{"title":"评估口服托法替尼治疗儿童脱发症的有效性和安全性","authors":"Robabeh Abedini, Saman Al-Zahawi, Soroosh Dehghan, Narges Ghandi, Maryam Nasimi, Zahra Razavi","doi":"10.1155/2024/3310042","DOIUrl":null,"url":null,"abstract":"<div>\n <p>Alopecia areata (AA) is a common chronic relapsing nonscarring alopecia. Severe forms of AA commonly manifest during childhood. Treatment of AA is challenging due to the variability of the disease course as well as unpredictable responses to treatment. There is no uniform approved treatment for cure or sustained remission in children till now. Tofacitinib emerged as a novel drug in the treatment of AA, but few studies have been conducted on its safety and efficacy in children. Limitation of this study includes retrospective nature, small sample size, and lack of prolonged follow-up. <i>Aim</i>. This retrospective study aimed to assess the efficacy and safety of oral tofacitinib in children with AA. <i>Method</i>. In this retrospective study, we included patients aged 18 years or younger with AA. The scalp blandness of included patients was greater than 20% and they were on oral tofacitinib for at least two months. The demographic data, clinical characteristics, tofacitinib efficacy, and adverse effects were recorded. The primary endpoint was the last recorded percent change in the Severity of Alopecia Tool (SALT) score during treatment. <i>Results</i>. We included 26 patients (12 males and 14 females) with AA with a mean age of 11.6 ± 4.42 (3–18) years. Eighteen of them were in the alopecia areata (AA) group, whereas eight patients had alopecia totalis (AT) or alopecia universalis (AU). The mean disease duration before starting treatment with tofacitinib was 3.9 ± 3.3 years. Most of the patients were on a tofacitinib daily dose of 5 mg (53.85%) and 10 mg (38.46%). Patients were on tofacitinib for 6.73 ± 3.79 months. The patients’ baseline SALT score was recorded as 68.58 ± 32.65 and the final SALT score was 17.65 ± 23.88. Thus, the patients achieved a 50.92% reduction in the SALT score. Interestingly, there were no statistically significant differences in clinical efficacy between subtypes of AA and AT/AU. <i>Conclusion</i>. Tofacitinib was significantly effective in treating AA and AT/AU in children, with mild tolerable adverse effects, although relapse during treatment and tapering was recorded. Future randomized clinical trials with longer follow-up periods are needed to evaluate the safety of oral tofacitinib in children.</p>\n </div>","PeriodicalId":11045,"journal":{"name":"Dermatologic Therapy","volume":"2024 1","pages":""},"PeriodicalIF":3.7000,"publicationDate":"2024-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1155/2024/3310042","citationCount":"0","resultStr":"{\"title\":\"Evaluation of the Efficacy and Safety of Oral Tofacitinib for the Treatment of Alopecia Areata in Children\",\"authors\":\"Robabeh Abedini, Saman Al-Zahawi, Soroosh Dehghan, Narges Ghandi, Maryam Nasimi, Zahra Razavi\",\"doi\":\"10.1155/2024/3310042\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div>\\n <p>Alopecia areata (AA) is a common chronic relapsing nonscarring alopecia. Severe forms of AA commonly manifest during childhood. Treatment of AA is challenging due to the variability of the disease course as well as unpredictable responses to treatment. There is no uniform approved treatment for cure or sustained remission in children till now. Tofacitinib emerged as a novel drug in the treatment of AA, but few studies have been conducted on its safety and efficacy in children. Limitation of this study includes retrospective nature, small sample size, and lack of prolonged follow-up. <i>Aim</i>. This retrospective study aimed to assess the efficacy and safety of oral tofacitinib in children with AA. <i>Method</i>. In this retrospective study, we included patients aged 18 years or younger with AA. The scalp blandness of included patients was greater than 20% and they were on oral tofacitinib for at least two months. The demographic data, clinical characteristics, tofacitinib efficacy, and adverse effects were recorded. The primary endpoint was the last recorded percent change in the Severity of Alopecia Tool (SALT) score during treatment. <i>Results</i>. We included 26 patients (12 males and 14 females) with AA with a mean age of 11.6 ± 4.42 (3–18) years. Eighteen of them were in the alopecia areata (AA) group, whereas eight patients had alopecia totalis (AT) or alopecia universalis (AU). The mean disease duration before starting treatment with tofacitinib was 3.9 ± 3.3 years. Most of the patients were on a tofacitinib daily dose of 5 mg (53.85%) and 10 mg (38.46%). Patients were on tofacitinib for 6.73 ± 3.79 months. The patients’ baseline SALT score was recorded as 68.58 ± 32.65 and the final SALT score was 17.65 ± 23.88. Thus, the patients achieved a 50.92% reduction in the SALT score. Interestingly, there were no statistically significant differences in clinical efficacy between subtypes of AA and AT/AU. <i>Conclusion</i>. Tofacitinib was significantly effective in treating AA and AT/AU in children, with mild tolerable adverse effects, although relapse during treatment and tapering was recorded. Future randomized clinical trials with longer follow-up periods are needed to evaluate the safety of oral tofacitinib in children.</p>\\n </div>\",\"PeriodicalId\":11045,\"journal\":{\"name\":\"Dermatologic Therapy\",\"volume\":\"2024 1\",\"pages\":\"\"},\"PeriodicalIF\":3.7000,\"publicationDate\":\"2024-09-23\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://onlinelibrary.wiley.com/doi/epdf/10.1155/2024/3310042\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Dermatologic Therapy\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://onlinelibrary.wiley.com/doi/10.1155/2024/3310042\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"DERMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Dermatologic Therapy","FirstCategoryId":"3","ListUrlMain":"https://onlinelibrary.wiley.com/doi/10.1155/2024/3310042","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"DERMATOLOGY","Score":null,"Total":0}
引用次数: 0
摘要
斑秃(AA)是一种常见的慢性复发性非瘢痕性脱发。严重的斑秃通常发生在儿童时期。由于病程多变以及对治疗的反应难以预测,AA 的治疗极具挑战性。到目前为止,还没有一种经批准的治疗方法可以治愈或持续缓解儿童的病情。托法替尼是治疗 AA 的新型药物,但有关其在儿童中的安全性和有效性的研究很少。本研究的局限性包括回顾性、样本量小以及缺乏长期随访。研究目的这项回顾性研究旨在评估口服托法替尼对 AA 儿童的疗效和安全性。研究方法在这项回顾性研究中,我们纳入了 18 岁或 18 岁以下的 AA 患者。纳入患者的头皮白化率大于 20%,且口服托法替尼至少两个月。研究记录了患者的人口统计学数据、临床特征、托法替尼疗效和不良反应。主要终点是治疗期间脱发严重程度工具(SALT)评分的最后记录变化百分比。研究结果我们共纳入了26名AA患者(12男14女),平均年龄为(11.6 ± 4.42)(3-18)岁。其中18人属于斑秃(AA)组,8人患有全秃(AT)或普秃(AU)。开始接受托法替尼治疗前的平均病程为(3.9 ± 3.3)年。大多数患者每天服用的托法替尼剂量为5毫克(53.85%)和10毫克(38.46%)。患者服用托法替尼的时间为(6.73 ± 3.79)个月。患者的基线 SALT 得分为 68.58 ± 32.65,最终 SALT 得分为 17.65 ± 23.88。因此,患者的 SALT 分数降低了 50.92%。有趣的是,AA 和 AT/AU 亚型之间的临床疗效没有明显的统计学差异。结论托法替尼对治疗儿童AA和AT/AU有明显疗效,不良反应轻微,可耐受,但在治疗和减量期间有复发记录。未来需要进行更长时间的随机临床试验,以评估儿童口服托法替尼的安全性。
Evaluation of the Efficacy and Safety of Oral Tofacitinib for the Treatment of Alopecia Areata in Children
Alopecia areata (AA) is a common chronic relapsing nonscarring alopecia. Severe forms of AA commonly manifest during childhood. Treatment of AA is challenging due to the variability of the disease course as well as unpredictable responses to treatment. There is no uniform approved treatment for cure or sustained remission in children till now. Tofacitinib emerged as a novel drug in the treatment of AA, but few studies have been conducted on its safety and efficacy in children. Limitation of this study includes retrospective nature, small sample size, and lack of prolonged follow-up. Aim. This retrospective study aimed to assess the efficacy and safety of oral tofacitinib in children with AA. Method. In this retrospective study, we included patients aged 18 years or younger with AA. The scalp blandness of included patients was greater than 20% and they were on oral tofacitinib for at least two months. The demographic data, clinical characteristics, tofacitinib efficacy, and adverse effects were recorded. The primary endpoint was the last recorded percent change in the Severity of Alopecia Tool (SALT) score during treatment. Results. We included 26 patients (12 males and 14 females) with AA with a mean age of 11.6 ± 4.42 (3–18) years. Eighteen of them were in the alopecia areata (AA) group, whereas eight patients had alopecia totalis (AT) or alopecia universalis (AU). The mean disease duration before starting treatment with tofacitinib was 3.9 ± 3.3 years. Most of the patients were on a tofacitinib daily dose of 5 mg (53.85%) and 10 mg (38.46%). Patients were on tofacitinib for 6.73 ± 3.79 months. The patients’ baseline SALT score was recorded as 68.58 ± 32.65 and the final SALT score was 17.65 ± 23.88. Thus, the patients achieved a 50.92% reduction in the SALT score. Interestingly, there were no statistically significant differences in clinical efficacy between subtypes of AA and AT/AU. Conclusion. Tofacitinib was significantly effective in treating AA and AT/AU in children, with mild tolerable adverse effects, although relapse during treatment and tapering was recorded. Future randomized clinical trials with longer follow-up periods are needed to evaluate the safety of oral tofacitinib in children.
期刊介绍:
Dermatologic Therapy has been created to fill an important void in the dermatologic literature: the lack of a readily available source of up-to-date information on the treatment of specific cutaneous diseases and the practical application of specific treatment modalities. Each issue of the journal consists of a series of scholarly review articles written by leaders in dermatology in which they describe, in very specific terms, how they treat particular cutaneous diseases and how they use specific therapeutic agents. The information contained in each issue is so practical and detailed that the reader should be able to directly apply various treatment approaches to daily clinical situations. Because of the specific and practical nature of this publication, Dermatologic Therapy not only serves as a readily available resource for the day-to-day treatment of patients, but also as an evolving therapeutic textbook for the treatment of dermatologic diseases.