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Quality of Life in Children with Congenital Bleeding Disorders: A Scoping Review of Current Research State and Knowledge Gaps

1Erasmus MC University Medical Center Rotterdam MC - Sophia Children's Hospital, Pediatric Hematology, Rotterdam, the Netherlands, 2Sanquin, Unit Transfusional Medicine, Amsterdam, the Netherlands, 3Erasmus University Medical Center, Rotterdam, the Netherlands, 4Erasmus MC University Medical Center Rotterdam MC - Sophia Children's Hospital, Pediatric hematology, Rotterdam, the Netherlands, 5Erasmus MC University Medical Center Rotterdam, Public Health, Rotterdam, the Netherlands

Abstract Number: PB1024

Meeting: ISTH 2020 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical

Background: Quality of Life (QoL) in children with bleeding disorders can be affected. To measure QoL in developing children, input of children and parents is needed. This can be done by generic questionnaires and disease-specific questionnaires, that contain questions referring to typical disease-related issues, such as bleeding symptoms.

Aims: The aim of this scoping review was to analyze current knowledge on QoL in children with all forms of congenital bleeding disorders in order to identify knowledge gaps.

Methods: We performed a systematic literature search on April 29th 2019. Articles were screened on title/abstract by two independent reviewers using a preset inclusion tool. Eligible articles were read in full, leading to a final inclusion of 37 articles.

Results: Two-third of studies were performed in Europe and North-America, and published after 2010 (86,4%). Only 34,3% included children < 4 years of age. In 31 articles (83.8%) patients had hemophilia, but only 9 included a homogeneous group of the same disease and severity. Fourteen articles pooled results of patients with different types of bleeding disease or severity. Thirteen different generic and five different disease-specific questionnaires were used. Most studies only used self-reports, sometimes combined with proxy-reports. Ten articles used a reference group. Mean and SD were most often used as descriptive statistics.

Conclusions: QoL research in children with congenital bleeding disorders is an upcoming research field, but it is mainly performed in school-aged children with hemophilia, who are treated in well developed countries. Pitfalls are the pooling of various bleeding disorder types and severity, the lack of parent perspectives, the lack of non-hemophilia disease-specific questionnaires. Too many different generic QoL questionnaires are used, results not comparable between themselves. QoL was rarely measured to evaluate the effect of a therapy. Parameters as mean or SD are of less use in heterogeneous groups.

To cite this abstract in AMA style:

Huisman EJ, Mussert C, Cnossen MH, Bai G, Raat H. Quality of Life in Children with Congenital Bleeding Disorders: A Scoping Review of Current Research State and Knowledge Gaps [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/quality-of-life-in-children-with-congenital-bleeding-disorders-a-scoping-review-of-current-research-state-and-knowledge-gaps/. Accessed November 30, 2023.

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