Hemophilia A future or investigational therapies
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Overview
Research is underway into novel therapies to help in the management and possible cure of hemophilia A.
Gene therapy
The goal of gene therapy is to correct the chromosomal deficit which results in Hemophilia A. Currently, there is no role for gene therapy in the management of hemophilia A, though the issue is being pursued in clinical trials. [1] Researchers are also studying the genomes of patients affected with inhibitors to determine why some patients develop inhibitors and others do not. Understanding a patient's genetic profile may help physicians predict a patient's risk for developing inhibitors to factor concentrates, and help guide their treatment. [2]
Immune Tolerance Induction (ITI) Therapy
- Recently, Immune Tolerance Induction (ITI) therapy is being researched as a means of helping patients with haemophilia and factor inhibitors. ITI involves overcoming the immune reaction and desensitizing the body to the foreign factor concentrate infusion. ITI is expensive, time-intensive, and requires the oversight of multiple medical professionals, and is best coordinated at a hemophilia treatment center (HTC). [3]
References
- ↑ Konkle BA, Josephson NC, Nakaya Fletcher S. Hemophilia A. 2000 Sep 21 [Updated 2014 Jun 5]. In: Pagon RA, Adam MP, Ardinger HH, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2016. Available from: http://www-ncbi-nlm-nih-gov.laneproxy.stanford.edu/books/NBK1404/
- ↑ Why We Do research on Hemophilia | Hemophilia | NCBDDD | CDC. Available at http://www.cdc.gov/ncbddd/hemophilia/research.html. Accessed on Sept 20, 2016
- ↑ Inhibitors | Hemophilia | NCBDDD | CDC. Available at http://www.cdc.gov/ncbddd/hemophilia/inhibitors.html. Accessed on Sept 20, 2016