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PROs used in clinical trials and core outcome sets
Methods
Patient population
GID was defined as disorders associated with ID with a genetic etiology
involving the central nervous system during childhood. Individuals with ID
without known etiology were also included due to shared comorbidities with
GID and grouped under the term GID. Individuals with inherited metabolic
disorders (IMDs) were excluded, as these individuals fall outside the scope
of the future generic core PRO set.
To identify PROs used for individuals with GID, we integrated the results of
two literature reviews, as described in Figure 1.
4
Figure 1. Steps for developing a generic core PRO set1: step 1.1 and 1.2 were undertaken in this
study.
1Figure 1: Reproduced/adapted from van Silfhout et al., 2024 (22) under the Creative Commons
license (https://creativecommons.org/licenses/by/4.0/).
PROs, patient reported outcomes; PROM(s), patient reported outcome measure(s); COS(s), core
outcome set(s); GID, genetic intellectual disability.
*Target number of participants.
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