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Facing clinical challenges associated with paroxysmal nocturnal haemoglobinuria

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Haematology, Diagnostics and Screening Watch Time: 37 mins

touchMDT Facing clinical challenges associated with paroxysmal nocturnal haemoglobinuria

Watch a leading haematologist, a primary care physician and a patient with paroxysmal nocturnal haemoglobinuria (PNH) share their perspectives on the clinical challenges associated with PNH.

Overview & Learning Objectives

Patient living with PNH

Primary care physician, haematologist and patient

A primary care physician and a haematologist discuss the prevalence, pathophysiology, classification and symptoms of PNH, and the considerations for diagnosis. In addition, Ms Jess Ratcliffe shares her experience of the clinical manifestations of PNH and her journey to diagnosis.

Expert Spotlight
Dr Will Evans
Centre for Academic Primary Care, University of Nottingham, Nottingham, UK
Prof. Régis Peffault de Latour
Hematology and Bone Marrow Transplant Department, Saint-Louis Hospital, Paris, France
Ms Jess Ratcliffe
Godalming, UK

Dr Will Evans, Prof. Régis Peffault de Latour, and Ms Jess Ratcliffe discuss the underlying pathophysiology, signs and symptoms, and diagnosis of PNH.

Listen on the Go

Learn more Back to MDT Hub Time: 11:42
 
Primary care physician and haematologist

A primary care physician and a haematologist discuss the available and emerging complement inhibitors for the management of patients with PNH, including mechanism of action, key clinical data, and potential benefits and limitations of the treatments.

Expert Spotlight
Dr Will Evans
Centre for Academic Primary Care, University of Nottingham, Nottingham, UK
Prof. Régis Peffault de Latour
Hematology and Bone Marrow Transplant Department, Saint-Louis Hospital, Paris, France

Dr Will Evans and Prof. Régis Peffault de Latour discuss the available and emerging complement inhibitors for the management of patients with PNH.

Listen on the Go

Learn more Back to MDT Hub Time: 12:37
 
Haematologist and patient

A haematologist and a patient with PNH discuss living with PNH and its impact on daily life from the patient’s perspective, including the treatment pathway, daily challenges and remaining unmet needs for people with the condition.

Expert Spotlight
Prof. Régis Peffault de Latour
Hematology and Bone Marrow Transplant Department, Saint-Louis Hospital, Paris, France
Ms Jess Ratcliffe
Godalming, UK

Prof. Régis Peffault de Latour and Ms Jess Ratcliffe discuss living with PNH and its impact on daily life from the patient’s perspective.

Listen on the Go

Learn more Back to MDT Hub Time: 12:37
 
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Learning Objectives & Overview
Overview

In this activity, a haematologist involved in caring for patients with PNH, and a primary care physician, share their perspectives on how to recognize and diagnose PNH and manage the condition with complement inhibitors. Plus, hear from a patient on her personal experience of living with PNH, from diagnosis to its impact upon daily life.

Learning Objectives

After watching this activity, participants should be better able to:

  • Summarize the signs and symptoms of PNH and how these inform the clinical diagnosis
  • Explain the benefits and limitations of current and emerging complement inhibitor therapies for PNH
  • Explain the burden of PNH and impact on patients’ quality of life
Faculty & Disclosures
Prof. Régis Peffault de Latour

Hematology and Bone Marrow Transplant Department, Saint-Louis Hospital, Paris, France

Prof. Régis Peffault de Latour is the head of the Hematology and Bone Marrow Transplant Department at Saint-Louis Hospital, Paris, France. read more

Prof. Peffault de Latour is also involved with the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, as well as the MaRIH – the French organization for rare haematological and immunological disorders. He is also the chair of the Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplantation group. 

Prof. Peffault de Latour’s research interest focuses on bone marrow transplantation, bone marrow failure and PNH. He is the principal investigator of many research studies on bone marrow failure. Prof. Peffault de Latour is the author or co-author of more than 300 papers and is a regular reviewer for the New England Journal of Medicine, Blood, Haematologica, British Journal of Haematology, Biology of Blood and Marrow Transplantation and Bone Marrow Transplantation.

Prof. Régis Peffault de Latour discloses: Advisory board/panel fees from Alexion, Apellis, Novartis and Sobi. Consultancy/speaker’s bureau fees from Alexion, Amgen, Apellis, Idem, Jazz, Novartis, Pfizer, Roche and Samsung. Grants/research support from Alexion, Novartis, Pfizer and Sobi.

Dr Will Evans

Centre for Academic Primary Care, University of Nottingham, Nottingham, UK

Dr Will Evans is an academic with the Primary Care Stratified Medicine (PRISM) research group, in the Centre for Academic Primary Care at the University of Nottingham, UK. His research interests include personalized medicine, rare diseases, genomics in primary care and large database analysis. read more

Dr Evans is also a primary care physician in Leeds, UK, and is a general practitioner with a special interest in genetics with the Yorkshire Regional Genetics Service.

Dr Evan’s eldest son has Niemann–Pick type C, an ultra-rare metabolic disease, and Dr Evans is the current chairman of the Board of Trustees of Niemann–Pick UK, the UK charity for the disease.

Dr Will Evans discloses: Advisory board/panel fees from Orphazyme and Intrabio (relationships terminated). Consultancy fees from Mendelian. Grants/research support from National Institute for Health and Care Research. Salary/contractual services from Leeds Teaching Hospitals NHS Trust, Mendelian (relationship terminated), National Institute for Health and Care Research, Oakwood surgery (relationship terminated) and the University of Nottingham. Speaker’s bureau fees from Takeda. Stock shares in Mendelian.

Ms Jess Ratcliffe

Godalming, UK

Ms Jess Ratcliffe is a personal development coach to startup leaders and teams, and was diagnosed with PNH in 2016, at the age of 25. read more

Jess started her first company, GaBoom, in 2010 before moving to San Francisco in 2014 to work for a small startup, Big Health, as a product manager. She returned to the UK in 2017, following her PNH diagnosis, and embarked on her current career as a personal development coach, working with both individuals and teams to unlock their potential.

Alongside her work, Jess is an avid golfer. Inspired by her experience of shielding during the start of the pandemic, Jess took up the sport during national lockdown in the UK.

Ms Jess Ratcliffe has no interests/relationships or affiliations to disclose in relation to this activity.

Downloads

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