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Treatment outcomes in persons with severe haemophilia B in the Nordic region : The B-NORD study

Kihlberg, Kristina LU ; Baghaei, Fariba ; Bruzelius, Maria ; Funding, Eva LU ; Andre Holme, Pål ; Lassila, Riitta ; Nummi, Vuokko LU ; Ranta, Susanna ; Osooli, Mehdi LU orcid and Berntorp, Erik LU , et al. (2021) In Haemophilia 27(3). p.366-374
Abstract

Introduction: Data on outcome in persons with haemophilia B (PwHB) are limited and mainly extrapolated from studies of haemophilia A (HA). Aim: To characterize treatment outcomes in persons with severe HB in the Nordic region, with a focus on joint health, compared with matched controls with HA. Methods: PwHB attending haemophilia centres in Denmark, Finland, Norway and Sweden were enrolled and matched with controls with HA. Joint assessment using Haemophilia Joint Health Score (HJHS) and ultrasound according to Haemophilia Early Arthropathy Detection protocol (HEAD-US) was conducted. Adherence was evaluated using the Validated Haemophilia Regimen Treatment Adherence Scale (VERITAS). Results: Seventy-nine males with HB, with median age... (More)

Introduction: Data on outcome in persons with haemophilia B (PwHB) are limited and mainly extrapolated from studies of haemophilia A (HA). Aim: To characterize treatment outcomes in persons with severe HB in the Nordic region, with a focus on joint health, compared with matched controls with HA. Methods: PwHB attending haemophilia centres in Denmark, Finland, Norway and Sweden were enrolled and matched with controls with HA. Joint assessment using Haemophilia Joint Health Score (HJHS) and ultrasound according to Haemophilia Early Arthropathy Detection protocol (HEAD-US) was conducted. Adherence was evaluated using the Validated Haemophilia Regimen Treatment Adherence Scale (VERITAS). Results: Seventy-nine males with HB, with median age of 30 years (range 1–75), were enrolled. Eleven patients (14%) had a history of or current inhibitor. Twenty-nine PwHB (37%) reported joint bleeds during the prior year, and 35% had previously undergone joint surgery. Ninety-five per cent were on prophylaxis, and 70% used recombinant concentrates, with a median factor consumption of 3,900 IU/kg/year for standard half-life products. Only two patients had a VERITAS score corresponding to ‘non-adherence'. Joint health, assessed with HJHS, showed a significant lower score among PwHB compared with HA controls, explained by a difference in the 18–49 age group, without observed differences in older or younger subgroups. The HEAD-US scores were overall low. Conclusion: The Nordic cohort of PwHB is well treated by prophylaxis, but the goal of zero bleeds for all is not reached. Our findings suggest that patients with severe HB suffer from a milder arthropathy than patients with severe HA.

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organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
adherence, arthropathy, coagulation factor IX, haemophilia B, joint score, phenotype, ultrasound
in
Haemophilia
volume
27
issue
3
pages
9 pages
publisher
Wiley-Blackwell
external identifiers
  • pmid:33780113
  • scopus:85103223406
ISSN
1351-8216
DOI
10.1111/hae.14299
language
English
LU publication?
yes
id
7f955723-bfd9-4465-96ab-9aafca9371d2
date added to LUP
2021-04-08 12:11:58
date last changed
2024-05-04 05:33:46
@article{7f955723-bfd9-4465-96ab-9aafca9371d2,
  abstract     = {{<p>Introduction: Data on outcome in persons with haemophilia B (PwHB) are limited and mainly extrapolated from studies of haemophilia A (HA). Aim: To characterize treatment outcomes in persons with severe HB in the Nordic region, with a focus on joint health, compared with matched controls with HA. Methods: PwHB attending haemophilia centres in Denmark, Finland, Norway and Sweden were enrolled and matched with controls with HA. Joint assessment using Haemophilia Joint Health Score (HJHS) and ultrasound according to Haemophilia Early Arthropathy Detection protocol (HEAD-US) was conducted. Adherence was evaluated using the Validated Haemophilia Regimen Treatment Adherence Scale (VERITAS). Results: Seventy-nine males with HB, with median age of 30 years (range 1–75), were enrolled. Eleven patients (14%) had a history of or current inhibitor. Twenty-nine PwHB (37%) reported joint bleeds during the prior year, and 35% had previously undergone joint surgery. Ninety-five per cent were on prophylaxis, and 70% used recombinant concentrates, with a median factor consumption of 3,900 IU/kg/year for standard half-life products. Only two patients had a VERITAS score corresponding to ‘non-adherence'. Joint health, assessed with HJHS, showed a significant lower score among PwHB compared with HA controls, explained by a difference in the 18–49 age group, without observed differences in older or younger subgroups. The HEAD-US scores were overall low. Conclusion: The Nordic cohort of PwHB is well treated by prophylaxis, but the goal of zero bleeds for all is not reached. Our findings suggest that patients with severe HB suffer from a milder arthropathy than patients with severe HA.</p>}},
  author       = {{Kihlberg, Kristina and Baghaei, Fariba and Bruzelius, Maria and Funding, Eva and Andre Holme, Pål and Lassila, Riitta and Nummi, Vuokko and Ranta, Susanna and Osooli, Mehdi and Berntorp, Erik and Astermark, Jan}},
  issn         = {{1351-8216}},
  keywords     = {{adherence; arthropathy; coagulation factor IX; haemophilia B; joint score; phenotype; ultrasound}},
  language     = {{eng}},
  month        = {{05}},
  number       = {{3}},
  pages        = {{366--374}},
  publisher    = {{Wiley-Blackwell}},
  series       = {{Haemophilia}},
  title        = {{Treatment outcomes in persons with severe haemophilia B in the Nordic region : The B-NORD study}},
  url          = {{http://dx.doi.org/10.1111/hae.14299}},
  doi          = {{10.1111/hae.14299}},
  volume       = {{27}},
  year         = {{2021}},
}