Patient-related Quality Of Life Research Articles

Methodological considerations in PISCES 3: a randomized, placebo-controlled study of intracerebral stem cells in subjects with disability following an ischemic stroke

Background and hypothesisAt present, there are no medical interventions proven to improve functional recovery in patients with subacute stroke. We hypothesize that the intraparenchymal administration of CTX0E03, a conditionally immortalized neural stem cell line, linked with a standardized rehabilitation therapy regimen for the upper limb, would improve functional outcomes in patients 6–12 months after an index ischemic stroke.Study designPISCES III was designed as a multicenter prospective, sham-controlled, outcome-blinded randomized clinical trial. Eligibility required a qualifying ischemic stroke 6–12 months prior to surgical intervention. Patients must be between 35 and 75 years of age and have residual moderate or moderately severe disability (mRS 3 or 4), with the preservation of some residual upper limb movement. All patients received a standardized regimen of home physical therapy following the intervention.Study outcomesThe primary outcome measure is improvement in the modified Rankin Scale (mRS) of disability at 6 months post treatment. Secondary outcomes include assessment of activities of daily living (Barthel Index), functional mobility (Timed Up and Go; Fugl Meyer Assessment), neurological impairment (NIHSS), upper limb function (Chedoke Arm and Hand Inventory), as well as patient related quality of life and global rating scales.DiscussionPISCES III was designed as a randomized trial directly comparing the effects of intraparenchymal injection of a conditional stem cell line vs. sham procedure in patients with subacute stroke. This is one of the first studies of this type to include a standardized minimum rehabilitation protocol. As there are a limited number of studies evaluating invasive stem cell administration in the chronic setting of CNS injury, study design considerations are discussed.

Cutaneous T-cell lymphoma: diagnosing subtypes and the challenges.

Cutaneous T-cell lymphoma is a rare type of extranodal non-Hodgkin's lymphoma that primarily affects the skin. The uncertain pathogenesis and variable clinical presentation make the diagnosis and management of cutaneous T-cell lymphoma a challenge. Cutaneous T-cell lymphoma is a chronic, relapsing illness with treatment aimed at symptomatic relief and improving patient related quality of life. Early-stage cutaneous T-cell lymphoma typically follows an indolent course, often being mistaken for benign dermatological conditions which can lead to a diagnostic delay. Advanced stage cutaneous T-cell lymphoma has a poor prognosis with significant morbidity. Accurate diagnosis and early involvement of a specialist team is paramount to ensure correct management and improved patient outcomes. Promising advances are being made to develop novel agents which could improve prognosis and quality of life. This article provides an overview of the two main subtypes of cutaneous T-cell lymphoma: mycosis fungoides and Sézary syndrome. Clinical presentation, histopathological correlation and diagnostic challenges are reviewed alongside example case studies.

Mini-Incision Parotidectomy-Our Technique.

Traditional parotidectomy incision was devised by Blair (1912) which was modified by Bailey (1941). Over the years various approaches and techniques have evolved to improve the aesthetic outcome and at the same time for complete disease clearance with reduced complications. In this study, we evaluated the feasibility of our mini-incision parotidectomy technique along with the surgical and quality of life (QOL) outcomes. This prospective case series was conducted at Apollo Hospitals, Bangalore over a period of 2years (June 2018-August 2020) and includes 20 patients. The surgical outcomes were assessed in terms of feasibility of mini-incision technique with respect to levels of parotid involved and functional outcomes in terms of presence or absence of complications like facial palsy (temporary or permanent), seroma and Frey's syndrome. Patient related quality of life (QOL) outcomes were assessed in terms of post-operative pain score, patient comfort score and cosmetic score by using numerical rating scale-11 (NRS-11). The mini-incision parotidectomy technique is feasible for lesions in all the parotid levels and conversion or lengthening of incision was not needed in any of the cases. 2(10%) patients had temporary facial palsy (House-Brackman grade III) which was recovered within 3weeks after surgery. One patient (5%) with adenoid cystic carcinoma had permanent facial palsy. Out of 20 patients 2(10%) had seroma and 1(5%) patient presented with Frey's syndrome. Mean post-operative pain score at 0, 6 and 24h were 4.8, 3.4 and 1.8 out of 10 respectively. Mean comfort score was 9 out of 10 and mean cosmetic score was 9.5 out of 10. Mini-incision parotidectomy technique can render improved functional as well as aesthetic outcomes after parotidectomy without compromising the surgical clearance of the disease process.

Stereotactic MR-guided on-table adaptive radiation therapy (SMART) for locally advanced pancreatic cancer.

TPS786 Background: Standard dose radiation therapy has been unsuccessful in inoperable pancreatic cancer; with a negative study (LAP07) for conventional chemoradiation and dropping of the stereotactic body radiation therapy arm in Alliance A021501. Recently, reports of using high dose ablative radiation therapy has been associated with increased survival in retrospective studies. Moreover, technological advances with MRI-guided radiation therapy offer improved targeting and the ability to change the radiation delivery on a daily fashion; allowing ablative radiation doses over one week. However, it is not clear whether this can be done safely on a multiinstitutional basis. Methods: We are conducting the largest prospective study of ablative radiation therapy in pancreatic cancer. The study is a single arm, multi-institutional phase II, industry sponsored study to investigate the safety and efficacy of Stereotactic, MR guided, on-table-Adaptive Radiation Therapy (SMART). Eligibility criteria include locally advanced and borderline resectable pancreatic cancer patients with ECOG PS of 0 or 1; who have non-metastatic disease after a minimum of 3 months of any systemic therapy; including investigational agents. Patients will receive MR-guided radiation therapy to a dose of 50 Gy / 5 fractions; with maximum tumor coverage delivered each fraction that allows keeping the gastrointestinal organs at risk to a dose of 33 Gy or less. Primary endpoint is grade 3 of higher gastrointestinal toxicity at 90 days. Secondary endpoints are overall survival at 2 years, distant progression free survival at 6 months, and changes in patient related quality of life at 3 and 12 months. Target sample size was calculated to show at a significance level 0.05, a reduction of the toxicity rate to 8% or lower by using SMART compared with 15.8%, the toxicity rate of conventionally delivered chemoradiation at a power level 0.8. Given an expected 15% drop-out, the enrollment goal is 133. Descriptive statistics will be used for secondary objectives. The study opened in January, 2019 and is currently opened at 4 centers; with other US and international sites pending. Sponsored by Viewray, Inc. Clinical trial information: NCT03621644.

Value For Money In The Treatment Of Patients With Type 2 Diabetes Mellitus: Assessing The Long-Term Cost-Effectiveness Of IDegLira Versus iGlarLixi In Italy.

ObjectiveItalian treatment guidelines for type 2 diabetes mellitus (T2DM) target good glycemic control but acknowledge the associated risk of hypoglycemia. Unlike traditional antidiabetic therapies, modern treatment options such as fixed-ratio combinations of basal insulin and glucagon-like peptide 1 receptor agonists are associated with improved glycemic control, reduced body weight and low risk of hypoglycemia. The cost-effectiveness of the fixed-ratio combinations of basal insulin and glucagon-like peptide 1 receptor agonists IDegLira and iGlarLixi was assessed for Italy in patients with T2DM uncontrolled on basal insulin, to evaluate how short-term clinical benefits translate into long-term health economic outcomes.MethodsThe IQVIA CORE Diabetes Model was used to project clinical and economic outcomes over patient lifetimes. Treatment effects were sourced from an indirect treatment comparison. The analysis captured direct medical costs (expressed in 2017 Euros) from the perspective of the Italian National Health Service (NHS) and patient-related quality of life. Sensitivity analyses were performed.ResultsIDegLira was associated with gains of 0.09 life years and 0.13 quality-adjusted life years (QALYs) relative to iGlarLixi, due to a lower cumulative incidence and delayed onset of diabetes-related complications. IDegLira was associated with an incremental cost of EUR 930 over patient lifetimes, leading to an incremental cost-effectiveness ratio of EUR 7,386 per QALY gained.ConclusionOver the lifetime of patients with T2DM uncontrolled on basal insulin, IDegLira was associated with improved clinical outcomes at higher costs relative to iGlarLixi. At a willingness-to-pay threshold of EUR 30,000 per QALY gained, IDegLira was considered to be cost-effective versus iGlarLixi from the perspective of the Italian NHS.

Patient-related quality of life after pelvic ring fractures in elderly.

Pelvic ring fractures, occurring in elderly patients are a challenging problem. Little known is about the patient-related outcome after these injuries. The primary objective of this study is to evaluate the quality of life after pelvic ring injuries in patients aged over 60years. Patients (≥60years) with pelvic ring fractures treated in our trauma department between 2004 and 2014 were included. Next to patient data, injury-related details as well as treatment details were assessed. After a follow-up of at least twoyears, the survival rate and the patient-related outcome were evaluated using the SF-36 and the EQ-5D score. One hundred ninety-six patients (138 women; 58 men; mean age 75.3 ± 7.8years) were identified. Ninety-six patients were treated operatively, 100 patients conservatively. The overall complication rate was significantly lower for conservatively compared to operatively treated patients (conservatively 18% vs. operatively 33%; p= 0.014). The total mortality rate over 2years is 29% with no significant difference of the two-year survival rate (2-year survival rate: operatively 77% vs. non-operatively 65%; p= 0.126). Fifty-five patients completed the SF-36 and EQ-5D score after a mean follow-up of 4.2 ± 2.9years. The mean physical component score of the SF-36 is 33.6 ± 8.3, and the mean mental component score is 45.3 ± 8.4. The mean EQ-5D VAS reached 62.5 ± 27.9. Elderly patients with pelvic ring fractures show a high mortality rate and a limited patient-related outcome. While the complication rate of conservatively treated patients is lower compared to operated patients, the twoyear survival rate is steady.

The effects of a four-week intensive scoliosis-specific exercise programme on patient-related quality of life in adult subjects: A >3 years follow-up study

The effects of a four-week intensive scoliosis-specific exercise programme on patient-related quality of life in adult subjects: A >3 years follow-up study

Patients’ preferences for the management of knee injuries: a discrete choice experiment

Objectives To elicit the preferences in two cohorts of patients with knee injuries who had been referred by their General Practitioner (GP) for Magnetic Resonance Imaging (MRI) or orthopaedic consultation; or were listed for an arthroscopy. Methods A discrete choice experiment using a postal questionnaire asked for patients’ preferences for, and trade-offs between, the attributes of waiting time for diagnosis, treatment time, change in knee problem and out of pocket expenses. Results Participants with knee injuries who responded to the questionnaire were: 148 of 218 (68%) in the GP cohort; and 95 of 121 (79%) in the arthroscopy cohort. Waiting time for diagnosis and treatment, as well as change in knee problem, were statistically significant important attributes. This applies less to out-of-pocket expenses although patients in the arthroscopy cohort were significantly more willing to pay extra for their treatment. There were no significant interactions between employment status and cost. For patient-related quality oflife, attributes for choosing a shorter time to diagnosis or treatment were statistically significant or borderline. Conclusion Patients with knee injuries find shorter waiting time for diagnosis and treatment to be important as well as improvements in their knee problem. Out-of-pocket expenses were less important, except for patients waiting for an arthroscopy who were willing to pay extra.

Efficacy of a text messaging (SMS) based intervention for adults with hypertension: protocol for the StAR (SMS Text-message Adherence suppoRt trial) randomised controlled trial

BackgroundInterventions to support people with hypertension in attending clinics and taking their medication have potential to improve outcomes, but delivery on a wide scale and at low cost is challenging. Some trials evaluating clinical interventions using short message service (SMS) text-messaging systems have shown important outcomes, although evidence is limited. We have developed a novel SMS system integrated with clinical care for use by people with hypertension in a low-resource setting. We aim to test the efficacy of the system in improving blood pressure control and treatment adherence compared to usual care.Methods/designThe SMS Text-message Adherence suppoRt trial (StAR) is a pragmatic individually randomised three-arm parallel group trial in adults treated for hypertension at a single primary care centre in Cape Town, South Africa. The intervention is a structured programme of clinic appointment, medication pick-up reminders, medication adherence support and hypertension-related education delivered remotely using an automated system with either informational or interactive SMS text-messages. Usual care is supplemented by infrequent non-hypertension related SMS text-messages. Participants are 1:1:1 individually randomised, to usual care or to one of the two active interventions using minimisation to dynamically adjust for gender, age, baseline systolic blood pressure, years with hypertension, and previous clinic attendance. The primary outcome is the change in mean systolic blood pressure at 12-month follow-up from baseline measured with research staff blinded to trial allocation. Secondary outcomes include the proportion of patients with 80% or more of days medication available, proportion of participants achieving a systolic blood pressure less than 140 mmHg and a diastolic blood pressure less than 90 mmHg, hospital admissions, health status, retention in clinical care, satisfaction with treatment and care, and patient related quality of life. Anonymised demographic data are collected on non-participants.DiscussionThe StAR trial uses a novel, low cost system based on widely available mobile phone technology to deliver the SMS-based intervention, manage communication with patients, and measure clinically relevant outcomes. The results will inform implementation and wider use of mobile phone based interventions for health care delivery in a low-resource setting.Trial registrationNCT02019823

Assessment of disease activity, damage and quality of life in systemic lupus erythematosus: New aspects

Systemic lupus erythematosus (SLE) is a multisystem auto-immune disorder that results from a combination of genetic, environmental and hormonal factors. The heterogeneity of disease presentation and course in different individuals and the variability in the disease progression/fluctuations within the same patient have made finding a unifying assessment tool difficult. It is currently accepted that assessment of patients with SLE cannot be accomplished with a single index. Formal evaluation of three aspects of the disease, disease activity, disease damage and patient-related quality of life (QoL), is required. In the recent decade, the pathogenesis of SLE at the cellular and molecular levels has been the subject of much research. Robust assessment tools are needed to correlate the presence of various serological markers with disease activity. In addition, multiple clinical trials of new therapies have necessitated validated measures that can give a sensitive response index. This review focusses on the SLE assessment tools currently in use and their translational application in clinical research and trials.

The influence of myocardial infarction and stroke on patient related quality of life

Aim: To investigate health-related quality of life in a large sample of patients with stroke and myocardial function.

Clinical and cost-effectiveness of newer immunosuppressive regimens in renal transplantation: a systematic review and modelling study

Clinical and cost-effectiveness of newer immunosuppressive regimens in renal transplantation: a systematic review and modelling study

Dual chamber versus single chamber ventricular pacemakers for sick sinus syndrome and atrioventricular block.

Dual chamber pacing or single chamber atrial pacing ('physiologic' pacing) is believed to have an advantage over single chamber ventricular pacing in that it resembles cardiac physiology more closely by maintaining atrioventricular (AV) synchrony and dominance of the sinus node, which in turn may reduce cardiovascular morbidity and mortality thus contributing to patient survival and quality of life. However, a significant proportion of pacemakers currently implanted are single chamber ventricular pacemakers. The objective of this review was to assess the short- and long-term clinical effectiveness of dual chamber pacemakers compared to single chamber ventricular pacemakers in adults with AV block, sick sinus syndrome or both. An additional objective was to assess separately any potential differences in effectiveness between dual chamber pacing and single chamber atrial pacing. The clinical effectiveness of single chamber atrial pacing versus single chamber ventricular pacing was not examined. The Cochrane Controlled Trials Register (The Cochrane Library Issue 3, 2002), MEDLINE (1966 to 2002), EMBASE (1980 to 2002) and the Science Citation Index (1980 to 2002) were searched on 19th August 2002. Citation lists and web sites were checked and researchers in the field contacted. Parallel group or crossover randomised controlled trials of at least 48 hours duration comparing dual chamber pacing and single chamber ventricular pacing, and investigating cardiovascular morbidity, mortality, patient related quality of life, exercise capacity and complication rates. Data was extracted onto pre-piloted data extraction forms. Quality assessment was undertaken using a checklist, with a sub-sample of quality data independently extracted by a second reviewer. Where appropriate data was available, meta-analysis was performed. Where meta-analysis was not possible, the number of studies showing a positive, neutral or negative direction of effect and statistical significance were simply counted. Five parallel and 26 crossover randomised controlled trials were identified. The quality of reporting was found to be poor. Pooled data from parallel studies shows a statistically non-significant preference for physiologic pacing (primarily dual chamber pacing) for the prevention of stroke, heart failure and mortality, and a statistically significant beneficial effect regarding the prevention of atrial fibrillation (odds ratio (OR) 0.79, 95% CI 0.68 to 0.93). Both parallel and crossover studies favour dual chamber pacing with regard to pacemaker syndrome (parallel: Peto OR 0.11, 95% CI 0.08 to 0.14; crossover: standardised mean difference (SMD) -0.74, 95% CI - 0.95 to -0.52). Pooled data from crossover studies shows a statistically significant trend towards dual chamber pacing being more favourable in terms of exercise capacity (SMD -0.24, 95% CI -0.03 to -0.45). No individual studies reported a significantly more favourable outcome with single chamber ventricular pacing. This review shows a trend towards greater effectiveness with dual chamber pacing compared to single chamber ventricular pacing, which supports the current British Pacing and Electrophysiology Group's Guidelines regarding atrioventricular block. Additional randomised controlled trial evidence from ongoing trials in this area will further inform the debate.