Primary prophylaxis in children with severe haemophilia A and B-Implementation over the last 20 years as illustrated in real-world data in the PedNet cohorts
Ljung, Rolf LU
; de Kovel, Marloes
and van den Berg, H Marijke
(2023)
In Haemophilia
29(2).
p.498-504
- Abstract
INTRODUCTION: The prophylactic regimen in children with severe haemophilia is suggested in various publications and guidelines. Few data exist on its implementation in clinical practice.
AIM: To investigate the implementation of primary prophylaxis based on real-life data from PedNet during the last 20 years.
METHODS: All children from the PedNet cohort (n = 1260) with severe haemophilia A (SHA) or severe haemophilia B (SHB), FVIII/IX < .01 IU/mL, born between 2000 and 2009 (Cohort I; SHA n = 662; SHB n = 88) and 2010-2019 (Cohort II; SHA n = 598; SHB n = 94) were included.
RESULTS: In SHA, the median age at start of prophylaxis was 17.3 months (IQR; 12.5-26.1) in Cohort I which decreased to 13.1 months (IQR;... (More)
INTRODUCTION: The prophylactic regimen in children with severe haemophilia is suggested in various publications and guidelines. Few data exist on its implementation in clinical practice.
AIM: To investigate the implementation of primary prophylaxis based on real-life data from PedNet during the last 20 years.
METHODS: All children from the PedNet cohort (n = 1260) with severe haemophilia A (SHA) or severe haemophilia B (SHB), FVIII/IX < .01 IU/mL, born between 2000 and 2009 (Cohort I; SHA n = 662; SHB n = 88) and 2010-2019 (Cohort II; SHA n = 598; SHB n = 94) were included.
RESULTS: In SHA, the median age at start of prophylaxis was 17.3 months (IQR; 12.5-26.1) in Cohort I which decreased to 13.1 months (IQR; 10.4-19.1) in Cohort II (p < .000). "Once-a-week" prophylaxis at start increased from 49% to 68% (SHA) and 38% to 70% (SHB). FVIII doses were reduced from median 43.5 (IQR; 34.6-49.0) to 30.9 IU/kg (IQR; 26.3-46.3), while dosing with FIX did not change. After 2010 approximately 60% of the patients with SHA and SHB started prophylaxis before any joint bleed. The number of CVADs needed in both cohorts was around 30%. Incidences of inhibitors were unchanged: SHA (∼31%) and SHB (∼10%). Sporadic cases were diagnosed significantly later (median 8.3 months; IQR; 3.7-11.9) and they had more joint bleeds before start of prophylaxis.
CONCLUSION: Primary prophylaxis nowadays starts at an earlier age: before any joint bleed (60% of patients with SHA and SHB). Approximately 70% started on a once-weekly schedule with significantly reduced doses in SHA but unchanged in SHB.
(Less)
- author
- Ljung, Rolf
LU
; de Kovel, Marloes
and van den Berg, H Marijke
- author collaboration
- organization
- publishing date
- 2023
- type
- Contribution to journal
- publication status
- published
- subject
- in
- Haemophilia
- volume
- 29
- issue
- 2
- pages
- 498 - 504
- publisher
- Wiley-Blackwell
- external identifiers
-
- pmid:36571801
- scopus:85145288351
- ISSN
- 1351-8216
- DOI
- 10.1111/hae.14729
- language
- English
- LU publication?
- yes
- additional info
- © 2022 The Authors. Haemophilia published by John Wiley & Sons Ltd.
- id
- 2a49edba-7103-4655-a592-779ab27bec6c
- date added to LUP
- 2023-01-02 12:59:17
- date last changed
- 2024-11-13 06:11:28
@article{2a49edba-7103-4655-a592-779ab27bec6c,
abstract = {{<p>INTRODUCTION: The prophylactic regimen in children with severe haemophilia is suggested in various publications and guidelines. Few data exist on its implementation in clinical practice.</p><p>AIM: To investigate the implementation of primary prophylaxis based on real-life data from PedNet during the last 20 years.</p><p>METHODS: All children from the PedNet cohort (n = 1260) with severe haemophilia A (SHA) or severe haemophilia B (SHB), FVIII/IX < .01 IU/mL, born between 2000 and 2009 (Cohort I; SHA n = 662; SHB n = 88) and 2010-2019 (Cohort II; SHA n = 598; SHB n = 94) were included.</p><p>RESULTS: In SHA, the median age at start of prophylaxis was 17.3 months (IQR; 12.5-26.1) in Cohort I which decreased to 13.1 months (IQR; 10.4-19.1) in Cohort II (p < .000). "Once-a-week" prophylaxis at start increased from 49% to 68% (SHA) and 38% to 70% (SHB). FVIII doses were reduced from median 43.5 (IQR; 34.6-49.0) to 30.9 IU/kg (IQR; 26.3-46.3), while dosing with FIX did not change. After 2010 approximately 60% of the patients with SHA and SHB started prophylaxis before any joint bleed. The number of CVADs needed in both cohorts was around 30%. Incidences of inhibitors were unchanged: SHA (∼31%) and SHB (∼10%). Sporadic cases were diagnosed significantly later (median 8.3 months; IQR; 3.7-11.9) and they had more joint bleeds before start of prophylaxis.</p><p>CONCLUSION: Primary prophylaxis nowadays starts at an earlier age: before any joint bleed (60% of patients with SHA and SHB). Approximately 70% started on a once-weekly schedule with significantly reduced doses in SHA but unchanged in SHB.</p>}},
author = {{Ljung, Rolf and de Kovel, Marloes and van den Berg, H Marijke}},
issn = {{1351-8216}},
language = {{eng}},
number = {{2}},
pages = {{498--504}},
publisher = {{Wiley-Blackwell}},
series = {{Haemophilia}},
title = {{Primary prophylaxis in children with severe haemophilia A and B-Implementation over the last 20 years as illustrated in real-world data in the PedNet cohorts}},
url = {{http://dx.doi.org/10.1111/hae.14729}},
doi = {{10.1111/hae.14729}},
volume = {{29}},
year = {{2023}},
}