Prognostic factors with high-dose melphalan for refractory multiple myeloma

Area of Science:

• Hematology
• Oncology
• Clinical Pharmacology

Background:

• Advanced multiple myeloma often exhibits resistance to standard chemotherapy regimens.
• Existing treatments like melphalan-prednisone and VAD (vincristine-adriamycin-dexamethasone) have limitations in refractory cases.

Purpose of the Study:

• To evaluate the efficacy and safety of high-dose melphalan (HDM) combined with dexamethasone in advanced multiple myeloma.
• To assess HDM's effectiveness in patients refractory to standard treatments.

Main Methods:

• Administered high-dose melphalan (HDM) and dexamethasone to 43 patients with advanced multiple myeloma.
• 36 patients were refractory to standard melphalan-prednisone and VAD chemotherapy.
• Analyzed response rates, complete remission, and survival based on patient performance status and renal function.

Main Results:

• 44% of patients achieved >75% tumor mass reduction, with 3 complete remissions.
• Response rates varied: 56% in relapsing, 50% in primary drug-resistant, and 23% in unresponsive patients.
• High early mortality (30%) occurred in patients with poor performance or impaired renal function.
• Selected patients with good performance and low lactic dehydrogenase showed a median survival of 18 months vs. 3 months for others (P < .001).

Conclusions:

• High-dose melphalan (HDM) can overcome resistance to standard chemotherapy and VAD in advanced multiple myeloma.
• Timely administration of HDM benefits selected patients, particularly those with favorable prognostic markers.
• Careful patient selection is crucial due to the treatment's significant toxicity and associated mortality.