Immune Checkpoint Inhibitors: A Pediatric Needs Assessment

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Objectives Immune checkpoint inhibitor (ICI) therapy is increasing in pediatric oncology. Immune-related adverse events (irAEs) can affect various organ systems, including rheumatic-irAEs (Rh-irAEs) such as inflammatory arthritis, myositis, sicca syndrome, systemic lupus erythematosus, sarcoidosis, and vasculitis.[1] Few cases report detail Rh-irAEs and their management in the pediatric population.[2] Our objective was to assess the familiarity of pediatric rheumatologists (PR) worldwide with ICI-induced Rh-irAEs, gauge their confidence managing these conditions, and identify knowledge gaps to guide future educational efforts. Methods We circulated a 21-question online survey to 2084 PR via the “Dr. Peter Dent Pediatric Rheumatology Bulletin Board.” Responses were collected from June 2024 to September 2024. We collected data on practitioner demographics, knowledge of ICIs and Rh-irAEs, confidence in managing Rh-irAEs, and preferred educational resources. Results Sixty-nine responses were received of which 55 (80%) were PR from academic centers, and 9 (13%) were from community practices (Table 1). 24 (35%) had >20 years of clinical experience. Despite global distribution, 56 (81%) of responses came from North America. 34 (49%) of respondents were not aware of ICIs and their related mechanisms, indications, and side effects, and 40 (58%) were not familiar with irAEs. 55 (80%) had never managed a patient with Rh-irAEs. Among those who had (14/69), the median number of cases managed was 2.75 (IQR 1.75). Confidence in managing these conditions was limited: 39 (57%) were “not confident at all” managing Rh-irAEs, 34 (49%) were “not confident at all” managing pre-existing autoimmune diseases (PAD) in ICI users, and 46 (67%) were “not confident at all” advising oncology colleagues on initiating or discontinuing ICIs in the context of Rh-irAEs or PADs. No one felt “completely confident” managing these conditions. Several knowledge gaps were identified: 59 (86%) in long-term management, 55 (80%) in acute management, and 51 (74%) in recognition and diagnosis. 43 (62%) indicated the need for pediatric-specific clinical guidelines. Of the 14 (20%) respondents with clinical experience treating Rh-irAEs, initial treatment approaches varied, with 4/14 (29%) using NSAIDs, 3/14 (21%) using prednisone, and 4/14 (29%) combining prednisone with methotrexate. Long-term management also varied, with 5/14 (36%) using methotrexate, and 3/14 (21%) using TNF inhibitors. Table 1: Respondent Demographics, Knowledge & Confidence Assessment Conclusion Significant knowledge gaps and a lack of confidence exist among PR in managing ICI-related Rh-irAEs. As ICI use increases in pediatric oncology, PR exposure to Rh-irAEs will follow. Targeted educational programs and clinical guidelines will be valuable to address these gaps and improve patient care. [1.] Ghosh N. Rheum Dis Clin North Am 2022; 48(2):411-28. [2.] Storwick JA. Pediatr Rheumatol Online J 2024;22(1):49.

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