OBJECTIVES : Haemophilia management and patients’ quality of life in Italy have improved mainly due to extensive use of prophylaxis with factor concentrates and development of specialized haemophilia centers. Data on current limitations of haemophilia management and treatment are needed to assess the potential impact of new treatment approaches. This analysis evaluated the management satisfaction and unmet needs from the perspective of patients with severe haemophilia without inhibitors and their specialist physicians.

METHODS : Online surveys (for patients≥18 years, caregivers of children<18 years, and physicians) were developed by a multidisciplinary working group and conducted from November 2019 with the support of the Italian Federation of Haemophilia Associations. The surveys collected data about demographic, clinical characteristics, disease management, treatment satisfaction, quality of life, working and daily life, barriers to treatment and sources of information.

RESULTS : As of May 2020, 208 subjects (119 adults/89 caregivers) and 35 physicians completed the survey. Severe haemophilia A (SHA) was reported in 57.2% and B (SHB) in 12% of cases. In the six months preceding survey enrolment, disease control for the majority of time was reported by more than 90% of patients and by 75.8% of physicians. However, a mean(SD) 6 month treated bleeding rate of 1.7(3.5) (2.1(4.2) adult/1.1(2.3) children) was reported by SHA and 0.8(2.2) (1.1(2.9) adult/0.5(1.5) children) by SHB. Treatment adherence ≥90% was reported by 88.8% (87.5% adults/90,2% children) of SHA, 90.9% (88.9% adults/92.3% children) of SHB and 32.4% of physicians. The treatment burden (number of infusions) was considered acceptable by only 37.4% (45.0% adults/28.6% children) of SHA, 70.8% (72.8% adults/69.3% children) of SHB and 39.4% of physicians.

CONCLUSIONS : This large national study provides an updated overview of haemophilia condition, highlighting unmet needs from different point of views. This information can guide future interventions to manage haemophilia and the assessment of impact of new treatment options.