The B-Natural study—The outcome of immune tolerance induction therapy in patients with severe haemophilia B
(2021) In Haemophilia 27(5). p.802-813- Abstract
Introduction: Inhibitors develop less frequently in haemophilia B (HB) than haemophilia A (HA). However, when present, the success of tolerization by immune tolerance induction (ITI) therapy is lower and the risk of complications higher. Aim: To evaluate the use and outcome of ITI in patients with HB and inhibitors. Methods: Subjects include singletons or siblings with a current/history of inhibitors enrolled in B-Natural—an observational study designed to increase understanding of clinical management of patients with HB. Patients were followed for 6 months and information on demographics, medical and social history, and treatment were recorded. Results: Twenty-nine patients with severe HB and inhibitors were enrolled in 24 centres.... (More)
Introduction: Inhibitors develop less frequently in haemophilia B (HB) than haemophilia A (HA). However, when present, the success of tolerization by immune tolerance induction (ITI) therapy is lower and the risk of complications higher. Aim: To evaluate the use and outcome of ITI in patients with HB and inhibitors. Methods: Subjects include singletons or siblings with a current/history of inhibitors enrolled in B-Natural—an observational study designed to increase understanding of clinical management of patients with HB. Patients were followed for 6 months and information on demographics, medical and social history, and treatment were recorded. Results: Twenty-nine patients with severe HB and inhibitors were enrolled in 24 centres. Twenty-two underwent one or more courses of ITI with or without immune suppression. Eight patients (36.4%) were successfully tolerized after the first course of ITI. One of these successes (12.5%) experienced allergic manifestations, whereas the corresponding number for the 10 treatment failures was five (50%). One of seven (14.2%) patients with large deletions and three of eight (37.5%) with nonsense mutations were tolerized at the first attempt, and all patients experiencing nephrosis either failed or were on-going. At study end, 11 (50%) were considered successfully tolerized after one or more ITI courses, three were unsuccessful, and eight were still undergoing treatment. Conclusion: Our data underscore the possibilities and difficulties of achieving tolerization in patients with HB with inhibitors. The type of mutation and complications appear to correlate with ITI outcome, but more accurate definitions of successful ITI are warranted.
(Less)
- author
- organization
- publishing date
- 2021
- type
- Contribution to journal
- publication status
- published
- subject
- keywords
- allergy, factor IX deficiency, haemophilia B, immune tolerance induction, inhibitors, nephrotic syndrome
- in
- Haemophilia
- volume
- 27
- issue
- 5
- pages
- 802 - 813
- publisher
- Wiley-Blackwell
- external identifiers
-
- scopus:85107740831
- pmid:34118102
- ISSN
- 1351-8216
- DOI
- 10.1111/hae.14357
- language
- English
- LU publication?
- yes
- id
- c5f9a282-5adc-4dd1-82f0-99179dac8134
- date added to LUP
- 2021-07-20 11:00:25
- date last changed
- 2024-04-20 09:38:31
@article{c5f9a282-5adc-4dd1-82f0-99179dac8134,
abstract = {{<p>Introduction: Inhibitors develop less frequently in haemophilia B (HB) than haemophilia A (HA). However, when present, the success of tolerization by immune tolerance induction (ITI) therapy is lower and the risk of complications higher. Aim: To evaluate the use and outcome of ITI in patients with HB and inhibitors. Methods: Subjects include singletons or siblings with a current/history of inhibitors enrolled in B-Natural—an observational study designed to increase understanding of clinical management of patients with HB. Patients were followed for 6 months and information on demographics, medical and social history, and treatment were recorded. Results: Twenty-nine patients with severe HB and inhibitors were enrolled in 24 centres. Twenty-two underwent one or more courses of ITI with or without immune suppression. Eight patients (36.4%) were successfully tolerized after the first course of ITI. One of these successes (12.5%) experienced allergic manifestations, whereas the corresponding number for the 10 treatment failures was five (50%). One of seven (14.2%) patients with large deletions and three of eight (37.5%) with nonsense mutations were tolerized at the first attempt, and all patients experiencing nephrosis either failed or were on-going. At study end, 11 (50%) were considered successfully tolerized after one or more ITI courses, three were unsuccessful, and eight were still undergoing treatment. Conclusion: Our data underscore the possibilities and difficulties of achieving tolerization in patients with HB with inhibitors. The type of mutation and complications appear to correlate with ITI outcome, but more accurate definitions of successful ITI are warranted.</p>}},
author = {{Astermark, Jan and Holstein, Katharina and Abajas, Yasmina L. and Kearney, Susan and Croteau, Stacy E. and Liesner, Riana and Funding, Eva and Kempton, Christine L. and Acharya, Suchitra and Lethagen, Stefan and LeBeau, Petra and Bowen, Joel and Berntorp, Erik and Shapiro, Amy D.}},
issn = {{1351-8216}},
keywords = {{allergy; factor IX deficiency; haemophilia B; immune tolerance induction; inhibitors; nephrotic syndrome}},
language = {{eng}},
number = {{5}},
pages = {{802--813}},
publisher = {{Wiley-Blackwell}},
series = {{Haemophilia}},
title = {{The B-Natural study—The outcome of immune tolerance induction therapy in patients with severe haemophilia B}},
url = {{http://dx.doi.org/10.1111/hae.14357}},
doi = {{10.1111/hae.14357}},
volume = {{27}},
year = {{2021}},
}