Self-reported alcohol use in the cystic fibrosis community.

J Cyst Fibros. 2019 Jul 11;:

Authors: Lowery EM, Afshar M, West N, Kovacs EJ, Smith B, Joyce C

Abstract
INTRODUCTION: Excessive alcohol use (EAU), a harmful pattern of drinking that includes binge drinking and heavy use, occurs in 25% (binge) and 6% (heavy use) of the US population, respectively. Little is known about alcohol use in individuals with cystic fibrosis (CF). The objective of this investigation is to examine alcohol consumption patterns in individuals with CF using a health survey administered from a social media platform.
METHODS: Individuals with CF, 18 years of age or older, were recruited for participation through social media and internet-based platforms.
RESULTS: 1135 individuals initially participated in the survey and 84% (n = 952) were eligible and completed the survey. Of the respondents, 77% (n = 729) currently consume alcohol, 18% (n = 171) formerly consumed alcohol, and 5% (n = 52) never consumed alcohol. Amongst the people with CF who currently consume alcohol, 54% (N = 391) met criteria for EAU. Thirty percent of current drinkers experienced symptoms of harmful alcohol use. Of those who met criteria for EAU, 7% wore oxygen, 6% had a lung transplant, 10% had liver disease and 32% had diabetes. Those with EAU reported more hospitalizations than those without EAU [244 (62%) vs 182 (54%), p = .034]. Characteristics associated with EAU after multivariable adjustment included younger age, unmarried status, male gender and younger age at initiation of drinking.
CONCLUSION: EAU is occurring at a much higher proportion in individuals with CF. A substantial percentage of CF individuals with EAU also have medical co-morbidities. Screening, brief intervention, and referral to treatment for EAU in CF clinics is warranted.

PMID: 31303381 [PubMed - as supplied by publisher]

Supersonic shear-wave elastography and APRI for the detection and staging of liver disease in pediatric cystic fibrosis.

J Cyst Fibros. 2019 Jul 11;:

Authors: Calvopina DA, Noble C, Weis A, Hartel GF, Ramm LE, Balouch F, Fernandez-Rojo MA, Coleman MA, Lewindon PJ, Ramm GA

Abstract
BACKGROUND: Current diagnostic methods for the diagnosis of Cystic fibrosis (CF)-associated liver disease (CFLD) are non-specific and assessment of disease progression is difficult prior to the advent of advanced disease with portal hypertension. This study investigated the potential of Supersonic shear-wave elastography (SSWE) to non-invasively detect CFLD and assess hepatic fibrosis severity in children with CF.
METHODS: 125 children were enrolled in this study including CFLD (n = 55), CF patients with no evidence of liver disease (CFnoLD = 41) and controls (n = 29). CFLD was diagnosed using clinical, biochemical and imaging best-practice guidelines. Advanced CFLD was established by the presence of portal hypertension and/or macronodular cirrhosis on ultrasound. Liver stiffness measurements (LSM) were acquired using SSWE and diagnostic performance for CFLD detection was evaluated alone or combined with aspartate aminotransferase-to-platelet ratio index (APRI).
RESULTS: LSM was significantly higher in CFLD (8.1 kPa, IQR = 6.7-11.9) versus CFnoLD (6.2 kPa, IQR = 5.6-7.0; P < 0.0001) and Controls (5.3 kPa, IQR = 4.9-5.8; P < 0.0001). LSM was also increased in CFnoLD versus Controls (P = 0.0192). Receiver Operating Characteristic (ROC) curve analysis demonstrated good diagnostic accuracy for LSM in detecting CFLD using a cut-off = 6.85 kPa with an AUC = 0.79 (Sensitivity = 75%, Specificity = 71%, P < 0.0001). APRI also discriminated CFLD (AUC = 0.74, P = 0.004). Classification and regression tree modelling combining LSM + APRI showed 14.8 times greater odds of accurately predicting CFLD (AUC = 0.84). The diagnostic accuracy of SSWE for discriminating advanced disease was excellent with a cut-off = 9.05 kPa (AUC = 0.95; P < 0.0001).
CONCLUSIONS: SSWE-determined LSM shows good diagnostic accuracy in detecting CFLD in children, which was improved when combined with APRI. SSWE alone discriminates advanced CFLD.

PMID: 31303380 [PubMed - as supplied by publisher]

When Is Forgetting Not Forgetting? A Discursive Analysis of Differences in Forgetting Talk Between Adults With Cystic Fibrosis With Different Levels of Adherence to Nebulizer Treatments.

Qual Health Res. 2019 Jul 13;:1049732319856580

Authors: Drabble SJ, O'Cathain A, Arden MA, Hutchings M, Beever D, Wildman M

Abstract
Forgetting is often cited as a reason why people struggle to adhere to treatments for chronic conditions. Interventions have tried to improve forgetting behavior using reminders. We used a discursive psychological approach to explore differences in how high and low adherers constructed forgetting their nebulizer treatments for cystic fibrosis. Interviews were conducted with 18 adults from a cystic fibrosis center in the United Kingdom. High adherers constructed forgetting treatments as occasional lapses in automaticity and temporary lapses in memory that they found easy to repair. Low adherers utilized forgetting to normalize more consistent nonadherence to treatments. However, it is important to contextualize forgetting as a discursive resource that helped these participants to negotiate moral discourses around adherence to treatment that reminder interventions cannot address; we therefore recommend a more behavioral, patient-focused, theory-driven approach to intervention development.

PMID: 31303116 [PubMed - as supplied by publisher]

Chronic rhinosinusitis in unified airway disease: surfactant proteins as mediators of respiratory immunity.

Swiss Med Wkly. 2019 Jul 01;149:w20104

Authors: Noutsios GT, Sharma S

Abstract
PURPOSE OF REVIEW: The aim of this review is to describe the co-occurrence of chronic rhinosinusitis (CRS) with other inflammatory illnesses of the lower respiratory system characterised by airway obstruction and hyperresponsiveness, such as asthma, cystic fibrosis (CF), and chronic obstructive pulmonary disease (COPD) in the context of the unified airway disease (UAD). We also sought to discuss the novel role of surfactant proteins as mediators of innate immunity in the sinonasal epithelium and their potential as therapeutic interventions.
RECENT FINDINGS: Different epidemiological and physiological studies in CRS and asthma have outlined that there are common clustering patterns in the phenotypes/endotypes of both diseases, reinforcing the notion of the UAD. Also, surfactant proteins A (SP-A) and SP-D have now emerged as novel innate immunity molecules in bacterial sinusitis and allergic fungal sinusitis patients, respectively.
SUMMARY: CRS and asthma coexist and are interconnected. Therefore, management of CRS and asthma must be jointly carried out as one functional entity. SP-A and SP-D bridge the innate and adaptive immunity mechanisms of the sinonasal epithelium to bring together a well-orchestrated mechanism that effectively fights pathogens. The use of SP-A to ameliorate the innate immune responses in CRS is a new concept and is likely to lead to new horizons in CRS therapeutic regimens. &nbsp.

PMID: 31302901 [PubMed - in process]

First clinical trials of novel ENaC targeting therapy, SPX-101, in healthy volunteers and adults with cystic fibrosis.

Pulm Pharmacol Ther. 2019 Jul 11;:101819

Authors: Couroux P, Farias P, Rizvi L, Griffin K, Hudson C, Crowder T, Tarran R, Tullis E

Abstract
BACKGROUND: ENaC inhibition has been investigated as a CF treatment; however, small molecule inhibitors of ENaC lack efficacy and/or have shown dose-limiting hyperkalemia. SPX-101 is a novel, investigational small peptide (SPLUNC1 mimetic) that regulates ENaC density with the potential for efficacy without systemic effects.
METHODS: Two trials are presented: The first was a Phase 1, 2-part, randomized, double-blind, placebo-controlled, ascending-dose study of nebulized SPX-101 in healthy adults. Part 1 evaluated 4 single doses of SPX-101 ranging from 20 to 240 mg. Part 2 evaluated a 14-day regimen of SPX-101 at 4 doses of SPX-101 ranging from 10 to 120 mg BID. Pharmacokinetics, adverse events, spirometry, vital signs, electrocardiograms, pulse oximetry, and clinical laboratory values were assessed. The second trial was a tolerability-confirming, Phase 1b, open-label study conducted in 5 adult subjects with CF. Ascending doses of SPX-101 inhalation solution (10 mg-120 mg BID) were administered for 7 days. Safety was assessed as described above.
RESULTS: All 64 healthy volunteers (32 in each Part) completed the single and multiple dose study. SPX-101 was well tolerated with little/no systemic exposure and with no hyperkalemia. Adverse events were generally mild with reported respiratory events associated with the purported pharmacological activity of SPX-101. Tolerability of SPX-101 was similarly observed in adults with CF; all 5 subjects treated with SPX-101 completed the study.
CONCLUSIONS: SPX-101 was well-tolerated across a range of doses and had little/no systemic exposure in healthy adults and adults with CF, thus supporting further study in patients with CF. CLINICALTRIAL.
GOV REGISTRATION: NCT03056989.

PMID: 31302339 [PubMed - as supplied by publisher]

Lower airway microbiota and mycobiota in children with severe asthma.

J Allergy Clin Immunol. 2018 02;141(2):808-811.e7

Authors: Goldman DL, Chen Z, Shankar V, Tyberg M, Vicencio A, Burk R

PMID: 29031597 [PubMed - indexed for MEDLINE]

Motion robust high resolution 3D free-breathing pulmonary MRI using dynamic 3D image self-navigator.

Magn Reson Med. 2018 06;79(6):2954-2967

Authors: Jiang W, Ong F, Johnson KM, Nagle SK, Hope TA, Lustig M, Larson PEZ

Abstract
PURPOSE: To achieve motion robust high resolution 3D free-breathing pulmonary MRI utilizing a novel dynamic 3D image navigator derived directly from imaging data.
METHODS: Five-minute free-breathing scans were acquired with a 3D ultrashort echo time (UTE) sequence with 1.25 mm isotropic resolution. From this data, dynamic 3D self-navigating images were reconstructed under locally low rank (LLR) constraints and used for motion compensation with one of two methods: a soft-gating technique to penalize the respiratory motion induced data inconsistency, and a respiratory motion-resolved technique to provide images of all respiratory motion states.
RESULTS: Respiratory motion estimation derived from the proposed dynamic 3D self-navigator of 7.5 mm isotropic reconstruction resolution and a temporal resolution of 300 ms was successful for estimating complex respiratory motion patterns. This estimation improved image quality compared to respiratory belt and DC-based navigators. Respiratory motion compensation with soft-gating and respiratory motion-resolved techniques provided good image quality from highly undersampled data in volunteers and clinical patients.
CONCLUSION: An optimized 3D UTE sequence combined with the proposed reconstruction methods can provide high-resolution motion robust pulmonary MRI. Feasibility was shown in patients who had irregular breathing patterns in which our approach could depict clinically relevant pulmonary pathologies. Magn Reson Med 79:2954-2967, 2018. © 2017 International Society for Magnetic Resonance in Medicine.

PMID: 29023975 [PubMed - indexed for MEDLINE]

Health-related Quality of Life of Macedonian Families Experiencing Cystic Fibrosis in Pediatric Practice.

Folia Med (Plovdiv). 2019 Jun 01;61(2):213-222

Authors: Nakov ZN, Fushtikj SN, Tonikj-Ribarska J, Jolevska ST

Abstract
BACKGROUND: Health-related quality of life (HRQoL) is a parameter that is examined in the area of clinical effectiveness. Like other chronic health conditions, paediatric cystic fibrosis (CF) impacts not only children but also their families.
AIM: The present study investigates for the first time the HRQoL of children and parents in the Republic of North Macedonia.
MATERIALS AND METHODS: The survey included 22 children (6 to 13 years of age) and their parents and 7 parents of children under 6 years of age by using the CFQ Revised and questions for current medical treatment.
RESULTS: Children (6-13 years) reported the highest score for the digestive condition (84.85), while the lowest score was given for social activity (59.74). The highest score for digestive condition was also obtained from the parents of children from 6-13 years and under age of 6. The parents of children (6-13 years) reported the lowest score (60.56) for treatment burden activity, while the lowest score (50.0) for eating condition was obtained from the parents of children under 6 years.
CONCLUSION: Nationality and gender have no significant impact on the HRQoL parameters. The highest scores for the digestive condition, respiratory function and physical condition are in a positive correlation with the fact that enzyme, antibiotic and physical therapy are given as a standard medical care. The lowest scores of the social aspect of the CF patients indicate the need for including a psychological support and support of social workers as a part of the standard medical care of these patients.

PMID: 31301654 [PubMed - in process]

Beyond cystic fibrosis transmembrane conductance regulator therapy: a perspective on gene therapy and small molecule treatment for cystic fibrosis.

Gene Ther. 2019 Jul 12;:

Authors: Schneider-Futschik EK

Abstract
Cystic fibrosis (CF) is a life-limiting disease caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) activity. The recent advent of the FDA-approved CFTR modulator drug ivacaftor, alone or in combination with lumacaftor or tezacaftor, has enabled treatment of the majority of patients suffering from CF. Even before the identification of the CFTR gene, gene therapy was put forward as a viable treatment option for this genetic condition. However, initial enthusiasm has been hampered as CFTR gene delivery to the lungs has proven to be more challenging than expected. This review covers the contemporary clinical and scientific knowledge base for small molecule CFTR modulator drug therapy, gene delivery vectors and CRISPR/Cas9 gene editing and highlights the prospect of these technologies for future treatment options.

PMID: 31300729 [PubMed - as supplied by publisher]

A cystic fibrosis child with lung function decline.

J Cyst Fibros. 2019 Jul 09;:

Authors: Fiscarelli EV, Ricciotti G, Rossitto M, Pompilio A, Tuccio Guarna Assanti V, Lucidi V

Abstract
Respiratory infections are a major threat to cystic fibrosis patients. Besides bacteria, many fungi colonize the cystic fibrosis respiratory tract where an important fungal biota has been described. We report here the case of a 7-year-old cystic fibrosis child with pulmonary exacerbation and Arthrographis kalrae isolated from bronchoalveolar lavage fluid. To the best of our knowledge, this is the first reported case of lung infection due to Arhtrographis kalrae in a cystic fibrosis pediatric patient.

PMID: 31300282 [PubMed - as supplied by publisher]

International prevalence and risk factors evaluation for Drug-Resistant Streptococcus pneumoniae pneumonia.

J Infect. 2019 Jul 09;:

Authors: Aliberti S, Cook GS, Babu BL, Reyes LF, Rodriguez A, Sanz F, Soni NJ, Anzueto A, Faverio P, Sadud RF, Muhammad I, Prat C, Vendrell E, Neves J, Kaimakamis E, Feneley A, Swarnakar R, Franzetti F, Carugati M, Morosi M, Monge E, Restrepo MI, GLIMP investigators

Abstract
OBJECTIVE: Streptococcus pneumoniae is the most frequent bacterial pathogen isolated in subjects with Community-acquired pneumonia (CAP) worldwide. Limited data are available regarding the current global burden and risk factors associated with drug-resistant Streptococcus pneumoniae (DRSP) in CAP subjects. We assessed the multinational prevalence and risk factors for DRSP-CAP in a multinational point-prevalence study.
DESIGN: The prevalence of DRSP-CAP was assessed by identification of DRSP in blood or respiratory samples among adults hospitalized with CAP in 54 countries. Prevalence and risk factors were compared among subjects that had microbiological testing and antibiotic susceptibility data. Multivariate logistic regressions were used to identify risk factors independently associated with DRSP-CAP.
RESULTS: 3,193 subjects were included in the study. The global prevalence of DRSP-CAP was 1.3% and continental prevalence rates were 7.0% in Africa, 1.2% in Asia, and 1.0% in South America, Europe, and North America, respectively. Macrolide resistance was most frequently identified in subjects with DRSP-CAP (0.6%) followed by penicillin resistance (0.5%). Subjects in Africa were more likely to have DRSP-CAP (OR: 7.6; 95%CI: 3.34-15.35, p<0.001) when compared to centres representing other continents. .
CONCLUSIONS: This multinational point-prevalence study found a low global prevalence of DRSP-CAP that may impact guideline development and antimicrobial policies.

PMID: 31299410 [PubMed - as supplied by publisher]

F508del disturbs the dynamics of the nucleotide binding domains of CFTR before and after ATP hydrolysis.

Proteins. 2019 Jul 12;:

Authors: Abreu B, Lopes EF, F Oliveira AS, Soares CM

Abstract
The cystic fibrosis transmembrane conductance regulator (CFTR) channel is an ion channel responsible for chloride transport in epithelia and it belongs to the class of ABC transporters. The deletion of phenylalanine 508 (F508del) in CFTR is the most common mutation responsible for cystic fibrosis. Little is known about the effect of the mutation in the isolated nucleotide binding domains (NBDs), on dimer dynamics, ATP hydrolysis and even on nucleotide binding. Using molecular dynamics simulations of the human CFTR NBD dimer, we showed that F508del increases, in the pre-hydrolysis state, the inter-motif distance in both ATP binding sites (ABP) when ATP is bound. Additionally, a decrease in the number of catalytically competent conformations was observed in the presence of F508del. We used the subtraction technique to study the first 300ps after ATP hydrolysis in the catalytic competent site and found that the F508del dimer evidences lower conformational changes than the wild type. Using longer simulation times, the magnitude of the conformational changes in both forms increases. Nonetheless, the F508del dimer shows lower C-α RMS values in comparison to the wild-type, on the F508del loop, on the residues surrounding the catalytic site and the portion of NBD2 adjacent to ABP1. These results provide evidence that F508del interferes with the NBD dynamics before and after ATP hydrolysis. These findings shed a new light on the effect of F508del on NBD dynamics and reveal a novel mechanism for the influence of F508del on CFTR. This article is protected by copyright. All rights reserved.

PMID: 31298435 [PubMed - as supplied by publisher]

The effect of CFTR modulators on a cystic fibrosis patient presenting with recurrent pancreatitis in the absence of respiratory symptoms: a case report.

BMC Gastroenterol. 2019 Jul 11;19(1):123

Authors: Johns JD, Rowe SM

Abstract
BACKGROUND: Cystic fibrosis (CF) is a genetic disorder of the epithelial CFTR apical chloride channel resulting in multi-organ manifestations, including pancreatic exocrine secretion. In the pancreas, CFTR abnormality results in abnormally viscous secretions that obstruct proximal ducts leading to fibrotic injury and ultimately pancreatic insufficiency in 85% of the CF population. CFTR modulators, including the potentiator ivacaftor, augment channel gating to restore 30-50% of CFTR-mediated anion transport. While CFTR modulation has been shown to alkalinize the pH of the alimentary tract and potentially augment pancreatic enzyme activity, the effect of ivacaftor on recurrent pancreatitis is emerging. Here we describe a case of a patient with CF (R117H/7 T/F508del) who presented with recurrent pancreatitis who was effectively treated with ivacaftor in the absence of respiratory symptoms.
CASE PRESENTATION: A 24-year-old white male with past medical history of recurrent acute pancreatitis presented for evaluation following a referral from an outside hospital. The patient reported a lifetime of gastrointestinal symptoms requiring over 20 hospitalizations for pancreatitis in the last 10 years. Prior U/S and CT imaging for pancreatitis ruled out gallstones or anatomical etiologies. Family history included a brother with CF carrier status who suffered from recurrent acute pancreatitis. Sweat chloride testing was suggestive of CFTR dysfunction (57 mmol/L). Genetic testing demonstrated disease causing CFTR mutations: R1117H/7 T/F508del. Patient was prescribed pancrelipase, however, he reported worsened gas and diarrhea symptoms. Pancrelipase was discontinued and the patient was prescribed ivacaftor 150 mg BID. After 6 weeks of ivacaftor treatment, patient reported improved gastrointestinal symptoms. For an additional 19 months, patient reported no episodes of pancreatitis until he discontinued ivacaftor. Over the next 3 weeks, patient experienced progressive nausea and sharp epigastric pain and laboratory studies confirmed pancreatitis. Patient was subsequently lost to follow up.
CONCLUSIONS: These findings support a possible relationship between the use of CFTR modulators, such as ivacaftor, in the management of recurrent pancreatitis in the setting of patients with cystic fibrosis and a CFTR mutation with residual CFTR activity or otherwise known to be responsive in vitro. Ivacaftor may be useful for recurrent pancreatitis, even in the absence of respiratory morbidity.

PMID: 31296159 [PubMed - in process]

Comparable Bioavailability and Disposition of Pefloxacin in Patients with Cystic Fibrosis and Healthy Volunteers Assessed via Population Pharmacokinetics.

Pharmaceutics. 2019 Jul 10;11(7):

Authors: Bulitta JB, Jiao Y, Landersdorfer CB, Sutaria DS, Tao X, Shin E, Höhl R, Holzgrabe U, Stephan U, Sörgel F

Abstract
Quinolone antibiotics present an attractive oral treatment option in patients with cystic fibrosis (CF). Prior studies have reported comparable clearances and volumes of distribution in patients with CF and healthy volunteers for primarily renally cleared quinolones. We aimed to provide the first pharmacokinetic comparison for pefloxacin as a predominantly nonrenally cleared quinolone and its two metabolites between both subject groups. Eight patients with CF (fat-free mass [FFM]: 36.3 ± 6.9 kg, average ± SD) and ten healthy volunteers (FFM: 51.7 ± 9.9 kg) received 400 mg pefloxacin as a 30 min intravenous infusion and orally in a randomized, two-way crossover study. All plasma and urine data were simultaneously modelled. Bioavailability was complete in both subject groups. Pefloxacin excretion into urine was approximately 74% higher in patients with CF compared to that in healthy volunteers, whereas the urinary excretion of metabolites was only slightly higher in patients with CF. After accounting for body size and composition via allometric scaling by FFM, pharmacokinetic parameter estimates in patients with CF divided by those in healthy volunteers were 0.912 for total clearance, 0.861 for nonrenal clearance, 1.53 for renal clearance, and 0.916 for volume of distribution. Nonrenal clearance accounted for approximately 90% of total pefloxacin clearance. Overall, bioavailability and disposition were comparable between both subject groups.

PMID: 31295857 [PubMed]

Role of Dietary Supplements in the Management of Parkinson's Disease.

Biomolecules. 2019 Jul 10;9(7):

Authors: Ciulla M, Marinelli L, Cacciatore I, Stefano AD

Abstract
The use of food supplements or functional food has significantly increased in the past decades, especially to compensate both the modern lifestyle and the food shortages of the industrialized countries. Despite food supplements are habitually intended to correct nutritional deficiencies or to support specific physiological functions, they are often combined with common drug therapies to improve the patient's health and/or mitigate the symptoms of many chronic diseases such as cardiovascular diseases, cystic fibrosis, cancer, liver and gastrointestinal diseases. In recent years, increased attentions are given to the patient's diet, and the use of food supplements and functional food rich in vitamins and antioxidants plays a very important role in the treatment and prevention of neurodegenerative diseases such as Parkinson's disease (PD). Natural compounds, phytochemicals, vitamins, and minerals can prevent, delay, or alleviate the clinical symptoms of PD in contrast to some of the main physiopathological mechanisms involved in the development of the disease, like oxidative stress, free radical formation, and neuroinflammation. The purpose of this review is to collect scientific evidences which support the use of specific biomolecules and biogenic elements commonly found in food supplements or functional food to improve the clinical framework of patients with PD.

PMID: 31295842 [PubMed - in process]

Editorial.

Curr Opin Pharmacol. 2018 12;43:151

Authors: Trist D, Williams M

PMID: 30502021 [PubMed - indexed for MEDLINE]

Integrated Approaches to Analyze Big Data in the Perinatal/Neonatal Space.

Breastfeed Med. 2018 04;13(S1):S5-S6

Authors: Knight R

PMID: 29624426 [PubMed - indexed for MEDLINE]

Breast Milk Fats and Lipids: Expanding Benefits to Fragile Infant Populations.

Breastfeed Med. 2018 04;13(S1):S26-S27

Authors: Freedman SD

PMID: 29624422 [PubMed - indexed for MEDLINE]

Vitamin K and cystic fibrosis: A gordian knot that deserves our attention.

Respir Med. 2019 Jul 06;155:36-42

Authors: Hatziparasides G, Loukou I, Moustaki M, Douros K

Abstract
Cystic fibrosis (CF) is an inherited genetic disorder with multiorgan involvement. Gastrointestinal tract dysfunction leads to fat and fat-soluble vitamins (A,D,E,K) malabsorption and deficiency of these vitamins. Subclinical vitamin K (VK) deficiency seems to be a common problem in CF patients. However, despite the rest of fat-soluble vitamins being routinely supplemented, this is not a universal clinical practice for VK. Inefficient levels of VK may have significant effects on blood coagulation and bone formation. There are also some data indicating that VK may play a key role on regulation of inflammation. Supplementing CF patients with VK seems rational, but the appropriate dosing regimens are still a matter of debate. This review will try to delineate the problem and communicate the latest opinions on this controversial issue.

PMID: 31295676 [PubMed - as supplied by publisher]

Impact of pharmacy services on cystic fibrosis transmembrane conductance regulator modulator prescribing at a pediatric cystic fibrosis center.

Pediatr Pulmonol. 2019 Jul 11;:

Authors: Wright BA, Singh SB, Schultz JL, Ramsey LJ, Spading KA, Mascardo LA, Starner TD

Abstract
BACKGROUND: This study was undertaken to determine if the presence of a clinical pharmacy team impacted patients' access to cystic fibrosis transmembrane conductance regulator (CFTR) modulators.
METHODS: A retrospective chart review of electronic medical records from the University of Iowa Hospitals and Clinics (UIHC) was conducted. Data were collected regarding the timing of prior authorization (PA) submissions and approvals from 2012 to 2018. The Wilcoxon rank-sum test was used to compare the meantime (days) between prescription and PA submission dates, and PA submission and approval date for all patients included in the analysis. Comparisons were made for pre- and postpharmacy services eras as well as the UIHC Specialty Pharmacy versus a non-UIHC Specialty Pharmacy.
RESULTS: Sixty-three patients were included in the final analysis. The average time between prescription date and PA submission was 12.5 days (standard deviation [SD] = 17.4 days) in the preclinical pharmacy services era and 3.5 days (SD = 5.8 days; P = .028) in the postclinical pharmacy services era. The average time to PA submission significantly decreased from 9.8 days (SD = 13.1 days) to 1.3 days (SD = 4.2 days; P < .0001) when prescriptions were filled by the UIHC Specialty Pharmacy vs a non-UIHC Specialty Pharmacy.
CONCLUSIONS: There was a significant benefit to CFTR modulator prescribing when clinical pharmacy services were incorporated in our cystic fibrosis (CF) care team, which will become increasingly important with the anticipation of new CF medications in the near future.

PMID: 31294925 [PubMed - as supplied by publisher]