In the human, blood normally coagulates within seconds of an injury or forms blood clots (platelets (tiny cells in the blood) congregate around ruptures in blood vessels to form a clot, through a complex process involving as many as twenty different plasma proteins (manufactured in the liver), known as coagulation factors. Plasma proteins C and S inhibit (anticoagulants — prevent blood clots) reverse the process to prevent excessive clotting. Major structural component of blood clots is Fibrin fiber, creates a fibrin mesh. The fibrin mesh is stronger and more stable then the temporary platelet scab. Days after platelets form a clot, fibrin mesh strengths even more, protecting the blood vessels from further damage or blood loss. A protein called plasma is formed that dissolves fibrin (after the injury heals) and the blood clot is slowly removed.
Congenital protein C or S deficiency is an inherited disorder (Often develops in the utero or in the first few days of life) caused by deficiency of the plasma proteins C and S, and attributed to vitamin K deficiency. Severe congenital protein C deficiency results in a hypercoagulable state (Abnormal tendency for blood clotting) or thrombosis and often goes under diagnosed. Also, deficiency related to a history of recurrent blood clots in the vein. Jesse Goodman, M.D., M.P.H, director of FDA’s Center for Biologics Evaluation and Research said: ” If left untreated, clotting may result in blindness, severe brain damage, multi – organ failure and death for these patients.” Protein C deficiency (Heterozygous – one normal gene and one defective) occurs in approximately one to two for every million births. Protein S deficiency occurs in about one in 20,000 people. Diagnosis includes medical and family history (Screened for the presence of the genetic defect.), physical examination and laboratory tests (Blood tests – detecting for deficiency in proteins C and S, and speed at which small blood vessels close off to stop bleeding and platelet function.) Complications related to protein C or S deficiency include: Pulmonary embolism (blocked artery in the lungs), recurrent pregnancy loss, repeated venous clots (clot in a vein deep within the body), and childhood stroke. Symptoms include swelling and redness of the leg, jaundice, edema (fluid collection in the tissue), mental confusion, and vomiting of blood.
In March 2007, Baxter International received Food and Drug Administration approval (under a priority review schedule) for the sale and marketing of CEPROTIN (Manufactured by Baxter Healthcare Corporation of Deerfield Illinois): First replacement therapy in patients (pediatrics and adults) with life — threatening blood clotting complications related to severe congenital Protein C deficiency. The drug is derived from protein C concentrate (healthy human blood donors) plasma. During clinical phase two and phase three study trials: study included eighteen patients (nine male and nine female) ranging in age from newborn to 25.7 years. CEPROTIN proven effective in 94 percent of patients and six percent of patients treated found “effective with complications,” because they required a dosage adjustment. During the study, seven patients took CEPROTIN as a preventive measure before surgery or anticoagulation therapy, and eight had taken the medication as a long – term preventive measure. CEPOTRIN was more effective treatment compared to modalities, such as fresh frozen plasma or conventional anticocoagulants. Also, more effectively reducing size and number of skin lesions in patients.
The most common adverse reactions observed were rash, itching and lightheadedness. CEPROTIN may not be advisable for patients on low sodium / renal impairment diet because exceeds 200 mg. Medication contains trace amounts of heparin, which may lead to Heparin induced thrombocytopenia (low blood platelet count). As plasma derived treatment therapeutics, potential to transmit infectious agents or viruses cannot be totally eliminated.
The Food and Drug Administration granted CEPROTIN orphan drug status (provide manufacturer with financial incentives to develop a drug or biologic to treat a rare disease) affecting fewer than 200,000 people in the United States. In July 2001, the European Medicines Evaluation Agency (EMEA) granted Baxter International license for the sale of CEPROTIN. Larry Guiheen, president, Baxter BioPharmaceuticals said: “Baxter has long – standard history of developing therapeutics for rare coagulation disorders. As part of that commitment, we have made CEPROTIN available to patients, most of them children, for compassionate use and through our clinical trials since 1998. We are pleased to make this therapy available to patients with this extremely rare condition.”
Starting on April 2, 2007, Baxter Pharmaceuticals will provide a toll free telephone to obtain further information regarding CEPROTIN (1 – 888 – 237 – 7684) from 7:00 a.m. to 6:00 p. m. Central Standard Time, Monday through Friday.