Hokland Peter, Daar Shahina, Khair Wael, Sheth Sujit, Taher Ali T, Torti Lorenza, Hantaweepant Chattree, Rund Deborah
Faculty of Health, Department of Clinical Medicine, Aarhus University, Aarhus, Denmark.
College of Medicine & Health Sciences, Sultan Qaboos University, Muscat, Oman.
Br J Haematol. 2023 Apr;201(2):199-214. doi: 10.1111/bjh.18671. Epub 2023 Feb 17.
The thalassaemias are a group of genetic disorders of haemoglobin which are endemic in the tropics but are now found worldwide due to migration. Basic standard of care therapy includes regular transfusions to maintain a haemoglobin level of around 10 g/dL, together with iron chelation therapy to prevent iron overload. Novel therapies, bone marrow transplantation, and gene therapy are treatment options that are unavailable in many countries with stressed economies. This Wider Perspectives article presents the strategies for management of an adolescent refugee patient with beta thalassaemia, as it would be performed by expert haematologists in six countries: Italy, Lebanon, Oman, the Sudan, Thailand and the United States. The experienced clinicians in each country have adapted their practice according to the resources available, which vary greatly. Even in the current modern era, providing adequate transfusions and chelation is problematic in many countries. On the other hand, ensuring adherence to therapy, particularly during adolescence, is a similar challenge seen in all countries. The concluding section highlights the disparities in available therapies and puts the role of novel therapies into a societal context.
地中海贫血是一组血红蛋白的遗传性疾病,在热带地区呈地方性流行,但由于人口迁移,现在在世界各地都有发现。基本的标准治疗方法包括定期输血以维持血红蛋白水平在10克/分升左右,同时进行铁螯合治疗以防止铁过载。新型疗法、骨髓移植和基因疗法是许多经济紧张的国家无法采用的治疗选择。这篇“更广阔视角”文章介绍了由意大利、黎巴嫩、阿曼、苏丹、泰国和美国六个国家的血液学专家对一名青少年β地中海贫血难民患者的管理策略。每个国家经验丰富的临床医生都根据各自国家差异很大的现有资源调整了他们的治疗方法。即使在当今现代社会,许多国家在提供充足的输血和螯合治疗方面仍存在问题。另一方面,确保患者坚持治疗,尤其是在青少年时期,是所有国家都面临的类似挑战。结论部分强调了现有治疗方法的差异,并将新型疗法的作用置于社会背景中。