Hemophilia A future or investigational therapies
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]Associate Editor(s)-in-Chief: Fahd Yunus, M.D. [2]
Overview
Research is underway into novel therapies to help in the management and possible cure of Hemophilia A and B. These include gene therapy, novel factor concentrates, and immune tolerance induction (ITI) therapy.[1]
Gene therapy
- The goal of gene therapy is to correct the chromosomal deficit which results in Hemophilia. Currently, there is no role for gene therapy in the management of hemophilia, though the issue is being pursued in clinical trials[2]
- 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[3]
Novel Factor Concentrates
- New factor concentrates are being designed with longer half-lives, allowing them to stay functionally active in the patient's system for a longer period of time
- If successful, these extended factor concentrates have the potential to provide less frequent and/or fewer injections to hemophiliacs receiving prophylaxis
- Though they do not provide a cure to hemophilia, these extended factor concentrate formulations could significantly alter the administration of prophylaxis to patients, and increase availability of prophylaxis to a wider percentage of patients with hemophilia [4]
Immune Tolerance Induction (ITI) Therapy
- Recently, Immune Tolerance Induction (ITI) therapy is being researched as a means of helping patients with hemophilia 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)[5]
References
- ↑ Nguyen TH, Anegon I (2016). "Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success". EMBO Mol Med. 8 (5): 439–41. doi:10.15252/emmm.201606325. PMC 5130315. PMID 27138565.
- ↑ 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
- ↑ Current issues in prophylaxis – World Federation of Hemophilia. Available at http://www.wfh.org/en/abd/prophylaxis/current-issues-in-prophylaxis. Accessed on Sept 20, 2016
- ↑ Inhibitors | Hemophilia | NCBDDD | CDC. Available at http://www.cdc.gov/ncbddd/hemophilia/inhibitors.html. Accessed on Sept 20, 2016