1 University of Cambridge School of Clinical Medicine, Cambridge, UK. Common Data Elements in Degenerative Cervical Myelopathy (CODE-DCM): Identifying the Patient and Carer Perspective Colin Munro1, Fahana Akter2, Peter Hutchinson3, Benjamin Davies3, Mark Kotter2,3 1 University of Cambridge School of Clinical Medicine, Cambridge, UK. 2 Welcome Trust and Medical Research Council Stem Cell Institute, Anne Maclaren Laboratory, University of Cambridge, Cambridge, UK. 3 Department of Academic Neurosurgery, University of Cambridge, Cambridge, UK.

Degenerative Cervical Myelopathy (DCM) DCM is a common, progressive disease that: Is caused by degenerative changes in the cervical spine. Severely affects quality of life1. Estimated prevalence of 1% in the over 50s = most common cause of adult spinal cord impairment.

The issues with current DCM research A large proportion of biomedical research is wasted due to mismatch between what patients want and the research that is done3. This is because of a lack of input from patients in setting research outcomes. Variability of outcomes between research studies makes comparisons difficult.

Variability of outcome reporting in DCM studies A large proportion of biomedical research is wasted due to mismatch between what patients want and the research that is done3. This is because of little input from patients in setting research outcomes. Variability of outcomes between research studies makes comparisons difficult.

Common Data Elements in Degenerative Cervical Myelopathy To address variable outcome reporting the COMET Initiative aims to promote the development of Core Outcome Sets for all conditions. Core Outcome Sets are agreed and standardised sets of outcomes; the minimum that should be recorded in clinical studies. This allows studies to be compared, contrasted and combined easily. CODE-DCM is a project aiming to create a Core Outcome Set for Degenerative Cervical Myelopathy. CODE-DCM aims to involve patients in the selection of the core outcomes so that research output corresponds to patient needs.

CODE-DCM: The patient and carer perspective Investigate how DCM affects patients from both patient and carer perspectives. This will guide the outcomes that are chosen for research studies of DCM. Two stage approach: Identify the broad areas in which DCM affects patients Investigate which of these areas are most important to patients

Methods Structured interviews with patients (N=5) and carers (N=4) Reported effects listed and categorised Online DELPHI survey of Myelopathy.org Shortlist of effects and categories Stage 1 Stage 2 Stage 1: identified the areas in which DCM affects a few individuals Stage 2: validates the results of Stage 1 and investigates which areas are most important to patients Details of Stage 1: Using the Delphi process, DCM patients (N=5) and their carers (N=4) were interviewed about the impact of the condition on patients. Results from the systematic reviews1 and patient research priorities2 were disseminated, and further discussion recorded. The groups were merged, results shared and categorised by mutual agreement.

Results - Stage 1 - Reported Effects Reduced grip strength Symptom variability day by day Numbness Depression Difficulty planning life Neck clicking Heavy legs Urinary incontinence Insomnia Neck pain Reduced walking distance Difficulty breathing when lying flat Falls Erectile dysfunction Leg pain Neck stiffness Anxiety Difficult to climb stairs Unable to get out of bed Clumsiness Faecal incontinence Pins and needles Arm pain Muscle weakness Difficulties with social interaction 41 distinct effects were reported by patients and 11 by carers. These were then grouped into 9 categories.

Results - Stage 1 - Categories Patients: Carers: From Stage 1: Patients reported 41 effects of DCM whilst carers reported 11. These effects were categorised into 9 groups: arm/leg function, immobility, pain, psychosocial, sleep, genitourinary issues, sensation, symptom variability and breathing. The proportion of effects, reported by both patients and carers, relating to each category is shown below:

Results - Stage 2 - Round one of DELPHI Results from Stage 1 have been validated in a larger cohort using an online survey of the myelopathy.org community. All DCM effects were reported by at least 10% of patients. Demographics Most common DCM effects Least common DCM effects No. of patients: 244 Fatigue (82.1%) Unable to get out of chair (24.3%) Average age: 57 Reduced grip strength (80.1%) Difficulty breathing when lying flat (19.4%) Male/Female ratio: 1:3 Pins and needles (79.1%) Unable to get out of bed (17.5%) Average mJOA: 11.6 Numbness (78.6%) Faecal incontinence (10.2%) Neck stiffness (77.6%) Erectile dysfunction (10.2%)

Results - Stage 2 - Round one of DELPHI Some additional symptoms were reported by the survey: headaches, allodynia, electric shock sensations (Lhermitte’s sign) and female sexual problems. The results will be fed into round 2 of the DELPHI.

Results - Stage 2 - Patient Priorities We asked respondents to rank the categories of how DCM affects them in order of their impact. The mean rank for pain was the highest, followed by arm/leg function and sensation. 40% of respondents ranked pain first compared with only 15% ranking arm/leg function first. Category Ranking Scores: pain (6.84), arm/leg function (6.16), sensation (5.59), sleep (5.53), immobility (5.52), disease variability (4.79), psychosocial (4.65), genitourinary issues (4.25) and breathing (3.85). 1 having the least impact and 9 the most Scores are calculated by: 10 subtracted by the mean rank

Mismatch between research focus and patient priorities in DCM Whilst 40% of patients ranked pain as having the greatest impact on their lives only 27% of research includes it as an outcome2. Quality of life aspects are also infrequently recorded in studies (29%) despite their importance to patients. Some effects of DCM are not investigated at all: symptom variability, breathing and sleep. Current research is not representing how patients are affected.

Conclusions DCM affects patients in a variety of ways, with differing accounts from patients and carers. Patients consider pain as the most important feature of DCM. There is substantial mismatch between patients’ priorities and conducted research. Symptom variability, breathing and sleep are not currently measured in DCM treatment studies2. Subsequent steps in the CODE-DCM project will increase our understanding of what is important to patients. The results from this project will hopefully give DCM research more of a patient centered focus.

References Oh et al. Comparing Quality of Life in Cervical Spondylotic Myelopathy with Other Chronic Debilitating Diseases Using the SF-36 Survey. World Neurosurgery. 2017. Davies BM et al. Reported Outcome Measures in Degenerative Cervical Myelopathy: A Systematic Review. PloS One. 2016;11(8). Chalmers et al. How to increase value and reduce waste when research priorities are set. Lancet. 2014;383(9912).

If anyone would like to get involved, please visit: Questions? If anyone would like to get involved, please visit: www.myelopathy.org/codedcm