Paediatric Clinical Immunology, Pellegrin Hospital, CIC1401, INSERM CICP, Bordeaux University Hospital, Bordeaux, France.
Centre de Référence National des Cytopénies Auto-immunes de l'Enfant, Bordeaux, France.
Blood. 2024 Apr 18;143(16):1576-1585. doi: 10.1182/blood.2023021884.
Autoimmune cytopenia (AIC) in children may be associated with positive antinuclear antibodies (ANA) and may progress to systemic lupus erythematosus (SLE). We evaluated the risk of progression to SLE of childhood-onset ANA-associated AIC. In the French national prospective OBS'CEREVANCE cohort, the long-term outcome of children with ANA-associated AIC (ANA titer ≥1/160) and a subgroup of children who developed SLE were described. ANA were positive in 355 of 1803 (20%) children with AIC. With a median follow-up of 5.8 (range, 0.1-29.6) years, 79 of 355 (22%) patients developed SLE at a median age of 14.5 (1.1-21.4) years; 20% of chronic immune thrombocytopenic purpura, 19% of autoimmune hemolytic anemia, and 45% of Evans syndrome. None of the patients with ANA-negative test developed SLE. Severe manifestations of SLE were observed in 21 patients, and 2 patients died. In multivariate analysis including patients with positive ANA within the first 3 months after AIC diagnosis, age >10 years at AIC diagnosis (relative risk [RR], 3.67; 95% confidence interval [CI], 1.18-11.4; P = .024) and ANA titer >1/160 (RR, 5.28; 95% CI, 1.20-23.17; P = .027) were associated with the occurrence of SLE after AIC diagnosis. ANA-associated AIC is a risk factor for progression to SLE, especially in children with an initial ANA titer >1/160 and an age >10 years at AIC diagnosis. ANA screening should be recommended in children with AIC, and patients with ANA should be monitored long-term for SLE, with special attention to the transition period. This trial was registered at www.ClinicalTrials.gov as #NCT05937828.
儿童自身免疫性血细胞减少症 (AIC) 可能与抗核抗体 (ANA) 阳性有关，并可能进展为系统性红斑狼疮 (SLE)。我们评估了儿童发病时与 ANA 相关的 AIC 进展为 SLE 的风险。在法国全国前瞻性 OBS'CEREVANCE 队列中，描述了 ANA 相关 AIC（ANA 滴度≥1/160）儿童的长期结局和发展为 SLE 的儿童亚组。在 1803 例 AIC 儿童中，355 例（20%）ANA 阳性。中位随访时间为 5.8 年（范围 0.1-29.6），355 例患者中有 79 例（22%）在中位年龄 14.5 岁（1.1-21.4 岁）时发展为 SLE；20%的为慢性免疫性血小板减少性紫癜，19%的为自身免疫性溶血性贫血，45%的为 Evans 综合征。没有任何检测结果为 ANA 阴性的患者发展为 SLE。21 例患者出现严重 SLE 表现，2 例死亡。在包括 AIC 诊断后 3 个月内 ANA 阳性的患者在内的多变量分析中，年龄>10 岁（相对风险 [RR]，3.67；95%置信区间 [CI]，1.18-11.4；P=0.024）和 ANA 滴度>1/160（RR，5.28；95% CI，1.20-23.17；P=0.027）与 AIC 诊断后 SLE 的发生相关。ANA 相关 AIC 是进展为 SLE 的危险因素，尤其是在初始 ANA 滴度>1/160 和 AIC 诊断时年龄>10 岁的儿童中。应建议对 AIC 患儿进行 ANA 筛查，对 ANA 阳性患儿应进行 SLE 的长期监测，特别注意过渡期。该试验在 ClinicalTrials.gov 上注册为 #NCT05937828。
