Reference Detail

Ref Type

Journal Article

PMID

(38844796)

Authors

Lau LMS, Khuong-Quang DA, Mayoh C, Wong M, Barahona P, Ajuyah P, Senapati A, Nagabushan S, Sherstyuk A, Altekoester AK, Fuentes-Bolanos NA, Yeung V, Sullivan A, Omer N, Diamond Y, Jessop S, Battaglia L, Zhukova N, Cui L, Lin A, Gifford AJ, Fleuren EDG, Dalla-Pozza L, Moore AS, Khaw SL, Eisenstat DD, Gottardo NG, Wood PJ, Tapp H, Alvaro F, McCowage G, Nicholls W, Hansford JR, Manoharan N, Kotecha RS, Mateos MK, Lock RB, Tyrrell V, Haber M, Trahair TN, Cowley MJ, Ekert PG, Marshall GM, Ziegler DS

Title

Precision-guided treatment in high-risk pediatric cancers.

Journal	Vol	Issue	Date	Pages	Journal Short Title
Nature medicine			2024 Jun 06		Nat Med

URL

Abstract Text

Recent research showed that precision medicine can identify new treatment strategies for patients with childhood cancers. However, it is unclear which patients will benefit most from precision-guided treatment (PGT). Here we report consecutive data from 384 patients with high-risk pediatric cancer (with an expected cure rate of less than 30%) who had at least 18 months of follow-up on the ZERO Childhood Cancer Precision Medicine Program PRecISion Medicine for Children with Cancer (PRISM) trial. A total of 256 (67%) patients received PGT recommendations and 110 (29%) received a recommended treatment. PGT resulted in a 36% objective response rate and improved 2-year progression-free survival compared with standard of care (26% versus 12%; P = 0.049) or targeted agents not guided by molecular findings (26% versus 5.2%; P = 0.003). PGT based on tier 1 evidence, PGT targeting fusions or commenced before disease progression had the greatest clinical benefit. Our data show that PGT informed by comprehensive molecular profiling significantly improves outcomes for children with high-risk cancers. ClinicalTrials.gov registration: NCT03336931.

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Molecular Profile	Treatment Approach

Gene Name	Source	Synonyms	Protein Domains	Gene Description	Gene Role

Therapy Name	Drugs	Efficacy Evidence	Clinical Trials

Drug Name	Trade Name	Synonyms	Drug Classes	Drug Description

Gene	Variant	Impact	Protein Effect	Variant Description	Associated with drug Resistance

Molecular Profile	Indication/Tumor Type	Response Type	Therapy Name	Approval Status	Evidence Type	Efficacy Evidence	References
RET M918T	medullary thyroid carcinoma	sensitive	Selpercatinib	Case Reports/Case Series	Actionable	In a clinical case study, Retevmo (selpercatinib) treatment resulted in a partial response in a pediatric patient with medullary thyroid carcinoma harboring RET M918T (PMID: 38844796; NCT03336931).	38844796
RET L790F	medullary thyroid carcinoma	predicted - sensitive	Selpercatinib	Case Reports/Case Series	Actionable	In a clinical case study, Retevmo (selpercatinib) treatment resulted in a partial response in a pediatric patient with medullary thyroid carcinoma harboring RET L790F (PMID: 38844796; NCT03336931).	38844796
BRAF V600D	ganglioglioma	predicted - sensitive	Dabrafenib + Trametinib	Case Reports/Case Series	Actionable	In a clinical case study, treatment with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in a partial response in a pediatric patient with ganglioglioma harboring BRAF V600D (PMID: 38844796; NCT03336931).	38844796