López de Hontanar Torres G, Zubicaray J, Sebastián E, Hernández-Martín A, Iriondo J, Sevilla J
Pediatric Hematology and Oncology Department, Hospital Infantil Universitario Niño Jesús, Madrid, Spain.
Dermatology Department, Hospital Infantil Universitario Niño Jesús, Madrid, Spain.
Front Pediatr. 2024 Dec 24;12:1516039. doi: 10.3389/fped.2024.1516039. eCollection 2024.
Immune thrombocytopenia (ITP) is a disease characterized by platelet destruction, presenting substantial challenges in clinical practice. The classic first line therapeutic management includes corticosteroids and intravenous immunoglobulins. Although it is less frequent in children than in adults, there is a significant percentage of patients, up to 47% according to the Pediatric and Adult Registry on Chronic ITP, who require second-line or further treatment, due to non-response to the first line treatment or persistence of disease, among other reasons. Several second line approaches for its treatment are currently in use, including increasing platelet production with thrombopoietin receptor agonists. We report the case of a 16-year-old patient with ITP and alopecia areata successfully treated with baricitinib, a reversible and selective JAK 1/2 inhibitor. Baricitinib is currently in use for the treatment of several autoimmune conditions and has been shown to increase platelet counts in these patients. This phenomenon has been linked to increased TPO signaling and reduced platelet destruction. There are promising preliminary results of adult ITP patients treated with baricitinib. This case report is the first reported use of baricitinib in ITP in the pediatric and adolescent setting, potentially leading to its use in this condition.
免疫性血小板减少症(ITP)是一种以血小板破坏为特征的疾病,在临床实践中面临诸多挑战。经典的一线治疗方法包括使用皮质类固醇和静脉注射免疫球蛋白。尽管儿童患者比成人患者少见,但仍有相当比例的患者(根据慢性ITP儿科和成人登记处的数据,高达47%)因对一线治疗无反应或疾病持续存在等原因而需要二线或进一步治疗。目前有几种二线治疗方法正在使用,包括使用血小板生成素受体激动剂来增加血小板生成。我们报告了一例16岁患有ITP和斑秃的患者,使用巴瑞替尼(一种可逆性和选择性JAK 1/2抑制剂)成功治疗的病例。巴瑞替尼目前用于治疗多种自身免疫性疾病,并且已显示可增加这些患者的血小板计数。这种现象与血小板生成素信号增加和血小板破坏减少有关。使用巴瑞替尼治疗成人ITP患者已有一些有前景的初步结果。本病例报告是首次报道在儿科和青少年ITP患者中使用巴瑞替尼,这可能会促使其在这种疾病中得到应用。