Management Of Cystic Fibrosis Patients Research Articles

Effect of Curcumin on Experimental Models of Cystic Fibrosis: A Systematic Review

Context: Cystic fibrosis (CF) is a genetic disorder characterized by CF transmembrane conductance regulator (CFTR) gene defects and chronic inflammation. Management of CF patients is challenging, and no definitive treatment is available. Although evidence suggests that curcumin is effective against inflammation and can regulate ion channels, its effect on CF patients is not well established. Objectives: This systematic review evaluated the available evidence for the application of curcumin in experimental models of CF. Evidence Acquisition: The data in this study were selected from articles published in PubMed, ScienceDirect, ProQuest, and Springer until July 2021. Articles on the use of curcumin in CF patients were included based on the purpose of the study. In this review, 11 articles were found in the final search. Then, a quality assessment was performed using SYRCLE’s RoB tool. Results: Eleven out of 2188 screened studies were eligible for this systematic review. Most in vitro studies (7 out of 8) reported that curcumin influences channel function through a variety of mechanisms, while only 1 study provided comparable results. Regarding anti-inflammatory function, 1 study showed that curcumin could suppress the toll-like receptor 2 (TLR2) gene and protein expression in the CF bronchial epithelial cell line. Two of 3 in vivo studies reported channel correction with curcumin. Conclusions: The available evidence in this systematic study demonstrates the usefulness of curcumin via different mechanisms among different study models. Given that few studies have been conducted in this field, this systematic review suggests more investigations be undertaken to facilitate generalization to human studies.

Prevalence and Impact of Rheumatologic Pain in Cystic Fibrosis Adult Patients.

BackgroundWith the improvement of cystic fibrosis (CF) patient survival, the prevalence of long-term complications increased, among them rheumatologic disorders.MethodsThe aim of this prospective study was to evaluate the prevalence of spinal and joint pain, and their impact on disability, anxiety, depression, and quality of life in CF adult patients.ResultsForty-seven patients were analyzed, 72% of men, mean aged 28 years, with a mean body mass index of 22 kg/m2 and a mean FEV1% of 63%. Twenty-two patients (47%) described rheumatologic pain either spinal (n = 15, 32%) and/or joint pain (n = 14, 30%). Patients with spinal and/or joint pain were shorter (p = 0.023), more frequently colonized with Staphylococcus aureus (p < 0.008), had more frequent ΔF508 homozygous mutations (p = 0.014), and a trend for more impairment of the 6-min walking distance (p = 0.050). The presence of rheumatologic pain tended to be associated with disability according to the Health Assessment Questionnaire (HAQ) and anxiety. Compared with patients with no pain patients with both spinal and joint pain exhibited a more pronounced impact on the St George's Respiratory Questionnaire (SGRQ).ConclusionRheumatologic pain is frequent in CF adult patients, and may affect daily living, anxiety and quality of life. Systematic assessment of rheumatologic pain should be included in the management of CF patients.

Alkyl-Quinolones derivatives as potential biomarkers for Pseudomonas aeruginosa infection chronicity in Cystic Fibrosis

In Cystic Fibrosis (CF), a rapid and standardized definition of chronic infection would allow a better management of Pseudomonas aeruginosa (Pa) infections, as well as a quick grouping of patients during clinical trials allowing better comparisons between studies. With this purpose, we compared the metabolic profiles of 44 in vitro cultures of Pa strains isolated from CF patients at different stages of infection in order to identify metabolites differentially synthetized according to these clinical stages. Compounds produced and secreted by each strain in the supernatant of a liquid culture were analysed by metabolomic approaches (UHPLC-DAD-ESI/QTOF, UV and UPLC-Orbitrap, MS). Multivariate analyses showed that first colonization strains could be differentiated from chronic colonization ones, by producing notably more Alkyl-Quinolones (AQs) derivatives. Especially, five AQs were discriminant: HQC5, HQNOC7, HQNOC7:1, db-PQS C9 and HQNOC9:1. However, the production of HHQ was equivalent between strain types. The HHQ/HQNOC9:1 ratio was then found to be significantly different between chronic and primo-colonising strains by using both UV (p = 0.003) and HRMS data (p = 1.5 × 10–5). Our study suggests that some AQ derivatives can be used as biomarkers for an improved management of CF patients as well as a better definition of the clinical stages of Pa infection.

Clinical Considerations for Routine Auditory and Vestibular Monitoring in Patients With Cystic Fibrosis.

Purpose Specific classes of antibiotics, such as aminoglycosides, have well-established adverse events producing permanent hearing loss, tinnitus, and balance and/or vestibular problems (i.e., ototoxicity). Although these antibiotics are frequently used to treat pseudomonas and other bacterial infections in patients with cystic fibrosis (CF), there are no formalized recommendations describing approaches to implementation of guideline adherent ototoxicity monitoring as part of CF clinical care. Method This consensus statement was developed by the International Ototoxicity Management Working Group (IOMG) Ad Hoc Committee on Aminoglycoside Antibiotics to address the clinical need for ototoxicity management in CF patients treated with known ototoxic medications. These clinical protocol considerations were created using consensus opinion from a community of international experts and available evidence specific to patients with CF, as well as published national and international guidelines on ototoxicity monitoring. Results The IOMG advocates four clinical recommendations for implementing routine and guideline adherent ototoxicity management in patients with CF. These are (a) including questions about hearing, tinnitus, and balance/vestibular problems as part of the routine CF case history for all patients; (b) utilizing timely point-of-care measures; (c) establishing a baseline and conducting posttreatment evaluations for each course of intravenous ototoxic drug treatment; and (d) repeating annual hearing and vestibular evaluations for all patients with a history of ototoxic antibiotic exposure. Conclusion Increased efforts for implementation of an ototoxicity management program in the CF care team model will improve identification of ototoxicity signs and symptoms, allow for timely therapeutic follow-up, and provide the clinician and patient an opportunity to make an informed decision about potential treatment modifications to minimize adverse events. Supplemental Material https://doi.org/10.23641/asha.16624366.

Serum-Based Proteomics Profiling in Adult Patients with Cystic Fibrosis

Cystic fibrosis (CF), the most common lethal autosomal recessive disorder among Caucasians, is caused by mutations in the CF transmembrane conductance regulator (CFTR) chloride channel gene. Despite significant advances in the management of CF patients, novel disease-related biomarkers and therapies must be identified. We performed serum proteomics profiling in CF patients (n = 28) and healthy subjects (n = 10) using the 2D-DIGE MALDI-TOF proteomic approach. Out of a total of 198 proteins identified, 134 showed a statistically significant difference in abundance and a 1.5-fold change (ANOVA, p < 0.05), including 80 proteins with increased abundance and 54 proteins with decreased abundance in CF patients. A multiple reaction monitoring-mass spectrometry analysis of six differentially expressed proteins identified by a proteomic approach (DIGE-MALD-MS) showed a significant increase in C3 and CP proteins and a decrease in APOA1, Complement C1, Hp, and RBP4proteins compared with healthy controls. Fifteen proteins were identified as potential biomarkers for CF diagnosis. An ingenuity pathway analysis of the differentially regulated proteins indicates that the central nodes dysregulated in CF subjects involve pro-inflammatory cytokines, ERK1/2, and P38 MAPK, which are primarily involved in catalytic activities and metabolic processes. The involved canonical pathways include those related to FXR/RXR, LXR/RXR, acute phase response, IL12, nitric oxide, and reactive oxygen species in macrophages. Our data support the current efforts toward augmenting protease inhibitors in patients with CF. Perturbations in lipid and vitamin metabolism frequently observed in CF patients may be partly due to abnormalities in their transport mechanism.

S0112 Evaluating the Outcomes of Endoscopic Retrograde Cholangiopancreatography in Patients With Cystic Fibrosis

INTRODUCTION: There is a known association of biliary stones with cystic fibrosis (CF). The CFTR expression in the gallbladder is described as one of the highest of all human tissues. Consequently, CFTR activity is imperative in the biliary tree and its absence can lead to significant biliary disease due to decreased secretion of biliary bicarbonate and disturbance of normal bile acid circulation. ERCP has an important role in the diagnosis and management of CF patients. However, there is very limited data evaluating the outcomes and the adverse events (AEs) post ERCP in these patients. Therefore, we conducted this study to evaluate ERCP-related AEs in CF patients using a large national cohort. METHODS: A retrospective cohort study was performed using the National Inpatient Sample (NIS) database 2011–2014. We identified all adult patients (≥18 year old) who underwent ERCP using ICD-9 codes. Primary outcomes comprised ERCP-related AEs including post ERCP pancreatitis (PEP), bleeding and perforation. Secondary outcomes included all-cause mortality (ACM) and length of hospital stay (LOS). Primary and secondary outcomes were compared between the study and control group using multivariate logistic regression analysis. RESULTS: We identified a total of 108,182 patients underwent ERCP, of which 73 (0.07%) had CF. CF patients were younger (31.75 ± 14.5 vs 58.48 ± 20.14), less females (49.7% vs 59.7%) and less likely to be African American (4.3% vs 9.1%) compared to the control group, (P < 0.05 for all. In addition, CF patients had less alcohol (1.4% vs 4.3%, P > 0.05) compared to the control group. Using multivariate logistic regression and after adjusting for potential cofounding factors including age, race, gender and Elixhauser comorbidities, patients with CF had no statistically significant difference in PEP (OR 0.99, 95% CI: 0.31–3.23) or bleeding (OR 3.22, 95% CI: 0.44–23.63). In addition, the control group had 0.04% (n = 48) total cases of post ERCP perforation reported and 1% (n = 1093) of ACM while none were reported in CF patients. Further, the adjusted LOS was slightly longer in the CF group compared to the control group (8.33 ± 6.37 vs 5.49 ± 5.04 day, P < 0.05). CONCLUSION: CF patients undergoing ERCP might have slightly increase in the LOS, however they have comparable outcomes and similar risk of AEs comparing to other patients who don’t carry this diagnosis.

Cystic Fibrosis Mutation Spectrum in North Macedonia: A Step Toward Personalized Therapy.

The most prevalent "rare" disease worldwide, cystic fibrosis (CF), is an autosomal recessive multisystem disease, caused by mutations in the CFTR gene. The knowledge of CFTR mutations present in certain population is important for designing a simple, fast and cost-effective genetic testing approach, also for better management of CF patients, including the administration of novel targeted therapies. Here, we present genetic results of 158 unrelated CF patients from the National CF Registry of the Republic of North Macedonia. Initially, patients were screened for the 11 most common CF mutations. Additional CF mutations and large deletions/duplications in the CFTR gene were analyzed using commercial kits. If the genotype was undetermined, all CFTR exons were analyzed using Sanger DNA sequencing or next generation sequencing (NGS) (since 2014). The most common CF mutation, c.l521_ 1523del (legacy name F508del), was found with an overall incidence of 75.9%. Additionally, 26 other pathogenic variants and three large deletions were identified in the CFTR gene as a genetic cause of CF. Two of these, c.1070 C>T (p.Ala357Val) and c.2779_2788dup CTTGCTATGG (p.Gly930AlafsTer48), were novel. According to the distribution and prevalence of the pathogenic variants detected in our patients, a fast and cost-effective method, based on a single base extension was designed as a first-line CF genetic test with a 90.0% detection rate within our population. Furthermore, the knowledge of CFTR mutation classes in our CF patients represents the first step toward personalized therapy for CF in our country.

Oral glucose tolerance test and continuous glucose monitoring to assess diabetes development in cystic fibrosis patients

IntroductionPatients with cystic fibrosis (CF) undergo a slow and progressive process toward diabetes. Oral glucose tolerance test (OGTT) is recommended to diagnose impaired glucose levels in these patients. Continuous glucose monitoring (CGM) measures glucose profiles under real-life conditions. ObjectiveTo compare OGTT and CGM results in CF patients. MethodsPaired OGTT and 6-day CGM profiles (146.2±9.1h/patient) were performed in 30 CF patients aged 10–18 years. ResultsAccording to OGTT, 14 patients had normal glucose tolerance (NGT), 14 abnormal glucose tolerance (AGT), and two cystic fibrosis-related diabetes (CFRD). In 27 patients (13 NGT, 13 AGT, 1 CFRD), CGM showed glucose values ranging from 140 to 200mg/dL during similar monitoring times (2%–14% with NGT, 1%–16.9% with AGT, and 3% with CFRD). Glucose peak levels ≥200mg/dL were seen in seven patients (3 NGT, 3 AGT, 1 CFRD).According to CGM, two patients had all glucose values under 140mg/dL (1 NGT, 1 AGT). Seventeen patients had glucose levels ranging from 140 to 200mg/dL (10 NGT, 6 AGT, 1 CFRD). Ten patients (3 NGT, 7 AGT) had glucose values ≥200mg/dL for ≤1% of the monitoring time and one (CFRD) for >1% of the monitoring time. ConclusionsOGTT results did not agree with those of the CGM. CGM allows for diagnosis of glucose changes not detected by OGTT. Such changes may contribute to optimize pre-diabetes management in CF patients.

Oral glucose tolerance test and continuous glucose monitoring to assess diabetes development in cystic fibrosis patients

IntroductionPatients with cystic fibrosis (CF) undergo a slow and progressive process toward diabetes. Oral glucose tolerance test (OGTT) is recommended to diagnose impaired glucose levels in these patients. Continuous glucose monitoring (CGM) measures glucose profiles under real-life conditions. ObjectiveTo compare OGTT and CGM results in CF patients. MethodsPaired OGTT and 6-day CGM profiles (146.2±9.1h/patient) were performed in 30 CF patients aged 10–18 years. ResultsAccording to OGTT, 14 patients had normal glucose tolerance (NGT), 14 abnormal glucose tolerance (AGT), and two cystic fibrosis-related diabetes (CFRD). In 27 patients (13 NGT, 13 AGT, 1 CFRD), CGM showed glucose values ranging from 140 to 200mg/dL during similar monitoring times (2%–14% with NGT, 1%–16.9% with AGT, and 3% with CFRD). Glucose peak levels ≥200mg/dL were seen in seven patients (3 NGT, 3 AGT, 1 CFRD).According to CGM, two patients had all glucose values under 140mg/dL (1 NGT, 1 AGT). Seventeen patients had glucose levels ranging from 140 to 200mg/dL (10 NGT, 6 AGT, 1 CFRD). Ten patients (3 NGT, 7 AGT) had glucose values ≥200mg/dL for ≤1% of the monitoring time and one (CFRD) for >1% of the monitoring time. ConclusionsOGTT results did not agree with those of the CGM. CGM allows for diagnosis of glucose changes not detected by OGTT. Such changes may contribute to optimize pre-diabetes management in CF patients.

The Emergence of Streptococcus anginosus Group as a Cystic Fibrosis Pathogen

Molecular profiling studies have identified potential emerging pathogens, such as Streptococcus anginosus group, that may play a role in cystic fibrosis (CF) lung disease by either directly causing infection or upregulating the virulence factors of classic CF pathogens, such as Pseudomonas aeruginosa. Routine surveillance of CF pathogens using traditional microbiology culture guides treatment and management of CF patients; however, routine CF culture protocols have not been modified to select for, detect, and further determine the role these emerging pathogens play in the progression of CF lung disease.

188 Expert consensus for the management of cystic fibrosis patients with severe pulmonary affectation using inhaled antibiotics

188 Expert consensus for the management of cystic fibrosis patients with severe pulmonary affectation using inhaled antibiotics

Combinations of colistin solutions and nebulisers for lung infection management in cystic fibrosis patients

In this work different nebulisers were investigated in order to assess their efficiency in combination with colistimethate sodium (CMS) inhalation products. Four nebulisers, namely I-neb®, Aeroneb® Go, eFlow®rapid and PARI LC® Sprint were studied in terms of delivered dose (DD), drug delivery rate (DDR) and respirable dose (RD) of CMS. The goal was to provide scientific data to physicians for prescribing the most appropriate nebuliser for the CMS specific user.All the apparatuses nebulised ColiFin 1MIU/3ml solution (80mg of CMS) with delivered doses between 31% and 41% of the loaded amount. Aeroneb Go showed the longest nebulisation time (more than 20min). When ColiFin 2MIU/4ml was nebulised with eFlow rapid or PARI LC Sprint, the CMS respirable dose was 45.3mg and 39.2mg, in times of 5.6 and 10.8min, respectively. I-neb, having a medication cup capacity limited to 0.4ml, loaded with Promixin 0.4MIU/0.4ml (32mg of CMS), provided in a time of 9min a RD of 21.5mg, a value slightly higher than those obtained by nebulising ColiFin 1MIU/3ml with the other nebulisers (range 15.9–17.6mg).The results illustrate that the clinical outcome depends on the comparative analysis of nebulisation efficiency (respirable dose) and convenience (time), not disregarding the ratios between the amount loaded, delivered and deposited at lung level.

A phase 3, multi-center, multinational, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of levofloxacin inhalation solution (APT-1026) in stable cystic fibrosis patients

RationaleFor patients with cystic fibrosis (CF), the use of inhaled antibiotics has become standard of care to suppress chronic Pseudomonas airways infection. There are limited antibiotic options formulated and approved for inhaled use and antibiotic efficacies attenuate over time, making additional inhaled antibiotic classes desirable. APT-1026 (levofloxacin inhalation solution, LIS) is a fluoroquinolone in development for management of chronic P. aeruginosa airways infection in patients with CF. ObjectivesTo compare the safety and efficacy of a 28-day course of treatment with LIS 240mg or placebo BID in persons ≥12years old with CF and chronic P. aeruginosa infection. MethodsA multinational, randomized (2:1), double-blinded study of LIS and placebo over 28days in CF patients ≥12years with chronic P. aeruginosa infection. Time to exacerbation was the primary endpoint. FEV1 (% predicted) and patient-reported quality of life were among secondary endpoints. Main resultsBaseline demographics for 330 subjects (LIS=220) were similar although significantly more patients randomized to LIS had experienced multiple exacerbations in the year prior to study entry. There was no statistically significant difference in protocol-defined pulmonary exacerbations between treatment arms. Relative change in FEV1% predicted from baseline was significantly greater for patients randomized to LIS compared to those randomized to placebo (mean difference 1.31%, p=0.01 [95% CI 0.27, 2.34%]). LIS was well-tolerated, with dysguesia the most frequent adverse event. ConclusionsLIS did not demonstrate a difference in time to next exacerbation when compared to placebo. Reasons for this result are discussed but may be due to an imbalance in the frequency of prior pulmonary exacerbations between the two groups. An improvement in FEV1 (% predicted) at 28days was observed and LIS was well tolerated. LIS is safe and has a potential role in the management of CF patients with chronic P. aeruginosa.

Mounier Kuhn Syndrome Presenting with Recurrent Atelectasis

Context: Mounier Kuhn syndrome is usually diagnosed in adulthood, and only a few cases have been described in childhood. Case report: We present the case of a seven-year old boy suffering from recurrent pneumonia and atelectasis. Previously performed chest X-rays showed bilateral hyperinflation and tracheobronchomegaly. Chest computed tomography (CT) confirmed the presence of distal enlargement of trachea and bronchi. Tracheobronchomegaly associated with recurrent respiratory tract infections is consistent with Mounier Kuhn syndrome. Pseudomonas aeruginosa was isolated from the sputum of the patient. He was then treated according to the guidelines for Pseudomonas aeruginosa management in cystic fibrosis patients considering the similarities in clinical presentations and pathophysiology of both diseases. Antibiotic treatment resulted in a remarkable reduction of events of pulmonary exacerbation and hospitalizations. There are no specific guidelines for treatment options in case of pulmonary exacerbation of Mounier Kuhn syndrome. Case reports discussing the choice and efficiency of antibiotic treatment are random. Conclusion: We share our experience of treating pulmonary exacerbation caused by Pseudomonas aeruginosa in a patient with Mounier Kuhn syndrome suggesting a possible treatment option of pseudomonas infections in this syndrome.

WS04.3 Deficiency of dehydroepiandrosterone sulfate in pediatric and adult patients with cystic fibrosis

Objectives Dehydroepiandrosterone (DHEA) is the most abundant adrenal steroid, and the serum concentrations of its sulfate ester, DHEA-S, are 20-fold higher than those of any other circulating steroid hormone. Decreased levels of DHEA-S are associated with pulmonary hypertension and cardiovascular diseases, immune abnormalities, osteoporosis, glucose intolerance and diabetes, deterioration of lipid metabolism. The aim of the study was to evaluate DHEA-S concentrations in children and adults with cystic fibrosis (CF). Methods 125 pediatric (6–17 years) and 38 adult patients (18–28 years) with CF were enrolled into the study. Plasma DHEA-S and ACTH levels were assayed with commercially available kits. Results DHEA-S concentrations were significantly below the age-adjusted median values in 68% of children and 73% of adults with CF. The hormone deficiency was more pronounced in subjects with cirrhosis and in the group of young women where, respectively, 100% and 82% of participants had DHEA-S concentrations below the age-adjusted median values. Pediatric patients (6–11 years) with minimal DHEA-S levels demonstrated significantly lower Body Mass Indexes compared to those of CF children who had normal hormone concentrations (14.4 vs 16.3 kg/m 2 p Conclusion Our data indicate that most pediatric and adult CF patients suffer from DHEA-S deficiency. Since DHEA is a well-tolerated and inexpensive drug, its supplementation might prove useful for the clinical management of CF patients with low levels of the hormone.

Role of nebulized amphotericin B in the management of allergic bronchopulmonary aspergillosis in cystic fibrosis: Case report and review of literature

Objectives:To review the data available in literature about nebulized amphotericin B (AMB) in the treatment of allergic bronchopulmonary aspergillosis (ABPA) in cystic fibrosis (CF) and to report our experience in the use of this drug, with a particular therapeutic scheme.Case Report:We used nebulized liposomal amphotericin B (L-AMB) in a patient affected by CF, complicated by ABPA. The previous combined treatment with oral steroids and azoles had no respiratory benefit and caused relevant side effects. Amphotericin B has always been well tolerated and permitted a slight steroid tapering. We also observed benefits in pulmonary function and laboratory tests.Conclusions:Few data are available in literature about the use of nebulized AMB in CF and there are no RCTs evaluating antifungals in CF-ABPA. In our opinion, the reported case suggests that nebulized L-AMB could represent a possible strategy in ABPA management in CF patients.

Proposal of a quantitative PCR-based protocol for an optimal Pseudomonas aeruginosa detection in patients with cystic fibrosis

BackgroundThe lung of patients with cystic fibrosis (CF) is particularly sensitive to Pseudomonas aeruginosa. This bacterium plays an important role in the poor outcome of CF patients. During the disease progress, first acquisition of P. aeruginosa is the key-step in the management of CF patients. Quantitative PCR (qPCR) offers an opportunity to detect earlier the first acquisition of P. aeruginosa by CF patients. Given the lack of a validated protocol, our goal was to find an optimal molecular protocol for detection of P. aeruginosa in CF patients.MethodsWe compared two formerly described qPCR formats in early detection of P. aeruginosa in CF sputum samples: a qPCR targeting oprL gene, and a multiplex PCR targeting gyrB and ecfX genes.ResultsTested in vitro on a large panel of P. aeruginosa isolates and others gram-negative bacilli, oprL qPCR exhibited a better sensitivity (threshold of 10 CFU/mL versus 730 CFU/mL), whereas the gyrB/ecfX qPCR exhibited a better specificity (90% versus 73%). These results were validated ex vivo on 46 CF sputum samples positive for P. aeruginosa in culture. Ex vivo assays revealed that qPCR detected 100 times more bacterial cells than culture-based method did.ConclusionBased on these results, we proposed a reference molecular protocol combining the two qPCRs, which offers a sensitivity of 100% with a threshold of 10 CFU/mL and a specificity of 100%. This combined qPCR-based protocol can be adapted and used for other future prospective studies.

265 Prevalence and treatment of reduced BMD in a cystic fibrosis cohort undergoing lung transplantation

Background: As life expectancy in cystic fibrosis (CF) increases, long term sequelae and their treatment assume greater importance. Osteoporosis is a prime example. Objectives: To examine the prevalence and treatment of reduced bone mineral density (BMD) in CF patients referred for lung transplantation as a basis for implementing improved clinical practice. Methods: Single centre retrospective analysis of bone health and its management in CF patients who underwent lung transplantation 01/2008−07/2012. We measured BMD via dual-energy X-ray absorptiometry (DEXA) scan and examined multiple risk factors for reduced BMD. Results: 41 patients with CF underwent primary lung transplantation, comprising 63% females. Mean age at transplant was 27 years (95% CI 24−30), mean BMI 18.35 (95% CI 17.56–19.13). 5/41 patients (12%) had a documented history of fracture. DEXA was undertaken in 39/41 (95%) of patients prior to transplantation, with t-scores reported in 27/41 patients. The mean t-score at L2-L4 was −1.4 (95% CI −1.0 to −1.8); and at the femoral neck −1.1 (95% CI −0.7 to −1.5). 13/27 (48%) patients met WHO criteria for osteopenia and 6/27 (22%) for osteoporosis. There was significant variability in the measurement and treatment of risk factors. Conclusions: Clinically significant reduced BMD is frequent in CF patients referred for consideration of lung transplantation. Treatment of reduced BMD and its risk factors was variable, prompting implementation of a number of clinical practice improvement measures including pre-emptive management prior to referral.

266 Cystic fibrosis related osteopenia: What is the etiology?

Background: As life expectancy in cystic fibrosis (CF) increases, long term sequelae and their treatment assume greater importance. Osteoporosis is a prime example. Objectives: To examine the prevalence and treatment of reduced bone mineral density (BMD) in CF patients referred for lung transplantation as a basis for implementing improved clinical practice. Methods: Single centre retrospective analysis of bone health and its management in CF patients who underwent lung transplantation 01/2008−07/2012. We measured BMD via dual-energy X-ray absorptiometry (DEXA) scan and examined multiple risk factors for reduced BMD. Results: 41 patients with CF underwent primary lung transplantation, comprising 63% females. Mean age at transplant was 27 years (95% CI 24−30), mean BMI 18.35 (95% CI 17.56–19.13). 5/41 patients (12%) had a documented history of fracture. DEXA was undertaken in 39/41 (95%) of patients prior to transplantation, with t-scores reported in 27/41 patients. The mean t-score at L2-L4 was −1.4 (95% CI −1.0 to −1.8); and at the femoral neck −1.1 (95% CI −0.7 to −1.5). 13/27 (48%) patients met WHO criteria for osteopenia and 6/27 (22%) for osteoporosis. There was significant variability in the measurement and treatment of risk factors. Conclusions: Clinically significant reduced BMD is frequent in CF patients referred for consideration of lung transplantation. Treatment of reduced BMD and its risk factors was variable, prompting implementation of a number of clinical practice improvement measures including pre-emptive management prior to referral.

Pre-hospital management of cystic fibrosis patients presenting with haemoptysis

This paper discusses the pre-hospital clinical management of cystic fibrosis (CF) patients suffering with haemoptysis. While clear guidelines and procedures exist within hospitals in regard to the management of such patients, the same cannot be said for the pre-hospital setting, with very limited clinical practice guidance. Massive haemoptysis is a serious threat to life, though for some patients such as the CF population, even a minor bleed can have devastating effect on quality of life. This paper proposes a pre-hospital clinical guideline based on a retrospective case series from a large Australian tertiary hospital. It is hoped this paper will provide important clinical information regarding CF patients suffering with haemoptysis as it is imperative the management of these patients is undertaken promptly and is well-informed.