Page 173 - Let them speak Towards a patient reported outcome measure set for

Evaluating the core PROM set
include long, abstract, or complex questions, making them unsuitable for
many individuals with GenID (13), and causing a burden for them and their
caregivers (14). Lastly, most PROMs are not properly psychometrically
tested in the GenID population, raising concerns about their reliability and
validity (15, 16).
A potential solution to these challenges is adopting a suitable core PROM
set for GenID, using instruments from the Patient-Reported Outcomes
Measurement Information System (PROMIS) (17). PROMIS efficiently and
reliably measures relevant PROs across different health domains. PROMIS
instruments are well-established for their robust psychometric properties
in other disease populations and the general population, and their minimal
burden on patients. PROMIS consists of item banks, which are large sets
of items that measure a single health construct, and are developed using
Item Response Theory (IRT). IRT orders items by difficulty, enabling the
creation of Short Form (SF) questionnaires or the use of Computerized
Adaptive Testing (CAT). When using CAT, subsequent items are tailored
to the respondent’s previous answers (18). This approach makes PROMIS
instruments shorter, patient-specific, and more reliable than other PROMs
(19, 20). Given the clinical heterogeneity in GenID, PROMIS CATs may be
particularly suitable for this population, as items are tailored to the specific
individual with GenID and their level of functioning. Gradually, studies
are adopting PROMIS instruments for specific GenID groups, such as
neurofibromatosis type 1 and TSC (21-23). PROMIS instruments also form
the basis of the Dutch generic PROM set, which is recommended for use in
medical care in the Netherlands to enhance comparability across disease
groups and minimize the use of many different PROMs (24, 25).
Previously, we identified relevant PROs through scoping reviews and a
qualitative study (26, 27), classified and conceptualized these PROs with an
expert group, and prioritized these PROs with key stakeholders to establish
a GenID core PRO set (28). The aim of the current study is to select a GenID
core PROM set to measure this core PRO set and evaluate its psychometric
properties in a Dutch sample of caregivers of children with GenID, adults
with GenID, and caregivers of adults with GenID (Figure 1), focusing on the
reliability, efficiency, and construct validity of the instruments.
7
171