PB1163 - Real-World Unmet Needs of Patients with Haemophilia A/B with or without Inhibitors: Historical Haemophilia Characteristics from Patients Entering a Non-Interventional Study

Tuesday, July 12, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Presenting Author(s)

Jerzy Windyga, MD, PhD

Professor
Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland
Warsaw, Mazowieckie, Poland

Co-Author(s)

HT

Huyen Tran

Professor
Australian Centre for Blood Diseases, Monash University
Melbourne, Victoria, Australia
TF

Teruhisa Fujii

Associate Professor
Division of Transfusion Medicine/Hemophilia Treatment Center, Hiroshima University Hospital, Hiroshima, Japan
Hiroshima, Hiroshima, Japan
CL

Chuhl-Joo Lyu

Professor
Department of Pediatrics, Yonsei University College of Medicine
Seoul, Seoul-t'ukpyolsi, Republic of Korea
LV

Laura Villarreal Martinez

Professor
Hospital Universitario Dr. José Eleuterio González
Monterrey, Nuevo Leon, Mexico
JS

Jameela Sathar

Consultant Haematologist
Department of Hematology, Hospital Ampang, Ampang, Selangor, Malaysia
Ampang, Selangor, Malaysia
OS

Oleksandra Stasyshyn

-
Institute of Blood Pathology and Transfusion Medicine, Lviv, Ukraine
Lviv, L'vivs'ka Oblast', Ukraine
NZ

Nadezhda Zozulya, PhD

Head of the Department
National Research Center for Hematology
Moscow, Moskva, Russia
Renée Brown Frandsen, MSc

Senior Statistician
Novo Nordisk A/S, Søborg, Denmark
Søborg, Hovedstaden, Denmark
CE

Christian Winther Eskelund

International Medical Manager
Novo Nordisk A/S
Søborg, Hovedstaden, Denmark
SA

Shashikant Apte

Director
Sahyadri Speciality Hospital, Pune, Maharashtra, India
Pune, Maharashtra, India
Johnny Mahlangu, MD, MMed

Professor in Haematology
University of the Witwatersrand and NHLS
Johannesburg, South Africa

Background: Many patients with haemophilia A (HA) and B (HB) continue to have unmet needs in disease management and treatment burden. Real-world evidence may help to further describe these unmet needs, ultimately improving haemophilia management.

Aims: To describe real-world unmet medical needs of patients with HA/HB with or without inhibitors using historical data.

Methods: Historical data were collected from patients entering the non-interventional study, explorer6 (NCT03741881). Male patients ≥12 years old with severe HA (FVIII activity < 1%), severe/moderate HB (FIX activity ≤2%) or HA/HB with inhibitors of any severity were included. Patients with previous/current concizumab or emicizumab treatment were excluded. Each patient was treated according their country’s standard-of-care (SoC). Historical data including demographics and treatment regimen were collected, analysed and described.

Results: In total, data from 231 patients (Figure 1) from 109 centres were collected. Median age was 28 years (range, 12–78 years). For the prophylaxis (PPX) group, the mean (median) annualised bleeding rates (ABR) for patients without inhibitors were HA 6.6 (2.0), HB 5.4 (2.0); and for patients with inhibitors HA 22.0 (9.0), HB 18.0 (10.0). For the episodic treatment group, the mean (median) ABRs for patients without inhibitors were HA 30.3 (24.0), HB 19.3 (12.0); and for patients with inhibitors HA 25.2 (14.0), HB 17.9 (18.0) (Table 1). At baseline, 65.6% of patients on episodic and 34.1% of patients on PPX had ≥1 target joint.

Conclusion(s): Here we present data from >30 countries, describing the historic haemophilia characteristics of patients with HA/HB, with or without inhibitors. Many patients had a high ABR and target joints despite receiving their country’s SoC treatment (episodic or PPX). This reflects a high unmet medical need, particularly for those receiving episodic treatment or those with inhibitors. Emerging therapy options could help improve health outcomes in these patients.