Dr Morro ML Touray

Background: Despite the established evidence and theoretical advances explaining human judgments under uncertainty, developments of mobile health (mHealth) Clinical Decision Support Systems (CDSS) have not explicitly applied the psychology of decision making to the study of user needs. We report on a user needs approach to develop a prototype of a mHealth CDSS for Parkinson’s Disease (PD), which is theoretically grounded in the psychological literature about expert decision making and judgement under uncertainty. Methods: A suite of user needs studies was conducted in 4 European countries (Greece, Italy, Slovenia, the UK) prior to the development of PD_Manager, a mHealth-based CDSS designed for Parkinson’s Disease, using wireless technology. Study 1 undertook Hierarchical Task Analysis (HTA) including elicitation of user needs, cognitive demands and perceived risks/benefits (ethical considerations) associated with the proposed CDSS, through structured interviews of prescribing clinicians (N=47). Study 2 carried out computational modelling of prescribing clinicians’ (N=12) decision strategies based on social judgment theory. Study 3 was a vignette study of prescribing clinicians’ (N=18) willingness to change treatment based on either self-reported symptoms data, devices-generated symptoms data or combinations of both. Results: Study 1 indicated that system development should move away from the traditional silos of ‘motor’ and ‘non-motor’ symptom evaluations and suggest that presenting data on symptoms according to goal-based domains would be the most beneficial approach, the most important being patients’ overall Quality of Life (QoL). The computational modelling in Study 2 extrapolated different factor combinations when making judgements about different questions. Study 3 indicated that the clinicians were equally likely to change the care plan based on information about the change in the patient’s condition from the patient’s self-report and the wearable devices. 3 Conclusions: Based on our approach, we could formulate the following principles of mHealth design: 1) enabling shared decision making between the clinician, patient and the carer; 2) flexibility that accounts for diagnostic and treatment variation among clinicians; 3) monitoring of information integration from multiple sources. Our approach highlighted the central importance of the patient-clinician relationship in clinical decision making and the relevance of theoretical as opposed to algorithm (technology)-based modelling of human judgment.

Background and aims: Ascites develops in about 90% with advanced cirrhosis; when refractory to medical therapy, standard of care is repeated large volume paracentesis (LVP) with albumin support. Refractory ascites (RA) confers a median life expectancy of six months without liver transplantation (LT). LVP is not an optimal palliative strategy. One alternative is long-term abdominal drains (LTAD), used in advanced malignant ascites, also enabling community management. Our ultimate aim is to improve end of life care (EoLC) in advanced cirrhosis and RA. This feasibility randomised controlled trial (RCT) aimed to resolve uncertainties in designing a definitive RCT. Method: Multicentre feasibility RCT with 1:1 randomisation between standard of care (LVP) vs. LTAD (Rocket Medical) in adults with RA, ineligible for LT. Both arms received prophylactic antibiotics. LTAD were inserted under ultrasound guidance. Community nurses undertook home visits to drain ascites dependent on symptoms; (maximum 6L/week), without albumin support. Follow-up was 12 weeks with home visits every two weeks for the following assessments: clinical, questionnaire based to include quality of life, palliative care needs, carer burden and health economics (HE). Here we report clinical and HE outcomes. Results: Thirty six patients were randomised; 19 LVP (two withdrew, wanting LTAD) and 17 LTAD (one withdrew-insufficient ascites). Mean age (years) LTAD vs. LVP 66 + 10.4 vs. 68 ± 12; predominately male (76% vs. 74%). Participants were well matched at baseline in liver tests and prognostic scores: LTAD vs. LVP (serum bilirubin (μmol/L) 26 ± 15.8 vs. 16 ±10, serum albumin (g/L) 33 ± 4.2 vs. 31 ± 3.3, serum creatinine (mmol/L) 113 ± 46.7 vs.118 ± 53.1; MELD 14 ± 4.6 vs. 16 ± 7.2). One LTAD participant required hospitalisation for repeated LVP. Serum albumin (g/L) in the LTAD arm declined to 29 ± 3.3 at week two, subsequently remaining stable LTAD vs. LVP (29 ± 5.6 vs. 31 ± 5.5). Serum creatinine remained stable in both arms. There were no LTAD related serious adverse reactions. LTAD related adverse reactions included mild cellulitis (n = 4) and small volume leakage around LTAD insertion site (n = 3), all resolving rapidly. Peritonitis was rare, LTAD (possible) n = 1 and LVP n = 2. Overall mortality was 36% (12/33). Mortality and median survival (days in those who died) were 7/16 (44%) vs. 5/17 (29%), 53 days (IQR 43) vs. 61 days (IQR 35) in LTAD vs. LVP respectively. All but one death was liver related. Those in LTAD arm spent ≈20% less time in hospital. All nine alive in the LTAD arm at end of study elected to keep LTAD in. Detailed clinical and HE analysis is underway. Conclusion: Preliminary data from the REDUCe study supports the safety and efficacy of palliative LTAD in RA due to advanced cirrhosis. LTAD allows successful management in the community with reduction in health resource utilisation. Proceeding to a definitive RCT is justified.

Objective: Complex cardiac devices including implantable cardioverter defibrillator (ICD) and cardiac resynchronisation therapy (CRT) devices can safely be implanted as a day case procedure as opposed to overnight stay. We assess how common day case complex device therapy is and the cost implications of more widespread adoption across the UK. Methods: A freedom of information request was sent to all centres performing complex cardiac devices across the UK to assess the adoption of this technique. Cost implications were assessed using Department of Health National Schedule of Reference Costs 2016–2017. Results: 100 UK centres were surveyed, 80% replied. Eighty per cent of UK centres already implant complex cardiac devices as a day case to some extent. 64.06% of centres have a protocol for this. 12.82% of centres do ˂25% of complex devices as a day case. 15.38% do 25%–50% as day case. 17.95% do 50%–75% as day case and 33.33% do ˃75% as day case. There was no relationship between centre volume and the proportion of devices done as a day case as opposed to overnight stay. The cost saving of performing a complex device as a day case as opposed to overnight stay was £412 per ICD, £525 per CRT-pacemaker and £2169 per CRT-defibrillator. Conclusions: Day case complex devices are already widespread across the UK, however, there is scope for increase. An increase in proportion of day case devices could translate to £5 583 265 in savings annually for the National Health Service if all centres performed 75% of devices as a day case.

Background: mhealth, predominantly wearable technology and mobile apps, have been considered in Parkinson’s Disease to provide valuable ecological data between face to face visits and improve monitoring of motor symptoms remotely. Objective: In this study we explore the feasibility of using a technology based mhealth platform comprising a smartphone in combination with a smartwatch and a pair of smart insoles, described in the present study as the PD_manager system, to collect clinically meaningful data. We also explore outcomes and disease related factors which are important determinants to establish feasibility. Finally, we further validate a tremor evaluation method with data collected while patients perform their daily activities. Methods: PD_manager trial was an open label parallel group randomized study. The mheath platform consists of a wristband, a pair of sensor insoles, a smartphone (with dedicated mobile Android apps and a knowledge platform) serving as the cloud backend. The compliance was assessed with statistical analysis and the factors affecting it using appropriate regression analysis. The correlation of the scores of our previous algorithm for tremor evaluation and the respective UPDRS estimations by clinicians were explored. Results: There were 65 of the 75 study participants (87%) who completed the protocol. They used the PD_manager system for a median 11.57 days (Std. dev. 3.15). The regression analysis suggests that the main factor associated with high usage was caregivers’ burden. Motor Aspects of Experiences of Daily Living and patients’ self-rated health status also influence the system’s usage. Our algorithm provided clinically meaningful data for the detection and evaluation of tremor. Conclusions: We found that PD patients, regardless of their demographics and disease characteristics, used the system for 11-14 days. The study further supports that mhealth can be an effective tool for the ecologically valid, passive, unobtrusive monitoring and evaluation of symptoms. Future studies will be required to demonstrate that an mhealth platform can improve disease management and care.

Background: Parkinson’s disease is a degenerative neurological condition causing multiple motor and non-motor symptoms that have a serious adverse effect on quality of life. Management is problematic due to the variable and fluctuating nature of symptoms, often hourly and daily. The PD_Manager mHealth platform aims to provide a continuous feed of data on symptoms to improve clinical understanding of the status of any individual patient and inform care planning. The objectives of this trial are to (1) assess patient (and family carer) perspectives of PD_ Manager regarding comfort, acceptability and ease of use; (2) assess clinician views about the utility of the data generated by PD_Manager for clinical decision making and the acceptability of the system in clinical practice. Methods/design: This trial is an unblinded, parallel, two-group, randomised controlled pilot study. A total of 200 persons with Parkinson’s disease (Hoehn and Yahr stage 3, experiencing motor fluctuations at least 2 h per day), with primary family carers, in three countries (110 Rome, 50 Venice, Italy; 20 each in Ioannina, Greece and Surrey, England) will be recruited. Following informed consent, baseline information will be gathered, including the following: age, gender, education, attitudes to technology (patient and carer); time since Parkinson’s diagnosis, symptom status and comorbidities (patient only). Randomisation will assign participants (1:1 in each country), to PD_Manager vs control, stratifying by age (1 ≤ 70 : 1 > 70) and gender (60% M: 40% F). The PD_Manager system captures continuous data on motor symptoms, sleep, activity, speech quality and emotional state using wearable devices (wristband, insoles) and a smartphone (with apps) for storing and transmitting the information. Control group participants will be asked to keep a symptom diary covering the same elements as PD_Manager records. After a minimum of two weeks, each participant will attend a consultation with a specialist doctor for review of the data gathered (by either means), and changes to management will be initiated as indicated. Patients, carers and clinicians will be asked for feedback on the acceptability and utility of the data collection methods. The PD_ Manager intervention, compared to a symptom diary, will be evaluated in a cost-consequences framework Discussion: Information gathered will inform further development of the PD_Manager system and a larger effectiveness trial. Trial registration: ISRCTN Registry, ISRCTN17396879. Registered on 15 March 2017.

Palliative care remains suboptimal in end-stage liver disease.

Background:

Abstract Objectives  To evaluate the cost effectiveness of the use of nine-valent pneumococcal polysaccharide conjugate vaccine in a routine infant immunisation programme based on the Pneumococcal Vaccine Trial (PVT) study in The Gambia. Methods  This was a clinical trial-based cost-effectiveness study conducted as part of the PVT study. The PVT was an intention-to-treat double-blind placebo-controlled trial of a nine-valent pneumococcal polysaccharide conjugate vaccine. The trial was conducted in the eastern parts of The Gambia, West Africa and recruited 17 437 children aged 40–364 days. A deterministic static cohort model was developed to evaluate direct benefits and costs of pneumococcal conjugate vaccine in The Gambia's routine immunisation programme. The incremental cost-effectiveness ratio (iCER) is defined as vaccinating infants against pneumococcal disease compared with no vaccination from a public provider's perspective using The Gambia's 2005 projected under-one-year population. Key findings  The results show the use of the vaccine in The Gambia's routine infant vaccination programme to be cost effective using an assumed price of US$5.00 per vial in single-dose vials. Compared with offering no vaccination, the incremental cost per DALYs averted would be 30 DALYs from the public provider perspective. At least 1569 and 340 invasive childhood pneumococcal illnesses and deaths respectively among the cohort would be prevented. In the absence of the vaccine 16 871 DALYs would be lost while with the use of the vaccine 7804 DALYs would be lost. Given the average treatment cost of pneumococcal illnesses to be US$191 (95% confidence interval 180 to 203) the introduction of the vaccine programme would lead to an additional cost of US$274 279 (about US$8.43/child). Conclusions  The availability of a cost-effective vaccine that can prevent thousands of pneumococcal illnesses and related deaths is a major development towards improving the disease burden in sub-Saharan African countries. This study supports the introduction of nine-valent pneumococcal vaccine into the infant immunisation programme of The Gambia as it is cost effective and will avert many preventable deaths and illnesses. Despite differences in distribution of serotypes between countries, the static model used in the analysis makes the results applicable to other developing countries, particularly those in sub-Saharan Africa.

Objectives The objectives were to investigate the impact of ‘time-effect’ on the estimation of quality-adjusted life years (QALYs) along prospective clinical trials’ outcomes using an assumed fixed time duration versus the actual time durations for each case. The ‘time’ duration is the length of time in a health state. Methods Two methods were used in the estimation of QALYs based using EQ-5D 3L scores collected at specific time-point intervals. One method used the actual time durations for each case based on CRF records, and the other used an assumed time duration and globally applied it to all the cases. Using SPSS® software program, we used paired-sample t-tests to assess whether the ‘time-effect’ can potentially affect trial results using CONSTRUCT trial data as reported in the trial results publications. The trial compared use of Infliximab with Cyclosporine for patients with Ulcerative Colitis and it involved some 270 participants. Key findings The results largely indicate statistically significant differences between the two methods of QALY estimations. QALYs at the respective time-points indicate no statistical difference between the two approaches. However, the difference in terms of total QALYs between the two QALY estimation approaches is statistically significant with considerable impact on costs/QALY. Conclusions Considering the possible impact of the time-effect on QALY estimations, the result implies that it can have significant implications for resources allocations decisions. In this respect, researchers have to pay due considerations to the approach they use and where possible, actual time durations must be used in QALY estimations along prospective clinical trials.