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

Summary 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...

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Veröffentlicht in:	The Lancet (British edition) 2008-02, Vol.371 (9610), p.395-403
Hauptverfasser:	Kuter, David J, Prof, Bussel, James B, MD, Lyons, Roger M, MD, Pullarkat, Vinod, MD, Gernsheimer, Terry B, MD, Senecal, Francis M, MD, Aledort, Louis M, MD, George, James N, MD, Kessler, Craig M, MD, Sanz, Miguel A, MD, Liebman, Howard A, MD, Slovick, Frank T, MD, de Wolf, J Th M, MD, Bourgeois, Emmanuelle, MD, Guthrie, Troy H, MD, Newland, Adrian, MA, Wasser, Jeffrey S, MD, Hamburg, Solomon I, MD, Grande, Carlos, MD, Lefrère, François, MD, Lichtin, Alan Eli, MD, Tarantino, Michael D, MD, Terebelo, Howard R, DO, Viallard, Jean-François, MD, Cuevas, Francis J, MD, Go, Ronald S, MD, Henry, David H, MD, Redner, Robert L, MD, Rice, Lawrence, MD, Schipperus, Martin R, MD, Guo, D Matthew, PhD, Nichol, Janet L, MS
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Aged Blood diseases Carrier Proteins - administration & dosage Carrier Proteins - adverse effects Carrier Proteins - therapeutic use Chronic Disease - drug therapy Chronic illnesses Clinical trials Double-Blind Method Drug Administration Schedule Drug therapy Female Humans Internal Medicine Male Medical treatment Middle Aged Platelet Count Purpura, Thrombotic Thrombocytopenic - blood Purpura, Thrombotic Thrombocytopenic - drug therapy Purpura, Thrombotic Thrombocytopenic - immunology Receptors, Fc - administration & dosage Receptors, Fc - therapeutic use Recombinant Fusion Proteins Splenectomy Thrombopoietin Treatment Outcome
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Zusammenfassung:	Summary 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 30×109 /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 50×109 /L to 200×109 /L. The primary objectives were to assess the efficacy of romiplostim as measured by a durable platelet response (platelet count ≥50×109 /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
ISSN:	0140-6736 1474-547X
DOI:	10.1016/S0140-6736(08)60203-2