Identifying Children with HEreditary Coagulation disorders (iCHEC): a protocol for a prospective cohort study.

Autor:	Stokhuijzen E; Pediatric Hematology, Academic Medical Center - Emma Children's Hospital, Amsterdam, The Netherlands.; Department of Plasma Proteins, Sanquin Research, Amsterdam, The Netherlands., Rand ML; Division of Hematology/Oncology and Translational Medicine Program, Hospital for Sick Children, Toronto, Ontario, Canada.; Departments of Laboratory Medicine & Pathobiology, Biochemistry, and Pediatrics, University of Toronto, Ontario, Canada., Cnossen MH; Department of Pediatric Hematology, Erasmus University Medical Center - Sophia Children's Hospital, Rotterdam, The Netherlands., Biss TT; Department of Haematology, Newcastle Upon Tyne Hospitals NHS Foundation Trust, Newcastle Upon Tyne, UK., James PD; Department of Medicine, Division of Hematology, Queens University, Kingston General Hospital, Kingston, Canada., Suijker MH; Pediatric Hematology, Academic Medical Center - Emma Children's Hospital, Amsterdam, The Netherlands., Peters M; Pediatric Hematology, Academic Medical Center - Emma Children's Hospital, Amsterdam, The Netherlands., van der Lee JH; Academic Medical Center - Pediatric Clinical Research Office, Woman-Child Center, Amsterdam, The Netherlands., Peters B; Pediatric Hematology, Academic Medical Center - Emma Children's Hospital, Amsterdam, The Netherlands., Meijer AB; Department of Plasma Proteins, Sanquin Research, Amsterdam, The Netherlands.; Department of Pharmaceutics, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands., Blanchette VS; Department of Pediatrics, University of Toronto, Toronto, Canada.; Division of Hematology/Oncology, Hospital for Sick Children, Toronto, Canada., Fijnvandraat K; Pediatric Hematology, Academic Medical Center - Emma Children's Hospital, Amsterdam, The Netherlands.; Department of Plasma Proteins, Sanquin Research, Amsterdam, The Netherlands.
Jazyk:	angličtina
Zdroj:	BMJ open [BMJ Open] 2018 May 03; Vol. 8 (5), pp. e020686. Date of Electronic Publication: 2018 May 03.
DOI:	10.1136/bmjopen-2017-020686
Abstrakt:	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.)
Databáze:	MEDLINE
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