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 Table of Contents  
REVIEW
Year : 2011  |  Volume : 2  |  Issue : 3  |  Page : 225-233

New Therapeutic Agents for Hemophilia: Extended Half-life, Enhanced Activity, and Oral Therapy


Executive Director Hematology Disease Area Lead Pfizer, Inc. and Professor of Medicine Robert Wood Johnson Medical School University of Medicine and Dentistry of New Jersey New Brunswick, NJ, USA

Date of Web Publication30-Jun-2014

Correspondence Address:
Marcus E Carr
M.E. Carr, Pfizer Inc., 500 Arcola Road, F-4218 Collegeville, PA 19426
USA
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Source of Support: None, Conflict of Interest: None


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  Abstract 

With the introduction of safe recombinant products and advances in the purification of plasma concentrates, the ravages of the HIV and hepatitis C epidemics in hemophilia patients have begun to subside in many countries. Indeed, the life spans for many patients with hemophilia A and B are approaching those of the non-hemophilic population. However, challenges remain, and new agents and treatments are in development to meet these challenges. Prophylactic therapy is reducing the burden of joint damage caused by joint bleeding. The challenge is to reduce the frequency and increase the convenience so that prophylaxis will become the primary mode of therapy for adults as well as children. For patients with inhibitors to factors VIII or IX, the challenge is to increase the efficacy of treatments and develop forms of prophylaxis so that treatment of these patients will no longer require immune tolerance therapy, simply a shift to a different agent. The global challenge is to make therapy available to all patients regardless of the location, as we move safely toward the ultimate goal of a genetic cure. In the near future, new devices to speed and simplify factor administration and increasing numbers of room temperature-stable clotting factors will increase user convenience. Long-acting clotting factors will enhance and simplify prophylactic therapy. Novel high-activity and long-acting clotting agents will enhance treatment and allow prophylaxis in inhibitor patients. Oral therapies will obviate the need for these factors in mild patients, convert the phenotype of many patients with moderate disease to a mild form, and augment factor replacement in both severe hemophilia patients and those with inhibitors. It is an exciting time to be involved in hemophilia care, but bringing these new agents to the clinical arena will require a combined effort of the industry, academic health centers, health care professionals, and patients.

Keywords: Hemophilia A, Hemophilia B, Factor VIII inhibitors, Factor IX inhibitors, Long-acting Clotting Factors, Recombinant Factor VIIa, Zymogen Factor Xa, Tissue Factor Pathway Inhibitor, Aptamers, Non-anticoagulant Sulfated Polysaccharides


How to cite this article:
Carr ME. New Therapeutic Agents for Hemophilia: Extended Half-life, Enhanced Activity, and Oral Therapy. J Appl Hematol 2011;2:225-33

How to cite this URL:
Carr ME. New Therapeutic Agents for Hemophilia: Extended Half-life, Enhanced Activity, and Oral Therapy. J Appl Hematol [serial online] 2011 [cited 2021 Dec 2];2:225-33. Available from: https://www.jahjournal.org/text.asp?2011/2/3/225/135591

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