Abdul Muqtadir Abbasi: PNH Treatment – Simplified for Medical Students
Abdul Muqtadir Abbasi, Assistant Consultant at King Faisal Specialist Hospital and Research Center, shared a post on LinkedIn:
“PNH Treatment: Simplified for Medical Students
The key to treating PNH is to answer one question:
Is the patient suffering from complement-mediated hemolysis, bone marrow failure, or both?
1. Complement inhibition – the main treatment for symptomatic PNH
In PNH, the complement system destroys RBCs because they lack CD55 and CD59.
We use drugs that block complement:
- Eculizumab / Ravulizumab: Block C5 – reduce intravascular hemolysis.
- Crovalimab: Also blocks C5.
- Pegcetacoplan / Iptacopan: Act upstream of C5 and can help control intravascular and extravascular hemolysis.
- Danicopan: An oral factor D inhibitor used as an add-on to a C5 inhibitor for selected patients with persistent extravascular hemolysis.
These treatments reduce hemolysis and can improve anemia, symptoms and thrombotic risk. They do NOT eliminate the PNH clone.
2. Supportive treatment
Depending on the patient:
- Folic acid; iron only if deficiency is confirmed.
- RBC transfusions when clinically indicated.
- Treat complications and monitor blood counts, hemolysis markers and renal function.
- Vaccination and infection-prevention measures are essential with complement inhibitors, especially against meningococcus.
3. What if the patient has bone marrow failure?
Complement inhibitors do not correct aplastic anemia.
If significant marrow failure is present, treatment may include immunosuppressive therapy or allogeneic stem-cell transplantation, depending on severity and suitability.
4. What about thrombosis?
PNH can cause life-threatening thrombosis, including at unusual sites.
- Urgent assessment and anticoagulation are generally required when thrombosis occurs, unless contraindicated.
- Complement inhibition is central to reducing ongoing PNH-related thrombotic risk.
- Duration of anticoagulation is individualized.
5. Does every patient need treatment?
No. A small PNH clone without hemolysis, symptoms or clinically significant complications may need monitoring rather than complement inhibition.
The exam takeaway:
- Symptomatic hemolytic PNH – consider complement inhibition.
- PNH with significant aplastic anemia – address marrow failure too.
- PNH-associated thrombosis – urgent anticoagulation assessment and complement inhibition.
- Small, clinically silent clone – monitor and individualize.
Treat the disease the patient has – not just the clone percentage.”
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