In Phase I, the maximum tolerated dose was determined as 2.97 mg/m2 and recommended Phase II dose was selected as 2.23 mg/m2, which was later converted to a 4.0 mg/m2 dose based on encouraging results related to the body's ability to tolerate the drug. In Phase II, the investigators observed an overall response rate of 92 percent with 55 percent of patients reaching VGPR or better, including 23 percent with a complete remission. As the treatment cycles progressed, the rate and depth of response increased.
Minor adverse events, such as fatigue, nausea, and rash, were noted in approximately 40 percent of patients. Serious adverse events were minimal and primarily consisted of decreased blood counts, nausea and vomiting, diarrhea, rash, and electrolyte disturbances. One patient died from pneumonia while on treatment, and seven patients discontinued treatment due to different side effects.
"As targeted therapies continue to evolve, we are now shifting our efforts to focus on making them safer and producing them in more convenient forms for patients. MLN9708 is a great example of how to accomplish this goal," said Shaji K. Kumar, MD, lead author and Professor of Medicine and Consultant in Hematology at the Mayo Clinic in Rochester, Minn. "We are now planning ongoing studies to examine this drug in combination with other myeloma drugs in Phase II and Phase III clinical trials. Once approved for treatment of myeloma, this drug will al
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American Society of Hematology