About Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria is an acquired clonal disorder of hematopoietic stem cells caused by somatic mutations in the PIGA gene, resulting in deficiency of GPI-anchored complement regulatory proteins (CD55 and CD59) on blood cell surfaces. The loss of these proteins renders red blood cells, white blood cells, and platelets vulnerable to complement-mediated destruction, leading to intravascular hemolysis, thrombosis in unusual sites, and cytopenias. PNH is closely associated with aplastic anemia and carries a significantly elevated risk of life-threatening venous thromboembolism.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Paroxysmal Nocturnal Hemoglobinuria trials.
Flow cytometry quantifying the PNH clone size (percentage of GPI-deficient granulocytes and red cells) is essential for most trial eligibility criteria; a clone size greater than 10% in granulocytes is typically required.
Current use of complement inhibitors (eculizumab, ravulizumab) affects eligibility for trials of novel complement pathway agents; document your treatment history and any breakthrough hemolysis events.
LDH levels, transfusion history, thrombotic event history, and FACIT-Fatigue scores are commonly used outcome measures in PNH trials and should be documented prospectively.
Patient Resources
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