scholarly journals Rivaroxaban versus warfarin for the management of left ventricle thrombus

2021 ◽  
Vol 73 (1) ◽  
Author(s):  
Monirah A. Albabtain ◽  
Yahya Alhebaishi ◽  
Ola Al-Yafi ◽  
Hatim Kheirallah ◽  
Adel Othman ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Retrospective Study ◽  
Left Ventricle ◽  
Surface Area ◽  
Median Time ◽  
Randomized Clinical Trials ◽  
Off Label Use ◽  
Duration Of Treatment ◽  
Off Label ◽  
Warfarin Group

Abstract Background Rivaroxaban has been recently introduced for the management of non-valvular intra-cardiac thrombosis with variable results. We aimed to compare the results of the off-label use of rivaroxaban versus warfarin in the management of patients with left ventricle (LV) thrombus. This research is a retrospective study conducted on 63 patients who had LV thrombus from January to December 2016. We compared patients treated with warfarin (n=35) to patients who had rivaroxaban (n=28), and study outcomes were time to thrombus resolution, bleeding, stroke, and mortality. Results The median duration of treatment was 9.5 (25th-75th percentiles: 6-32.5) months for rivaroxaban and 14 (3-41) months for warfarin. Thrombus resolution occurred in 24 patients in the warfarin group (68.6%) and 20 patients in the rivaroxaban group (71.4%). The median time to resolution in the warfarin group was 9 (4-20) months and 3 (2-11.5) months in the rivaroxaban group. Thrombus resolution was significantly faster in patients on rivaroxaban (p= 0.019). Predictors of thrombus resolution were thrombus surface area (HR: 1.21; CI 95% (1.0-1.46); p= .048) and the use of rivaroxaban (HR: 1.92; CI 95% (1.01-3.65); p= 0.048). There was no difference in stroke, bleeding, and mortality between both groups. Conclusion Rivaroxaban was as effective and safe as warfarin in managing patients with left ventricle thrombus. Larger randomized clinical trials are recommended to confirm our findings.

Safety and Evidence of Off-Label Use of Approved Drugs at the National Cancer Center Hospital in Japan

2020 ◽  
pp. OP.20.00131
Author(s):  
Seiko Bun ◽  
Kan Yonemori ◽  
Hiroko Sunadoi ◽  
Rena Nishigaki ◽  
Emi Noguchi ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Pharmaceutical Products ◽  
National Cancer Center Hospital ◽  
Phase Iii ◽  
Cancer Center ◽  
Off Label Use ◽  
Pharmaceutical Development ◽  
Off Label ◽  
Center Hospital ◽  
Label Use

PURPOSE: In Japan, for pharmaceutical products to be covered by public medical insurance, their efficacy and safety must first be confirmed in clinical trials. To our knowledge, this study is the first investigation into the off-label use of pharmaceutical products at a high-volume cancer treatment center in Japan. The objective of this study is to explore the framework necessary for future pharmaceutical development and regulatory approval in the field of oncology by surveying the frequency of and indications for off-label use of pharmaceutical products at the National Cancer Center Hospital in Tokyo, Japan. MATERIALS AND METHODS: The pharmaceutical products used off-label in daily practice from 2003 to 2015 at the National Cancer Center Hospital were retrospectively examined based on applications that had been submitted to an internal review committee requesting off-label use. RESULTS: A total of 1,390 applications were submitted during the study period. The most frequently used supporting documents were the results of phase II trials, followed by case series and phase III trials. The cancer most frequently treated with off-label drugs was sarcoma (15.1%), followed by urologic cancer (9.2%) and GI cancer (7.6%). CONCLUSION: As reported in previous studies, pharmaceutical products were generally used off-label for the treatment of rare cancers, for which large-scale clinical trials are difficult to conduct. Continued discussion of the types of frameworks that are needed to guide pharmaceutical development is necessary.

scholarly journals Interleukin-6 Inhibition in Inflammatory Diseases: Results Achieved and Tasks to Accomplish

2017 ◽  
Vol 2 (2_suppl) ◽  
pp. S20-S28 ◽  
Author(s):  
Toshio Tanaka ◽  
Masashi Narazaki
Keyword(s):  
Clinical Trials ◽  
Host Defense ◽  
Interleukin 6 ◽  
Therapeutic Strategy ◽  
Tissue Injury ◽  
Inflammatory Diseases ◽  
Off Label Use ◽  
Polyarticular Juvenile Idiopathic Arthritis ◽  
Pathological Effect ◽  
Off Label

Interleukin-6 (IL-6) is a prototypical cytokine featuring functional pleiotropy and redundancy. Under situations of stress, such as infection or tissue injury, IL-6 is rapidly synthesized and plays a major role in host defense. However, uncontrolled excessive or persistent production of IL-6 has a pathological effect in various diseases. Thus, IL-6 blockade was expected to become a novel therapeutic strategy for IL-6–mediated inflammatory diseases, and the first-in-class IL-6 inhibitor tocilizumab, which blocks IL-6 activity by inhibiting IL-6 binding to its receptor, was developed. Clinical trials of tocilizumab have verified its efficacy and tolerable safety profile in several diseases, and it has been approved for the treatment of patients with rheumatoid arthritis, Castleman's disease, systemic and polyarticular juvenile idiopathic arthritis, and giant cell arteritis. Off-label use and clinical trials strongly indicate that tocilizumab will be applicable for a wide variety of acute and chronic inflammatory diseases.

scholarly journals Trastuzumab and Metastatic Breast Cancer: Trastuzumab Use in Australia—Monitoring the Effect of an Expensive Medicine Access Program

2007 ◽  
Vol 25 (24) ◽  
pp. 3688-3693 ◽  
Author(s):  
Sallie-Anne Pearson ◽  
Clare L. Ringland ◽  
Robyn L. Ward
Keyword(s):  
Breast Cancer ◽  
Clinical Trials ◽  
Metastatic Breast Cancer ◽  
Clinical Practice ◽  
Metastatic Breast ◽  
Cardiac Monitoring ◽  
Off Label Use ◽  
Off Label ◽  
Trastuzumab Therapy ◽  
Drug Wastage

Purpose Data from clinical trials are used for drug registration; however, many cancer medicines are ultimately used off-label. This study examines the extent to which the clinical practice use of trastuzumab for the treatment of metastatic breast cancer differs from its use under trial conditions. Methods This study involved all women (N = 1,469) with metastatic breast cancer who received trastuzumab in Australia between December 2001 and March 2005. Given that Australia operates a universal health care system, administrative databases could be examined to determine the duration of therapy, rate of off-label use, compliance with cardiac monitoring, and the extent of drug wastage (volume and cost). Results A total of 433 enrollees (29.5%) received trastuzumab as monotherapy and 1,036 enrollees (70.5%) received the drug in combination with chemotherapy. A total of 321 women (22%) received off-label trastuzumab. The median duration of trastuzumab therapy was longer than that on trial: 5.6 v 3.1 months for enrollees receiving monotherapy and 12.5 v 6.9 months for concomitant chemotherapy. Only 47 (3%) of enrollees received cardiac monitoring before and during trastuzumab therapy. We estimated 24% of trastuzumab dispensed was discarded, at a cost of $21.1 million Australian. Alternative administration schedules and the addition of another vial size potentially reduce wastage to 6% of volume dispensed. Conclusion Debates about the use of expensive cancer medicines should consider postmarketing assessments as well as trial experience. The longer duration of trastuzumab use in clinical practice and the high rates of off-label use provide incentive for new clinical trials. Strategies to improve cardiac monitoring and to minimize drug wastage are issues that require immediate attention.

scholarly journals Medicines for the Treatment Of COVID-19: Awaiting the Evidence

2020 ◽  
Vol 33 (7-8) ◽  
pp. 500
Author(s):  
Natalia Marto ◽  
Emília C. Monteiro
Keyword(s):  
Clinical Trials ◽  
Severe Acute Respiratory Syndrome ◽  
The Novel ◽  
Off Label Use ◽  
Promising Candidate ◽  
Off Label ◽  
Health Authorities ◽  
Investigational Drugs ◽  
The World ◽  
Repurposed Drugs

The novel severe acute respiratory syndrome coronavirus 2 is the cause of Coronavirus Disease 2019, a new illness with no effective treatment or vaccine that has reached pandemic proportions. In this document, we analyze how health authorities and agencies around the world position themselves regarding the off-label use of repurposed drugs or new investigational drugs to treat Coronavirus Disease 2019. We review the most promising candidate medicines, including available evidence, clinical recommendations and current options for access. Our concluding remarks stress the importance of administering off-label and investigational drugs in the setting of clinical trials, or at least in standardized scenarios, to generate as much scientific knowledge as achievable while engaging in the best efforts to treat patients and save lives.

Recommendations for Clinical Trials of Off-Label Drugs Used to Treat Advanced-Stage Cancer

2012 ◽  
Vol 30 (6) ◽  
pp. 661-666 ◽  
Author(s):  
C. Daniel Mullins ◽  
Russ Montgomery ◽  
Amy P. Abernethy ◽  
Arif Hussain ◽  
Steven D. Pearson ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Drug Administration ◽  
Randomized Clinical Trials ◽  
Technology Policy ◽  
Food And Drug Administration ◽  
Decision Makers ◽  
Public And Private ◽  
Off Label ◽  
The Us ◽  
Before And After

Purpose To provide recommendations to trialists and sponsors that guide the design and implementation of prospective postapproval clinical trials for oncology drugs used outside US Food and Drug Administration–labeled indications for treatment of late-stage cancers. Methods A meeting was hosted by the Center for Medical Technology Policy in Baltimore, MD, on November 12, 2009. Discussions during the meeting and key informant interviews were conducted before and after this stakeholder meeting. Peer review by multidisciplinary stakeholders was followed by a public comment period. Input was received from patient advocacy groups, medical oncologists, pharmaceutical companies, the US Food and Drug Administration, Centers for Medicare and Medicaid Services, the National Cancer Institute, foreign government agencies involved in health technology assessment, public and private payers, drug compendia, clinical research entities, statisticians, academics, and the American Society of Clinical Oncology. Results To address the needs of patients and their clinical providers, compendia, payers, and policy makers, recommendations are proposed to guide the design of future prospective trials for off-label use of oncology drugs across four areas: trial design and data analysis, patient and site recruitment, comparators, and outcomes. Conclusion The US Food and Drug Administration provides guidance to the pharmaceutical industry and others designing randomized clinical trials for regulatory approval. However, a gap exists for postregulatory decision makers, including patients, prescribers, and payers, because regulatory trials do not answer the questions most relevant to them. Therefore, guidance is needed for trials performed in the postapproval environment for these postapproval decision makers.

Chapter 6 - Clinical trials and off-label use of drugs

2016 ◽  
Vol 17 (3-4) ◽  
pp. 409-430
Author(s):  
Giovanni Maria Cavo
Keyword(s):  
Clinical Trials ◽  
Off Label Use ◽  
Off Label ◽  
Label Use

scholarly journals Retrospective study of 151 patients with cutaneous leishmaniasis treated with meglumine antimoniate

2005 ◽  
Vol 38 (3) ◽  
pp. 213-217 ◽  
Author(s):  
Armando de Oliveira Schubach ◽  
Keyla B. Feldman Marzochi ◽  
João Soares Moreira ◽  
Tânia Maria Pacheco Schubach ◽  
Marcelo Lodi Araújo ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Retrospective Study ◽  
Cutaneous Leishmaniasis ◽  
Median Time ◽  
Continuous Treatment ◽  
Low Doses ◽  
Intermittent Treatment ◽  
Meglumine Antimoniate ◽  
Adults And Children ◽  
Rest Intervals

We retrospectively analyzed a series of 151 cases of cutaneous leishmaniasis treated between 1967 and 1982. One-hundred-and-thirty-nine (92%) patients presented with active lesions and were treated with daily doses of meglumine antimoniate: 81 adults received a 5-ml vial IM and 58 children received 1 to 5ml. Forty-five (32.4%) patients underwent continuous treatment with meglumine antimoniate for 25 to 116 days without rest intervals, and 94 (67.6%) intermittent treatment with 2 to 5 series of meglumine antimoniate. Intermittent series could include schedules of daily IM applications for 10 to 25 days each and intervals varying from 10 to 60 days. Antimony dose was calculated for 66 (47.5%) patients and ranged from 3.9 to 28.7 Sb5+/kg/day. Of these, 35 patients received >10mg and 31 patients <10mg Sb5+/kg/day. Median time of healing was longer for lesions on the legs and feet - 67.5 days versus 48.7 days (p < 0.001) for other sites. However, there were no significant differences in the median time of healing between adults and children, intermittent and continuous regimens or high and low antimony doses. Fifty-one patients were reassessed 5 to 14 years after treatment and showed no evidence of disease. These results support further investigation (clinical trials) on treatment using low doses of antimony.

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