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Long-Term Outcomes Reported With Escalated Melphalan in Multiple Myeloma

By: Celeste L. Dixon
Posted: Tuesday, August 7, 2018

The final outcomes of a trial studying a preparative regimen of escalated melphalan, 280 mg/m2, with amifostine cytoprotection prior to autologous hematopoietic stem cell transplantation for multiple myeloma were “disappointing,” according to researchers in a Bone Marrow Transplantation article. The median progression-free survival of the 57 patients who entered the study between 1999 and 2003 was just 22 months, with a 7-year progression-free survival rate of 10%, noted Parameswaran Hari, MD, MRCP, MS, of the Medical College of Wisconsin, Milwaukee, and colleagues. The 7-year overall survival rate was 39%, with a median overall survival of 67 months.

The administration of this dose of melphalan, significantly higher than the standard 200 mg/m2, was safe and contributed to an overall response rate of 90% at 100 days post treatment. However, the 280 mg/m2 dose used in the trial was preceded by two doses of amifostine as a cytoprotectant. “The possibility that a myeloma stem cell population was protected cannot be excluded,” wrote the authors.

Dr. Hari and colleagues explained that their study took place prior to “more modern transplant studies using melphalan 200,” which can utilize the superior induction and maintenance techniques that are now available. “Recent developments such as the availability of propylene glycol-free melphalan and…of melphalan-based combinations (such as busulfan/melphalan and busulfan/melphalan/bortezomib) indicate that there are many avenues to improve conditioning in myeloma to effect deeper responses,” the team concluded on a hopeful note.



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