Department of Clinical Immunology and Rheumatology, St. John's Medical College Hospital, St. John's National Academy of Medical Sciences, Sarjapur Road, Bengaluru, 560034, India.
Rheumatic Disease Clinic, Mumbai, India.
Clin Rheumatol. 2024 Jul;43(7):2245-2252. doi: 10.1007/s10067-024-07019-x. Epub 2024 Jun 3.
Determine domain-based-outcomes and steroid-sparing efficacy of generic tofacitinib in IIM.
This is a multicenter retrospective study wherein clinical phenotype, autoantibody profile, prior immunosuppressives, and outcomes at 3, 6, and 12 months were retrieved for IIM patients prescribed tofacitinib. Overall clinical response was assessed as complete or partial remission as per physician judgment. Changes in cutaneous and calcinosis domain were recorded as per physician global assessment (PGA), lung domain as per medical research council (MRC) dyspnea scale, and muscle strength by Manual Muscle Testing-8 (MMT-8).
Forty-two patients of IIM with mean age 38.7 ± 16 years; (76.2% (N = 32) women), median duration of illness 48 (19;88) months were included. Commonest indication for initiating tofacitinib was either for refractory or as steroid sparing for cutaneous domain (N = 25/42, 59.5%) followed by calcinosis (N = 16/42, 38%). Overall complete and/or partial remission was achieved in 23/37 (64.8%), 30/35 (85.7%), and 29/30 (96.6%) patients at 3, 6, and 12 months, respectively. At 12-month follow-up, there was a reduction in prednisolone dose, with absolute decrease from a daily dose of 17.5 mg (IQR 5;50) to 2.5 mg (IQR 0;5) (p < 0.001). Individual domain assessments revealed improvement in cutaneous domain [16/25 (64%)] and calcinosis [6/15 (40%)]. Adverse effects included herpes zoster (N = 2/42, 4.8%) and dyslipidemia (N = 4/42, 9.5%).
Treatment with generic tofacitinib significantly reduces the daily dose of corticosteroids and is effective in cutaneous domain including calcinosis in IIM.
• This multicenter retrospective study is the first real-world data from India, elucidating steroid sparing efficacy of generic tofacitinib in patients with inflammatory myositis. • Domain-based outcome assessment suggests good clinical improvement especially in cutaneous domain, even those with refractory disease. • Modest benefits were evident in calcinosis, but its effect on the muscle and pulmonary domain appears limited.
评估通用托法替布治疗特发性炎性肌病（IIM）的疗效和对激素的影响。
这是一项多中心回顾性研究，我们对接受托法替布治疗的 IIM 患者的临床表型、自身抗体谱、既往免疫抑制剂使用情况和治疗 3、6、12 个月时的结局进行了回顾。根据医生的判断，将整体临床反应评估为完全或部分缓解。根据医生整体评估（PGA）记录皮肤和钙沉积域的变化，根据医学研究委员会（MRC）呼吸困难量表记录肺部域的变化，使用手动肌肉测试-8（MMT-8）记录肌肉力量。
共纳入 42 例特发性炎性肌病患者，平均年龄 38.7±16 岁（76.2%（N=32）为女性），中位病程 48（19；88）个月。启动托法替布治疗的最常见指征是难治性疾病或为了治疗皮肤域的激素（N=25/42，59.5%），其次是钙沉积（N=16/42，38%）。治疗 3、6 和 12 个月时，37 例患者中有 23 例（64.8%）、35 例患者中有 30 例（85.7%）、30 例患者中有 29 例（96.6%）达到完全或部分缓解。在 12 个月的随访中，泼尼松龙剂量降低，从每日 17.5mg（IQR 5；50）降至 2.5mg（IQR 0；5）（p<0.001）。各域评估显示皮肤域（16/25，64%）和钙沉积域（6/15，40%）均有改善。不良事件包括带状疱疹（N=2/42，4.8%）和血脂异常（N=4/42，9.5%）。
使用通用托法替布治疗可显著降低皮质类固醇的日剂量，并有效治疗 IIM 的皮肤域，包括钙沉积。
