Phillips, Bob

White Rose Research Online

This is a repository copy of Towards evidence-based medicine for paediatricians.White Rose Research Online URL for this paper:http://eprints.whiterose.ac.uk/121691/Version: Published VersionArticle:Phillips, Bob orcid.org/0000-0002-4938-9673 (2017) Towards evidence-based medicine forpaediatricians. Archives of Disease in Childhood. p. 988. ISSN 1468-2044 https://doi.org/10.1136/archdischild-2017-313806eprints@whiterose.ac.ukhttps://eprints.whiterose.ac.uk/Reuse This article is distributed under the terms of the Creative Commons Attribution (CC BY) licence. This licence allows you to distribute, remix, tweak, and build upon the work, even commercially, as long as you credit the authors for the original work. More information and the full terms of the licence here: https://creativecommons.org/licenses/ Takedown If you consider content in White Rose Research Online to be in breach of UK law, please notify us by emailing eprints@whiterose.ac.uk including the URL of the record and the reason for the withdrawal request. 988  Arch Dis Child October 2017 Vol 102 No 10Archimedes questions begin with a scenario. This is because the patient, their situation and the question they provoke, is the hub around which the wheel of evidence-based practice revolves. It’s the patient’s situation that triggers the asking of a structured clinical question,1 and the selection of patient-oriented outcomes which are important to the particular patient; this means listening carefully to what this particular family and patient are telling you. The acquisition and appraisal of studies that follow fundamentally link to this, and the end result should be bringing those thoughts back to the patient and discussing what this means for them.That discussion of what the evidence is and how to act on it is the core of shared decision-making.My experience of shared decision-making has evolved over my years of working in paediatric oncology. Most often, I’ve had conversations which are most shared and least guided when we’re talking about symptom-relieving approaches where a variety of near-equally effec-tive options are open. (Next most often it’s in the situation of relapsing disease where a wide range of uncertainty in many dimensions appears.) For a massive range of not-yet-senior clinicians, the realms of non-life-saving interventions are where I would suggest you take a brave step into the world of shared decision-making.Yes, really.What this will mean is to reject evidence based medicine  as a satisfying, brain stretching but ultimately pointless intellectual exer-cise. It means to view listening to your patient and their relatives, to understand and to incorporate their views into your conception of the medical actions that could be undertaken, as a fundamental way of practising medicine. It might be tricky to imagine yourself doing this is some straight-jacketed specialities, but you really can make a differ-ence. Why not take it up with the next young person you meet who doesn’t like their laxative, or family considering ways to maintain the loveliness of their new baby’s soft and uncannulated skin?Bob PhillipsCorrespondence to Dr Bob Phillips, Centre for Reviews and Dissemination, University of York, Heslington, York YO10 5DD, UK;  bob. phillips@ doctors. org. ukCompeting interests None declared.Provenance and peer review Commissioned; internally peer reviewed.Received 26 July 2017Revised 8 August 2017Accepted 14 August 2017 ► http:// dx. doi. org/ 10. 1136/ archdischild- 2017- 313230 ► http:// dx. doi. org/ 10. 1136/ archdischild- 2017- 313217Arch Dis Child 2017;102:988. doi:10.1136/archdischild-2017-313806RefeRenCe 1 Haroon M, Phillips R. ’There is nothing like looking, if you want to find something’ – asking questions and searching for answers – the evidence based approach. Arch Dis Child Educ Pract Ed 2010;95:34–9.Sharing decision-makingArchimedesTowards evidence-based medicine for paediatriciansEdited by Bob Phillipsgroup.bmj.com on September 25, 2017 - Published by http://adc.bmj.com/Downloaded from paediatriciansTowards evidence-based medicine forBob Phillipsdoi: 10.1136/archdischild-2017-3138062017 102: 988 Arch Dis Child  http://adc.bmj.com/content/102/10/988.2Updated information and services can be found at: These include:References #BIBLhttp://adc.bmj.com/content/102/10/988.2This article cites 1 articles, 1 of which you can access for free at: serviceEmail alertingbox at the top right corner of the online article. Receive free email alerts when new articles cite this article. Sign up in theCollectionsTopic Articles on similar topics can be found in the following collections  (268)ADC ArchimedesNoteshttp://group.bmj.com/group/rights-licensing/permissionsTo request permissions go to:http://journals.bmj.com/cgi/reprintformTo order reprints go to:http://group.bmj.com/subscribe/To subscribe to BMJ go to:group.bmj.com on September 25, 2017 - Published by http://adc.bmj.com/Downloaded from 

Towards evidence-based medicine for paediatricians

Bob Phillips

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Archives of Disease in Childhood

Towards evidence based medicine for paediatricians

This is a repository copy of Towards evidence based medicine for paediatricians.White Rose Research Online URL for this paper:http://eprints.whiterose.ac.uk/121690/Version: Published VersionArticle:Phillips, Bob orcid.org/0000-0002-4938-9673 (2017) Towards evidence based medicine forpaediatricians. Archives of Disease in Childhood. p. 780. ISSN 1468-2044 https://doi.org/10.1136/archdischild-2017-313580eprints@whiterose.ac.ukhttps://eprints.whiterose.ac.uk/Reuse This article is distributed under the terms of the Creative Commons Attribution (CC BY) licence. This licence allows you to distribute, remix, tweak, and build upon the work, even commercially, as long as you credit the authors for the original work. More information and the full terms of the licence here: https://creativecommons.org/licenses/ Takedown If you consider content in White Rose Research Online to be in breach of UK law, please notify us by emailing eprints@whiterose.ac.uk including the URL of the record and the reason for the withdrawal request. 780  Arch Dis Child August 2017 Vol 102 No 8Towards evidence based medicine for paediatriciansEdited by Bob PhillipsArchimedesWe’re all well aware of the problems of doing randomised clinical trials in paediatrics but are we as aware of some alternative approaches that have been used?1‘Sequential design’ studies look at comparing a series of treatments against each other, switching to the ‘better’ arm and comparing against the next candidate as time progresses. They need quickly and easily available outcomes and tend to be usable only for short-course treat-ments but they’ve been estimated to reduce sample sizes by about 25%.‘Adaptive design’ begins by pitting all the contenders against each other and dropping off the weakest. It can then roll onwards with the final two into a more ‘traditional’ trial design. Again, this needs pretty rapid outcomes and fairly short-course treatments, but reduces numbers to only half of those needed in repeated traditional design trials.‘Response adapted’ designs are intriguing. These include the with-drawal-enriched design—where everyone gets the treatment to start with, then those who show response are randomised to having placebo or carrying on with therapy. This is tricky with ethics—if you’ve shown it to work can you stop?—and has the risk of carryover effects, and doesn’t work for curative treatments. Another variation is the three-stage design, which starts with a randomisation to treatment/placebo. Stage 2 is taking the non-responders to placebo, and randomising those again to placebo/treatment, and stage 3, where only those who have responded to treatment are taken and randomised to placebo/carry-on (like the withdrawal design). This works well with chronic conditions where therapy is not disease modifying, but changes outcomes for a short period.‘Placebo-phase’ randomisations are appealing for therapies with a good chance of success. These randomise participants to all receive the treatment … the randomisation is about ‘when’ to get it … with variable placebo phases before treatment is commenced. This offers the opportunity to treat every child, and still manage to estimate the effect of a therapy. This does rely on (a) you not requiring immediate disease-altering treatment and (b) the disease process not changing massively over time so the ‘later’ ones have more disease to get rid of than the earlier ones.‘Bayesian approaches’ are the oddest to think about. They start from the principle that we do not have a 50% knowledge about a therapy, but based on adult evidence and possibly prior phase II child studies, we actually believe it’s about 60%–70% (or whatever) effective. The trial analysis then ‘builds’ on this ‘prior knowledge’ to come up with an answer based on the assumptions and the data. This can greatly reduce the amount of people needed in the trial, but can feel very suspect. Bob PhillipsCorrespondence to Dr Bob Phillips, University of York, Centre for Reviews and Dissemination, York YO10 5DD, UK;  bob. phillips@ doctors. org. ukCompeting interests None declared.Provenance and peer review Commissioned; internally peer reviewed.Received 20 June 2017Accepted 21 June 2017 ► http://dx.doi.org/10.1136/archdischild-2017-313107 ► http://dx.doi.org/10.1136/archdischild-2017-313242Arch Dis Child 2017;102:780. doi:10.1136/archdischild-2017-313580REFERENCE 1 Baiardi P, Giaquinto C, Girotto S, et al. TEDDY Network of Excellence.  innovative study design for paediatric clinical trials. Eur J Clin Pharmacol 2011;67(Suppl 1):109–15.Can our children’s trials work better than they do?group.bmj.com on September 25, 2017 - Published by http://adc.bmj.com/Downloaded from paediatriciansTowards evidence based medicine forBob Phillipsdoi: 10.1136/archdischild-2017-3135802017 102: 780 Arch Dis Child  http://adc.bmj.com/content/102/8/780.2Updated information and services can be found at: These include:References #BIBLhttp://adc.bmj.com/content/102/8/780.2This article cites 1 articles, 0 of which you can access for free at: serviceEmail alertingbox at the top right corner of the online article. Receive free email alerts when new articles cite this article. Sign up in theCollectionsTopic Articles on similar topics can be found in the following collections  (268)ADC ArchimedesNoteshttp://group.bmj.com/group/rights-licensing/permissionsTo request permissions go to:http://journals.bmj.com/cgi/reprintformTo order reprints go to:http://group.bmj.com/subscribe/To subscribe to BMJ go to:group.bmj.com on September 25, 2017 - Published by http://adc.bmj.com/Downloaded from 

IN BOX:Archimedes seeks to assist practising clinicians by providing “evidence-based” answers to common questions that are not at the forefront of research but are at the core of practice (format adapted from BestBETS published in the Emergency Medicine Journal). A full description of the format is available online at http://bit.ly/ArchiTemplate.Readers wishing to submit their own questions—with best evidence answers—are encouraged to review those already proposed at www.bestbets.org. If your question still hasn’t been answered, feel free to submit your summary according to the instructions for authors at http://bit.ly/ArchiInstructions Cases and controlsI’ve noticed that there are a fair few phrases in the world where there actual meaning can be unclear or uncertain, or possibly interpreted differently by different folk. Take “maybe later” when used by parent to child: this clearly means “no” to the parent, and “yes but not now” to the child. But the world of science can’t be confused …  can it?Just take a gander through the field of “case control” titled studies and you may find yourself upset to discover it can. Now I am fairly clear that what I mean by case/control is a design where the participants are chosen because they have developed (cases) or haven’t got (controls) the outcome of interest; they died, developed neuroblastoma or had exclusion from school. The analysis then is about finding out if these groups had different levels of exposure to a proposed causative factor, such as blood transfusions, bacon, or X-factor viewing.What is not a case control study is a study where the groups are chosen for the exposure to a treatment or not. This is a comparative cohort study.Now as is so often the case when appraising papers, it doesn’t sometimes matter the phrase the authors have written. It’s what they did that counts – so discount their title if the design doesn’t fit it - and appraise what it is not what it’s labelled as being.the Corresponding Author has the right to grant on behalf of all authors and does grant on behalf of all authors, an exclusive licence (or non exclusive for government employees) on a worldwide basis to the BMJ Publishing Group Ltd and its Licensees to permit this article (if accepted) to be published in Archives of Disease in Childhood editions and any other BMJPGL products to exploit all subsidiary rights, as set out in our licence (http://group.bmj.com/products/journals/instructions-for-authors/licence-forms

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Towards evidence-based medicine for paediatricians

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