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

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http://hdl.handle.net/10138/340344

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Kihlberg , K , Baghaei , F , Bruzelius , M , Funding , E , Holme , P A , Lassila , R , Nummi , V , Ranta , S , Osooli , M , Berntorp , E & Astermark , J 2021 , ' Treatment outcomes in persons with severe haemophilia B in the Nordic region : The B-NORD study ' , Haemophilia , vol. 27 , no. 3 , pp. 366-374 . https://doi.org/10.1111/hae.14299

Title: Treatment outcomes in persons with severe haemophilia B in the Nordic region : The B-NORD study
Author: Kihlberg, Kristina; Baghaei, Fariba; Bruzelius, Maria; Funding, Eva; Holme, Pål Andre; Lassila, Riitta; Nummi, Vuokko; Ranta, Susanna; Osooli, Mehdi; Berntorp, Erik; Astermark, Jan
Contributor organization: HUS Comprehensive Cancer Center
Clinicum
Department of Oncology
Hematologian yksikkö
Research Program in Systems Oncology
Faculty of Medicine
University of Helsinki
Date: 2021-05
Language: eng
Number of pages: 9
Belongs to series: Haemophilia
ISSN: 1351-8216
DOI: https://doi.org/10.1111/hae.14299
URI: http://hdl.handle.net/10138/340344
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 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.
Subject: adherence
arthropathy
coagulation factor IX
haemophilia B
joint score
phenotype
ultrasound
PHASE-3
REGIMENS
ADHERENCE
DEFICIENCY
CHILDREN
PROPHYLAXIS
RECOMBINANT FACTOR-IX
FUSION PROTEIN
INHIBITORS
LESS SEVERE
3122 Cancers
Peer reviewed: Yes
Rights: cc_by_nc_nd
Usage restriction: openAccess
Self-archived version: publishedVersion


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