Ravelli Angelo, Rosina Silvia, MacMahon Jayne M, Baird Talia, Rebollo-Giménez Ana Isabel, Hinze Claas, McCann Liza J, Reed Ann M, Rider Lisa G, Arvigo Matilde, Bader-Meunier Brigitte, Bruno Claudio, Caifeng Li, Campanilho-Marques Raquel, Cuccato Sara, Fiorillo Chiara, Hahn Nikki A, Huber Adam M, Jansen Marc, Kasapcopur Ozgur, Katsikas Maria Martha, Kim Susan, Livermore Polly, Maillard Sue, Malattia Clara, Migowa Angela Nyangore, Miyamae Takako, Murphy Ruth, Nicolai Rebecca, Papadopoulou Charalampia, Pilkington Clarissa, Sanner Helga, Sawhney Sujata, Smolewska Elzbieta, Tarvin Stacey E, Tiller Georgina, Toplak Natasa, Wedderburn Lucy R, Bovis Francesca, Consolaro Alessandro, Feldman Brian M
Direzione Scientifica, IRCCS Istituto Giannina Gaslini, Genoa, Italy; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DINOGMI), University of Genova, Genoa, Italy.
UOC Reumatologia e Malattie Autoinfiammatorie, IRCCS Istituto Giannina Gaslini, Genoa, Italy.
Ann Rheum Dis. 2025 Jul;84(7):1055-1067. doi: 10.1016/j.ard.2025.04.024. Epub 2025 May 22.
Despite the recent prognostic improvement, a sizeable proportion of patients with juvenile dermatomyositis (JDM) respond suboptimally to contemporary therapies. This study aimed to develop recommendations for treating JDM to target.
A Steering Committee formulated a set of provisional recommendations based on evidence derived from a systematic literature review and a retrospective chart review of patients. These were discussed, amended, and voted on by an international Task Force, including 28 paediatric rheumatologists, 2 specialists in neuromuscular diseases, 1 dermatologist, 1 physical therapist, 1 research nurse, 2 patients with JDM, and 1 parent of a patient with JDM. Items that achieved at least an 80% majority vote were accepted as final recommendations.
Although the literature review did not reveal trials that compared a treat-to-target strategy with a nonsteered approach, it provided indirect evidence about specific end points that could serve as targets that facilitated development of recommendations. The group reached consensus on 7 overarching principles and 12 recommendations. It was agreed that both patients/parents and treaters should share decisions in setting treatment targets and therapeutic strategies, with inactive disease as the preferred target and minimal disease activity an alternative one. Inactive disease is targeted to be achieved within 12 months after treatment start. Interim targets include minimal and moderate clinical improvement within 6 weeks and 3 months, respectively, and normalisation of muscle strength within 6 months. High-dose glucocorticoids remain fundamental in the initial management, but progressive tapering and discontinuation within 12 months through optimisation of concomitant immunomodulatory therapy was advised. A research agenda was formulated.
The Task Force developed recommendations for treating JDM to target, being aware that the evidence is not strong and needs to be expanded by future research. Implementation of the recommendations in clinical practice will help to reach optimal outcomes for JDM.
尽管近期预后有所改善，但仍有相当一部分青少年皮肌炎（JDM）患者对当代治疗反应欠佳。本研究旨在制定针对JDM治疗目标的建议。
指导委员会根据系统文献综述和对患者的回顾性病历审查得出的证据制定了一套临时建议。这些建议由一个国际特别工作组进行讨论、修订和投票，该工作组包括28名儿科风湿病学家、2名神经肌肉疾病专家、1名皮肤科医生、1名物理治疗师、1名研究护士、2名JDM患者以及1名JDM患者的家长。获得至少80%多数票的项目被接受为最终建议。
尽管文献综述未发现将治疗达标策略与非定向方法进行比较的试验，但它提供了关于可作为目标的特定终点的间接证据，这有助于制定建议。该小组就7项总体原则和12项建议达成了共识。一致认为患者/家长和治疗者都应共同参与设定治疗目标和治疗策略，以疾病静止为首选目标，最小疾病活动度为替代目标。目标是在治疗开始后12个月内实现疾病静止。 interim targets include minimal and moderate clinical improvement within 6 weeks and 3 months, respectively, and normalisation of muscle strength within 6 months. 高剂量糖皮质激素在初始治疗中仍然是基础，但建议通过优化联合免疫调节治疗在12个月内逐步减量并停用。制定了一项研究议程。
特别工作组制定了针对JDM治疗目标的建议，同时意识到证据并不充分，需要未来的研究加以扩充。在临床实践中实施这些建议将有助于为JDM患者实现最佳治疗效果。
