Department of Paediatric Pulmonology, Immunology and Critical Care Medicine, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany.
Department of Paediatrics, Division of Gastroenterology, Nephrology and Metabolic Diseases, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany.
Pediatr Rheumatol Online J. 2023 Sep 26;21(1):108. doi: 10.1186/s12969-023-00875-y.
The objective of this initiative was to develop a treat-to-target (T2T) approach for the management of patients with Familial Mediterranean Fever (FMF), including the definition of a complex treatment target, and establish strategies that improve patient care and long-term outcome.
An initial set of statements as well as a flow chart visualising the proposed concept was developed. To adapt the preliminary statements to the current state of knowledge, a systematic literature search was performed and the modified statements were subject to a Delphi approach. To ensure the applicability of the statements in daily practice, an online survey was conducted among paediatric rheumatologists in Germany. In addition, data from the national AID-NET registry were analysed with respect to therapeutic response.
This T2T initiative yielded a total of 26 statements guiding FMF management with respect to diagnosis, treatment targets, treatment strategies and monitoring. The online survey identified cut-off values for inflammatory markers indicating treatment intensification and appropriate measures in case of colchicine intolerance or non-adherence. The analysis of data derived from the national AID-NET showed that colchicine therapy was successfully terminated in 61% of patients (27 out of 44) with heterozygous MEFV mutations. Multidimensional treatment targets incorporating objective and subjective reported outcome measures were developed. These provide the basis for stratifying patients into the following treatment paths: continue colchicine, persisting attacks / inflammation, colchicine intolerance, persisting arthritis, colchicine reduction and adjustment/reduction of biologics.
The proposed consensus treatment plan for the management of FMF incorporates multidimensional targets allowing transparent treatment decisions, which will promote personalised disease management and increase adherence to therapy.
本倡议的目的是为家族性地中海热(FMF)患者的管理制定一个达标治疗(T2T)方法,包括定义复杂的治疗目标,并制定改善患者护理和长期预后的策略。
制定了一套初始陈述和流程图,以可视化提出的概念。为了使初步陈述适应当前的知识状态,进行了系统的文献检索,并对修改后的陈述进行了 Delphi 方法评估。为了确保陈述在日常实践中的适用性,在德国对儿科风湿病学家进行了在线调查。此外,还分析了国家 AID-NET 登记处的数据,以评估治疗反应。
这项 T2T 倡议共产生了 26 项陈述,指导 FMF 管理的诊断、治疗目标、治疗策略和监测。在线调查确定了指示治疗强化和在秋水仙碱不耐受或不依从时采取适当措施的炎症标志物的临界值。从国家 AID-NET 获得的数据分析表明,在杂合子 MEFV 突变的 44 名患者中(27 名),秋水仙碱治疗成功终止。制定了包含客观和主观报告结果测量的多维治疗目标。这些为患者分层进入以下治疗路径提供了基础:继续秋水仙碱、持续发作/炎症、秋水仙碱不耐受、持续关节炎、秋水仙碱减少以及调整/减少生物制剂。
拟议的 FMF 管理共识治疗计划纳入了多维目标,允许透明的治疗决策,这将促进个性化疾病管理并提高治疗依从性。