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Therapy Name Busulfan + CPX-351 + Fludarabine + Melphalan + Midostaurin
Synonyms
Therapy Description

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Drug Name Trade Name Synonyms Drug Classes Drug Description
Busulfan Busulfex Busulphan Busulfex (busulfan) inhibits DNA replication and RNA transcription, likely through induction of DNA alkylation (PMID: 27481448). Busulfex (busulfan) in combination with cyclophosphamide is FDA approved as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for patients with chronic myelogenous leukemia (FDA.gov).
CPX-351 Vyxeos Liposome-encapsulated Daunorubicin-Cytarabine Vyxeos (CPX-351) is a liposomal formulation of cytarabine and daunorubicin with anti-tumor activities (PMID: 25223583). Vyxeos (CPX-351) is FDA approved for use in patients with newly-diagnosed therapy-related acute myeloid leukemia (AML) or AML with myelodysplasia-related changes (FDA.gov).
Fludarabine Fludara FAMP|Fludarabine phosphate Flurdara (fludarabine) is converted to 2-fluoro-ara-ATP intracellularly, which potentially inhibits DNA polymerase alpha, ribonucleotide reductase and DNA primase, leading to decreased DNA synthesis and reduced tumor growth (NCI Drug Dictionary)
Melphalan Alkeran Chemotherapy - Alkylating 18 Alkeran (melphalan) is an antineoplastic alkylating agent, which cross-links DNA and induces cell toxicity and is FDA approved for multiple myeloma and epithelial ovarian carcinoma (FDA.gov).
Midostaurin Rydapt PKC412|CGP 41251 CSF1R Inhibitor 28 FLT3 Inhibitor 69 KIT Inhibitor 57 PKC alpha Inhibitor 6 PKC beta Inhibitor 6 PKC Inhibitor (Pan) 11 VEGFR2 Inhibitor 37 Rydapt (midostaurin) is a multi-kinase inhibitor with activity against FLT3, KIT, PDGFRB, KDR (VEGFR2) and PKC, with higher selectivity for conventional PKC isoforms, which induces cell cycle arrest and apoptosis (PMID: 12124173, PMID: 23127174, PMID: 15914319). Rydapt (midostaurin) is FDA approved for FLT3-mutant AML in combination with chemotherapy, and is approved for use in aggressive systemic mastocytosis, systemic mastocytosis with associated hematological neoplasm, or mast cell leukemia as a single therapy (FDA.gov).

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References

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Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT04982354 Phase Ib/II Busulfan + CPX-351 + Fludarabine + Melphalan + Midostaurin Induction Therapy for Patients With FLT3 Mutated Acute Myeloid Leukemia Withdrawn USA 0


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