Non‐severe haemophilia: Is it benign? – Insights from the PROBE study

Abstract

Introduction There are limited data on the impact of haemophilia on health status and health‐related quality of life (HRQL) in people with non‐severe (mild and moderate) haemophilia. Aim To evaluate the health status of people living with mild or moderate haemophilia. Methods Data on respondents with no bleeding disorder (NoBD), mild and moderate haemophilia patients were drawn from the PROBE study. Respondents were enrolled using network patient organizations. This analysis was performed as a cross‐sectional study. Primary outcomes were reported bleeding, acute and chronic pain, activities of daily living and HRQL. Results A total of 862 respondents with NoBD (n = 173), mild (n = 102) and moderate (n = 134) haemophilia were eligible, with a median age of 33, 42 and 43, respectively. In relation to haemophilia‐related sequalae, 53% of male and 29% of female patients with mild and 83% of males with moderate haemophilia had more than 2‐3 bleeds in the last 12 months. Reporting of acute and chronic pain is less in those with NoBD compared to the mild and moderate cohorts for both genders. Multivariate analysis demonstrates significant reductions in quality of life using VAS, EQ‐5D‐5L and PROBE for males with mild and moderate haemophilia (P ≤ .001) with only PROBE indicating a significant reduction for females with mild (P = .002). Conclusion People affected by mild or moderate haemophilia report a significant HRQL impact due to haemophilia‐related bleeding. Future research is needed to identify the optimal care management of patients with mild and moderate haemophilia.

Identified unmet needs and proposed solutions in mild‐to‐moderate haemophilia: A summary of opinions from a roundtable of haemophilia experts

“…The experts convened were not intended to provide global expertise. However, some of the data presented in this supplement are global in nature, 15 and these data could inform other countries to apply the findings locally and make assessments. In addition, although the roundtable was a structured meeting, the discussions on which this paper is based report on the aggregate reflections of the group; a formal process for the adoption of a consensus was not employed.…”

“…During a roundtable meeting, participants were tasked with defining the unmet needs of this population and developing potential solutions following a discussion of pertinent data and findings (some unpublished), including the Pain, Functional Impairment, and Quality of Life (P‐FiQ), 9,10 the Bridging Hemophilia B Experiences, Results and Opportunities Into Solutions (B‐HERO‐S), 10‐14 the Community Having Opportunity to Influence Care Equity (CHOICE) (unpublished data), and the Patient Reported Outcomes, Burdens, and Experiences (PROBE) 15 studies. Select data from these studies are presented in this supplement 10,15 …”

“…Select data from these studies are presented in this supplement. 10,15 The roundtable participants met on 1 June 2019 in Chicago, Illinois for 8.5 hours. Importantly, this was a US-based roundtable meeting; thus, the following observations were developed from this viewpoint.…”

“…As reviewed in the data presented throughout this supplement, patients with haemophilia are often limited in their activities of daily living. 10,15 Pain and limited mobility can impair the ability of patients with mild-to-moderate haemophilia to complete tasks in an efficient manner and require the need for assistance with these activities. This could affect a patient's self-esteem and ultimately lead to depression.…”

Current challenges for men and women with mild‐to‐moderate haemophilia

“…This study identified issues with pain, joint range of motion and activities of daily living. Importantly, problems with access to treatment for women were also found 21 …”