Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Academic Article uri icon



  • BACKGROUND: Chronic immune thrombocytopenic purpura (ITP) is characterised by accelerated platelet destruction and decreased platelet production. Short-term administration of the thrombopoiesis-stimulating protein, romiplostim, has been shown to increase platelet counts in most patients with chronic ITP. We assessed the long-term administration of romiplostim in splenectomised and non-splenectomised patients with ITP. METHODS: In two parallel trials, 63 splenectomised and 62 non-splenectomised patients with ITP and a mean of three platelet counts 30x10(9)/L or less were randomly assigned 2:1 to subcutaneous injections of romiplostim (n=42 in splenectomised study and n=41 in non-splenectomised study) or placebo (n=21 in both studies) every week for 24 weeks. Doses of study drug were adjusted to maintain platelet counts of 50x10(9)/L to 200x10(9)/L. The primary objectives were to assess the efficacy of romiplostim as measured by a durable platelet response (platelet count > or =50x10(9)/L during 6 or more of the last 8 weeks of treatment) and treatment safety. Analysis was per protocol. These studies are registered with ClinicalTrials.gov, numbers NCT00102323 and NCT00102336. FINDINGS: A durable platelet response was achieved by 16 of 42 splenectomised patients given romplostim versus none of 21 given placebo (difference in proportion of patients responding 38% [95% CI 23.4-52.8], p=0.0013), and by 25 of 41 non-splenectomised patients given romplostim versus one of 21 given placebo (56% [38.7-73.7], p<0.0001). The overall platelet response rate (either durable or transient platelet response) was noted in 88% (36/41) of non-splenectomised and 79% (33/42) of splenectomised patients given romiplostim compared with 14% (three of 21) of non-splenectomised and no splenectomised patients given placebo (p<0.0001). Patients given romiplostim achieved platelet counts of 50x10(9)/L or more on a mean of 13.8 (SE 0.9) weeks (mean 12.3 [1.2] weeks in splenectomised group vs 15.2 [1.2] weeks in non-splenectomised group) compared with 0.8 (0.4) weeks for those given placebo (0.2 [0.1] weeks vs 1.3 [0.8] weeks). 87% (20/23) of patients given romiplostim (12/12 splenectomised and eight of 11 non-splenectomised patients) reduced or discontinued concurrent therapy compared with 38% (six of 16) of those given placebo (one of six splenectomised and five of ten non-splenectomised patients). Adverse events were much the same in patients given romiplostim and placebo. No antibodies against romiplostim or thrombopoietin were detected. INTERPRETATION: Romiplostim was well tolerated, and increased and maintained platelet counts in splenectomised and non-splenectomised patients with ITP. Many patients were able to reduce or discontinue other ITP medications. Stimulation of platelet production by romiplostim may provide a new therapeutic option for patients with ITP.


  • Kuter, David J
  • Bussel, James B
  • Lyons, Roger M
  • Pullarkat, Vinod
  • Gernsheimer, Terry B
  • Senecal, Francis M
  • Aledort, Louis M
  • George, James N
  • Kessler, Craig M
  • Sanz, Miguel A
  • Liebman, Howard A
  • Slovick, Frank T
  • de Wolf, J Th M
  • Bourgeois, Emmanuelle
  • Guthrie, Troy H
  • Newland, Adrian
  • Wasser, Jeffrey S
  • Hamburg, Solomon I
  • Grande, Carlos
  • Lefrère, François
  • Lichtin, Alan Eli
  • Tarantino, Michael D
  • Terebelo, Howard R
  • Viallard, Jean-François
  • Cuevas, Francis J
  • Go, Ronald S
  • Henry, David H
  • Redner, Robert L
  • Rice, Lawrence
  • Schipperus, Martin R
  • Guo, D Matthew
  • Nichol, Janet L

publication date

  • February 2, 2008



  • Carrier Proteins
  • Purpura, Thrombotic Thrombocytopenic
  • Receptors, Fc


Scopus Document Identifier

  • 38649120593

Digital Object Identifier (DOI)

  • 10.1016/S0140-6736(08)60203-2

PubMed ID

  • 18242413

Additional Document Info


  • 371


  • 9610