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