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December 8, 2025BloodOpen Access

Treatment-requiring paroxysmal nocturnal hemoglobinuria in association with myeloproliferative neoplasms (MPNs): Clinical correlations and outcomes

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Authors

APAnimesh PardananiATAyalew TefferiNGNaseema Gangat

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Overview

Observational analysis reveals hemolysis and thrombosis in patients with paroxysmal nocturnal hemoglobinuria and myeloproliferative neoplasms, suggesting the need for improved treatment strategies.

Key Points

  • Hemolysis was controlled in only 22% of patients receiving eculizumab, despite treatment for paroxysmal nocturnal hemoglobinuria.
  • The cohort included 11 patients, with 54% experiencing transfusion-dependent anemia during the median follow-up of 8.4 years.
  • Next-generation sequencing identified various mutations in paroxysmal nocturnal hemoglobinuria and myeloproliferative neoplasms patients, with thrombosis seen in 18% of the cohort.
  • The study highlights the predominance of primary myelofibrosis, especially in patients with CALR mutations, indicating a need for improved therapeutic options.

Cite This Study

Pardanani et al. (2025) studied this question.

synapsesocial.com/papers/69362f4e4fa91c937236d82ahttps://doi.org/10.1182/blood-2025-3188
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