Development of a novel observer-reported outcome measure for the assessment of Respiratory Syncytial Virus (RSV) infection symptoms in pediatric clinical trials
Abstract Background Respiratory syncytial virus (RSV) is a seasonal infection affecting most children by 2 years of age and the leading cause of lower respiratory tract infection requiring hospitalization in infants. Novel antiviral medications are in development to improve the clinical outcomes of...
| Main Authors: | , , , , , , , |
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| Format: | Article |
| Language: | English |
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SpringerOpen
2018-02-01
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| Series: | Journal of Patient-Reported Outcomes |
| Subjects: | |
| Online Access: | http://link.springer.com/article/10.1186/s41687-018-0034-9 |
| _version_ | 1819079850539876352 |
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| author | Sandy Lewis Carla DeMuro Stan L. Block Shelly Senders Paul Wisman Seth Toback Jason W. Chien Valerie Williams |
| author_facet | Sandy Lewis Carla DeMuro Stan L. Block Shelly Senders Paul Wisman Seth Toback Jason W. Chien Valerie Williams |
| author_sort | Sandy Lewis |
| collection | DOAJ |
| description | Abstract Background Respiratory syncytial virus (RSV) is a seasonal infection affecting most children by 2 years of age and the leading cause of lower respiratory tract infection requiring hospitalization in infants. Novel antiviral medications are in development to improve the clinical outcomes of RSV; however, no clinical outcome assessments (COAs) for RSV have been developed in alignment with the United States Food and Drug Administration patient-reported outcome guidance to assist in the evaluation of new therapies. To address this need, an observer-reported outcome (ObsRO) measure designed to assess observable RSV symptoms was created. Methods The literature was reviewed to evaluate existing COAs and identify constructs of interest. Individual caregiver interviews elicited concepts that informed item development, and candidate items were subsequently evaluated in two rounds of cognitive testing. Separate cohorts of caregivers of RSV-infected nonhospitalized and hospitalized infants participated. Therapeutic-area experts provided input throughout the instrument development process. Results Caregivers of 39 children < 24 months old with RSV (31 nonhospitalized, 8 hospitalized) participated in in-depth, individual interviews during concept elicitation and cognitive debriefing, resulting in 21 concepts identified as potentially observable and relevant to young children with RSV. The item pool was reduced to 12 cardinal symptoms and behavior impacts reported to be directly observable by caregivers, with 10 daytime and 9 nighttime symptoms to capture diurnal variation in severity. Conclusions The RSV Caregiver Diary assesses RSV symptom severity and change from the parent or caregiver perspective in a standardized manner to measure treatment benefit. Following psychometric evaluation and refinement, this tool is expected to be suitable for assisting in the clinical development of RSV therapeutics. |
| first_indexed | 2024-12-21T19:35:32Z |
| format | Article |
| id | doaj.art-8dcb8cdb9c544022a54a2396aeabcb47 |
| institution | Directory Open Access Journal |
| issn | 2509-8020 |
| language | English |
| last_indexed | 2024-12-21T19:35:32Z |
| publishDate | 2018-02-01 |
| publisher | SpringerOpen |
| record_format | Article |
| series | Journal of Patient-Reported Outcomes |
| spelling | doaj.art-8dcb8cdb9c544022a54a2396aeabcb472022-12-21T18:52:36ZengSpringerOpenJournal of Patient-Reported Outcomes2509-80202018-02-01211910.1186/s41687-018-0034-9Development of a novel observer-reported outcome measure for the assessment of Respiratory Syncytial Virus (RSV) infection symptoms in pediatric clinical trialsSandy Lewis0Carla DeMuro1Stan L. Block2Shelly Senders3Paul Wisman4Seth Toback5Jason W. Chien6Valerie Williams7RTI Health SolutionsRTI Health SolutionsKentucky Pediatric & Adult ResearchSenders PediatricsPediatric Research of CharlottesvillebiomerieuxGilead Sciences Inc.RTI Health SolutionsAbstract Background Respiratory syncytial virus (RSV) is a seasonal infection affecting most children by 2 years of age and the leading cause of lower respiratory tract infection requiring hospitalization in infants. Novel antiviral medications are in development to improve the clinical outcomes of RSV; however, no clinical outcome assessments (COAs) for RSV have been developed in alignment with the United States Food and Drug Administration patient-reported outcome guidance to assist in the evaluation of new therapies. To address this need, an observer-reported outcome (ObsRO) measure designed to assess observable RSV symptoms was created. Methods The literature was reviewed to evaluate existing COAs and identify constructs of interest. Individual caregiver interviews elicited concepts that informed item development, and candidate items were subsequently evaluated in two rounds of cognitive testing. Separate cohorts of caregivers of RSV-infected nonhospitalized and hospitalized infants participated. Therapeutic-area experts provided input throughout the instrument development process. Results Caregivers of 39 children < 24 months old with RSV (31 nonhospitalized, 8 hospitalized) participated in in-depth, individual interviews during concept elicitation and cognitive debriefing, resulting in 21 concepts identified as potentially observable and relevant to young children with RSV. The item pool was reduced to 12 cardinal symptoms and behavior impacts reported to be directly observable by caregivers, with 10 daytime and 9 nighttime symptoms to capture diurnal variation in severity. Conclusions The RSV Caregiver Diary assesses RSV symptom severity and change from the parent or caregiver perspective in a standardized manner to measure treatment benefit. Following psychometric evaluation and refinement, this tool is expected to be suitable for assisting in the clinical development of RSV therapeutics.http://link.springer.com/article/10.1186/s41687-018-0034-9Respiratory syncytial virusClinical outcomes assessmentObserver-reported outcomeCaregiver diary |
| spellingShingle | Sandy Lewis Carla DeMuro Stan L. Block Shelly Senders Paul Wisman Seth Toback Jason W. Chien Valerie Williams Development of a novel observer-reported outcome measure for the assessment of Respiratory Syncytial Virus (RSV) infection symptoms in pediatric clinical trials Journal of Patient-Reported Outcomes Respiratory syncytial virus Clinical outcomes assessment Observer-reported outcome Caregiver diary |
| title | Development of a novel observer-reported outcome measure for the assessment of Respiratory Syncytial Virus (RSV) infection symptoms in pediatric clinical trials |
| title_full | Development of a novel observer-reported outcome measure for the assessment of Respiratory Syncytial Virus (RSV) infection symptoms in pediatric clinical trials |
| title_fullStr | Development of a novel observer-reported outcome measure for the assessment of Respiratory Syncytial Virus (RSV) infection symptoms in pediatric clinical trials |
| title_full_unstemmed | Development of a novel observer-reported outcome measure for the assessment of Respiratory Syncytial Virus (RSV) infection symptoms in pediatric clinical trials |
| title_short | Development of a novel observer-reported outcome measure for the assessment of Respiratory Syncytial Virus (RSV) infection symptoms in pediatric clinical trials |
| title_sort | development of a novel observer reported outcome measure for the assessment of respiratory syncytial virus rsv infection symptoms in pediatric clinical trials |
| topic | Respiratory syncytial virus Clinical outcomes assessment Observer-reported outcome Caregiver diary |
| url | http://link.springer.com/article/10.1186/s41687-018-0034-9 |
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