Pediatric autoimmune hemolytic anemia is associated with a high incidence of underlying immune disorders

Pediatric autoimmune hemolytic anemia (AIHA) is a heterogeneous disease with significant morbidity due to the underlying condition and its treatment. Evidence-based guidelines for evaluation and management are lacking. Data from 399 patients with AIHA followed at 15 pediatric centers were collected to identify factors associated with secondary diagnoses, recurrent/chronic course, therapeutic efficacy, and mortality. Most had AIHA associated with secondary diagnoses including Evans syndrome (37%, 142/385), other autoimmunity (22%, 86/392), and inborn errors of immunity (IEI, 18%, 68/379). Of 305 patients tested, 82% had abnormal functional immune results. Genetic testing for an IEI was sent in 31% (109/348) with pathogenic findings identified in 32% of those tested. Patients with IEI or other autoimmunity more frequently had abnormal immunoglobulin and complement testing. Prevalence of IEI was not different between those presenting with or without infection. The median number of treatments for the first AIHA episode was 2 (range: 0-17). Of those with warm AIHA, 31% received steroid-sparing therapy during the first episode. Patients with recurrent AIHA (42%) had a higher rate of abnormal immune tests (OR=2.29, p=0.012), Evans syndrome (OR= 4.85; p<0.001), IEI (OR=3.88, p<0.001), and other autoimmune disorders (OR=3.29; p<0.001). With median follow up of 4.9 years (range: 0-19.4 years), 72/257 (28%) with warm AIHA continued to have active disease on treatment. Of the 399 patients, 10 died, all of whom had secondary diagnoses. Expansive immune evaluation, monitoring, and targeted treatments directed at immune diagnoses are needed for pediatric AIHA, highlighting the need for evidence-based pediatric AIHA guidelines.

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Journal
Blood Advances
Published
2026-07-02
DOI
https://doi.org/10.1182/bloodadvances.2026020023
Primary Topic
Blood groups and transfusion
Type
article
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article

Pediatric autoimmune hemolytic anemia is associated with a high incidence of underlying immune disorders

Sanjay Shah, Olivia Noe, Caitlin Montcrieff, Wendy B. London et al.
Blood Advances
Blood groups and transfusion
article

Pediatric autoimmune hemolytic anemia is associated with a high incidence of underlying immune disorders

Sanjay Shah, Olivia Noe, Caitlin Montcrieff, Wendy B. London, MacGregor Steele, John Leister, Cassandra J. Everly, Shilpa Nataraj, Joshua A Kalter, Jennifer Rothman, Rachael F. Grace, Michele P. Lambert, Dana Ghanem, Emily Harris, Hannah Elkus, Kristin A. Shimano, Sherif M. Badawy, Sureka Pavalagantharajah, Stephanie FritchLilla, Stacey Rifkin-Zenenberg, Kirsty Hillier, Gerald C. Tiu, Caitlyn McComb, Ellis Bloom, Robert J. Klaassen, Jessica Chumsky, Danielle Charland, Tatiana Kalashnikova, Allison Sarah Remiker, Nan Chen, Taizo A. Nakano, Manpreet Kochhar, Lia Phillips, Aila Khan, Erika Barbosa Semedo Tavares, Vicky Breakey, Alison Matsunaga, Russell Pierce Valle
article en

Abstract

Pediatric autoimmune hemolytic anemia (AIHA) is a heterogeneous disease with significant morbidity due to the underlying condition and its treatment. Evidence-based guidelines for evaluation and management are lacking. Data from 399 patients with AIHA followed at 15 pediatric centers were collected to identify factors associated with secondary diagnoses, recurrent/chronic course, therapeutic efficacy, and mortality. Most had AIHA associated with secondary diagnoses including Evans syndrome (37%, 142/385), other autoimmunity (22%, 86/392), and inborn errors of immunity (IEI, 18%, 68/379). Of 305 patients tested, 82% had abnormal functional immune results. Genetic testing for an IEI was sent in 31% (109/348) with pathogenic findings identified in 32% of those tested. Patients with IEI or other autoimmunity more frequently had abnormal immunoglobulin and complement testing. Prevalence of IEI was not different between those presenting with or without infection. The median number of treatments for the first AIHA episode was 2 (range: 0-17). Of those with warm AIHA, 31% received steroid-sparing therapy during the first episode. Patients with recurrent AIHA (42%) had a higher rate of abnormal immune tests (OR=2.29, p=0.012), Evans syndrome (OR= 4.85; p<0.001), IEI (OR=3.88, p<0.001), and other autoimmune disorders (OR=3.29; p<0.001). With median follow up of 4.9 years (range: 0-19.4 years), 72/257 (28%) with warm AIHA continued to have active disease on treatment. Of the 399 patients, 10 died, all of whom had secondary diagnoses. Expansive immune evaluation, monitoring, and targeted treatments directed at immune diagnoses are needed for pediatric AIHA, highlighting the need for evidence-based pediatric AIHA guidelines.

Blood Advances
Children's Hospital of Wisconsin (US), Boston Children's Hospital (US), Lurie Children's Hospital (US), Hackensack University Medical Center (US), Children's Hospital of Philadelphia (US), Long Island University (US), Palo Alto University (US), University of California, San Francisco (US), Medical College of Wisconsin (US), Children's Hospital of Eastern Ontario (CA), University of Minnesota System (US), Alberta Children's Hospital (CA), Children's Hospital Colorado (US), Hasbro Children's Hospital (US), Dana-Farber Cancer Institute (US), Phoenix Children's Hospital (US), Lurie Children's Hospital (US), Duke Medical Center (US), Children's Minnesota (US), UCSF Benioff Children's Hospital (US), Children's Hospital at Montefiore (US), Boston Children's Museum (US), Duke University Hospital (US), Dana-Farber/Boston Children's Cancer and Blood Disorders Center (US), Robert H. Lurie Comprehensive Cancer Center of Northwestern University, University of Colorado Denver (US), Stanford University (US), McMaster University (CA)
Good health and well-being
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Blood groups and transfusion
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