Quality of life in a large multinational haemophilia B cohort (The B-Natural study) – Unmet needs remain
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Quality of life in a large multinational haemophilia B cohort (The B-Natural study) – Unmet needs remain. / Berntorp, Erik; LeBeau, Petra; Ragni, Margaret V.; Borhany, Munira; Abajas, Yasmina L.; Tarantino, Michael D.; Holstein, Katharina; Croteau, Stacy E.; Liesner, Raina; Tarango, Cristina; Carvalho, Manuela; McGuinn, Catherine; Funding, Eva; Kempton, Christine L.; Bidlingmaier, Christoph; Cohen, Alice; Oldenburg, Johannes; Kearney, Susan; Knoll, Christine; Kuriakose, Philip; Acharya, Suchitra; Reiss, Ulrike M.; Kulkarni, Roshni; Witkop, Michelle; Lethagen, Stefan; Krouse, Rebecca; Shapiro, Amy D.; Astermark, Jan.
I: Haemophilia, Bind 28, Nr. 3, 2022, s. 453-461.Publikation: Bidrag til tidsskrift › Tidsskriftartikel › Forskning › fagfællebedømt
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T1 - Quality of life in a large multinational haemophilia B cohort (The B-Natural study) – Unmet needs remain
AU - Berntorp, Erik
AU - LeBeau, Petra
AU - Ragni, Margaret V.
AU - Borhany, Munira
AU - Abajas, Yasmina L.
AU - Tarantino, Michael D.
AU - Holstein, Katharina
AU - Croteau, Stacy E.
AU - Liesner, Raina
AU - Tarango, Cristina
AU - Carvalho, Manuela
AU - McGuinn, Catherine
AU - Funding, Eva
AU - Kempton, Christine L.
AU - Bidlingmaier, Christoph
AU - Cohen, Alice
AU - Oldenburg, Johannes
AU - Kearney, Susan
AU - Knoll, Christine
AU - Kuriakose, Philip
AU - Acharya, Suchitra
AU - Reiss, Ulrike M.
AU - Kulkarni, Roshni
AU - Witkop, Michelle
AU - Lethagen, Stefan
AU - Krouse, Rebecca
AU - Shapiro, Amy D.
AU - Astermark, Jan
N1 - Publisher Copyright: © 2022 The Authors. Haemophilia published by John Wiley & Sons Ltd.
PY - 2022
Y1 - 2022
N2 - 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.
AB - 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.
KW - EQ-5D
KW - FIX
KW - haemophilia B
KW - inhibitor
KW - prophylaxis
KW - QoL
U2 - 10.1111/hae.14525
DO - 10.1111/hae.14525
M3 - Journal article
C2 - 35263495
AN - SCOPUS:85126024130
VL - 28
SP - 453
EP - 461
JO - Haemophilia
JF - Haemophilia
SN - 1351-8216
IS - 3
ER -
ID: 307817572