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Inhibitor development in previously untreated patients with severe haemophilia: A comparison of included patients and outcomes between a clinical study and a registry-based study.

Carla J JonkerKatrien Oude RengerinkArno W HoesPeter G M MolH Marijke van den Berg
Published in: Haemophilia : the official journal of the World Federation of Hemophilia (2020)
In the registry-based study, patient numbers and completeness of follow-up were higher. The risk of developing an inhibitor to a single product was comparable. Although the sample size of this study was too small to conclude on differences in high- or low-titre inhibitors, this suggests that a registry could serve as a more suitable source for evaluation of high-titre inhibitors in the setting of factor VIII deficiency.
Keyphrases
  • end stage renal disease
  • newly diagnosed
  • ejection fraction
  • prognostic factors
  • type diabetes
  • clinical trial
  • case report
  • replacement therapy
  • patient reported