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为中等收入经济体的儿童建立并维持血液和骨髓移植服务:一份代表欧洲血液与骨髓移植协作组儿科疾病工作组撰写的经验驱动型立场文件

Setting up and sustaining blood and marrow transplant services for children in middle-income economies: an experience-driven position paper on behalf of the EBMT PDWP.

作者信息

Faulkner Lawrence, Verna Marta, Rovelli Attilio, Agarwal Rajat Kumar, Dhanya Rakesh, Parmar Lalith, Sedai Amit, Kumari Ankita, Ramprakash Stalin, Raghuram C P, Mehta Pallavi, Elizabeth Sandeep, Khalid Sadaf, Batool Aliya, Ghilani Sarah Khan, Fatima Itrat, Zara Tatheer, Marwah Priya, Soni Rajpreet, Trivedi Deepa, Conter Valentino, Canesi Marta, Othman Dosti, Faeq Vian, Kleinschmidt Katharina, Yesillipek Akif, Lam Catherine G, Howard Scott C, Corbacioglu Selim

机构信息

Cure2Children Foundation, Florence, Italy.

Sankalp India Foundation, Bengaluru, India.

出版信息

Bone Marrow Transplant. 2021 Mar;56(3):536-543. doi: 10.1038/s41409-020-0983-5. Epub 2020 Sep 7.

Abstract

Severe blood disorders and cancer are the leading cause of death and disability from noncommunicable diseases in the global pediatric population and a major financial burden. The most frequent of these conditions, namely sickle cell disease and severe thalassemia, are highly curable by blood or bone marrow transplantation (BMT) which can restore a normal health-related quality of life and be cost-effective. This position paper summarizes critical issues in extending global access to BMT based on ground experience in the start-up of several BMT units in middle-income countries (MICs) across South-East Asia and the Middle East where close to 700 allogeneic BMTs have been performed over a 10-year period. Basic requirements in terms of support systems, equipment, and consumables are summarized keeping in mind WHO's model essential lists and recommendations. BMT unit setup and maintenance costs are summarized as well as those per transplant. Low-risk BMT is feasible and safe in MICs with outcomes comparable to high-income countries but at a fraction of the cost. This report might be of assistance to health care institutions in MICs interested in developing hematopoietic stem cell transplantation services and strengthening context appropriate tertiary care and higher medical education.

摘要

严重血液疾病和癌症是全球儿童非传染性疾病导致死亡和残疾的主要原因,也是一项重大经济负担。其中最常见的病症,即镰状细胞病和重型地中海贫血,可通过血液或骨髓移植(BMT)得到有效治疗,这种治疗方法能够恢复与健康相关的正常生活质量,且具有成本效益。本立场文件基于东南亚和中东几个中等收入国家(MICs)启动多个BMT单位的实际经验,总结了扩大全球BMT可及性的关键问题,在过去十年中,这些国家已进行了近700例异基因BMT。在考虑世界卫生组织的基本药物清单和建议的基础上,总结了支持系统、设备和耗材方面的基本要求。总结了BMT单位的设置和维护成本以及每次移植的成本。低风险BMT在中等收入国家是可行且安全的,其结果与高收入国家相当,但成本仅为其一小部分。本报告可能有助于中等收入国家有兴趣开展造血干细胞移植服务、加强适合当地情况的三级医疗服务和高等医学教育的医疗机构。

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