TY - JOUR
T1 - Update on Pediatric Cancer Surveillance Recommendations for Patients with Neurofibromatosis Type 1, Noonan Syndrome, CBL Syndrome, Costello Syndrome, and Related RASopathies
AU - Perrino, Melissa R.
AU - Das, Anirban
AU - Scollon, Sarah R.
AU - Mitchell, Sarah G.
AU - Greer, Mary Louise C.
AU - Yohe, Marielle E.
AU - Hansford, Jordan R.
AU - Kalish, Jennifer M.
AU - Schultz, Kris Ann P.
AU - MacFarland, Suzanne P.
AU - Kohlmann, Wendy K.
AU - Lupo, Philip J.
AU - Maxwell, Kara N.
AU - Pfister, Stefan M.
AU - Weksberg, Rosanna
AU - Michaeli, Orli
AU - Jongmans, Marjolijn C.J.
AU - Tomlinson, Gail E.
AU - Brzezinski, Jack
AU - Tabori, Uri
AU - Ney, Gina M.
AU - Gripp, Karen W.
AU - Gross, Andrea M.
AU - Widemann, Brigitte C.
AU - Stewart, Douglas R.
AU - Woodward, Emma R.
AU - Kratz, Christian P.
N1 - Publisher Copyright:
© 2024 American Association for Cancer Research.
PY - 2024/11/1
Y1 - 2024/11/1
N2 - Neurofibromatosis type 1 (NF1), Noonan syndrome, and related syndromes, grouped as RASopathies, result from dysregulation of the RAS-MAPK pathway and demonstrate varied multisystemic clinical phenotypes. Together, RASopathies are among the more prevalent genetic cancer predisposition syndromes and require nuanced clinical management. When compared with the general population, children with RASopathies are at significantly increased risk of benign and malignant neoplasms. In the past decade, clinical trials have shown that targeted therapies can improve outcomes for low-grade and benign neoplastic lesions but have their own challenges, highlighting the multidisciplinary care needed for such individuals, specifically those with NF1. This perspective, which originated from the 2023 American Association for Cancer Research Childhood Cancer Predisposition Workshop, serves to update pediatric oncologists, neurologists, geneticists, counselors, and other health care professionals on revised diagnostic criteria, review previously published surveillance guidelines, and harmonize updated surveillance recommendations for patients with NF1 or RASopathies.
AB - Neurofibromatosis type 1 (NF1), Noonan syndrome, and related syndromes, grouped as RASopathies, result from dysregulation of the RAS-MAPK pathway and demonstrate varied multisystemic clinical phenotypes. Together, RASopathies are among the more prevalent genetic cancer predisposition syndromes and require nuanced clinical management. When compared with the general population, children with RASopathies are at significantly increased risk of benign and malignant neoplasms. In the past decade, clinical trials have shown that targeted therapies can improve outcomes for low-grade and benign neoplastic lesions but have their own challenges, highlighting the multidisciplinary care needed for such individuals, specifically those with NF1. This perspective, which originated from the 2023 American Association for Cancer Research Childhood Cancer Predisposition Workshop, serves to update pediatric oncologists, neurologists, geneticists, counselors, and other health care professionals on revised diagnostic criteria, review previously published surveillance guidelines, and harmonize updated surveillance recommendations for patients with NF1 or RASopathies.
UR - http://www.scopus.com/inward/record.url?scp=85208249297&partnerID=8YFLogxK
U2 - 10.1158/1078-0432.CCR-24-1611
DO - 10.1158/1078-0432.CCR-24-1611
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C2 - 39196581
AN - SCOPUS:85208249297
SN - 1078-0432
VL - 30
SP - 4834
EP - 4843
JO - Clinical Cancer Research
JF - Clinical Cancer Research
IS - 21
ER -
