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September 10, 2025Acta HaematologicaOpen Access

Paroxysmal nocturnal hemoglobinuria with large clones in non-hypoplastic myelodysplastic syndrome- report of two cases

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Authors

JBJanine Briggeler-ManiEHEmmanuel HäfligerASAnnatina Schnegg‐Kaufmann

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Overview

Report on two cases of hemolytic paroxysmal nocturnal hemoglobinuria in patients with non-hypoplastic myelodysplastic syndrome, highlighting treatment with complement inhibitors.

Key Points

  • Two cases of paroxysmal nocturnal hemoglobinuria were identified in patients with myelodysplastic syndrome, showing effective treatment.
  • In both cases, patients displayed significant anemia and received treatment with complement inhibitors ravulizumab and pegcetacoplan, indicating their efficacy.
  • Assessment of paroxysmal nocturnal hemoglobinuria clones in myelodysplastic syndromes is crucial for appropriate diagnosis and management.
  • Complement inhibitors are underrepresented in studies for non-hypoplastic myelodysplastic syndrome, yet standard doses appear effective.

Cite This Study

Briggeler-Mani et al. (2025) studied this question.

synapsesocial.com/papers/68c23cf6b210217d6478c040https://doi.org/10.1159/000548287
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