Two hundred and twenty-five patients with severe and moderate for

Two hundred and twenty-five patients with severe and moderate forms of haemophilia A and B from three centres were invited

to participate in the study. Spearman’s rank correlation test was used for validation, and internal consistency of the HAL was calculated with Cronbach’s alpha. Eighty-four patients (39%) (18–80 years old) filled out the questionnaires. The internal consistency of the Swedish version of HAL was high, with Cronbach’s alpha being 0.98–0.71. Function of the legs had the highest consistency and transportation had the lowest. The correlation was excellent between the HAL sum score and AIMS 2 physical (r = 0.84, P < 0.01), IPA autonomy indoors (r = 0.83, P < 0.01) and autonomy outdoors (r = 0.89, NVP-BEZ235 P < 0.01). The Swedish version of HAL has both internal consistency and convergent

validity and may complement other functional tests to gather information on the patient’s self-perceived ability. “
“The objective of the present study was to evaluate the Selleckchem GSK1120212 pharmacokinetic (PK) and pharmacodynamic (PD) profiles of the new recombinant FVIII compound turoctocog alfa and a Glyco-PEGylated FVIII derivative thereof (N8-GP) in Haemophilia A dogs. Six haemophilic dogs divided into two groups were included in the study. Each dog was administered a dose of 125 U kg−1, blood samples were collected at predetermined time points for both pharmacokinetic (FVIII measured by one-stage aPTT assay) and pharmacodynamic [whole blood clotting time (WBCT)] evaluations. After intravenous administration to haemophilic dogs, the plasma concentration at the first sampling point was comparable for turoctocog alfa and N8-GP, and the clearance was estimated to be 6.5 and 3.9 mL h−1kg−1 for turoctocog alfa and N8-GP respectively. Both turoctocog alfa and N8-GP

were able to reduce the WBCT time to normal levels (<20 min), however, the reduced clearance was reflected in the WBCT, 上海皓元医药股份有限公司 which returned to baseline at a later time point for N8-GP as compared with dogs dosed with turoctocog alfa. The clearance was 40% reduced for N8-GP as compared with turoctocog alfa. Simulations of a multiple dosing regimen in dogs, suggest that to maintain WBCT <20 min N8-GP can be dosed at reduced intervals, e.g. with 4 days between doses, whereas turoctocog alfa will have to be dosed with 2½ day between doses. Data thereby supports N8-GP as an alternative to standard rFVIII replacement therapy, with a more convenient dosing regimen. "
“Summary.  A predictive standardized bleeding questionnaire (Vicenza score), previously validated for identifying individuals with type 1 von Willebrand’s disease (VWD), has never been prospectively validated in tertiary care paediatric settings.

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