PB1282 - Real-World Unmet Needs Of Adult Patients With Severe Hemophilia A Without Inhibitors In Poland: How To Select The Optimal Therapy?

Tuesday, June 27, 2023

18:30 – 19:30 ET

Room: Exhibition

Presenting Author(s)

Jerzy Windyga, Prof. dr med. (he/him/his)

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

Co-Author(s)

JZ

Joanna Zdziarska

Assistant Department of Hematology
Jagiellonian University Medical College
Cracow, Malopolskie, Poland
ES

Ewa Stefanska-Windyga

Head Outpatient Clinic for Persons with Disorders of Hemostasis
Institute of Hematology and Transfusion Medicine
Warsaw, Mazowieckie, Poland
WN

Weronika Nowak

Assistant Institute of Hemostasis, Department of Hematology
Medical University of Lodz
Lodz, Lodzkie, Poland
JK

Justyna Kozinska

Assistant Department and Clinic of Hemato-oncology and Bone Marrow Transplantation
Medical University of Lublin
Lublin, Lubelskie, Poland
JR

Joanna Rupa-Matysek

Assistant Prof. Department of Hematology and Bone Marrow Transplantation
Poznan University of Medical Sciences
Poznań, Wielkopolskie, Poland
AM

Andrzej Mital

Assistant Prof. Department of Hematology and Transplantology
Medical University of Gdansk
Gdańsk, Pomorskie, Poland
AS

Anna Sikorska

Assistant Outpatient Clinic for Persons with Disorders of Hemostasis
Institute of Hematology and Transfusion Medicine
Warsaw, Mazowieckie, Poland
JT

Jacek Trelinski

Head Institute of Hemostasis, Department of Hematology
Medical University of Lodz
Lodz, Lodzkie, Poland

Background: The standard of care for people with severe hemophilia A without inhibitors (PwsHAw/o-inh) is long-term prophylaxis (LT-PPX) with factor VIII (FVIII) or emicizumab. The most recent Polish data revealed that emicizumab had favourable efficacy profile as compared with previously used bypassing agents (BPAs) in 34 adult patients with hemophilia A complicated by FVIII inhibitor (PwHAinh) and led to annualized bleeding rate (ABR) reduction from 24 to 0.28 (p < 0.0001). However, due to much higher costs of emicizumab as compared with FVIII concentrates, in contrast to PwHAinh, emicizumab is not used in PwsHAw/o-inh in Poland.

Aims: To identify the key unmet medical needs in PwsHAw/o-inh treated with FVIII concentrates.

Methods: The study was carried out in six Hemophilia Treatment Centres (HTC) managing 75% (690/915) of all PwsHAw/o-inh in Poland. Healthcare professionals from these HTCs provided data on PwsHAw/o-inh using specifically designed electronic case report forms (eCRF).

Results: In total data from 120 patients aged 19-80 (median 45) years were collected. 93/120 (78%) received LT-PPX and 27 (23%) were treated on-demand. Within 12 months prior to study entry, 77/120 (64%) patients experienced bleeds requiring treatment, including 41/77 (53%) with 6 or more annual bleeds requiring treatment. 35/120 (29%) of patients reported difficult peripheral venous access. The most common comorbidity was hemophilic arthropathy reported by 108/120 (90%) of studied patients, followed by hepatitis C (66/120, 55%) and arterial hypertension (40/120, 33%). Among 79/120 (66%) of professionally active patients, 31 (39%) reported hemophilia-associated absenteeism from work (on average 10±7 days/per year).

Conclusion(s): The results of the current study revealed that replacement therapy with FVIII is suboptimal in a significant proportion of adult patients with severe hemophilia A without inhibitors in Poland. We believe that PwsHAw/o-inh reporting difficult venous access and/or high ABR while on FVIII long-term prophylaxis could benefit from switching to emicizumab.