Schifferli Alexandra, Le Gavrian Gautier, Aladjidi Nathalie, Moulis Guillaume, Godeau Bertrand, Leblanc Thierry, Héritier Sébastien, Fernandes Helder, Kühne Thomas
Department of Hematology/Oncology, University Children's Hospital Basel, Basel, Switzerland.
Pediatric Hematologic Unit, Centre d'Investigation Clinique Plurithématique INSERM 1401, University Hospital of Bordeaux, Bordeaux, France.
Blood Adv. 2024 Dec 24;8(24):6183-6194. doi: 10.1182/bloodadvances.2024014381.
Adolescents and young adults (AYAs) with immune thrombocytopenia (ITP) exhibit distinct clinical features and needs, defying categorization as either adults or children. Previous findings revealed a 50% risk of chronic disease at 12 months, yet the long-term course remains unclear. This study aimed to delineate the clinical and laboratory characteristics of AYAs with chronic primary ITP. Data from patients aged 12 to 25 years with chronic disease at 1 year were extracted from 3 registries (Pediatric and Adult Registry on Chronic ITP, CEREVANCE, and Cytopénies Auto-immunes Registre Midi-Pyrénéen), covering the period from 2004 to 2021. Sustained complete remission off treatment (SCROT) occurring beyond 12 months was defined as platelet count of >100 × 109/L without treatment for at least 12 months, independently of the previous treatment strategy. A total of 427 AYAs (64% female) with chronic primary ITP were included. Clinical information was available for ∼100% of patients at initial diagnosis and at 6- and 12-month follow-ups (FUs); and for 88%, 77%, and 59% at 24, 36, and 48 months, respectively. Over time, clinical features improved gradually, with fewer patients requiring treatment. Throughout the FU period, second-line drug use increased steadily among treated patients, without affecting the need for corticosteroids and IV immunoglobulins. The proportion of new patients achieving SCROT at 24-, 36-, and 48-month FU was 10% (38/375), 9.5% (31/327), and 12% (30/250), respectively, including 23 who underwent splenectomy. AYAs achieving SCROT between 12 and 36 months displayed higher platelet counts in the first year (excluding the initial period) and received fewer IV immunoglobulin treatments beyond 12 months compared with those with ongoing disease.
患有免疫性血小板减少症（ITP）的青少年和青年（AYA）表现出独特的临床特征和需求，难以简单归类为成人或儿童。既往研究发现，12个月时慢性病风险为50%，但其长期病程仍不明确。本研究旨在描述慢性原发性ITP的AYA的临床和实验室特征。从3个登记处（慢性ITP儿科和成人登记处、CEREVANCE以及南比利牛斯自身免疫性血细胞减少登记处）提取了12至25岁、病程1年的慢性病患者的数据，涵盖2004年至2021年期间。治疗停药后持续完全缓解（SCROT）定义为血小板计数>100×10⁹/L且至少12个月未接受治疗，与先前的治疗策略无关。共纳入427例慢性原发性ITP的AYA（64%为女性）。在初始诊断时以及6个月和12个月随访时，约100%的患者可获得临床信息；在24、36和48个月时，分别有88%、77%和59%的患者可获得临床信息。随着时间推移，临床特征逐渐改善，需要治疗的患者减少。在整个随访期间，接受治疗的患者二线药物使用稳步增加，但不影响对皮质类固醇和静脉注射免疫球蛋白的需求。在24个月、36个月和48个月随访时达到SCROT的新患者比例分别为10%（38/375）、9.5%（31/327）和12%（30/250），其中23例接受了脾切除术。与疾病持续的患者相比，在12至36个月达到SCROT的AYA在第一年（不包括初始期）血小板计数更高，且12个月后接受静脉注射免疫球蛋白治疗的次数更少。
