Treatment outcomes in persons with severe haemophilia B in the Nordic region: The B-NORD study
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Treatment outcomes in persons with severe haemophilia B in the Nordic region : The B-NORD study. / Kihlberg, Kristina; Baghaei, Fariba; Bruzelius, Maria; Funding, Eva; Andre Holme, Pål; Lassila, Riitta; Nummi, Vuokko; Ranta, Susanna; Osooli, Mehdi; Berntorp, Erik; Astermark, Jan.
I: Haemophilia, Bind 27, Nr. 3, 2021, s. 366-374.Publikation: Bidrag til tidsskrift › Tidsskriftartikel › Forskning › fagfællebedømt
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TY - JOUR
T1 - Treatment outcomes in persons with severe haemophilia B in the Nordic region
T2 - The B-NORD study
AU - Kihlberg, Kristina
AU - Baghaei, Fariba
AU - Bruzelius, Maria
AU - Funding, Eva
AU - Andre Holme, Pål
AU - Lassila, Riitta
AU - Nummi, Vuokko
AU - Ranta, Susanna
AU - Osooli, Mehdi
AU - Berntorp, Erik
AU - Astermark, Jan
PY - 2021
Y1 - 2021
N2 - 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.
AB - 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.
KW - adherence
KW - arthropathy
KW - coagulation factor IX
KW - haemophilia B
KW - joint score
KW - phenotype
KW - ultrasound
U2 - 10.1111/hae.14299
DO - 10.1111/hae.14299
M3 - Journal article
C2 - 33780113
AN - SCOPUS:85103223406
VL - 27
SP - 366
EP - 374
JO - Haemophilia, Supplement
JF - Haemophilia, Supplement
SN - 1355-0691
IS - 3
ER -
ID: 259621644