SU-CPSSA-WKSP - CPSSA Workshop: Safety

Sunday, April 21, 2024

08:00 – 11:30 CEST

Location: N117-N118

Speaker(s)

Pratima Chowdary, MD (she/her/hers)

Centre Director
KD Comprehensive Care Haemophilia Centre, Royal Free Hospital
London, United Kingdom
Amy Dunn, MD (she/her/hers)

Professor of Pediatrics
Nationwide Children's Hospital/Ohio State University
Ohio, United States
Alfonso Iorio, MD, PhD, FRCP(C) (he/him/his)

Professor, Department of Health Research Methods, Evidence, and Impact (HEI)
McMaster University
Hamilton, Canada
Radoslaw Kaczmarek, PhD (he/him/his)

Assistant Research Professor of Pediatrics
Wells Center for Pediatric Research, Indiana University School of Medicine
Indianapolis, United States
Peter Lenting, PhD

Director of Research
French National Institute of Health and Medical Research - INSERM
Le Kremlin-Bicetre, France
Brian O'Mahony, FACSLM

CEO
Irish Haemophilia Society
Dublin, Ireland
RP

Rucha Patil

Scientist, ICMR
National Institute of Immunohematology
Mumbai, India
Glenn Pierce, MD, PhD

Vice-President, Medical
World Federation of Hemophilia
La Jolla, United States
Alok Srivastava, MD, FRACP, FRCPA, FRCP

Professor
Christian Medical College
Vellore, India

This popular safety workshop, developed by the CPSSAC of the WFH, will focus on the safety, efficacy and access of new and current therapies for people with bleeding disorders. The latest clinical trial data on pipeline therapies, real world data from recently approved therapies and treatment options in low- and middle-income countries will be highlighted.

Presentations:

08:00 – 08:15 CEST

PART 1: Introduction - Welcome and recap of safety, efficacy and access issues
Speaker: Radoslaw Kaczmarek, PhD (he/him/his) – Wells Center for Pediatric Research, Indiana University School of Medicine
08:15 – 09:00 CEST

PART 2: Safety & efficacy - Pipeline developments: Phase 1-3 clinical trial data
08:17 – 08:32 CEST

Investigator therapies for hemophilia A and B
Speaker: Pratima Chowdary, MD (she/her/hers) – KD Comprehensive Care Haemophilia Centre, Royal Free Hospital
08:32 – 08:47 CEST

Investigator therapies for VWD and rare bleeding disorders
Speaker: Peter Lenting, PhD – French National Institute of Health and Medical Research - INSERM
08:47 – 09:00 CEST

Q&A
09:00 – 10:00 CEST

PART 2: Safety & efficacy - New therapies: real-world data and clinical practice patterns
09:02 – 09:17 CEST

Emicizumab/Hemlibra: Low-dose
Speaker: Rucha Patil – National Institute of Immunohematology
09:17 – 09:32 CEST

Fc-VWF-XTEN Fusion Protein/ALTUVIIIO: 1st year real-world data
Speaker: Amy Dunn, MD (she/her/hers) – Nationwide Children's Hospital/Ohio State University
09:32 – 09:47 CEST

Emicizumab optimization app: how to maximize the vial
Speaker: Alfonso Iorio, MD, PhD, FRCP(C) (he/him/his) – McMaster University
09:47 – 10:00 CEST

Q&A
10:00 – 10:20 CEST

Break
10:20 – 10:40 CEST

PART 2: Safety & efficacy - Cryoprecipitate: cautions and limitations
10:22 – 10:32 CEST

Cryoprecipitate: cautions and limitations
Speaker: Glenn Pierce, MD, PhD – World Federation of Hemophilia
10:32 – 10:40 CEST

Q&A
10:40 – 11:30 CEST

PART 3: Access
10:42 – 10:52 CEST

Treatment options for low- and middle-income countries
Speaker: Brian O'Mahony, FACSLM – Irish Haemophilia Society
10:52 – 11:02 CEST

ACCESS Gene therapy: real-world evidence on access and economics
Speaker: Brian O'Mahony, FACSLM – Irish Haemophilia Society
11:02 – 11:10 CEST

Q&A
11:10 – 11:20 CEST

Comparison of ISTH and WFH hemophilia treatment guidelines
Speaker: Alok Srivastava, MD, FRACP, FRCPA, FRCP – Christian Medical College
11:20 – 11:30 CEST

Q&A