New insights into childhood autoimmune hemolytic anemia - a French national observational study of 265 children

Q: What outcomes were reported after a median follow-up of 3 years?

After a median follow-up of 3 years, 4% of children died, 28% remained treatment-dependent, and 39% achieved continuous complete remission.

Q: Which direct antiglobulin test results were linked to continuous complete remission survival?

Multivariate analysis associated IgG and IgG+C3d direct antiglobulin tests with a lower rate of survival with continuous complete remission.

Oct 4, 2026

Uploaded by Kurz

Autoimmune hemolytic anemia in children remains rare and incompletely described regarding how it begins and how it evolves. A nationwide French observational study enrolled 265 patients from pediatric hematology units, documenting initial presentation, classification into primary or secondary disease, and outcomes up to last follow-up. Direct antiglobulin test profiles, Evans’ syndrome occurrence, remission and survival relationships were analyzed to identify prognostic subgroups, including children in continuous complete remission.

LanguageEnglish

CategoryHealthcare

FormatPDF

Pages9

Reading Time23 min

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Abstract

Background

Design and Methods

Results

Conclusions

Introduction

New insights into childhood autoimmune hemolytic anemia: a French national observational study of 265 children Nathalie Aladjidi,1 Guy Leverger,2 Thierry Leblanc,3 Marie Quitterie Picat,4 Gérard Michel,5 Yves Bertrand,6 Brigitte Bader-Meunier,7 Alain Robert,8 Brigitte Nelken,9 Virginie Gandemer,10 Hélène Savel,4 Jean Louis Stephan,11 Fanny Fouyssac,12 Julien Jeanpetit,1 Caroline Thomas,13 Pierre Rohr lich,14 André Baruchel,3 Alain Fischer,7 Geneviève Chêne,4 and Y. Perel,1 for the Centre de Référence National des Cytopénies Auto-immunes de l’Enfant (CEREVANCE) 1CHU Bordeaux, Centre de Référence National des Cytopénies Auto-immunes de l’Enfant (CEREVANCE), 2Paris-Trousseau, 3ParisRobert Debré, 5Marseille, 6Lyon, 7Paris-Necker, 8Toulouse, 9Lille, 10Rennes, 11Saint Etienne, 12Nancy, 13Nantes, 14Besançon, 4Unité de Soutien Méthodologique à la Recherche Clinique (USMR) and CIC-EC7, CHU Bordeaux Acknowledgments: the authors wish to thank all patients and families and all CEREVANCE physicians for their participation (cited in appendix). Funding: this work was initiated and financially supported in part by grants from the Association Française pour le Syndrome d’Evans (AFSE). The clinical research and methodology staff were supported by the GIS - Institut des Maladies Rares - INSERM, and the French Health Ministry (Programme Hospitalier de Recherche Clinique 2005, Rare Diseases Plan 2007). Manuscript received on 28 October 2010. Revised version arrived on December 16, 2010. Manuscript accepted on January 3, 2011. Correspondence: Nathalie Aladjidi, MD, Pediatric Hematology Unit, Centre de Référence National des Cytopénies Auto-immunes de l’Enfant (CEREVANCE) Hôpital des Enfants, Hôpital Pellegrin, CHU Bordeaux, Place Amelie Raba Leon, 33 000 Bordeaux, France; Phone: +33.5.57820261; Fax: +33.5.57820279; E-mail: nathalie.aladjidi@chu-bor[deaux.fr](deaux.fr) The online version of this article has a Supplementary Appendix. ABSTRACT Background Autoimmune hemolytic anemia is a rare condition in children. Little is known about its initial presentation and the subsequent progression of the disease. Design and Methods Since 2004, a national observational study has been aiming to thoroughly describe cases and identify prognostic factors. Patients from all French hematologic pediatric units have been included if they had a hemoglobin concentration less than 11 g/dL, a positive direct antiglobulin test and hemolysis. Evans’ syndrome was defined by the association of autoimmune hemolytic anemia and immunological thrombocytopenic purpura. Data from patients' medical records were registered from birth to last follow-up. Autoimmune hemolytic anemia was classified as primary or secondary. Remission criteria, qualifying the status of anemia at last follow-up, were used with the aim of identifying a subgroup with a favorable prognosis in continuous complete remission. Results The first 265 patients had a median age of 3 .8 years at diagnosis. In 74% of cases the direct antiglobulin test was IgG/IgG+C3d. Consanguinity was reported in 8% of cases and first degree familial immunological diseases in 15% of cases. Evans’ syndrome was diagnosed in 37% of cases. Autoimmune hemolytic anemia was post-infectious in 10%, immunological in 53% and primary in 37% of cases. After a median follow-up of 3 years, 4% of children had died, 28% were still treatment-dependent and 39% were in continuous complete remission. In multivariate analysis, IgG and IgG+C3d direct antiglobulin tests were associated with a lower rate of survival with continuous complete remission (adjusted hazard ratio, 0.43; 95% confidence interval, 0.21-0.86) . Conclusions This nationwide French cohort is the largest reported study of childhood autoimmune hemolytic anemia. The rarity of this condition is confirmed. Subgroups with genetic predisposition and underlying immune disorders were identified. Key words: autoimmune hemolytic anemia, children, progn

FAQs

What was the purpose of the national observational study of childhood autoimmune hemolytic anemia?

To thoroughly describe clinical cases and identify prognostic factors in children with autoimmune hemolytic anemia, including disease progression from initial presentation to last follow-up.

How were patients selected and how was autoimmune hemolytic anemia classified?

Patients were included if hemoglobin was < 11 g/dL, the direct antiglobulin test was positive, and hemolysis was present. Autoimmune hemolytic anemia was classified as primary or secondary, and Evans’ syndrome was defined by the association with immunological thrombocytopenic purpura.

What outcomes were reported after a median follow-up of 3 years?

After a median follow-up of 3 years, 4% of children died, 28% remained treatment-dependent, and 39% achieved continuous complete remission.

Which direct antiglobulin test results were linked to continuous complete remission survival?

Multivariate analysis associated IgG and IgG+C3d direct antiglobulin tests with a lower rate of survival with continuous complete remission.

Oct 4, 2026

Uploaded by Kurz