Department of Traditional Chinese Medicine, Peking Union Medical College Hospital, Peking Union Medical College and Chinese Academy of Medical Sciences, Beijing, China.
Peking Union Medical College Hospital, Peking Union Medical College and Chinese Academy of Medical Sciences, Beijing, China.
JAMA Dermatol. 2021 Jan 1;157(1):74-78. doi: 10.1001/jamadermatol.2020.3095.
Nail involvement is common in synovitis, acne, pustulosis, hyperostosis, and osteitis (SAPHO) syndrome, which has a strong association with quality of life in patients with SAPHO. Tofacitinib is an oral Janus kinase inhibitor that has been previously shown to be effective for nail psoriasis.
To assess the efficacy and safety of tofacitinib for the treatment of nail involvement in SAPHO syndrome.
Participants received tofacitinib, 5 mg, twice daily, for 12 weeks.
DESIGN, SETTING, AND PARTICIPANTS: This open-label, single-arm, prospective pilot study included 13 patients with SAPHO syndrome accompanied by nail lesions and active palmoplantar pustulosis who were recruited from Peking Union Medical College Hospital from September 2019 to December 2019. Follow-up was completed in March 2020. Analysis began March 2020.
The primary end point was the percentage of the change from baseline in Nail Psoriasis Severity Index scores at week 12. Secondary end points included the percentage of the change from baseline in Palmoplantar Psoriasis Area and Severity Index scores, change from baseline in Visual Analogue Scale scores in global osteoarticular pain, Dermatology Life Quality Index scores, and inflammatory markers. Adverse events were recorded throughout the study.
Thirteen female Asian patients (means [SD] age, 39.7 [12.3] years) were included, all of whom completed the study. At week 12, significant improvements were observed in Nail Psoriasis Severity Index scores (median, -67% [interquartile range (IQR), -56% to -77%]; P < .001) and Palmoplantar Psoriasis Area and Severity Index scores (median, -71% [IQR, -58% to -78%]; P < .001). Significant improvement was also noted in Dermatology Life Quality Index scores (median, -12 [IQR, -8.5 to -15]; P < .001) at week 12. A significant decrease in Visual Analogue Scale scores in global osteoarticular pain was observed at week 8 (median, -4 [IQR, 0 to -5]; P = .02) but was not significant at week 12. Inflammatory marker levels were decreased, as indicated by erythrocyte sedimentation rate (median, -8 mm/h [IQR, -4 mm/h to -11 mm/h]; P < .001) and high-sensitivity C-reactive protein levels (median, -1.6 [IQR, -0.3 to -4.1]; P = .01). No severe adverse events were observed.
In this pilot study, tofacitinib yielded significant remission of nail lesions and palmoplantar psoriasis accompanied by an improvement in quality of life in patients with SAPHO syndrome. Additional follow-up studies to evaluate the long-term efficacy and safety of tofacitinib for nail involvement in SAPHO syndrome are warranted.
Chinese Clinical Trial Registry number: ChiCTR1900025941.
重要性：指甲受累在滑膜炎、痤疮、脓疱病、骨质增生和骨炎（SAPHO）综合征中很常见，SAPHO 患者的生活质量与指甲受累有很强的关联。托法替尼是一种口服 Janus 激酶抑制剂，先前已被证明对指甲银屑病有效。
目的：评估托法替尼治疗 SAPHO 综合征指甲受累的疗效和安全性。
干预措施：参与者接受托法替尼，每日两次，每次 5mg，治疗 12 周。
设计、地点和参与者：这项开放标签、单臂、前瞻性试验研究纳入了 2019 年 9 月至 2019 年 12 月期间从北京协和医院招募的 13 名伴有指甲病变和活动性掌跖脓疱病的 SAPHO 综合征患者。随访于 2020 年 3 月完成。分析于 2020 年 3 月开始。
主要终点：第 12 周时 Nail Psoriasis Severity Index 评分从基线的变化百分比。次要终点包括 Palmoplantar Psoriasis Area and Severity Index 评分从基线的变化百分比、全球骨关节炎疼痛的视觉模拟量表评分从基线的变化、皮肤病生活质量指数评分和炎症标志物。整个研究过程中记录了不良事件。
结果：纳入了 13 名亚洲女性患者（平均[标准差]年龄，39.7[12.3]岁），均完成了研究。在第 12 周时，Nail Psoriasis Severity Index 评分（中位数，-67%[四分位距（IQR），-56%至-77%]；P<0.001）和 Palmoplantar Psoriasis Area and Severity Index 评分（中位数，-71%[IQR，-58%至-78%]；P<0.001）均有显著改善。在第 12 周时，皮肤病生活质量指数评分（中位数，-12[IQR，-8.5 至-15]）也有显著改善（P<0.001）。在第 8 周时观察到全球骨关节炎疼痛的视觉模拟量表评分（中位数，-4[IQR，0 至-5]；P=0.02）显著降低，但在第 12 周时无统计学意义。炎症标志物水平下降，红细胞沉降率（中位数，-8mm/h[IQR，-4mm/h 至-11mm/h]；P<0.001）和高敏 C 反应蛋白水平（中位数，-1.6[IQR，-0.3 至-4.1]；P=0.01）。未观察到严重不良事件。
结论和相关性：在这项初步研究中，托法替尼使 SAPHO 综合征患者的指甲病变和掌跖脓疱病显著缓解，并改善了生活质量。需要进一步的随访研究来评估托法替尼治疗 SAPHO 综合征指甲受累的长期疗效和安全性。
试验注册：中国临床试验注册中心编号：ChiCTR1900025941。
