| Title |
Qualitative research with patients and caregivers of patients with PIK3CA related overgrowth spectrum: content validity of clinical outcome assessments
|
|---|---|
| Published in |
Journal of Patient-Reported Outcomes, July 2022
|
| DOI | 10.1186/s41687-022-00481-8 |
| Pubmed ID | |
| Authors |
Kimberly Raymond, Susan Vallow, Cory Saucier, Kristi Jackson, Michelle K. White, Andrew Lovley, Denise D’Alessio |
| Abstract |
PIK3CA-Related Overgrowth Spectrum (PROS) are rare syndromes caused by a mutation in the PIK3CA gene, including fibroadipose hyperplasia or overgrowth; congenital lipomatous overgrowth, vascular malformations, epidermal nevi, scoliosis/skeletal and spinal (CLOVES); megalencephaly-capillary malformation (MCAP or M-CM); fibro-adipose vascular anomaly (FAVA); Klippel-Trenaunay syndrome (KT; also known as, Klippel-Trenaunay-Weber syndrome); capillary, lymphatic, and venous malformations (CLVM); and lymphatic malformation (LM). Characterized by malformations and tissue overgrowth, PROS manifests at birth or in early childhood. Pain and functional limitations associated with these conditions may greatly impact the health-related quality of life (HRQoL) of persons with PROS including physical functioning, work/school, social functioning, and emotional well-being. Selected clinical outcome assessments (COAs), identified during a literature review, were tested with adults with PROS, and children with PROS and their caregivers to determine comprehensibility, relevance, and appropriateness for measuring symptom severity and HRQoL. Tested were the Patient Global Impression of Symptom Severity (PGI-S), Brief Pain Inventory (BPI), Wong-Baker FACES, Patient-Reported Outcomes Measurement Information System (PROMIS) Profile, PROMIS Pediatric Short Form Sleep Disturbance, and PROMIS Dyspnea Severity. Qualitative interviews tested the self-report adult, self-report pediatric, and observer-report COAs with adults with PROS, and children with PROS and their caregivers. Ten adults (≥ 18 years old) with PROS, and 20 children (6-17 years old) with PROS and their caregivers, participated. All reported positive feedback on item relevance. Adults and children over the age of 12 comprehended and responded to self-reported items. Secondary objectives examined the age children could self-report their conditions using pediatric versions and assessed available observer-report versions of the COAs with caregivers. Some participants under the age of 12 had trouble understanding some terminology. Further, adults and children with cognitive impairment associated with MCAP/M-CM sometimes had difficulty with self-report. Caregivers were able to report their child's symptoms and impacts using observer-report COAs. Participant feedback prompted further consideration of the measurement of pain in this population, including variability of pain over time, location of pain, and type. This study provided valuable information from patients about PROS, supporting the content validity of the COAs, with recommended revisions. COAs are easily understood by persons with PROS and caregivers and are appropriate for measuring symptoms and disease-related impacts across diverse PROS syndromes in clinical trials. |
X Demographics
The data shown below were collected from the profiles of 2 X users who shared this research output. Click here to find out more about how the information was compiled.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Unknown | 2 | 100% |
Demographic breakdown
| Type | Count | As % |
|---|---|---|
| Science communicators (journalists, bloggers, editors) | 1 | 50% |
| Members of the public | 1 | 50% |
Mendeley readers
The data shown below were compiled from readership statistics for 15 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Unknown | 15 | 100% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Other | 1 | 7% |
| Student > Doctoral Student | 1 | 7% |
| Student > Bachelor | 1 | 7% |
| Researcher | 1 | 7% |
| Student > Postgraduate | 1 | 7% |
| Other | 0 | 0% |
| Unknown | 10 | 67% |
| Readers by discipline | Count | As % |
|---|---|---|
| Medicine and Dentistry | 3 | 20% |
| Business, Management and Accounting | 1 | 7% |
| Biochemistry, Genetics and Molecular Biology | 1 | 7% |
| Social Sciences | 1 | 7% |
| Psychology | 1 | 7% |
| Other | 0 | 0% |
| Unknown | 8 | 53% |
Attention Score in Context
This research output has an Altmetric Attention Score of 1. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 14 July 2022.
All research outputs
#17,789,675
of 22,851,489 outputs
Outputs from Journal of Patient-Reported Outcomes
#352
of 496 outputs
Outputs of similar age
#285,293
of 434,068 outputs
Outputs of similar age from Journal of Patient-Reported Outcomes
#21
of 34 outputs
Altmetric has tracked 22,851,489 research outputs across all sources so far. This one is in the 19th percentile – i.e., 19% of other outputs scored the same or lower than it.
So far Altmetric has tracked 496 research outputs from this source. They receive a mean Attention Score of 3.7. This one is in the 22nd percentile – i.e., 22% of its peers scored the same or lower than it.
Older research outputs will score higher simply because they've had more time to accumulate mentions. To account for age we can compare this Altmetric Attention Score to the 434,068 tracked outputs that were published within six weeks on either side of this one in any source. This one is in the 30th percentile – i.e., 30% of its contemporaries scored the same or lower than it.
We're also able to compare this research output to 34 others from the same source and published within six weeks on either side of this one. This one is in the 29th percentile – i.e., 29% of its contemporaries scored the same or lower than it.