Hemophilia future or investigational therapies
|
Hemophilia Microchapters |
|
Diagnosis |
|---|
|
Treatment |
|
Case Studies |
|
Hemophilia future or investigational therapies On the Web |
|
American Roentgen Ray Society Images of Hemophilia future or investigational therapies |
|
Risk calculators and risk factors for Hemophilia future or investigational therapies |
Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1];Associate Editor(s)-in-Chief: Sabawoon Mirwais, M.B.B.S, M.D.[2]
Overview
Future and investigational therapies of hemophilia include co-transplantation of endothelial colony-forming cells (ECFCs) and placenta-derived mesenchymal stromal cells (PMSCs) at the neonatal age and prevention of the anti-factor VIII memory B-cell response.
Future and Investigational Therapies
Future and investigational therapies of hemophilia include:
Co-transplantation of endothelial colony-forming cells (ECFCs) and placenta-derived mesenchymal stromal cells (PMSCs)
- This is done at the neonatal age.[1]
- It is a potential strategy to achieve stable and long-term engraftment.[1]
- This holds great promise for the cell-based treatment of hemophilia A.
Prevention of the anti-factor VIII memory B-cell response
- It is an investigational therapy to counter the formation of inhibitors.
- This is achieved via selective inhibition of the Bruton's tyrosine kinase, (R)-5-amino-1-(1-cyanopiperidin-3-yl)-3-(4-[2,4-difluorophenoxyl] phenyl)-1H pyrazole-4-carboxamide (PF-06250112), to inhibit B-cell receptor signaling prior to challenge with exogenous factor VIII.[2]
References
- ↑ 1.0 1.1 Gao K, Kumar P, Cortez-Toledo E, Hao D, Reynaga L, Rose M, Wang C, Farmer D, Nolta J, Zhou J, Zhou P, Wang A (January 2019). "Potential long-term treatment of hemophilia A by neonatal co-transplantation of cord blood-derived endothelial colony-forming cells and placental mesenchymal stromal cells". Stem Cell Res Ther. 10 (1): 34. doi:10.1186/s13287-019-1138-8. PMC 6341603. PMID 30670078.
- ↑ Delignat S, Russick J, Gangadharan B, Rayes J, Ing M, Voorberg J, Kaveri SV, Lacroix-Desmazes S (December 2018). "Prevention of the anti-factor VIII memory B-cell response by inhibition of the Bruton's tyrosine kinase in experimental hemophilia A". Haematologica. doi:10.3324/haematol.2018.200279. PMID 30545924.