The systemic administration of intravenous melphalan.

Area of Science:

• Oncology
• Pharmacology

Background:

• Melphalan (L-phenylalanine mustard) is an alkylating agent used orally for breast cancer, ovarian cancer, and multiple myeloma.
• Intravenous (IV) melphalan is available in Europe and Canada but remains investigational in the US.
• The role and efficacy of IV melphalan in cancer chemotherapy require further definition.

Purpose of the Study:

• To evaluate the activity and toxicity of IV melphalan in various malignancies.
• To compare IV melphalan administration with oral melphalan.
• To explore the potential of both lower and higher doses of IV melphalan in cancer treatment.

Main Methods:

• Review of existing clinical data on IV melphalan administration.
• Evaluation of IV melphalan at lower doses (30-70 mg/m2) as a single agent and in combination.
• Assessment of high-dose IV melphalan ( > 140 mg/m2) with bone marrow reinfusion.

Main Results:

• IV melphalan demonstrated striking activity in previously untreated rhabdomyosarcoma patients.
• High-dose IV melphalan achieved high response rates in nonhematologic tumors, including resistant types like melanoma and colon carcinoma.
• High-dose melphalan regimens improved response rates and survival in poor-prognosis patients with non-Hodgkin's lymphoma, Hodgkin's disease, multiple myeloma, and neuroblastoma.

Conclusions:

• IV melphalan exhibits significant activity across various cancers, particularly rhabdomyosarcoma.
• High-dose IV melphalan offers survival benefits for specific poor-prognosis hematologic malignancies.
• Further clinical trials are essential to fully define the therapeutic potential and optimal patient selection for IV melphalan.