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 Kuriakose; Suchitra Acharya; Ulrike M Reiss; Roshni Kulkarni; Michelle Witkop; Stefan Lethagen; Rebecca Krouse; Amy D Shapiro; Jan Astermark

doi:10.1111/hae.14525

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

Afdeling for Blodsygdomme

Abstrakt

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.

Originalsprog	Engelsk
Tidsskrift	Haemophilia
Vol/bind	28
Udgave nummer	3
Sider (fra-til)	453-461
Antal sider	9
ISSN	1351-8216
DOI	https://doi.org/10.1111/hae.14525
Status	Udgivet - maj 2022

Adgang til dokumentet

10.1111/hae.14525

Andre filer og links

Link to publication in Scopus

Citationsformater

Berntorp, E., LeBeau, P., Ragni, M. V., Borhany, M., Abajas, Y. L., Tarantino, M. D., Holstein, K., Croteau, S. E., Liesner, R., Tarango, C., Carvalho, M., McGuinn, C., Funding, E., Kempton, C. L., Bidlingmaier, C., Cohen, A., Oldenburg, J., Kearney, S., Knoll, C., ... Astermark, J. (2022). Quality of life in a large multinational haemophilia B cohort (The B-Natural study) - Unmet needs remain. Haemophilia, 28(3), 453-461. https://doi.org/10.1111/hae.14525

Berntorp, E, LeBeau, P, Ragni, MV, Borhany, M, Abajas, YL, Tarantino, MD, Holstein, K, Croteau, SE, Liesner, R, Tarango, C, Carvalho, M, McGuinn, C, Funding, E, Kempton, CL, Bidlingmaier, C, Cohen, A, Oldenburg, J, Kearney, S, Knoll, C, Kuriakose, P, Acharya, S, Reiss, UM, Kulkarni, R, Witkop, M, Lethagen, S, Krouse, R, Shapiro, AD & Astermark, J 2022, 'Quality of life in a large multinational haemophilia B cohort (The B-Natural study) - Unmet needs remain', Haemophilia, bind 28, nr. 3, s. 453-461. https://doi.org/10.1111/hae.14525

@article{3ff3f963cc5c418e9b3f9eadca66c4ca,

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

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.",

keywords = "Adolescent, Child, Child, Preschool, Cohort Studies, Hemophilia B/drug therapy, Humans, Quality of Life, Severity of Illness Index, Surveys and Questionnaires, Visual Analog Scale",

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

note = "{\textcopyright} 2022 The Authors. Haemophilia published by John Wiley & Sons Ltd.",

year = "2022",

month = may,

doi = "10.1111/hae.14525",

language = "English",

volume = "28",

pages = "453--461",

journal = "Haemophilia",

issn = "1351-8216",

publisher = "Wiley-Blackwell Publishing Ltd",

number = "3",

}

TY - JOUR

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

PY - 2022/5

Y1 - 2022/5

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 - Adolescent

KW - Child

KW - Child, Preschool

KW - Cohort Studies

KW - Hemophilia B/drug therapy

KW - Humans

KW - Quality of Life

KW - Severity of Illness Index

KW - Surveys and Questionnaires

KW - Visual Analog Scale

UR - http://www.scopus.com/inward/record.url?scp=85126024130&partnerID=8YFLogxK

U2 - 10.1111/hae.14525

DO - 10.1111/hae.14525

M3 - Journal article

C2 - 35263495

VL - 28

SP - 453

EP - 461

JO - Haemophilia

JF - Haemophilia

SN - 1351-8216

IS - 3

ER -

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

Abstrakt

Adgang til dokumentet

Andre filer og links

Fingeraftryk

Citationsformater