Quality of life in a large multinational haemophilia B cohort (The B-Natural study) - Unmet needs remain

Erik Berntorp, Petra LeBeau, Margaret V Ragni, Munira Borhany, Yasmina L Abajas, Michael D Tarantino, Katharina Holstein, Stacy E Croteau, Raina Liesner, Cristina Tarango, Manuela Carvalho, Catherine McGuinn, Eva Funding, Christine L Kempton, Christoph Bidlingmaier, Alice Cohen, Johannes Oldenburg, Susan Kearney, Christine Knoll, Philip KuriakoseSuchitra Acharya, Ulrike M Reiss, Roshni Kulkarni, Michelle Witkop, Stefan Lethagen, Rebecca Krouse, Amy D Shapiro, Jan Astermark

Abstract

INTRODUCTION: The B-Natural study is a multicentre, multinational, observational study of haemophilia B (HB) designed to increase understanding of clinical manifestations, treatment and quality of life (QoL).

AIM: To characterise and compare QoL in HB across disease severity groups and individuals with inhibitors to identify gaps in treatment.

METHODS: A total of 224 individuals from 107 families were enrolled from a total of 24 centres in North America (n = 16), Europe (n = 7) and Asia (n = 1). Of these, 68 (30.4%) subjects had severe (<1 IU/dL), median age 15.6 years, 114 (50.9%) moderate (1-5 IU/dL), age 13.3 years, and 42 (18.8%) mild (>5-< 40 IU/dL), age 12.1 years, disease. Twenty-nine participants had inhibitors or a history of inhibitors. Three versions of the EQ-5D instrument were used as a measure of QoL: proxy (ages 4-7), youth (ages 8-15) and self (age 16+). Each instrument included a visual analogue scale ranging from 100 (best health) to 0 (worst health) to assess current day's health (EQ VAS). Range-of-motion (ROM) for elbows, knees and ankles was assessed using a four-point scale, from which a composite score was calculated.

RESULTS: In all severity groups, a proportion of subjects showed less than optimal QoL. The majority of the mild and moderate severe participants reported a normal EQ-5D health profile (79% and 72%, respectively), whereas about half (47%) of the severe participants and only 13% of the inhibitor participants reported this profile.

CONCLUSION: The B-Natural study reveals impacted QoL in all disease severities of HB including those with inhibitors. Unmet needs remain and include nonsevere HB.

OriginalsprogEngelsk
TidsskriftHaemophilia
Vol/bind28
Udgave nummer3
Sider (fra-til)453-461
Antal sider9
ISSN1351-8216
DOI
StatusUdgivet - maj 2022

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