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The myeloma chemotherapy that is also the standard high-dose conditioning before autologous transplant, and, delivered directly into the liver's blood supply or the eye, treats uveal melanoma metastases and retinoblastoma.
Approved 1964 (oral, myeloma; melphalan-prednisone was the standard for decades). High-dose melphalan 200 mg/m² with autologous stem-cell rescue remains standard consolidation in myeloma (IFM 2009, DETERMINATION). Hepzato Kit (2023): percutaneous hepatic perfusion for uveal melanoma liver metastases (FOCUS trial, 36% response). Intra-arterial and intravitreal melphalan are standard in retinoblastoma. Melflufen is a peptide-conjugated prodrug.
Bifunctional alkylator producing DNA interstrand cross-links; active in myeloma at conventional dose and as high-dose conditioning; regional delivery for eye melanoma liver metastases and retinoblastoma.
1.Melphalan (including hepatic delivery system) slips into a pocket on its target.
Oral anticancer drug with an injectable equivalent used for the same indication, so Medicare covers the oral form under Part B (the oral anticancer drug benefit) as well as under Part D; the pharmacy bills whichever applies. High-dose IV melphalan for transplant conditioning is inpatient (Part A). The Hepzato Kit (hepatic delivery) is a hospital procedure with its own coding.
Multi-source generic; covered under the medical benefit without prior authorisation in most plans, as part of standard regimens. Hepzato Kit requires prior authorisation.
Sources: Medicare.gov: Chemotherapy · Medicare.gov: Prescription drugs (outpatient, Part B) · Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.
Sources: NICE search: melphalan. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
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| Region | Year | Indication |
|---|---|---|
| US | 1964 | Multiple myeloma; non-resectable epithelial ovarian carcinoma |
| US | 2016 | High-dose conditioning before autologous HSCT in myeloma (Evomela) |
| US | 2023 | Uveal melanoma with unresectable hepatic metastases via hepatic delivery system (Hepzato Kit) |
Read for the class this product belongs to (other alkylating drugs) unless the answer names the product. Population figures from the cohorts named, not a prediction for one person. Blank where no source gives a recovery figure.
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Query for this drug: (TITLE:"Melphalan" OR ABSTRACT:"Melphalan" OR TITLE:"including hepatic delivery system" OR ABSTRACT:"including hepatic delivery system" OR TITLE:"Alkeran" OR ABSTRACT:"Alkeran" OR TITLE:"Evomela" OR ABSTRACT:"Evomela" OR TITLE:"Hepzato Kit" OR ABSTRACT:"Hepzato Kit") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Melphalan (including hepatic delivery system), not a curated reading list.
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