1. To evaluate the diagnostic accuracy of a newly developed pediatric BAT.2. To analyze the influence of age, sex and type of bleeding disorder on the total bleeding score and the score of individual bleeding items.
ID
Source
Brief title
Condition
- Coagulopathies and bleeding diatheses (excl thrombocytopenic)
- Blood and lymphatic system disorders congenital
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The main study outcome is the diagnostic accuracy of a newly developed
pediatric BAT. This will be analysed by calculating the area under the Receiver
operating characteristic curve (ROC) .
Secondary outcome
Linear regression will be used to model the association of bleeding score with
age, sex and type of bleeding disorder in an multivariatble model
Background summary
The diagnosis of an inherited bleeding disorder is made based on the results
from a patient*s family history, bleeding history and laboratory test results.
Especially in children it may be difficult for the doctor to obtain a clear
bleeding history, as bleeding symptoms can be subtle, children face less
hemostatic challenges compared to adults and, for example, bruises in toddlers
may be caused by normal activity. In order to standardize bleeding histories,
bleeding assessment tools (BATs) have been developed, yielding a score based on
the medical treatment history of the most severe bleeding episode for specific
bleeding symptoms. Although it has been shown that high bleeding scores are
associated with the presence of a bleeding disorder, the current pediatric BAT
lacks sensitivity, efficiency and validity. The primary aim of the study is to
develop a refined pediatric BAT that can be used as a screening tool for
inherited bleeding disorders in tertiary clinics. The secondary aim of the
study is to analyze the influence of age, sex and type of bleeding disorder on
the total bleeding score and the score of individual bleeding items.
Study objective
1. To evaluate the diagnostic accuracy of a newly developed pediatric BAT.
2. To analyze the influence of age, sex and type of bleeding disorder on the
total bleeding score and the score of individual bleeding items.
Study design
This is a prospective cohort study. The cohort will include pediatric patients
presenting with bleeding symptoms at 5 tertiary hematology clinics in The
Netherlands, Canada and The United Kingdom. Data collection will last until the
required amount of 200 patient inclusions has been reached. This is expected to
take approximately 1 year.
Study burden and risks
Risks imposed by participation in this study are considered negligible.
Patients will undergo the same diagnosticprocedures/tests as usual (so, when
they would not participate in this study).
Meibergdreef 9
Amsterdam 1105 AZ
NL
Meibergdreef 9
Amsterdam 1105 AZ
NL
Listed location countries
Age
Inclusion criteria
1. Age 0-18 years
2. Presenting with (1) the history, signs and symptoms of bleeding, and/or (2) abberant laboratory blood parameters and/or (3) a diagnosis of a bleeding disorder in relatives
Exclusion criteria
1. Patients with a previous diagnosis of an inherited bleeding disorder
2. Patients with a known, acquired cause of bleeding (ie. renal or liver disease, use of medication that is known to cause an increased bleeding tendency)
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
In other registers
| Register | ID |
|---|---|
| CCMO | NL56790.018.16 |
| OMON | NL-OMON21443 |