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MELPHALAN HYDROCHLORIDE
At a glance
| Generic name | MELPHALAN HYDROCHLORIDE |
|---|---|
| Modality | Small molecule |
| Phase | FDA-approved |
| First approval | 1992 |
Approved indications
Boxed warnings
- WARNING Melphalan should be administered under the supervision of a qualified physician experienced in the use of cancer chemotherapeutic agents. Severe bone marrow suppression with resulting infection or bleeding may occur. Controlled trials comparing intravenous (IV) to oral melphalan have shown more myelosuppression with the IV formulation. Hypersensitivity reactions, including anaphylaxis, have occurred in approximately 2% of patients who received the IV formulation. Melphalan is leukemogenic in humans. Melphalan produces chromosomal aberrations in vitro and in viv o and, therefore, should be considered potentially mutagenic in humans.
Common side effects
- Acute hypersensitivity reactions including anaphylaxis
- Bone marrow suppression
- Leukopenia
- Thrombocytopenia
- Anemia
- Nausea and vomiting
- Diarrhea
- Oral ulceration
- Urticaria
- Pruritus
- Edema
- Skin rashes
Serious adverse events
- Irreversible bone marrow failure
- Hepatic veno-occlusive disease
- Cardiac arrest
- Pulmonary fibrosis with fatal outcomes
- Interstitial pneumonitis
- Skin necrosis requiring skin grafting
- Hepatitis
- Jaundice
- Vasculitis
- Hemolytic anemia
Key clinical trials
- Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma (PHASE3)
- Ibrutinib Before and After Stem Cell Transplant in Treating Patients With Relapsed or Refractory Diffuse Large B-cell Lymphoma (PHASE3)
- Venetoclax or Placebo in Combination With Reduced-Intensity Conditioning Hematopoietic Cell (Bone Marrow/Blood Stem Cell) Transplant and as Maintenance Therapy After Transplant in Patients With Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial) (PHASE2)
- MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) (PHASE2)
- Intra-Arterial Chemotherapy for Newly Diagnosed, Residual, or Recurrent Atypical Choroid Plexus Papilloma and Choroid Plexus Carcinoma Prior to Second-Look Surgery (PHASE1)
- Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases (PHASE1, PHASE2)
- Hepzato Kit and Opdualag for Metastatic Melanoma and Liver Metastasis (PHASE1, PHASE2)
- Auto Transplant High Dose Melphalan vs High Dose Melphalan+Bortezomib in Pts With Multiple Myeloma Age 65 Years or Older (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |