Amid Ali, Saliba Antoine N, Taher Ali T, Klaassen Robert J
Division of Hematology/Oncology, Department of Pediatrics, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.
Division of Hematology/Oncology, Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon.
Arch Dis Child. 2015 Nov;100(11):1051-7. doi: 10.1136/archdischild-2014-308112. Epub 2015 Aug 19.
Over the past few decades, there has been a remarkable improvement in the survival of patients with thalassaemia in developed countries. Availability of safe blood transfusions, effective and accessible iron chelating medications, the introduction of new and non-invasive methods of tissue iron assessment and other advances in multidisciplinary care of thalassaemia patients have all contributed to better outcomes. This, however, may not be true for patients who are born in countries where the resources are limited. Unfortunately, transfusion-transmitted infections are still major concerns in these countries where paradoxically thalassaemia is most common. Moreover, oral iron chelators and MRI for monitoring of iron status may not be widely accessible or affordable, which may result in poor compliance and suboptimal iron chelation. All of these limitations will lead to reduced survival and increased thalassaemia-related complications and subsequently will affect the patient's quality of life. In countries with limited resources, together with improvement of clinical care, strategies to control the disease burden, such as public education, screening programmes and appropriate counselling, should be put in place. Much can be done to improve the situation by developing partnerships between developed countries and those with limited resources. Future research should also particularly focus on patient's quality of life as an important outcome of care.
在过去几十年里，发达国家地中海贫血患者的生存率有了显著提高。安全输血的可及性、有效且可获得的铁螯合药物、新的非侵入性组织铁评估方法的引入以及地中海贫血患者多学科护理的其他进展，都促成了更好的治疗结果。然而，对于出生在资源有限国家的患者来说，情况可能并非如此。不幸的是，在这些地中海贫血最为常见的国家，输血传播感染仍是主要问题。此外，口服铁螯合剂和用于监测铁状态的磁共振成像可能无法广泛获得或负担得起，这可能导致依从性差和铁螯合效果不理想。所有这些限制都将导致生存率降低和地中海贫血相关并发症增加，进而影响患者的生活质量。在资源有限的国家，除了改善临床护理外，还应制定控制疾病负担的策略，如公众教育、筛查项目和适当的咨询。通过发展发达国家与资源有限国家之间的伙伴关系，可以做很多事情来改善这种情况。未来的研究还应特别关注患者的生活质量，将其作为护理的一项重要成果。
