ZUG, Switzerland, June 10, 2011 /PRNewswire/ --
- Interim Results From Largest Study Of Adult ITP Patients To Date
Amgen today announced interim results from an international, single-arm study ("209 study") evaluating the safety and efficacy of Nplate(R) (romiplostim) in adults with primary immune thrombocytopenia (ITP) - a rare blood disorder - demonstrated that Nplate induced a rapid platelet response with a good safety profile in adult ITP patients with low platelet counts and bleeding symptoms (Abstract #0223).
"This is the largest ever study in adult ITP with over 400 patients enrolled. These interim data highlight again Nplate's ability to treat adult patients successfully, including those with varying severity of the condition," said Dr Ann Janssens, Department of Hematology, University Hospitals Leuven, Belgium. "Nplate has consistently demonstrated that it can significantly increase and maintain platelet counts in adult ITP patients, highlighting its importance as a well-tolerated treatment option."
Interim results from the first 235 patients show: - Nplate was able to induce a platelet response in a large majority of adult patients (90 percent) with primary ITP treated with Nplate for up to 201 weeks. - Median time to response was one week. - Over the course of the study, a doubling of the platelet count to greater than or equal to 50,000 platelets per microliter was achieved by 86 percent of patients who received Nplate. - A platelet count increase of greater than or equal to 20,000 platelets per microliter from baseline was achieved by 91 percent of patients who received Nplate.
Incidence and type of adverse events (AEs) in patients treated with Nplate were consistent with those reported in previous studies. The most common side effects were mild and included headache (28 percent of patients), fatigue (23 percent) and arthralgia (19 percent). No neutralizing antibodies to Nplate or thrombopoietin (TPO) or hematopoieitic malignancies or MDS events were reported.
209 Study Design
This is an open-label, single-arm study of Nplate for the treatment of adults with primary ITP. Nplate was administered once weekly, with dose adjustments to maintain platelet counts of greater than or equal to 50,000 platelets per microliter. The primary study objective was incidence of AEs and antibody formation. Secondary study objectives were to evaluate platelet responses defined as either (1) doubling of baseline count and a platelet count greater than or equal to 50,000 platelets per microliter or (2) a platelet count increase of greater than or equal 20,000 platelets per microliter from baseline.(1) As of June 2009, 235 patients had been treated for a median of 18 weeks with a maximum duration of 201 weeks. Sixty percent of patients had previously undergone splenectomy.
About Adult ITP
In patients with ITP, platelets - blood elements needed to prevent bleeding - are destroyed by the patient's own immune system. Recent data also suggest that low platelet counts in the blood may be caused by the inability of the body's natural processes to produce platelets. Low platelet counts leave adult ITP patients open to sudden serious bleeding events. The risk for serious bleeding events increases when platelet counts drop to less than 30,000 platelets per microliter; normal counts range from 150,000 to 400,000 platelets per microliter. ITP has historically been considered a disease of platelet destruction although recent data suggest that the body's natural platelet production processes in ITP are unable to compensate for low levels of platelets in the blood. Increasing the rate of platelet production may address low platelet levels associated with ITP. Adult chronic ITP has an incidence of 5.8-6.6 per 100,000 in the United States and an estimated 2.0 per 100,000 in the European Union.(2,3)
For more information about ITP please visit itpvillage.com
Nplate is the first platelet producer approved in most regions, including the European Union (EU), Canada, Australia, Russia, Mexico, Switzerland and the U.S. Nplate also has received orphan designation for chronic ITP in the U.S. (2003), the EU (2005), Switzerland (2005), Japan (2006) and Mexico (2010).
Nplate is the first FDA approved treatment specifically for adult chronic ITP. It is also being investigated for potential use in children ages 12 months to 18 years old with persistent severe thrombocytopenia, myelodysplastic syndromes (MDS) and chemotherapy-induced thrombocytopenia (CIT).
In the EU, Nplate is indicated for the treatment of splenectomized adult chronic ITP patients who are refractory to other treatments (e.g. corticosteroids, immunoglobulins). Nplate may be considered as a second-line treatment for adult non-splenectomized ITP patients for whom surgery is contraindicated.
For more information about Nplate, please visit http://www.Nplate.com.
Important EU Nplate Safety Information
The most common side effects are headache, fatigue, arthralgia, myalgia, injection site bruising, injection site pain, oedema peripheral, dizziness, muscle spasms, nausea, contusion, diarrhoea, bone marrow disorder, influenza-like illness, insomnia and pruritus.
Reoccurrence of thrombocytopenia and bleeding after cessation of treatment and increased bone marrow reticulin have been associated with Nplate treatment in the clinical trials. Thrombotic/thromboembolic complications, progression of existing hematopoietic malignancies or MDS, and effects on red and white blood cells are all potential risks associated with Nplate treatment. As with all therapeutic proteins, patients may develop antibodies to the therapeutic protein.
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(1) Data on file. Interim Results from an International Multi-center, Single-arm Study Evaluating the Safety and Efficacy of Romiplostim in Adults with Primary Immune Thrombocytopenia (ITP). Abstract# 0223
(2) McMillan R. Therapy for Adults with Refractory Chronic Immune Thrombocytopenic Purpura. Ann Intern Med 1997;126:307-314
(3) Fogarty PF et al. Curr Opin Hematol 2007;14:515-519.