BRIEF SUMMARY OF PRESCRIBING INFORMATION
Nplate® (romiplostim), for subcutaneous injection
WARNINGS AND PRECAUTIONS
Risk of Progression of Myelodysplastic Syndromes to Acute
Myelogenous Leukemia
Progression from myelodysplastic syndromes (MDS) to
acute myelogenous leukemia (AML) has been observed
in clinical trials with Nplate®. A randomized, double-blind,
placebo-controlled trial enrolling patients with severe
thrombocytopenia and International Prognostic Scoring
System (IPSS) low or intermediate-1 risk MDS was terminated
due to more cases of AML observed in the Nplate® treatment
arm. At the time of an interim analysis, among 219 MDS
patients randomized 2:1 to treatment with Nplate® or
placebo (147 Nplate®: 72 placebo), 11 patients showed
progression to AML, including nine on the Nplate® arm versus
two on the placebo arm. In addition, in peripheral blood
counts, the percentage of circulating myeloblasts increased
to greater than 10% in 28 patients, 25 of whom were
in the romiplostim treatment arm. Of the 28 patients
who had an increase in circulating myeloblasts to greater than
10%, eight of these patients were diagnosed to have AML,
and 20 patients had not progressed to AML. In four
patients, increased peripheral blood blast cell counts
decreased to baseline after discontinuation و