Investigational Treatment for Epilepsy

2013 ◽  
pp. 179-182
Author(s):  
William O. Tatum
Keyword(s):  
Investigational Treatment

Efficacy and safety of prophylaxis with once-weekly BAY 79–4980 compared with thrice-weekly rFVIII-FS in haemophilia A patients

2012 ◽  
Vol 108 (11) ◽  
pp. 913-922 ◽  
Author(s):  
Uri Martinowitz ◽  
Jerzy Windyga ◽  
Giovanni Di Minno ◽  
Andrzej Hellmann ◽  
Ingrid Pabinger ◽  
...  
Keyword(s):  
Controlled Study ◽  
Control Group ◽  
Investigational Drug ◽  
Haemophilia A ◽  
Double Blind ◽  
Investigational Treatment ◽  
Joint Health ◽  
Secondary Endpoints ◽  
Recombinant Fviii ◽  
Randomised Controlled

SummaryThe benefits of prophylaxis of haemophilia A patients regarding joint health and quality-of-life are well established. However, adherence to an up to every-other-day infusion regimen is a barrier to widespread adoption of prophylaxis. BAY 79–4980 is an investigational drug consisting of rFVIII-FS (sucrose-formulated recombinant FVIII) reconstituted with liposome solvent. Previous clinical studies showed extended protection from bleeding after a single injection of BAY 79–4980 (13.3 ± 6.2 days) compared with rFVIII-FS (7.2 ± 1.7 days). The effect of once-a-week prophylaxis with BAY 79–4980 (35 IU/kg) compared with three times-per-week rFVIII-FS (25 IU/kg) in previously treated, severe haemophilia A patients was evaluated in a 52-week, double-blind, two-arm, randomised, controlled study. The primary and secondary endpoints were protection from total bleeds and joint bleeds, respectively. Short- and long-term safety and tolerability of BAY 79–4980 including effects on lipid levels were assessed. A total of 139 and 131 subjects were evaluable for safety and efficacy analyses, respectively. A large difference in efficacy between treatment groups was observed with 72.1% (49/68) in the rFVIII-FS control group demonstrating <9 bleeds/year compared with 38.1% (24/63) of BAY 79–4980-treated subjects. A similar difference was seen in annualised joint bleeds, with 43 subjects (63.2%) in the control group demonstrating <5 joint bleeds/year compared with 24 subjects (38.1%) treated with BAY 79–4980. The distribution of bleeds seven days post-prophylactic treatment with BAY 79–4980 showed that 61% of bleeds occurred after day 4 post dosing. There were no safety concerns identified. The investigational treatment arm was prematurely discontinued due to failure to achieve the primary endpoint.

Corticosteroid-induced magnetic resonance imaging changes in patients with recurrent malignant glioma.

1994 ◽  
Vol 12 (9) ◽  
pp. 1886-1889 ◽  
Author(s):  
C J Watling ◽  
D H Lee ◽  
D R Macdonald ◽  
J G Cairncross
Keyword(s):  
Magnetic Resonance ◽  
Malignant Glioma ◽  
Symptom Control ◽  
Corticosteroid Treatment ◽  
Cross Sectional Area ◽  
Sectional Area ◽  
Recurrent Malignant Glioma ◽  
Cross Sectional ◽  
Investigational Treatment ◽  
Area And Volume

PURPOSE We studied corticosteroid-induced magnetic resonance (MR) scan changes in patients with recurrent malignant glioma to determine if corticosteroid therapy started concurrently with investigational treatment might yield false-positive responses. PATIENTS AND METHODS Ten symptomatic patients not on corticosteroids when malignant glioma recurred had a baseline MR scan performed before corticosteroid treatment, followed by serial scans at weekly intervals for 1 month while on dexamethasone (16 mg/d). The maximum cross-sectional areas and volumes of the gadolinium-enhancing regions (tumor) and T2-weighted abnormalities (tumor plus edema) were compared quantitatively and qualitatively for each series of scans. RESULTS Nine of 10 patients (90%) had a measurable reduction in the size of the gadolinium-enhancing region or T2-weighted abnormality with corticosteroid treatment. The maximum cross-sectional area and volume of the gadolinium-enhancing region decreased by at least 25% in three of 10 patients (30%). The maximum cross-sectional area and volume of the T2-weighted abnormality decreased by at least 25% in five of 10 patients (50%). Maximum measurable radiologic improvement was evident within 2 weeks in most patients. MR scans were judged improved by the reporting neuroradiologist in seven of 10 (70%). These subjective visual improvements were also evident within 2 weeks, but generally described as slight or modest. CONCLUSION Corticosteroid-induced MR scan reductions in tumor size may confound the assessment of response of recurrent malignant gliomas to investigational agents. For patients who start corticosteroids for symptom control, investigational treatment should be delayed until a new baseline MR image is established 2 weeks later. Response is then judged by comparing subsequent MR scans with the new corticosteroid-influenced baseline image.

scholarly journals Turning a stroke into a TIA: curative thrombolysis with combined intravenous and intra-arterial tPA

CJEM ◽  
2006 ◽  
Vol 8 (01) ◽  
pp. 54-57 ◽  
Author(s):  
David J. Gladstone ◽  
Richard I. Aviv ◽  
Babak Jahromi ◽  
Sandra E. Black ◽  
Devra Baryshnik ◽  
...  
Keyword(s):  
Ischemic Stroke ◽  
Acute Ischemic Stroke ◽  
Standard Treatment ◽  
Treatment Approach ◽  
Novel Treatment ◽  
Intravenous Tissue Plasminogen Activator ◽  
Investigational Treatment ◽  
Tissue Plasminogen ◽  
Proximal Middle Cerebral Artery ◽  
Emergency Revascularization

ABSTRACT Intravenous tissue plasminogen activator (tPA) is standard treatment for eligible patients with acute ischemic stroke, but may be less effective for very severe strokes caused by proximal intracranial artery occlusions. We report the case of a woman with a devastating stroke who recovered completely following emergency revascularization of an occluded proximal middle cerebral artery using a novel treatment approach that combines both intravenous (IV) and intra-arterial (IA) tPA. This case illustrates the potential value of the combined IV–IA thrombolytic approach, which is an emerging investigational treatment strategy for selected patients with severe acute ischemic stroke.

Mechanisms and the Current State of Deep Brain Stimulation in Neuropsychiatry

CNS Spectrums ◽  
2003 ◽  
Vol 8 (7) ◽  
pp. 522-526 ◽  
Author(s):  
Benjamin D. Greenberg ◽  
Ali R. Rezai
Keyword(s):  
Deep Brain Stimulation ◽  
Brain Stimulation ◽  
Movement Disorders ◽  
Mechanisms Of Action ◽  
Therapeutic Effects ◽  
Current State ◽  
Investigational Treatment ◽  
Preclinical And Clinical Studies ◽  
The Brain ◽  
Deep Brain

ABSTRACTDeep brain stimulation (DBS) is established as a therapy for movement disorders, and it is an investigational treatment in other neurologic conditions. DBS precisely targets neuroanatomical targets deep within the brain that are proposed to be centrally involved in the pathophysiology of some neuropsychiatric illnesses. DBS is nonablative, offering the advantages of reversibility and adjustability. This might permit therapeutic effectiveness to be enhanced or side effects to be minimized. Preclinical and clinical studies have shown effects of DBS locally, at the stimulation target, and at a distance, via actions on fibers of passage or across synapses. Although its mechanisms of action are not fully elucidated, several effects have been proposed to underlie the therapeutic effects of DBS in movement disorders, and potentially in other conditions as well. The mechanisms of action of DBS are the focus of active investigation in a number of clinical and preclinical laboratories. As in severe movement disorders, DBS may offer a degree of hope for patients with intractable neuropsychiatric illness. It is already clear that research intended to realize this potential will require a very considerable commitment of resources, energy, and time across disciplines including psychiatry, neurosurgery, neurology, neuropsychology, bioengineering, and bioethics. These investigations should proceed cautiously.

scholarly journals Control of malignant ascites using HIPEC in 2 patients with ovarian cancer: A case-report

2020 ◽  
Author(s):  
Vladislav Nikulin ◽  
Amir Abdullaev ◽  
Mikhail Davydov ◽  
Alexey Rumyantsev ◽  
Vladislav Kirsanov ◽  
...  
Keyword(s):  
Ovarian Cancer ◽  
Randomized Clinical Trials ◽  
Stage Iv ◽  
Figo Stage ◽  
Malignant Ascites ◽  
Investigational Treatment ◽  
Long Time ◽  
Platinum Refractory

Abstract Objective: to demonstrate an efficacy of Hyperthermic Intraperitoneal Chemotherapy (HIPEC) in management of malignant ascites (MA) in patients with platinum-refractory ovarian cancer (OC).Background: MA in OC patients can dramatically affect quality of life. HIPEC is an investigational treatment modality that can be effective in MA setting but evidence-based data supporting this method are lacking. Cases presentation: 2 women 50-year-old, FIGO stage IV and 60-year-old, FIGO stage IIIC presented at our center, both had recurrent MA. Patients were treated with HIPEC after platinum-refractory recurrence. The first one had total control of MA with no evidence of disease at the time of last follow-up examination. The 2nd had 9 months of disease control – a relatively long time considering her MA recurrence rate.Conclusions: HIPEC can be successfully used for MA management in selected patients with epithelial OC refractory to standard chemotherapy, however more data are needed from randomized clinical trials.

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