Shannon van Hoorn

53 PROMs in autosomal inherited bleeding disorders: A systematic literature review 2 about the quality of life of patients with autosomal inherited bleeding disorders should be interpret with this limitation in mind. Secondly, from all the used PROMs, only one PROM (i.e., the Hemophilia Activity List) has undergone any degree of development and validation in patients with inherited bleeding disorders 26,28. The use of non-validated or inadequately targeted PROMs in a study that has not considered their psychometric properties may have adverse consequences. This includes ethical concerns surrounding patients having to complete measures that are incapable of capturing the patient’s perspective and can therefore have unreliable or biased results. Thirdly, the various autosomal inherited bleedings disorders were not equally represented in this systematic review and most studies focused on patients with VWD. The latter is also partly due to the substantial amount of studies originating from the WiN study group, which might have led to bias as a result of partially overlapping sets of participants. Fourthly, the NIH risk of bias assessment raised some key issues including lack of effect sizes that allow for comparison between study populations and controls. This means, that even if a study finds significant differences in PROs, the relevance of this result might be questionable. Therefore, caution should be observed in the generalization of findings. Additionally, only a minority of studies adjusted for key confounding variables such as demographic data including age and socio-economic status, while these factors have been previously described as predictors of HRQoL 58-60. Fifthly, only few studies compared the study population with the general population or patients with and without a certain symptom. This limits the capacity to attribute causality. Lastly, additional unpublished work or studies published in languages other than English were not included in this review, which may have biased the results. Our systematic review highlights the need for future studies using established standards to analyze PROs in patients with autosomal inherited bleeding disorders. The use of a validated method to evaluate and compare PROs across populations, conditions, research studies and clinical practices is important to improve quality of care for the individual patient. An example of such a method is the Patient-Reported Outcomes Measurement Information System (PROMIS®).

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