Page 61 - Let them speak Towards a patient reported outcome measure set for

of the often time- and labor-intensive care delivered for individuals with GID (32). Additionally,
PRO data gathered with PROMs could inform the development of clinical guidelines and
influence decisions regarding funding healthcare (24, 33).
Protocol for developing a core PRO set
Currently, various PROs are measured for GID, especially in research. A recent study identified
more than 200 different PROMs, which were being utilized in 312 clinical trials involving
individuals with GID (34). Although this overload of PROs measured is not surprising due to
lack of definition (e.g., syndrome-specific symptoms). Lastly, it is unknown
the wide spectrum of manifestations of GID, it does hamper the possibility to combine and
whether the PROs measured are relevant to individuals with GID.
compare PRO data across different genetic or other subgroups (30). Additionally, analyzing and
interpreting PRO data can be complex due to the multidimensionality of PROs (e.g., adaptive
functioning), differences in terminology used for similar PROs (e.g., participation, engagement
To overcome these challenges, we aim to develop a comprehensive,
in meaningful activity, community involvement), and unclear PROs due to lack of definition
universally applicable core PRO set for both children and adults with GID to
(e.g., syndrome-specific symptoms). Lastly, it is unknown whether the PROs measured are
be used in care and research. The frequently shared comorbidities in GID
relevant to individuals with GID.
can result in similar patient reported problems across disorders, enabling
To overcome these challenges, we aim to develop a comprehensive, universally applicable core
the development of a generic core PRO set. This corresponds to the trend
PRO set for both children and adults with GID to be used in care and research. The frequently
seen toward standardized measurement of generic PROs across conditions
shared comorbidities in GID can result in similar patient reported problems across disorders,
in the general population, where PROs such as anxiety, depression, pain,
enabling the development of a generic core PRO set. This corresponds to the trend seen toward
3
and fatigue are often shared (35). In this protocol, the steps for developing
standardized measurement of generic PROs across conditions in the general population, where
PROs such as anxiety, depression, pain, and fatigue are often shared (35). In this protocol, the
the generic core PRO set are described, following the Core Outcome Set-
steps for developing the generic core PRO set are described, following the Core Outcome Set-
Standardized Protocol Items (COS-STAP) Statement (36) in order to align
Standardized Protocol Items (COS-STAP) Statement (36) in order to align with the official steps
with the official steps for developing a Core Outcome Set (COS).
for developing a Core Outcome Set (COS).
Figure 1. Potential benefits of utilizing PROMs in care, research, and health policy.
Figure 1. Potential benefits of utilizing PROMs in care, research, and health policy.
Methods and results
Stakeholders
Methods and results
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Stakeholders
The project team consists of a Study Management Group and a Study
Advisory Board. The Study Management Group consists of researchers;
one researcher (NvS) who will coordinate the day-to-day management
of the project, two PROM experts and medical psychologists (MvM, LH),
and two GID experts including an ID physician and a genetic metabolic
pediatrician (AvE, CvK). The Study Advisory Board consists of GID
experts. The Study Management Group will also collaborate with patient
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