Casale Maddalena, Baldini Marina, Del Monte Patrizia, Gigante Antonia, Grandone Anna, Origa Raffaella, Poggi Maurizio, Gadda Franco, Lai Rosalba, Marchetti Monia, Forni Gian Luca
Ematologia e Oncologia Pediatrica, Dipartimento della Donna, del Bambino e di Chirurgia Generale e Specialistica, Università della Campania "Luigi Vanvitelli", Napoli.
Centro Malattie Rare, UOC Medicina Interna, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milano.
Recenti Prog Med. 2022 Sep;113(9):495-554. doi: 10.1701/3865.38516.
The treatment of endocrinopathies in haemoglobinopathies is a continually expanding research area; therefore, recommendations supporting the appropriateness of treatments are a pressing need for the medical community.
The Management Committee of SITE selected and gathered a multidisciplinary and multiprofessional team including experts in haemoglobinopathies and experts in endocrinopathies, who have been flanked by experts with methodological and organizational expertise, in order to formulate recommendations based on the available scientific evidence integrated by clinical experience. The project followed the systematic approach for the production of clinical practice guidelines according to the methodology suggested by the National Center for Clinical Excellence, Quality and Safety of Care (CNEC).
Out of 14 topics 100 clinical questions were addressed and 206 recommendations were elaborated. Strength of recommendations, panel agreement, general description of the topic, and interpretation of evidence were reported.
Good Practice Recommendations are the final outcome of translational research and allow to transfer the latest research knowledge to the daily clinical practice of endocrine complications in haemoglobinopathies.
血红蛋白病内分泌病变的治疗是一个不断扩展的研究领域;因此,为医学界提供支持治疗适宜性的建议迫在眉睫。
SITE管理委员会挑选并组建了一个多学科、多专业团队,其中包括血红蛋白病专家和内分泌病变专家,并辅以具备方法学和组织专业知识的专家,以便根据现有科学证据并结合临床经验制定建议。该项目遵循了国家临床卓越、质量与安全中心(CNEC)建议的方法学,采用系统方法制定临床实践指南。
针对14个主题提出了100个临床问题,并制定了206条建议。报告了建议的强度、专家小组的共识、主题的总体描述以及证据解读。
良好实践建议是转化研究的最终成果,有助于将最新研究知识应用于血红蛋白病内分泌并发症的日常临床实践。