Prophylaxis use of clotting factor replacement products in people with non-severe haemophilia : A review of the literature
(2023) In Haemophilia 29(1). p.33-44- Abstract
Introduction: People with non-severe haemophilia appear to be under-treated in many countries, and this may lead to joint damage and worsen quality of life. Aim: To review literature for clotting factor replacement prophylaxis in people with non-severe haemophilia A and B (HA/HB) in relation to long-term outcomes to support clinical decision-making. Methods: A targeted literature search was performed to identify studies published between 2000 and 2021 that included prophylaxis in people with non-severe HA/HB and long-term outcomes, including annualized bleeding rates, joint health and quality of life. Results: Although eligible articles included 2737 and 2272 people with mild or moderate HA, respectively, only 22% (n = 609) and 29% (n =... (More)
Introduction: People with non-severe haemophilia appear to be under-treated in many countries, and this may lead to joint damage and worsen quality of life. Aim: To review literature for clotting factor replacement prophylaxis in people with non-severe haemophilia A and B (HA/HB) in relation to long-term outcomes to support clinical decision-making. Methods: A targeted literature search was performed to identify studies published between 2000 and 2021 that included prophylaxis in people with non-severe HA/HB and long-term outcomes, including annualized bleeding rates, joint health and quality of life. Results: Although eligible articles included 2737 and 2272 people with mild or moderate HA, respectively, only 22% (n = 609) and 29% (n = 668) reported treatment regimens. A total of 549 people with moderate HA were treated with factor replacement prophylaxis and were from high-income countries. On the contrary, nearly all people with mild HA received desmopressin (n = 599). Details of treatment regimens for women with haemophilia and people with HB were sparse. Three studies provided long-term outcomes for people with moderate haemophilia who received prophylaxis with factor concentrate, supporting early prophylaxis in people with a frequent bleeding phenotype regardless of their endogenous clotting factor level to preserve joint health. Conclusion: There remain large knowledge gaps when considering how to provide optimal treatment for people with non-severe haemophilia. Nonetheless, there is a strong rationale that prophylaxis should be considered early in life according to similar strategies as for severe haemophilia for those with a frequent severe bleeding phenotype.
(Less)
- author
- Iorio, Alfonso ; Königs, Christoph ; Reding, Mark T. ; Rotellini, Dawn ; Skinner, Mark W. ; Mancuso, Maria Elisa and Berntorp, Erik LU
- organization
- publishing date
- 2023
- type
- Contribution to journal
- publication status
- published
- subject
- keywords
- arthropathy, bleeding, episodic/on-demand treatment, non-severe (mild or moderate) haemophilia A or B, prophylactic FVIII or FIX, women with haemophilia (symptomatic carriers)
- in
- Haemophilia
- volume
- 29
- issue
- 1
- pages
- 33 - 44
- publisher
- Wiley-Blackwell
- external identifiers
-
- scopus:85139714802
- pmid:36224704
- ISSN
- 1351-8216
- DOI
- 10.1111/hae.14676
- language
- English
- LU publication?
- yes
- id
- edf6e4ca-d655-4b29-9ae3-2eef6f0f9271
- date added to LUP
- 2022-12-13 14:29:49
- date last changed
- 2024-06-13 21:35:43
@article{edf6e4ca-d655-4b29-9ae3-2eef6f0f9271,
abstract = {{<p>Introduction: People with non-severe haemophilia appear to be under-treated in many countries, and this may lead to joint damage and worsen quality of life. Aim: To review literature for clotting factor replacement prophylaxis in people with non-severe haemophilia A and B (HA/HB) in relation to long-term outcomes to support clinical decision-making. Methods: A targeted literature search was performed to identify studies published between 2000 and 2021 that included prophylaxis in people with non-severe HA/HB and long-term outcomes, including annualized bleeding rates, joint health and quality of life. Results: Although eligible articles included 2737 and 2272 people with mild or moderate HA, respectively, only 22% (n = 609) and 29% (n = 668) reported treatment regimens. A total of 549 people with moderate HA were treated with factor replacement prophylaxis and were from high-income countries. On the contrary, nearly all people with mild HA received desmopressin (n = 599). Details of treatment regimens for women with haemophilia and people with HB were sparse. Three studies provided long-term outcomes for people with moderate haemophilia who received prophylaxis with factor concentrate, supporting early prophylaxis in people with a frequent bleeding phenotype regardless of their endogenous clotting factor level to preserve joint health. Conclusion: There remain large knowledge gaps when considering how to provide optimal treatment for people with non-severe haemophilia. Nonetheless, there is a strong rationale that prophylaxis should be considered early in life according to similar strategies as for severe haemophilia for those with a frequent severe bleeding phenotype.</p>}},
author = {{Iorio, Alfonso and Königs, Christoph and Reding, Mark T. and Rotellini, Dawn and Skinner, Mark W. and Mancuso, Maria Elisa and Berntorp, Erik}},
issn = {{1351-8216}},
keywords = {{arthropathy; bleeding; episodic/on-demand treatment; non-severe (mild or moderate) haemophilia A or B; prophylactic FVIII or FIX; women with haemophilia (symptomatic carriers)}},
language = {{eng}},
number = {{1}},
pages = {{33--44}},
publisher = {{Wiley-Blackwell}},
series = {{Haemophilia}},
title = {{Prophylaxis use of clotting factor replacement products in people with non-severe haemophilia : A review of the literature}},
url = {{http://dx.doi.org/10.1111/hae.14676}},
doi = {{10.1111/hae.14676}},
volume = {{29}},
year = {{2023}},
}