Clinical correlations of paroxysmal nocturnal hemoglobinuria with myeloproliferative neoplasms, highlighting thrombosis risks.
Key Points
This study aims to characterize the clinical phenotype and treatment outcomes of patients with paroxysmal nocturnal hemoglobinuria and myeloproliferative neoplasms.
Database search of Mayo Clinic to identify patients with treatment-requiring PNH and MPN
Clinical features recorded included hemolysis, thrombosis, and therapeutic interventions
Next-generation sequencing and cytogenetic analysis were performed in selected cases.
Identified 11 patients with treatment-requiring PNH and MPN including cases of primary myelofibrosis and polycythemia vera.
82% experienced hemolytic anemia, prompting PNH workup; 18% were due to thrombosis.
First-line treatment with complement inhibitors showed only a 22% control rate of hemolysis, with some achieving durable responses.