Outcome of Children and Adolescents With Relapsed/Refractory/Progressive Malignancies Treated With Molecularly Informed Targeted Drugs in the Pediatric Precision Oncology Registry INFORM.
Anna-Elisa HeipertzKristian W PajtlerElke PfaffKathrin SchrammMirjam Blattner-JohnsonTill MildeBarbara C JonesCecilia ZulianiCaroline HutterOlli LohiAntonios KattamisIwona Dachowska-KalwakAnna NilssonNicolas U GerberKarin P S LangenbergBianca F GoemansChristian Michel ZwaanJan J MolenaarNatalie JägerUta DirksenRuth WittStefan M PfisterDavid T W JonesDominik T SchneiderOlaf WittCornelis M van TilburgPublished in: JCO precision oncology (2023)
Patients with respective molecular alterations benefit from treatment with ALK, NTRK, and BRAF inhibitors as previously described. No survival benefit was observed with MTT for mutations in the MEK, CDK, other kinase, or mTOR signaling pathways. The noninterventional character of a registry has to be taken into account when interpreting these data and underlines the need for innovative interventional biomarker-driven clinical trials in pediatric oncology.
Keyphrases
- clinical trial
- palliative care
- signaling pathway
- pi k akt
- acute lymphoblastic leukemia
- multiple sclerosis
- acute myeloid leukemia
- diffuse large b cell lymphoma
- cell proliferation
- multiple myeloma
- advanced non small cell lung cancer
- cancer therapy
- randomized controlled trial
- machine learning
- combination therapy
- young adults
- phase ii
- drug delivery
- free survival
- data analysis
- epidermal growth factor receptor
- deep learning
- childhood cancer
- double blind