scholarly journals Variation in treatment preferences of pulmonary exacerbations among Australian and New Zealand cystic fibrosis physicians

2021 ◽  
Vol 8 (1) ◽  
pp. e000956
Author(s):  
Grace Currie ◽  
Anna Tai ◽  
Tom Snelling ◽  
André Schultz
Keyword(s):  
Cystic Fibrosis ◽  
Clinical Trials ◽  
New Zealand ◽  
Treatment Options ◽  
Early Response ◽  
Treatment Preferences ◽  
Dornase Alfa ◽  
Clinical Scenarios ◽  
Professional Opinion ◽  
Pulmonary Exacerbations

BackgroundDespite advances in cystic fibrosis (CF) management and survival, the optimal treatment of pulmonary exacerbations remains unclear. Understanding the variability in treatment approaches among physicians might help prioritise clinical uncertainties to address through clinical trials.MethodsPhysicians from Australia and New Zealand who care for people with CF were invited to participate in a web survey of treatment preferences for CF pulmonary exacerbations. Six typical clinical scenarios were presented; three to paediatric and another three to adult physicians. For each scenario, physicians were asked to choose treatment options and provide reasons for their choices.ResultsForty-nine CF physicians (31 paediatric and 18 adult medicine) participated; more than half reported 10+ years of experience. There was considerable variation in primary antibiotic selection; none was preferred by more than half of respondents in any scenario. For secondary antibiotic therapy, respondents consistently preferred intravenous tobramycin and a third antibiotic was rarely prescribed, except in one scenario describing an adult patient. Hypertonic saline nebulisation and twice daily chest physiotherapy was preferred in most scenarios while dornase alfa use was more variable. Most CF physicians (>80%) preferred to change therapy if there was no early response. Professional opinion was the most common reason for antibiotic choice.ConclusionsVariation exists among CF physicians in their preferred choice of primary antibiotic and use of dornase alfa. These preferences are driven by professional opinion, possibly reflecting a lack of evidence to base policy recommendations. Evidence from high-quality clinical trials is needed to inform physician decision making.

scholarly journals A first-year dornase alfa treatment impact on clinical parameters of patients with cystic fibrosis: the Brazilian cystic fibrosis multicenter study

2013 ◽  
Vol 31 (4) ◽  
pp. 420-430 ◽  
Author(s):  
Tatiana Rozov ◽  
Fernando Antônio A. e Silva ◽  
Maria Angélica Santana ◽  
Fabíola Villac Adde ◽  
Rita Heloisa Mendes
Keyword(s):  
Cystic Fibrosis ◽  
Emergency Room ◽  
Age Groups ◽  
Forced Expiratory Volume ◽  
First Year ◽  
Age Group ◽  
Emergency Room Visits ◽  
Dornase Alfa ◽  
Pulmonary Exacerbations ◽  
One Year

OBJECTIVE: To describe the clinical impact of the first year treatment with dornase alfa, according to age groups, in a cohort of Brazilian Cystic Fibrosis (CF) patients. METHODS: The data on 152 eligible patients, from 16 CF reference centers, that answered the medical questionnaires and performed laboratory tests at baseline (T0), and at six (T2) and 12 (T4) months after dornase alfa initiation, were analyzed. Three age groups were assessed: six to 11, 12 to 13, and >14 years. Pulmonary tests, airway microbiology, emergency room visits, hospitalizations, emergency and routine treatments were evaluated. Student's t-test, chi-square test and analysis of variance were used when appropriated. RESULTS: Routine treatments were based on respiratory physical therapy, regular exercises, pancreatic enzymes, vitamins, bronchodilators, corticosteroids, and antibiotics. In the six months prior the study (T0 phase), hospitalizations for pulmonary exacerbations occurred in 38.0, 10.0 and 61.4% in the three age groups, respectively. After one year of intervention, there was a significant reduction in the number of emergency room visits in the six to 11 years group. There were no significant changes in forced expiratory volume in one second (VEF1), in forced vital capacity (FVC), in oxygen saturation (SpO2), and in Tiffenau index for all age groups. A significant improvement in Shwachman-Kulczychi score was observed in the older group. In the last six months of therapy, chronic or intermittent colonization by P. aeruginosa was detected in 75.0, 71.4 and 62.5% of the studied groups, respectively, while S. aureus colonization was identified in 68.6, 66.6 and 41.9% of the cases. CONCLUSIONS: The treatment with dornase alfa promoted the maintenance of pulmonary function parameters and was associated with a significant reduction of emergency room visits due to pulmonary exacerbations in the six to 11 years age group, with better clinical scores in the >14 age group, one year after the intervention.

Systemic treatment preferences for patients with advanced desmoid-type fibromatosis (DF) in Europe.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. e22523-e22523
Author(s):  
Thomas Van Cann ◽  
Annelies Requilé ◽  
Patrick Schoffski
Keyword(s):  
Clinical Trials ◽  
Randomized Trials ◽  
Systemic Treatment ◽  
Treatment Options ◽  
Local Treatment ◽  
Control Treatment ◽  
Treatment Preferences ◽  
Homogeneous Population ◽  
Symptomatic Disease ◽  
Wait And See

e22523 Background: The treatment of DF as a rare, benign mesenchymal tumor is poorly standardized and based on physician’s choice. Only a few clinical trials have assessed the different treatment options in DF and randomized evidence is virtually non-existing. We evaluated systemic treatment preferences for patients (pts) with advanced DF among European sarcoma experts, aiming to provide a reference for a generally acceptable control treatment for potential prospective randomized trials. Methods: A 7-item structured questionnaire was sent to physicians in Europe and Israel involved in the multidisciplinary care and experimental treatment of mesenchymal tumors, to assess their systemic treatment choices for DF. Results: The questionnaire was sent to 266 experts (117 institutions, 21 countries), 54 experts (52 institutions, 14 countries) responded. Wait and see was the most common primary approach for pts with advanced DF. (Symptomatic) Disease progression and failure or non-availability of local treatment options were cited as common reasons for considering systemic therapy. Treatment preferences for pts with sporadic vs hereditary DF were similar, with a tendency of earlier use of chemotherapy in hereditary DF. At least 28 different agents or regimens are used in DF. Tamoxifen +/- nonsteroidal anti-inflammatory agents (NSAIDs) or NSAIDs alone are most commonly used in first line, followed by methotrexate- or anthracycline-containing regimen. Tyrosine kinase inhibitors are often used in further lines; other drugs are sporadically used. Clinical trial activity in DF was restricted to only one country/one multi-centric study. Conclusions: DF pts in Europe and Israel have broad off label access to systemic agents, but only to a few clinical trials. Treatment approaches are diverse, poorly standardized and based on weak evidence. There is an urgent need for practice-defining, prospective randomized trials. Such trials should ideally select a homogeneous population of pts with well documented, progressive, symptomatic disease and/or functional impairment after wait and see and/or local treatments. NSAIDs +/- tamoxifen could be considered a generally accepted standard control treatment for clinical trials.

Interpreting Randomized Clinical Trials in Gynecologic Oncology Surgery: Does One Size Fit All?

2019 ◽  
pp. 342-350
Author(s):  
Leigh A. Cantrell ◽  
Jacobus Pfisterer ◽  
John Boggess ◽  
Linda R. Duska
Keyword(s):  
Clinical Trials ◽  
Clinical Research ◽  
Randomized Clinical Trials ◽  
Early Stage ◽  
Treatment Options ◽  
Gynecologic Oncology ◽  
Advanced Ovarian Cancer ◽  
Clinical Scenarios ◽  
Early Stage Cervical Cancer ◽  
New Treatment

Randomized clinical trials (RCTs) are considered the gold standard of clinical research. They are designed to eliminate bias and to produce objective and generalizable results about new treatment paradigms. Although RCTs have recognized limitations, including long completion time and high cost, they also have transformed clinical research and improved the quality of health care by rigorously evaluating countless new treatment options. Surgical RCTs present their own unique set of limitations including an inability to standardize surgical technique and expertise; an inability to overcome enrollment bias by enrolling surgeons; and a lack of generalizability with respect to institutional resources and abilities. Here, we discuss surgical RCTs in two domains: upfront management of advanced ovarian cancer and surgical management of early-stage cervical cancer. Familiarity with the abundant retrospective data available for both of these clinical scenarios as well as recognition of the strengths and limitations of surgical RCTs are critical to determine the best treatment for an individual patient.

Comparative Effectiveness of Vancomycin Versus Linezolid for the Treatment of Acute Pulmonary Exacerbations of Cystic Fibrosis

2019 ◽  
Vol 54 (3) ◽  
pp. 197-204 ◽  
Author(s):  
Nicholas M. Fusco ◽  
Calvin J. Meaney ◽  
Carla A. Frederick ◽  
William A. Prescott
Keyword(s):  
Cystic Fibrosis ◽  
Lung Function ◽  
Adverse Drug Events ◽  
Treatment Options ◽  
Forced Expiratory Volume ◽  
Median Percentage ◽  
Median Percentage Change ◽  
Pulmonary Exacerbations ◽  
Viable Treatment

Background: Data are limited regarding the preferred antibiotics for treatment of acute pulmonary exacerbations (APEs) of cystic fibrosis (CF), when methicillin-resistant Staphylococcus aureus (MRSA) is suspected. Objective: To compare the rate of return to baseline lung function among individuals with APEs of CF treated with either vancomycin or linezolid. Methods: This retrospective study included individuals hospitalized for APEs of CF from May 1, 2015, to April 30, 2017 who were infected with MRSA and treated with vancomycin or linezolid. The primary outcome was the return to baseline lung function, as measured by forced expiratory volume in 1 s (FEV1). Descriptive and inferential statistics were used. All tests were 2-tailed with α set at 0.05. Results: A total of 122 encounters were included (vancomycin: n = 66; linezolid: n = 66). No difference existed in return to baseline FEV1 between vancomycin (53 [80.3%]) and linezolid (50 [75.8%]; P = 0.53); nor was there a difference in median percentage change in FEV1 from admission to follow-up between vancomycin (24.7%) and linezolid (20.7%; P = 0.61). Adverse drug events occurred more frequently in patient encounters treated with vancomycin (10 [15.2%]) compared with linezolid (2 [3%]; P = 0.002). Conclusion and Relevance: Our study observed no difference in the effectiveness of vancomycin compared with linezolid in terms of change in lung function for APEs of CF. The rate of adverse drug events was low. In individuals with CF infected with MRSA who are experiencing an APE, either vancomycin or linezolid appear to be viable treatment options.

Inhaled Antibiotics in Cystic Fibrosis: A Review

1996 ◽  
Vol 30 (7-8) ◽  
pp. 840-850 ◽  
Author(s):  
Cynthia Toso ◽  
Dennis M Williams ◽  
Peader G Noone
Keyword(s):  
Cystic Fibrosis ◽  
Clinical Trials ◽  
Pulmonary Function ◽  
Adjunctive Therapy ◽  
Pulmonary Function Tests ◽  
Response To Therapy ◽  
Suppressive Therapy ◽  
Individual Response ◽  
Inhaled Antibiotics ◽  
Pulmonary Exacerbations

OBJECTIVE: To provide an overview of aerosol drug therapy, including physical considerations and aerosol drug delivery systems, and to review clinical experience with inhaled antibiotics in cystic fibrosis (CF) when used as adjunctive therapy to intravenous therapy for acute pulmonary exacerbations and chronic, suppressive therapy. DATA SOURCES: A MEDLINE search (1966-1995) of English-language literature describing the use of inhaled antibiotics for the management of CF. STUDY SELECTION AND DATA EXTRACTION: All articles were considered for possible inclusion in the review. Pertinent information as judged by the authors was selected for discussion. DATA SYNTHESIS: The use of inhaled antibiotics as adjunctive therapy to systemic therapy for acute exacerbations did not improve pulmonary function tests, increase hospital discharge rate, or permanently eradicate sputum Pseudomonas. Clinical trials of inhaled antibiotics as suppressive therapy yielded variable results. Individually, four trials documented a significant improvement in pulmonary function, three trials documented a slower decline in pulmonary function, and four trials reported a reduced frequency of hospitalizations. However, the trials were unable to collectively document a prolonged beneficial effect of inhaled antibiotics on pulmonary function, sputum bacterial density, and frequency of hospitalizations. CONCLUSIONS: Clinical trials conducted thus far suggest no role for inhaled antibiotics in the treatment of acute pulmonary exacerbations in patients with CF. Aerosolized antibiotics used as suppressive therapy may be useful in certain patients, but their use should be limited to select patients based on individual response to therapy. Additional long-term, well-controlled trials of inhaled antibiotics as suppressive therapy are needed before routine use can be recommended.

The Role of Dornase Alfa in the Treatment of Cystic Fibrosis

1996 ◽  
Vol 30 (6) ◽  
pp. 656-661 ◽  
Author(s):  
Gena W Cramer ◽  
John A Bosso ◽  
Wanda T Maldonado ◽  
Chantal Guévremont
Keyword(s):  
Quality Of Life ◽  
Cystic Fibrosis ◽  
Clinical Trials ◽  
Positive Impact ◽  
Infection Rates ◽  
Long Term Effects ◽  
Dornase Alfa

Objective To review the current utility and proper role of dornase alfa (recombinant human DNase or rhDNase), which has been approved for use in cystic fibrosis. Several aspects related to these issues are addressed including the drug's mechanism of action, administration and dosing, and clinical safety and efficacy. We also critically examine the agent's role in the treatment of cystic fibrosis and consider the controversies involved with its use. data source A MEDLINE search was conducted to identify pertinent literature, including review articles and clinical trials. Study Selection Studies examining the efficacy and safety of dornase alfa in patients with cystic fibrosis. Data Extraction Results from published, prospective, randomized trials are presented and critiqued. Data Synthesis Production of viscous respiratory secretions is a hallmark phenomenon of cystic fibrosis, leading to a variety of symptoms. Dornase alfa targets this symptom and decreases the viscosity of these secretions. Clinical trials have indicated a small but statistically significant improvement in forced expiratory volume in 1 second and forced vital capacity. Enhancement in a patient's dyspnea and quality of life has varied between the trials, with few of the studies noting no statistically significant improvement. Adverse reactions are minimal and did not result in any patient withdrawals from the trials. A positive impact on infection rates, length of hospitalization, and need for intravenous antibiotic therapy was noted in one trial. However, reports of similar results have not yet been published, and thus the clinical significance or impact of this phenomenon is not fully understood. Moreover, results of more long-term use and in patients whose conditions are less stable have yet to undergo the scrutiny of peer/editorial review. Administration of the drug, which must be maintained continuously, is relatively expensive. Conclusions Dornase alfa appears to produce small but sustained improvements in lung function in patients with cystic fibrosis. It may also slow the progression of pulmonary disease. Infection rates appear to be reduced, which may well have important long-term consequences. However, evidence to date has not clarified the most appropriate use of dornase alfa in the treatment of cystic fibrosis. Whether quality of life is affected in a meaningful and measurable way is yet to be clarified. A trial of the drug in patients with cystic fibrosis who have obvious lung disease is reasonable, but continued treatment should be based on clear clinical response. Therefore, questions about the drug's exact role in the overall management of cystic fibrosis remain to be answered. Although benefits received may not prove to be cost-effective, long-term effects on disease progression may well justify use of this agent.

The impact of a physician’s recommendation and gender on informed decision making: A randomized controlled study in a simulated decision situation

2020 ◽  
Author(s):  
Marie Eggeling ◽  
Anna Meinhardt ◽  
Ulrike Cress ◽  
Joachim Kimmerle ◽  
Martina Bientzle
Keyword(s):  
Decision Making ◽  
Cruciate Ligament ◽  
Treatment Options ◽  
General Information ◽  
Treatment Preference ◽  
Controlled Study ◽  
Treatment Preferences ◽  
Hypothetical Scenario ◽  
And Gender ◽  
The Impact

Objective: This study examined the influence of physicians’ recommendations and gender on the decision-making process in a preference-sensitive situation. Methods: N = 201 participants were put in a hypothetical scenario in which they suffered from a rupture of the anterior cruciate ligament (ACL). They received general information on two equally successful treatment options for this injury (surgery vs. physiotherapy) and answered questions regarding their treatment preference, certainty and satisfaction regarding their decision, and attitude toward the treatment options. Then participants watched a video that differed regarding physician’s recommendation (surgery vs. physiotherapy) and physician’s gender (female vs. male voice and picture). Afterward, they indicated again their treatment preference, certainty, satisfaction, and attitude, as well as the physician’s professional and social competence.Results: Participants changed their treatment preferences in the direction of the physician’s recommendation (P<.001). Decision certainty (P<.001) and satisfaction (P<.001) increased more strongly if the physician’s recommendation was congruent with the participant’s prior attitude than if the recommendation was contrary to the participant’s prior attitude. Finally, participants’ attitudes toward the recommended treatment became more positive (surgery recommendation: P<.001; physiotherapy recommendation: P<.001). We found no influence of the physician’s gender on participants’ decisions, attitudes, or competence assessments.Conclusion: This research indicates that physicians should be careful with recommendations when aiming for shared decisions, as they might influence patients even if the patients have been made aware that they should take their personal preferences into account. This could be particularly problematic if the recommendation is not in line with the patient’s preferences.

Preliminary Report of In vitro and In vivo Effectiveness of Dornase Alfa on SARS-CoV-2 Infection (Preprint)

2020 ◽  
Author(s):  
Hacer Kuzu Okur ◽  
Koray Yalcin ◽  
Cihan Tastan ◽  
Sevda Demir ◽  
Bulut Yurtsever ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Respiratory Tract ◽  
Mononuclear Cells ◽  
Multiple Organ ◽  
Peripheral Blood Mononuclear ◽  
Dornase Alfa ◽  
Tract Infections ◽  
Adult Peripheral Blood

UNSTRUCTURED Dornase alfa, the recombinant form of the human DNase I enzyme, breaks down neutrophil extracellular traps (NET) that include a vast amount of DNA fragments, histones, microbicidal proteins and oxidant enzymes released from necrotic neutrophils in the highly viscous mucus of cystic fibrosis patients. Dornase alfa has been used for decades in patients with cystic fibrosis to reduce the viscoelasticity of respiratory tract secretions, to decrease the severity of respiratory tract infections, and to improve lung function. Previous studies have linked abnormal NET formations to lung diseases, especially to acute respiratory distress syndrome (ARDS). Coronavirus disease 2019 (COVID-19) pandemic affected more than two million people over the world, resulting in unprecedented health, social and economic crises. The COVID-19, viral pneumonia that progresses to ARDS and even multiple organ failure, is caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). High blood neutrophil levels are an early indicator of SARS-CoV-2 infection and predict severe respiratory diseases. A similar mucus structure is detected in COVID-19 patients due to the accumulation of excessive NET in the lungs. Here, we show our preliminary results with dornase alfa that may have an in-vitro anti-viral effect against SARS-CoV-2 infection in a bovine kidney cell line, MDBK without drug toxicity on healthy adult peripheral blood mononuclear cells. In this preliminary study, we also showed that dornase alfa can promote clearance of NET formation in both an in-vitro and three COVID-19 cases who showed clinical improvement in radiological analysis (2-of-3 cases), oxygen saturation (SpO2), respiratory rate, disappearing of dyspnea and coughing.

scholarly journals Therapeutic clinical trials to combat COVID-19 pandemic in India: analysis from trial registry

2020 ◽  
Vol 0 (0) ◽  
Author(s):  
Angelika Batta ◽  
Raj Khirasaria ◽  
Vinod Kapoor ◽  
Deepansh Varshney
Keyword(s):  
Clinical Trials ◽  
De Novo ◽  
Treatment Options ◽  
Drug Repurposing ◽  
Trial Registry ◽  
Therapeutic Treatment ◽  
Clinical Trial Registry ◽  
Clear Cut ◽  
Therapeutic Trials ◽  
Microsoft Office

AbstractObjectivesWith the emergence of Novel corona virus, hunt for finding a preventive and therapeutic treatment options has already begun at a rapid pace with faster clinical development programs. The present study was carried out to give an insight of therapeutic interventional trials registered under clinical trial registry of India (CTRI) for COVID-19 pandemic.MethodsAll trials registered under CTRI were evaluated using keyword “COVID” from its inception till 9th June 2020. Out of which, therapeutic interventional studies were chosen for further analysis. Following information was collected for each trial: type of therapeutic intervention (preventive/therapeutic), treatment given, no. of centers (single center/multicentric), type of institution (government/private), study design (randomized/single-blinded/double-blinded) and sponsors (Government/private). Microsoft Office Excel 2007 was used for tabulation and analysis.ResultsThe search yielded total of 205 trials, out of which, 127 (62%) trials were interventional trials. Out of these, 71 (56%) were AYUSH interventions, 36 (28.3%) tested drugs, 9 (7%) tested a nondrug intervention, rest were nutraceuticals and vaccines. About 66 (56%) were therapeutic trials. Majority were single-centered trials, i.e. 87 (73.7%). Trials were government funded in 57 (48.3%) studies. Majority were randomized controlled trials, i.e. 67 (56.8%). AYUSH preparations included AYUSH-64, Arsenic Album, SamshamaniVati etc.ConclusionsThe number of therapeutic interventional clinical trials was fair in India. A clear-cut need exists for an increase in both quantity and quality of clinical trials for COVID-19. Drug repurposing approach in all systems of medicine can facilitate prompt clinical decisions at lower costs than de novo drug development.

scholarly journals Diagnosis and treatment of neurogenic dysphagia – S1 guideline of the German Society of Neurology

2021 ◽  
Vol 3 (1) ◽  
Author(s):  
Rainer Dziewas ◽  
Hans-Dieter Allescher ◽  
Ilia Aroyo ◽  
Gudrun Bartolome ◽  
Ulrike Beilenhoff ◽  
...  
Keyword(s):  
Neurological Diseases ◽  
Treatment Options ◽  
German Society ◽  
Diagnosis And Treatment ◽  
Dietary Interventions ◽  
Neurogenic Dysphagia ◽  
Treatment Interventions ◽  
Term Care ◽  
High Resolution Manometry ◽  
Clinical Scenarios

Abstract Introduction Neurogenic dysphagia defines swallowing disorders caused by diseases of the central and peripheral nervous system, neuromuscular transmission, or muscles. Neurogenic dysphagia is one of the most common and at the same time most dangerous symptoms of many neurological diseases. Its most important sequelae include aspiration pneumonia, malnutrition and dehydration, and affected patients more often require long-term care and are exposed to an increased mortality. Based on a systematic pubmed research of related original papers, review articles, international guidelines and surveys about the diagnostics and treatment of neurogenic dysphagia, a consensus process was initiated, which included dysphagia experts from 27 medical societies. Recommendations This guideline consists of 53 recommendations covering in its first part the whole diagnostic spectrum from the dysphagia specific medical history, initial dysphagia screening and clinical assessment, to more refined instrumental procedures, such as flexible endoscopic evaluation of swallowing, the videofluoroscopic swallowing study and high-resolution manometry. In addition, specific clinical scenarios are captured, among others the management of patients with nasogastric and tracheotomy tubes. The second part of this guideline is dedicated to the treatment of neurogenic dysphagia. Apart from dietary interventions and behavioral swallowing treatment, interventions to improve oral hygiene, pharmacological treatment options, different modalities of neurostimulation as well as minimally invasive and surgical therapies are dealt with. Conclusions The diagnosis and treatment of neurogenic dysphagia is challenging and requires a joined effort of different medical professions. While the evidence supporting the implementation of dysphagia screening is rather convincing, further trials are needed to improve the quality of evidence for more refined methods of dysphagia diagnostics and, in particular, the different treatment options of neurogenic dysphagia. The present article is an abridged and translated version of the guideline recently published online (https://www.awmf.org/uploads/tx_szleitlinien/030-111l_Neurogene-Dysphagie_2020-05.pdf).

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