RESUMO
BACKGROUND: Malignant melanoma is the fifth most common cancer in the UK, with rates continuing to rise, resulting in considerable burden to patients and the NHS. OBJECTIVES: The objectives were to evaluate the effectiveness and cost-effectiveness of current and alternative follow-up strategies for stage IA and IB melanoma. REVIEW METHODS: Three systematic reviews were conducted. (1) The effectiveness of surveillance strategies. Outcomes were detection of new primaries, recurrences, metastases and survival. Risk of bias was assessed using the Cochrane Collaboration's Risk-of-Bias 2.0 tool. (2) Prediction models to stratify by risk of recurrence, metastases and survival. Model performance was assessed by study-reported measures of discrimination (e.g. D-statistic, Harrel's c-statistic), calibration (e.g. the Hosmer-Lemeshow 'goodness-of-fit' test) or overall performance (e.g. Brier score, R2). Risk of bias was assessed using the Prediction model Risk Of Bias ASsessment Tool (PROBAST). (3) Diagnostic test accuracy of fine-needle biopsy and ultrasonography. Outcomes were detection of new primaries, recurrences, metastases and overall survival. Risk of bias was assessed using the Quality Assessment of Diagnostic Accuracy Studies-2 (QUADAS-2) tool. Review data and data from elsewhere were used to model the cost-effectiveness of alternative surveillance strategies and the value of further research. RESULTS: (1) The surveillance review included one randomised controlled trial. There was no evidence of a difference in new primary or recurrence detected (risk ratio 0.75, 95% confidence interval 0.43 to 1.31). Risk of bias was considered to be of some concern. Certainty of the evidence was low. (2) Eleven risk prediction models were identified. Discrimination measures were reported for six models, with the area under the operating curve ranging from 0.59 to 0.88. Three models reported calibration measures, with coefficients of ≥ 0.88. Overall performance was reported by two models. In one, the Brier score was slightly better than the American Joint Committee on Cancer scheme score. The other reported an R2 of 0.47 (95% confidence interval 0.45 to 0.49). All studies were judged to have a high risk of bias. (3) The diagnostic test accuracy review identified two studies. One study considered fine-needle biopsy and the other considered ultrasonography. The sensitivity and specificity for fine-needle biopsy were 0.94 (95% confidence interval 0.90 to 0.97) and 0.95 (95% confidence interval 0.90 to 0.97), respectively. For ultrasonography, sensitivity and specificity were 1.00 (95% confidence interval 0.03 to 1.00) and 0.99 (95% confidence interval 0.96 to 0.99), respectively. For the reference standards and flow and timing domains, the risk of bias was rated as being high for both studies. The cost-effectiveness results suggest that, over a lifetime, less intensive surveillance than recommended by the National Institute for Health and Care Excellence might be worthwhile. There was considerable uncertainty. Improving the diagnostic performance of cancer nurse specialists and introducing a risk prediction tool could be promising. Further research on transition probabilities between different stages of melanoma and on improving diagnostic accuracy would be of most value. LIMITATIONS: Overall, few data of limited quality were available, and these related to earlier versions of the American Joint Committee on Cancer staging. Consequently, there was considerable uncertainty in the economic evaluation. CONCLUSIONS: Despite adoption of rigorous methods, too few data are available to justify changes to the National Institute for Health and Care Excellence recommendations on surveillance. However, alternative strategies warrant further research, specifically on improving estimates of incidence, progression of recurrent disease; diagnostic accuracy and health-related quality of life; developing and evaluating risk stratification tools; and understanding patient preferences. STUDY REGISTRATION: This study is registered as PROSPERO CRD42018086784. FUNDING: This project was funded by the National Institute for Health Research Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol 25, No. 64. See the NIHR Journals Library website for further project information.
Malignant melanoma is the deadliest of skin cancers; in the UK, > 2500 people die from it every year. Initially, the cancer is removed surgically, which cures it for most people, but, for some, the cancer returns. For this reason, after a melanoma is removed, patients are followed up to see if the melanoma reoccurs or if new melanomas have developed. It is felt that early cancer detection improves the chance of future treatment working. A key question is how best to follow up patients after initial melanoma surgery. This study concentrates on the earliest stage of melanoma (American Joint Committee on Cancer stage I), which accounts for more than 7 out of 10 of all melanoma diagnoses. The study also investigates if new ways of follow-up could be at least as good as current practice and a better use of NHS money. We systematically reviewed studies comparing different ways of organising follow-up, and then methods to identify those patients at high risk of developing a further melanoma and how good different tests are at detecting this cancer. We then compared different possible follow-up strategies. For each strategy, we considered its impact on quality and length of life, and how well it used NHS resources. We found little evidence to support a change in how follow-up should be organised currently. There were some ways of organising follow-up that might be better than current care, but further research is needed. We found that new research on whether or not follow-up should be performed by a cancer nurse specialist, rather than a dermatologist or surgeon, would be worthwhile. We also found that more research could be worthwhile on how frequently melanoma recurs and spreads, as well as how accurately a diagnosis of further cancer is made and how to identify those most at risk of further melanoma spread.
Assuntos
Melanoma , Neoplasias Cutâneas , Análise Custo-Benefício , Humanos , Melanoma/diagnóstico , Melanoma/cirurgia , Modelos Econômicos , Qualidade de Vida , Neoplasias Cutâneas/diagnóstico , Neoplasias Cutâneas/cirurgia , UltrassonografiaRESUMO
BACKGROUND: One-fifth of all disabled children have mobility limitations. Early provision of powered mobility for very young children (aged < 5 years) is hypothesised to trigger positive developmental changes. However, the optimum age at which to introduce powered mobility is unknown. OBJECTIVE: The aim of this project was to synthesise existing evidence regarding the effectiveness and cost-effectiveness of powered mobility for very young children, compared with the more common practice of powered mobility provision from the age of 5 years. REVIEW METHODS: The study was planned as a mixed-methods evidence synthesis and economic modelling study. First, evidence relating to the effectiveness, cost-effectiveness, acceptability, feasibility and anticipated outcomes of paediatric powered mobility interventions was reviewed. A convergent mixed-methods evidence synthesis was undertaken using framework synthesis, and a separate qualitative evidence synthesis was undertaken using thematic synthesis. The two syntheses were subsequently compared and contrasted to develop a logic model for evaluating the outcomes of powered mobility interventions for children. Because there were insufficient published data, it was not possible to develop a robust economic model. Instead, a budget impact analysis was conducted to estimate the cost of increased powered mobility provision for very young children, using cost data from publicly available sources. DATA SOURCES: A range of bibliographic databases [Cumulative Index to Nursing and Allied Health Literature (CINHAL), MEDLINE, EMBASE™ (Elsevier, Amsterdam, the Netherlands), Physiotherapy Evidence Database (PEDro), Occupational Therapy Systematic Evaluation of Evidence (OTseeker), Applied Social Sciences Index and Abstracts (ASSIA), PsycINFO, Science Citation Index (SCI; Clarivate Analytics, Philadelphia, PA, USA), Social Sciences Citation Index™ (SSCI; Clarivate Analytics), Conference Proceedings Citation Index - Science (CPCI-S; Clarivate Analytics), Conference Proceedings Citation Index - Social Science & Humanities (CPCI-SSH; Clarivate Analytics), Cochrane Central Register of Controlled Trials (CENTRAL), Cochrane Database of Systematic Reviews, Database of Abstracts of Reviews of Effects (DARE), NHS Economic Evaluation Database (NHS EED), Health Technology Assessment (HTA) Database and OpenGrey] was systematically searched and the included studies were quality appraised. Searches were carried out in June 2018 and updated in October 2019. The date ranges searched covered from 1946 to September 2019. RESULTS: In total, 89 studies were included in the review. Only two randomised controlled trials were identified. The overall quality of the evidence was low. No conclusive evidence was found about the effectiveness or cost-effectiveness of powered mobility in children aged either < 5 or ≥ 5 years. However, strong support was found that powered mobility interventions have a positive impact on children's movement and mobility, and moderate support was found for the impact on children's participation, play and social interactions and on the safety outcome of accidents and pain. 'Fit' between the child, the equipment and the environment was found to be important, as were the outcomes related to a child's independence, freedom and self-expression. The evidence supported two distinct conceptualisations of the primary powered mobility outcome, movement and mobility: the former is 'movement for movement's sake' and the latter destination-focused mobility. Powered mobility should be focused on 'movement for movement's sake' in the first instance. From the budget impact analysis, it was estimated that, annually, the NHS spends £1.89M on the provision of powered mobility for very young children, which is < 2% of total wheelchair service expenditure. LIMITATIONS: The original research question could not be answered because there was a lack of appropriately powered published research. CONCLUSIONS: Early powered mobility is likely to have multiple benefits for very young children, despite the lack of robust evidence to demonstrate this. Age is not the key factor; instead, the focus should be on providing developmentally appropriate interventions and focusing on 'movement for movement's sake'. FUTURE WORK: Future research should focus on developing, implementing, evaluating and comparing different approaches to early powered mobility. STUDY REGISTRATION: This study is registered as PROSPERO CRD42018096449. FUNDING: This project was funded by the National Institute for Health Research (NIHR) Health Technology programme and will be published in full in Health Technology Assessment; Vol. 24, No. 50. See the NIHR Journals Library website for further project information.
The aim of this study was to find out the benefits and costs of providing very young children, aged < 5 years, with powered mobility devices. Examples of powered mobility devices are electrically powered wheelchairs and modified ride-on toys. We looked at many research papers about children and powered mobility. We found many benefits of powered mobility. We then combined all of the information to see if using powered mobility before the age of 5 years had any specific benefits for children. The evidence tells us that powered mobility has a positive effect on children's movement, and it can boost children's social interactions with other people, and their independence. Children using powered mobility were able to go to their friends by themselves, move around a play space as they wanted and take part in physical activities and games. We found that the fit between the child, the powered mobility device and the child's everyday environment was important. When the fit was not good, children experienced a lot of problems. Some children and families felt that powered mobility did not suit their needs, leading to children using a manual wheelchair instead and thereby missing out on education, social opportunities and play. Barriers to powered mobility were found in the physical environment (e.g. inaccessible buildings) and the social environment (e.g. adults supervising children too closely) and often affected children's independence. We found that the advantages and disadvantages of powered mobility were similar in younger and older children, even though the activities they took part in were different. We also found that each year the NHS spends < 2% of its wheelchair service budget on powered mobility for very young children. In conclusion, powered mobility can benefit very young children, but it requires a good fit with the child's environment.