Search

Introduction: It is challenging to obtain a reliable bleeding history in children who are referred for a suspected inherited bleeding disorder. Bleeding symptoms may be subtle as children face fewer haemostatic challenges compared with adults. In order to standardise bleeding histories, questionnaires have been developed, called bleeding assessment tools (BATs). Although it has been shown that high bleeding scores are associated with the presence of a mucocutaneous bleeding disorder, these BATs lack sensitivity, efficiency and flexibility in the paediatric setting. We developed a new BAT (the iCHEC (identifying Children with HEreditary Coagulation disorders) BAT) to improve on these characteristics. We aim to evaluate the diagnostic accuracy of the iCHEC BAT as a screening tool for children who are suspected for having a bleeding disorder., Methods and Analysis: This is a prospective cohort study. Children (age 0-18 years) suspected for a bleeding disorder who present at tertiary haematology clinics, and/or their parents/guardians, will be asked to complete the iCHEC BAT. Sensitivity was increased by inclusion of paediatric-specific bleeding symptoms and novel qualitative questions per bleeding symptom. Efficiency was improved by developing a self-administered (online) version of the questionnaire. Flexibility for changes in the bleeding phenotype of developing children was improved by including questions that define when the bleeding symptoms occurred in the past. The diagnostic accuracy of the specific bleeding items will be evaluated by receiver operator characteristic curves, using classification based on the results from laboratory assessment as the reference standard. Analysis of the discriminative power of individual bleeding symptoms will be assessed., Ethics and Dissemination: The study has been approved by the medical ethics committees of all participating centres in the Netherlands, Canada and the UK. All paediatric subjects and/or their parents/guardians will provide written informed consent. Study results will be submitted for publication in peer-reviewed journals., Competing Interests: Competing interests: None declared., (© Article author(s) (or their employer(s) unless otherwise stated in the text of the article) 2018. All rights reserved. No commercial use is permitted unless otherwise expressly granted.)

Background: The half-life and mean residence time (MRT) of infused recombinant factor VIII (FVIII) concentrate are associated with pre-infusion levels of von Willebrand factor (VWF) in severely affected hemophilia A patients. It is currently unknown if individual FVIII concentrate half-life and MRT can be extended by increasing endogenous VWF levels. Aim: Our aim was to evaluate the effect of a 1-deamino-8-D-arginine vasopressin (DDAVP)-induced rise in VWF concentration on the pharmacokinetics of infused FVIII in hemophilia A patients., Methods: Four adult hemophilia A patients participated in this cross-over, placebo-controlled study. Each patient received either intravenous DDAVP or placebo, one hour prior to administration of 50 IU/kg plasma-derived immune-affinity purified FVIII concentrate., Results: The combined administration of DDAVP and FVIII concentrate was well tolerated. The levels of VWF Antigen (Ag) doubled after DDAVP, whereas they remained stable after placebo infusion. This rise in VWF Ag resulted in a slight modification of the pharmacokinetic parameters of FVIII concentrate. The MRT of FVIII concentrate increased in all patients (mean from 17.6 h to 19.9 h, p < 0.001, 95% CI for MRT change: +4.7 to -0.3 h). However, in vivo recoveries tended to decrease following DDAVP administration., Conclusions: Collectively, these data show that administration of DDAVP did not improve the pharmacokinetics of FVIII concentrate in a clinically significant manner., Relevance for Patients: Our results indicate that no clinical benefit is to be expected from the modification in FVIII pharmacokinetics resulting from DDAVP-administration prior to infusion of FVIII concentrate in hemophilia A patients.

Background: Given the low mortality of pediatric patients diagnosed with thyroid disease, quality of life (QoL) after thyroid surgery is very important. To organize the best possible patient care we analyzed our experience with respect to QoL and clinical outcome., Methods: This is a single center, retrospective cohort study. Data of patients who underwent thyroid surgery < 19 years between January 2000 and December 2012 were collected. QoL was measured using the child health questionnaire child form (CHQ-CF87, < 18 years) and the World Health Organization quality of life assessment (WHOQOL-100, ≥ 18 years)., Results: Forty patients were included (mean age 13.7 years; 29 females (72.5%)). Twenty-six patients underwent total thyroidectomy (including 7 repeat surgeries), 14 underwent hemithyroidectomy. QoL assessment in 26 patients revealed lower physical QoL in patients with a current age < 18 years (n = 11) (p < .001), but higher overall and physical QoL in patients ≥ 18 years (n = 15) compared with controls (p = .01 and p = .036 respectively). Patients ≥ 18 years, who underwent total thyroidectomy experienced lower overall and physical QoL compared with those who underwent hemithyroidectomy (p = .035 and p = .005 respectively)., Conclusions: Surgery for thyroid disease during childhood significantly affects QoL. However, QoL seems to improve with increasing age, and hemi-thyroidectomy has less negative effects on QoL than total thyroidectomy., (Copyright © 2015 Elsevier Inc. All rights reserved.)