PB0654 - Combined Point of Care Tools Improve Treatment Adherence and Health-Related Quality of Life in Patients with Hemophilia under regular prophylaxis: an Observational Mulicenter Prospective Study

Monday, June 26, 2023

18:30 – 19:30 ET

Room: Exhibition

Presenting Author(s)

Mariasanta Napolitano, 310885

Associate Professor of hematology
Department of Health Promotion, Mother and Child Care, Internal Medicine and Medical Specialties (ProMISE), University of Palermo, Palermo, Italy
Palerno, Sicilia, Italy

Co-Author(s)

MD

Matteo Nicola Dario Di Minno

full professor
Department of Clinical Medicine and Surgery, Federico II University
Napoli, Campania, Italy
IC

Ilenia Lorenza Calcaterra

Assistant professor
Department of Clinical Medicine and Surgery, Federico II University
Napoli, Campania, Italy
rm

renato marino

MD
University Hospital of Bari
bari, Puglia, Italy
gm

giuseppe malcangi

MD
university hospital of Bari
bari, Puglia, Italy
MM

Marta Mattana

PhD student
University of Palermo
Palermo, Sicilia, Italy
Simona Raso, MD,PhD (she/her/hers)

MD, PhD
Department of Hematology and Rare Diseases, Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello, Palermo, Italy.
Palermo, Sicilia, Italy
GG

Gaetano Giffrida

MD
university hospital of Catania
Catania, Sicilia, Italy
mM

maria Francesca Mansueto

Dr
University Hospital "P. Giaccone", Palermo
Palermo, Sicilia, Italy
DS

Dalila Scaturro

Assistant Professor
Department of Surgical, Oncological and Stomatologica Science,University of Palermo
Palermo, Sicilia, Italy
GM

Giulia Letizia Mauro

Full professor
Department of Surgical, Oncological and Stomatologica Science, University of Palermo
Palermo, Sicilia, Italy
SS

Sergio Siragusa

Professor of Hematology
Haematology Unit, Thrombosis and Haemostasis Reference Regional Center, University of Palermo, Palermo, Italy
Palermo, Sicilia, Italy

Background: Assessment of joints by ultrasounds (US) is a point of care tool for the detection of early joint arthropathy; population pharmacokinetic (pop-PK) studies allow to optimise prophylaxis regimens, these tools may improve adherence to haemophilia treatment contributing to reduce bleeding rates . Adherence to continuous intravenous replacement therapy (RT) is challenging, extended half life (EHL) molecules have been developed also to overcome this limit.

Aims: Aim of the current prospective multicenter study was to evaluate the impact of combined US assessment and pop-PK study on adherence to treatment and health related quality of life in patients with haemophilia A(HA) and B (HB) under regular prophylaxis.

Methods: This study was performed, after IRB approval, at four tertiary centers from June 2020 to December 2022. Enrolled patients signing informed consent underwent the following: US joints evaluation calculating HEAD-US protocol, treatment adherence evaluation by VERITAS-Pro questionnaire, health -related quality of life assessment by the standardized EQ-5D,EQ-VAS and pop-PK study as needed (treatment switch, life style,weight changes); US and PK results were shared with each patient and discussed during encounters, at enrolment (T0) and at 12 (T12) and 24 (T24) months follow-up.Statistical analysis was performed using the SPSS software version 25.0 (SPSS Chicago, IL). Statistical tests were 2-sided, with a significance threshold of 0.05.

Results: Sixty males have currently completed 24 months follow-up, main results are summarised in Table 1 Thirthy two patients affected by HA and 1 with HB switched to EHL at enrollment or within T12,the remaining HB subjects were already under EHL at T0.At T24, an improved HEAD-US score, significantly higher adherence (p < 0.05)and quality of life scores(p < 0.05), were observed, in particular in patients switched to EHL.

Conclusion(s): Improved treatment adherence and quality of life is reached n patients under RT ,by promoting disaese awareness and patient education trough combined point of care tools.