J Family Med Prim Care. 2021 May;10(5):1994-1997. doi: 10.4103/jfmpc.jfmpc_2421_20. Epub 2021 May 31.

ABSTRACT

AIMS: To assess the clinical profile and nutritional status of infants with cystic fibrosis (CF) and track their nutritional outcomes with treatment.

MATERIALS AND METHODS: This retrospective study was conducted in a tertiary-care institute in South India. Demographic and clinical information were collected. The nutritional status and treatment outcome was assessed by Z-scores for weight-for-age (WAZ), length-for-age (LAZ), and weight-for-length (WLZ) at diagnosis and follow-up.

RESULTS: Nineteen infants with CF had mean follow-up duration of 9.7 ± 8.7 months. There was a mean delay of 2.9 ± 2.1 months from symptom onset to diagnosis, by which time infants were severely malnourished (mean WAZ -4.68 ± 1.8). Pneumonia, summer dehydration with electrolyte abnormalities (42.1%), and a combination of anemia, hypoalbuminemia, and malnutrition (42.1%) were the predominant features. Significant weight loss had been recorded in undiagnosed infants by second month of life before symptom onset. At follow-up, there was a remarkable improvement in WAZ (P 0.001), but not LAZ and WLZ. There was a high mortality rate of 37% in these infants.

CONCLUSIONS: Malnutrition is a significant morbidity in infants with CF in India. There was significant improvement of WAZ with treatment, but it lagged behind the recommended targets. There is an opportunity for identification of CF infants at the time of vaccination at six and ten weeks of age, by the primary care physician and pediatrician. Screening of young infants having failure to thrive in the immunization clinic may be a strategy for early diagnosis of infants with severe CF phenotype.

PMID:34195137 | PMC:PMC8208186 | DOI:10.4103/jfmpc.jfmpc_2421_20

J Clin Transl Endocrinol. 2021 Jun 12;24:100259. doi: 10.1016/j.jcte.2021.100259. eCollection 2021 Mar.

ABSTRACT

AUTOMATIC REFERRALS WITHIN A CYSTIC FIBROSIS MULTIDISCIPLINARY CLINIC IMPROVE PATIENT EVALUATION AND MANAGEMENT.

BACKGROUND: Cystic fibrosis (CF) affects multiple systems beyond the pulmonary system, including the gastrointestinal and endocrine systems. Many CF clinics focus on pulmonary effects, initiating referrals to other specialties only when a condition has been identified by the primary pulmonary team. Unfortunately, many extrapulmonary manifestations of cystic fibrosis may be overlooked. Thus, implementing a multidisciplinary clinic with automatic referrals to designated subspecialists may improve patient care.

METHODS: This retrospective review of medical records examined the effects of integrating a pediatric endocrinologist into the University of Massachusetts Memorial Medical Center Pediatric CF Clinic in March 2017. In this new CF/Endocrinology clinic, all patients scheduled to see a pulmonologist were automatically referred to pediatric endocrinology. We compared rates of referrals to pediatric endocrinology, oral glucose tolerance tests (OGTTs), and bone density (DEXA) scans before (2013-2016) and after (2017-2020) implementation of this clinic. We also recorded endocrine disorders being evaluated and/or treated after implementation.

RESULTS: The rate of referral to pediatric endocrinology increased from before (4%) to after (82%) (p < 0.0001) implementation of the CF/Endocrinology Clinic. OGTT and DEXA scan screening rates also increased from 7% to 65% (p < 0.0001) and from 6% to 63% (p = 0.0011), respectively. Before implementation, patients were evaluated by endocrinology primarily for CF-related diabetes. After implementation, the diversity of endocrine conditions under evaluation and/or management increased substantially; the most common were vitamin D insufficiency/deficiency (37.2% of clinic patients), glycemic dysregulation (36.8%), and poor weight gain/failure to thrive (17.5%).

CONCLUSION: Implementing a multidisciplinary CF clinic with automatic referrals to pediatric endocrinology improves patient care by promoting early detection and management of endocrine concerns that may have been overlooked and by increasing OGTT and DEXA screening rates.

PMID:34195009 | PMC:PMC8237349 | DOI:10.1016/j.jcte.2021.100259

Cureus. 2021 May 27;13(5):e15283. doi: 10.7759/cureus.15283.

ABSTRACT

Cystic fibrosis (CF) commonly affects those of European descent; however, it can also be found in those of Asian, African, and Caribbean descent. Patients with CF may have significant lung disease, and their perioperative management can be challenging for the anesthesiologist. In this case report, we describe the use of serratus anterior plane block (SAPB) and IV sedation as an alternative to general anesthesia with an endotracheal tube in a patient with CF pulmonary exacerbation presenting to the operating room for a video-assisted thoracic surgery (VATS).

PMID:34194884 | PMC:PMC8235998 | DOI:10.7759/cureus.15283

J Cyst Fibros. 2021 Jun 27:S1569-1993(21)01289-3. doi: 10.1016/j.jcf.2021.06.005. Online ahead of print.

ABSTRACT

BACKGROUND: The potential effects of ivacaftor during pregnancy and breastfeeding on the offspring are still unknown. This study aimed to investigate pre-/postnatal age-related entry into the brain and lungs and transfer of maternally administered drug by the placental and via the milk.

METHODS: In acute experiments Sprague Dawley rats at embryonic day (E) 19, postnatal days (P) 4, 9, 16, and adult were administered an intraperitoneal injection of ivacaftor (40 mg/kg) traced with [3H] ivacaftor. To determine tissue entry, plasma, cerebrospinal fluid (CSF), lungs and brains were collected, and radioactivity measured using liquid scintillation counting. For long term experiments pregnant dams were orally treated at 25 mg/kg/day for 7 days and pups collected at E19. For postnatal pups, dams received treatment for 7 or 14 days and pups were collected at P6, 9, 13 and 16. To estimate placental and milk transfer concentration of ivacaftor in pup & maternal plasma was determined by liquid chromatography-mass spectrometry.

RESULTS: At all ages, entry of ivacaftor into lungs, following either acute or prolonged exposure, was much higher than into brain & CSF. Brain entry appeared higher at earlier ages. Transfer across the placenta and breast milk. was estimated to be around ~40% of maternal plasma.

CONCLUSIONS: Fetal and postnatal rats were exposed to maternally administered ivacaftor via placental and milk transfer. Preferential entry in the lungs at all ages suggests the possibility that exposing CF babies to maternally administered ivacaftor could be beneficial for limiting progression of CF pathology in early development.

PMID:34193363 | DOI:10.1016/j.jcf.2021.06.005

JPGN Rep. 2021 May 27;2(3):e061. doi: 10.1097/PG9.0000000000000061. eCollection 2021 Aug.

ABSTRACT

The European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) published recommendations regarding protection for the paediatric endoscopist during the coronavirus 2019 (COVID-19) pandemic.The aim of this survey was to investigate whether European paediatric gastroenterology centres applied the recommendations and how this extraordinary situation was handled by the different centres.

RESULTS: Twelve paediatric European gastroenterology centres participated. Nine centres (75%) screened their patients for possible COVID-19 infection before the procedure, the same amount of hospitals changed their practice based on the ESPGHAN recommendations. Six-seven percentage of the centres reduced the staff in the endoscopy suite, 83% of the units used FFP2/3 masks and protective goggles during the procedure and 75% wore waterproof gowns.

CONCLUSION: Uniform guidelines could not be applied by all European hospitals at a certain time point of the viral spread, as different regions of Europe were not only affected differently by COVID-19, but also had different access to personal protective equipment.

PMID:34192294 | PMC:PMC8162040 | DOI:10.1097/PG9.0000000000000061

J Palliat Med. 2021 Jun 30. doi: 10.1089/jpm.2021.0186. Online ahead of print.

ABSTRACT

Background: Advance care planning (ACP) is recommended for all patients with cystic fibrosis (CF), yet clear implementation guidelines do not exist. Methods: The University of North Carolina Adult CF Care Team developed a process to implement semistructured multidisciplinary outpatient ACP meetings as routine care for patients with CF. Premeeting and post-meeting surveys were used to elicit patients' attitudes toward ACP. Results: Twenty-seven adults with CF completed a face-to-face ACP meeting, and 13 completed both surveys. Following the multidisciplinary ACP meeting, overall scores for understanding of ACP topics improved by 4.5 points (p = 0.003). Conclusion: We successfully implemented sustainable ACP meetings for adults with CF and found increased comfort with ACP and documentation of wishes after ACP meetings. It is important for CF care providers to meet the needs of this patient population by ensuring that ACP is in place before crisis situations.

PMID:34191614 | DOI:10.1089/jpm.2021.0186

Pediatr Pulmonol. 2021 Jun 30. doi: 10.1002/ppul.25554. Online ahead of print.

ABSTRACT

BACKGROUND: Cystic fibrosis (CF) patients who grow Pseudomonas aeruginosa on respiratory culture are commonly prescribed inhaled tobramycin (TIS) to eradicate the organism. The objective of this study was to determine the impact of a pharmacy technician/pharmacist team, in conjunction with an integrated health-system specialty pharmacy (IHSSP), on the time from positive culture to prescribing and access to TIS in a pediatric CF clinic.

METHODS: A retrospective study of CF patients positive for P. aeruginosa who were prescribed TIS for eradication.

RESULTS: The study included 20 patients in the pre-group and 42 patients in the post-group. Total median (IQR) days from positive culture to TIS being shipped to the patient from the pharmacy was significantly different: 15 (10.25-21) days in the pre-group and 9 (7-14) days in the post groups (p=0.005). The time from positive culture to TIS prescribing was significantly different: 6 (5-12.75) days in the pre-group and 5 (3.75-6) days in the post-group (p=0.01). In the post-group median time from prescription to the patient receiving the TIS was significantly different between the two groups 2 (2-5) days IHSSP group versus 6 (3-9) external specialty pharmacy group (p=0.003)). Time from prescription to prior authorization approval was the same in both groups.

CONCLUSIONS: The addition of the pharmacy team reduced time from culture to TIS being received by the patient. Patients able to fill at the IHSSP received their medication sooner than an external specialty pharmacy. The study shows the benefit of an integrated pharmacy model in conjunction with an IHSSP. This article is protected by copyright. All rights reserved.

PMID:34191410 | DOI:10.1002/ppul.25554

Protein Sci. 2021 Jun 30. doi: 10.1002/pro.4150. Online ahead of print.

ABSTRACT

Membrane proteins play key roles in cellular signaling and transport, represent the majority of drug targets, and are implicated in many diseases. Their relevance renders them important subjects for structural, biophysical, and functional investigations. However, obtaining membrane proteins in high purities is often challenging with conventional purification steps alone. To address this issue, we present here an approach to increase the purity of α-helical transmembrane proteins. Our approach exploits the Thioredoxin (Trx) tag system, which is able to confer some of its favorable properties, such as high solubility and thermostability, to its fusion partners. Using Trx fusions of transmembrane helical hairpin constructs derived from the human cystic fibrosis transmembrane conductance regulator (CFTR) and a bacterial ATP synthase, we establish conditions for the successful implementation of the selective heat treatment procedure to increase sample purity. We further examine systematically its efficacy with respect to different incubation times and temperatures using quantitative gel electrophoresis. We find that minute-timescale heat treatment of Trx-tagged fusion constructs with temperatures ranging from 50-90°C increases the purity of the membrane protein samples from ~60% to 98% even after affinity purification. We show that this single-step approach is even applicable in cases where regular selective heat purification from crude extracts, as reported for Trx fusions to soluble proteins, fails. Overall, our approach is easy to integrate into existing purification strategies and provides a facile route for increasing the purity of membrane protein constructs after purification by standard chromatography approaches. This article is protected by copyright. All rights reserved.

PMID:34191368 | DOI:10.1002/pro.4150

J Bras Pneumol. 2021 Jun 23;47(3):e20210017. doi: 10.36416/1806-3756/e20210017. eCollection 2021.

ABSTRACT

OBJECTIVE: To identify microorganisms in sputum samples of patients with stable non-cystic fibrosis bronchiectasis and to determine risk factors related to the isolation of Pseudomonas aeruginosa (PA) in those patients.

METHODS: Consecutive patients were recruited from a tertiary hospital outpatient clinic in the city of Fortaleza, Brazil. The patients were submitted to spirometry, six-minute walk test, HRCT, and sputum collection. Data on serum fibrinogen levels, disease severity, sputum color, and history of azithromycin treatment were collected.

RESULTS: The study included 112 patients, and females predominated (68%). The mean age was 51.6 ± 17.4 years. Most patients presented with mild-to-moderate disease (83%). The mean six-minute walk distance was 468.8 ± 87.9 m. Mean FEV1 and FVC, in % of predicted values, were 60.4 ± 21.8% and 69.9 ± 18.5%, respectively. The mean serum fibrinogen level was 396.1 ± 76.3 mg/dL. PA was isolated in 47 patients, other potentially pathogenic microorganisms (PPMs) were isolated in 31 patients, and non-PPMs were isolated in 34 patients. Purulent sputum was identified in 77 patients (68%). The patients with PA, when compared with those without it, presented with more severe disease, higher serum fibrinogen levels, and lower FVC%. In addition, purulent sputum and long-term azithromycin treatment were more common in those with PA. The multivariate regression analysis showed that the independent factors associated with PA were serum fibrinogen level > 400 mg/dL (OR = 3.0; 95% CI: 1.1-7.7) and purulent sputum (OR = 4.3; 95% CI: 1.6-11.3).

CONCLUSIONS: In our sample, the prevalence of PA in sputum was 42%. Sputum color and inflammatory markers were able to predict the isolation of PA, emphasizing the importance of routine sputum monitoring.

PMID:34190862 | DOI:10.36416/1806-3756/e20210017

Compr Physiol. 2021 Jun 30;11(3):2135-2190. doi: 10.1002/cphy.c200023.

ABSTRACT

Pediatric pulmonary hypertension (PPH) is a multifactorial disease with diverse etiologies and presenting features. Pulmonary hypertension (PH), defined as elevated pulmonary artery pressure, is the presenting feature for several pulmonary vascular diseases. It is often a hidden component of other lung diseases, such as cystic fibrosis and bronchopulmonary dysplasia. Alterations in lung development and genetic conditions are an important contributor to pediatric pulmonary hypertensive disease, which is a distinct entity from adult PH. Many of the causes of pediatric PH have prenatal onset with altered lung development due to maternal and fetal conditions. Since lung growth is altered in several conditions that lead to PPH, therapy for PPH includes both pulmonary vasodilators and strategies to restore lung growth. These strategies include optimal alveolar recruitment, maintaining physiologic blood gas tension, nutritional support, and addressing contributing factors, such as airway disease and gastroesophageal reflux. The outcome for infants and children with PH is highly variable and largely dependent on the underlying cause. The best outcomes are for neonates with persistent pulmonary hypertension (PPHN) and reversible lung diseases, while some genetic conditions such as alveolar capillary dysplasia are lethal. © 2021 American Physiological Society. Compr Physiol 11:2135-2190, 2021.

PMID:34190343 | DOI:10.1002/cphy.c200023

Hum Fertil (Camb). 2021 Jun 30:1-8. doi: 10.1080/14647273.2021.1946173. Online ahead of print.

ABSTRACT

Infertility is a common health problem that affects around 1 in 6 couples in the United States, where half of these cases are attributed to male factors. Genetics play an important role in infertility and it is estimated that up to 50% of cases are due to genetic factors. Despite this, many male infertility cases are still idiopathic. This study aimed to identify the presence of possibly pathogenic rare variants in a set of candidate genes related to azoospermia in 69 Jordanian men using a next-generation sequencing-based panel covering more than a hundred male infertility related genes. A total of 9 variants were found and validated. Among them, two variants included reported pathogenic variants in CFTR and one novel pathogenic variant in the USP9Y gene. We also report the detection of 6 other variants with uncertain significance in other genes. Interestingly, male cases with CFTR variants did not show the expected cystic fibrosis phenotypes except for infertility. This work helps to uncover the contribution of additional genetic factors to the aetiology of male infertility and highlights the importance to obtain more reliable information about the presence of genetic variation in the Jordanian population.

PMID:34190021 | DOI:10.1080/14647273.2021.1946173

J Clin Psychol Med Settings. 2021 Jun 29. doi: 10.1007/s10880-021-09798-w. Online ahead of print.

ABSTRACT

Increased life expectancy for individuals with complex pediatric-onset conditions means most of this population survive into adulthood. While this is great news for individuals and their families, the traditional adult medical model must adapt to extend the care provided by specialty pediatric practices to primary care. In this paper, we introduce a model of integrated behavioral health (IBH) in a primary care practice for adults with childhood onset medical and developmental conditions. Our discussion includes the role of IBH providers (i.e., psychologists, psychiatrists, and social workers) as members of the integrated team, patient engagement and response to treatment, and innovative ways we strive to meet patient needs. Our review of electronic health records of patients seen at the UR Medicine Complex Care Center suggest that IBH is feasible and highly utilized, with 216 patients (40%) having had contact with an IBH provider on the team at least once. We discuss the challenges of meeting the longer-term needs of this complex patient population and our directions for future growth including creating peer and caregiver support networks, expanding services offered, and continued collaboration with community partners.

PMID:34189638 | DOI:10.1007/s10880-021-09798-w

Biochem Biophys Rep. 2021 Jun 16;27:101054. doi: 10.1016/j.bbrep.2021.101054. eCollection 2021 Sep.

ABSTRACT

Parathyroid hormone (PTH) has previously been shown to enhance the transepithelial secretion of Cl- and HCO3 - across the intestinal epithelia including Caco-2 monolayer, but the underlying cellular mechanisms are not completely understood. Herein, we identified the major signaling pathways that possibly mediated the PTH action to its known target anion channel, i.e., cystic fibrosis transmembrane conductance regulator anion channel (CFTR). Specifically, PTH was able to induce phosphorylation of protein kinase A and phosphoinositide 3-kinase. Since the apical HCO3 - efflux through CFTR often required the intracellular H+/HCO3 - production and/or the Na+-dependent basolateral HCO3 - uptake, the intracellular pH (pHi) balance might be disturbed, especially as a consequence of increased endogenous H+ and HCO3 - production. However, measurement of pHi by a pH-sensitive dye suggested that the PTH-exposed Caco-2 cells were able to maintain normal pH despite robust HCO3 - transport. In addition, although the plasma membrane Na+/K+-ATPase (NKA) is normally essential for basolateral HCO3 - uptake and other transporters (e.g., NHE1), PTH did not induce insertion of new NKA molecules into the basolateral membrane as determined by membrane protein biotinylation technique. Thus, together with our previous data, we concluded that the PTH action on Caco-2 cells is dependent on PKA and PI3K with no detectable change in pHi or NKA abundance on cell membrane.

PMID:34189282 | PMC:PMC8220001 | DOI:10.1016/j.bbrep.2021.101054

Transl Pediatr. 2021 May;10(5):1486-1496. doi: 10.21037/tp-20-89.

ABSTRACT

The field of in utero gene therapy (IUGT) represents a crossroad of technologic advancements and medical ethical boundaries. Several strategies have been developed for IUGT focusing on either modifying endogenous genes, replacing missing genes, or modifying gene transcription products. The list of candidate diseases such as hemoglobinopathies, cystic fibrosis, lysosomal storage disorders continues to grow with new strategies being developed as our understanding of their respective underlying molecular pathogenesis increases. Treatment in utero has several distinct advantages to postnatal treatment. Biologic and physiologic phenomena enable the delivery of a higher effective dose, generation of immune tolerance, and the prevention of phenotypic onset for genetic diseases. Therapeutic technology for IUGT including CRISPR-Cas9 systems, zinc finger nucleases (ZFN), and peptide nucleic acids (PNAs) has already shown promise in animal models and early postnatal clinical trials. While the ability to detect fetal diagnoses has dramatically improved with developments in ultrasound and next-generation sequencing, treatment options remain experimental, with several translational gaps remaining prior to implementation in the clinical realm. Complicating this issue, the potential diseases targeted by this approach are often debilitating and would otherwise prove fatal if not treated in some manner. The leap from small animals to large animals, and subsequently, to humans will require further vigorous testing of safety and efficacy.

PMID:34189107 | PMC:PMC8192997 | DOI:10.21037/tp-20-89

Nat Commun. 2021 Jun 29;12(1):4024. doi: 10.1038/s41467-021-24337-9.

ABSTRACT

Pseudomonas aeruginosa can cause nosocomial infections, especially in ventilated or cystic fibrosis patients. Highly pathogenic isolates express the phospholipase ExoU, an effector of the type III secretion system that acts on plasma membrane lipids, causing membrane rupture and host cell necrosis. Here, we use a genome-wide screen to discover that ExoU requires DNAJC5, a host chaperone, for its necrotic activity. DNAJC5 is known to participate in an unconventional secretory pathway for misfolded proteins involving anterograde vesicular trafficking. We show that DNAJC5-deficient human cells, or Drosophila flies knocked-down for the DNAJC5 orthologue, are largely resistant to ExoU-dependent virulence. ExoU colocalizes with DNAJC5-positive vesicles in the host cytoplasm. DNAJC5 mutations preventing vesicle trafficking (previously identified in adult neuronal ceroid lipofuscinosis, a human congenital disease) inhibit ExoU-dependent cell lysis. Our results suggest that, once injected into the host cytoplasm, ExoU docks to DNAJC5-positive secretory vesicles to reach the plasma membrane, where it can exert its phospholipase activity.

PMID:34188051 | DOI:10.1038/s41467-021-24337-9

Infect Genet Evol. 2021 Jun 26:104982. doi: 10.1016/j.meegid.2021.104982. Online ahead of print.

ABSTRACT

Pseudomonas aeruginosa (P. aeruginosa) displays high drug resistance and biofilm-mediated adaptability, which makes its infections difficult to treat. Alternative intervention methods and targets have made such infections treatment manageable. One of the biofilm components, functional amyloids of Pseudomonas (Fap) is correlated positively with virulence and mucoidy phenotype found in infection in cystic fibrosis (CF) patients. Extracellular accessibility, conservation across P. aeruginosa isolates and linkage with lung infections phenotype in CF patients, makes Fap a promising intervention target. Furthermore, the reported effect of bacterial amyloid on neuronal function and immune response makes it a targetable candidate. In the current study, Fap C protein and its immediate interactions were explored to extract antigenic T-cell and B-cell epitopes. A combination of epitopes and peptide adjuvants has been linked to derive vaccine candidate structures. The vaccine candidates were validated for antigenicity, allergenicity, physiochemical properties, stability and interactions with TLRs and MHC alleles. Immunosimulation studies have demonstrated that vaccines elicit Th1 dominated response, which can assist in good prognosis of infection in CF patients.

PMID:34186254 | DOI:10.1016/j.meegid.2021.104982

Clin Microbiol Infect. 2021 Jun 26:S1198-743X(21)00341-4. doi: 10.1016/j.cmi.2021.06.021. Online ahead of print.

ABSTRACT

OBJECTIVES: To determine the presence and genotypic macrolide susceptibility of Mycoplasma amphoriforme, and presence of Ureaplasma spp., and Mycoplasma fermentans among clinical samples from England previously investigated for Mycoplasma pneumoniae.

METHODS: Quantitative and conventional PCR were used to retrospectively screen a collection of 160 clinical samples, previously submitted to Public Health England (PHE) for detection of M. pneumoniae, between October 2016 and December 2017. Samples which were positive for M. amphoriforme DNA were further investigated for mutations associated with genotypic macrolide resistance by sequencing of domain V of the 23s rRNA.

RESULTS: Mycoplasma amphoriforme were detected in 10/160 (6.3%) samples, Ureaplasma parvum were detected in 4/160 samples (2.5%) with 0/160 M. fermentans detections. Of the nine individuals (two samples were from the same patient) in which M. amphoriforme were detected, eight were male (10 - 60 years age range) and one was female (30 - 40 years age range). One individual, with cystic fibrosis was positive for both M. amphoriforme and U. parvum. All M. amphoriforme DNA were genotypically susceptible for macrolides.

CONCLUSIONS: Mycoplasma amphoriforme were found in clinical samples including the lower respiratory tract samples of patients with pneumonia. In the absence of other respiratory pathogens, these data suggest a potential role of this organism in human disease, with no evidence of acquired macrolide resistance. Ureaplasma parvum was detected in cerebrospinal fluid and respiratory tract samples. These data suggest the need to consider these atypical respiratory pathogens in future diagnostic investigations.

PMID:34186210 | DOI:10.1016/j.cmi.2021.06.021

Chest. 2021 Jun 26:S0012-3692(21)01267-8. doi: 10.1016/j.chest.2021.06.039. Online ahead of print.

NO ABSTRACT

PMID:34186037 | DOI:10.1016/j.chest.2021.06.039

Nurs Womens Health. 2021 Jun 26:S1751-4851(21)00122-7. doi: 10.1016/j.nwh.2021.05.006. Online ahead of print.

ABSTRACT

OBJECTIVE: To describe the development, evaluation, and psychometric properties of a new instrument that measures fertility preservation (FP) knowledge in women with cystic fibrosis (CF) titled the Knowledge of FP in Women With CF Instrument (KFP-WCFI).

DESIGN: The 10-item KFP-WCFI was developed and evaluated through a cross-sectional survey.

SETTING: Participants were recruited nationally from CF Foundation-accredited CF clinics and via snowball sampling.

PARTICIPANTS: Fifty women with CF ages 18 through 35 years completed the instrument.

MEASUREMENTS: Construct validity was assessed using confirmatory factor analysis (CFA). In the CFA, the model fit was evaluated using standardized root mean square residual, root mean square error of approximation, and comparative fit index. Cronbach's alpha was used to examine internal consistency reliability. The criterion validity was assessed using inferential statistics.

RESULTS: The CFA with two subscales, General Fertility Knowledge and Transplant-Related Fertility Knowledge, demonstrated good fit, with a standardized root mean square residual of 0.07, root mean square error of approximation of 0.06, and comparative fit index of 0.97, indicating good construct validity of the instrument. This instrument demonstrated internal consistency reliability with a Cronbach's alpha of .91 for the General Fertility Knowledge subscale and .64 for the Transplant-Related Fertility Knowledge subscale. Women who reported a pregnancy scored higher than women who did not report a pregnancy (p = .02), suggesting criterion validity.

CONCLUSION: The newly developed KFP-WCFI appears to be a valid and reliable instrument that can be used to measure self-assessed FP knowledge in women with CF.

PMID:34186019 | DOI:10.1016/j.nwh.2021.05.006

Diabetes Technol Ther. 2021 Jun 29. doi: 10.1089/dia.2021.0201. Online ahead of print.

ABSTRACT

Background Diabetes technologies are associated with improvements in glycemic control and health-related quality of life among people with type 1 diabetes (T1D). Use and perceptions of continuous glucose monitors (CGM) and insulin pumps within the CF community have not been well-studied. Methods A 30-item online survey was sent to a CF community group including people with CF (pwCF) and parents of children with CF (cwCF) addressing CFRD diagnosis, CGM and insulin pump use, and perceptions of diabetes technologies. Results The response rate was 11% (n=120; 83 pwCF, 35 cwCF). Sixty-one percent of pwCF and 34% of cwCF reported a diagnosis of CFRD. CGM use was reported by 75% (n=47) of respondents with CFRD but was discontinued by 19% (n=9), most commonly due to cost and increased worry about glycemia. Insulin pump therapy was reported by 29% (n=18 of 62) of respondents with CFRD and was discontinued by 28% (n=5), most commonly due to pain or skin irritation. Overall, 91% agreed or strongly agreed that CGM facilitated CFRD management. Eighty-one percent agreed with at least 5 of 7 positive statements about CGM as compared to 22% for insulin pumps. Potential embarrassment over device wear, concerns about cost, and pain were commonly held negative perceptions of both technologies. Conclusions As compared to T1D and despite perceived benefits, rates of sustained diabetes technology use are low in the CFRD community. Better insurance coverage to mitigate cost, better patient education, and confirmation that these technologies improve health and patient-reported outcomes may increase uptake.

PMID:34185606 | DOI:10.1089/dia.2021.0201