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PMID | (32923911) | ||||||||||||
Authors | Ortiz MV, Gerdemann U, Raju SG, Henry D, Smith S, Rothenberg SM, Cox MC, Proust S, Bender JG, Frazier AL, Anderson P, Pappo AS | ||||||||||||
Title | Activity of the Highly Specific RET Inhibitor Selpercatinib (LOXO-292) in Pediatric Patients With Tumors Harboring RET Gene Alterations. | ||||||||||||
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Abstract Text |
Molecular Profile | Treatment Approach |
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Gene Name | Source | Synonyms | Protein Domains | Gene Description | Gene Role |
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Therapy Name | Drugs | Efficacy Evidence | Clinical Trials |
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Drug Name | Trade Name | Synonyms | Drug Classes | Drug Description |
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Gene | Variant | Impact | Protein Effect | Variant Description | Associated with drug Resistance |
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RET | D378_G385delinsE | indel | unknown | RET D378_G385delinsE results in a deletion of eight amino acids within the extracellular domain of the Ret protein from amino acid 378 to 385, combined with the insertion of a glutamic acid (E) at the same location (UniProt.org). D378_G385delinsE has been identified in the scientific literature (PMID: 32923911), but has not been biochemically characterized and therefore, its effect on Ret protein function is unknown (PubMed, Aug 2024). |
Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
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RET M918T | medullary thyroid carcinoma | sensitive | Selpercatinib | Case Reports/Case Series | Actionable | In a clinical case study, a pediatric patient with previously treated metastatic medullary thyroid cancer harboring germ line RET M918T mutation achieved stable disease and remained on treatment after 5.2 months with Retevmo (selpercatinib) therapy (PMID: 32923911). | 32923911 |
RET D378_G385delinsE | medullary thyroid carcinoma | predicted - sensitive | Selpercatinib | Case Reports/Case Series | Actionable | In a clinical case study, a pediatric patient with heavily pretreated medullary thyroid cancer harboring RET D378_G385delinsE achieved a partial response after 8 weeks of Retevmo (selpercatinib) treatment, with an 86% reduction in tumor size after 40 weeks, and remained progression-free after 27 months (PMID: 32923911; NCT03157128). | 32923911 |