METHODS: This is a retrospective study of all pemphigus patients aged <18 years, diagnosed between 2000 and 2023, from three tertiary medical centers in Israel. The diagnosis was confirmed by positive immunofluorescence.
RESULTS: Twelve pediatric pemphigus patients were included (mean age 10.7 ± 4.3 years, male:female ratio 1:1). Mean diagnostic delay was 11.1 ± 12.6 months (range 1.8-36 months). Most patients had pemphigus vulgaris with mucosal involvement (58.3%). First-line treatment for all patients included systemic corticosteroids (sCS), with a treatment duration (including tapering down) of 28 ± 18.4 months. Hospitalization did not yield better outcomes. Only three patients achieved sustained complete response with sCS treatment (25.0%), and the rest required additional therapeutics, most commonly rituximab. Rituximab showed a good safety profile and therapeutic response. Follow-up was recorded up to 18.1 years after diagnosis (mean: 5.6 years). Three of five patients with information available more than 5 years after the pemphigus diagnosis still exhibited disease symptoms.
CONCLUSIONS: Pediatric pemphigus is associated with a significant diagnostic delay. While sCS can induce remission in most patients as a first-line treatment, long-term disease control requires additional immunomodulators. Long-term follow-up reveals a chronic yet mostly benign disease course in this population and advocates for the use of rituximab in pediatric pemphigus patients.
方法:这是一项对所有年龄<18岁的天疱疮患者的回顾性研究,在2000年至2023年之间诊断,来自以色列的三个三级医疗中心。免疫荧光阳性证实了诊断。
结果:纳入12例小儿天疱疮患者(平均年龄10.7±4.3岁,男性:女性比例为1:1)。平均诊断延迟为11.1±12.6个月(范围1.8-36个月)。大多数患者患有粘膜受累的寻常型天疱疮(58.3%)。所有患者的一线治疗包括全身性皮质类固醇(sCS),治疗持续时间(包括逐渐减少)为28±18.4个月。住院没有产生更好的结果。只有3名患者在sCS治疗中获得了持续的完全缓解(25.0%),其余的需要额外的治疗,最常见的是利妥昔单抗。利妥昔单抗显示出良好的安全性和治疗反应。随访记录到诊断后18.1年(平均:5.6年)。在天疱疮诊断超过5年后,获得信息的五名患者中有三名仍表现出疾病症状。
结论:小儿天疱疮与显著的诊断延迟有关。虽然sCS可以作为一线治疗在大多数患者中引起缓解,长期疾病控制需要额外的免疫调节剂.长期随访揭示了该人群的慢性但大多数是良性疾病过程,并主张在小儿天疱疮患者中使用利妥昔单抗。