Title: Research Assessment Outcome Measures for Malignant Bowel Obstruction (RAMBO)
Funder: Marie Curie

Abstract: Background: Malignant bowel obstruction (MBO) affects between 10-50% of ovarian and 15% of gastrointestinal cancers. It one of the most distressing complications of cancer; causing symptoms which include nausea, vomiting, abdominal distension, colic, pain and constipation. It often requires hospitalisation for persistent unresolved symptoms. MBO reduces quality of life. Non-surgical palliative approaches to symptom control usually involve a combination of antiemetics, acid suppression and/or parenteral antisecretory agents (anticholinergics or somatostatin analogues), with gut decompression (nasogastric or gastrostomy tube). Current evidence is inadequate; clinical guidelines favour somatostatin analogues but recommendations are based largely on case series and inadequately-powered clinical trials. Recently two adequately powered, well-conducted clinical trials demonstrated no objective benefit and one suggested evidence of harm. Importantly, these trials included a placebo arm to account for the natural history of MBO, which is largely unknown except that spontaneous resolution with recurrence is common. However, these trials do not appear to have impacted on subsequent guidelines or clinical recommendations. A recent systematic review of somatostatin analogues, using objective tools to evaluate data quality, concluded that there was moderate evidence of no benefit, and only poor evidence for their use. The authors acknowledged the challenges of evaluating current data given the varied clinical outcomes used and the lack of an agreed core outcome set for consistent clinical and research evaluation of MBO. Most importantly, there are no data to indicate that the current commonly used outcome measures reflect what is important to patients and carers, and the benefit they consider to be clinically meaningful. This issue was first highlighted in 2007 but has not been addressed; attention is long overdue. Aim/ Research Questions 1. To review the current scope of outcome measures used to evaluate MBO though answering the following questions: What outcome measures are currently used to evaluate MBO within the context of clinical studies and practice? What symptoms and effects do patients with MBO, their carers and clinicians consider most important, and what improvement would be considered clinically meaningful? How do currently used outcomes map to those important to patients, carers and clinicians, and, in consequence, what are the gaps and limitations in current practice? 2. To develop a set of core outcomes that is specific to MBO Methods Methodology Systematic review: Databases and the grey literature will be searched using MeSH terms and key words. Results will be screened for inclusion against predefined criteria. Included studies will be appraised for quality and bias, by two independent reviewers, with access to a third for arbitration. A range of evaluation methodologies (e.g. RCTs, case series/reports, qualitative studies) are anticipated for inclusion. Evidence will be synthesised following a thematic approach and where possible combined and compared using meta-analytic techniques. Qualitative interviews will be undertaken with patients who have experienced MBO and their carers and clinicians (palliative care, oncology, primary care, surgery). They will explore their experiences of MBO with a focus on the aspects of the symptoms and changes in them that are most important to them. Thematic analysis will be used to identify the most important components of their experience. Core Outcomes common to both review and interview findings, and all identified from the interviews will go forward to the expert panel. Expert panel/Delphi survey: The data extracted on outcomes will be discussed by an expert panel and fed into a Delphi survey to reach consensus on a set of core outcomes to propose to the Delphi panel to refine and rank in order to achieve consensus. The expert panel and survey will include people with experience of MBO academics, clinicians (palliative care, oncology, primary care, surgeons). Outcomes This review will identify what outcomes are currently used to evaluate MBO. The qualitative study will identify what matters most to patients with MBO and their carers in terms of the target symptoms and degree of change, which is clinically important in response to an intervention. The development of a core outcome set for MBO in palliative care has been registered with COMET and will facilitate consistency and comparison between future studies. This resource would also facilitate assessments of, and responses to, the management of MBO in clinical practice

Lay abstract: Malignant bowel obstruction (MBO) is caused when the cancer causes a blockage in the bowel. It is a common problem faced by patients with advanced cancer. The symptoms are very distressing for patients and their families. These include being sick often, which may contain faeces. People with MBO may feel sick and bloated, have stomach pains and be constipated. MBO stops patients from keeping food or water down making it hard for the person to be cared for at home. An operation is often not possible and treatment aims to help symptoms using injections and/or tubes to drain extra fluid. There have been some studies about how to manage MBO, but they have measured different ways that the patient is affected. This makes it impossible to compare studies and agree on the best treatment. Also, we have no idea if the things we measure are important to the patient and their families, or whether they are just the ones that researchers, doctors or nurses think are important. As an urgent first step, we want to agree a way of measuring MBO symptoms that are important to patients, so treatments can immediately be tested in hospitals, hospices and in patients’ homes. To do this we shall start with what we already know by looking at work that other researchers have done. We shall then ask patients and carers what is most important to them. We will compare this to understand in detail what we might be missing. Finally, we will work with patients, family representatives, researchers and clinicians to agree together on what we should measure. We will use the best techniques to do this work and have registered this proposal with the COMET group (who oversees such work) as a mark of quality.

Research activity codes: 7.3 MANAGEMENT AND DECISION MAKING;7.2 END OF LIFE CARE
Health categories: CANCER AND NEOPLASMS;GENERIC HEALTH RELEVANCE