PB0670 - Bleed Outcomes in the Moderate and Mild Hemophilia A Population Without Prophylactic Treatment in CHESS II and CHESS PAEDs

Monday, July 11, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Presenting Author(s)

Kate Khair, PhD

Director of Research
Haemnet
London, England, United Kingdom

Co-Author(s)

FN

Francis Nissen

Senior Data Scientist
F. Hoffmann-La Roche Ltd
Basel, Basel-Stadt, Switzerland
EF

Enrico Ferri Grazzi

Director, HEOR
HCD Economics Ltd.
Daresbury, England, United Kingdom
JO

Jamie O'Hara

Chief Executive Officer
HCD Economics
London, England, United Kingdom
RO

Richard Ofori-Asenso

Real-world Data Scientist
Roche Products Ltd.
Welwyn Garden City, England, United Kingdom
MA

Martynas Aizenas

Global Access Strategy Leader, Hematology
F. Hoffmann-La Roche Ltd
Basel, Basel-Stadt, Switzerland
HZ

Hanlu Zhang

Data Scientist
F. Hoffmann-La Roche Ltd.
Basel, Basel-Stadt, Switzerland
KM

Katya Moreno

Therapeutic Area Head Hematology and Global Product Development / Medical Affairs
F. Hoffmann-La Roche Ltd
Basel, Basel-Stadt, Switzerland
TB

Tom Burke

Principal Health Economist
HCD Economics and Faculty of Health and Social Care, University of Chester
Daresbury, England, United Kingdom
BN

Beatrice Nolan

Consultant Hematologist
Children's Health Ireland at Crumlin
Dublin, Dublin, Ireland

Background: Hemophilia A (HA) is a congenital bleeding disorder caused by a deficiency in clotting factor (F)VIII and can cause uncontrolled bleeding and musculoskeletal dysfunction. Published data on the unmet clinical need in moderate and mild HA are limited.

Aims: To assess bleed outcomes in people with moderate or mild HA.

Methods: CHESS II and CHESS PAEDs are retrospective, burden-of-illness studies. CHESS II was conducted in adults (≥18 years), with hemophilia of any severity, across eight European countries. CHESS PAEDs focused on children (0–17 years), with moderate or severe hemophilia, across five European countries. Both studies obtained informed consent and ethical approval for data use. Data were captured by specialists via web-based forms. Twelve months of retrospective data on all bleeding episodes, target joints and pain were examined.

Results: In CHESS II, 174/190 adults (91.6%) with moderate HA and 92/94 adults (97.9%) with mild HA did not receive routine prophylaxis (Table 1). Of those not receiving prophylaxis: 147/174 (84.5%) with moderate HA had ≥1 bleed/year, including 60/174 (34.5%) who had ≥3 bleeds/year; 69/92 (75.0%) with mild HA had ≥1 bleed/year, including 7/92 (7.6%) who had ≥3 bleeds/year.
In CHESS PAEDs, of the 282 children with moderate HA included, 146/282 (51.8%) did not receive routine prophylaxis (Table 2); of those, 108/146 (74.0%) had ≥1 bleed/year, including 59/146 (40.4%) who had ≥3 bleeds/year.
In those not receiving prophylaxis, the number of target joints and pain severity increased with a higher number of bleeds.

Conclusion(s): Most adults with moderate or mild HA in CHESS II and more than half of children with moderate HA in CHESS PAEDs did not receive routine prophylaxis; more than one third of adults and ~40% of children with moderate HA not receiving prophylaxis experienced ≥3 bleeds/year. Additional research is warranted to investigate potential reasons for limited use of prophylaxis in these populations.