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Inhibitor incidence...
Inhibitor incidence in an unselected cohort of previously untreated patients with severe haemophilia B : a PedNet study
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- Male, Christoph (författare)
- Medical University of Vienna
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- Andersson, Nadine G (författare)
- Lund University,Lunds universitet,Pediatrisk hematologi,Forskargrupper vid Lunds universitet,Paediatric Haematology Research Unit,Lund University Research Groups,Skåne University Hospital
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- Rafowicz, Anne (författare)
- Bicêtre Hospital
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- Liesner, Ri (författare)
- Great Ormond Street Hospital
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- Kurnik, Karin (författare)
- Technical University of Munich
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- Fischer, Kathelijn (författare)
- University Medical Center Utrecht
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- Platokouki, Helen (författare)
- Aghia Sophia Children's Hospital
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- Santagostino, Elena (författare)
- Maggiore Hospital Policlinico,IRCCS Multimedica
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- Chambost, Hervé (författare)
- La Timone University Hospital
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- Nolan, Beatrice (författare)
- Children's Health Ireland (CHI) at Crumlin
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- Königs, Christoph (författare)
- University Hospital Frankfurt
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- Kenet, Gili (författare)
- Sheba Medical Center
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- Ljung, Rolf (författare)
- Lund University,Lunds universitet,Pediatrik, Lund,Sektion V,Institutionen för kliniska vetenskaper, Lund,Medicinska fakulteten,Pediatrisk hematologi,Forskargrupper vid Lunds universitet,Paediatrics (Lund),Section V,Department of Clinical Sciences, Lund,Faculty of Medicine,Paediatric Haematology Research Unit,Lund University Research Groups
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- van den Berg, Marijke (författare)
- PedNet Haemophilia Research Foundation
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(creator_code:org_t)
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- 2020-01-09
- 2021
- Engelska.
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Ingår i: Haematologica. - : Ferrata Storti Foundation (Haematologica). - 1592-8721 .- 0390-6078. ; 106:1, s. 123-129
- Relaterad länk:
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http://dx.doi.org/10... (free)
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https://haematologic...
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https://lup.lub.lu.s...
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https://doi.org/10.3...
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Abstract
Ämnesord
Stäng
- The incidence of FIX inhibitors in severe hemophilia B (SHB) is not well defined. Frequencies of 3-5% have been reported but most studies to date were small, including patients with different severities, and without prospective follow-up for inhibitor incidence. Study objective was to investigate inhibitor incidence in patients with SHB followed up to 500 exposure days (ED), the frequency of allergic reactions, and the relationship with genotypes. Consecutive previously untreated patients (PUPs) with SHB enrolled into the PedNet cohort were included. Detailed data was collected for the first 50 ED, followed by annual collection of inhibitor status and allergic reactions. Presence of inhibitors was defined by at least two consecutive positive samples. Additionally, data on factor IX gene mutation was collected. 154 PUPs with SHB were included; 75% were followed until 75 ED, and 43% until 500 ED. Inhibitors developed in 14 patients (7 high-titre). Median number of ED at inhibitor manifestation was 11 (IQR 6.5-36.5). Cumulative inhibitor incidence was 9.3% (95%CI 4.4-14.1) at 75 ED, and 10.2% (5.1-15.3) at 500 ED. Allergic reactions occurred in 4 (28.6%) inhibitor patients. Missense mutations were most frequent (46.8%) overall but not associated with inhibitors. Nonsense mutations and deletions with large structural changes comprised all mutations among inhibitor patients and were associated with an inhibitor risk of 26.9% and 33.3%, respectively. In an unselected, well-defined cohort of PUPs with SHB, cumulative inhibitor incidence was 10.2% at 500 ED. Nonsense mutations and large deletions were strongly associated with the risk of inhibitor development. The PedNet Registry is registered at clinicaltrials.gov; identifier: NCT02979119.
Ämnesord
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Hematologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Hematology (hsv//eng)
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Male, Christoph
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Andersson, Nadin ...
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Rafowicz, Anne
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Liesner, Ri
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Kurnik, Karin
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Fischer, Katheli ...
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Platokouki, Hele ...
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Santagostino, El ...
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Chambost, Hervé
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Nolan, Beatrice
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Königs, Christop ...
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Kenet, Gili
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Ljung, Rolf
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van den Berg, Ma ...
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- MEDICIN OCH HÄLSOVETENSKAP
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och Klinisk medicin
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och Hematologi
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Haematologica
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Lunds universitet