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Molecular Profile ALK act mut
Therapy Cyclophosphamide + Lorlatinib + Topotecan
Indication/Tumor Type neuroblastoma
Response Type sensitive

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
ALK act mut neuroblastoma sensitive Cyclophosphamide + Lorlatinib + Topotecan Phase I Actionable In a Phase I trial, the combination of Lorbrena (lorlatinib), Topotecan, and Cytoxan (cyclophosphamide) treatment was well tolerated and resulted in a modified response rate of 63% (5/8) in pediatric patients with neuroblastoma harboring an ALK activating mutation, including ALK F1174C, F1174L, R1275Q, or R1275L (PMID: 37012551; NCT03107988). 37012551
PubMed Id Reference Title Details
(37012551) Lorlatinib with or without chemotherapy in ALK-driven refractory/relapsed neuroblastoma: phase 1 trial results. Full reference...