scholarly journals BASELINE FRAILTY EVALUATION IN DRUG DEVELOPMENT

2016 ◽  
pp. 1-2
Author(s):  
F. CERRETA ◽  
J. ANKRI ◽  
D. BOWEN ◽  
A. CHERUBINI ◽  
A.J. CRUZ JENTOFT ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Older Adult ◽  
Inappropriate Prescribing ◽  
Evidence Base ◽  
Age Group ◽  
Market Authorisation ◽  
Frail Older Adult ◽  
Age Limits ◽  
Physician Patient ◽  
Family Preference

Older people are high consumers of medicines, but despite this they have often been excluded from clinical trials. Reasons for exclusion have frequently been poorly justifiable, and have included predefined arbitrary upper age limits, lists of different comorbidities, polypharmacy or physician/patient/family preference. This selection bias is even more evident for the frail older adults. Consequently, the evidence base in this age group is often limited at the time of market authorisation. The benefit/risk analysis for the frail older adult may well be different from that of younger people, and a lack of data may result in inappropriate prescribing.

Disparities in older adult accrual to cancer trials: Analysis from the alliance for clinical trials in oncology (A151736)

2021 ◽  
Author(s):  
Noam A. VanderWalde ◽  
Travis Dockter ◽  
Daniel V. Wakefield ◽  
Daniel Satele ◽  
Jeff Sloan ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Older Adult ◽  
Cancer Trials

scholarly journals Improving the External Validity of Clinical Trials: The Case of Multiple Chronic Conditions

2013 ◽  
Vol 3 (2) ◽  
pp. 30-35 ◽  
Author(s):  
Fortin Martin ◽  
Smith M. Susan
Keyword(s):  
Clinical Trials ◽  
External Validity ◽  
Chronic Conditions ◽  
Evidence Base ◽  
Specific Reference ◽  
Multiple Chronic Conditions ◽  
Patient Centered ◽  
Department Of Health ◽  
Patient Groups ◽  
Intervention Trials

The U.S. Department of Health and Human Services vision and strategic framework on multiple chronic conditions (MCCs) incorporates recommendations designed to facilitate research that will improve our knowledge about interventions and systems that will benefit individuals with MCCs (or multimorbidity). The evidence base supporting the management of patients with MCCs will be built through intervention trials specifically designed to address multimorbidity and identification of MCCs in participants across the clinical trial range. This article specifically focuses on issues relating to external validity with specific reference to trials involving patients with MCCs. The exclusion of such patients from clinical trials has been well documented. Randomized control trials (RCTs) are considered the “gold standard” of evidence, but may have drawbacks in relation to external validity, particularly in relation to multimorbidity. It may, therefore, be necessary to consider a broader range of research methods that can provide converging evidence on intervention effects to address MCCs. Approaches can also be taken to increase the usefulness of RCTs in general for providing evidence to inform multimorbidity management. Additional improvements to RCTs would include better reporting of inclusion and exclusion criteria and participant characteristics in relation to MCCs. New trials should be considered in terms of how they will add to the existing evidence base and should inform how interventions may work in different settings and patient groups. Research on treatments and interventions for patients with MCCs is badly needed. It is important that this research includes patient-centered measures and that generalizability issues be explicitly addressed.

scholarly journals Evidence-based practice of stereotactic radiosurgery: Outcomes from an educational course for neurosurgery and radiation oncology residents

2021 ◽  
Vol 12 ◽  
pp. 77
Author(s):  
Swathi Chidambaram ◽  
Sergio W. Guadix ◽  
John Kwon ◽  
Justin Tang ◽  
Amanda Rivera ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Stereotactic Radiosurgery ◽  
Radiation Oncology ◽  
Evidence Based Practice ◽  
Evidence Base ◽  
Comfort Level ◽  
Evidence Based ◽  
Senior Faculty ◽  
Exact Test ◽  
Before And After

Background: As the field of brain and spine stereotactic radiosurgery (SRS) continues to grow, so will the need for a comprehensive evidence base. However, it is unclear to what degree trainees feel properly equipped to use SRS. We assess the perceptions and comfort level reported by neurosurgery and radiation oncology residents concerning the evidence-based practice of SRS. Methods: A continuing medical education (CME) course provided peer-reviewed updates regarding treatment with intracranial and spinal SRS. Presentations were given by neurosurgery and radiation oncology residents with mentorship by senior faculty. To gauge perceptions regarding SRS, attendees were surveyed. Responses before and after the course were analyzed using the Fisher’s exact test in R statistical software. Results: Participants reported the greatest knowledge improvements concerning data registries (P < 0.001) and clinical trials (P = 0.026). About 82% of all (n = 17) radiation oncology and neurosurgery residents either agreed or strongly agreed that a brain and spine SRS rotation would be beneficial in their training. However, only 47% agreed or strongly agreed that one was currently part of their training. In addition, knowledge gains in SRS indications (P = 0.084) and ability to seek collaboration with colleagues (P = 0.084) showed notable trends. Conclusion: There are clear knowledge gaps shared by potential future practitioners of SRS. Specifically, knowledge regarding SRS data registries, indications, and clinical trials offer potential areas for increased educational focus. Furthermore, the gap between enthusiasm for increased SRS training and the current availability of such training at medical institutions must be addressed.

scholarly journals Patterns and Predictors of Emicizumab Adherence in People with Hemophilia

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 2178-2178
Author(s):  
Ang Li ◽  
Catlin Goodfriend ◽  
John Sokol ◽  
Rebecca Kruse-Jarres
Keyword(s):  
Clinical Trials ◽  
Half Life ◽  
Clinical Care ◽  
Multivariable Analysis ◽  
Skewed Distribution ◽  
Prior Exposure ◽  
Age Group ◽  
Active Inhibitor ◽  
Routine Prophylaxis

Introduction: Emicizumab is a subcutaneously administered, humanized bispecific monoclonal antibody that is recently approved for hemostatic prophylaxis in people with hemophilia A (PWH) with or without factor VIII inhibitors. We hypothesize that the new route and frequency of administration would lead to better treatment adherence compared to factor or bypass products in PWH outside of clinical trials. We performed the current study to test the hypothesis and to examine potential predictors of non-adherence associated with emicizumab treatment. Methods: We performed a retrospective cohort study at the Washington Center for Bleeding Disorders. Inclusion criteria included PWH with moderate to severe hereditary hemophilia (FVIII <5%), clinician recommendation for routine prophylaxis, receipt of clinical care and medication from a hemophilia treatment center (HTC), at least 12 months of prior exposure to factor or bypass products, and at least 3 months of emicizumab treatment. For patients who were previously enrolled in emicizumab clinical trials, adherence data were collected from the time of first commercial product administration. Adherence percentage (%) was estimated as days of drug dispensed / days elapsed x 100 and non-adherence % was determined as 100 - adherence %. Relevant patient, condition, treatment, and socioeconomic variable data were collected from review of medical records. To assess the difference in drug adherence, we compared the adherence % of emicizumab versus that of factor or bypass product in the same patients using the paired t test. To assess predictors of non-adherence %, we used a generalized linear model (GLM) with log link and gamma distribution to account for the right skewed distribution of the outcome. A multivariable GLM was built to incorporate the most significant predictors of non-adherence. Results: We identified 56 PWH that initiated commercial emicizumab from 1/2018 to 5/2019 at our HTC. Five patients were excluded for fewer than 3 months of follow-up on treatment and 3 patients were excluded for not having prior exposure to factor therapy. The remaining 48 patients had a median duration on emicizumab of 7 months (IQR 5-9) at the time of study. The most common dosing frequency was weekly administration (77%). The median age at treatment index date was 17 years (IQR 9-36), 65% were Caucasian, and 46% had Medicaid or Medicare insurance. The majority of patients had a diagnosis of severe hemophilia (90%), 42% had a history of inhibitor (15% active inhibitor), and 25% were previously enrolled in an emicizumab interventional trial. Prior to emicizumab initiation, 46% of patients had 5 or more self-reported annualized bleeds. The most common reason for emicizumab initiation was patient preference (35%), followed by breakthrough bleeding (33%), difficult venipuncture (21%), and shortened factor half-life (10%). Among 12 patients who previously received only on demand treatment, their adherence on emicizumab was 89%. Among 36 patients who previously received routine prophylaxis, their adherence was significantly higher on emicizumab (98%) than factor/bypass products (89%) (p=0.002). Specifically, 18 out of 48 patients (38%) had factor/bypass adherence <75% (or refused prophylaxis) and 2 out of 48 patients (4%) had emicizumab adherence <75%. Various factors were associated with increased emicizumab non-adherence (Table 1). Age group had the strongest association where young and older adult PWH had more non-adherence % than children and adolescents (Figure 1). On multivariable analysis, age group, active inhibitor, and prior factor/bypass agent non-adherence (episodic or <75% usage) were significantly associated with increased emicizumab non-adherence. Conclusions: In the current study, we found that PWH requiring routine prophylaxis were more likely to be adherent to emicizumab than previous factor or bypass agents. Age group (young adult), active inhibitor, and prior non-adherence to factor product were significant predictors for decreased emicizumab adherence but the differences were small. Given the long half-life of the drug, the significance of non-perfect adherence on bleeding outcomes needs to be studied prospectively with longer clinical follow-up. Disclosures No relevant conflicts of interest to declare.

scholarly journals Prospective Population-Based Analysis of Characteristics and Therapy Options in AML: The Case of Catalonia (PERIS Project)

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 32-33
Author(s):  
Montserrat Hoyos ◽  
Ana Garrido ◽  
Montserrat Arnan ◽  
Marina Diaz-Beyá ◽  
Olga Salamero ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Board Of Directors ◽  
Hematopoietic Cell Transplantation ◽  
Clinical Laboratory ◽  
Laboratory Data ◽  
Population Based ◽  
Advanced Age ◽  
Age Group ◽  
Advisory Committees ◽  
Low Intensity

Introduction:Acute myeloid leukemia (AML) real-world incidence has been investigated in a limited number of European countries such as Sweden, Denmark and a few others. These prospective population-based analyses give a more precise idea of AML as a health problem than registry data from cooperative groups or other sources, that usually include selected cases as part of research studies and/or therapy trials. In October 2016, the Autonomous Government of Catalonia funded a project (PERIS SLT002/16/00433) to prospectively collect all AML cases from our territory. Objective:To investigate the incidence, characteristics and treatment decisions in all consecutive AML patients diagnosed in Catalonia between January 2017 and December 2019. Methods:Inclusion criteria were diagnosis of AML according the WHO 2016 criteria, both primary and secondary (APL excluded) with an age &gt;18 years. The project was disseminated to all hospitals from Catalonia regardless of their size, having at least one hematologist. A specific informatics tool was implemented for remote reporting of the cases. All data were anonymized. In parallel, a circuit for centralized bio banking of patients' samples was designed. The database included the main clinical, laboratory data as well as the initial therapeutic approach. Cases included in our CETLAM group cooperative studies were automatically linked to the trial database for collecting detailed information. Statistical analyses were performed with R packages. Results:Assuming an incidence of AML of 4 cases per 100,000 inhabitants (based on previous reported data from others), we expected 912 cases during three years in the 7,6 million population of Catalonia. Our prospective registry included 750 consecutive AML patients, 82% of the expected cases. The remaining 18% could be explained by the exclusion of APL, age below 18 years, or underreporting. Seventy percent of patients (n=527) were diagnosed and treated in the 5 large University Hospitals from Barcelona and the two adjacent cities (Badalona and Hospitalet). Table 1 shows the main characteristics of the patients. Among the 390 patients up to 70 years, 272 (70%) were enrolled in the CETLAM AML-12 protocol that included intensive chemotherapy (ICT) and risk adapted hematopoietic cell transplantation (HCT). Forty-one additional patients (11%) in this age group received other ICT in different clinical trials. A remaining 73 patients (20%) were treated with other intensive or non-intensive approaches outside trials. In the group of 360 patients older than 70 years only a 33% (n= 119) were treated under the risk-adapted CETLAM AML-16 protocol for elderly AML patients. This trial included ICT as in the CETLAM-12 in case of favorable genetic features; this was received by 13 of the 119 patients (11%) enrolled. The remaining patients of CETLAM-16 were treated with low-intensity chemotherapy (oral fludarabine, subcutaneous (SC) cytarabine and G-CSF or azacytidine) and 97 additional elderly patients were included in other clinical trials mostly with targeted and hypomethylating agents (27%). Other active therapies outside trials (usually low-intensity) were administered in 50 additional patients (14%) whereas the remaining 94 patients (26%) only received supportive measures (transfusions, hydroxyurea, antibiotics, palliation, or no treatment), because of one or more of the following: advanced age, poor AML features or severe clinical condition. Overall survival (OS) of the whole series at 2 years was 31±2% (CI: 27-35). Patients younger than 70 years had a 2-year OS of 47±3% (CI: 41-53) compared to 11±3% (CI: 7-17) for those above 70 years (p&lt;0.001) (Image 1). Conclusions:This prospective study is highly representative of the diagnosis and treatment of AML in Catalonia. The median age at diagnosis was 70 years. Of note, 81% of patients up to 70 years were enrolled in ICT trials. The proportion of patients in trials in the elderly group was lower although still remarkable (60%). In this advanced age group, a 26% of patients were treated with supportive measures only. Despite the high inclusion rate in clinical trials, only one third of newly diagnosed AML patients have the probability to survive at 2 years, with a dismal outcome in those above 70 years. Therefore, the investigation of novel and more effective treatments remains mandatory. This series will be detailed and updated during the meeting. Disclosures Salamero: Pfizer:Consultancy;Jazz Pharmaceuticals:Consultancy, Honoraria;Daichii Sankyo:Honoraria;Novartis:Consultancy, Honoraria;Celgene:Consultancy, Honoraria.Olivera:BAYER:Consultancy;Pfizer:Consultancy, Speakers Bureau;Daiichi Sankyo:Consultancy, Speakers Bureau;Boehringer Ingelheim:Consultancy, Speakers Bureau.Sureda Balari:Celgene:Consultancy, Honoraria;Merck Sharpe and Dohme:Consultancy, Honoraria, Speakers Bureau;Sanofi:Consultancy, Honoraria;Novartis:Consultancy, Honoraria;Gilead/Kite:Consultancy, Honoraria;Janssen:Consultancy, Honoraria;Incyte:Consultancy;Roche:Honoraria;BMS:Speakers Bureau;Celgene/Bristol-Myers Squibb:Consultancy, Honoraria;Takeda:Consultancy, Honoraria, Speakers Bureau.Ribera:Pfizer, Amgen:Research Funding;Pfizer, Amgen, Ariad, Novartis:Consultancy, Speakers Bureau.Sierra:Jazz Pharmaceuticals:Research Funding;Pfizer:Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau;Daiichi Sankyo:Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau;Abbvie:Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau;Novartis:Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau;Astellas:Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees;Roche:Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees;Gilead-Kite:Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees.

scholarly journals 94 Association between Dual-Task Timed “UP and GO” Test and Occurrence of Falls According to Age Group (Young-Older or Old-Older Adults): A Longitudinal Observation Study

2019 ◽  
Vol 48 (Supplement_4) ◽  
pp. iv18-iv27
Author(s):  
Tsuyoshi Asai ◽  
Kensuke Oshima ◽  
Yoshihiro Fukumoto ◽  
Shogo Misu
Keyword(s):  
Older Adults ◽  
Older Adult ◽  
Dual Task ◽  
Community Dwelling ◽  
Age Group ◽  
Adult Group ◽  
Observation Study ◽  
Timed Up And Go ◽  
Community Dwelling Older Adults

Abstract Aim To elucidate the association between the occurrence of falls and timed “up and go” (TUG) test score in a dual-task condition among community-dwelling older adults by age group. Methods This longitudinal observation study included 987 community-dwelling older adults at baseline. A TUG test (single-TUG) and a TUG test while counting aloud backward from 100 (dual-TUG) were conducted at baseline. The dual-task cost (DTC) value was computed from these results. Data on fall history were obtained using a self-administered questionnaire at the 1-year follow-up. At follow-up, 322 participants had dropped out and six participants had missing data for falls. The final analysis included 658 individuals (follow-up rate: 658/987, 67%) divided into a young-older adult group (aged 60–74 years) and an old-older adult group (aged 75 years or older). Associations between the occurrence of falls and TUG-related values were analyzed by age group using multivariate logistic regression models. Results For old-older adults, there were significant associations between the occurrence of falls and DTC value (odds ratio [OR] 0.981, 95% confidence interval [CI]: 0.963–0.999, p = 0.040) and single-TUG score (OR 1.129, 95% CI: 1.006–1.268, p = 0.039). However, no significant associations were observed for young-older adults. Conclusions Slower single-TUG test score and lower DTC value are associated with the occurrence of falls among old-older adults but not among young-older adults. Dual task assessment is useful for predicting falls in TUG fall assessment for old-older adults.

scholarly journals Treatment with CPAP in Elderly Patients with Obstructive Sleep Apnoea

2020 ◽  
Vol 9 (2) ◽  
pp. 546 ◽  
Author(s):  
Tomas Posadas ◽  
Grace Oscullo ◽  
Enrique Zaldívar ◽  
Alberto Garcia-Ortega ◽  
José Daniel Gómez-Olivas ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Elderly Patients ◽  
Obstructive Sleep Apnoea ◽  
The Elderly ◽  
Sleep Apnoea ◽  
Age Group ◽  
Obstructive Sleep ◽  
Receive Treatment ◽  
Therapeutic Tools

The population pyramid is changing as a result of the ever-increasing life expectancy, which makes it crucial to acquire an in-depth understanding of the diseases that most often affect the elderly. Obstructive sleep apnoea (OSA) affects 15%–20% of the population aged over 65 years. Despite this prevalence, there have been very few specific studies on the management of OSA in this age group, even though over 60% of the patients aged over 65-70 years who attend sleep units with suspicion of OSA receive treatment with continuous positive airway pressure (CPAP), on the basis of an extrapolation of the positive results achieved by CPAP in clinical trials involving middle-aged males. However, the latter’s form of presentation, evolution and, probably, prognosis comparing with OSA are not the same as those of elderly patients. Recent clinical trials performed on an exclusive series of elderly patients have shed light on the possible role of CPAP treatment in elderly patients with OSA, but there are still many questions that need to be answered. The physiological increase in the number of sleep-related disorders with the passing of years, and the lack of validated diagnostic and therapeutic tools for this age group are probably the greatest obstacles to define, diagnose and treat OSA in the elderly.

The research evidence base for homeopathy: a fresh assessment of the literature

Homeopathy ◽  
2003 ◽  
Vol 92 (02) ◽  
pp. 84-91
Author(s):  
RT Mathie
Keyword(s):  
Clinical Trials ◽  
Research Evidence ◽  
Controlled Trial ◽  
Relative Number ◽  
Evidence Base ◽  
Weight Of Evidence ◽  
Clinical Effects ◽  
Upper Respiratory Tract ◽  
Medical Conditions ◽  
Double Blind

Abstract Background. The claims made for the clinical effects of homeopathy are controversial. The results of several meta-analyses of clinical trials are positive, but they fail in general to highlight specific medical conditions that respond well to homeopathy. Aims. This review examines the cumulative research from randomised and/or double-blind clinical trials (RCTs) in homeopathy for individual medical conditions reported since 1975, and asks the question: What is the weight of the original evidence from published RCTs that homeopathy has an effect that is statistically significantly different from that in a comparative group? Method. Analysis of the 93 substantive RCTs that compare homeopathy either with placebo or another treatment. Results. 50 papers report a significant benefit of homeopathy in at least one clinical outcome measure, 41 that fail to discern any inter-group differences, and two that describe an inferior response with homeopathy. Considering the relative number of research articles on the 35 different medical conditions in which such research has been carried out, the weight of evidence currently favours a positive treatment effect in eight: childhood diarrhoea, fibrositis, hayfever, influenza, pain (miscellaneous), side-effects of radio- or chemotherapy, sprains and upper respiratory tract infection. Based on published research to date, it seems unlikely that homeopathy is efficacious for headache, stroke or warts. Insufficient research prevents conclusions from being drawn about any other medical conditions. Conclusions. The available research evidence emphasises the need for much more and better-directed research in homeopathy. A fresh agenda of enquiry should consider beyond (but include) the placebo-controlled trial. Each study should adopt research methods and outcome measurements linked to a question addressing the clinical significance of homeopathy's effects.

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