# Melphalan (including hepatic delivery system)

Source: https://onco.cc/drugs/melphalan/  
OnCo record `melphalan` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

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.

## Summary

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.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-08
- Tags: gap-fill; generic
- Brand: Alkeran / Evomela / Hepzato Kit
- Modality: Alkylating agent (nitrogen mustard)
- Mechanism: 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.
- Approvals: 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)

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Melphalan
- Label: Hepzato Kit (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Hepzato
- Label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=melphalan

## Connected records

- cancers: [Diffuse large B-cell lymphoma](https://onco.cc/cancers/dlbcl/), [High-risk neuroblastoma](https://onco.cc/cancers/neuroblastoma-high-risk/), [Multiple myeloma](https://onco.cc/cancers/multiple-myeloma/), [Newly diagnosed multiple myeloma, transplant-eligible](https://onco.cc/cancers/myeloma-transplant-eligible/), [Newly diagnosed multiple myeloma, transplant-ineligible](https://onco.cc/cancers/myeloma-transplant-ineligible/), [Ovarian cancer](https://onco.cc/cancers/ovarian/), [Peripheral T-cell lymphomas (including cutaneous T-cell lymphoma)](https://onco.cc/cancers/peripheral-t-cell-lymphoma/), [Plasma cell leukaemia](https://onco.cc/cancers/plasma-cell-leukaemia/), [Relapsed and refractory classical Hodgkin lymphoma](https://onco.cc/cancers/relapsed-refractory-hodgkin-lymphoma/), [Retinoblastoma](https://onco.cc/cancers/retinoblastoma/), [Uveal melanoma](https://onco.cc/cancers/uveal-melanoma/)
- technologies: [Autologous stem cell transplant (high-dose therapy)](https://onco.cc/technologies/autologous-stem-cell-transplant/), [Cytotoxic chemotherapy](https://onco.cc/technologies/cytotoxic-chemotherapy/), [Isolated limb perfusion and infusion](https://onco.cc/technologies/isolated-limb-perfusion/), [Oral cryotherapy (ice chips) to prevent mucositis](https://onco.cc/technologies/oral-cryotherapy-mucositis/), [Percutaneous hepatic perfusion (chemosaturation)](https://onco.cc/technologies/percutaneous-hepatic-perfusion/)
- trials: [A Study of Melphalan With or Without Siltuximab in People With Multiple Myeloma Having an Autologous Stem Cell Transplant](https://onco.cc/trials/nct06679829/), [An Open-Label Expanded Access Study of the Melphalan/Hepatic Delivery System (HDS) in Patients With Hepatic Dominant Ocular Melanoma](https://onco.cc/trials/nct05022901/), [BMT CTN 0803](https://onco.cc/trials/bmt-ctn-0803/), [DETERMINATION](https://onco.cc/trials/determination/), [Evaluation of the Safety and Efficacy of Treatment w/High Dose Melphalan Given Directly Into the Liver Followed by Treatment w/Approved Cancer Treatme](https://onco.cc/trials/nct06607458/), [FOCUS](https://onco.cc/trials/nct02678572/), [High-Risk Neuroblastoma Study 2 of SIOP-Europa-Neuroblastoma (SIOPEN)](https://onco.cc/trials/nct04221035/), [Panobinostat, Gemcitabine Hydrochloride, Busulfan, and Melphalan Before Stem Cell Transplant in Treating Patients With Refractory or Relapsed Multiple Myeloma](https://onco.cc/trials/nct02506959/)
- terms: [Alkylating agents and antimetabolites](https://onco.cc/terms/alkylating-agent/), [Alkylating agents and therapy-related myeloid neoplasms](https://onco.cc/terms/rejuv-second-alkylating-agents-and-myeloid-neoplasms/), [Autologous stem cell transplant (ASCT)](https://onco.cc/terms/autologous-transplant/), [Choices made at the time of treatment that change the second cancer risk](https://onco.cc/terms/rejuv-second-choices-made-at-treatment/), [Conditioning regimen (myeloablative, reduced-intensity)](https://onco.cc/terms/conditioning-regimen/), [Fertility before lymphoma treatment: what to ask for, and when](https://onco.cc/terms/lymphoma-tx-fertility-preservation/), [Platinum drugs and PARP inhibitors: the newer leukaemia risk](https://onco.cc/terms/rejuv-second-platinum-and-parp-inhibitors/), [Stem cell transplant in lymphoma: what it is still for](https://onco.cc/terms/lymphoma-tx-transplant-role/)
- drugs: [Tasonermin](https://onco.cc/drugs/tasonermin/)
- companies: [Acrotech Biopharma](https://onco.cc/companies/acrotech-biopharma/)

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