scholarly journals Findings from a feasibility study of estradiol for hypogonadal women with cystic fibrosis-related bone disease

2021 ◽  
Vol 7 (1) ◽  
Author(s):  
Malinda Wu ◽  
Rabindra Tirouvanziam ◽  
Neha Arora ◽  
Vin Tangpricha
Keyword(s):  
Quality Of Life ◽  
Cystic Fibrosis ◽  
Lung Function ◽  
Feasibility Study ◽  
Bone Disease ◽  
Inflammatory Markers ◽  
Critical Role ◽  
Estrogen Replacement ◽  
Mineral Density

Abstract Background Advancements in therapies for patients with cystic fibrosis (CF) have decreased mortality, leading to increased prevalence of chronic complications including bone disease. CF-related bone disease (CFBD) is characterized by low bone mineral density (BMD) and fragility fractures. Estrogen deficiency increases bone resorption, resulting in decreased BMD that can be restored with estrogen replacement. Current CF guidelines recommend treating female hypogonadal patients with CFBD with estrogen replacement, but no prospective study has investigated the effects of estrogen supplementation on CFBD. Estrogen is known to modulate inflammatory markers and autoimmune diseases. We proposed to test the hypothesis that estrogen status plays a critical role in optimizing bone health, modulating inflammation, preserving lung function, and maximizing quality of life in premenopausal women with CF. Methods We planned a randomized, placebo-controlled, investigator- and patient-blinded, pilot trial with two parallel arms. Eligible subjects were women with CF 18–50 years old with hypogonadism and low BMD who were not taking systemic glucocorticoids, had not had a prior transplant, and did not have contraindications to oral estradiol. Subjects would be block randomized to receive oral estradiol or placebo for 6 months. The primary outcome was feasibility metrics. Secondary outcomes included relative changes in estradiol, bone turnover markers, lung function, inflammatory markers, and quality of life metrics. The study was funded through departmental funds. Results Of 233 subjects screened, 86 subjects were women with CF 18–50 years old and none were eligible for participation. Most subjects were excluded due to absent DXA report (24%), normal BMD (22%), or use of systemic estrogen (16%). Due to difficulty recruiting the planned 52 subjects, the trial was closed for recruitment and no subjects were randomized. Conclusion This study was designed to investigate the feasibility of a safety and efficacy trial of estrogen therapy for women with CF. Unfortunately, due to eligibility criteria, the study was unable to recruit subjects. This feasibility study highlights the need for improved BMD screening in young women with CF. Future study designs may require the incorporation of a screening DXA as part of subject recruitment. Trial registration The study was registered on ClinicalTrials.gov (NCT03724955).

scholarly journals Effects of aerobic interval training on glucose tolerance in children and adolescents with cystic fibrosis: a randomized trial protocol

Trials ◽  
2019 ◽  
Vol 20 (1) ◽  
Author(s):  
Karolinne Souza Monteiro ◽  
Matheus de Paiva Azevedo ◽  
Lucas Menescal Jales ◽  
Fernanda Elizabeth Pereira da Silva ◽  
Ricardo Fernando Arrais ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cystic Fibrosis ◽  
Lung Function ◽  
Glucose Tolerance ◽  
Children And Adolescents ◽  
Interval Training ◽  
Moderate Intensity ◽  
Registration System ◽  
Aerobic Interval Training

Abstract Background Individuals with cystic fibrosis (CF) may develop CF-related diabetes (CFDR). This comorbidity is related to a poorer quality of life, microvascular complications, a decline in lung function, and an increase in exacerbations, as well as delayed growth and puberty. Evidence exists that physical exercise contributes to glycemic control in individuals with non-CF-related diabetes. This exercise is usually continuous with moderate intensity and long duration, which can cause muscle dyspnea and fatigue in CF individuals. Aerobic interval training (AIT) emerges as a safe and effective alternative for treating these individuals. The objective of this study is to evaluate the effects of AIT on glucose tolerance in children and adolescents with CF. Methods This study will be a two-arm, prospectively registered, randomized controlled trial with blind assessors and twenty 6- to 18-year-old individuals with cystic fibrosis (CF) from two different Brazilian states. People with CF will be randomly allocated to either the experimental or control group using block randomization, stratified by puberty stage,. Participants from both groups will receive an educational intervention and will be asked to continue their usual daily treatment for the full duration of the study. Those in the experimental group will perform AIT on a cycle ergometer at home three times a week, for 8 consecutive weeks. The sample characterization will include an assessment of puberty stage, socioeconomic status, dyspnea, and anthropometry. The primary outcome will be the change in glucose tolerance, while the secondary outcomes will include lung function, exercise tolerance, respiratory muscle strength, quality of life, and CF exacerbations. All outcomes will be assessed at baseline, week 9, and week 17. Discussion This is the first study to evaluate the effects of AIT on glucose tolerance in children and adolescents with CF. This study will serve as a basis for guiding clinical practice and decision-making in treating glucose intolerance and CF-related diabetes (CFRD) in children and adolescents with CF. Trial registration ClinicalTrials.gov Protocol Registration System: NCT03653949. Registered on August 31, 2018.

Telephone monitoring and home visits significantly improved the quality of life, treatment adherence and lung function in children with cystic fibrosis

2017 ◽  
Vol 106 (11) ◽  
pp. 1882-1882 ◽  
Author(s):  
Elisavet-Anna Chrysochoou ◽  
Elpis Hatziagorou ◽  
Fotis Kirvassilis ◽  
John Tsanakas
Keyword(s):  
Quality Of Life ◽  
Cystic Fibrosis ◽  
Lung Function ◽  
Treatment Adherence ◽  
Home Visits ◽  
Telephone Monitoring

scholarly journals Estrogen Supplementation Is Associated With Higher Quality of Life Scores in Women With Cystic Fibrosis

2021 ◽  
Vol 5 (Supplement_1) ◽  
pp. A732-A732
Author(s):  
Malinda Wu ◽  
Neha Arora ◽  
Viranuj Sueblinvong ◽  
William R Hunt ◽  
Vin Tangpricha
Keyword(s):  
Quality Of Life ◽  
Cystic Fibrosis ◽  
Estrogen Therapy ◽  
Cross Sectional Study ◽  
Rank Test ◽  
Sectional Study ◽  
Mineral Density ◽  
Cross Sectional ◽  
Domain Scores

Abstract Purpose: With rapid advancements in therapeutic options for patients with cystic fibrosis (CF), the median predicted survival has increased to 47 years along with the prevalence of non-pulmonary complications for patients with CF. Women with CF suffer irregular menses, sexual dysfunction and low bone mineral density. With increasing pregnancies among women with CF, they may consider contraception. Estrogen supplementation may modulate these outcomes and others. The purpose of this study was to explore the effects of supplemental estrogen use on quality of life (QOL) in CF. Methods: Women with CF ages 16-50 years were administered a validated CF-specific QOL survey (CFQ-R) during a routine CF clinic visit through an IRB-approved cross-sectional study. The QOL domain scores of subjects taking and not taking estrogen were compared pairwise by Kruskal Wallis tests and overall by Wilcoxon signed rank test. Results: The estrogen exposed and estrogen unexposed subjects with CF had similar age, BMI, FEV1, race, CF mutation, pancreatic insufficiency, diabetes and relationship status. The estrogen exposed subjects were taking 20-30 mcg of ethinyl estradiol in oral contraceptive pills. The estrogen exposed subjects had consistently higher QOL scores than the estrogen unexposed subjects (p=0.001). The estrogen exposed subjects had significantly higher scores in 7 of the 12 CFQ-R categories: physical, vitality, treatment burden and role domains and weight, respiratory and digestion symptom scales (p<0.05). When correcting for multiple comparisons, the estrogen-supplemented women had higher role domain scores than women not taking estrogen supplement (p=0.03). Conclusions: Estrogen supplementation was associated with improved quality of life in women with CF. This cross-sectional study highlights the need for further investigation into the potential benefits of estrogen supplementation. The dose, route, formulation and timing of estrogen therapy may modulate the beneficial effects for women with CF. Support: This work was supported by the National Center for Advancing Translational Sciences of the National Institutes of Health under award TL1TR002382 and UL1TR002378, and Cystic Fibrosis Foundation award WU20D0. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH or CFF.

scholarly journals Management of chronic Pseudomonas aeruginosa infection with inhaled levofloxacin in people with cystic fibrosis

2021 ◽  
Author(s):  
J Stuart Elborn ◽  
Patrick A Flume ◽  
Donald R Van Devanter ◽  
Claudio Procaccianti
Keyword(s):  
Quality Of Life ◽  
Cystic Fibrosis ◽  
Pseudomonas Aeruginosa ◽  
Lung Function ◽  
Standard Care ◽  
Bacterial Infections ◽  
Pulmonary Infections ◽  
Pseudomonas Aeruginosa Infection

People with cystic fibrosis (CF) are highly susceptible to bacterial infections of the airways. By adulthood, chronic Pseudomonas aeruginosa ( Pa) is the most prevalent infective organism and is difficult to eradicate owing to its adaptation to the CF lung microenvironment. Long-term suppressive treatment with inhaled antimicrobials is the standard care for reducing exacerbation frequency, improving quality of life and increasing measures of lung function. Levofloxacin (a fluoroquinolone antimicrobial) has been approved as an inhaled solution in Europe and Canada, for the treatment of adults with CF with chronic P. aeruginosa pulmonary infections. Here, we review the clinical principles relating to the use of inhaled antimicrobials and inhaled levofloxacin for the management of P. aeruginosa infections in patients with CF.

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