Department of Hematology/Oncology, University Children's Hospital Basel, Basel, Switzerland.
Centre de Référence National des Cytopénies Autoimmunes de l'Enfant (CEREVANCE), Pediatric Hematologic Unit, Centre d'Investigation Clinique Plurithématique (CICP) INSERM 1401, University Hospital of Bordeaux, Bordeaux, France.
Br J Haematol. 2023 Oct;203(1):36-42. doi: 10.1111/bjh.19081.
Defining immune thrombocytopenia (ITP) in two age groups-children and adults-overlooks the specific clinical features and needs of adolescents and young adults (AYAS). We previously reported a high risk of chronic disease at 12 months (50%); however, data on the course of chronic ITP, the risk of refractoriness and treatment strategies in AYAS are limited. Data from patients aged 12-25 years with chronic primary ITP at 12 months were extracted from three large registries between 2004 and 2021. Clinical and laboratory data were evaluated until 48 months of follow-up (FU). Refractory ITP was defined as the administration of ≥3 different lines of therapy. A total of 427 AYAS (64% female) with chronic ITP were included. Overall, 7% and 14% were classified as 'refractory' at 12 and 48 months of FU respectively. The proportion of males was greater in the refractory group than in the non-refractory group (43% vs. 35%). AYAS with refractory disease displayed lower median platelet counts, more bleeding and a higher need for treatment at initial diagnosis and FU than non-refractory patients. This study reveals that refractory ITP is uncommon in AYAS; however, AYAS with refractory ITP display a high disease burden at all time points, including at initial diagnosis.
将免疫性血小板减少症 (ITP) 定义为两个年龄组——儿童和成人——忽略了青少年和年轻成年人 (AYAS) 的特定临床特征和需求。我们之前报告过，12 个月时慢性疾病的风险很高（50%）；然而，关于 AYAS 慢性 ITP 的病程、难治性风险和治疗策略的数据有限。从 2004 年至 2021 年期间的三个大型登记处中提取了年龄在 12-25 岁、12 个月时患有慢性原发性 ITP 的患者的数据。在随访 48 个月期间，评估了临床和实验室数据。难治性 ITP 的定义为接受了≥3 种不同的治疗方案。共纳入了 427 例慢性 ITP 的 AYAS（64%为女性）。总体而言，难治性 ITP 在 12 个月和 48 个月的 FU 中分别有 7%和 14%被归类为“难治性”。难治性组中男性的比例高于非难治性组（43% vs. 35%）。难治性疾病的 AYAS 最初诊断和 FU 时的血小板计数中位数较低、出血较多、治疗需求较高。这项研究表明，难治性 ITP 在 AYAS 中并不常见；然而，难治性 ITP 的 AYAS 在所有时间点都表现出较高的疾病负担，包括在初始诊断时。
