Plenary Overview: Novel therapeutics

Location: Pacifico

CE: 1

Chair(s)

Margareth Ozelo, MD, PhD (she/her/hers)

Professor
Hemocentro Unicamp, University of Campinas
Campinas, Brazil

Speaker(s)

SB

Shrinivas Bishu, MD

Assistant Professor
Division of Gastroenterology Department of Medicine
University of Michigan
Francisco Careta

Professor
Federal University of Espirito Santo
Alegre, Brazil
Niamh Larkin, MSc, BSc, RGN, RNP, RANP

Haemophilia Advance Nurse Practitioner
National Coagulation Centre
St. James's Hospital
Dublin 8, Ireland
Margareth Ozelo, MD, PhD (she/her/hers)

Professor
Hemocentro Unicamp, University of Campinas
Campinas, Brazil
David Stephensen, MSc PhD (he/him/his)

Physiotherapist
Kent Haemophilia and Thrombosis Centre, UK,and the Haemophilia Centre at the Royal London Hospital

Learning Objectives:

Overview
Patient perspective with severe hemophilia on emicizumab
Patient perspective gene therapy, Compared with prophylaxis, gene therapy is superior because it maintains factor levels at constant values that virtually stop spontaneous bleeding. However, it is still not possible to predict the factor values that each person will be able to reach or for how long they will be able to maintain it. In addition, long-term steroid treatments can have negative impacts.
Changing role of the haemophilia team in the era of novel therapeutics, The development of novel therapeutics has positively impacted the direction of haemophilia care into the future, however the underlying principles remain consistent. Personalized therapeutic care and patient partnerships aim to optimize treatment schedules but issues continue such as compliance, bleeding episodes and management of arthropathy and pain. The haemophilia team and the therapeutic landscape will continue to evolve in sync.
Physiotherapist perspective: Joint issues / activity choice, The emergence of novel therapies for the treatment of people with bleeding disorders has markedly reduced their frequency of bleeding and burden of disease, but has it changed the course of their existing joint disease? Monitoring early signs of joint disease progression, supporting and enabling physical activity and participation choices and goals are important challenges to ensure that in the era of novel therapies, people with a bleeding disorder have the ‘freedom to move’.

Presentations:

10:00 - 10:01 ART

Chair
Chair: Margareth C. Ozelo, MD, PhD (she/her/hers) – Hemocentro Unicamp, University of Campinas
10:01 - 10:10 ART

Overview
Speaker: Margareth C. Ozelo, MD, PhD (she/her/hers) – Hemocentro Unicamp, University of Campinas
10:10 - 10:15 ART

Patient with severe hemophilia on emicizumab
Speaker: Shrinivas Bishu, MD – University of Michigan
10:15 - 10:20 ART

Patient with gene therapy
Speaker: Francisco Careta – Federal University of Espirito Santo
10:20 - 10:30 ART

Changing role of the hemophilia team in the era of novel therapeutics
Speaker: Niamh Larkin, MSc, BSc, RGN, RNP, RANP – St. James's Hospital
10:30 - 10:40 ART

Recognizing and managing early joint damage
Speaker: David Stephensen, MSc PhD (he/him/his) – Kent Haemophilia and Thrombosis Centre, UK,and the Haemophilia Centre at the Royal London Hospital
10:40 - 11:00 ART

Discussion
Chair: Margareth C. Ozelo, MD, PhD (she/her/hers) – Hemocentro Unicamp, University of Campinas