The impact of vanzacaftor/tezacaftor/deutivacaftor on parent and child perceived psychosocial functioning in people with cystic fibrosis: a single center experience
Presenter Status
Staff
Abstract Type
Clinical Research
Primary Mentor or Principal Investigator
Ashley Severin
Presentation Type
Poster
Start Date
20-5-2026 11:00 AM
End Date
20-5-2026 12:00 PM
Abstract Text
Background:
(ETI) is a highly effective modulator therapy (HEMT) for children with cystic fibrosis (CwCF). CwCF who are currently prescribed ETI are eligible to transition to vanzacaftor/tezacaftor/deutivacaftor (VTD). While ETI is associated with improvement in health outcomes for most CwCF, there are emerging data regarding neuropsychiatric adverse events (NAEs). VTD is a once daily HEMT shown to be non-inferior to ETI with a similar safety profile, particularly concerning NAEs.
Objectives/Goal:
The objective of this study was to assess the impact of VTD on parent and child perceived psychosocial functioning and sleep for CwCF.
Methods/Design:
The Pediatric Symptom Checklist (PSC) is a 35-item caregiver questionnaire measuring emotional and behavioral problems of the child. Items are rated on a 3-point Likert scale. Scores 28 or higher are considered elevated for ages 6-16. The Patient-Reported Outcomes Measurement Information System (PROMIS) Sleep-Related Impairment and Sleep Disturbance Short Forms are 4-item questionnaires assessing sleep quality and impact of sleep on daily functioning. Raw scores are converted to T-scores, with scores ≥60 indicating sleep disturbances or impairment. Parents of children with CF (PoCwCF) at Children’s Mercy Kansas City CF Center and eligible for VTD were contacted for enrollment. Baseline questionnaires were sent electronically prior to VTD initiation. Subsequent questionnaires will be sent at 3, 6 and 12 months. The PSC and PROMIS questionnaires were completed by PoCwCF aged 6-16 years. The Youth-PSC (Y-PSC) and PROMIS questionnaires were completed by CwCF ages 13-16 years.
Results:
Eighty-five CwCF, aged 6-16 years were eligible for enrollment. Currently, 33 PoCwCF (39%) enrolled and completed baseline questionnaires. Average age of CwCF included was 11 years (± 3.5) and 20 (61%) CwCF were female. Prior to VTD, 31 (94%) CwCF were prescribed ETI, 1 LUM/IVA, and 1 was not on CFTR modulator therapy. The median total score of the baseline PSC was 10.5 (0-33). Questions with the highest scores were 1) distracted easily, 2) fidgety, unable to sit still, and 3) has trouble concentrating. Only 1 score greater than 28 was noted. The median total score for the PROMIS Sleep Disturbance was 62.05 (41.4-68.1) and Sleep Impairment was 53.6 (40- 66.4). The PROMIS Sleep Disturbance had 17 (52%) abnormal scores, while the Sleep Impairment had 6 (18%). At present, 4 CwCF have completed questionnaires. The median total PSC score was 8.5 (6-9). The median total score for the PROMIS Sleep Disturbance was 55.8 (53.7-57.9) and Sleep Impairment was 50.35 (38.3-55.1)
Conclusions:
Baseline PSC data showed 97% of scores in normal range with only one score considered elevated. The highest scoring questions highlight common trends in behavioral adverse events, correlating with concerns raised by the CF community. The increased scores for sleep indicate poor sleep quality and difficulties with sleep onset and maintenance among CwCF. These results provide only baseline data and subsequent questionnaires post-VTD initiation will be crucial in identifying trends and assessing the impact of VTD on psychosocial and sleep functioning. Data collection is ongoing with 3-month follow up data expected Spring 2025. We anticipate participation in this study will increase as CwCF gain access to VTD. Limitations in enrollment include lack of insurance coverage and CwCF not transitioning from ETI to VTD.
The impact of vanzacaftor/tezacaftor/deutivacaftor on parent and child perceived psychosocial functioning in people with cystic fibrosis: a single center experience
Background:
(ETI) is a highly effective modulator therapy (HEMT) for children with cystic fibrosis (CwCF). CwCF who are currently prescribed ETI are eligible to transition to vanzacaftor/tezacaftor/deutivacaftor (VTD). While ETI is associated with improvement in health outcomes for most CwCF, there are emerging data regarding neuropsychiatric adverse events (NAEs). VTD is a once daily HEMT shown to be non-inferior to ETI with a similar safety profile, particularly concerning NAEs.
Objectives/Goal:
The objective of this study was to assess the impact of VTD on parent and child perceived psychosocial functioning and sleep for CwCF.
Methods/Design:
The Pediatric Symptom Checklist (PSC) is a 35-item caregiver questionnaire measuring emotional and behavioral problems of the child. Items are rated on a 3-point Likert scale. Scores 28 or higher are considered elevated for ages 6-16. The Patient-Reported Outcomes Measurement Information System (PROMIS) Sleep-Related Impairment and Sleep Disturbance Short Forms are 4-item questionnaires assessing sleep quality and impact of sleep on daily functioning. Raw scores are converted to T-scores, with scores ≥60 indicating sleep disturbances or impairment. Parents of children with CF (PoCwCF) at Children’s Mercy Kansas City CF Center and eligible for VTD were contacted for enrollment. Baseline questionnaires were sent electronically prior to VTD initiation. Subsequent questionnaires will be sent at 3, 6 and 12 months. The PSC and PROMIS questionnaires were completed by PoCwCF aged 6-16 years. The Youth-PSC (Y-PSC) and PROMIS questionnaires were completed by CwCF ages 13-16 years.
Results:
Eighty-five CwCF, aged 6-16 years were eligible for enrollment. Currently, 33 PoCwCF (39%) enrolled and completed baseline questionnaires. Average age of CwCF included was 11 years (± 3.5) and 20 (61%) CwCF were female. Prior to VTD, 31 (94%) CwCF were prescribed ETI, 1 LUM/IVA, and 1 was not on CFTR modulator therapy. The median total score of the baseline PSC was 10.5 (0-33). Questions with the highest scores were 1) distracted easily, 2) fidgety, unable to sit still, and 3) has trouble concentrating. Only 1 score greater than 28 was noted. The median total score for the PROMIS Sleep Disturbance was 62.05 (41.4-68.1) and Sleep Impairment was 53.6 (40- 66.4). The PROMIS Sleep Disturbance had 17 (52%) abnormal scores, while the Sleep Impairment had 6 (18%). At present, 4 CwCF have completed questionnaires. The median total PSC score was 8.5 (6-9). The median total score for the PROMIS Sleep Disturbance was 55.8 (53.7-57.9) and Sleep Impairment was 50.35 (38.3-55.1)
Conclusions:
Baseline PSC data showed 97% of scores in normal range with only one score considered elevated. The highest scoring questions highlight common trends in behavioral adverse events, correlating with concerns raised by the CF community. The increased scores for sleep indicate poor sleep quality and difficulties with sleep onset and maintenance among CwCF. These results provide only baseline data and subsequent questionnaires post-VTD initiation will be crucial in identifying trends and assessing the impact of VTD on psychosocial and sleep functioning. Data collection is ongoing with 3-month follow up data expected Spring 2025. We anticipate participation in this study will increase as CwCF gain access to VTD. Limitations in enrollment include lack of insurance coverage and CwCF not transitioning from ETI to VTD.
Comments
Full text not provided by primary author
Poster Board Number: 15