Gender diverse youth with cystic fibrosis have unique health needs. Providers should be aware of ... more Gender diverse youth with cystic fibrosis have unique health needs. Providers should be aware of existing health disparities in this population as well as aspects of gender-affirming care including hormone therapy, chest binding, and use of affirming language. This communication provides an introduction to these concerns.
Bacteriophage therapy, which uses lytic viruses as antimicrobials, has received renewed interest ... more Bacteriophage therapy, which uses lytic viruses as antimicrobials, has received renewed interest to address the emerging antimicrobial resistance (AMR) crisis. Cystic fibrosis (CF), a disease complicated by recurrentP. aeruginosapulmonary infections that cause lung function decline, is an example where AMR is already a clinical problem. While bacteria evolve bacteriophage resistance, we developed a strategy to select bacteriophages that target bacterial cell surface receptors that contribute to antibiotic resistance or virulence. Thus, in addition to killing bacteria, these phages steer surviving, evolved bacteria to antibiotic re-sensitivity or attenuated virulence. Here, we present outcomes from nine CF adults treated with nebulized bacteriophage therapy for AMRP. aeruginosausing this personalized approach. Results showed that phage therapy: 1) reduced sputumP. aeruginosa, 2) showed evidence for predicted trade-offs in most subjects, and 3) improved lung function, which may reflec...
With recent therapeutic advances in care, people with cystic fibrosis (CF) are living longer and ... more With recent therapeutic advances in care, people with cystic fibrosis (CF) are living longer and healthier lives. Development of the cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies has led to improved function of the CFTR protein resulting in improved lung function, decreased rates of pulmonary exacerbations and improved nutritional status for the majority of people with CF. Given improved quality and quantity of life, more people with CF are considering becoming pregnant than ever before. Since the first reported pregnancy in a woman with CF in 1960, the management of pregnancy in CF has been of increased interest and is an active field of research.In this review, we aim to discuss the management of pregnancy in CF. We discuss the optimisation of preconception health, management of maintenance CF therapies, and use of CFTR modulators during pregnancy and lactation. We also describe the management of pulmonary exacerbations during pregnancy as well as ...
Most people with cystic fibrosis (CF) are diagnosed following abnormal newborn screening (NBS), w... more Most people with cystic fibrosis (CF) are diagnosed following abnormal newborn screening (NBS), which begins with measurement of immunoreactive trypsinogen (IRT) values. A case report found low concentrations of IRT in an infant with CF exposed to the CF transmembrane conductance regulator (CFTR) modulator, elexacaftor–tezacaftor–ivacaftor (ETI), in utero. However, IRT values in infants born to mothers taking ETI have not been systematically assessed. We hypothesized that ETI-exposed infants have lower IRT values than newborns with CF, CFTR-related metabolic syndrome/CF screen positive, inconclusive diagnosis (CRMS/CFSPID), or CF carriers. IRT values were collected from infants born in Indiana between 1 January 2020, and 2 June 2022, with ≥1 CFTR mutation. IRT values were compared to infants born to mothers with CF taking ETI followed at our institution. Compared to infants identified with CF (n = 51), CRMS/CFSPID (n = 21), and CF carriers (n = 489), ETI-exposed infants (n = 19) had...
WS05.01 AAV mediated gene therapy for cystic fibrosis: interim results from a phase 1/2 clinical trial
Journal of Cystic Fibrosis
P094 A Phase II study to evaluate the safety, tolerability, pharmacodynamics and pharmacokinetics of BI 1291583 in patients with cystic fibrosis bronchiectasis (the Clairafly™ study)
Journal of Cystic Fibrosis
P312 Provider perspectives and practices related to sexual and reproductive care provision for males with cystic fibrosis
Journal of Cystic Fibrosis
Safety and efficacy of vanzacaftor–tezacaftor–deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials
The Lancet Respiratory Medicine
170 Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis and at least one F508del allele: 144-week interim results from an open-label extension study
Journal of Cystic Fibrosis
Clinician attitudes and practices on pregnancy planning and care in cystic fibrosis
Journal of Cystic Fibrosis
Bone mineral density screening by DXA for people with cystic fibrosis: A registry analysis of patient and program factors influencing rates of screening
Journal of Cystic Fibrosis, 2022
BACKGROUND Current guidelines recommend screening for Cystic Fibrosis (CF) related bone disease (... more BACKGROUND Current guidelines recommend screening for Cystic Fibrosis (CF) related bone disease (CFBD) using dual-energy x-ray absorptiometry (DXA) for all people ≥ 18 years of age and select people ≥ 8 years of age. However, adherence to these guidelines is variable. This study aims to evaluate screening practices among adult programs in the US and identify patient and program-based characteristics which may influence screening. METHODS The CF Foundation Patient Registry (CFFPR) was used to identify all people over the age of 18 who were seen at adult CF programs and received screening for CFBD using DXA at least once between 2014 and 2018. Associations with patient and program level characteristics were assessed using the Chi Square test. Patient level variables were also examined using standardized difference to assess for meaningful clinical differences in rates of screening. RESULTS From 2014 to 2018, a total of 15,134 people over the age of 18 were identified in the CFFPR. Of these people, 9,023 (60%) received a DXA during the time period. The median rate of screening by program was 66% and programs in the highest quartile of screening obtained DXAs on >76% of their population. At the program level, larger size and increased adherence to other guideline practices such as OGTT screening and 4 visits, 4 cultures in a year correlated with higher rates of screening for CFBD. At the patient-level, people with lower lung function (FEV1 <90%) and those with CF related diabetes were more likely to be screened. People without health insurance were less likely to receive recommended screening. CONCLUSION Screening practices for CFBD vary widely across adult programs in the US despite recommendations to screen all people over the age of 18. Factors identified in this analysis may be used to identify those people at highest risk of missing appropriate screening. CFBD has significant implications for our patients and therefore routine screening should be emphasized as part of standard care moving forward.
Background: Given the variability in pulmonary exacerbation (PEx) management within and between C... more Background: Given the variability in pulmonary exacerbation (PEx) management within and between Cystic Fibrosis (CF) Care Centers, it is possible that some approaches may be superior to others. A challenge with comparing different PEx management approaches is lack of a community consensus with respect to treatment-response metrics. In this analysis, we assess the feasibility of using different response metrics in prospective randomized studies comparing PEx treatment protocols. Methods: Response parameters were compiled from the recent STOP (Standardized Treatment of PEx) feasibility study. Pulmonary function responses (recovery of best prior 6-month and 12-month FEV 1 % predicted and absolute and relative FEV 1 % predicted improvement from treatment initiation) and sign and symptom recovery from treatment initiation (measured by the Chronic Respiratory Infection Symptom Score [CRISS]) were studied as categorical and continuous variables. The proportion of patients retreated within 30 days after the end of initial treatment was studied as a categorical variable. Sample sizes required to adequately power prospective 1:1 randomized superiority and non-inferiority studies employing candidate endpoints were explored. Results: The most sensitive endpoint was mean change in CRISS from treatment initiation, followed by mean absolute FEV 1 % predicted change from initiation, with the two responses only modestly correlated (R 2 = .157; P b 0.0001). Recovery of previous best FEV 1 was a problematic endpoint due to missing data and a substantial proportion of patients beginning PEx treatment with FEV 1 exceeding their previous best measures (12.1% N 12-month best, 19.6% N 6-month best). Although mean outcome measures deteriorated approximately 2-weeks post-treatment follow-up, the effect was non-uniform: 62.7% of patients experienced an FEV 1 worsening versus 49.0% who experienced a CRISS worsening. Conclusions: Results from randomized prospective superiority and non-inferiority studies employing mean CRISS and FEV 1 change from treatment initiation should prove compelling to the community. They will need to be large, but appear feasible.
Uploads
Papers by Raksha Jain