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The B-Natural study...
The B-Natural study—The outcome of immune tolerance induction therapy in patients with severe haemophilia B
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- Astermark, Jan (författare)
- Lund University,Lunds universitet,Klinisk koagulationsmedicin, Malmö,Forskargrupper vid Lunds universitet,Clinical Coagulation, Malmö,Lund University Research Groups,Skåne University Hospital
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- Holstein, Katharina (författare)
- University Medical Center Hamburg-Eppendorf
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- Abajas, Yasmina L. (författare)
- University of North Carolina
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- Kearney, Susan (författare)
- Children's Hospitals and Clinics of Minnesota
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- Croteau, Stacy E. (författare)
- Boston Children's Hospital
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- Liesner, Riana (författare)
- Great Ormond Street Hospital
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- Funding, Eva (författare)
- Copenhagen University Hospital
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- Kempton, Christine L. (författare)
- Emory University
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- Acharya, Suchitra (författare)
- Northwell Haemostasis and Thrombosis Centre
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- Lethagen, Stefan (författare)
- Swedish Orphan Biovitrum
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- LeBeau, Petra (författare)
- Rho, Inc.
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- Bowen, Joel (författare)
- Indiana Haemophilia and Thrombosis Centre
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- Berntorp, Erik (författare)
- Lund University,Lunds universitet,Klinisk koagulationsmedicin, Malmö,Forskargrupper vid Lunds universitet,Clinical Coagulation, Malmö,Lund University Research Groups
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- Shapiro, Amy D. (författare)
- Indiana Haemophilia and Thrombosis Centre
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(creator_code:org_t)
- 2021-06-12
- 2021
- Engelska.
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Ingår i: Haemophilia. - : Wiley. - 1351-8216 .- 1365-2516. ; 27:5, s. 802-813
- Relaterad länk:
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http://dx.doi.org/10... (free)
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https://doi.org/10.1...
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https://lup.lub.lu.s...
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https://doi.org/10.1...
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Abstract
Ämnesord
Stäng
- 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.
Ämnesord
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Hematologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Hematology (hsv//eng)
Nyckelord
- allergy
- factor IX deficiency
- haemophilia B
- immune tolerance induction
- inhibitors
- nephrotic syndrome
Publikations- och innehållstyp
- art (ämneskategori)
- ref (ämneskategori)
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Till lärosätets databas
- Av författaren/redakt...
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Astermark, Jan
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Holstein, Kathar ...
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Abajas, Yasmina ...
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Kearney, Susan
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Croteau, Stacy E ...
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Liesner, Riana
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visa fler...
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Funding, Eva
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Kempton, Christi ...
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Acharya, Suchitr ...
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Lethagen, Stefan
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LeBeau, Petra
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Bowen, Joel
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Berntorp, Erik
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Shapiro, Amy D.
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visa färre...
- Om ämnet
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- MEDICIN OCH HÄLSOVETENSKAP
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MEDICIN OCH HÄLS ...
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och Klinisk medicin
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och Hematologi
- Artiklar i publikationen
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Haemophilia
- Av lärosätet
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Lunds universitet