Immune checkpoint inhibitors and the pediatric rheumatologist: a pediatric needs assessment
Abstract Background The use of immune checkpoint inhibitor (ICI) therapy is increasing in pediatric oncology. ICIs can cause rheumatic-immune related adverse events (Rh-irAEs) such as inflammatory arthritis and myositis. Few case reports detail Rh-irAEs and their management in the pediatric populati...
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BMC
2025-07-01
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| Series: | Pediatric Rheumatology Online Journal |
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| Online Access: | https://doi.org/10.1186/s12969-025-01127-x |
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| author | John Storwick Carrie Ye Shahin Jamal Nancy Maltez Mercedes Chan |
| author_facet | John Storwick Carrie Ye Shahin Jamal Nancy Maltez Mercedes Chan |
| author_sort | John Storwick |
| collection | DOAJ |
| description | Abstract Background The use of immune checkpoint inhibitor (ICI) therapy is increasing in pediatric oncology. ICIs can cause rheumatic-immune related adverse events (Rh-irAEs) such as inflammatory arthritis and myositis. Few case reports detail Rh-irAEs and their management in the pediatric population. Our objective was to assess the familiarity of pediatric rheumatologists (PRs) worldwide with Rh-irAEs, gauge confidence in managing these conditions, and identify knowledge gaps to guide future educational efforts. Methods We circulated an online survey to 2084 PRs 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 participants responded, of which 55 (80%) were PRs from academic centers. Despite global distribution, 56 (81%) responses came from North America. Thirty-four (49%) respondents were not aware of ICIs and their related mechanisms, indications, and side effects, and 40 (58%) were not familiar with irAEs. Fifty-five (80%) had never managed a patient with Rh-irAEs. Among those who had (14/69, 21%), the median number of cases managed was 2.0 (IQR 0.0). Thirty-nine respondents were “not confident at all” managing Rh-irAEs, 34 were “not confident at all” managing pre-existing autoimmune diseases (PAD) in ICI users, and 46 were “not confident at all” advising oncology colleagues on initiating or discontinuing ICIs in the context of Rh-irAEs or pre-existing autoimmune diseases (PAD). No respondents felt “completely confident” managing these conditions. Participants identified knowledge gaps in long-term management, acute management, and recognition and diagnosis. Forty-three indicated the need for pediatric-specific clinical guidelines. Of the 14 respondents with clinical experience treating Rh-irAEs, treatment varied, with 4 using nonsteroidal anti-inflammatory drugs, 3 using prednisone, and 4 combining prednisone with methotrexate. Long-term management also varied, with 5 using methotrexate, and 3 using tumor necrosis factor inhibitors. Conclusions Significant knowledge gaps and a lack of confidence exist among PRs managing ICI-related Rh-irAEs. As ICI use increases in pediatric oncology, PRs’ exposure to Rh-irAEs will follow. Targeted educational programs and clinical guidelines may be valuable to address these gaps. |
| format | Article |
| id | doaj-art-4e5aafa4ee5048c7b659866322764b76 |
| institution | Kabale University |
| issn | 1546-0096 |
| language | English |
| publishDate | 2025-07-01 |
| publisher | BMC |
| record_format | Article |
| series | Pediatric Rheumatology Online Journal |
| spelling | doaj-art-4e5aafa4ee5048c7b659866322764b762025-08-20T03:46:07ZengBMCPediatric Rheumatology Online Journal1546-00962025-07-012311810.1186/s12969-025-01127-xImmune checkpoint inhibitors and the pediatric rheumatologist: a pediatric needs assessmentJohn Storwick0Carrie Ye1Shahin Jamal2Nancy Maltez3Mercedes Chan4Division of Pediatric Rheumatology, British Columbia Children’s Hospital, The University of British ColumbiaDepartment of Medicine, University of AlbertaDepartment of Medicine, University of British Columbia, Arthritis Research CanadaDepartment of Medicine, University of OttawaDivision of Pediatric Rheumatology, British Columbia Children’s Hospital, The University of British ColumbiaAbstract Background The use of immune checkpoint inhibitor (ICI) therapy is increasing in pediatric oncology. ICIs can cause rheumatic-immune related adverse events (Rh-irAEs) such as inflammatory arthritis and myositis. Few case reports detail Rh-irAEs and their management in the pediatric population. Our objective was to assess the familiarity of pediatric rheumatologists (PRs) worldwide with Rh-irAEs, gauge confidence in managing these conditions, and identify knowledge gaps to guide future educational efforts. Methods We circulated an online survey to 2084 PRs 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 participants responded, of which 55 (80%) were PRs from academic centers. Despite global distribution, 56 (81%) responses came from North America. Thirty-four (49%) respondents were not aware of ICIs and their related mechanisms, indications, and side effects, and 40 (58%) were not familiar with irAEs. Fifty-five (80%) had never managed a patient with Rh-irAEs. Among those who had (14/69, 21%), the median number of cases managed was 2.0 (IQR 0.0). Thirty-nine respondents were “not confident at all” managing Rh-irAEs, 34 were “not confident at all” managing pre-existing autoimmune diseases (PAD) in ICI users, and 46 were “not confident at all” advising oncology colleagues on initiating or discontinuing ICIs in the context of Rh-irAEs or pre-existing autoimmune diseases (PAD). No respondents felt “completely confident” managing these conditions. Participants identified knowledge gaps in long-term management, acute management, and recognition and diagnosis. Forty-three indicated the need for pediatric-specific clinical guidelines. Of the 14 respondents with clinical experience treating Rh-irAEs, treatment varied, with 4 using nonsteroidal anti-inflammatory drugs, 3 using prednisone, and 4 combining prednisone with methotrexate. Long-term management also varied, with 5 using methotrexate, and 3 using tumor necrosis factor inhibitors. Conclusions Significant knowledge gaps and a lack of confidence exist among PRs managing ICI-related Rh-irAEs. As ICI use increases in pediatric oncology, PRs’ exposure to Rh-irAEs will follow. Targeted educational programs and clinical guidelines may be valuable to address these gaps.https://doi.org/10.1186/s12969-025-01127-xImmune checkpoint inhibitorsIrAEICINeeds assessmentPediatricsRheumatic immune related adverse events |
| spellingShingle | John Storwick Carrie Ye Shahin Jamal Nancy Maltez Mercedes Chan Immune checkpoint inhibitors and the pediatric rheumatologist: a pediatric needs assessment Pediatric Rheumatology Online Journal Immune checkpoint inhibitors IrAE ICI Needs assessment Pediatrics Rheumatic immune related adverse events |
| title | Immune checkpoint inhibitors and the pediatric rheumatologist: a pediatric needs assessment |
| title_full | Immune checkpoint inhibitors and the pediatric rheumatologist: a pediatric needs assessment |
| title_fullStr | Immune checkpoint inhibitors and the pediatric rheumatologist: a pediatric needs assessment |
| title_full_unstemmed | Immune checkpoint inhibitors and the pediatric rheumatologist: a pediatric needs assessment |
| title_short | Immune checkpoint inhibitors and the pediatric rheumatologist: a pediatric needs assessment |
| title_sort | immune checkpoint inhibitors and the pediatric rheumatologist a pediatric needs assessment |
| topic | Immune checkpoint inhibitors IrAE ICI Needs assessment Pediatrics Rheumatic immune related adverse events |
| url | https://doi.org/10.1186/s12969-025-01127-x |
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