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Management of infection in PNH patients treated with eculizumab or other complement inhibitors: Unmet clinical needs

Corrado Girmenia1, Wilma Barcellini2, Paola Bianchi2

  • 1Hematology, Department of Hematology, Oncology and Dermatology, AOU Policlinico Umberto I, Sapienza University of Rome, Rome, Italy.

Blood Reviews
|September 18, 2022
PubMed

Insights

Experts identified key unmet needs in managing infectious risks for paroxysmal nocturnal hemoglobinuria (PNH) patients on complement inhibitors (CIs). Recommendations focus on enhancing infection prevention and non-pharmacological strategies for better CI therapy outcomes.

Area of Science:

  • Hematology
  • Immunology
  • Clinical Medicine

Background:

  • Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired blood disorder.
  • Terminal complement inhibitors (CIs) like eculizumab are effective PNH treatments.
  • Infectious complications pose a significant risk for PNH patients on CIs.

Purpose of the Study:

  • To identify and address unmet clinical needs (UCNs) in managing infectious risk in PNH patients.
  • To establish consensus on optimizing infection prevention and management strategies.
  • To propose advancements in clinical practice for complement inhibitor therapy.

Main Methods:

  • A panel of experts was convened to discuss UCNs using the Delphi technique.
  • Clinically relevant UCNs for PNH patients receiving or considering CIs were selected.
  • Consensus opinions and proposals for clinical practice were developed.

Main Results:

  • Key UCNs identified include optimizing infection prevention measures.
  • Developing non-pharmacological infectious risk-mitigation strategies is crucial.
  • Improving the management of disease exacerbation during infectious complications is needed.

Conclusions:

  • Addressing these UCNs can improve the safety and efficacy of CI therapy in PNH.
  • The findings will inform future clinical practice and research in PNH management.
  • Enhanced strategies are needed to mitigate infectious risks associated with CIs.

Keywords:
ChemoprophylaxisComplement inhibitorsEculizumabInfectionsParoxysmal nocturnal hemoglobinuriaVaccination

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