RT Book, Section
A1 Lichtman, Marshall A.
A1 Kaushansky, Kenneth
A1 Prchal, Josef T.
A1 Levi, Marcel M.
A1 Burns, Linda J.
A1 Linch, David C.
SR Print(0)
ID 1189334473
T1 Paroxysmal Nocturnal Hemoglobinuria
T2 Williams Manual of Hematology, 10e
YR 2022
FD 2022
PB McGraw-Hill Education
PP New York, NY
SN 9781264269204
LK hemonc.mhmedical.com/content.aspx?aid=1189334473
RD 2024/10/13
AB Paroxysmal  nocturnal  hemoglobinuria  (PNH)  is  an  acquired  hematopoietic  stem/progenitor  cell  (HSPC)  disorder  characterized  by  deficiency  of  glycosyl  phosphatidylinositol  (GPI)-anchored  proteins  (GPI-APs)  on  the  surface  of  hematopoietic  cells.  Two  complement  regulatory  proteins  (CD55  and  CD59)  are  GPI  anchored,  and  deficiency  of  these  two  proteins  on  erythrocytes  derived  from  the  mutant  HSPC  leads  to  complement-mediated  intravascular  hemolysis  that  is  the  clinical  hallmark  of  the  disease.  Marrow  failure  and  thrombophilia  also  complicate  the  disease.