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BACKGROUND: Parkinson's disease (PD) is the second most common neurodegenerative disorder and, according to the Global Burden of Disease estimates in 2015, was the fastest growing neurological disorder globally with respect to associated prevalence, disability, and deaths. Information regarding the awareness, diagnosis, phenotypic characteristics, epidemiology, prevalence, risk factors, treatment, economic impact and lived experiences of people with PD from the African perspective is relatively sparse in contrast to the developed world, and much remains to be learned from, and about, the continent. METHODS: Transforming Parkinson's Care in Africa (TraPCAf) is a multi-faceted, mixed-methods, multi-national research grant. The study design includes multiple sub-studies, combining observational (qualitative and quantitative) approaches for the epidemiological, clinical, risk factor and lived experience components, as appropriate, and interventional methods (clinical trial component). The aim of TraPCAf is to describe and gain a better understanding of the current situation of PD in Africa. The countries included in this National Institute for Health and Care Research (NIHR) Global Health Research Group (Egypt, Ethiopia, Ghana, Kenya, Nigeria, South Africa and Tanzania) represent diverse African geographies and genetic profiles, with differing resources, healthcare systems, health and social protection schemes, and policies. The research team is composed of experts in the field with vast experience in PD, jointly led by a UK-based and Africa-based investigator. DISCUSSION: Despite the increasing prevalence of PD globally, robust data on the disease from Africa are lacking. Existing data point towards the poor awareness of PD and other neurological disorders on the continent and subsequent challenges with stigma, and limited access to affordable services and medication. This multi-site study will be the first of its kind in Africa. The data collected across the proposed sub-studies will provide novel and conclusive insights into the situation of PD. The selected country sites will allow for useful comparisons and make results relevant to other low- and middle-income countries. This grant is timely, as global recognition of PD and the public health challenge it poses builds. The work will contribute to broader initiatives, including the World Health Organization's Intersectoral global action plan on epilepsy and other neurological disorders. TRIAL REGISTRATION: https://doi.org/10.1186/ISRCTN77014546 .
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Saúde Global , Doença de Parkinson , Humanos , Doença de Parkinson/epidemiologia , Doença de Parkinson/terapia , Atenção à Saúde , África do Sul , NigériaRESUMO
BACKGROUND: There is limited evidence on what shapes the acceptability of population level dietary and active-travel policies in England. This information would be useful in the decision-making process about which policies should be implemented and how to increase their effectiveness and sustainability. To fill this gap, we explored public and policymakers' views about factors that influence public acceptability of dietary and active-travel policies and how to increase public acceptability for these policies. METHODS: We conducted online, semi-structured interviews with 20 members of the public and 20 policymakers in England. A purposive sampling frame was used to recruit members of the public via a recruitment agency, based on age, sex, socioeconomic status and ethnicity. Policymakers were recruited from existing contacts within our research collaborations and via snowball sampling. We explored different dietary and active-travel policies that varied in their scope and focus. Interviews were transcribed verbatim and analysed using thematic reflexive analysis with both inductive and deductive coding. RESULTS: We identified four themes that informed public acceptability of dietary and active-travel policies: (1) perceived policy effectiveness, i.e., policies that included believable mechanisms of action, addressed valued co-benefits and barriers to engage in the behaviour; (2) perceived policy fairness, i.e., policies that provided everyone with an opportunity to benefit (mentioned only by the public), equally considered the needs of various population subgroups and rewarded 'healthy' behaviours rather than only penalising 'unhealthy' behaviours; (3) communication of policies, i.e., policies that were visible and had consistent and positive messages from the media (mentioned only by policymakers) and (4) how to improve policy support, with the main suggestion being an integrated strategy addressing multiple aspects of these behaviours, inclusive policies that consider everyone's needs and use of appropriate channels and messages in policy communication. CONCLUSIONS: Our findings highlight that members' of the public and policymakers' support for dietary and active-travel policies can be shaped by the perceived effectiveness, fairness and communication of policies and provide suggestions on how to improve policy support. This information can inform the design of acceptable policies but can also be used to help communicate existing and future policies to maximise their adoption and sustainability.
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Dieta , Política de Saúde , Humanos , Pesquisa Qualitativa , Formulação de Políticas , ComunicaçãoRESUMO
Direct posterior dental restorations are commonly provided following management of dental caries. Amalgam use has been phased down and the feasibility of a phase-out by 2030 is being explored. Alternative direct restorative materials differ in their outcomes and provision. This research aimed to elicit the UK population's preferences for different attributes of restorations and their willingness to pay (WTP) for restorative services and outcomes. A discrete choice experiment (DCE) was designed with patient and public involvement and distributed to a representative sample of the UK general population using an online survey. Respondents answered 17 choice tasks between pairs of scenarios that varied in levels of 7 attributes (wait for filling, clinician type, filling color, length of procedure, likely discomfort after filling, average life span of filling, and cost). An opt-out (no treatment) was included. Mixed logit models were used for data analysis. Marginal WTP for attribute levels and relative attribute importance were calculated. In total, 1,002 respondents completed the DCE. Overall, respondents were willing to pay £39.52 to reduce a 6-wk wait for treatment to 2 wk, £13.55 to have treatment by a dentist rather than a therapist, £41.66 to change filling color from silvery/gray to white, £0.27 per minute of reduced treatment time, £116.52 to move from persistent to no postoperative pain, and £5.44 per year of increased restoration longevity. Ability to pay affected willingness to pay, with low-income respondents more likely to opt out of treatment and value restoration color (white) and increased longevity significantly lower than those with higher income. Clinicians should understand potential drivers of restoration choice, so they can be discussed with individual patients to obtain consent. It is important that policy makers consider general population preferences for restorative outcomes and services, with an awareness of how income affects these, when considering the potential phase-out of amalgam restorations.
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Cárie Dentária , Humanos , Inquéritos e Questionários , Assistência Odontológica , Amálgama Dentário , RendaRESUMO
AIMS: Many parents in contact with children's social care services misuse alcohol however do not meet the threshold for specialist alcohol treatment, and typically do not receive appropriate support for their needs. Brief alcohol interventions have been found to be effective in healthcare settings, however, it is unknown whether the brief intervention structure delivered within health settings would transfer well into children's social care. This paper aims to examine the characteristics of brief intervention for alcohol misusing parents which social care practitioners consider to be important and acceptable to implement in this sector. METHODS: We assessed preferences for, and acceptability of, brief alcohol intervention with parents in contact with children's social care using a discrete choice experiment. We recruited 205 children's social care practitioners from London and the North East of England. Data were analysed using mixed logit which accounted for repeated responses. FINDINGS: Six attributes showed statistically significant coefficients, suggesting that a brief intervention with these attributes would encourage implementation. These were: level of alcohol-related risk targeted; intervention recipient; timing of intervention; duration of sessions; number of sessions and intervention structure. The attribute of most importance identified based on the attribute with the largest coefficient in the conditional logit model was risk level. CONCLUSIONS: Brief alcohol interventions delivered to parents in social care should focus on the impact upon children and the wider family, they should be a flexible part of on-going casework and should be more intensive and less structured.
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Intervenção em Crise , Pais , Consumo de Bebidas Alcoólicas/prevenção & controle , Criança , Inglaterra , Humanos , Londres , Apoio SocialRESUMO
INTRODUCTION: A European Union amalgam phase-down has recently been implemented. Publicly funded health care predominates in the United Kingdom with the system favoring amalgam use. The current use of amalgam and its alternatives has not been fully investigated in the United Kingdom. OBJECTIVES: The study aimed to identify direct posterior restorative techniques, material use, and reported postoperative complication incidence experienced by primary care clinicians and differences between clinician groups. METHODS: A cross-sectional survey was distributed to primary care clinicians through British dentist and therapist associations (11,092 invitations). The questionnaire sought information on current provision of direct posterior restorations and perceived issues with the different materials. Descriptive statistical and hypothesis testing was performed. RESULTS: Dentists' response rate was 14% and therapists' estimated minimum response rate was 6% (total N = 1,513). The most commonly used restorative material was amalgam in molar teeth and composite in premolars. When placing a direct posterior mesio-occluso-distal restoration, clinicians booked on average 45% more time and charged 45% more when placing composite compared to amalgam (P < 0.0001). The reported incidences of food packing and sensitivity following the placement of direct restorations were much higher with composite than amalgam (P < 0.0001). Widely recommended techniques, such as sectional metal matrix use for posterior composites, were associated with reduced food packing (P < 0.0001) but increased time booked (P = 0.002). CONCLUSION: Amalgam use is currently high in the publicly funded sector of UK primary care. Composite is the most used alternative, but it takes longer to place and is more costly. Composite also has a higher reported incidence of postoperative complications than amalgam, but time-consuming techniques, such as sectional matrix use, can mitigate against food packing, but their use is low. Therefore, major changes in health service structure and funding and posterior composite education are required in the United Kingdom and other countries where amalgam use is prevalent, as the amalgam phase-down continues. KNOWLEDGE TRANSFER STATEMENT: This study presents data on the current provision of amalgam for posterior tooth restoration and its directly placed alternatives by primary care clinicians in the United Kingdom, where publicly funded health care with copayment provision predominates. The information is important to manage and plan the UK phase-down and proposed phase-out of amalgam and will be of interest to other, primarily developing countries where amalgam provision predominates in understanding some of the challenges faced.
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Resinas Compostas , Restauração Dentária Permanente , Resinas Compostas/uso terapêutico , Estudos Transversais , Amálgama Dentário/efeitos adversos , Materiais Dentários/química , Restauração Dentária Permanente/métodosRESUMO
INTRODUCTION: Amalgam use has recently been phased down, and the potential for a phase-out is being investigated. OBJECTIVES: The study aimed to identify knowledge of the phase-down and opinions of a potential phase-out of amalgam by UK primary care clinicians and assess their confidence in using different materials in different situations. METHODS: An anonymized, prepiloted cross-sectional e-survey was used to assess primary care clinicians' knowledge and opinions of the amalgam phase-down and potential phase-out and their confidence in using amalgam and the alternatives in different situations. In total, 11,902 invitations were distributed through British dentist and therapist associations. Prior hypotheses were tested alongside descriptive statistics. RESULTS: Response rate was 13% (n = 1,513). Knowledge of the amalgam phase-down was low, with just 3% clinicians correctly identifying all patient groups in whom amalgam use should be avoided in the United Kingdom. Postgraduate education on posterior composite placement was high (88%), but a large majority had personal and patient-centered concerns over the suitability of the alternatives and lacked confidence when placing composite in comparison to amalgam in difficult situations (P < 0.0001). Logistic regressions revealed that the best predictors of high confidence in placing mesio-occluso-distal composites and composites in difficult situations were being a private general dentist or being primarily a composite user. CONCLUSION: Primary care clinicians have major personal and patient-centered concerns regarding the amalgam phase-down (of which they have limited knowledge) and potential phase-out. Many lack confidence in using the alternative, composite, to restore posterior teeth in difficult situations, whereas confidence in using amalgam in similar situations is high. Effective education of clinicians and understanding patients' needs, alongside policy changes, are required to enable a successful amalgam phase-down and potential phase-out. KNOWLEDGE TRANSFER STATEMENT: This study shows that UK primary care clinicians are worried about the phase-down of amalgam for themselves and their patients. Many lack confidence in the alternative, composite, when used in difficult situations, which is in stark contrast to amalgam. Knowledge of the phase-down is limited. There is a need for more effective education of clinicians, an understanding of patients' values, and policy changes to ensure the success of the phase-down and potential phase-out of amalgam.
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Resinas Compostas , Amálgama Dentário , Estudos Transversais , Amálgama Dentário/uso terapêutico , Humanos , Reino UnidoRESUMO
BACKGROUND: This study aims to compare patient preference for, and subsequent change in, oral health behaviour for three forms of risk information given at dental check-ups (verbal advice compared to verbal advice accompanied by a traffic light (TL) risk card; or compared to verbal advice with a quantitative light fluorescence (QLF) photograph of the patient's mouth). METHODS: A multi-centre, parallel-group, patient-randomised clinical trial was undertaken between August 2015 and September 2016. Computer-generated random numbers using block stratification allocated patients to three arms. The setting was four English NHS dental practices. Participants were 412 dentate adults at medium/high risk of poor oral health. Patients rated preference and willingness to pay (WTP) for the three types of information. The primary outcome was WTP. After receiving their check-up, patients received the type of information according to their group allocation. Follow-up was by telephone/e-mail at 6 and 12 months. Mean and median WTP for the three arms were compared using Wilcoxon signed-rank tests. Tobit regression models were used to investigate factors affecting WTP and preference for information type. Secondary outcomes included self-rated oral health and change in oral health behaviours (tooth-brushing, sugar consumption and smoking) and were investigated using multivariate generalised linear mixed models. RESULTS: A total of 412 patients were randomised (138 to verbal, 134 to TL and 140 to QLF); 391 revisited their WTP scores after the check-up (23 withdrew). Follow-up data were obtained for 185 (46%) participants at 6 months and 153 (38%) participants at 12 months. Verbal advice was the first preference for 51% (209 participants), QLF for 35% (145 participants) and TL for 14% (58 participants). TL information was valued lower than either verbal or QLF information (p < 0.0001). Practice attended was predictive of verbal as first preference, and being older. Practice attended, preferring TL the most and having fewer than 20 teeth were associated with increased WTP; and living in a relatively deprived area or having low literacy decreased WTP. There were no significant differences in behaviour change on follow-up. CONCLUSIONS: Although a new NHS dental contract based on TL risk stratification is being tested, patients prefer the usual verbal advice. There was also a practice effect which will needs to be considered for successful implementation of this government policy. TRIAL REGISTRATION: ISRCTN, ISRCTN71242343. Retrospectively registered on 27 March 2018.
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Assistência Odontológica/organização & administração , Implementação de Plano de Saúde , Saúde Bucal , Educação de Pacientes como Assunto/métodos , Preferência do Paciente , Adolescente , Adulto , Idoso , Assistência Odontológica/normas , Feminino , Política de Saúde , Humanos , Masculino , Pessoa de Meia-Idade , Educação de Pacientes como Assunto/organização & administração , Educação de Pacientes como Assunto/normas , Medicina Estatal/normas , Reino Unido , Adulto JovemRESUMO
BACKGROUND: Following primary breast cancer treatment, the early detection of ipsilateral breast tumour recurrence (IBTR) or ipsilateral secondary cancer in the treated breast and detection of new primary cancers in the contralateral breast is beneficial for survival. Surveillance mammography is used to detect these cancers, but the optimal frequency of surveillance and the length of follow-up are unclear. OBJECTIVES: To identify feasible management strategies for surveillance and follow-up of women after treatment for primary breast cancer in a UK setting, and to determine the effectiveness and cost-effectiveness of differing regimens. METHODS: A survey of UK breast surgeons and radiologists to identify current surveillance mammography regimens and inform feasible alternatives; two discrete systematic reviews of evidence published from 1990 to mid 2009 to determine (i) the clinical effectiveness and cost-effectiveness of differing surveillance mammography regimens for patient health outcomes and (ii) the test performance of surveillance mammography in the detection of IBTR and metachronous contralateral breast cancer (MCBC); statistical analysis of individual patient data (West Midlands Cancer Intelligence Unit Breast Cancer Registry and Edinburgh data sets); and economic modelling using the systematic reviews results, existing data sets, and focused searches for specific data analysis to determine the effectiveness and cost-utility of differing surveillance regimens. RESULTS: The majority of survey respondents initiate surveillance mammography 12 months after breast-conserving surgery (BCS) (87%) or mastectomy (79%). Annual surveillance mammography was most commonly reported for women after BCS or after mastectomy (72% and 53%, respectively). Most (74%) discharge women from surveillance mammography, most frequently 10 years after surgery. The majority (82%) discharge from clinical follow-up, most frequently at 5 years. Combining initiation, frequency and duration of surveillance mammography resulted in 54 differing surveillance regimens for women after BCS and 56 for women following mastectomy. The eight studies included in the clinical effectiveness systematic review suggest surveillance mammography offers a survival benefit compared with a surveillance regimen that does not include surveillance mammography. Nine studies were included in the test performance systematic review. For routine IBTR detection, surveillance mammography sensitivity ranged from 64% to 67% and specificity ranged from 85% to 97%. For magnetic resonance imaging (MRI), sensitivity ranged from 86% to 100% and specificity was 93%. For non-routine IBTR detection, sensitivity and specificity for surveillance mammography ranged from 50% to 83% and from 57% to 75%, respectively, and for MRI from 93% to 100% and from 88% to 96%, respectively. For routine MCBC detection, one study reported sensitivity of 67% and specificity of 50% for both surveillance mammography and MRI, although this was a highly select population. Data set analysis showed that IBTR has an adverse effect on survival. Furthermore, women experiencing a second tumour measuring >20 mm in diameter were at a significantly greater risk of death than those with no recurrence or those whose tumour was <10 mm in diameter. In the base-case analysis, the strategy with the highest net benefit, and most likely to be considered cost-effective, was surveillance mammography alone, provided every 12 months at a societal willingness to pay for a quality-adjusted life-year of either £20,000 or £30,000. The incremental cost-effectiveness ratio for surveillance mammography alone every 12 months compared with no surveillance was £4727. LIMITATIONS: Few studies met the review inclusion criteria and none of the studies was a randomised controlled trial. The limited and variable nature of the data available precluded any quantitative analysis. There was no useable evidence contained in the Breast Cancer Registry database to assess the effectiveness of surveillance mammography directly. The results of the economic model should be considered exploratory and interpreted with caution given the paucity of data available to inform the economic model. CONCLUSIONS: Surveillance is likely to improve survival and patients should gain maximum benefit through optimal use of resources, with those women with a greater likelihood of developing IBTR or MCBC being offered more comprehensive and more frequent surveillance. Further evidence is required to make a robust and informed judgement on the effectiveness of surveillance mammography and follow-up. The utility of national data sets could be improved and there is a need for high-quality, direct head-to-head studies comparing the diagnostic accuracy of tests used in the surveillance population. FUNDING: The National Institute for Health Research Health Technology Assessment programme.
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Neoplasias da Mama/diagnóstico , Análise Custo-Benefício , Mamografia/economia , Adulto , Neoplasias da Mama/economia , Neoplasias da Mama/epidemiologia , Bases de Dados Factuais , Feminino , Pesquisas sobre Atenção à Saúde , Humanos , Incidência , Imageamento por Ressonância Magnética/economia , Mamografia/métodos , Mamografia/estatística & dados numéricos , Pessoa de Meia-Idade , Recidiva Local de Neoplasia , Prevalência , Anos de Vida Ajustados por Qualidade de Vida , Sistema de Registros , Fatores de Risco , Sensibilidade e Especificidade , Análise de Sobrevida , Ultrassonografia/economia , Reino Unido/epidemiologiaRESUMO
OBJECTIVES: To assess the clinical effectiveness and cost-effectiveness of non-surgical treatments for women with stress urinary incontinence (SUI) through systematic review and economic modelling. DATA SOURCES: The Cochrane Incontinence Group Specialised Register, electronic databases and the websites of relevant professional organisations and manufacturers, and the following databases: CINAHL, EMBASE, BIOSIS, Science Citation Index and Social Science Citation Index, Current Controlled Trials, ClinicalTrials.gov and the UKCRN Portfolio Database. STUDY SELECTION: The study comprised three distinct elements. (1) A survey of 188 women with SUI to identify outcomes of importance to them (activities of daily living; sex, hygiene and lifestyle issues; emotional health; and the availability of services). (2) A systematic review and meta-analysis of non-surgical treatments for SUI to find out which are most effective by comparing results of trials (direct pairwise comparisons) and by modelling results (mixed-treatment comparisons - MTCs). A total of 88 randomised controlled trials (RCTs) and quasi-RCTs reporting data from 9721 women were identified, considering five generic interventions [pelvic floor muscle training (PFMT), electrical stimulation (ES), vaginal cones (VCs), bladder training (BT) and serotonin-noradrenaline reuptake inhibitor (SNRI) medications], in many variations and combinations. Data were available for 37 interventions and 68 treatment comparisons by direct pairwise assessment. Mixed-treatment comparison models compared 14 interventions, using data from 55 trials (6608 women). (3) Economic modelling, using a Markov model, to find out which combinations of treatments (treatment pathways) are most cost-effective for SUI. DATA EXTRACTION: Titles and abstracts identified were assessed by one reviewer and full-text copies of all potentially relevant reports independently assessed by two reviewers. Any disagreements were resolved by consensus or arbitration by a third person. RESULTS: Direct pairwise comparison and MTC analysis showed that the treatments were more effective than no treatment. Delivering PFMT in a more intense fashion, either through extra sessions or with biofeedback (BF), appeared to be the most effective treatment [PFMT extra sessions vs no treatment (NT) odds ratio (OR) 10.7, 95% credible interval (CrI) 5.03 to 26.2; PFMT + BF vs NT OR 12.3, 95% CrI 5.35 to 32.7]. Only when success was measured in terms of improvement was there evidence that basic PFMT was better than no treatment (PFMT basic vs NT OR 4.47, 95% CrI 2.03 to 11.9). Analysis of cost-effectiveness showed that for cure rates, the strategy using lifestyle changes and PFMT with extra sessions followed by tension-free vaginal tape (TVT) (lifestyle advice-PFMT extra sessions-TVT) had a probability of greater than 70% of being considered cost-effective for all threshold values for willingness to pay for a QALY up to 50,000 pounds. For improvement rates, lifestyle advice-PFMT extra sessions-TVT had a probability of greater than 50% of being considered cost-effective when society's willingness to pay for an additional QALY was more than 10,000 pounds. The results were most sensitive to changes in the long-term performance of PFMT and also in the relative effectiveness of basic PFMT and PFMT with extra sessions. LIMITATIONS: Although a large number of studies were identified, few data were available for most comparisons and long-term data were sparse. Challenges for evidence synthesis were the lack of consensus on the most appropriate method for assessing incontinence and intervention protocols that were complex and varied considerably across studies. CONCLUSIONS: More intensive forms of PFMT appear worthwhile, but further research is required to define an optimal form of more intensive therapy that is feasible and efficient for the NHS to provide, along with further definitive evidence from large, well-designed studies.
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Modelos Econômicos , Incontinência Urinária por Estresse/terapia , Inibidores da Captação Adrenérgica/economia , Inibidores da Captação Adrenérgica/uso terapêutico , Biorretroalimentação Psicológica , Análise Custo-Benefício , Terapia por Estimulação Elétrica/economia , Terapia por Exercício/economia , Terapia por Exercício/métodos , Feminino , Humanos , Estilo de Vida , Cadeias de Markov , Diafragma da Pelve/fisiologia , Anos de Vida Ajustados por Qualidade de Vida , Fatores de Risco , Inibidores Seletivos de Recaptação de Serotonina/economia , Inibidores Seletivos de Recaptação de Serotonina/uso terapêutico , Estresse Psicológico/etiologia , Slings Suburetrais/economia , Resultado do Tratamento , Reino Unido/epidemiologia , Incontinência Urinária por Estresse/economia , Incontinência Urinária por Estresse/epidemiologia , Incontinência Urinária por Estresse/psicologiaRESUMO
This paper presents a summary of the evidence review group (ERG) report into the clinical and cost-effectiveness of romiplostim for the treatment of adults with chronic immune or idiopathic thrombocytopenic purpura (ITP) based upon a review of the manufacturer's submission to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal (STA) process. The submission's evidence came from two relatively high-quality randomised controlled trials (RCTs). The ERG found no evidence that any important data were missed or that data extraction was inaccurate. In both RCTs more patients in the romiplostim than in the placebo group achieved a durable platelet response [non-splenectomised patients: romiplostim 25/41 (61%), placebo 1/21 (5%), odds ratio (OR) 24.45, 95% confidence interval (CI) 3.34 to 179.18; splenectomised patients: romiplostim 16/42 (38%), placebo 0/21 (0%), OR 8.5 (95% CI 1.15 to 372)] and an overall platelet response [non-splenectomised patients: romiplostim 36/41 (88%), placebo 3/21 (14%), OR 34.74, 95% CI 7.77 to 155.38; splenectomised patients: romiplostim 33/42 (79%), placebo 0/21 (0%), OR 16.6 (95% CI 2.37 to 706]. The difference in mean period with a platelet response was 13.9 weeks (95% CI 10.5 to 17.4) in favour of romiplostim in the RCT of non-splectomised patients and 12.1 weeks (95% CI 8.7 to 15.6) in favour of romiplostim in the RCT of splectomised patients. The manufacturer's economic model evaluated the cost-effectiveness of romiplostim compared with standard care. The ERG had concerns about the way the decision problem was addressed in the economic model and about the non-adjustment of findings for confounding factors. In non-splenectomised patients, using romiplostim as a first option treatment, the base-case incremental cost-effectiveness ratio (ICER) was 14,840 pounds per quality-adjusted life-year (QALY). In splenectomised patients the ICER was 14,655 pounds per QALY. Additional sensitivity analyses performed by the ERG identified two issues of importance: whether individuals entered the model on watch and rescue or on active therapy in the comparator arm (ICER 21,674 pounds per QALY for non-splenectomised patients, 29,771 pounds per QALY for splenectomised patients); whether it was assumed that any unused medicine would be wasted. Combining all of the separate sensitivity analyses, and assuming that watch and rescue was not the first-line treatment, increased the ICERs further (non-splenectomised 37,290 pounds per QALY; splenectomised 131,017 pounds per QALY). In conclusion, the manufacturer's submission and additional work conducted by the ERG suggest that romiplostim has short-term efficacy for the treatment of ITP, but there is no robust evidence on long-term effectiveness or cost-effectiveness of romiplostim compared with relevant comparators.
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Imunossupressores/economia , Imunossupressores/uso terapêutico , Púrpura Trombocitopênica Idiopática/tratamento farmacológico , Receptores Fc/uso terapêutico , Proteínas Recombinantes de Fusão/economia , Proteínas Recombinantes de Fusão/uso terapêutico , Trombopoetina/economia , Trombopoetina/uso terapêutico , Pesquisa Comparativa da Efetividade , Análise Custo-Benefício , Humanos , Púrpura Trombocitopênica Idiopática/imunologia , Anos de Vida Ajustados por Qualidade de Vida , Ensaios Clínicos Controlados Aleatórios como Assunto , EsplenectomiaRESUMO
OBJECTIVE: To provide evidence on outcomes of importance to women who have stress urinary incontinence (SUI). The secondary aim was to identify additional outcomes that ought to be collected in future primary studies or in systematic reviews of the literature. DESIGN: Questionnaire survey of a cohort of women with SUI. SETTING: UK. SAMPLE: A total of 188 women with SUI. METHODS: Areas of importance to women who suffer from SUI were assessed using a patient generated index (PGI). In addition to the PGI, the questionnaire included the King's Health Questionnaire (KHQ) and the EuroQol-5D (EQ-5D). MAIN OUTCOME MEASURES: PGI, EQ-5D and the KHQ. RESULTS: In total, 38 different areas were reported by respondents on the PGI. PGI and EQ-5D scores were positively correlated and significant. Correlations between the seven domains of the KHQ and PGI were all negative, but only two were statistically significant: personal relationships and severity measures. CONCLUSIONS: The PGI succeeded in capturing a diverse range of outcomes of importance to women suffering with SUI. Given the limited correlation between the KHQ domains and the PGI and, in addition, that the areas mentioned in the PGI were not found to map well to the EQ-5D, the PGI in this instance may be capturing concerns of women who suffer from SUI, which are not captured by quality-of-life measures such as the EQ-5D.