Real-world data on the clinical use of secukinumab in pediatric generalized pustular psoriasis: a 48-week retrospective study

Generalized pustular psoriasis (GPP) is a recurrent life-threatening disease and usually uncommon in children. Secukinumab, an interleukin (IL) -17 inhibitor, has been shown to be effective in the treatment of GPP in adult patients. However, there is currently a lack of evidence for its long-term efficacy and safety in the pediatric GPP population. This is a descriptive retrospective study that collected data from 18 pediatric patients with moderate to severe GPP treated with secukinumab in our department from July 2019 to August 2020, with 48 weeks of follow-up. Patient demographics and treatment characteristics were assessed. Response to secukinumab was evaluated by body temperature changes, laboratory examination assessments, Generalized Pustular Psoriasis Physician Global Assessment (GPPASI) scores, Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) scores, Children's Dermatology Life Quality Index (CDLQI) scores, and Pediatric Quality of Life Inventory (pedsQL) scores. Adverse events were also recorded. All patients achieved GPPASI 50 at week 2 and GPPASI 75 at week 4. From week 12 to week 48, most patients achieved GPPASI 100 and only five patients did not reach GPPASI 100 due to minimal relapse. At week 2, the mean absolute GPPASI had a sharply decrease from 31.7 to 5.1 and improved further to 1.3 in week 4. Patients’ body temperature returned to normal at day 3. Pustules subsided within 3 days, erythema and scales decreased rapidly in the second week. The average CDLQI score decreased rapidly from 13.6 to 1.8 at week 4 and to 0 at week 48. For pedsQL scores, Physical Health Summary Score and Psychosocial Health Summary Score (mean change from baseline) improved gradually over time. All patients were followed for 48 weeks, and no serious adverse events were observed. Side effects that occurred in nine patients were mild and manageable. In conclusion, GPP systemic inflammation and skin symptoms did rapidly improve with the treatment of secukinumab, and this curative effect was maintained for 48 weeks without any unexpected safety signal. Our study shows that secukinumab could be an effective treatment option for pediatric patients with GPP.

泛发性脓疱型银屑病（Generalized pustular psoriasis, GPP）是一种反复发作的致命性疾病，在儿童群体中较为罕见。司库奇尤单抗（Secukinumab）作为一种白细胞介素（interleukin, IL）-17抑制剂，已被证实可有效治疗成人GPP患者，但目前针对儿童GPP人群，其长期疗效与安全性的相关证据仍较为匮乏。本研究为描述性回顾性研究，收集了2019年7月至2020年8月于我科室接受司库奇尤单抗治疗的18例中重度儿童GPP患者的数据，随访周期为48周。研究评估了患者的人口学特征与治疗相关特征，并通过体温变化、实验室检查指标、泛发性脓疱型银屑病医师总体评估（Generalized Pustular Psoriasis Physician Global Assessment, GPPASI）评分、泛发性脓疱型银屑病医师总体评估（Generalized Pustular Psoriasis Physician Global Assessment, GPPGA）评分、儿童皮肤病生活质量指数（Children's Dermatology Life Quality Index, CDLQI）评分以及儿童生活质量量表（Pediatric Quality of Life Inventory, pedsQL）评分，对患者接受司库奇尤单抗治疗后的应答情况进行评估，同时记录不良事件发生情况。所有患者均在第2周达到GPPASI 50，第4周达到GPPASI 75。第12周至第48周期间，多数患者实现GPPASI 100，仅5例患者因轻度复发未达到GPPASI 100。第2周时，患者的平均绝对GPPASI评分从31.7显著下降至5.1，并在第4周进一步改善至1.3。患者体温于治疗第3天恢复正常，脓疱于3天内消退，红斑与鳞屑在第二周快速减少。平均CDLQI评分从基线的13.6快速降至第4周的1.8，并在第48周时降至0。在pedsQL评分方面，躯体健康总评分与心理社会健康总评分（较基线的平均变化值）随时间推移逐步改善。所有患者均完成了48周的随访，未观察到严重不良事件。9例患者出现的不良反应均为轻度且可耐受。综上，司库奇尤单抗治疗可快速改善儿童GPP患者的全身炎症反应与皮肤症状，且该疗效可维持48周，未出现任何未预期的安全性信号。本研究表明，司库奇尤单抗可作为儿童GPP患者的有效治疗选择。