重型地中海贫血多发国家，严重缺乏骨髓移植中心。 第二治愈儿童基金会与来自结构化合作和知识转换计划的未受训专业人员共同资助了一个可行性研究，评估在服务不到位的中低收入国家发展一个BMT中心的安全性、疗效和成本。 本预期分析收录了2008年9月到2010年10月间先后在巴基斯坦接受BMT的24名病人。 其中，17名病人在成立的骨髓移植中心位于巴基斯坦卡拉奇市国家血液病研究所,完成手术；最先的7名在市立政府医院位于巴基斯坦医学研究院伊斯兰堡儿童医院,完成手术。 分析中，对比了病人的年龄、营养状态、发育、疾病、疾病状态和MBT后的随访时间。 所有病人都有一名相匹配的同胞捐献者。 移植手术时，他们的年龄都在10岁以下，并都接受了相同的预处理方案。 所有困难家庭在骨髓移植手术后的8个月期间都可以得到资助计划的帮助。 第二治愈儿童基金会提供了专业上和经济上的支持以及结构化网络数据管理和合作平台。 在进行平均19.6个月的随访中（(8.7月-31.5月），在已成立的和新运作的卫生中心，地中海贫血症救活率保险估计为85.6% 和85.7%，总生存率为94.1%和85.7%。 在统计上无明显区别。 其他结果指标，如传染性并发症、植入参数、移植相关并发症和BMT后表现分数等，都没有区别。 新运作卫生中心匹配相关移植手术的平均成本为11,513 USD （7,518至 21,176），包括BMT前评估费。 在结构化合作策略下，重型地中海贫血（TM）患者骨髓移植手术可以在诸如巴基斯坦等中低收入国家的新运作卫生中心进行，安全、有效，而且价格合理。 通过这种手术，大多数病人都可以存活下来。 该观察对提高全球地中海贫血患者接受治疗具有重要意义。