Impact of haemophilia on patients with mild‐to‐moderate disease: Results from the P‐FiQ and B‐HERO‐S studies

Abstract

Multiple studies have been conducted to determine the psychosocial and physical impact of haemophilia on patients and their caregivers. 1-9 Many of these studies focused only on men or severe haemophilia 1,3,4,6-9 ; however, epidemiologic studies suggest that joint bleeding occurs in patients with mild-to-moderate haemophilia, 10-12 including women and girls. 13-15 Importantly, some bleeds result in impairments in joint function and significantly influence quality of life. 16-18

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

“…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needs of this population and developing potential solutions following a discussion of pertinent data and findings (some unpublished), including the Pain, Functional Impairment, and Quality of Life (P-FiQ), 9,10 the Bridging Hemophilia B Experiences, Results and Opportunities Into Solutions (B-HERO-S), [10][11][12][13][14] the Community Having Opportunity to Influence Care Equity (CHOICE) (unpublished data), and the Patient Reported Outcomes, Burdens, and Experiences (PROBE) 15 studies.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.…”

“…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.…”

“…when urgent situations arise; many women who participated in the B-HERO-S survey reported issues with access to treatment. 10,14 In addition, many centres may not have specific clinics to focus on addressing comprehensive care of girls and women, including adolescent and adult gynaecology. In some circumstances, antifibrinolytic medication, desmopressin, and/or hormone therapy regulation can be used to help control heavy menstrual bleeding.…”

“…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.…”

“…HTC teams could be further trained on unique long-term (life span) issues in mild-to-moderate haemophilia identified by epidemiologic studies and recent health-related quality of life surveys, such as decreased joint range of motion and functional impairment. 10,27 This may help to address the discrepancy between current diagnostic criteria and joint outcomes observed in this group of patients and develop more focused comprehensive care approaches. In addition, HTCs could undertake a targeted analysis of patient satisfaction survey outcomes, such as the National Patient Satisfaction Survey, 28 specific to male and female patients with mild-to-moderate haemophilia.…”