Integrating Clinical, Functional, and Patient-Reported Outcomes in Haemophilia Care: A Delphi-Based Consensus on a...

Area of Science:

• Hematology
• Clinical Management
• Patient Outcomes

Background:

• Effective haemophilia management requires multidimensional evaluation of treatment adequacy.
• Current clinical practice lacks standardized tools for combining clinical, functional, and patient-reported outcomes.
• A structured Monitoring Tool for haemophilia A and B was developed and validated using expert consensus.

Purpose of the Study:

• To develop and validate a structured monitoring tool for haemophilia A and B.
• To provide a framework for assessing treatment adequacy in routine follow-up visits.
• To support structured treatment reassessment and optimize therapeutic decision-making.

Main Methods:

• A two-round Delphi process involving Italian haemophilia specialists.
• Experts rated the relevance of clinical domains (pharmacokinetics, bleeding, joint health, adherence, QoL) and individual items.
• Consensus was defined by interquartile range (IQR) < 8; section/item weights and Likert scores were used to create a composite score (0-100).

Main Results:

• Consensus was achieved for all domains and items across haemophilia types and treatments.
• For prophylaxis, bleeding episodes and joint health received the highest domain weights.
• For on-demand treatment, bleeding episodes and joint health were dominant; pharmacokinetics was excluded.
• Dynamic joint health indicators and longitudinal QoL changes received the highest item weights.
• The scoring system categorized results as Excellent, Suboptimal, Poor, or Critical.

Conclusions:

• The Delphi-validated Monitoring Tools offer a structured, weighted, and clinically relevant framework for assessing haemophilia treatment adequacy.
• These tools enable multidimensional outcome assessment for more consistent, personalized therapeutic decisions.
• Prospective validation in clinical cohorts is recommended.