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Novel drugs to treat hemophilia.

21:51 EDT 18th April 2014 | BioPortfolio

Summary of "Novel drugs to treat hemophilia."

Importance of the field: Hemophilia management requires replacement of the deficient clotting factor. Inhibitor development complicates treatment, and the implementation of and adherence to prophylaxis, considered the standard of care for severe hemophilia, is hampered by the high cost and the need for frequent intravenous infusions. New, less expensive therapies are needed to reduce morbidities, enhance convenience and improve outcomes. Areas covered in this review: We conducted a search of the Medline database from 1990 to 2010 for English language articles pertaining to hemophilia drug therapy. More than 4000 citations were obtained and examined for topic relevance. Additionally, proceedings from the American Society of Hematology, International Society on Thrombosis and Hemostasis, World Federation of Hemophilia, National Hemophilia Foundation and European Association for Haemophilia and Allied Disorders and the ClinicalTrials.gov website were reviewed for relevant publications and information. What the reader will gain: This article summarizes current research efforts focused on developing less immunogenic and more potent and/or longer-acting clotting factor concentrates that uniquely promote hemostasis, are more affordable and do not require intravenous administration. Take home message: The availability of orally administered clotting factors will truly transform the lives of hemophilia patients and their families and caregivers.

Affiliation

Hemophilia and Thrombophilia Center, Rush University Medical Center, 1653 West Congress Parkway, Chicago, IL 60612-3833, USA 312 942 8114 ; 312 942 8975 ; lvalentino@rush.edu.

Journal Details

This article was published in the following journal.

Name: Expert opinion on emerging drugs
ISSN: 1744-7623
Pages:

Links

PubMed Articles [4687 Associated PubMed Articles listed on BioPortfolio]

Sequential therapy with activated prothrombin complex concentrates and recombinant activated factor VII to treat unresponsive bleeding in patients with hemophilia and inhibitors: a single center experience.

Currently, the greatest challenge in hemophilia treatment is managing hemophilia patients with inhibitors. The two main bypassing agents that are used to treat hemophilia patients with inhibitors are...

Attempts to treat patients with hemophilia, the "royal disease".

FXIII - Mechanisms of Action in the Treatment of Hemophilia A.

Hemophilia is characterised by abnormal thrombin generation and impaired clot stability. FXIII promotes clot stability and may be a useful adjunct treatment for hemophilia.

Turoctocog alfa for the treatment of hemophilia A.

Patients with hemophilia A lack functional coagulation factor VIII (FVIII), causing excessive spontaneous bleeding episodes and bleeding during trauma or surgery. Plasma-derived or synthetic recombina...

Successful renal replacement therapy for a patient with severe hemophilia after surgical treatment of intracranial hemorrhage and hydrocephalus.

A 21-year-old Japanese male with severe hemophilia A was developed end-stage renal failure. He was placed on combination therapy with peritoneal dialysis (PD) and hemodialysis (HD). Eight months later...

Clinical Trials [1039 Associated Clinical Trials listed on BioPortfolio]

Study Evaluating ReFacto in Hemophilia A

To identify the causative mutations in previously untreated patients with hemophilia A enrolled in the ReFacto® clinical safety and efficacy study CTN 93-R833-0XX/C9741-28, using two esta...

Females With Severe or Moderate Hemophilia A or B: A Multi-Center Study

To collect and analyze data on females with hemophilia so as to better define the difference between the study population and the male population with hemophilia.

Covalent Tolerance Induction to Factor VIII-Prediction of Inhibitors in Hemophilia

To correlate the HLA type and genetic defect with hemophilia A.

Epidemiology and Immunology of Hemophilia A Inhibitors

To determine the risk factors associated with inhibitor formation in hemophilia A and to study the mechanism of tolerance in the murine hemophilia A model.

Rituximab to Treat Severe Hemophilia A

Hemophilia A is a serious blood clotting disorder caused by a lack of factor VIII, a specialized protein needed for normal blood clotting to occur. Individuals with this disease may experi...

Medical and Biotech [MESH] Definitions

A deficiency of blood coagulation factor IX inherited as an X-linked disorder. (Also known as Christmas Disease, after the first patient studied in detail, not the holy day.) Historical and clinical features resemble those in classic hemophilia (HEMOPHILIA A), but patients present with fewer symptoms. Severity of bleeding is usually similar in members of a single family. Many patients are asymptomatic until the hemostatic system is stressed by surgery or trauma. Treatment is similar to that for hemophilia A. (From Cecil Textbook of Medicine, 19th ed, p1008)

The classic hemophilia resulting from a deficiency of factor VIII. It is an inherited disorder of blood coagulation characterized by a permanent tendency to hemorrhage.

A hereditary deficiency of blood coagulation factor XI (also known as plasma thromboplastin antecedent or PTA or antihemophilic factor C) resulting in a systemic blood-clotting defect called hemophilia C or Rosenthal's syndrome, that may resemble classical hemophilia.

Agents used to treat AIDS and/or stop the spread of the HIV infection. These do not include drugs used to treat symptoms or opportunistic infections associated with AIDS.

Chinese herbal or plant extracts which are used as drugs to treat diseases or promote general well-being. The concept does not include synthesized compounds manufactured in China.

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