scholarly journals A multicentre randomiSed controlled TRial of IntraVEnous immunoglobulin compared with standard therapy for the treatment of transverse myelitis in adults and children (STRIVE)

2017 ◽  
Vol 21 (31) ◽  
pp. 1-50 ◽  
Author(s):  
Michael Absoud ◽  
Peter Brex ◽  
Olga Ciccarelli ◽  
Onyinye Diribe ◽  
Gavin Giovannoni ◽  
...  
Keyword(s):  
Spinal Cord ◽  
Randomised Controlled Trial ◽  
Standard Therapy ◽  
Outcome Measure ◽  
Controlled Trial ◽  
Transverse Myelitis ◽  
Health Technology ◽  
Controlled Trials ◽  
Adults And Children ◽  
Randomised Controlled

Background Transverse myelitis (TM) is an immune-mediated disorder of the spinal cord that affects adults and children and that causes motor, sensory and autonomic dysfunction. There is a prolonged recovery phase, which may continue for many years. Neuromyelitis optica (NMO) is an uncommon relapsing inflammatory central nervous system condition in which TM can be the first presenting symptom. As TM and NMO affect many patients in the prime of their working life, the disorder can impose a significant demand on health resources. There are currently no robust controlled trials in children or adults to inform the optimal treatment of TM. However, treatment with intravenous immunoglobulin (IVIG) is being effectively used in the management of a range of neurological conditions. Although other interventions such as plasma exchange (PLEX) in addition to intravenous (IV) methylprednisolone therapy can be beneficial in TM, PLEX is costly and technically challenging to deliver in the acute setting. IVIG is more readily accessible and less costly. Objective To evaluate whether additional and early treatment with IVIG is of extra benefit in TM compared with standard therapy with IV steroids. Design A multicentre, single-blind, parallel-group randomised controlled trial of IVIG compared with standard therapy for the treatment of TM in adults and children. Participants Patients aged ≥ 1 year diagnosed with either acute first-onset TM or first presentation of NMO. Target recruitment was 170 participants (85 participants per arm). Interventions Participants were randomised 1 : 1 to treatment with IV methylprednisolone only or treatment with IV methylprednisolone plus 2 g/kg of IVIG in divided doses within 5 days of the first commencement of steroid therapy. Main outcome measures Primary outcome measure – American Spinal Injury Association (ASIA) Impairment Scale at 6 months post randomisation, with a good outcome defined by a two-grade change. Secondary and tertiary outcome measures – ASIA motor and sensory scales, Expanded Disability Status Scale, health outcome, quality of life, Client Service Receipt Inventory and International Spinal Cord Injury Pain, Bladder and Bowel Basic Data Sets. Results In total, 26 participants were screened and two were randomised into the study. With the limited sample size, treatment effect could not be determined. However, we identified barriers to accrual that included strict inclusion criteria, the short enrolment window, challenges associated with the use of the ASIA Impairment Scale as an outcome measure and estimation of the incidence of TM. Conclusions The study did not reach the end point and the effect of IVIG in TM/NMO could not be determined. Investigators should be aware of the potential challenges associated with carrying out a rare disease trial with a short enrolment window. The study question is one that still necessitates investigation. Preliminary work to ameliorate the effect of the barriers encountered in this study is vital. Trial registration EudraCT 2014-002335-34, ClinicalTrials.gov NCT02398994 and Current Controlled Trials ISRCTN12127581. Funding This project was funded by the NIHR Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 21, No. 31. See the NIHR Journals Library website for further project information. Funding was also received from Biotest AG, Germany (supply of IVIG) and the Transverse Myelitis Society (excess research cost to facilitate study initiation).

scholarly journals Saline in Acute Bronchiolitis RCT and Economic evaluation: hypertonic saline in acute bronchiolitis – randomised controlled trial and systematic review

2015 ◽  
Vol 19 (66) ◽  
pp. 1-130 ◽  
Author(s):  
Mark L Everard ◽  
Daniel Hind ◽  
Kelechi Ugonna ◽  
Jennifer Freeman ◽  
Mike Bradburn ◽  
...  
Keyword(s):  
Systematic Review ◽  
Randomised Controlled Trial ◽  
Adverse Events ◽  
Supportive Care ◽  
Technology Assessment ◽  
Controlled Trial ◽  
Health Technology ◽  
Controlled Trials ◽  
Acute Bronchiolitis ◽  
Randomised Controlled

BackgroundAcute bronchiolitis is the most common cause of hospitalisation in infancy. Supportive care and oxygen are the cornerstones of management. A Cochrane review concluded that the use of nebulised 3% hypertonic saline (HS) may significantly reduce the duration of hospitalisation.ObjectiveTo test the hypothesis that HS reduces the time to when infants were assessed as being fit for discharge, defined as in air with saturations of > 92% for 6 hours, by 25%.DesignParallel-group, pragmatic randomised controlled trial, cost–utility analysis and systematic review.SettingTen UK hospitals.ParticipantsInfants with acute bronchiolitis requiring oxygen therapy were allocated within 4 hours of admission.InterventionsSupportive care with oxygen as required, minimal handling and fluid administration as appropriate to the severity of the disease, 3% nebulised HS every ± 6 hours.Main outcome measuresThe trial primary outcome was time until the infant met objective discharge criteria. Secondary end points included time to discharge and adverse events. The costs analysed related to length of stay (LoS), readmissions, nebulised saline and other NHS resource use. Quality-adjusted life-years (QALYs) were estimated using an existing utility decrement derived for hospitalisation in children, together with the time spent in hospital in the trial.Data sourcesWe searched MEDLINE, EMBASE, the Cochrane Central Register of Controlled Trials and other databases from inception or from 2010 onwards, searched ClinicalTrials.gov and other registries and hand-searchedChest,PaediatricsandJournal of Paediatricsto January 2015.Review methodsWe included randomised/quasi-randomised trials which compared HS versus saline (± adjunct treatment) or no treatment. We used a fixed-effects model to combine mean differences for LoS and assessed statistical heterogeneity using theI2statistic.ResultsThe trial randomised 158 infants to HS (n = 141 analysed) and 159 to standard care (n = 149 analysed). There was no difference between the two arms in the time to being declared fit for discharge [median 76.6 vs. 75.9 hours, hazard ratio (HR) 0.95, 95% confidence interval (CI) 0.75 to 1.20] or to actual discharge (median 88.5 vs. 88.7 hours, HR 0.97, 95% CI 0.76 to 1.23). There was no difference in adverse events. One infant developed bradycardia with desaturation associated with HS. Mean hospital costs were £2595 and £2727 for the control and intervention groups, respectively (p = 0.657). Incremental QALYs were 0.0000175 (p = 0.757). An incremental cost-effectiveness ratio of £7.6M per QALY gained was not appreciably altered by sensitivity analyses. The systematic review comprised 15 trials (n = 1922) including our own. HS reduced the mean LoS by –0.36 days (95% CI –0.50 to –0.22 days). High levels of heterogeneity (I2 = 78%) indicate that the result should be treated cautiously.ConclusionsIn this trial, HS had no clinical benefit on LoS or readiness for discharge and was not a cost-effective treatment for acute bronchiolitis. Claims that HS achieves small reductions in LoS must be treated with scepticism.Future workWell-powered randomised controlled trials of high-flow oxygen are needed.Study registrationThis study is registered as NCT01469845 and CRD42014007569.Funding detailsThis project was funded by the NIHR Health Technology Assessment (HTA) programme and will be published in full inHealth Technology Assessment; Vol. 19, No. 66. See the HTA programme website for further project information.

scholarly journals A randomised controlled trial to examine the effects of cinacalcet on bone and cardiovascular parameters in haemodialysis patients with advanced secondary hyperparathyroidism

2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Helen Eddington ◽  
Rajkumar Chinnadurai ◽  
Helen Alderson ◽  
Sara T. Ibrahim ◽  
Constantina Chrysochou ◽  
...  
Keyword(s):  
Randomised Controlled Trial ◽  
Secondary Hyperparathyroidism ◽  
Standard Therapy ◽  
Augmentation Index ◽  
Controlled Trial ◽  
Left Ventricular ◽  
Agatston Score ◽  
Open Label ◽  
Mineral And Bone Disorder ◽  
Randomised Controlled

Abstract Background Secondary hyperparathyroidism may lead to increased cardiovascular risk. The use of cinacalcet may improve bone and cardiovascular health with improved parathormone (PTH) and phosphate control. Methods This is an open-label prospective randomised controlled trial to compare progression of cardiovascular and chronic kidney disease mineral and bone disorder (CKD-MBD) parameters. Patients were randomised to receive cinacalcet alongside standard therapy or standard therapy alone. Thirty-six haemodialysis patients who had > 90 days on dialysis, iPTH > 300 pg/mL, calcium > 2.1 mmol/L and age 18–75 years were included. Following randomization, all 36 patients underwent an intensive 12-week period of bone disease management aiming for iPTH 150-300 pg/mL. The primary outcome was change in vascular calcification using CT agatston score. Secondary outcomes included pulse wave velocity (PWV), left ventricular mass index (LVMI), carotid intima-media thickness (CIMT), augmentation index (Aix) and bone measurements. The above measurements were obtained at baseline and 12 months. Results There was no evidence of a group difference in the progression of calcification (median change (IQR) cinacalcet: 488 (0 to1539); standard therapy: 563 (50 to 1214)). In a post hoc analysis combining groups there was a mean (SD) phosphate reduction of 0.3 mmol/L (0.7) and median (IQR) iPTH reduction of 380 pg/mL (− 754, 120). Regression of LVMI and CIMT was seen (P = 0.03 and P = 0.001) and was significantly associated with change of phosphate on multi-factorial analyses. Conclusions With a policy of intense CKD-MBD parameter control, no significant benefit in bone and cardiovascular markers was seen with the addition of cinacalcet to standard therapy over one year. Tight control of hyperphosphataemia and secondary hyperparathyroidism may lead to a reduction in LVMI and CIMT but this needs further investigation. Although the sample size was small, meticulous trial supervision resulted in very few protocol deviations with therapy.

scholarly journals Computerised cognitive–behavioural therapy for adults with intellectual disability: randomised controlled trial

2017 ◽  
Vol 211 (2) ◽  
pp. 95-102 ◽  
Author(s):  
Patricia Cooney ◽  
Catherine Jackman ◽  
David Coyle ◽  
Gary O'Reilly
Keyword(s):  
Intellectual Disability ◽  
Randomised Controlled Trial ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Controlled Trial ◽  
Behavioural Therapy ◽  
Primary Outcome Measure ◽  
Cognitive Behavioural ◽  
Randomised Controlled

BackgroundDespite the evidence base for computer-assisted cognitive–behavioural therapy (CBT) in the general population, it has not yet been adapted for use with adults who have an intellectual disability.AimsTo evaluate the utility of a CBT computer game for adults who have an intellectual disability.MethodA 2 × 3 (group × time) randomised controlled trial design was used. Fifty-two adults with mild to moderate intellectual disability and anxiety or depression were randomly allocated to two groups: computerised CBT (cCBT) or psychiatric treatment as usual (TAU), and assessed at pre-treatment, post-treatment and 3-month follow-up. Forty-nine participants were included in the final analysis.ResultsA significant group x time interaction was observed on the primary outcome measure of anxiety (Glasgow Anxiety Scale for people with an Intellectual Disability), favouring cCBT over TAU, but not on the primary outcome measure of depression (Glasgow Depression Scale for people with a Learning Disability). A medium effect size for anxiety symptoms was observed at post-treatment and a large effect size was observed after follow-up. Reliability of Change Indices indicated that the intervention produced clinically significant change in the cCBT group in comparison with TAU.ConclusionsAs the first application of cCBT for adults with intellectual disability, this intervention appears to be a useful treatment option to reduce anxiety symptoms in this population.

scholarly journals Understanding how a community-based intervention for people with spinal cord injury in Bangladesh was delivered as part of a randomised controlled trial: a process evaluation

Spinal Cord ◽  
2020 ◽  
Vol 58 (11) ◽  
pp. 1166-1175
Author(s):  
Hueiming Liu ◽  
Mohammad Sohrab Hossain ◽  
Md. Shofiqul Islam ◽  
Md. Akhlasur Rahman ◽  
Punam D. Costa ◽  
...  
Keyword(s):  
Spinal Cord ◽  
Randomised Controlled Trial ◽  
Healthcare Professionals ◽  
Controlled Trial ◽  
Home Visits ◽  
Case Managers ◽  
Community Based ◽  
Cord Injury ◽  
Telephone Calls ◽  
Randomised Controlled

Abstract Design Mixed methods study Setting Community, Bangladesh Objectives To understand how a community-based intervention for people with spinal cord injury (SCI) in Bangladesh was delivered as part of a randomised controlled trial and to gauge the perceptions of participants and healthcare professionals to the intervention. Methods A community-based intervention was administered to 204 participants as part of a large randomised controlled trial (called the CIVIC trial). Case-managers followed-up participants with regular telephone calls and home visits over the first 2 years after discharge. The following data were collected alongside the trial: (i) chart audit of telephone calls and home visits (ii) recordings of 20 telephone calls (iii) interviews with 14 Intervention participants and four healthcare professionals including three case-managers. Results Participants received the target number of telephone calls and home visits. Pressure injuries were identified as a problem during at least one telephone call by 43% of participants. Participants and case-managers valued regular telephone calls and home visits, and believed that calls and visits prevented complications and alleviated social isolation. Participants trusted case-managers and were confident in the care and advice provided. Case-managers expressed concerns that people with SCI in Bangladesh face many problems impacting on well-being and motivation stemming from poverty, limited employment opportunities, societal attitudes and inaccessible environments. Conclusion A community-based intervention involving regular telephone calls and home visits was administered as intended and was well received by the recipients of the care. Nonetheless, people with SCI in Bangladesh face economic and social problems which cannot be fully addressed by this type of intervention alone.

scholarly journals Effects of Testosterone and Evoked Resistance Exercise after Spinal Cord Injury (TEREX-SCI): study protocol for a randomised controlled trial

BMJ Open ◽  
2017 ◽  
Vol 7 (4) ◽  
pp. e014125 ◽  
Author(s):  
Ashraf S Gorgey ◽  
Refka E Khalil ◽  
Ranjodh Gill ◽  
Laura C O'Brien ◽  
Timothy Lavis ◽  
...  
Keyword(s):  
Spinal Cord ◽  
Spinal Cord Injury ◽  
Randomised Controlled Trial ◽  
Resistance Exercise ◽  
Study Protocol ◽  
Controlled Trial ◽  
Cord Injury ◽  
Randomised Controlled
Sign in / Sign up

Export Citation Format

Share Document