641 - Immune Checkpoint Inhibitors: a Pediatric Needs Assessment

Publication Number: 641.5276

John A. Storwick, University of British Columbia Faculty of Medicine, Vancouver, BC, Canada; Carrie Ye, University of Alberta, Edmonton, AB, Canada; Shahin Jamal, University of British Columbia Faculty of Medicine, Vancouver, BC, Canada; Nancy Maltez, University of Ottawa Faculty of Medicine, Ottawa, ON, Canada; Mercedes O. Chan, University of British Columbia Faculty of Medicine, Vancouver, BC, Canada

Background: Immune checkpoint inhibitors (ICIs) are new therapies being increasingly used 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 case reports detail Rh-irAEs and their management in the pediatric population [2].

Objective: This study aimed to assess pediatric rheumatologists' (PR) familiarity worldwide with ICI-induced Rh-irAEs, their confidence in managing these cases, and existing knowledge gaps to guide future education.

Design/Methods: A 21-question online survey was distributed to 2084 PRs via the "Dr. Peter Dent Pediatric Rheumatology Bulletin Board" from June 2024 to September 2024. Data collected included practitioner demographics, knowledge and confidence in managing Rh-irAEs, and preferred educational resources.

Results: Of 69 responses, 55 (80%) were PR from academic centers. 24 (35%) respondents had >20 years of clinical experience; despite global distribution, 56 (81%) were from North America. (Table 1).
34 respondents (49%) lacked awareness of ICIs' mechanisms and side effects, and 40 (58%) were unfamiliar with irAEs. Most PRs (80%) had never managed a Rh-irAE case; for those with experience (14/69), the median number of cases was 2.75 (IQR 1.75). Confidence was low, with 39 (57%) being “not confident at all” in managing Rh-irAEs, 34 (49%) being “not confident at all” in managing pre-existing autoimmune diseases (PAD) in ICI users, and 46 (67%) being “not confident at all” in advising oncology colleagues on initiating or discontinuing ICIs in the context of Rh-irAEs or PADs. No respondent felt “completely confident” managing these conditions. Knowledge gaps were notable: 59 (86%) in long-term management, 55 (80%) in acute management, and 51 (74%) in diagnosis and recognition. Furthermore, 43 (62%) highlighted a need for pediatric-specific guidelines. Among 14 respondents with Rh-irAE experience, initial treatments varied: 4/14 (29%) used NSAIDs, 3 /14 (21%) prednisone, and 4/14 (29%) combined prednisone with methotrexate. Long-term management also varied.

Conclusion(s): Notable knowledge gaps and low confidence exist among PRs in managing ICI-related Rh-irAEs. As ICI use increases in pediatric oncology, academic pediatricians and pediatric rheumatologists exposure to Rh-irAEs will increase. Targeted educational programs and clinical guidelines will be valuable to address these gaps, improve Rh-irAE identification in pediatric practices, and improve patient care.

Table 1: Respondent Demographics, Knowledge, and Confidence Assessment