Shannon van Hoorn

82 Chapter 3 This study also has several limitations. Firstly, this Delphi study used a long-list of 146 health outcomes based on the long-list of previous research performed in patients with hemophilia 12,19. This list was enriched with the findings of a systematic literature review on relevant patient-reported outcomes in patients with autosomal inherited bleeding disorders 14. In our aim to present the participants with a list of all possibly relevant health outcomes, no critical appraisal was performed on the health outcomes mentioned in the long-list of the previous studies. Therefore, our study may have included health outcomes that were similar or indistinguishable from each other in the perception of the participants. In addition, some health outcomes included within the long-list could not strictly be classified as a health outcome, i.e. the health outcome may not represent a change in health status that can be measured clinically, observed or self-reported 21,22. Secondly, within this Delphi study a very strict definition of importance was used to determine which health outcomes should be included in the second or third round. We defined importance as a median of five (very important) and unimportance as a median below five across all participants within a panel. This strict definition of importance was proven necessary after the first Delphi round. Without this strict definition of importance, it would not have been possible to achieve consensus on a feasible number of important health outcomes within a reasonable number of Delphi rounds. However, this may also have led to the exclusion of health outcomes early on in the Delphi process that, given a different definition of importance and more Delphi rounds, could have been identified as important for patients with autosomal inherited bleeding disorders as seen from the patient, caregiver and healthcare professional perspective. Thirdly, we were unable to report race or ethnicity as a social-cultural determinant of health. Therefore, we could not assess its influence on the patient, caregiver and healthcare professional perspectives on the importance of the various health outcomes. Lastly, difficulties were experienced during the recruitment of patients, caregivers and healthcare professionals. To obtain a reliable outcome of a Delphi panel, the panel must consist of a minimum of 30 experts 23, in this case patients, caregivers and healthcare professionals. To meet this criteria, the first Delphi round was extended multiple times resulting in a first Delphi round that lasted from February 1st 2021 till November 30th 2021. As a result,

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