METHODS: Multicentric retrospective observational study including patients <18 years treated with VMF alone for refractory BRAFV600E mutated LCH in 13 countries between October 1, 2013 and December 31, 2018.
RESULTS: Fifty-seven patients: 56% female, median age 2.1 years (0.2-14.6), median treatment duration 4.1 months (1.4-29.7). Forty-one patients (72%) had at least one CAE: photosensitivity (40%), keratosis pilaris (32%), rash (26%), xerosis (21%), and neutrophilic panniculitis (16%). No skin tumor was observed. Five percent of CAEs were grade 3. None were grade 4 or led to permanent VMF discontinuation. Dose reduction was necessary for 12% of patients, temporary treatment discontinuation for 16%, none leading to loss of efficacy. VMF dose, median RPL, and efficacy were not correlated with CAE occurrence.
CONCLUSIONS: At doses used for pediatric LCH, CAEs are frequent but rarely severe and have little impact on the continuation of treatment when managed appropriately. Regular dermatological follow-up is essential to manage CAEs and screen for possible induced skin tumors.
方法:多中心回顾性观察性研究,包括2013年10月1日至2018年12月31日在13个国家/地区仅接受VMF治疗难治性BRAFV600E突变LCH的患者。
结果:57例患者:56%为女性,中位年龄2.1岁(0.2-14.6),中位治疗持续时间4.1个月(1.4-29.7)。41例患者(72%)至少有一种CAE:光敏性(40%),毛发角化病(32%),皮疹(26%),干燥症(21%),和中性粒细胞脂膜炎(16%)。未观察到皮肤肿瘤。百分之五的CAE是3级。没有一个是4级或导致永久停止VMF。12%的患者需要减少剂量,暂时停止治疗16%,没有导致功效丧失。VMF剂量,RPL中位数,疗效与CAE发生无关。
结论:在用于小儿LCH的剂量下,CAE是常见的,但很少严重,如果管理得当,对继续治疗的影响很小。定期的皮肤病学随访对于管理CAE和筛查可能诱发的皮肤肿瘤至关重要。