Long-term follow-up for up to 5 years on the risk of leukaemic progression in thrombocytopenic patients with lower-risk myelodysplastic syndromes treated with romiplostim or placebo in a randomised double-blind trial

What Was Tested

The paper reports a study of Romiplostim, published as a randomized controlled trial.

Treatment options for thrombocytopenia in myelodysplastic syndromes are scarce.

Who Took Part

PubMed tags the participants as both men and women.

This study is a 5-year follow-up of a phase 2, multicentre, double-blind trial of romiplostim treatment in patients with lower-risk myelodysplastic syndromes.

Eligible patients were recruited at 109 centres in North America, Europe, Russia, and Australia, were aged 18-90 years, and had platelets of 20 × 109 per L or less with or without a history of bleeding or 50 × 109 platelets per L or less with a history of bleeding.

How the Study Was Run

The researchers set it up as a randomized, double-blind, placebo-controlled and multicenter study.

Because people in this Romiplostim study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

Blinding on both sides kept the participants and researchers unaware of who received Romiplostim.

The comparison arm used a placebo, so changes seen with Romiplostim could be set against what happens without it.

Running at several centres spread the Romiplostim study over different clinics and patient groups.

The primary outcomes for this long-term follow-up were survival and progression to acute myeloid leukaemia.

The primary outcome was assessed per protocol in all patients with available data.

This study is registered with ClinicalTrials.gov, NCT00614523.

What Was Measured

Topics PubMed links to this paper include Disease Progression, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Platelet Count, Prognosis, Receptors, Fc and Recombinant Fusion Proteins.

What This Means in Plain Language

The paper is a randomized, double-blind and placebo-controlled study of Romiplostim.

The abstract goes on to report its results in about 3 sentences; this page does not restate them, so read the paper for the findings and their limits.

The publication is from 2018 (The Lancet. Haematology), a journal based in England.

More context: the Romiplostim compound page, all human trials.