scholarly journals Daily Practice Managing Resistant Multiple Sclerosis Spasticity With Delta-9-Tetrahydrocannabinol: Cannabidiol Oromucosal Spray: A Systematic Review of Observational Studies

2019 ◽  
Vol 11 ◽  
pp. 117957351983199 ◽  
Author(s):  
Katja Akgün ◽  
Ute Essner ◽  
Cordula Seydel ◽  
Tjalf Ziemssen
Keyword(s):  
Multiple Sclerosis ◽  
Systematic Review ◽  
Clinical Practice ◽  
Real World ◽  
Observational Studies ◽  
Rating Scale ◽  
Real Life ◽  
Daily Practice ◽  
Patient Characteristics ◽  
Numeric Rating Scale

Background/purpose: Spasticity is one of the most common symptoms in people with multiple sclerosis (MS). Conventional anti-spasticity agents have limitations in their efficacy and tolerability. Delta-9-tetrahydrocannabinol: cannabidiol (THC:CBD) spray, a cannabinoid-based medicine, is approved as an add-on therapy for MS spasticity not adequately controlled by other anti-spasticity medications. The results from randomized controlled trials (RCTs) have demonstrated a reduction in the severity of spasticity and associated symptoms. However, RCTs do not always reflect real-life outcomes. We systematically reviewed the complementary evidence from non-interventional real-world studies. Methods: A systematic literature review was conducted to identify all non-RCT publications on THC:CBD spray between 2011 and 2017. Data on study design, patient characteristics, effectiveness, and safety outcomes were extracted from those publications meeting our inclusion criteria. Results: In total, we reviewed 14 real-world publications including observational studies and treatment registries. The proportion of patients reaching the threshold of minimal clinical important difference (MCID), with at least a 20% reduction of the spasticity Numeric Rating Scale (NRS) score after 4 weeks ranged from 41.9% to 82.9%. The reduction in the mean NRS spasticity score after 4 weeks was maintained over 6-12 months. The average daily dose was five to six sprays. Delta-9-tetrahydrocannabinol: cannabidiol was well tolerated in the evaluated studies in the same way as in the RCTs. No new or unexpected adverse events or safety signals were reported in everyday clinical practice. Conclusions: The data evaluated in this systematic review provide evidence for the efficacy and safety of THC:CBD in clinical practice and confirm results obtained in RCTs.

scholarly journals Real-World Safety of CFTR Modulators in the Treatment of Cystic Fibrosis: A Systematic Review

2020 ◽  
Vol 10 (1) ◽  
pp. 23
Author(s):  
Renée V. E. Dagenais ◽  
Victoria C. Su ◽  
Bradley S. Quon
Keyword(s):  
Systematic Review ◽  
Cystic Fibrosis ◽  
Clinical Trials ◽  
Real World ◽  
Observational Studies ◽  
Case Reports ◽  
Case Series ◽  
Patient Characteristics ◽  
Online Databases ◽  
Cftr Modulators

Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies target the underlying cause of cystic fibrosis (CF), and are generally well-tolerated; however, real-world studies indicate the frequency of discontinuation and adverse events (AEs) may be higher than what was observed in clinical trials. The objectives of this systematic review were to summarize real-world AEs reported for market-available CFTR modulators (i.e., ivacaftor (IVA), lumacaftor/ivacaftor (LUM/IVA), tezacaftor/ivacaftor (TEZ/IVA), and elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA)), and to identify ways in which the pharmacist on CF healthcare teams may contribute to mitigating and managing these AEs. The MEDLINE, EMBASE, CINAHL, and Web of Science Core Collection online databases were searched from 2012 to 1 Aug 2020. Full manuscripts or conference abstracts of observational studies, case series, and case reports were eligible for inclusion. The included full manuscripts and conference abstracts comprised of 54 observational studies, 5 case series, and 9 case reports. The types of AEs reported generally aligned with what have been observed in clinical trials. LUM/IVA was associated with a higher frequency of respiratory-related AE and discontinuation in real-world studies. A signal for mental health and neurocognitive AEs was identified with all 4 CFTR modulators. A systematic approach to monitoring for AEs in people with CF on CFTR modulators in the real-world setting is necessary to help better understand potential AEs, as well as patient characteristics that may be associated with higher risk of certain AEs. Pharmacists play a key role in the safe initiation and monitoring of people with CF on CFTR modulator therapies.

scholarly journals Real-World Outcomes of Ivacaftor Treatment in People with Cystic Fibrosis: A Systematic Review

2021 ◽  
Vol 10 (7) ◽  
pp. 1527
Author(s):  
Jamie Duckers ◽  
Beth Lesher ◽  
Teja Thorat ◽  
Eleanor Lucas ◽  
Lisa J. McGarry ◽  
...  
Keyword(s):  
Systematic Review ◽  
Cystic Fibrosis ◽  
Real World ◽  
Safety Profile ◽  
Clinical Trial Data ◽  
Patient Characteristics ◽  
Healthcare Resource Utilization ◽  
Patient Reported ◽  
Clinical Benefits

Cystic fibrosis (CF) is a rare, progressive, multi-organ genetic disease. Ivacaftor, a small-molecule CF transmembrane conductance regulator modulator, was the first medication to treat the underlying cause of CF. Since its approval, real-world clinical experience on the use of ivacaftor has been documented in large registries and smaller studies. Here, we systematically review data from real-world observational studies of ivacaftor treatment in people with CF (pwCF). Searches of MEDLINE and Embase identified 368 publications reporting real-world studies that enrolled six or more pwCF treated with ivacaftor published between January 2012 and September 2019. Overall, 75 publications providing data from 57 unique studies met inclusion criteria and were reviewed. Studies reporting within-group change for pwCF treated with ivacaftor consistently showed improvements in lung function, nutritional parameters, and patient-reported respiratory and sino-nasal symptoms. Benefits were evident as early as 1 month following ivacaftor initiation and were sustained over long-term follow-up. Decreases in pulmonary exacerbations, Pseudomonas aeruginosa prevalence, and healthcare resource utilization also were reported for up to 66 months following ivacaftor initiation. In studies comparing ivacaftor treatment to modulator untreated comparator groups, clinical benefits similarly were reported as were decreases in mortality, organ-transplantation, and CF-related complications. The safety profile of ivacaftor observed in these real-world studies was consistent with the well-established safety profile based on clinical trial data. Our systematic review of real-world studies shows ivacaftor treatment in pwCF results in highly consistent and sustained clinical benefit in both pulmonary and non-pulmonary outcomes across various geographies, study designs, patient characteristics, and follow-up durations, confirming and expanding upon evidence from clinical trials.

scholarly journals Professionals’, patients’ and families’ views on the use of opioids for chronic breathlessness: A systematic review using the framework method and pillar process

2021 ◽  
pp. 026921632110321
Author(s):  
Florence Reedy ◽  
Mark Pearson ◽  
Sarah Greenley ◽  
Joseph Clark ◽  
David C Currow ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Practice ◽  
A Priori ◽  
Synthesis Method ◽  
Patient Characteristics ◽  
Severe Illness ◽  
Pharmacological Interventions ◽  
Eligibility Criteria ◽  
Treatment Regimens ◽  
Appraisal Tool

Background: In combination with non-pharmacological interventions, opioids may safely reduce chronic breathlessness in patients with severe illness. However, implementation in clinical practice varies. Aim: To synthesise the published literature regarding health professionals’, patients’ and families’ views on the use of opioids for chronic breathlessness, identifying issues which influence implementation in clinical practice. Design: Systematic review and synthesis using the five-stage framework synthesis method. Data sources: Three electronic databases (MEDLINE, Embase via OVID, ASSIA via Proquest) were searched (March 2020) using a predefined search strategy. Studies were also citation chained from key papers. Papers were screened against a priori eligibility criteria. Data were extracted from included studies using the framework synthesis method. Qualitative and quantitative data were synthesised using the pillar process. Included studies were critically appraised using the Mixed-Methods Appraisal Tool. Results: After de-duplication, 843 papers were identified. Following screening, 22 studies were included. Five themes were developed: (i) clinician/patient characteristics, (ii) education/knowledge/experience, (iii) relationship between clinician/family, (iv) clinician/patient fear of opioids and (v) regulatory issues. Conclusions: There are significant barriers and enablers to the use of opioids for the symptomatic reduction of chronic breathlessness based on the knowledge, views and attitudes of clinicians, patients and families. Clinicians’ interactions with patients and their families strongly influences adherence with opioid treatment regimens for chronic breathlessness. Clinicians’, patients’ and families’ knowledge about the delicate balance between benefits and risks is generally poor. Education for all, but particularly clinicians, is likely to be a necessary (but insufficient) factor for improving implementation in practice.

scholarly journals Music intervention to relieve anxiety and pain in adults undergoing cardiac surgery: a systematic review and meta-analysis

Open Heart ◽  
2021 ◽  
Vol 8 (1) ◽  
pp. e001474
Author(s):  
Ellaha Kakar ◽  
Ryan J Billar ◽  
Joost van Rosmalen ◽  
Markus Klimek ◽  
Johanna J M Takkenberg ◽  
...  
Keyword(s):  
Systematic Review ◽  
Cardiac Surgery ◽  
Rating Scale ◽  
Meta Analysis ◽  
Numeric Rating Scale ◽  
Hospital Length ◽  
Hospital Length Of Stay ◽  
Music Intervention ◽  
Opioid Use ◽  
Beneficial Effects

ObjectivesPrevious studies have reported beneficial effects of perioperative music on patients’ anxiety and pain. We performed a systematic review and meta-analysis of randomised controlled trials investigating music interventions in cardiac surgery.MethodsFive electronic databases were systematically searched. Primary outcomes were patients’ postoperative anxiety and pain. Secondary outcomes were hospital length of stay, opioid use, vital parameters and time on mechanical ventilation. PRISMA guidelines were followed and PROSPERO database registration was completed (CRD42020149733). A meta-analysis was performed using random effects models and pooled standardised mean differences (SMD) with 95% confidence intervals were calculated.ResultsTwenty studies were included for qualitative analysis (1169 patients) and 16 (987 patients) for meta-analysis. The first postoperative music session was associated with significantly reduced postoperative anxiety (SMD = –0.50 (95% CI –0.67 to –0.32), p<0.01) and pain (SMD = –0.51 (95% CI –0.84 to –0.19), p<0.01). This is equal to a reduction of 4.00 points (95% CI 2.56 to 5.36) and 1.05 points (95% CI 0.67 to 1.41) on the State-Trait Anxiety Inventory and Visual Analogue Scale (VAS)/Numeric Rating Scale (NRS), respectively, for anxiety, and 1.26 points (95% CI 0.47 to 2.07) on the VAS/NRS for pain. Multiple days of music intervention reduced anxiety until 8 days postoperatively (SMD = –0.39 (95% CI –0.64 to –0.15), p<0.01).ConclusionsOffering recorded music is associated with a significant reduction in postoperative anxiety and pain in cardiac surgery. Unlike pharmacological interventions, music is without side effects so is promising in this population.

A Pilot Study of the Government Pharmaceutical Organization (GPO) Cannabis Extract for Multiple Sclerosis (MS) Spasticity Treatment in Thailand

2021 ◽  
Vol 104 (3) ◽  
pp. 460-465
Keyword(s):  
Multiple Sclerosis ◽  
Pilot Study ◽  
Rating Scale ◽  
Final Analysis ◽  
Numeric Rating Scale ◽  
Secondary Outcome ◽  
Pharmaceutical Organization ◽  
Cannabis Extract ◽  
The Government ◽  
Numeric Rating

Background: The prevalence of spasticity in multiple sclerosis (MS) patients is nearly 90%. Most patients do not respond to current anti-spastic medications. Objective: To evaluate the efficacy and safety of Government Pharmaceutical Organization cannabis extract (GPOCE) in the treatment of spasticity in MS patients in Thailand. Materials and Methods: This prospective pilot study in patients diagnosed with MS whose spasticity was not relieved under current spasticity treatments, was performed between November 2019 and June 2020. The GPOCE formulation of THC:CBD 1:1 was administered to all patients. The treatment outcomes were determined at 12 weeks and compared with their baseline. Results: Seven patients participated in the present study. Among these, two patients withdrew after receiving only a small dose of GPOCE. Finally, five patients were included in the final analysis. The primary outcome was a reduction in the Modified Ashworth Score (MAS), which decreased among participants from a baseline of 15 (IQR 12 to 19) to 6 (IQR 1 to 12) (p=0.043). The key secondary outcome was a clinically relevant response (CRR), which was defined as a reduction of the spasticity Numeric Rating Scale (NRS) of more than thirty percent compared to baseline. Four patients (80%) achieved CRR. Moreover, the overall spasticity NRS decreased from a median of 6 (IQR5 to 7) to 2 (IQR2 to 3). A reduction of other NRS parameters, including fatigue, pain, tremor, sleep, spasm, anxiety, and depression, was also observed after treatment. Moreover, GPOCE was generally well tolerated. Conclusion: GPOCE is useful in treating spasticity in patients with MS. The safety profile is acceptable under the supervision of a health care provider. Keywords: Multiple sclerosis (MS), Cannabis extract, Spasticity

scholarly journals Real-world adherence to oral anticoagulants in atrial fibrillation patients: a study protocol for a systematic review and meta-analysis

BMJ Open ◽  
2018 ◽  
Vol 8 (12) ◽  
pp. e025102 ◽  
Author(s):  
Clara L Rodríguez-Bernal ◽  
Aníbal García-Sempere ◽  
Isabel Hurtado ◽  
Yared Santa-Ana ◽  
Salvador Peiró ◽  
...  
Keyword(s):  
Atrial Fibrillation ◽  
Systematic Review ◽  
Real World ◽  
Observational Studies ◽  
Meta Analysis ◽  
Oral Anticoagulants ◽  
Medication Possession Ratio ◽  
Essential Element ◽  
Study Endpoint ◽  
Mortality And Morbidity

IntroductionAtrial fibrillation (AF) is one of the leading causes of cerebrovascular mortality and morbidity. Oral anticoagulants (OACs) have been shown to reduce the incidence of cardioembolic stroke in patients with AF, adherence to treatment being an essential element for their effectiveness. Since the release of the first non-vitamin K antagonist oral anticoagulant, several observational studies have been carried out to estimate OAC adherence in the real world using pharmacy claim databases or AF registers. This systematic review aims to describe secondary adherence to OACs, to compare adherence between OACs and to analyse potential biases in OAC secondary adherence studies using databases.Methods and analysisWe searched on PubMed, SCOPUS and Web of Science databases (completed in 26 September 2018) to identify longitudinal observational studies reporting days’ supply adherence measures with OAC in patients with AF from refill databases or AF registers. The main study endpoint will be the percentage of patients exceeding the 80% threshold in proportion of days covered or the medication possession ratio. Two reviewers will independently screen potential studies and will extract data in a structured format. A random-effects meta-analysis will be carried out to pool study estimates. The risk of bias will be assessed using the Newcastle-Ottawa Scale for observational studies and we will also assess some study characteristics that could affect days’ supply adherence estimates.Ethics and disseminationThis systematic review using published aggregated data does not require ethics approval according to Spanish law and international regulations. The final results will be published in a peer-review journal and different social stakeholders, non-academic audiences and patients will be incorporated into the diffusion activities.PROSPERO registration numberCRD42018095646.

scholarly journals Ocrelizumab in Multiple Sclerosis: A Real-World Study From Spain

2021 ◽  
Vol 11 ◽  
Author(s):  
Angel P. Sempere ◽  
Leticia Berenguer-Ruiz ◽  
Ines Borrego-Soriano ◽  
Amparo Burgos-San Jose ◽  
Luis Concepcion-Aramendia ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Practice ◽  
Real World ◽  
Median Number ◽  
Progressive Multiple Sclerosis ◽  
Primary Progressive Multiple Sclerosis ◽  
Clinical Practice Setting ◽  
Number Of Treatment ◽  
Relapsing Multiple Sclerosis

Objectives: The aim of this study was to describe the tolerability, safety, and effectiveness of ocrelizumab for primary progressive multiple sclerosis (PPMS) and relapsing multiple sclerosis (RMS) in a clinical practice setting.Methods: In this retrospective observational study, we analyzed clinical and MRI data in all patients with PPMS and RMS who had received at least one infusion of ocrelizumab in two health areas in south-eastern Spain. Patients involved in any ocrelizumab trial and those patients with a follow-up shorter than 6 months were excluded.Results: The cohort included 70 patients (42 women) who had received ocrelizumab; 30% had PPMS and 70%, RMS. At baseline, patients' mean age was 47.1 years in the PPMS group and 39.2 years in the RMS group, while the median EDSS was 3.0 and 2.5, respectively. Median follow-up was 13.6 months. The median number of treatment cycles was three. Most patients remained free from clinical and MRI activity after ocrelizumab initiation. Baseline MRI showed T1 Gd-enhancing lesions in 57% of the patients; by the first MRI control at 4–6 months, all patients except one were free of T1 Gd-enhancing lesions (69/70, 98.6% P &lt; 0.001). The proportion of patients with NEDA was 94% in the group of RMS patients who were followed for at least 1 year. Ocrelizumab was generally well-tolerated; the most common adverse events were infusion-related reactions and infections, none of which were serious.Conclusions: Our real-world study supports the tolerability, safety, and effectiveness of ocrelizumab in clinical practice.

Alemtuzumab treatment of multiple sclerosis in real-world clinical practice: A report from a single Italian center

2020 ◽  
Vol 38 ◽  
pp. 101504 ◽  
Author(s):  
M. di Ioia ◽  
V. Di Stefano ◽  
D. Farina ◽  
V. Di Tommaso ◽  
D. Travaglini ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Practice ◽  
Real World

scholarly journals Apixaban for Stroke Prevention in Atrial Fibrillation: Why are Event Rates Higher in Clinical Practice than in Randomized Trials?—A Systematic Review

2020 ◽  
Vol 120 (09) ◽  
pp. 1323-1329 ◽  
Author(s):  
Tim A. C. de Vries ◽  
Jack Hirsh ◽  
Ke Xu ◽  
Imaad Mallick ◽  
Vinai C. Bhagirath ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Practice ◽  
Thromboembolic Event ◽  
Meta Analysis ◽  
Patient Characteristics ◽  
Thromboembolic Events ◽  
Off Label Use ◽  
Off Label ◽  
Event Rates ◽  
Label Use

Abstract Background Recent reports suggest an important contribution from frequent off-label use of apixaban 2.5 mg twice daily to the higher rates of thromboembolic events observed in observational studies (OSs) relative to in randomized controlled trials (RCTs), and consequently, advocate against such use in all patients. Objectives To examine factors contributing to the higher thromboembolic event rates, we estimated the prevalence of off-label use in contemporary practice, and compared patient characteristics and rates of stroke/systemic embolism, major bleeding, and mortality by apixaban dose and by study design in a systematic review and meta-analysis. Results and Discussion We identified 18 OSs and 2 RCTs that included 155,228 and 11,928 patients, respectively. Patients in OSs more often received apixaban 2.5 mg twice daily (31.3% vs. 5.1%), were older (mean age 73.8 vs. 69.8 years), and had higher CHA2DS2-VASc scores (mean 3.6 vs. 2.9) versus those in RCTs. We observed a consistent pattern of higher rates of thromboembolic events, bleeding, and mortality in patients treated with 2.5 versus 5 mg twice daily apixaban in both OSs and RCTs. Conclusion The higher risk profiles of patients in OSs versus RCTs, and higher rates of both bleeding and mortality not attributable to thromboembolism in patients treated with apixaban 2.5 versus 5 mg twice daily suggest that differences in patient characteristics are additional important contributors to the higher than expected thromboembolic event rates in clinical practice.

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