ORIGINAL RESEARCH article
Front. Immunol.
Sec. Primary Immunodeficiencies
This article is part of the Research TopicDiseases with Immune Dysregulation in AfricaView all 11 articles
Predictive factors for severity and poor treatment response in children with Evans syndrome: A retrospective cohort study
Provisionally accepted
Monia BEN KHALED1,2*
Marwa Ben Ayed1,2
Zaid Zaroui1,2Ilhem Ben Fraj1,2,3Samia Rekaya1,2,3
NAJLA MEKKI4Yosra Chebbi3,5
Ben Taieb Aicha1,2Amani Merdassi1,2Ikram Zaiter1,2Takwa Lamouchi1,2Ridha Kouki2Houda Kaabi6Hamida Slama6Wafa Achour3,5
Mohamed Bejaoui1,2Fethi Mellouli1,2
Imen Ben-Mustapha4Monia Ouederni1,2- 1Faculty of Medicine, Tunis El Manar University, Tunis, Tunisia
- 2Pediatric Immuno-Hematology Department, Bone Marrow Transplantation Center of Tunis, Tunis, Tunisia., Tunis, Tunisia
- 3Laboratory of Child and Immunocompromised Microbiology", LR18ES39, Faculty of Medicine of Tunis, University of Tunis El Manar, Tunisia, Tunis, Tunisia
- 4Laboratory of Transmission, Control and Immunobiology of Infections, LR16IPT02, Institut Pasteur de Tunis, University of Tunis El Manar, Tunis, Tunisia
- 5Laboratory Department, Bone Marrow Transplantation Center of Tunis, Tunis, Tunisia
- 6National Center of Blood Transfusion, Tunis, Tunisia
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Evans syndrome (ES) is a rare disorder characterized by autoimmune cytopenias affecting multiple blood cell lineages. In children, management remains particularly challenging due to the absence of clear guidelines for acute treatment and escalation to second-line therapies. We conducted a retrospective, longitudinal study (2010-2024) including pediatric patients (<18 years) with ES to identify predictors of severe presentation, need for second-line therapy, and fatal outcome. Predictors were identified using Cox Regression. Fifty patients were included (sequential = 27, concomitant = 23), with a median age at diagnosis of 4.1 years (IQR:1.5– 8.5). Secondary ES was observed in 41(82%) cases, among which 38 (76%) had IEI. Severe clinical presentation at diagnosis occurred in 50% of patients and was independently associated with age < 24months and hemoglobin level < 80g/L. Corticosteroid dependence was observed in 34 cases (68%), with second-line therapy required in 31 patients (62%, cumulative risk=88%). This was associated with the presence of hepatomegaly and abnormal IgM levels. At last follow-up, 38(76%) patients were in remission and 19(38%) had relapsed. Fatal outcome (8 patients) was associated with age < 24 months at diagnosis (p=0.04), family history of IEI (p=10⁻³), splenomegaly (p=0.02), and hepatomegaly (p=0.05). Pediatric ES is therefore a severe condition particularly in infants. Outcomes are strongly influenced by underlying immune dysregulation, highlighting the need for early etiological evaluation to guide timely and appropriate therapeutic strategies including the early use of targeted or curative approaches.
Keywords: autoimmune cytopenias, Evans syndrome, Immune dysregulation, immunosuppressive therapy, Pediatrics, predictors, Primary Immunodeficiencies, Survival
Received: 08 Sep 2025; Accepted: 09 Feb 2026.
Copyright: © 2026 BEN KHALED, Ben Ayed, Zaroui, Ben Fraj, Rekaya, MEKKI, Chebbi, Aicha, Merdassi, Zaiter, Lamouchi, Kouki, Kaabi, Slama, Achour, Bejaoui, Mellouli, Ben-Mustapha and Ouederni. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Monia BEN KHALED
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