Paroxysmal nocturnal hemoglobinuria Flashcards
1
Q
What is the role for prophylactic AC?
A
no longer used since advent of eculizumab
2
Q
Disease association to know for boards with pNH
A
aplastic anemia
3
Q
Diagnosis of PNH
A
flow cytometry (looking for CD55 and CD59 deficiency)
4
Q
cell markers to know
A
CD55 + CD59
5
Q
VTE management in PNH patient
A
lovenox bridge to warfarin (confirm)
6
Q
First and second line for PNH
A
1) folate supplementation
2) IF large clone size –> eculiziumab (targets CD5, inhibiting complement) indefinitely (doesn’t correct underlying mutation)
3) second line = allo HCT
7
Q
Presentation of PNH
A
- fatigue
- jaundice
- hemoglobinuria
- abdominal pain/ED/pHTN (due to NO depletion)
8
Q
Hereditary or somatic?
A
acquired defect (somatic cell)
9
Q
Labs in PNH
A
- nonspecific - variable RC size, variable retic count, elevated LDH,
- this is why its a hard diagnosis to make
***can also see pancytopenic because it’s a somatic mutation in an HSCT
10
Q
What does initiation of therapy depend on in PNH?
A
Size of the clone
