Haematology Watch Time: 19 mins

touchEXPERT OPINIONS Paroxysmal nocturnal hemoglobinuria: from biology to bedside

Prof. Régis Peffault de Latour (French Reference Center for Aplastic Anemia and PNH, Saint-Louis Hospital, Paris, France) appraises the most important recent and emerging data, and discusses their potential impact on the clinical management of patients living with PNH.

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Current treatment landscape

Régis Peffault de Latour summarizes the aetiology of paroxysmal nocturnal hemoglobinuria (PNH), existing diagnostic criteria and approaches to clinical management. He also provides an overview of key data supporting the use of complement cascade inhibitors currently approved for use in PNH.

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Identifying the unmet needs

Régis Peffault de Latour explains the scientific rationale underpinning complement cascade inhibition to manage intra- and extravascular hemolysis leading to thrombosis formation. He also describes best practice for measuring quality of life for patients living with PNH using the FACIT-F scale.

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New and emerging therapies

Régis Peffault de Latour appraises the different therapeutic agents currently available for complement cascade inhibition and discusses their potential impact on the treatment landscape for PNH.

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Overview & Learning Objectives
Overview

Prof. Régis Peffault de Latour (French Reference Center for Aplastic Anemia and PNH, Saint-Louis Hospital, Paris, France) appraises the most important recent and emerging data, and discusses their potential impact on the clinical management of patients living with PNH.

This activity is intended for hematologists and other healthcare professionals involved in the treatment of patients with PNH.

Learning Objectives

After watching this touchEXPERT OPINIONS, you should be able to:

  • Discuss the current treatment landscape for PNH
  • Describe the unmet medical needs for PNH with respect to current treatment options
  • Summarize the evolving treatment landscape and future approaches for the management of PNH
About Prof. Régis Peffault de Latour
Prof. Régis Peffault de Latour

Saint-Louis Hospital, Paris, France

Régis Peffault de Latour is based at Saint-Louis Hospital, Paris, where he is Professor of Hematology in the Department of Hematology and Bone Marrow Transplantation.

As a leading clinician with extensive experience encompassing clinical trials and scientific research in the fields of rare immunological and hematological disorders, Prof. Peffault de Latour leads the French Reference Center for Aplastic Anemia and PNH, and is Chair of the French Network for Rare Immunological and Hematological Disorders (MARIH). He also Chairs the EuroBloodNet Bone Marrow Failures (BMF) group, and the European Society for Blood and Bone Marrow Transplantation (EBMT) Severe Aplastic Anaemia Working Party (SAAWP).

Disclosures: Consultancy and honoraria for Alexion Pharmaceuticals, Novartis International AG and Pfizer.

This content is intended for healthcare professionals only. Please confirm that you are a healthcare professional.

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