The B-Natural study-The outcome of immune tolerance induction therapy in patients with severe haemophilia B
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The B-Natural study-The outcome of immune tolerance induction therapy in patients with severe haemophilia B. / Astermark, Jan; Holstein, Katharina; Abajas, Yasmina L; Kearney, Susan; Croteau, Stacy E; Liesner, Riana; Funding, Eva; Kempton, Christine L; Acharya, Suchitra; Lethagen, Stefan; LeBeau, Petra; Bowen, Joel; Berntorp, Erik; Shapiro, Amy D.
in: HAEMOPHILIA, Jahrgang 27, Nr. 5, 09.2021, S. 802-813.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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T1 - The B-Natural study-The outcome of immune tolerance induction therapy in patients with severe haemophilia B
AU - Astermark, Jan
AU - Holstein, Katharina
AU - Abajas, Yasmina L
AU - Kearney, Susan
AU - Croteau, Stacy E
AU - Liesner, Riana
AU - Funding, Eva
AU - Kempton, Christine L
AU - Acharya, Suchitra
AU - Lethagen, Stefan
AU - LeBeau, Petra
AU - Bowen, Joel
AU - Berntorp, Erik
AU - Shapiro, Amy D
N1 - © 2021 The Authors. Haemophilia published by John Wiley & Sons Ltd.
PY - 2021/9
Y1 - 2021/9
N2 - 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.
AB - 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.
KW - Factor VIII/genetics
KW - Hemophilia A/drug therapy
KW - Hemophilia B/drug therapy
KW - Humans
KW - Immune Tolerance
KW - Immunosuppression Therapy
U2 - 10.1111/hae.14357
DO - 10.1111/hae.14357
M3 - SCORING: Journal article
C2 - 34118102
VL - 27
SP - 802
EP - 813
JO - HAEMOPHILIA
JF - HAEMOPHILIA
SN - 1351-8216
IS - 5
ER -