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

AbstractBACKGROUND:
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.
AuthorsDavid J Kuter, James B Bussel, Roger M Lyons, Vinod Pullarkat, Terry B Gernsheimer, Francis M Senecal, Louis M Aledort, James N George, Craig M Kessler, Miguel A Sanz, Howard A Liebman, Frank T Slovick, J Th M de Wolf, Emmanuelle Bourgeois, Troy H Guthrie Jr, Adrian Newland, Jeffrey S Wasser, Solomon I Hamburg, Carlos Grande, François Lefrère, Alan Eli Lichtin, Michael D Tarantino, Howard R Terebelo, Jean-François Viallard, Francis J Cuevas, Ronald S Go, David H Henry, Robert L Redner, Lawrence Rice, Martin R Schipperus, D Matthew Guo, Janet L Nichol
JournalLancet (London, England) (Lancet) Vol. 371 Issue 9610 Pg. 395-403 (Feb 2 2008) ISSN: 1474-547X [Electronic] England
PMID18242413 (Publication Type: Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)
Chemical References
  • Carrier Proteins
  • Receptors, Fc
  • Recombinant Fusion Proteins
  • romiplostim
  • Thrombopoietin
Topics
  • Adult
  • Aged
  • Carrier Proteins (administration & dosage, adverse effects, therapeutic use)
  • Chronic Disease (drug therapy)
  • Double-Blind Method
  • Drug Administration Schedule
  • Female
  • Humans
  • Male
  • Middle Aged
  • Platelet Count
  • Purpura, Thrombotic Thrombocytopenic (blood, drug therapy, immunology)
  • Receptors, Fc (administration & dosage, therapeutic use)
  • Recombinant Fusion Proteins
  • Splenectomy
  • Thrombopoietin
  • Treatment Outcome

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