PET imaging for guiding definition of therapeutic reference ranges – new drugs, new guidelines

2016 ◽  
Vol 49 (03) ◽  
Author(s):  
G Gründer
Keyword(s):  
Pet Imaging ◽  
New Drugs ◽  
Reference Ranges ◽  
New Guidelines ◽  
Definition Of

scholarly journals Practical and conceptual issues of clinical trial registration for Brazilian researchers

2015 ◽  
Vol 134 (1) ◽  
pp. 28-33 ◽  
Author(s):  
Carolina Gomes Freitas ◽  
Thomas Fernando Coelho Pesavento ◽  
Maurício Reis Pedrosa ◽  
Rachel Riera ◽  
Maria Regina Torloni
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
International Committee ◽  
New Drugs ◽  
Trial Registration ◽  
Clinical Trial Registry ◽  
Clinical Trial Registration ◽  
Registration Process ◽  
Trial History ◽  
Definition Of

CONTEXT AND OBJECTIVE: Clinical trial registration is a prerequisite for publication in respected scientific journals. Recent Brazilian regulations also require registration of some clinical trials in the Brazilian Clinical Trials Registry (ReBEC) but there is little information available about practical issues involved in the registration process. This article discusses the importance of clinical trial registration and the practical issues involved in this process. DESIGN AND SETTING: Descriptive study conducted by researchers within a postgraduate program at a public university in São Paulo, Brazil. METHODS: Information was obtained from clinical trial registry platforms, article reference lists and websites (last search: September 2014) on the following topics: definition of a clinical trial, history, purpose and importance of registry platforms, the information that should be registered and the registration process. RESULTS: Clinical trial registration aims to avoid publication bias and is required by Brazilian journals indexed in LILACS and SciELO and by journals affiliated to the International Committee of Medical Journal Editors (ICMJE). Recent Brazilian regulations require that all clinical trials (phases I to IV) involving new drugs to be marketed in this country must be registered in ReBEC. The pros and cons of using different clinical trial registration platforms are discussed. CONCLUSIONS: Clinical trial registration is important and various mechanisms to enforce its implementation now exist. Researchers should take into account national regulations and publication requirements when choosing the platform on which they will register their trial.

scholarly journals Rare Treatments for Rare Dyslipidemias: New Perspectives in the Treatment of Homozygous Familial Hypercholesterolemia (HoFH) and Familial Chylomicronemia Syndrome (FCS)

2021 ◽  
Vol 23 (11) ◽  
Author(s):  
Laura D’Erasmo ◽  
Simone Bini ◽  
Marcello Arca
Keyword(s):  
Familial Hypercholesterolemia ◽  
Treatment Options ◽  
New Drugs ◽  
Lipid Lowering ◽  
Homozygous Familial Hypercholesterolemia ◽  
Published Evidence ◽  
Novel Drugs ◽  
Life Threatening ◽  
History Of ◽  
Definition Of

Abstract Purpose of Review This review aims to summarize the most recent published literature concerning lomitapide and volanesorsen that are approved for the use in HoFH and FCS patients, respectively. Moreover, it will briefly revise the published evidence on novel, non-approved treatments that are under evaluation for the management of these rare forms of dyslipidemias Recent Findings The definition of rare dyslipidemias identifies a large number of severe disorders of lipid metabolism of genetic origin. Among them were homozygous familial hypercholesterolemia (HoFH) (OMIM #143890) and familial chylomicronemia syndrome (FCS) (OMIM #238600), which are characterized by a markedly impaired cholesterol- and triglyceride-containing lipoproteins metabolism. They are being particularly associated with poor health outcomes and quality of life. Considering the severity of these diseases, common lipid-lowering drugs are often ineffective or do not allow to achieve the recommended lipid targets to prevent the development of complications. Nowadays, several new drugs have been found to effectively treat HoFH and FCS with an acceptable safety profile. Summary Treating patients with HoFH and FCS remains very challenging. However, novel treatment options are emerging and might be considered in addition to conventional therapy for managing these diseases. These novel drugs will possibly change the natural history of these two rare and life-threatening diseases.

Treatment of Advanced Colorectal Cancer (CRC) in Daily Practice: Results of a Survey in two Italian Regions, Piemonte and Valle D'aosta

1998 ◽  
Vol 84 (5) ◽  
pp. 562-566 ◽  
Author(s):  
Alessandro Comandone ◽  
Roberto Berardo ◽  
Roberto Faggiuolo ◽  
Antonella Boglione ◽  
Paola Bergnolo ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
Performance Status ◽  
Daily Practice ◽  
New Drugs ◽  
Therapeutic Choice ◽  
Italian Regions ◽  
Important Health ◽  
Definition Of ◽  
No Gold Standard

Aims and background Colorectal cancer (CRC) is one of the most important health problems in Western countries: it is the fourth cancer in terms of incidence and the second cause of cancer death. Surgery is the main therapeutic choice and there is broad consensus on the role of adjuvant chemotherapy (CT) after resection. Unfortunately, 50% of the patients will relapse and die of the disease. Palliative CT based on 5-fluorouracil (5FU) may induce a 9-48% response rate with a median survival of 11.5 months. At present there is no gold standard for CT in advanced CRC and the situation has become more complicated since the advent of new drugs (Raltitrexed, Irinotecan, Oxaliplatin). The aim of this study was the identification of the different approaches to treatment of advanced CRC among the clinicians (oncologists, radiologists, internal medicine specialists, surgeons) who practice CT. Methods and study design Forty-six clinicians from two Italian Regions (Piemonte and Valle d'Aosta) were interviewed by telephone. Results 5FU modulated with Lederfolin according to the classic Machover scheme is the main option in daily practice. More sophisticated therapies are reserved to patients with a good performance status (PS) and are prescribed only in the larger centers. The planned therapies usually consist of six courses. Restaging may be performed after three or six courses. A marked difference has been recorded in the evaluation of a situation of no change (NC): 25.5% of the clinicians evaluate stable disease as a positive result. In the event of disease progression or relapse, 35% of the clinicians do not prescribe second-line CT. In case of further treatment, the options are totally subjective. Conclusions A national survey on this issue is necessary under the auspices of AIOM (Associazione Italiana Oncologia Medica) and involving oncologists, epidemiologists and statisticians, in order to define the reasons for variations in therapy in advanced CRC and determine the differences between clinicians of different age, specialization and location. This survey could lead to a definition of guidelines for the treatment of advanced CRC.

Standardization of Light Transmission Aggregometry for Diagnosis of Platelet Disorders: An Inter-Laboratory External Quality Assessment

2019 ◽  
Vol 119 (07) ◽  
pp. 1154-1161 ◽  
Author(s):  
Karina Althaus ◽  
Barbara Zieger ◽  
Tamam Bakchoul ◽  
Kerstin Jurk ◽  
Keyword(s):  
Platelet Function ◽  
Light Transmission ◽  
Platelet Rich Plasma ◽  
Reference Ranges ◽  
Technical Problems ◽  
Platelet Function Disorders ◽  
Light Transmission Aggregometry ◽  
Laboratory Survey ◽  
Definition Of

AbstractSeveral in vitro platelet function tests are available for the diagnosis of inherited platelet function disorders. Currently, the light transmission aggregometry (LTA) is recommended as one of the first-step tests. LTA is available in most specialized hemostasis laboratories. Although the LTA is accepted as a ‘gold standard’ assay for the evaluation of platelet function, its standardization in the clinical practice is still challenging. The GTH-based THROMKID-Plus Study Group has performed an inter-laboratory trial in Germany and Austria. Five different agonists were selected according to the Scientific and Standardization Committee/International Society on Thrombosis and Haemostasis recommendations and shipped in 3 different sets (one should represent a healthy control and two should simulate platelet function disorders) to 15 specialized laboratories in Germany and Austria. Agonists were analyzed by APACT or PAP4/8 aggregometer using platelet-rich plasma from healthy donors. In addition, laboratory-internal platelet agonists were tested in platelet-rich plasma from a healthy donor. All laboratories (9 used APACT, 6 used PAP4/PAP8) showed very consistent data regarding the maximum percentage of aggregation induced by the tested agonists and identified the differential diagnosis of the simulated platelet function disorders with one exception, which was due to technical problems. In contrast, there was a high variability of the laboratory-internal inductors regarding reagent type, concentrations and pathological cut-off values. Our study showed that the shipment of agonists is suitable for an inter-laboratory survey of LTA. However, there is still a remarkable need for standardization of agonist reagents and their concentration as well as for definition of reference ranges.

scholarly journals Pharmacotherapy and child psychiatry: is there a way forward?

2008 ◽  
Vol 14 (1) ◽  
pp. 10-16 ◽  
Author(s):  
A. James
Keyword(s):  
Pharmaceutical Industry ◽  
Child Psychiatry ◽  
New Drugs ◽  
Pharmaceutical Companies ◽  
Drug Treatments ◽  
New Guidelines ◽  
Post Trial ◽  
Relationship Of ◽  
The Relationship

Recent controversy over the use of serotonin reuptake inhibitors in children and adolescents has focused attention on the role of the pharmaceutical industry in the treatment of young people. Failure of pharmaceutical companies to fully disclose negative outcome trials has led to new guidelines for publication of all trial results. Scrutiny is on the conduct of trials and the relationship of the pharmaceutical industry with prescribing doctors and post-trial surveillance of new drugs. It is argued that drug treatments in child psychiatry are a powerful therapeutic tool but vigilance is needed to ensure that data on the efficacy and safety of drugs are freely available.

Definition of reference ranges for the platelet distribution width (PDW): a local need

2010 ◽  
Vol 48 (2) ◽  
Author(s):  
Mariela Granero Farias ◽  
Elias Guilherme Schunck ◽  
Suzane Dal Bó ◽  
Simone Martins de Castro
Keyword(s):  
Reference Ranges ◽  
Platelet Distribution Width ◽  
Distribution Width ◽  
Definition Of

Evaluation of Clinical Efficacy of New Medical Treatments in Advanced Colorectal Cancer. Results of a Workshop Organized by the Eortc Gitccg

1998 ◽  
Vol 84 (3) ◽  
pp. 335-347 ◽  
Author(s):  
Jacques Wils ◽  
Tarek Sahmoud ◽  
Alberto Sobrero ◽  
Harry Bleiberg ◽  
Sam Ahmedzai ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
Advanced Colorectal Cancer ◽  
New Drugs ◽  
European Organization ◽  
Medical Treatments ◽  
General Acceptance ◽  
Tract Cancer ◽  
Definition Of ◽  
The Impact

During the last few years several factors have contributed to an increasing change in the medical treatment of advanced colorectal cancer. Among them are the more general acceptance of the impact of chemotherapy on quality of life and survival in first as well as in second-line treatment, the introduction of new drugs and the definition of novel endpoints which can roughly be defined as “patient benefit”. For this reason the European Organization for Research and Treatment of Cancer (EORTC) Gastrointestinal Tract Cancer Cooperative Group (GITCCG) felt it was appropriate to organize a workshop with experts from different countries and national groups to discuss in depth several aspects concerning the treatment of patients with advanced colorectal cancer.

scholarly journals Diagnosis and Treatment of Acute Pancreatitis Due to Hypertriglyceridemia in Italy: A Survey among Physicians of the Italian Association for the Study of the Pancreas: A Brief Report

2020 ◽  
Vol 11 (2) ◽  
pp. 20-26
Author(s):  
Raffaele Pezzilli
Keyword(s):  
Acute Pancreatitis ◽  
Epigastric Pain ◽  
Imaging Techniques ◽  
Elevated Serum ◽  
Serum Triglyceride ◽  
New Drugs ◽  
Italian Association ◽  
Disease Definition ◽  
Definition Of ◽  
Epidemiological Characteristics

Introduction: The Italian Association for the Study of the Pancreas promoted a survey on exploring the point of view of Italian pancreatologists regarding the diagnosis and the treatment of acute pancreatitis (AP) due to hypertriglyceridemia (HAP). Method: A questionnaire was administered, and it contained four sections regarding epidemiological characteristics of the participants, how the participants arrived at a diagnosis of the disease, how they treated familial hypertriglyceridemia, and whether they knew of the new drugs developed for the treatment of this disease. Definition of AP and HAP: In this survey, all participants followed this definition of AP: The diagnosis of AP requires two of the following three features: abdominal pain consistent with acute pancreatitis (acute onset of a persistent and severe epigastric pain, often radiating to the back); serum pancreatic enzymes at least three times greater than the upper limit of normal; and characteristic findings of acute pancreatitis using imaging techniques. On the other hand, HAP is characterized by serum triglyceride concentration of >1000 mg/dL as the diagnostic cut-off, even though a value of >500 mg/dL has been used for a more inclusive definition, since moderately elevated triglyceride levels have also been suggested as a risk factor for AP. Results. Nine percent of all managed patients with AP had HAP; 5.0 ± 7.7 patients per year had a recurrence of HAP, and the number of recurrences was about one. A diagnosis of hypertriglyceridemia was made by the majority of Italian physicians due to the presence of elevated serum triglycerides at a level of ≥880 mg/dL. Twenty-five physicians treated their patients with fibrates, 23 with statins, 11 with omega-3, one with medium-chain triglycerides, and six with plasmapheresis. Finally, fewer than 50% of the physicians knew of the new drugs to treat dyslipidemia. Conclusions: The results of this survey show that an educational program is important, and we also need an Italian National Registry both for improving knowledge regarding this disease and for identifying the causal factors in our country.

scholarly journals Elevated blood pressure level based on 2017 ACC/AHA guideline in relation to stroke risk in rural areas of Liaoning province

2019 ◽  
Vol 19 (1) ◽  
Author(s):  
Yanxia Xie ◽  
Mingfeng Ma ◽  
Zhao Li ◽  
Xiaofan Guo ◽  
Guozhe Sun ◽  
...  
Keyword(s):  
Blood Pressure ◽  
Rural Areas ◽  
Rural China ◽  
Blood Pressure Level ◽  
Liaoning Province ◽  
Stroke Incidence ◽  
Cox Proportional Hazard ◽  
Cox Proportional Hazard Models ◽  
New Guidelines ◽  
Definition Of

Abstract Background The new ACC/AHA hypertension guideline lower the definition of hypertension from 140/90 mmHg to 130/80 mmHg and eliminate the category of prehypertension thus increasing the prevalence of hypertension. A purpose of this study is to explore the applicability of the new guidelines in rural China. Methods In total, 3229 participants aged ≥35 years and free of stroke at baseline were followed for up to 4.8 years during 2012 to 2017 in a rural community-based prospective cohort study of Xifeng County. The hazard ratio (HR) and 95% Confidence interval (CI) of different blood pressure (BP) levels for risk of incident stroke were analyzed by multivariable Cox proportional hazard models. Results During the follow-up, 81 new strokes occurred among the 3229 participants. Compared with normal BP (Systolic BP (SBP)<120 mmHg and Diastolic BP (DBP)<80 mmHg), stage 2 hypertension (SBP ≥ 140 mmHg or DBP ≥ 90 mmHg) had approximately 2.1 greater risks for stroke (HR: 2.10, 95% CI: 1.13 to 3.91, P = 0.020). However, there was no significant association between elevated (SBP:120-129 mmHg and DBP<80 mmHg), stage1 hypertension (SBP:130-139 mmHg or DBP:80-89 mmHg) and stroke incidence (HR: 0.93, 95% CI: 0.33 to 2.61, P = 0.888; HR: 0.96, 95% CI: 0.46 to 2.02, P = 0.920, respectively). An increase of the SBP by 1-SD increases the risk for stroke by 56% (HR: 1.56, 95%CI: 1.29 to 1.88, P < 0.001). An increase of the SBP by 20 mmHg increases the risk for stroke by 51% (HR: 1.51, 95%CI: 1.27 to 1.80, P < 0.001). Conclusions Compared with normal BP, the stage 2 hypertension based on 2017 ACC/AHA guideline significantly increases the risk of stroke incidence, but this association was not observed between elevated, stage1 hypertension and stroke incidence in Chinese rural adults.

Acute Cholestatic Hepatitis Associated with Celecoxib

2002 ◽  
Vol 36 (12) ◽  
pp. 1887-1889 ◽  
Author(s):  
Antonio Grieco ◽  
Luca Miele ◽  
Andrea Giorgi ◽  
Ignazio M Civello ◽  
Giovanni Gasbarrini
Keyword(s):  
English Language ◽  
Elevated Serum ◽  
Biliary Tree ◽  
Cholestatic Hepatitis ◽  
New Drugs ◽  
Alcoholic Beverages ◽  
Drug Induced ◽  
Herbal Products ◽  
Fourth Report ◽  
Definition Of

OBJECTIVE: To report a case of acute cholestatic hepatitis associated with the selective cyclooxygenase-2 inhibitor celecoxib. CASE SUMMARY: A 41-year-old white man was hospitalized for jaundice after 2 doses of celecoxib 200 mg for pain associated with right-knee trauma. Laboratory workup showed hyperbilirubinemia, mildly elevated serum transaminase concentrations, and cholestasis. Abdominal imaging showed no dilation of the biliary tree. Histology showed cholestasis, with bile plugs in dilated bile canaliculi and a mild portal infiltrate that are highly suggestive of drug-induced cholestasis. DISCUSSION: This is the fourth report in the English-language literature describing cholestatic hepatitis temporally related to celecoxib use, the second supported by histologic findings typical of drug-induced cholestasis, and the first in a patient who denied use of alcoholic beverages and was taking no other drugs or herbal products at the time of the reaction. The Naranjo probability scale indicated that celecoxib was a probable cause of acute cholestatic hepatitis in this patient. CONCLUSIONS: Cholestatic hepatitis is a well-recognized adverse effect of several drugs. Although celecoxib is considered to have a very low potential for hepatic toxicity, well-documented reports of adverse reactions can contribute significantly to the definition of more accurate safety profiles for new drugs introduced into clinical practice.

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