巴西替尼治疗儿童慢性免疫性血小板减少症及相关自身免疫性疾病:一例报告

IF 2.1 3区 医学 Q2 PEDIATRICS Frontiers in Pediatrics Pub Date : 2024-12-24 eCollection Date: 2024-01-01 DOI:10.3389/fped.2024.1516039
G López de Hontanar Torres, J Zubicaray, E Sebastián, A Hernández-Martín, J Iriondo, J Sevilla
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引用次数: 0

摘要

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,在临床实践中提出了重大挑战。经典的一线治疗管理包括皮质类固醇和静脉注射免疫球蛋白。尽管这种情况在儿童中的发生率低于成人,但根据儿童和成人慢性ITP登记处的数据,由于一线治疗无效或疾病持续存在等原因,有很大比例的患者(高达47%)需要二线或进一步治疗。目前正在使用几种二线治疗方法,包括使用血小板生成素受体激动剂增加血小板产量。我们报告一例16岁的ITP和斑秃患者成功治疗baricitinib,一种可逆和选择性JAK 1/2抑制剂。Baricitinib目前用于治疗几种自身免疫性疾病,并已被证明可增加这些患者的血小板计数。这种现象与TPO信号的增加和血小板破坏的减少有关。baricitinib治疗成人ITP患者有很好的初步结果。该病例报告是首次报道在儿童和青少年ITP中使用巴西替尼,可能导致在这种情况下使用巴西替尼。
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Baricitinib in pediatric chronic immune thrombocytopenia and associated autoimmune conditions: a case report.

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.

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来源期刊
Frontiers in Pediatrics
Frontiers in Pediatrics Medicine-Pediatrics, Perinatology and Child Health
CiteScore
3.60
自引率
7.70%
发文量
2132
审稿时长
14 weeks
期刊介绍: Frontiers in Pediatrics (Impact Factor 2.33) publishes rigorously peer-reviewed research broadly across the field, from basic to clinical research that meets ongoing challenges in pediatric patient care and child health. Field Chief Editors Arjan Te Pas at Leiden University and Michael L. Moritz at the Children''s Hospital of Pittsburgh are supported by an outstanding Editorial Board of international experts. This multidisciplinary open-access journal is at the forefront of disseminating and communicating scientific knowledge and impactful discoveries to researchers, academics, clinicians and the public worldwide. Frontiers in Pediatrics also features Research Topics, Frontiers special theme-focused issues managed by Guest Associate Editors, addressing important areas in pediatrics. In this fashion, Frontiers serves as an outlet to publish the broadest aspects of pediatrics in both basic and clinical research, including high-quality reviews, case reports, editorials and commentaries related to all aspects of pediatrics.
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