TY - JOUR
T1 - Paroxysmal nocturnal hemoglobinuria
T2 - Current treatments and unmet needs
AU - Bektas, Meryem
AU - Copley-Merriman, Catherine
AU - Khan, Shahnaz
AU - Sarda, Sujata P.
AU - Shammo, Jamile M.
N1 - Funding Information:
This research was developed under a research contract between RTI Health Solutions and Apellis Pharmaceuticals and was funded by Apellis Pharmaceuticals. Bektas, Copley-Merriman, and Khan are employees of RTI Health Solutions. Sarda is an employee of Apellis Pharmaceuticals. Shammo consults for Apellis Pharmaceuticals.
Publisher Copyright:
© 2020, Academy of Managed Care Pharmacy.
PY - 2020/12
Y1 - 2020/12
N2 - The current standard of care for paroxysmal nocturnal hemoglobinuria (PNH) are the C5 inhibitors eculizumab and ravulizumab, both monoclonal antibodies designed to target the complement protein C5, thereby preventing its cleavage and the formation of the terminal attack complex. C5 inhibitors have yielded substantial improvements in the treatment of PNH and changed the mortality and morbidity, as well as healthrelated quality of life of patients with the disease. These treatments target underlying intravascular hemolysis; however, they do not address extravascular hemolysis, resulting in incomplete response and remaining symptoms in some patients. Therefore, despite treatment with a C5 inhibitor, some patients still experience anemia with associated fatigue, transfusion needs, and impaired health-related quality of life.
AB - The current standard of care for paroxysmal nocturnal hemoglobinuria (PNH) are the C5 inhibitors eculizumab and ravulizumab, both monoclonal antibodies designed to target the complement protein C5, thereby preventing its cleavage and the formation of the terminal attack complex. C5 inhibitors have yielded substantial improvements in the treatment of PNH and changed the mortality and morbidity, as well as healthrelated quality of life of patients with the disease. These treatments target underlying intravascular hemolysis; however, they do not address extravascular hemolysis, resulting in incomplete response and remaining symptoms in some patients. Therefore, despite treatment with a C5 inhibitor, some patients still experience anemia with associated fatigue, transfusion needs, and impaired health-related quality of life.
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U2 - 10.18553/JMCP.2020.26.12-B.S14
DO - 10.18553/JMCP.2020.26.12-B.S14
M3 - Article
C2 - 33356783
AN - SCOPUS:85099076993
SN - 2376-0540
VL - 26
SP - S14-S20
JO - Journal of Managed Care and Specialty Pharmacy
JF - Journal of Managed Care and Specialty Pharmacy
ER -
