Pediatric autoimmune hemolytic anemia is associated with a high incidence of underlying immune disorders
Year: 2026
Type: article
Study design: Observational Study
Abstract: 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 (142/385 [37%]), other autoimmunity (86/392 [22%]), and inborn errors of immunity (IEI; 68/379 [18%]). Of 305 patients tested, 82% had abnormal functional immune results. Genetic testing for IEI was performed in 109 of 348 patients (31%), 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 and 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 (odds ratio [OR], 2.29; P = .012), Evans syndrome (OR, 4.85; P< .001), IEI (OR, 3.88; P< .001), and other autoimmune disorders (OR, 3.29; P< .001). With a median follow-up of 4.9 years (range, 0-19.4), 72 of 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.
Source: Blood Advances
Authors: Emily M. Harris, MacGregor Steele, Tatiana Kalashnikova, Sherif M. Badawy, Sureka Pavalagantharajah, Kirsty Hillier, Robert J. Klaassen, Joshua A. Kalter, Jennifer A. Rothman, Caitlyn McComb, Sanjay J. Shah, Kristin A. Shimano, Ellis J. Bloom, Aila Khan, Hannah Elkus, Vicky R. Breakey, Stephanie A. Fritch Lilla, John Leister, Manpreet Kochhar, Olivia Young, Lia Phillips, Jessica Chumsky, Dana Ghanem, Danielle Charland, Taizo A. Nakano, Allison Remiker, Cassandra J. Everly, Alison Matsunaga, Gerald C. Tiu, Russell P. Valle, Shilpa Nataraj, Stacey Rifkin-Zenenberg, Erika Tavares, Caitlin Montcrieff, Nan Chen, Wendy B. London, Michele P. Lambert, Rachael F. Grace
Institutions: Harvard University, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, University of Calgary, Alberta Children's Hospital, Northwestern University, Lurie Children's Hospital, McMaster University, NYU Langone Health, New York University, Children's Hospital of Eastern Ontario, Children's Hospital Colorado, Duke University, Duke Medical Center, Phoenix Children's Hospital, University of Arizona College of Medicine- Phoenix, University of California, San Francisco, UCSF Benioff Children's Hospital, Children's Hospital of Philadelphia, Children's Minnesota, University of Minnesota, Brown University, Hasbro Children's Hospital, NYU Grossman Long Island School of Medicine, Medical College of Wisconsin, Hackensack Meridian Health
Language: English
Subfield: Immunology
Field: Immunology and Microbiology
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $3,800
Funders: Amgen, Bristol-Myers Squibb, Sanofi, F. Hoffmann-La Roche, Medpace, Memorial Sloan-Kettering Cancer Center, Takeda Pharmaceuticals U.S.A., Vertex Pharmaceuticals, Agios Pharmaceuticals, bluebird bio, Argenx, Horizon Therapeutics, Novo Nordisk, Swedish Orphan Biovitrum, National Institutes of Health, Bayer Canada
OpenAlex ID: W7167013771