Adult Immune (Idiopathic) Thrombocytopenic Purpura (ITP) is a chronic and potentially serious autoimmune disorder characterized by low platelet counts in the blood. In it, platelets are destroyed by the patient's own immune system. ITP has historically been considered a disease of platelet destruction; however, recent data also suggest that the most prominent defect is the body's inability to compensate for platelet destruction by increasing production. Newer approaches to treating ITP fous on increasing platelet production. The U.S. Food and Drug Administration has approved Nplate (romiplostim) in August 2008. This drug is the first product that directly stimulates the bone marrow to produce needed platelets in patients with a rare blood disorder that can lead to serious bleeding. Nplate revealed a number of adverse reactions, from bone-marrow abnormalities to dangerous blood clots. A Risk Evaluation and Mitigation Strategy (REMS) has been developed to address the risks of Nplate therapy. Under the Food and Drug Administration Amendments Act of 2007, FDA has determined that a REMS is necessary for the benefits of Nplate to outweigh the risks of the product. The REMS will include a Medication Guide for patients and requires that all prescribers and patients enroll in a special program to track the long term safety of Nplate therapy.
Where a splenectomy was not performed, a recent study presented at the 14th congress of the European Hematology Association from a study comparing romiplostim (Nplate®) to the medical standard of care (SOC) in non-splenectomised patients with chronic immune thrombocytopenic purpura (ITP).
The study involved 234 adults with chronic ITP, who were treated with either romiplostim (n=157) or medical SOC (n=77); the latter was prescribed by the investigator according to standard institutional practices or therapeutic guidelines; the only treatments not allowed were investigational agents or other thrombopoietic agents. A total of 13 (8%) patients who received romiplostim and 27 (35%) of those in the SOC group underwent splenectomy or discontinued the study prior to reporting a splenectomy. Furthermore, 12% and 27% experienced treatment failure (platelet counts ≤20,000 platelets/microL for four consecutive weeks, a major bleeding event, and/or a change in therapy due to intolerable side effects or bleeding symptoms) or discontinued the study, respectively.
Where a splenectomy was not performed, a recent study presented at the 14th congress of the European Hematology Association from a study comparing romiplostim (Nplate®) to the medical standard of care (SOC) in non-splenectomised patients with chronic immune thrombocytopenic purpura (ITP).
The study involved 234 adults with chronic ITP, who were treated with either romiplostim (n=157) or medical SOC (n=77); the latter was prescribed by the investigator according to standard institutional practices or therapeutic guidelines; the only treatments not allowed were investigational agents or other thrombopoietic agents. A total of 13 (8%) patients who received romiplostim and 27 (35%) of those in the SOC group underwent splenectomy or discontinued the study prior to reporting a splenectomy. Furthermore, 12% and 27% experienced treatment failure (platelet counts ≤20,000 platelets/microL for four consecutive weeks, a major bleeding event, and/or a change in therapy due to intolerable side effects or bleeding symptoms) or discontinued the study, respectively.
http://www.fda.gov/cder/foi/label/2008/125268lbl.pdf
Prescribing information
Nplate™ is only available through the Nplate™ NEXUS (Network of Experts Understanding and Supporting Nplate™ and patients) Program. This program is designed to promote informed risk-benefit decisions before initiating treatment and while patients are on treatment to assure appropriate use of Nplate™ in patients with chronic ITP who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. Nplate™ should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding. It should not be used in an attempt to normalize platelet counts. Nplate™ is not indicated for the treatment of thrombocytopenia due to myelodysplastic syndrome (MDS) or any cause of thrombocytopenia other than chronic ITP.
http://www.fda.gov/cder/foi/label/2008/125268lbl.pdf
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Kuter DJ, Bussel JB, Lyons RM, et al. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: A double-blind randomised controlled trial. Lancet. 2008;371(9610):395-403.
- Stasi R, Evangelista ML, Amadori S. Novel thrombopoietic agents: A review of their use in idiopathic thrombocytopenic purpura. Drugs. 2008;68(7):901-912.
- Tiu RV, Sekeres MA. The role of AMG-531 in the treatment of thrombocytopenia in idiopathic thrombocytopenic purpura and myelodysplastic syndromes. Expert Opin Biol Ther. 2008;8(7):1021-1030.
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