Page 93 - Let them speak Towards a patient reported outcome measure set for

PROs used in clinical trials and core outcome sets
22q11.2 deletion syndrome (32), obesity in Prader-Willi syndrome (33), and
early-onset dementia in Down syndrome (34) can all impact the ability to
perform daily activities. Therefore, different clinical manifestations may
still lead to the same PROs. This also accords with earlier studies, which
showed that PROs often overlap across different conditions, such as
cancer, congenital heart disease, and inflammatory bowel disease (35, 36).
Unclear or no PROs
Some of the identified PROs were unclear (i.e., ‘syndrome-specific
symptoms’), which made it difficult to know what was exactly being
measured. Additionally, many of the identified PROs turned out to be no PRO
at all since they did not represent health aspects reported by patients (e.g.,
‘behavior’, ‘self-efficacy’, or ‘social support’). This highlights the challenge
still faced by many researchers and clinicians who lack clarity on how to
classify outcomes; as PROs or as other outcomes (e.g., observer reported
outcomes or clinician reported outcomes).
Need for harmonization; a generic core PRO set
This review demonstrates the need for harmonization of PROs used for GID.
There is not only a myriad of PROs used for GID, but these PROs also often
include different terminologies, multiple domains, and unclear definitions,
making it difficult to combine or compare them properly and thus questioning
their usefulness. Additionally, there still seems to be uncertainty about what
constitutes a PRO and what constitutes other outcomes. Therefore, a clear
and well-defined generic core PRO set is needed for GID for use in both care
and research, as no such set currently exists. This generic core PRO set
will ensure consistent measurement and assessment of PROs, reducing
the great variety of PROs used for GID in care and research. Additionally, it
will also become more clear which outcomes do not classify as PROs. This
generic core PRO set may be supplemented with condition-specific PROs
for particular GIDs, if necessary. The use of a generic set of PROs also
aligns with the Dutch government’s initiative to measure the same PROs
across conditions (31).
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