Paroxysmal nocturnal hemoglobinuria
ICD-10 D59.5 · ICD-11 3A21.0
This protocol addresses paroxysmal nocturnal hemoglobinuria (PNH) in patients whose prior proximal complement inhibitor therapy has not achieved its intended haematological goals, defining the clinical situation that calls for escalation to the next line of management.
Previous Line — Failure Condition
Treatment with a proximal complement inhibitor — including danicopan, iptacopan, or pegcetacoplan — failed to achieve improvement in hemoglobin levels accompanied by a drop in reticulocytes to the normal range. Non-achievement of these haematological targets defines the escalation point to this protocol.
Next-Line Approach (partial overview)
The protocol outlines a transplant-based strategy that represents the only potentially curative treatment approach in the appropriate clinical context. The full protocol details the specific indications and conditions under which this approach applies.
References
The only potentially curative treatment approach for PNH is allogeneic stem cell transplantation.
An indication for allogeneic stem cell transplantation arises in PNH in the context of severe aplastic anemia if there is already an indication for transplantation due to the aplastic anemia alone (refer to Onkopedia guideline on aplastic anemia).
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