Wellness in the modulator era: An observational study of the impact of CFTR modulator therapy on the well-being of people with cystic fibrosis.

BACKGROUND: People with cystic fibrosis (PwCF) have experienced substantial improvements in health following use of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies. However, less is known about how modulator therapies impact well-being. METHODS: We used a cross-sectional observational study to identify relationships between CFTR modulator therapies, health-related quality of life (HRQoL), and well-being. Adult PwCF and caregivers of children with CF completed the Wellness in the Modulator Era (Well-ME) survey between June 22 and July 31, 2022. HRQoL was measured with PROMIS Global 10/Global 7 + 2 Parent Proxy. We used a mixed methods analysis to compare experiences and concerns of PwCF who currently (n = 665), no longer (n = 51), or never (n = 184) took modulator therapy. RESULTS: Adult PwCF taking a modulator (n = 416) reported better PROMIS global physical health than those who no longer (n = 37) or never took a modulator (n = 94) and better PROMIS global mental health than those who never took a modulator. Caregiver-reported HRQoL was similar across children with CF who currently, no longer, or never took a modulator. PwCF taking a modulator reported larger improvements in physical health, quality of life, social well-being, and treatment burden than those who no longer or never took a modulator. Nearly one-quarter (23 %) of PwCF taking modulator therapy reported worsening of mental well-being. CONCLUSIONS: This study expands our knowledge of well-being among PwCF in the CFTR modulator era as reported by patients and parents. Findings lay the groundwork for establishing future research priorities, policy efforts, and communications in areas that improve well-being for PwCF.

An in vivo analysis of implanted programmable device interference during magnetically controlled growing rod lengthenings: a story of 129 lengthenings.

PURPOSE: Early-onset scoliosis (EOS) is often treated with magnetically controlled growing rods (MCGR) which can be lengthened with a magnetic external remote control (ERC). Many individuals with EOS have concomitant medical conditions which are managed with other implanted programmable devices (IPD). Some providers are concerned that the magnetic field generated during MCGR lengthening may interfere with other IPD, such as ventriculoperitoneal shunts (VPS), intrathecal baclofen pumps (ITBP), vagal nerve stimulators (VNS), and cochlear implants (CI). The aim of this study was to evaluate the safety of MCGR lengthenings in patients with EOS and other IPD. METHODS: This single-center, single-surgeon case series followed 12 patients with 13 IPD as they underwent treatment with MCGR. Post MCGR lengthening, monitoring of patient symptoms, and interrogation of IPD were conducted to evaluate for magnetic interference. RESULTS: After 129 MCGR lengthenings, post-lengthening VPS interrogation found 2 instances of potential interference in settings (both in Medtronic Strata shunts); however, no pre-lengthening interrogation was completed to confirm if these changes occurred prior to or during the lengthening procedure. ITBP interrogation found no changes, and there were no patient-reported adverse effects related to VNS or CI function. CONCLUSION: It is safe and effective to utilize MCGR in patients with IPD. However, the possibility of magnetic interference must be considered, particularly in individuals with VPS. We recommend approaching with the ERC from a caudal direction to minimize potential interference and all patients be monitored during treatment. If possible, IPD settings should be assessed pre-lengthening, confirmed afterwards and readjusted if necessary. LEVEL OF EVIDENCE: Level IV.

Positive and negative impacts of elexacaftor/tezacaftor/ivacaftor: Healthcare providers' observations across US centers.

BACKGROUND: Elexacaftor/tezacaftor/ivacaftor (ETI) has been associated with unprecedented clinical improvements, transforming the management of cystic fibrosis (CF). However, side effects with implications for safety and well-being have been reported, including neuropsychiatric changes. This study aimed to better characterize the emerging positive and negative impacts of ETI. METHODS: The Cystic Fibrosis Foundation's Mental Health Advisory Committee distributed a 26-item survey to US CF care teams to assess clinician observations of patient-reported experiences with ETI. Survey responses measured the prevalence of these effects in five domains: (1) positive physical and psychological effects, (2) sleep difficulties, (3) cognitive difficulties, (4) worsening mental health, and (5) concerns about the future and finances. RESULTS: Seventy-five healthcare providers responded from a pediatric, adult, and combined centers. Positive physical effects of ETI and increased optimism were reported in the upper quartiles (50%-100%) and rated as having a significant impact on daily functioning. Sleep and cognitive difficulties were reported in 1%-24%, with slight impacts on functioning, and psychological symptoms (e.g., increased stress, depression, anxiety) and new psychiatric medications were reported in 1%-24%, with moderate impacts. Concerns about the future were reported in 1%-24%, with minimal impacts. CONCLUSION: Across US centers, providers most often observed positive physical effects of ETI. However, a variety of negative side effects were also reported, including sleep disruptions and worsening psychological functioning, which should be systematically monitored by CF teams. These national-level data are a first step in evaluating the prevalence and consequences of these side effects and can directly inform future studies.

National integration of mental health screening and treatment into specialized care for cystic fibrosis: What predicts success?

OBJECTIVES: The CF Foundation sponsored competitive awards for Mental Health Coordinators (MHCs) from 2016 to 2018 to implement the international guidelines for mental health screening and treatment in US CF centers. Longitudinal surveys evaluated success in implementing these guidelines using the Consolidated Framework for Implementation Research (CFIR). METHODS: MHCs completed annual surveys assessing implementation from preparation/basic implementation (e.g., using recommended screeners) to full implementation/sustainability (e.g., providing evidence-based treatments). Points were assigned to questions through consensus, with higher scores assigned to more complex tasks. Linear regression and mixed effects models were used to: (1) examine differences in centers and MHC characteristics, (2) identify predictors of success, (3) model the longitudinal trajectory of implementation scores. RESULTS: A total of 122 MHCs (88.4% responded): Cohort 1, N = 80; Cohort 2, N = 30; and Cohort 3, N = 12. No differences in center characteristics were found. Significant improvements in implementation were observed across centers over time. Years of experience on a CF team was the only significant predictor of success; those with 1-5 years or longer reported the highest implementation scores. Change over time was predicted by >5 years of experience. CONCLUSIONS: Implementation of the mental health guidelines was highly successful over time. Funding for MHCs with dedicated time was critical. Longitudinal modeling indicated that CF centers with diverse characteristics could implement them, supported by evidence from the CF Patient Registry showing nearly universal uptake of mental health screening in the United States. Years of experience predicted better implementation, suggesting that education and training of MHCs and retention of experienced providers are critical to success.

A patient's experience as a research participant.

The author presents a brief synopsis of an interview with a research participant, highlighting the individual's insights and recommendations both for participants and for researchers.

Breast cancer in Native American women treated at an urban-based Indian health referral center 1982-2003.

BACKGROUND: Breast cancer incidence and survival varies by race and ethnicity. There are limited data regarding breast cancer in Native American women. METHODS: A retrospective chart review was performed of 139 women diagnosed with breast cancer and treated at Phoenix Indian Medical Center in Phoenix, AZ between January 1, 1982 and December 31, 2003. Data points included tribal affiliation, and quantum (percentage American Indian Heritage) along with patient, tumor, and treatment characteristics. RESULTS: Most patients (79%) presented initially with physical symptoms. There were no significant differences based on tribal affiliation; however, higher quantum predicted both larger tumor size and more advanced stage at diagnosis. Obesity also significantly correlated with larger tumor size and more advanced stage. Treatment was inadequate in 21%; this was attributed to traditional beliefs, patient refusal, or financial issues. CONCLUSIONS: When compared to national averages, Native American women presented at a later stage, underutilized screening, and had greater delays to treatment.