Centre de Référence National des Cytopénies Auto-immunes de l'Enfant (CEREVANCE), Bordeaux, France.
Division of Pediatric Hematology-Oncology, Charles-Bruneau Cancer Center, Department of Pediatrics, Sainte-Justine University Hospital, Université de Montréal, Montréal, QC, Canada.
Blood. 2022 Jul 21;140(3):253-261. doi: 10.1182/blood.2022015508.
Splenectomy is effective in ∼70% to 80% of pediatric chronic immune thrombocytopenia (cITP) cases, and few data exist about it in autoimmune hemolytic anemia (AIHA) and Evans syndrome (ES). Because of the irreversibility of the procedure and the lack of predictions regarding long-term outcomes, the decision to undertake splenectomy is difficult in children. We report here factors associated with splenectomy outcomes from the OBS'CEREVANCE cohort, which prospectively includes French children with autoimmune cytopenia (AIC) since 2004. The primary outcome was failure-free survival (FFS), defined as the time from splenectomy to the initiation of a second-line treatment (other than steroids and intravenous immunoglobulins) or death. We included 161 patients (cITP, n = 120; AIHA, n = 19; ES, n = 22) with a median (minimum-maximum) follow-up of 6.8 years (1.0-33.3) after splenectomy. AIC subtype was not associated with FFS. We found that immunopathological manifestations (IMs) were strongly associated with unfavorable outcomes. Diagnosis of an IM before splenectomy was associated with a lower FFS (hazard ratio [HR], 0.39; 95% confidence interval [CI], 0.21-0.72, P = .003, adjusted for AIC subtype). Diagnosis of an IM at any timepoint during follow-up was associated with an even lower FFS (HR, 0.22; 95% CI, 0.12-0.39; P = 2.8 × 10-7, adjusted for AIC subtype) as well as with higher risk of recurrent or severe bacterial infections and thrombosis. In conclusion, our results support the search for associated IMs when considering a splenectomy to refine the risk-benefit ratio. After the procedure, monitoring IMs helps to identify patients with higher risk of unfavorable outcomes.
脾切除术在儿童慢性免疫性血小板减少症（cITP）病例中有效率约为 70%至 80%，而在自身免疫性溶血性贫血（AIHA）和 Evans 综合征（ES）中则很少有相关数据。由于该手术具有不可逆性，且缺乏对长期结果的预测，因此儿童是否进行脾切除术的决策非常困难。我们在此报告了 OBS'CEREVANCE 队列中脾切除术结果的相关因素，该队列自 2004 年以来前瞻性地纳入了法国自身免疫性血细胞减少症（AIC）患儿。主要结局是无失败生存（FFS），定义为从脾切除术到开始二线治疗（除类固醇和静脉注射免疫球蛋白外）或死亡的时间。我们纳入了 161 例患者（cITP，n=120；AIHA，n=19；ES，n=22），脾切除术后的中位（最小-最大）随访时间为 6.8 年（1.0-33.3）。AIC 亚型与 FFS 无关。我们发现免疫病理表现（IMs）与不良结局密切相关。脾切除术前诊断为 IM 与较低的 FFS 相关（风险比[HR]，0.39；95%置信区间[CI]，0.21-0.72，P=0.003，调整了 AIC 亚型）。在随访期间的任何时间点诊断为 IM 与更低的 FFS 相关（HR，0.22；95%CI，0.12-0.39；P=2.8×10-7，调整了 AIC 亚型），且与复发性或严重细菌感染和血栓形成的风险增加相关。总之，我们的结果支持在考虑脾切除术时寻找相关的 IMs，以优化风险效益比。手术后，监测 IMs有助于识别具有不良结局高风险的患者。
