�The U.S. Food and Drug Administration approved Nplate (romiplostim), the first merchandise that directly stimulates the bone vegetable marrow to bring forth needed platelets in patients with a rare blood disorder that can jumper lead to serious bleeding.
The condition, which usually develops in adults, is known as chronic immune thrombocytopenic purpura (ITP), a disease that results in a low telephone number of platelets, the blood components that help with clotting. In patients with chronic ITP, the immune system is believed to destroy platelets and the patient's bone marrow is often ineffective to remunerate for this loss.
"This product is important in that it offers a new glide slope to the treatment of patients with an uncommon blood disorder who are often very ill," said Janet Woodcock, M.D., conductor, Center for Drug Evaluation and Research, FDA.
The estimated 140,000 citizenry with chronic ITP are prone to bruising and at risk for life-threatening bleeding. Current medical intervention includes corticosteroids and immune gamma globulin. Surgery to remove the spleen, a procedure known as a splenectomy, may help some patients. Nplate is approved only for patients with chronic ITP who do not respond sufficiently to current treatments.
FDA based its blessing on two randomized clinical trials of about one hundred twenty-five patients world Health Organization had standard at least one prior ITP treatment. One study enrolled patients who still had their spleen, the other enrolled patients wHO did non.
During sise months of treatment, patients who received Nplate had significantly higher platelet counts and kept up those higher counts compared to those who did not have the drug. The response to Nplate was higher in those patients world Health Organization still had their spleen than in those patients who had undergone a splenectomy. In those patients who did not welcome Nplate, but one experienced a sustained increase in platelet counts.
Safety concerns with Nplate include unchewable deposits in the bone marrow and the possibility that at one time Nplate is stopped, blood platelet counts could drop down the stairs what they were in front beginning intervention.
Additional risks include blood clots due to inordinate increases in platelets and, if Nplate were disposed to patients with an abnormal rakehell condition known as myelodysplasia, a danger for a form of blood malignant neoplastic disease known as acute leukaemia. Myelodysplasia, which is associated with low platelet counts, predisposes some patients to leukemia. In a study of 44 patients wHO had myelodysplasia and received Nplate, four patients developed leukemia. Further clinical trials in patients with predisposing conditions for leukemia will be required to determine whether the development of leukemia may relate to the use of Nplate. Nplate is approved only for habit among patients with chronic ITP.
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.
Nplate is manufactured by Amgen, Inc. of Thousand Oaks, Calif.
http://www.fda.gov
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Monday, 1 September 2008
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