组织针对罕见血液疾病的国家应对措施：意大利儿童镰状细胞病的经验。
Organizing national responses for rare blood disorders: the Italian experience with sickle cell disease in childhood.
作者信息
Colombatti Raffaella, Perrotta Silverio, Samperi Piera, Casale Maddalena, Masera Nicoletta, Palazzi Giovanni, Sainati Laura, Russo Giovanna
出版信息
Orphanet J Rare Dis. 2013 Oct 20;8:169. doi: 10.1186/1750-1172-8-169.
BACKGROUND
Sickle cell disease (SCD) is the most frequent hemoglobinopathy worldwide but remains a rare blood disorder in most western countries. Recommendations for standard of care have been produced in the United States, the United Kingdom and France, where this disease is relatively frequent because of earlier immigration from Africa. These recommendations have changed the clinical course of SCD but can be difficult to apply in other contexts. The Italian Association of Pediatric Hematology Oncology (AIEOP) decided to develop a common national response to the rising number of SCD patients in Italy with the following objectives: 1) to create a national working group focused on pediatric SCD, and 2) to develop tailored guidelines for the management of SCD that could be accessed and practiced by those involved in the care of children with SCD in Italy.
METHODS
Guidelines, adapted to the Italian social context and health system, were developed by 22 pediatric hematologists representing 54 AIEOP centers across Italy. The group met five times for a total of 128 hours in 22 months; documents and opinions were circulated via web.
RESULTS
Recommendations regarding the prevention and treatment of the most relevant complications of SCD in childhood adapted to the Italian context and health system were produced.
CONCLUSIONS
Creating a network of physicians involved in the day-to-day care of children with SCD is feasible in a country where it remains rare. Providing hematologists, primary and secondary care physicians, and caregivers across the country with web-based guidelines for the management of SCD tailored to the Italian context is the first step in building a sustainable response to a rare but emerging childhood blood disorder and in implementing the World Health Organization's suggestion "to design (and) implement … comprehensive national integrated programs for the prevention and management of SCD".
背景
镰状细胞病（SCD）是全球最常见的血红蛋白病，但在大多数西方国家仍然是一种罕见的血液疾病。在美国、英国和法国，由于早期来自非洲的移民，这种疾病相对较为常见，已经制定了相关的护理标准建议。这些建议改变了SCD的临床病程，但在其他情况下可能难以应用。意大利儿科血液肿瘤学会（AIEOP）决定针对意大利SCD患者数量的增加制定一项全国性的共同应对措施，目标如下：1）成立一个专注于儿童SCD的全国工作组；2）制定针对SCD管理的量身定制的指南，供意大利参与儿童SCD护理的人员参考和实施。
方法
由代表意大利54个AIEOP中心的22名儿科血液学家制定了适用于意大利社会背景和卫生系统的指南。该小组在22个月内共会面5次，总计128小时；文件和意见通过网络进行传阅。
结果
制定了针对意大利背景和卫生系统的、关于儿童期SCD最相关并发症预防和治疗的建议。
结论
在一个SCD仍然罕见的国家，建立一个参与SCD患儿日常护理的医生网络是可行的。为全国的血液学家、初级和二级护理医生以及护理人员提供针对意大利情况量身定制的基于网络的SCD管理指南，是对一种罕见但正在出现的儿童血液疾病建立可持续应对措施以及实施世界卫生组织“设计（并）实施……全面的国家综合SCD预防和管理计划”建议的第一步。
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