rFIXFc prophylaxis improves pain and levels of physical activity in haemophilia B : Post hoc analysis of B-LONG using haemophilia-specific quality of life questionnaires
(2022) In Haemophilia 28(1). p.18-26- Abstract
Introduction: Recurrent bleeding in severe haemophilia B causes painful hemarthroses and reduces capacity for physical activity. Recombinant factor IX Fc fusion protein (rFIXFc) prophylaxis results in low annualised bleeding rates, with the potential to improve patients’ health-related quality of life (HRQoL). Aim: To present a post hoc analysis of data from B-LONG describing change over time in patient-reported outcomes associated with pain and physical activity. Methods: Patients (≥12 years) who received weekly dose-adjusted or interval-adjusted rFIXFc prophylaxis and completed the Haemophilia-Specific QoL questionnaire for adolescents (Haemo-QoL) or adults (Haem-A-QoL) at baseline (BL) and end of study (EoS). Individual level changes... (More)
Introduction: Recurrent bleeding in severe haemophilia B causes painful hemarthroses and reduces capacity for physical activity. Recombinant factor IX Fc fusion protein (rFIXFc) prophylaxis results in low annualised bleeding rates, with the potential to improve patients’ health-related quality of life (HRQoL). Aim: To present a post hoc analysis of data from B-LONG describing change over time in patient-reported outcomes associated with pain and physical activity. Methods: Patients (≥12 years) who received weekly dose-adjusted or interval-adjusted rFIXFc prophylaxis and completed the Haemophilia-Specific QoL questionnaire for adolescents (Haemo-QoL) or adults (Haem-A-QoL) at baseline (BL) and end of study (EoS). Individual level changes in items of the ‘Physical Health’ and ‘Sports and Leisure’ domains, categorised as ‘never/rarely/seldom’ or ‘sometimes/often/all the time’, were analysed using McNemar's test to compare distribution of responses at EoS versus BL. Results: At EoS versus BL, a significantly greater proportion of patients did not experience painful swellings (64% vs. 44%; P =.004), painful joints (44% vs. 28%; P =.003) or pain when moving (54% vs. 41%; P =.026). Additionally, at EoS versus BL, patients were less likely to avoid participating in sports like football (30% vs. 8%; P =.002), avoid sports due to their haemophilia (47% vs. 27%; P =.007), or experience difficulty walking as far as they wanted (63% vs. 43%; P =.001). The proportion of patients who played sports as much as the general population was numerically increased (52% vs. 37%; P =.033) at EoS versus BL. Conclusion: Results of the analysis suggest that over time, rFIXFc prophylaxis is associated with significant improvements in pain and physical functioning. This contributes to previous evidence of overall HRQoL improvements in patients with haemophilia B treated with rFIXFc.
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- author
- Astermark, Jan LU ; Hermans, Cédric ; Ezzalfani, Monia ; Aballéa, Samuel ; Santagostino, Elena ; Hakimi, Zalmai and Nazir, Jameel
- organization
- publishing date
- 2022
- type
- Contribution to journal
- publication status
- published
- subject
- keywords
- factor IX, haemophilia B, Pain, patient reported outcomes measures, physical activity, quality of Life, rFIXFc protein
- in
- Haemophilia
- volume
- 28
- issue
- 1
- pages
- 18 - 26
- publisher
- Wiley-Blackwell
- external identifiers
-
- scopus:85118879107
- pmid:34761474
- ISSN
- 1351-8216
- DOI
- 10.1111/hae.14455
- language
- English
- LU publication?
- yes
- additional info
- Publisher Copyright: © 2021 The Authors. Haemophilia published by John Wiley & Sons Ltd.
- id
- 7194cd08-c40f-4d3d-b7c9-8a86587fb184
- date added to LUP
- 2021-12-02 15:36:08
- date last changed
- 2024-04-20 18:06:09
@article{7194cd08-c40f-4d3d-b7c9-8a86587fb184,
abstract = {{<p>Introduction: Recurrent bleeding in severe haemophilia B causes painful hemarthroses and reduces capacity for physical activity. Recombinant factor IX Fc fusion protein (rFIXFc) prophylaxis results in low annualised bleeding rates, with the potential to improve patients’ health-related quality of life (HRQoL). Aim: To present a post hoc analysis of data from B-LONG describing change over time in patient-reported outcomes associated with pain and physical activity. Methods: Patients (≥12 years) who received weekly dose-adjusted or interval-adjusted rFIXFc prophylaxis and completed the Haemophilia-Specific QoL questionnaire for adolescents (Haemo-QoL) or adults (Haem-A-QoL) at baseline (BL) and end of study (EoS). Individual level changes in items of the ‘Physical Health’ and ‘Sports and Leisure’ domains, categorised as ‘never/rarely/seldom’ or ‘sometimes/often/all the time’, were analysed using McNemar's test to compare distribution of responses at EoS versus BL. Results: At EoS versus BL, a significantly greater proportion of patients did not experience painful swellings (64% vs. 44%; P =.004), painful joints (44% vs. 28%; P =.003) or pain when moving (54% vs. 41%; P =.026). Additionally, at EoS versus BL, patients were less likely to avoid participating in sports like football (30% vs. 8%; P =.002), avoid sports due to their haemophilia (47% vs. 27%; P =.007), or experience difficulty walking as far as they wanted (63% vs. 43%; P =.001). The proportion of patients who played sports as much as the general population was numerically increased (52% vs. 37%; P =.033) at EoS versus BL. Conclusion: Results of the analysis suggest that over time, rFIXFc prophylaxis is associated with significant improvements in pain and physical functioning. This contributes to previous evidence of overall HRQoL improvements in patients with haemophilia B treated with rFIXFc.</p>}},
author = {{Astermark, Jan and Hermans, Cédric and Ezzalfani, Monia and Aballéa, Samuel and Santagostino, Elena and Hakimi, Zalmai and Nazir, Jameel}},
issn = {{1351-8216}},
keywords = {{factor IX; haemophilia B; Pain; patient reported outcomes measures; physical activity; quality of Life; rFIXFc protein}},
language = {{eng}},
number = {{1}},
pages = {{18--26}},
publisher = {{Wiley-Blackwell}},
series = {{Haemophilia}},
title = {{rFIXFc prophylaxis improves pain and levels of physical activity in haemophilia B : Post hoc analysis of B-LONG using haemophilia-specific quality of life questionnaires}},
url = {{http://dx.doi.org/10.1111/hae.14455}},
doi = {{10.1111/hae.14455}},
volume = {{28}},
year = {{2022}},
}