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Article ; Online: Effect of ester to amide or N-methylamide substitution on bacterial membrane depolarization and antibacterial activity of novel cyclic lipopeptides.

Bionda, Nina / Fleeman, Renee M / Shaw, Lindsey N / Cudic, Predrag

2013 Volume 8, Issue 8, Page(s) 1394–1402

Abstract: ... of cyclic lipodepsipeptide 1 and its analogues--amide 2, N-methylamide 3, and linear peptide 4--to interact ... of the depsipeptide bond in 1 with an N-methylamide bond in 3, or its hydrolysis to peptide 4, lead to a complete loss ...

Abstract	Cyclic lipopeptides derived from the fusaricidin/LI-F family of naturally occurring antibiotics represent particularly attractive candidates for the development of new antibacterial agents. In comparison with natural products, these derivatives may offer better stability under physiologically relevant conditions and lower nonspecific toxicity, while preserving their antibacterial activity. In this study we assessed the ability of cyclic lipodepsipeptide 1 and its analogues--amide 2, N-methylamide 3, and linear peptide 4--to interact with the cytoplasmic membranes of selected Gram-positive bacteria. We also investigated their bacteriostatic/bactericidal modes of action and in vivo potency by using a Galleria mellonella model of MRSA infection. Cyclic lipopeptides 1 and 2 depolarize the cytoplasmic membranes of Gram-positive bacteria in a concentration-dependent manner. The degree of membrane depolarization was influenced by the structural and physical properties of 1 and 2, with the more flexible and hydrophobic peptide 1 being most efficient. However, membrane depolarization does not correlate with bacterial cell lethality, suggesting that membrane-targeting activity is not the main mode of action for this class of antibacterial peptides. Conversely, substitution of the depsipeptide bond in 1 with an N-methylamide bond in 3, or its hydrolysis to peptide 4, lead to a complete loss of antibacterial activity and indicate that the conformation of cyclic lipopeptides plays a role in their antibacterial activities. Cyclic lipopeptides 1 and 2 are also capable of improving the survival of G. mellonella larvae infected with MRSA at varying efficiencies, reflecting their in vitro activities. Gaining more insight into the structure-activity relationship and mode of action of these cyclic lipopeptides may enable the development of new antibiotics of this class with improved antibacterial activity.
MeSH term(s)	Amides/chemistry ; Animals ; Anti-Bacterial Agents/chemical synthesis ; Anti-Bacterial Agents/chemistry ; Anti-Bacterial Agents/pharmacology ; Cell Wall/drug effects ; Circular Dichroism ; Esters ; Gram-Positive Bacteria/drug effects ; Gram-Positive Bacteria/metabolism ; Larva/microbiology ; Lepidoptera/growth & development ; Lipopeptides/chemical synthesis ; Lipopeptides/chemistry ; Lipopeptides/pharmacology ; Methicillin-Resistant Staphylococcus aureus/drug effects ; Methicillin-Resistant Staphylococcus aureus/isolation & purification ; Models, Animal
Chemical Substances	Amides ; Anti-Bacterial Agents ; Esters ; Lipopeptides
Language	English
Publishing date	2013-06-18
Publishing country	Germany
Document type	Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
ZDB-ID	2218496-X
ISSN	1860-7187 ; 1860-7179
ISSN (online)	1860-7187
ISSN	1860-7179
DOI	10.1002/cmdc.201300173
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Article ; Online: Care delivery and self-management strategies for children with epilepsy.

Fleeman, Nigel / Bradley, Peter M / Panebianco, Mariangela / Sharma, Anika

The Cochrane database of systematic reviews

2022 Volume 4, Page(s) CD006245

Abstract: Background: Epilepsy is a neurological disorder affecting both children and adults. Epileptic seizures are the result of excessive and abnormal cortical cell electrical activity in the brain. In response to criticism that epilepsy care for children has ... ...

Abstract	Background: Epilepsy is a neurological disorder affecting both children and adults. Epileptic seizures are the result of excessive and abnormal cortical cell electrical activity in the brain. In response to criticism that epilepsy care for children has little impact on long-term outcomes, healthcare professionals and administrators have developed various service models and strategies to address perceived inadequacies. This is an updated version of a Cochrane Review previously published in 2018. Objectives: To assess the effects of any specialised or dedicated intervention for epilepsy versus usual care in children and adolescents with epilepsy and their families. Search methods: We searched the following databases on 14 January 2020: the Cochrane Register of Studies (CRS Web), MEDLINE (Ovid, 1946 to 13 January 2020), PsycINFO (1887 to 14 January 2020), CINAHL Plus (1937 to 14 January 2020), ClinicalTrials.gov, and the World Health Organization International Clinical Trials Registry Platform. The Cochrane Register of Studies (CRS Web) includes the Cochrane Epilepsy Group Specialised Register and the Cochrane Central Register of Controlled Trials (CENTRAL). We also contacted experts in the field seeking information on unpublished and ongoing studies and checked the websites of epilepsy organisations and the reference lists of included studies. Selection criteria: We included randomised controlled trials recruiting children and adolescents with epilepsy. Data collection and analysis: Two review authors independently selected trials for inclusion and extracted the relevant data. We assessed the following outcomes: 1. Seizure frequency and severity; 2. Appropriateness and volume of medication prescribed (including evidence of drug toxicity); 3. Participants' reported knowledge of information and advice received from professionals; 4. Participants' reports of health and quality of life; 5. Objective measures of general health status; 6. Objective measures of social or psychological functioning (including the number of days spent on sick leave/absence from school or work, and employment status); and 7. Costs of care or treatment. The results of the data extraction and quality assessment for each study were presented in structured tables and as a narrative summary. All summary statistics were extracted for each outcome. Main results: We included nine studies of eight interventions in the review, reporting on seven distinct self-management programmes for educating or counselling children with epilepsy and their parents, and one new model of care. Based largely on self-reported outcomes, each programme showed some benefits for the well-being of children with epilepsy; however, all of the included studies had methodological flaws. No single programme was evaluated with different study samples, and in no instance was the same outcome measured and reported in the same way across studies, precluding any possible meta-analysis, even if the interventions were considered sufficiently similar to include in meta-analysis. We chose the outcomes for which data might be important for decisions about the interventions as per guidance in the Cochrane Handbook for Systematic Reviews of Interventions. We found moderate certainty evidence that one of the educational interventions reduced seizure frequency. There was low certainty evidence that two other educational interventions reduced seizure severity, seizure control, and seizure cure rates. The evidence for all other outcomes (drug adherence, knowledge, self-efficacy and self-perception of epilepsy on quality of life) was mixed. Authors' conclusions: Whilst each of the programmes evaluated in this review showed some benefit to children with epilepsy, their impact was extremely variable. No programme showed benefits across the full range of outcomes, and all studies had methodological problems. There is currently insufficient evidence in favour of any single programme. Further evidence from randomised controlled trials using validated measures and considering clinical meaningfulness as well as statistical significance of results is required.
MeSH term(s)	Adolescent ; Adult ; Child ; Epilepsy/psychology ; Epilepsy/therapy ; Humans ; Medication Adherence ; Quality of Life ; Seizures ; Self Care ; Self-Management ; Systematic Reviews as Topic
Language	English
Publishing date	2022-04-27
Publishing country	England
Document type	Journal Article ; Meta-Analysis ; Review ; Research Support, Non-U.S. Gov't ; Systematic Review
ISSN	1469-493X
ISSN (online)	1469-493X
DOI	10.1002/14651858.CD006245.pub5
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Article ; Online: Care delivery and self-management strategies for children with epilepsy.

Fleeman, Nigel / Bradley, Peter M

The Cochrane database of systematic reviews

2018 Volume 3, Page(s) CD006245

Abstract: Background: In response to criticism that epilepsy care for children has little impact, healthcare professionals and administrators have developed various service models and strategies to address perceived inadequacies.: Objectives: To assess the ... ...

Abstract	Background: In response to criticism that epilepsy care for children has little impact, healthcare professionals and administrators have developed various service models and strategies to address perceived inadequacies. Objectives: To assess the effects of any specialised or dedicated intervention for epilepsy versus usual care in children with epilepsy and in their families. Search methods: We searched the Cochrane Epilepsy Group Specialized Register (27 September 2016), the Cochrane Central Register of Controlled Trials (CENTRAL; 2016, Issue 9) in the Cochrane Library, MEDLINE (1946 to 27 September 2016), Embase (1974 to 27 September 2016), PsycINFO (1887 to 27 September 2016) and CINAHL Plus (1937 to 27 September 2016). In addition, we also searched clinical trials registries for ongoing or recently completed trials, contacted experts in the field to seek information on unpublished and ongoing studies, checked the websites of epilepsy organisations and checked the reference lists of included studies. Selection criteria: We included randomised controlled trials (RCTs), cohort studies or other prospective studies with a (matched or unmatched) control group (controlled before-and-after studies), or time series studies. Data collection and analysis: We used standard methodological procedures expected by Cochrane. Main results: Our review included six interventions reported through seven studies (of which five studies were designed as RCTs). They reported on different education and counselling programmes for children and parents; teenagers and parents; or children, adolescents and their parents. Each programme showed some benefits for the well-being of children with epilepsy, but all had methodological flaws (e.g. in one of the studies designed as an RCT, randomisation failed), no single programme was independently evaluated with different study samples and no interventions were sufficiently homogeneous enough to be included in a meta-analysis,. Authors' conclusions: While each of the programmes in this review showed some benefit to children with epilepsy, their impacts were extremely variable. No programme showed benefits across the full range of outcomes, and all studies had major methodological problems. At present there is insufficient evidence in favour of any single programme.
MeSH term(s)	Adaptation, Psychological ; Adolescent ; Child ; Controlled Before-After Studies ; Counseling ; Delivery of Health Care ; Epilepsy/psychology ; Epilepsy/therapy ; Humans ; Parents/education ; Patient Education as Topic/methods ; Randomized Controlled Trials as Topic ; Self Care ; Self Disclosure ; Treatment Outcome
Language	English
Publishing date	2018-03-01
Publishing country	England
Document type	Journal Article ; Research Support, Non-U.S. Gov't ; Review ; Systematic Review
ISSN	1469-493X
ISSN (online)	1469-493X
DOI	10.1002/14651858.CD006245.pub4
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Article ; Online: Correction: Onasemnogene Abeparvovec for Treating Pre-symptomatic Spinal Muscular Atrophy: An External Assessment Group Perspective of the Partial Review of NICE Highly Specialised Technology Evaluation 15.

Chaplin, Marty / Bresnahan, Rebecca / Fleeman, Nigel / Mahon, James / Houten, Rachel / Beale, Sophie / Boland, Angela / Dundar, Yenal / Marsden, Ashley / Munot, Pinki

PharmacoEconomics - open

2023 Volume 7, Issue 6, Page(s) 1015

Language	English
Publishing date	2023-12-13
Publishing country	Switzerland
Document type	Published Erratum
ZDB-ID	2874287-4
ISSN	2509-4254 ; 2509-4262
ISSN (online)	2509-4254
ISSN	2509-4262
DOI	10.1007/s41669-023-00462-7
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Article ; Online: Onasemnogene Abeparvovec for Treating Pre-symptomatic Spinal Muscular Atrophy: An External Assessment Group Perspective of the Partial Review of NICE Highly Specialised Technology Evaluation 15.

Chaplin, Marty / Bresnahan, Rebecca / Fleeman, Nigel / Mahon, James / Houten, Rachel / Beale, Sophie / Boland, Angela / Dundar, Yenal / Marsden, Ashley / Munot, Pinki

PharmacoEconomics - open

2023 Volume 7, Issue 6, Page(s) 863–875

Abstract: As part of the National Institute for Health and Care Excellence (NICE) highly specialised technology (HST) evaluation programme, Novartis submitted evidence to support the use of onasemnogene abeparvovec as a treatment option for patients with pre- ... ...

Abstract	As part of the National Institute for Health and Care Excellence (NICE) highly specialised technology (HST) evaluation programme, Novartis submitted evidence to support the use of onasemnogene abeparvovec as a treatment option for patients with pre-symptomatic 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the survival of motor neuron (SMN) 1 gene and up to three copies of the SMN2 gene. The Liverpool Reviews and Implementation Group at the University of Liverpool was commissioned to act as the External Assessment Group (EAG). This article summarises the EAG's review of the evidence submitted by the company and provides an overview of the NICE Evaluation Committee's final decision, published in April 2023. The primary source of evidence for this evaluation was the SPR1NT trial, a single-arm trial including 29 babies. The EAG and committee considered that the SPR1NT trial results suggested that onasemnogene abeparvovec is effective in treating pre-symptomatic SMA; however, long-term efficacy data were unavailable and efficacy in babies aged over 6 weeks remained uncertain. Cost-effectiveness analyses conducted by the company and the EAG (using a discounted price for onasemnogene abeparvovec) explored various assumptions; all analyses generated incremental cost-effectiveness ratios (ICERs) that were less than £100,000 per quality-adjusted life-year (QALY) gained. The committee recommended onasemnogene abeparvovec as an option for treating pre-symptomatic 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene in babies aged ≤ 12 months only if the company provides it according to the commercial arrangement (i.e. simple discount patient access scheme).
Language	English
Publishing date	2023-09-20
Publishing country	Switzerland
Document type	Journal Article ; Review
ZDB-ID	2874287-4
ISSN	2509-4254 ; 2509-4262
ISSN (online)	2509-4254
ISSN	2509-4262
DOI	10.1007/s41669-023-00439-6
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Article ; Online: Estimated health effects from domestic use of gaseous fuels for cooking and heating in high-income, middle-income, and low-income countries: a systematic review and meta-analyses.

Puzzolo, Elisa / Fleeman, Nigel / Lorenzetti, Federico / Rubinstein, Fernando / Li, Yaojie / Xing, Ran / Shen, Guofeng / Nix, Emily / Maden, Michelle / Bresnahan, Rebecca / Duarte, Rui / Abebe, Lydia / Lewis, Jessica / Williams, Kendra N / Adahir-Rohani, Heather / Pope, Daniel

The Lancet. Respiratory medicine

2024 Volume 12, Issue 4, Page(s) 281–293

Abstract: Background: Exposure to household air pollution from polluting domestic fuel (solid fuel and kerosene) represents a substantial global public health burden and there is an urgent need for rapid transition to clean domestic fuels. Gas for cooking and ... ...

Abstract	Background: Exposure to household air pollution from polluting domestic fuel (solid fuel and kerosene) represents a substantial global public health burden and there is an urgent need for rapid transition to clean domestic fuels. Gas for cooking and heating might possibly affect child asthma, wheezing, and respiratory health. The aim of this review was to synthesise the evidence on the health effects of gaseous fuels to inform policies for scalable clean household energy. Methods: In this systematic review and meta-analysis, we summarised the health effects from cooking or heating with gas compared with polluting fuels (eg, wood or charcoal) and clean energy (eg, electricity and solar energy). We searched PubMed, Scopus, Web of Science, MEDLINE, Cochrane Library (CENTRAL), Environment Complete, GreenFile, Google Scholar, Wanfang DATA, and CNKI for articles published between Dec 16, 2020, and Feb 6, 2021. Studies eligible for inclusion had to compare gas for cooking or heating with polluting fuels (eg, wood or charcoal) or clean energy (eg, electricity or solar energy) and present data for health outcomes in general populations. Studies that reported health outcomes that were exacerbations of existing underlying conditions were excluded. Several of our reviewers were involved in screening studies, data extraction, and quality assessment (including risk of bias) of included studies; 20% of studies were independently screened, extracted and quality assessed by another reviewer. Disagreements were reconciled through discussion with the wider review team. Included studies were appraised for quality using the Liverpool Quality Assessment Tools. Key health outcomes were grouped for meta-analysis and analysed using Cochrane's RevMan software. Primary outcomes were health effects (eg, acute lower respiratory infections) and secondary outcomes were health symptoms (eg, respiratory symptoms such as wheeze, cough, or breathlessness). This study is registered with PROSPERO, CRD42021227092. Findings: 116 studies were included in the meta-analysis (two [2%] randomised controlled trials, 13 [11%] case-control studies, 23 [20%] cohort studies, and 78 [67%] cross-sectional studies), contributing 215 effect estimates for five grouped health outcomes. Compared with polluting fuels, use of gas significantly lowered the risk of pneumonia (OR 0·54, 95% CI 0·38-0·77; p=0·00080), wheeze (OR 0·42, 0·30-0·59; p<0·0001), cough (OR 0·44, 0·32-0·62; p<0·0001), breathlessness (OR 0·40, 0·21-0·76; p=0·0052), chronic obstructive pulmonary disease (OR 0·37, 0·23-0·60; p<0·0001), bronchitis (OR 0·60, 0·43-0·82; p=0·0015), pulmonary function deficit (OR 0·27, 0·17-0·44; p<0·0001), severe respiratory illness or death (OR 0·27, 0·11-0·63; p=0·0024), preterm birth (OR 0·66, 0·45-0·97; p=0·033), and low birth weight (OR 0·70, 0·53-0·93; p=0·015). Non-statistically significant effects were observed for asthma in children (OR 1·04, 0·70-1·55; p=0·84), asthma in adults (OR 0·65, 0·43-1·00; p=0·052), and small for gestational age (OR 1·04, 0·89-1·21; p=0·62). Compared with electricity, use of gas significantly increased risk of pneumonia (OR 1·26, 1·03-1·53; p=0·025) and chronic obstructive pulmonary disease (OR 1·15, 1·06-1·25; p=0·0011), although smaller non-significant effects were observed for higher-quality studies. In addition, a small increased risk of asthma in children was not significant (OR 1·09, 0·99-1·19; p=0·071) and no significant associations were found for adult asthma, wheeze, cough, and breathlessness (p>0·05). A significant decreased risk of bronchitis was observed (OR 0·87, 0·81-0·93; p<0·0001). Interpretation: Switching from polluting fuels to gaseous household fuels could lower health risk and associated morbidity and mortality in resource-poor countries where reliance on polluting fuels is greatest. Although gas fuel use was associated with a slightly higher risk for some health outcomes compared with electricity, gas is an important transitional option for health in countries where access to reliable electricity supply for cooking or heating is not feasible in the near term. Funding: WHO.
MeSH term(s)	Infant, Newborn ; Adult ; Child ; Female ; Humans ; Air Pollution, Indoor/analysis ; Heating/adverse effects ; Cross-Sectional Studies ; Charcoal/analysis ; Premature Birth ; Asthma/epidemiology ; Asthma/etiology ; Pulmonary Disease, Chronic Obstructive ; Cooking ; Dyspnea ; Cough ; Bronchitis ; Pneumonia
Chemical Substances	Charcoal (16291-96-6)
Language	English
Publishing date	2024-02-01
Publishing country	England
Document type	Meta-Analysis ; Systematic Review ; Journal Article
ZDB-ID	2686754-0
ISSN	2213-2619 ; 2213-2600
ISSN (online)	2213-2619
ISSN	2213-2600
DOI	10.1016/S2213-2600(23)00427-7
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Article ; Online: Heated Humidified High-Flow Nasal Cannula for Preterm Infants: An Updated Systematic Review and Meta-analysis.

Fleeman, Nigel / Dundar, Yenal / Shah, Prakesh S / Shaw, Ben Nj

International journal of technology assessment in health care

2019 Volume 35, Issue 4, Page(s) 298–306

Abstract: ... The post-extubation analysis included ten RCTs (n = 1,201), and the analysis of primary respiratory support ... included ten RCTs (n = 1,676). There were no statistically significant differences for outcomes measuring ...

Abstract	Background: Heated humidified high-flow nasal cannula (HHHFNC) is gaining popularity as a mode of respiratory support. We updated a systematic review and meta-analyses examining the efficacy and safety of HHHFNC compared with standard treatments for preterm infants. The primary outcome was the need for reintubation for preterm infants following mechanical ventilation (post-extubation analysis) or need for intubation for preterm infants not previously intubated (analysis of primary respiratory support). Methods: We searched PubMed, MEDLINE, Embase, and the Cochrane Library for randomized controlled trials (RCTs) of HHHFNC versus standard treatments. Meta-analysis was conducted using Review Manager 5.3. Results: The post-extubation analysis included ten RCTs (n = 1,201), and the analysis of primary respiratory support included ten RCTs (n = 1,676). There were no statistically significant differences for outcomes measuring efficacy, including the primary outcome. There were statistically significant differences favoring HHHFNC versus nasal cannula positive airway pressure (NCPAP) for air leak (post-extubation, risk ratio [RR] 0.29, 95 percent confidence interval [CI] 0.11 to 0.76, I2 = 0) and nasal trauma (post-extubation: 0.35, 95 percent CI 0.27 to 0.46, I2 = 5 percent; primary respiratory support: RR 0.52, 95 percent CI 0.37 to 0.74; I2 = 27 percent). Studies, particularly those of primary respiratory support, included very few preterm infants with gestational age (GA) <28 weeks. Conclusions: HHHFNC may offer an efficacious and safe alternative to NCPAP for some infants but evidence is lacking for preterm infants with GA ≤28 weeks.
MeSH term(s)	Cannula ; Continuous Positive Airway Pressure/adverse effects ; Continuous Positive Airway Pressure/methods ; Female ; Gestational Age ; Hot Temperature ; Humans ; Humidity ; Infant, Newborn ; Infant, Premature ; Intubation, Intratracheal/statistics & numerical data ; Male ; Oxygen Inhalation Therapy/adverse effects ; Oxygen Inhalation Therapy/methods ; Randomized Controlled Trials as Topic
Language	English
Publishing date	2019-07-11
Publishing country	England
Document type	Journal Article ; Meta-Analysis ; Systematic Review
ZDB-ID	632573-7
ISSN	1471-6348 ; 0266-4623
ISSN (online)	1471-6348
ISSN	0266-4623
DOI	10.1017/S0266462319000424
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Article ; Online: A systematic review of health state utility values for thyroid cancer.

Houten, Rachel / Fleeman, Nigel / Kotas, Eleanor / Boland, Angela / Lambe, Tosin / Duarte, Rui

Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation

2020 Volume 30, Issue 3, Page(s) 675–702

Abstract: Purpose: Health state utility values are commonly used to inform economic evaluations and determine the cost-effectiveness of an intervention. The aim of this systematic review is to summarise the utility values available to represent the health-related ...

Abstract	Purpose: Health state utility values are commonly used to inform economic evaluations and determine the cost-effectiveness of an intervention. The aim of this systematic review is to summarise the utility values available to represent the health-related quality of life (HRQoL) of patients with thyroid cancer. Methods: Eight electronic databases were searched from January 1999 to April 2019 for studies which included assessment of HRQoL for patients with thyroid cancer. Utility estimates derived from multiple sources (EuroQol questionnaire 5-dimension (EQ-5D), time trade-off [TTO] and standard gamble [SG] methods) were extracted. In addition, utility estimates were generated by mapping from SF-36 and EORTC QLQ-30 to the EQ-5D-3L UK value set using published mapping algorithms. Results: Searches identified 33 eligible studies. Twenty-six studies reported HRQoL for patients with differentiated thyroid cancer and seven studies for patients with general thyroid cancer. We identified studies which used different methods and tools to quantify the HRQoL in patients with thyroid cancer, such as the EQ-5D-3L, SF-36, EORTC QLQ-30 and SG and TTO techniques to estimate utility values. Utility estimates range from 0.205 (patients with low-risk differentiated thyroid cancer) to utility values approximate to the average UK population (following successful thyroidectomy surgery and radioiodine treatment). Utility estimates for different health states, across thyroid cancer sub-types and interventions are presented. Conclusion: A catalogue of utility values is provided for use when carrying out economic modelling of thyroid cancer; by including mapped values, this approach broadens the scope of health states that can be considered within cost-effectiveness modelling.
MeSH term(s)	Female ; Humans ; Male ; Patient Acceptance of Health Care/statistics & numerical data ; Quality of Life/psychology ; Surveys and Questionnaires ; Thyroid Neoplasms/epidemiology
Language	English
Publishing date	2020-10-24
Publishing country	Netherlands
Document type	Journal Article ; Systematic Review
ZDB-ID	1161148-0
ISSN	1573-2649 ; 0962-9343
ISSN (online)	1573-2649
ISSN	0962-9343
DOI	10.1007/s11136-020-02676-2
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Article ; Online: Trastuzumab Deruxtecan for Treating HER2-Positive Unresectable or Metastatic Breast Cancer After Two or More Anti-HER2 Therapies: An Evidence Review Group Perspective of a NICE Single Technology Appraisal.

Houten, Rachel / Fleeman, Nigel / Mahon, James / Chaplin, Marty / Edwards, Katherine / Beale, Sophie / Boland, Angela / Dundar, Yenal / Marsden, Ashley / Malik, Zafar / Palmieri, Carlo

PharmacoEconomics - open

2023 Volume 7, Issue 3, Page(s) 345–358

Abstract: The National Institute for Health and Care Excellence (NICE) provides guidance to improve health and social care in England and Wales. NICE invited Daiichi Sankyo to submit evidence for the use of trastuzumab deruxtecan (T-DXd) for treating human ... ...

Abstract	The National Institute for Health and Care Excellence (NICE) provides guidance to improve health and social care in England and Wales. NICE invited Daiichi Sankyo to submit evidence for the use of trastuzumab deruxtecan (T-DXd) for treating human epidermal growth factor 2 (HER2)-positive unresectable or metastatic breast cancer (UBC/MBC) after two or more anti-HER2 therapies, in accordance with NICE's Single Technology Appraisal process. The Liverpool Reviews and Implementation Group, part of the University of Liverpool, was commissioned to act as the Evidence Review Group (ERG). This article summarises the ERG's review of the evidence submitted by the company and provides an overview of the NICE Appraisal Committee's (AC's) final decision made in May 2021. Results from the company's base-case fully incremental analysis showed that, compared with T-DXd, eribulin and vinorelbine were dominated and the incremental cost-effectiveness ratio (ICER) per quality-adjusted life year (QALY) gained versus capecitabine was £47,230. The ERG scenario analyses generated a range of ICERs, with the highest being a scenario relating to a comparison of T-DXd versus capecitabine (£78,142 per QALY gained). The ERG considered that due to a lack of appropriate clinical effectiveness evidence, the relative effectiveness of T-DXd versus any comparator treatment could not be determined with any degree of certainty. The NICE AC agreed that the modelling of overall survival was highly uncertain and concluded that treatment with T-DXd could not be recommended for routine use within the National Health Service (NHS). T-DXd was, however, recommended for use within the Cancer Drugs Fund, provided Managed Access Agreement conditions were followed.
Language	English
Publishing date	2023-04-21
Publishing country	Switzerland
Document type	Journal Article ; Review
ZDB-ID	2874287-4
ISSN	2509-4254 ; 2509-4262
ISSN (online)	2509-4254
ISSN	2509-4262
DOI	10.1007/s41669-023-00405-2
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Article ; Online: Pegcetacoplan for Treating Paroxysmal Nocturnal Haemoglobinuria: An Evidence Review Group Perspective of a NICE Single Technology Appraisal.

Bresnahan, Rebecca / Houten, Rachel / Greenhalgh, Janette / Nevitt, Sarah / Mahon, James / Beale, Sophie / Boland, Angela / Bhattacharyya, Devarshi / Dundar, Yenal / McEntee, Joanne / Gandhi, Shreyans / Fleeman, Nigel / Chaplin, Marty

PharmacoEconomics - open

2023 Volume 7, Issue 4, Page(s) 525–536

Abstract: As part of the Single Technology Appraisal (STA) process, the UK National Institute for Health and Care Excellence (NICE) invited Apellis Pharmaceuticals/Sobi to submit evidence for the clinical and cost effectiveness of pegcetacoplan versus eculizumab ... ...

Abstract	As part of the Single Technology Appraisal (STA) process, the UK National Institute for Health and Care Excellence (NICE) invited Apellis Pharmaceuticals/Sobi to submit evidence for the clinical and cost effectiveness of pegcetacoplan versus eculizumab and pegcetacoplan versus ravulizumab for treating paroxysmal nocturnal haemoglobinuria (PNH) in adults whose anaemia is uncontrolled after treatment with a C5 inhibitor. The Liverpool Reviews and Implementation Group at the University of Liverpool was commissioned as the Evidence Review Group (ERG). The company pursued a low incremental cost-effectiveness ratio (ICER) Fast Track Appraisal (FTA). This was a form of STA processed in a shorter time frame and designed for technologies with company base-case ICER < £10,000 per quality-adjusted life-year (QALY) gained and most plausible ICER < £20,000 per QALY gained. This article summarises the ERG's review of the company's evidence submission, and the NICE Appraisal Committee's (AC's) final decision. The company presented clinical evidence from the PEGASUS trial that assessed the efficacy of pegcetacoplan versus eculizumab. At Week 16, patients in the pegcetacoplan arm had statistically significantly greater change from baseline in haemoglobin levels and a higher rate of transfusion avoidance than patients in the eculizumab arm. Using the PEGASUS trial and Study 302 data (a non-inferiority trial that assessed ravulizumab versus eculizumab), the company conducted an anchored matching-adjusted indirect comparison (MAIC) to indirectly estimate the efficacy of pegcetacoplan versus ravulizumab. The company identified key differences between trial designs and populations that could not be adjusted for using anchored MAIC methods. The company and ERG agreed that the anchored MAIC results were not robust and should not inform decision making. In the absence of robust indirect estimates, the company assumed that ravulizumab had equivalent efficacy to eculizumab in the PEGASUS trial population. Results from the company base-case cost-effectiveness analysis showed that treatment with pegcetacoplan dominated eculizumab and ravulizumab. The ERG considered that the long-term effectiveness of pegcetacoplan was uncertain and ran a scenario assuming that after 1 year the efficacy of pegcetacoplan would be the same as eculizumab; treatment with pegcetacoplan continued to dominate eculizumab and ravulizumab. The AC noted that treatment with pegcetacoplan had lower total costs than treatment with eculizumab or ravulizumab because it is self-administered and reduces the need for blood transfusions. If the assumption that ravulizumab has equivalent efficacy to eculizumab does not hold, then this will affect the estimate of the cost effectiveness of pegcetacoplan versus ravulizumab; however, the AC was satisfied that the assumption was reasonable. The AC recommended pegcetacoplan as an option for the treatment of PNH in adults who have uncontrolled anaemia despite treatment with a stable dose of a C5 inhibitor for ≥ 3 months. Pegcetacoplan was the first technology recommended by NICE via the low ICER FTA process.
Language	English
Publishing date	2023-05-17
Publishing country	Switzerland
Document type	Journal Article ; Review
ZDB-ID	2874287-4
ISSN	2509-4254 ; 2509-4262
ISSN (online)	2509-4254
ISSN	2509-4262
DOI	10.1007/s41669-023-00408-z
Database	MEDical Literature Analysis and Retrieval System OnLINE

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