Results of the first open-label study comparing romiplostim treatment with standard- of-care therapies in nonsplenectomized adult patients with chronic immune thrombocytopenia (ITP) show that both the incidence of treatment failure and need for splenectomy were reduced among romiplostim-treated patients.
David J. Kuter, MD, DPhil, of Massachusetts General Hospital, Boston, and his associates compared the incidence of treatment failure and the incidence of splenectomy, the primary end points, in 157 patients with ITP who received weekly subcutaneous injections of romiplostim and 77 patients who received the standard of care. The findings are reported in the November 11, 2010 issue of the New England Journal of Medicine.
Of 157 patients who received romiplostim, 18 (11%) experienced treatment failure compared with 23 (30%) of 77 standard-of-care patients. The incidence of splenectomy was also significantly lower with romiplostim versus the standard of care (9% vs 36%).
Significant differences were found in secondary end points too. Patients receiving romiplostim experienced a significantly longer time to splenectomy than did SOC-treated patients, had a higher mean platelet count, and a 2.3 times greater platelet response rate.
Patients in the romiplostim group also had a significantly lower incidence of bleeding events, required fewer blood transfusions, and reported greater improvements in quality of life.
Adverse events associated with romiplostim treatment were similar to those in previous studies, were generally mild or moderate in severity, and did not result in treatment discontinuation. Headache and fatigue were the most commonly reported adverse events. Serious adverse events occurred in 23% of romiplostim-treated patients compared with 37% of those receiving the SOC.
"Romiplostim may offer the potential for long-term effective treatment in patients who wish either to avoid or defer a splenectomy," commented coauthor Mathias Rummel, MD, of the Hospital of the Justus-Liebig University, Giessen, Germany.