RT Book, Section
A1 Lichtman, Marshall A.
A1 Kaushansky, Kenneth
A1 Prchal, Josef T.
A1 Levi, Marcel M.
A1 Burns, Linda J.
A1 Armitage, James O.
SR Print(0)
ID 1133364028
T1 Paroxysmal Nocturnal Hemoglobinuria
T2 Williams Manual of Hematology, 9e
YR 2017
FD 2017
PB McGraw-Hill Education
PP New York, NY
SN 9781259642470
LK hemonc.mhmedical.com/content.aspx?aid=1133364028
RD 2024/04/19
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  the  complement-mediated  intravascular  hemolysis  that  is  the  clinical  hallmark  of  the  disease.  Marrow  failure  and  thrombophilia  also  complicate  the  disease.It  is  the  only  hemolytic  anemia  caused  by  an  acquired  (ie,  somatic)  mutation  that  is  intrinsic  to  the  red  cell.  The  defect  has  its  origins  in  the  HSPC  from  which  the  circulating  red  cells  are  derived.