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Table 5 Recommended changes of tested COAs for use in a PROS population

From: Qualitative research with patients and caregivers of patients with PIK3CA related overgrowth spectrum: content validity of clinical outcome assessments

Study finding Recommended change to COA for use in PROS
The ability to comprehend and self-report differed based on age and syndrome of the participant. Participants under 12 years of age were not able to consistently self-report; adult and child participants’ ability to self-report may need to be determined on an individualized basis in relation to their syndrome Pediatric versions of COAs included in this battery are best used for individuals ages 12 -17, while observer-report versions are most appropriate for those below the age of 12
  Individuals suffering from cognitive impairment might not be able to self-report, in which case caregiver observer-report would be most appropriate
The variability of patients’ symptom experience within and across days suggested the need for frequent assessment of symptoms. Also, due to the variability of experiences of pain and other symptoms throughout the day, many participants were struggling with questions that asked them to report averages The BPI item measuring “average” pain (BPI-SF item 5) may not be appropriate in this population, due to participants’ difficulty selecting a response on this item. The BPI item measuring “worst pain” (BPI-SF Item 3) may be most appropriate to assess pain intensity, as participants reported this item as relevant, understandable, and easy to select a response
  The PGI-S was reported by both adults and children as difficult to respond to due to variation of symptoms, both within and across days, and the sheer number of symptoms experienced as part of this condition. Frequent assessment may be useful to capture both good and bad days due to fluctuating symptoms. For example, Ecological Momentary Assessments (EMAs) or Experience Sampling Methods (ESMs) could be appropriate, if tested for use in this population to gather daily changes in symptoms. Additionally, a more specific recall period, such as “past 24-h,” would standardize the period being measured for each participant
Participants acknowledged different aspects of their pain, both in relation to pain type and pain location. Each of these aspects may vary within a particular day and across days Aspects of pain that participants thought important to describe their pain at a given point in time included pain type, location, intensity, and severity. The battery of COAs tested did not include an item to measure pain type. Future use of these measures in this population should consider whether an item related to pain type is important for its purpose
  Given that pain in PROS often occurs in multiple areas of the body, an option to report more than one location should be captured. Patients also identified pain type as an important concept to measure. Including the BPI pain type item may therefore provide a more comprehensive measure of pain in PROS patients
While children understood the Wong Baker FACES scale, nearly half reported difficulty selecting a response as they struggled to identify with the face associated with the numerical rating scale While the Wong-Baker FACES scale is validated for use in ages 3 and up, the BPI may be a more appropriate tool for capturing the nature of pain experienced by children with PROS, aged 12 and over