scholarly journals Accessing Investigational Products Outside of a Trial: Considerations for Neuromuscular Providers

2021 ◽  
Vol 23 (12) ◽  
Author(s):  
R. S. Bedlack ◽  
M. E. Cudkowicz ◽  
S. Paganoni ◽  
H. Khan-Tareen ◽  
J. A. Andrews
Keyword(s):  
Neuromuscular Diseases ◽  
Recent Survey ◽  
Inclusion Criteria ◽  
Design Features ◽  
Off Label ◽  
Expanded Access ◽  
Patient Request ◽  
The Right ◽  
Access Programs ◽  
Lack Of Knowledge

Abstract Purpose of Review People with fatal neuromuscular diseases such as ALS want to access investigational products. Trials are our preferred pathway for this, but most people with these diseases will not be able to participate due to restrictive inclusion criteria, travel burdens, or design features they will not accept. This leaves FDA Expanded Access Programs (EAPs), the Right To Try (RTT) pathway, and self-purchase of alternative and off-label treatments (AOTs). Recent Findings A recent survey highlighted physician barriers to the above pathways, including lack of knowledge and concerns about time burdens and risks. Emerging resources are highlighted that can mitigate some of these concerns. Summary With the information in this chapter, we hope that neuromuscular clinicians will feel more knowledgeable and confident in supporting patient request for investigational products.

Emotion Recognition Deficits in Psychiatric Disorders as a Target of Non-invasive Neuromodulation: A Systematic Review

2021 ◽  
pp. 155005942199168
Author(s):  
Yuji Yamada ◽  
Takuma Inagawa ◽  
Naotsugu Hirabayashi ◽  
Tomiki Sumiyoshi
Keyword(s):  
Systematic Review ◽  
Social Cognition ◽  
Emotion Recognition ◽  
Psychiatric Disorders ◽  
Parietal Lobe ◽  
Repetitive Transcranial Magnetic Stimulation ◽  
Inclusion Criteria ◽  
Cognitive Benefits ◽  
The Right ◽  
English Articles

Background. Social cognition deficits are a core feature of psychiatric disorders, such as schizophrenia and mood disorder, and deteriorate the functionality of patients. However, no definite strategy has been established to treat social cognition (eg, emotion recognition) impairments in these illnesses. Here, we provide a systematic review of the literature regarding transcranial direct current stimulation (tDCS) and repetitive transcranial magnetic stimulation (rTMS) for the treatment of social cognition deficits in individuals with psychiatric disorders. Methods. A literature search was conducted on English articles identified by PubMed, PsycINFO, and Web of Science databases, according to the guidelines of the PRISMA statement. We defined the inclusion criteria as follows: (1) randomized controlled trials (RCTs), (2) targeting patients with psychiatric disorders (included in F20-F39 of the 10th revision of the International Statistical Classification of Diseases and Related Health Problems [ICD-10]), (3) evaluating the effect of tDCS or rTMS, (4) reporting at least one standardized social cognition test. Results. Five papers (3 articles on tDCS and 2 articles on rTMS) met the inclusion criteria which deal with schizophrenia or depression. The significant effects of tDCS or rTMS targeting the left dorsolateral prefrontal cortex on the emotion recognition domain were reported in patients with schizophrenia or depression. In addition, rTMS on the right inferior parietal lobe was shown to ameliorate social perception impairments of schizophrenia. Conclusions. tDCS and rTMS may enhance some domains of social cognition in patients with psychiatric disorders. Further research is warranted to identify optimal parameters to maximize the cognitive benefits of these neuromodulation methods.

scholarly journals Canadian Recommendations for Clinical Trials of Pharmacologic Interventions in Rheumatoid Arthritis: Inclusion Criteria and Study Design: Table 1.

2011 ◽  
Vol 38 (10) ◽  
pp. 2095-2104 ◽  
Author(s):  
JACOB KARSH ◽  
EDWARD C. KEYSTONE ◽  
BOULOS HARAOUI ◽  
J. CARTER THORNE ◽  
JANET E. POPE ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Disease Activity ◽  
Study Design ◽  
New Drugs ◽  
Nominal Group ◽  
Consensus Method ◽  
Inclusion Criteria ◽  
Design Features

Objective.Current clinical trial designs for pharmacologic interventions in rheumatoid arthritis (RA) do not reflect the innovations in RA diagnosis, treatment, and care in countries where new drugs are most often used. The objective of this project was to recommend revised entry criteria and other study design features for RA clinical trials.Methods.Recommendations were developed using a modified nominal group consensus method. Canadian Rheumatology Research Consortium (CRRC) members were polled to rank the greatest challenges to clinical trial recruitment in their practices. Initial recommendations were developed by an expert panel of rheumatology trialists and other experts. A scoping study methodology was then used to examine the evidence available to support or refute each initial recommendation. The potential influence of CRRC recommendations on primary outcomes in future trials was examined. Recommendations were finalized using a consensus process.Results.Recommendations for clinical trial inclusion criteria addressed measures of disease activity [Disease Activity Score 28 using erythrocyte sedimentation rate (DAS28-ESR) > 3.2 PLUS ≥ 3 tender joints using 28-joint count (TJC28) PLUS ≥ 3 swollen joint (SJC28) OR C-reactive protein (CRP) or ESR > upper limit of normal PLUS ≥ 3 TJC28 PLUS ≥ 3 SJC28], functional classification, disease classification and duration, and concomitant RA treatments. Additional recommendations regarding study design addressed rescue strategies and longterm extension.Conclusion.There is an urgent need to modify clinical trial inclusion criteria and other study design features to better reflect the current characteristics of people living with RA in the countries where the new drugs will be used.

scholarly journals Is It Time to Rethink the Expanded‐Access Programs for HIV Infection?

2007 ◽  
Vol 196 (7) ◽  
pp. 974-977 ◽  
Author(s):  
Valerianna Amorosa ◽  
Pablo Tebas
Keyword(s):  
Hiv Infection ◽  
Expanded Access ◽  
Access Programs

scholarly journals Restorative minimally invasive interventions in patients with colostomy

2018 ◽  
Vol 99 (6) ◽  
pp. 1004-1008
Author(s):  
F Sh Akhmetzyanov ◽  
N A Valiev ◽  
A N Daminov ◽  
B Sh Bikbov
Keyword(s):  
Surgical Intervention ◽  
Surgical Trauma ◽  
Distal Stump ◽  
Inclusion Criteria ◽  
Exclusion Criteria ◽  
Intestinal Continuity ◽  
Intestinal Stoma ◽  
The Right ◽  
Medical Indications ◽  
Oncology Center

Aim. To show the benefits of performing mini-access restorative phase on the colon in patients with colostomy. Methods. A retrospective analysis of the results of closure stomy operations in 2011-2017 in two emergency cancer departments of Republican Clinical Oncology Center MH RT was conducted. The inclusion criteria of the study were: 1) surgical intervention for acute intestinal obstruction in colorectal cancer performed in RCOC; 2) mandatory removal of the primary tumor during the first surgery; 3) the presence of a functioning intestinal stoma formed during the first surgery; 4) fixation of both stumps in one stoma. The exclusion criteria were refusal to restore the continuity of the colon for medical indications and the patient's refusal to undergo the surgery. Results. The study included 11 males (44 %) and 14 females (56 %). The age interval was 49 to 81 years, of which 12 patients were over 70 years old (48 %), the average age was 67.7 ± 5.4 years. The tumor removed at the first stage was localized in the right parts of the colon in 5 patients (20 %), and in the left in 20 patients (80 %). By the stages of the malignant process, the patients were distributed as follows: stage B - 14 cases (56 %), stage C - 10 (40 %), stage D - 1 (4 %). The average duration of the surgery was 53 ± 14.38 minutes (40 to 123). The postoperative period was 10.8 ± 1.92 days on average (5 to 18). Conclusion. Restoration of intestinal continuity through mini-paracolostomy access is technically rational, as due to the mini-access the patient undergoes minor surgical trauma; the anatomical proximity of the anastomosed bowel stumps excludes the difficulties associated with the search for the distal stump in the traditional method of recovery; the duration of the postoperative hospital stay decreases significantly.

Cost Comparison of Fan-out Wafer Level Packaging and Flip Chip Packaging

2017 ◽  
Vol 2017 (DPC) ◽  
pp. 1-37 ◽  
Author(s):  
Amy Lujan
Keyword(s):  
Flip Chip ◽  
Cost Effective ◽  
Cost Comparison ◽  
Cost Modeling ◽  
Wafer Level ◽  
Design Features ◽  
Wafer Level Packaging ◽  
Flip Chip Packaging ◽  
The Right ◽  
The Cost

In recent years, there has been increased focus on fan-out wafer level packaging with the growing inclusion of a variety of fan-out wafer level packages in mobile products. While fan-out wafer level packaging may be the right solution for many designs, it is not always the lowest cost solution. The right packaging choice is the packaging technology that meets design requirements at the lowest cost. Flip chip packaging, a more mature technology, continues to be an alternative to fan-out wafer level packaging. It is important for many in the electronic packaging industry to be able to determine whether flip chip or fan-out wafer level packaging is the most cost-effective option. This paper will compare the cost of flip chip and fan-out wafer level packaging across a variety of designs. Additionally, the process flows for each technology will be introduced and the cost drivers highlighted. A variety of package sizes, die sizes, and design features will be covered by the cost comparison. Yield is a key component of cost and will also be considered in the analysis. Activity based cost modeling will be used for this analysis. With this type of cost modeling, a process flow is divided into a series of activities, and the total cost of each activity is accumulated. The cost of each activity is determined by analyzing the following attributes: time required, labor required, material required (consumable and permanent), capital required, and yield loss. The goal of this cost comparison is to determine which design features drive a design to be packaged more cost-effectively as a flip chip package, and which design features result in a lower cost fan-out wafer level package.

scholarly journals Access to Investigational Drugs: FDA Expanded Access Programs or “Right-to-Try” Legislation?

2015 ◽  
Vol 8 (5) ◽  
pp. 526-532 ◽  
Author(s):  
M. E. Blair Holbein ◽  
Jelena P. Berglund ◽  
Kevin Weatherwax ◽  
David E. Gerber ◽  
Joan E. Adamo
Keyword(s):  
Expanded Access ◽  
Investigational Drugs ◽  
Access Programs

Neuromuscular Disorders and Anesthesia

2018 ◽  
Author(s):  
Mariel Manlapaz ◽  
Perin Kothari
Keyword(s):  
Perioperative Management ◽  
Neuromuscular Disorders ◽  
Neuromuscular Diseases ◽  
Tonic Contraction ◽  
Endocrine Dysfunction ◽  
Muscle Involvement ◽  
Disease States ◽  
Anesthetic Drugs ◽  
Anesthetic Considerations ◽  
The Right

The various neuromuscular diseases present with different airway, cardiovascular, pulmonary, and anesthetic considerations. It is useful to categorize these different diseases into nerve, neuromuscular junction, and primary muscle diseases. Understanding their pathophysiology is paramount in choosing the right anesthetic drugs (for example, depolarizing versus nondepolarizing and regional versus general anesthesia). Knowing their manifestations such as autonomic dysfunction, skeletal/cardiac/smooth/bulbar muscle involvement, or tendency for tonic contraction, allows for expectant perioperative management. Finally appreciating their association with certain disease states such as malignant hyperthermia or endocrine dysfunction can prevent complications. A brief review of myotonic dystrophy is presented here, followed by a brief summary of anesthetic considerations for various neuromuscular diseases.

scholarly journals Melanoma of the Right Foot Simulating Kaposi’s Disease

2015 ◽  
Vol 2015 ◽  
pp. 1-3
Author(s):  
K. A. Kouassi ◽  
K. Kassi ◽  
K. Kouamé ◽  
M. A. Oussou ◽  
I. Kouassi ◽  
...  
Keyword(s):  
Malignant Tumor ◽  
Clinical Presentation ◽  
Histopathological Examination ◽  
Partial Resection ◽  
Sub Saharan Africa ◽  
Visceral Metastasis ◽  
Atypical Clinical Presentation ◽  
Sub Saharan ◽  
The Right ◽  
Lack Of Knowledge

Melanoma is a malignant tumor rarely being described in sub-Saharan Africa. We reported an unusual and atypical clinical presentation. It was a 59-year-old patient who was hospitalized for a monomelic black tumor evolving for 10 years. Histopathological examination confirmed the melanocytic origin of this tumor. Paraclinical assessment did not find any visceral metastasis. A partial resection of the tumor was performed. The patient left the hospital against medical consent due to lack of technical facilities. The delay in the consultation and the lack of knowledge of melanoma by doctors and patients might contribute to the severity and the difficulties of its management.

Impact of Thrombophilia On Arterial Ischemic Stroke or Cerebral Venous Sinus Thromboses in Children: A Systematic Review & Meta-Analysis of Observational Studies.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 3993-3993
Author(s):  
Lisa K Lütkhoff ◽  
Manuela Albisetti ◽  
Timothy J. Bernard ◽  
Mariana Bonduel ◽  
Leonardo R. Brandao ◽  
...  
Keyword(s):  
Risk Factors ◽  
Ischemic Stroke ◽  
Random Effects ◽  
Antiphospholipid Antibodies ◽  
Observational Studies ◽  
Meta Analysis ◽  
Inclusion Criteria ◽  
Arterial Ischemic Stroke ◽  
Off Label ◽  
The Impact

Abstract Abstract 3993 Poster Board III-929 Background The incidence of stroke in children is estimated at about 2.6 per 100,000 per year. Risk factors include congenital heart malformations, trauma, hemolytic anemias, collagen tissue diseases, inborn metabolic disorders, and infectious diseases. Apart from acquired thrombophilic risk factors, such as the presence of antiphospholipid antibodies, inherited thrombophilias (IT) have been found to be associated with stroke in infants and children. However, results of single studies on the risk of stroke onset associated with IT have been contradictory or inconclusive, mainly due to lack of statistical power. The aim of this study was to estimate the impact of thrombophilia (IT) on risk of childhood stroke via meta-analysis of published observational studies. Methods and Results A systematic search of electronic databases (Medline, EMBASE, OVID, Web of Science, The Cochrane Library) for studies published from 1970 to 2009 was conducted using key words in combination both as MeSH terms and text words. Citations were independently screened by two authors and those meeting the a priori defined inclusion criteria were retained. Data on year of publication, study design, country of origin, number of patients/controls, ethnicity, stroke type (arterial ischemic stroke [AIS]; cerebral venous sinus thrombosis [CSVT]) were abstracted. Publication bias indicator and heterogeneity across studies were evaluated, and summary odds ratios (ORs) and 95% confidence intervals (CIs) were calculated using both fixed-effects and random-effects models. Twenty-one of 185 references found met inclusion criteria. 1698 patients (AIS: 1291; CSVT: 407) and 2913 controls aged neonate to 18 years were enrolled. No significant heterogeneity was discerned across studies, and no publication bias was detected. A statistically significant association with stroke onset was demonstrated for each IT trait evaluated, with no difference found between AIS (table) and CSVT. Summary ORs/CIs (random-effects model) for AIS & CSVT cohorts were as follows: Protein C-deficiency (8.76/4.53-16.96), FV G1691A (3.34/2.66-4.26), FII G20210A (2.50/1.67-3.74), MTHFR T677T (1.61/1.21-2.14), antiphospholipid antibodies (5.84/3.06-11.18), elevated lipoprotein (a) (6.24/4.51-8.64), and combined ITs (8.85/3.32-23.57). Carrier rates reported for antithrombin- or protein S deficiency among patients were 1.5% and 1.6% as compared with 0.06% (p<0.001) and 0.4% (p=0.003) in healthy controls. Conclusions The present meta-analysis indicates that IT serve as risk factors for incident stroke. However, the impact of IT upon outcome and recurrence risk needs to be further investigated. Disclosures: Manco-Johnson: Baxter BioScience: Honoraria; Bayer HealthCare: Honoraria; CSL Behring: Honoraria; NovoNordisk: Honoraria; Octapharma: Honoraria. Off Label Use: Enoxaparin (LMWH) is used off-label in children to prevent symptomatic thromboembolism.

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