Systemic Inflammatory Diseases in Children With Sickle Cell Disease: A French Multicenter Observational Study on Diagnostic and Therapeutic Issues

IF 2.3 3区医学 Q2 HEMATOLOGY Pediatric Blood & Cancer Pub Date : 2025-01-27 DOI:10.1002/pbc.31563

Caroline Vinit, Corinne Guitton, Mariane De Montalembert, Patricia Benhaim, Lahoueri Amor-Chelihi, Brigitte Bader-Meunier, Florence Missud, Isabelle Melki, Vincent Gajdos, Cécile Arnaud, Annie Kamden, Oussama Charara, Véronique Hentgen, Sylvie Nathanson, Coralie Bloch, Ulrich Meinzer, Pierre Quartier, Isabelle Kone-Paut, Loïc De Pontual, Luu-Ly Pham

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Abstract

Background

Systemic inflammatory diseases (SIDs) have been reported in patients with sickle cell disease (SCD), but clinical data in children are scarce.

Objectives

To identify clinical and laboratory features at diagnosis of SID in children with SCD and to describe their evolution.

Methods

Data from children with SCD and SIDs were retrospectively collected in a French multicenter study from 1991 to 2018. Information included clinical characteristics, inflammatory markers, autoantibodies patterns, treatments, and complications. Inflammatory marker levels were compared at SID diagnosis and at the last follow-up. Statistical analyses were performed using Cran R software.

Results

Among a cohort of 3800 children with SCD, 43 SIDs were identified in 35 study participants: autoimmune liver disease (AILD, n = 13), inflammatory bowel disease (IBD, n = 7), juvenile idiopathic arthritis (JIA, n = 6), systemic lupus erythematosus (n = 4), autoimmune hemolytic anemia (n = 3), Sjögren syndrome (n = 1), histiocytic necrotizing lymphadenitis (n = 2), vasculitis (n = 2), myasthenia gravis (n = 1), sarcoidosis (n = 1), idiopathic inflammatory granulomatous uveitis (n = 1), mixed connective tissue disease (n = 2). Prevalence of SID was 0.9% in our cohort of children with SCD. The median time between initial symptoms and SID diagnosis was 10 (3–20) months, notably longer in children with JIA, IBD, and Sjögren syndrome. Sixteen patients (46%) exhibited hypergammaglobulinemia (>20 g/L) at diagnosis. No significant differences were observed for other inflammatory parameters. Twenty-one children (60%) received systemic steroids and 13 (37%) biological therapies. Three patients (9%) underwent hematopoietic stem cell transplantation. Nine patients (26%) had severe infections; one died.

Conclusion

Delayed diagnosis was frequent due to overlapping clinical presentations between SCD and SID. Clinicians must be aware of warning signs associated with elevated inflammatory markers, hypergammaglobulinemia, or specific antibodies. Therapeutic strategies remain challenging.

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镰状细胞病儿童的全身性炎性疾病：一项法国多中心的诊断和治疗观察研究

背景：镰状细胞病（SCD）患者有全身性炎症性疾病（SIDs）的报道，但儿童的临床资料很少。目的：探讨SCD患儿SID诊断的临床和实验室特征，并描述其演变过程。方法：回顾性收集1991年至2018年法国一项多中心研究中SCD和SIDs患儿的数据。信息包括临床特征、炎症标志物、自身抗体模式、治疗和并发症。比较SID诊断时和最后一次随访时的炎症标志物水平。采用Cran R软件进行统计分析。结果：在3800名患有SCD的儿童队列中，35名研究参与者中发现了43名SIDs：自身免疫性肝病（AILD， 13例）、炎症性肠病（IBD， 7例）、幼年特发性关节炎（JIA， 6例）、系统性红斑狼疮（4例）、自身免疫性溶血性贫血（3例）、Sjögren综合征（1例）、组织细胞坏死性淋巴结炎（2例）、血管炎（2例）、重症肌无力（1例）、结节病（1例）、特发性炎性肉芽肿性葡萄膜炎（1例）、混合结缔组织病（n = 2）。在我们的SCD患儿队列中，SID的患病率为0.9%。从初始症状到SID诊断的中位时间为10（3-20）个月，JIA、IBD和Sjögren综合征患儿的时间更长。16例患者（46%）在诊断时表现为高γ -球蛋白血症（bbb20 g/L）。其他炎症参数无显著差异。21名儿童（60%）接受了全身类固醇治疗，13名儿童（37%）接受了生物治疗。3例（9%）患者接受了造血干细胞移植。严重感染9例（26%）；人死亡。结论：SCD与SID临床表现重叠，常导致诊断延误。临床医生必须意识到与炎症标志物升高、高γ球蛋白血症或特异性抗体相关的警告信号。治疗策略仍然具有挑战性。

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来源期刊

Pediatric Blood & Cancer 医学-小儿科

CiteScore

4.90

自引率

9.40%

发文量

546

审稿时长

1.5 months

期刊介绍： Pediatric Blood & Cancer publishes the highest quality manuscripts describing basic and clinical investigations of blood disorders and malignant diseases of childhood including diagnosis, treatment, epidemiology, etiology, biology, and molecular and clinical genetics of these diseases as they affect children, adolescents, and young adults. Pediatric Blood & Cancer will also include studies on such treatment options as hematopoietic stem cell transplantation, immunology, and gene therapy.