Interactive web-based pulmonary rehabilitation programme: a randomised controlled feasibility trial

Open access articleObjectives: The aim of this study was to determine if an interactive web-based pulmonary rehabilitation (PR) programme is a feasible alternative to conventional PR. Design: Randomised controlled feasibility trial. Setting: Participants with a diagnosis of chronic obstructive pulmonary disease were recruited from PR assessments, primary care and community rehabilitation programmes. Patients randomised to conventional rehabilitation started the programme according to the standard care at their referred site on the next available date. Participants: 103 patients were recruited to the study and randomised: 52 to conventional rehabilitation (mean (±SD) age 66 (±8) years, Medical Research Council (MRC) 3 (IQR2–4)); 51 to the web arm (mean (±SD) age 66 (±10) years, MRC 3 (IQR2–4)). Participants had to be willing to participate in either arm of the trial, have internet access and be web literate. Interventions: Patients randomised to the web-based programme worked through the website, exercising and recording their progress as well as reading educational material. Conventional PR consisted of twice weekly, 2 hourly sessions (an hour for exercise training and an hour for education). Outcome measures: Recruitment rates, eligibility, patient preference and dropout and completion rates for both programmes were collected. Standard outcomes for a PR assessment including measures of exercise capacity and quality of life questionnaires were also evaluated. Results: A statistically significant improvement (p≤0.01) was observed within each group in the endurance shuttle walk test (WEB: mean change 189 ±211.1; PR classes: mean change 184.5±247.4 s) and Chronic Respiratory disease Questionnaire-Dyspnoea (CRQ-D; WEB: mean change 0.7±1.2; PR classes: mean change 0.8±1.0). However, there were no significant differences between the groups in any outcome.Dropout rates were higher in the web-based programme (57% vs 23%). Conclusions: An interactive web-based PR programme is feasible and acceptable when compared with conventional PR. Future trials maybe around choicebased PR programmes for select patients enabling stratification of patient care

Cost-effectiveness of left ventricular assist devices (LVADs) for patients with advanced heart failure : analysis of the British NHS Bridge to Transplant (BTT) program

Background: A previous cost-effectiveness analysis showed that bridge to transplant (BTT) with early design left ventricular assist devices (LVADs) for advanced heart failure was more expensive than medical management while appearing less beneficial. Older LVADs were pulsatile, but current second and third generation LVADs are continuous flow pumps. This study aimed to estimate comparative cost-effectiveness of BTT with durable implantable continuous flow LVADs compared to medical management in the British NHS. Methods and results: A semi-Markov multi-state economic model was built using NHS costs data and patient data in the British NHS Blood and Transplant Database (BTDB). Quality-adjusted life years (QALYs) and incremental costs per QALY were calculated for patients receiving LVADs compared to those receiving inotrope supported medical management. LVADs cost £80,569 ($127,887) at 2011 prices and delivered greater benefit than medical management. The estimated probabilistic incremental cost-effectiveness ratio (ICER) was £53,527 ($84,963)/QALY (95%CI: £31,802–£94,853; $50,479–$150,560) (over a lifetime horizon). Estimates were sensitive to choice of comparator population, relative likelihood of receiving a heart transplant, time to transplant, and LVAD costs. Reducing the device cost by 15% decreased the ICER to £50,106 ($79,533)/QALY. Conclusions: Durable implantable continuous flow LVADs deliver greater benefits at higher costs than medical management in Britain. At the current UK threshold of £20,000 to £30,000/QALY LVADs are not cost effective but the ICER now begins to approach that of an intervention for end of life care recently recommended by the British NHS. Cost-effectiveness estimates are hampered by the lack of randomized trials

Cost-effectiveness of left ventricular assist devices (LVADs) for patients with advanced heart failure : analysis of the British NHS Bridge to Transplant (BTT) program

Background: A previous cost-effectiveness analysis showed that bridge to transplant (BTT) with early design left ventricular assist devices (LVADs) for advanced heart failure was more expensive than medical management while appearing less beneficial. Older LVADs were pulsatile, but current second and third generation LVADs are continuous flow pumps. This study aimed to estimate comparative cost-effectiveness of BTT with durable implantable continuous flow LVADs compared to medical management in the British NHS. Methods and results: A semi-Markov multi-state economic model was built using NHS costs data and patient data in the British NHS Blood and Transplant Database (BTDB). Quality-adjusted life years (QALYs) and incremental costs per QALY were calculated for patients receiving LVADs compared to those receiving inotrope supported medical management. LVADs cost £80,569 ($127,887) at 2011 prices and delivered greater benefit than medical management. The estimated probabilistic incremental cost-effectiveness ratio (ICER) was £53,527 ($84,963)/QALY (95%CI: £31,802–£94,853; $50,479–$150,560) (over a lifetime horizon). Estimates were sensitive to choice of comparator population, relative likelihood of receiving a heart transplant, time to transplant, and LVAD costs. Reducing the device cost by 15% decreased the ICER to £50,106 ($79,533)/QALY. Conclusions: Durable implantable continuous flow LVADs deliver greater benefits at higher costs than medical management in Britain. At the current UK threshold of £20,000 to £30,000/QALY LVADs are not cost effective but the ICER now begins to approach that of an intervention for end of life care recently recommended by the British NHS. Cost-effectiveness estimates are hampered by the lack of randomized trials

Six weeks of home enteral nutrition versus standard care after esophagectomy or total gastrectomy for cancer: study protocol for a randomized controlled trial

Background: Each year approximately 3000 patients in the United Kingdom undergo surgery for esophagogastric cancer. Jejunostomy feeding tubes, placed at the time of surgery for early postoperative nutrition, have been shown to have a positive impact on clinical outcomes in the short term. Whether feeding out of hospital is of benefit is unknown. Local experience has identified that between 15 and 20% of patients required ‘rescue’ jejunostomy feeding for nutritional problems and weight loss while at home. This weight loss and poor nutrition may contribute to the detrimental effect on the overall quality of life (QoL) reported in these patients. Methods/Design: This randomized pilot and feasibility study will provide preliminary information on the routine use of jejunostomy feeding after hospital discharge in terms of clinical benefits and QoL. Sixty participants undergoing esophagectomy or total gastrectomy will be randomized to receive either a planned program of six weeks of home jejunostomy feeding after discharge from hospital (intervention) or treatment-as-usual (control). The intention of this study is to inform a multi-centre randomized controlled trial. The primary outcome measures will be recruitment and retention rates at six weeks and six months. Secondary outcome measures will include disease specific and general QoL measures, nutritional parameters, total and oral nutritional intake, hospital readmission rates, and estimates of healthcare costs. Up to 20 participants will also be enrolled in a qualitative sub-study that will explore participants’ and carers’ experiences of home tube feeding. The results will be disseminated by presentation at surgical, gastroenterological and dietetic meetings and publication in appropriate peer review journals. A patient-friendly lay summary will be made available on the University of Leicester and the University Hospitals of Leicester NHS Trust websites. The study has full ethical and institutional approval and started recruitment in July 2012. Trial registration: UKClinical Research Network ID #12447 (Main study); UKCRN ID#13361 (Qualitative sub study); ClinicalTrials.gov #NCT01870817 (First registered 28 May 2013

A randomised controlled trial of six weeks of home enteral nutrition versus standard care after oesophagectomy or total gastrectomy for cancer: report on a pilot and feasibility study.

BACKGROUND: Poor nutrition in the first months after oesophago-gastric resection is a contributing factor to the reduced quality of life seen in these patients. The aim of this pilot and feasibility study was to ascertain the feasibility of conducting a multi-centre randomised controlled trial to evaluate routine home enteral nutrition in these patients. METHODS: Patients undergoing oesophagectomy or total gastrectomy were randomised to either six weeks of home feeding through a jejunostomy (intervention), or treatment as usual (control). Intervention comprised overnight feeding, providing 50 % of energy and protein requirements, in addition to usual oral intake. Primary outcome measures were recruitment and retention rates at six weeks and six months. Nutritional intake, nutritional parameters, quality of life and healthcare costs were also collected. Interviews were conducted with a sample of participants, to ascertain patient and carer experiences. RESULTS: Fifty-four of 112 (48 %) eligible patients participated in the study over the 20 months. Study retention at six weeks was 41/54 patients (76 %) and at six months was 36/54 (67 %). At six weeks, participants in the control group had lost on average 3.9 kg more than participants in the intervention group (95 % confidence interval [CI] 1.6 to 6.2). These differences remained evident at three months (mean difference 2.5 kg, 95 % CI -0.5 to 5.6) and at six months (mean difference 2.5 kg, 95 % CI -1.2 to 6.1). The mean values observed in the intervention group for mid arm circumference, mid arm muscle circumference, triceps skin fold thickness and right hand grip strength were greater than for the control group at all post hospital discharge time points. The economic evaluation suggested that it was feasible to collect resource use and EQ-5D data for a full cost-effectiveness analysis. Thematic analysis of 15 interviews identified three main themes related to the intervention and the trial: 1) a positive experience, 2) the reasons for taking part, and 3) uncertainty of the study process. CONCLUSIONS: This study demonstrated that home enteral feeding by jejunostomy was feasible, safe and acceptable to patients and their carers. Whether home enteral feeding as 'usual practice' is a cost-effective therapy would require confirmation in an appropriately powered, multi-centre study. TRIAL REGISTRATION: UK Clinical Research Network ID 12447 (main trial, first registered 30 May 2012); UK Clinical Research Network ID 13361 (qualitative substudy, first registered 30 May 2012); ClinicalTrials.gov NCT01870817 (first registered 28 May 2013)

Impact of Potential Symptoms and Risks Associated with Acid Sphingomyelinase Deficiency on Patients and Caregivers: A Best-Worst Scaling Study

Carol Mansfield,1 Lubomyra Nalysnyk,2 Dhaivat Joshi,2 Joshua Coulter,1 Ruth Pulikottil-Jacob3 1Health Preference Assessment, RTI Health Solutions, Research Triangle Park, North Carolina, USA; 2Health Economics and Value Assessment, Sanofi, Cambridge, MA, USA; 3Health Economics and Value Assessment, Sanofi, Thames Valley Park, Reading, UKCorrespondence: Ruth Pulikottil-Jacob, Sanofi, Thames Valley Park, Reading, RG6 1AD, UK, Tel +44-7525594087, Email [email protected]: Acid sphingomyelinase deficiency (ASMD) is a rare, progressive, and potentially fatal disease affecting major organs; its symptoms present heterogeneously. Data on the most bothersome symptoms for patients with ASMD types B or A/B and their caregivers or parents are limited. We conducted a survey to quantify the relative impact of potential ASMD symptoms and risks for patients and parents/caregivers.Patients and Methods: Twenty respondents, recruited via National Niemann-Pick Disease Foundation (United States) and Niemann-Pick United Kingdom, took a preference survey: 11 patients who had a self-reported diagnosis of ASMD types B or A/B and 9 parents who had a child with ASMD types B or A/B. Using object-case best-worst scaling, we explored the most and least bothersome among a set of 15 ASMD symptoms/risks selected based on clinical input and qualitative research with patients and caregivers. In 15 experimentally designed questions containing five items each, respondents ranked the symptoms/risks, irrespective of their experiences with them. Data were analyzed using a conditional multinomial logit model.Results: Patients reported constant abdominal pain, severe pain in bones and joints, and severe fatigue to be the most bothersome potential symptoms or risks, followed by a chance of bleeding in the spleen. The next most bothersome potential symptom was constant shortness of breath. Easy bruising and noticeable abdominal enlargement were among the least bothersome symptoms. The most bothersome symptom for parents was bleeding in the spleen.Conclusion: Patients and parents had similar perceptions of the most bothersome potential symptoms/risks. Despite the small sample size typical of rare disease studies, understanding patient preferences is important for such diseases and can inform shared decision-making.Keywords: best-worst scaling, patient, caregiver, rare diseas

Cost-effectiveness of new left ventricular assist devices (LVADS) for patients with advanced heart failure : analysis of the UK NHS bridge to transplant (BTT) programme

Background: Public health measures aim to prevent people from reaching tertiary prevention or palliative or heroic care. Nevertheless these remain the only options for some people. There is an expanding population with very severe heart failure and although heart transplant is the optimal treatment, this option is dependent on a diminishing supply of donor hearts. For more than a decade, the NHS has supported the transplant programme for patients awaiting a donor heart with a ‘bridge to transplant’ programme - surgical implantation of a mechanical pump to support left ventricular function called a Left Ventricular Assist Device (LVAD). Alternatively individuals waiting for a heart receive medical management with drugs such as intravenous inotropes

Comparative cost-effectiveness of the HeartWare versus HeartMate II left ventricular assist devices used in the United Kingdom National Health Service bridge-to-transplant program for patients with heart failure

Background: Patients with advanced heart failure may receive a left ventricular assist device (LVAD) as part of a bridge-to-transplant (BTT) strategy. The United Kingdom National Health Service (UK NHS) has financed a BTT program in which the predominant LVADs used have been the HeartMate II (HM II; Thoratec, Pleasanton, CA) and HeartWare (HW; HeartWare International, Inc. Framingham, MA). We aimed to compare the cost-effectiveness of the use of these within the NHS program. Methods: Individual patient data from the UK NHS Blood and Transplant Data Base were analyzed with Kaplan-Meier and competing outcomes methodologies. Outcomes were time to death, time to heart transplant (HT), and cumulative incidences of HT, death on LVAD support, and LVAD explantation. A semi-Markov multistate economic model was built to assess cost-effectiveness. The perspective was from the NHS, discount rates were 3.5%. Outcomes were quality-adjusted life-years (QALYs) and incremental cost (2011 prices in GB£) per QALY (ICER) for HW vs HM II. Results: Survival was better with HW support than with HM II. Cumulative incidence of HT was low for both groups (11% at ~2 years). HW patients accrued 4.99 lifetime QALYs costing £258,913 ($410,970), HM II patients accrued 3.84 QALYs costing £231,871 ($368,048); deterministic and probabilistic ICERs for HW vs HM II were £23,530 ($37,349) and £20,799 ($33,014), respectively. Conclusions: Patients In the UK BTT program who received the HW LVAD had a better clinical outcome than those who received the HM II, and the HW was more cost-effective. This result needs to be reassessed in a randomized controlled trial comparing the 2 devices