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

Background: An appropriate and effective management of haemophilia is currently based on a multidimensional evaluation of treatment adequacy. Current clinical practice however is still lacking standardised tools able to combine clinical, functional, and patient-reported outcomes. In this study a structured Monitoring Tool for haemophilia A and B was developed and validated through a Delphi-based expert consensus process. This study represents an expert consensus-based validation of a monitoring framework, rather than a clinicalvalidation in patient cohorts. The tool is intended for use by haemophilia treaters during routine follow-up visits to support structured treatment reassessment. Score categories reflect the need for clinical re-evaluation or potential treatment optimisation, rather than disease severity. Methods: Italian haemophilia specialists were asked to participate to a panel over a two-round Delphi process. Experts rated the relevance of several predefined clinical domains—pharmacokinetics, bleeding episodes, joint health, adherence and quality of life (QoL)—and the individual items within each domain for patients on prophylactic or on-demand treatment. Consensus was defined by responses within an interquartile range (IQR) < 8. Section and item weights and Likert-based scoring values were used to reach a composite score between 0 and 100. Results: Consensus was achieved for all domains and items across haemophilia types and treatments, prophylaxis and on demand (Haemophilia A: 16 and 12 participants; Haemophilia B: 12 and 9, respectively). With reference to prophylaxis domains, bleeding episodes received the highest domain weight (31–32%), followed by joint health (27–29%) and adherence/QoL (21–23%) and pharmacokinetics (18–19%). For on-demand treatment, pharmacokinetics was excluded; bleeding episodes (38–40%) and joint health (35–37%) remained dominant. At the item level, dynamic joint health indicators (HJHS and HEAD-US changes) and longitudinal QoL changes consistently received the highest weights. The final scoring system categorised results as Excellent (0–25), Suboptimal (26–50), Poor (51–75), or Critical (76–100). Conclusions: The Delphi-validated Monitoring Tools provide a structured, weighted, and clinically relevant framework for assessing treatment adequacy in haemophilia A and B across prophylactic and on-demand settings. These tools allow multidimensional outcome assessment and may support a more consistent, personalised therapeutic decision-making. A prospective validation of the tool in clinical cohorts is warranted.