Recombinant factor IX Fc prophylaxis reduces pain and increases levels of physical activity, with sustained, long-term improvements in patients with hemophilia B : post hoc analysis of phase III trials using patient-reported outcomes
(2023) In Therapeutic advances in hematology 14.- Abstract
Background: Pain is a common symptom of hemophilia that may adversely affect patients’ quality of life (QoL). Previous post hoc analyses of prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc) have been published for adults and adolescents, demonstrating improvements in health-related QoL (HRQoL) when assessed by the haemophilia-specific QoL (HaemAQoL) questionnaire. Objective: To describe in depth the evolution of QoL, pain- and activity-related domains and questions for pediatric, adolescent, and adult patients with hemophilia B treated with rFIXFc prophylaxis. Design: A post hoc analysis of data from a series of clinical trials. Methods: This post hoc, long-term analysis assessed patient-reported outcomes (PROs) from the... (More)
Background: Pain is a common symptom of hemophilia that may adversely affect patients’ quality of life (QoL). Previous post hoc analyses of prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc) have been published for adults and adolescents, demonstrating improvements in health-related QoL (HRQoL) when assessed by the haemophilia-specific QoL (HaemAQoL) questionnaire. Objective: To describe in depth the evolution of QoL, pain- and activity-related domains and questions for pediatric, adolescent, and adult patients with hemophilia B treated with rFIXFc prophylaxis. Design: A post hoc analysis of data from a series of clinical trials. Methods: This post hoc, long-term analysis assessed patient-reported outcomes (PROs) from the Kids B-LONG (NCT01440946: pediatric) and B-LONG (NCT01027364: adults and adolescents) parent studies and the B-YOND (NCT01425723: all age groups) extension study. Results: Ninety-two adult and adolescent patients that started in the B-LONG study were assessed, with a median (range) duration of follow-up of 58.9 (0.0–78.4) months. The Haem-A-QoL total score was significantly reduced from baseline by 4.45 (p ⩽ 0.01), as were the subdomains ‘physical health’ (9.10; p = 0.001), ‘sports and leisure’ (11.25; p ⩽ 0.01), ‘treatment’ (2.69; p = 0.05), and ‘view of self’ (5.81; p = 0.002). Thirty pediatric patients that started in the Kids B-LONG study were assessed, with a median (min–max) duration of follow-up of 36.7 (9.0–59.9) months. The high level of satisfaction demonstrated by the PROs at baseline was maintained. Conclusion: rFIXFc prophylaxis reduced perceived pain and increased levels of physical activity with sustained, long-term improvements in QoL in adult and adolescent patients with hemophilia B and maintained high QoL scores in pediatric patients.
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- author
- Astermark, Jan LU ; Hermans, Cédric ; Ezzalfani, Monia ; Sidhom, Alaeddine ; Barbier, Sylvaine ; Kragh, Nana ; Falk, Aletta and Eriksson, Daniel
- organization
- publishing date
- 2023-01-01
- type
- Contribution to journal
- publication status
- published
- subject
- keywords
- factor IX, hemophilia B, pain, patient-reported outcome measures, physical activity, quality of life, rFIXFc protein
- in
- Therapeutic advances in hematology
- volume
- 14
- publisher
- SAGE Publications
- external identifiers
-
- pmid:37283819
- scopus:85161348703
- ISSN
- 2040-6207
- DOI
- 10.1177/20406207231170701
- language
- English
- LU publication?
- yes
- id
- b425d8a1-c4ce-4fe2-bc8f-402e4c56628a
- date added to LUP
- 2023-08-24 15:46:29
- date last changed
- 2024-04-20 01:37:24
@article{b425d8a1-c4ce-4fe2-bc8f-402e4c56628a,
abstract = {{<p>Background: Pain is a common symptom of hemophilia that may adversely affect patients’ quality of life (QoL). Previous post hoc analyses of prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc) have been published for adults and adolescents, demonstrating improvements in health-related QoL (HRQoL) when assessed by the haemophilia-specific QoL (HaemAQoL) questionnaire. Objective: To describe in depth the evolution of QoL, pain- and activity-related domains and questions for pediatric, adolescent, and adult patients with hemophilia B treated with rFIXFc prophylaxis. Design: A post hoc analysis of data from a series of clinical trials. Methods: This post hoc, long-term analysis assessed patient-reported outcomes (PROs) from the Kids B-LONG (NCT01440946: pediatric) and B-LONG (NCT01027364: adults and adolescents) parent studies and the B-YOND (NCT01425723: all age groups) extension study. Results: Ninety-two adult and adolescent patients that started in the B-LONG study were assessed, with a median (range) duration of follow-up of 58.9 (0.0–78.4) months. The Haem-A-QoL total score was significantly reduced from baseline by 4.45 (p ⩽ 0.01), as were the subdomains ‘physical health’ (9.10; p = 0.001), ‘sports and leisure’ (11.25; p ⩽ 0.01), ‘treatment’ (2.69; p = 0.05), and ‘view of self’ (5.81; p = 0.002). Thirty pediatric patients that started in the Kids B-LONG study were assessed, with a median (min–max) duration of follow-up of 36.7 (9.0–59.9) months. The high level of satisfaction demonstrated by the PROs at baseline was maintained. Conclusion: rFIXFc prophylaxis reduced perceived pain and increased levels of physical activity with sustained, long-term improvements in QoL in adult and adolescent patients with hemophilia B and maintained high QoL scores in pediatric patients.</p>}},
author = {{Astermark, Jan and Hermans, Cédric and Ezzalfani, Monia and Sidhom, Alaeddine and Barbier, Sylvaine and Kragh, Nana and Falk, Aletta and Eriksson, Daniel}},
issn = {{2040-6207}},
keywords = {{factor IX; hemophilia B; pain; patient-reported outcome measures; physical activity; quality of life; rFIXFc protein}},
language = {{eng}},
month = {{01}},
publisher = {{SAGE Publications}},
series = {{Therapeutic advances in hematology}},
title = {{Recombinant factor IX Fc prophylaxis reduces pain and increases levels of physical activity, with sustained, long-term improvements in patients with hemophilia B : post hoc analysis of phase III trials using patient-reported outcomes}},
url = {{http://dx.doi.org/10.1177/20406207231170701}},
doi = {{10.1177/20406207231170701}},
volume = {{14}},
year = {{2023}},
}