Patient-Centred Management of Well-Controlled Haemophilia: Obtaining Opinions and Definitions Through a Delphi Consensus

This is the authors' abstract. We don't add key points for this paper.

Background/Objectives: For people with haemophilia, health-related quality of life mainly depends on the arthropathy caused by repeated joint bleeding. Prophylaxis is the standard of care in patients with severe bleeding phenotypes, but globally, none of the measures used to assess patients’ outcomes consider their desires and life expectations. We propose the concept of the “patient-centred management of well-controlled haemophilia” to define individual responses to prophylaxis. The aims of this work are (1) to achieve agreement about the definition of the “patient-centred management of well-controlled haemophilia” by a steering committee of experts, and (2) to share a series of statements that should define the “patient-centred management of well-controlled haemophilia” with other haemophilia clinicians looking for a consensus in this scenario. Methods: An eight-expert group was established to define the concept of the “patient-centred management of well-controlled haemophilia”. Seven major aspects were identified, and a final version of 42 statements was established and distributed to a 75-expert panel for consensus gathering using the Delphi methodology. Results: Forty-eight experts participated in the first round (participation rate: 64%); two sentences from domain 3 were split, leading to a total of 44 statements across the seven domains. Consensus was achieved in 92.85% of cases. Five items and three statements advanced to the second round. Eleven statements were reconsidered in the second round (response rate: 100%). The questionnaire presented high internal consistency. Conclusions: New treatments offer promising solutions for patients, but there is a paucity of models to measure global outcomes. Patient-centred medicine requires multidimensional assessment, and the “patient-centred management of well-controlled haemophilia” concept is intended to enable this.