Characteristics of self-medication in French students.
PMID: 33039100 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - September 28, 2020 Category: Psychiatry & Psychology Authors: Bagheri H Tags: Therapie Source Type: research

Novel forms of injectable buprenorphine and French model of opioid use disorder treatment.
PMID: 33039103 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - September 28, 2020 Category: Psychiatry & Psychology Authors: Lapeyre-Mestre M Tags: Therapie Source Type: research

Possible apixaban-induced agranulocytosis: A life-threatening adverse drug reaction causing by a widely prescribed drug.
ad S PMID: 33039102 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - September 26, 2020 Category: Psychiatry & Psychology Authors: Chen P, Zamy M, Nili-Asgari R, Cymbalista F, Dhôte R, Abad S Tags: Therapie Source Type: research

TNF- α inhibitors and psychiatric adverse drug reactions in the spectrum of bipolar (manic) or psychotic disorders: Analysis from the French pharmacovigilance database.
CONCLUSION: Depressive disorders are well-known ADR of TNF-α inhibitors, but we report, here, 41 reports of psychiatric ADR diagnosed as manic or psychotic disorders or in the spectrum of bipolar or psychotic disorders with these treatments. Epidemiological studies are needed to confirm this signal. PMID: 33039101 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - September 25, 2020 Category: Psychiatry & Psychology Authors: Yelnik CM, Gaboriau L, Petitpain N, Théophile H, Delaporte E, Carton L, Gautier S, Lambert M Tags: Therapie Source Type: research

Do immunosuppressive agents hamper the vaccination response in patients with rheumatic diseases? A review of the literature.
Abstract Patients with auto-immune disease are more susceptible to infection than similar populations without auto-immune disease. Vaccination seems to be one of the most effective methods to prevent patients from possible infections, but may be impaired by concomitant immunomodulators. The aim of this review was to evaluate the effect of immunosuppressive drugs on vaccination efficiency. We found that the majority of studies confirms that neither the use of corticosteroids and conventional synthetic disease-modifying antirheumatic drugs (csDMARDs) nor the use of biological agents, except rituximab, reduce the eff...
Source: Therapie - September 2, 2020 Category: Psychiatry & Psychology Authors: Ben Nessib D, Fazaa A, Miladi S, Sellami M, Ouenniche K, Souabni L, Kassab S, Chekili S, Ben Abdelghani K, Laatar A Tags: Therapie Source Type: research

Adherence to biologic disease-modifying antirheumatic drugs in adult patients with rheumatic diseases.
CONCLUSION: This work has revealed the low rate of adherence to biological treatments in Tunisian patient with RD. Predictors of poor adherence were the absence of coxitis and a rate of administration of bDMARDs less than one injection per month. Detection of these factors could help us to adapt our strategies to improve adherence that are essentially based on therapeutic education program. PMID: 32948325 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - September 1, 2020 Category: Psychiatry & Psychology Authors: Fazaa A, Makhlouf Y, Ben Ouhiba A, Miladi S, Sellami M, Ouenniche K, Souabni L, Kassab S, Chekili S, Zakraoui L, Ben Abdelghani K, Laatar A Tags: Therapie Source Type: research

Severe polyuria secondary to acyclovir induced-proximal tubulopathy.
er AS PMID: 32919761 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - August 27, 2020 Category: Psychiatry & Psychology Authors: Yvin E, Beloncle F, Thépot S, Lagarce L, Augusto JF, Garnier AS Tags: Therapie Source Type: research

COVID-19  and NSAIDs: Primum non nocere.
COVID-19 and NSAIDs: Primum non nocere. Therapie. 2020 Jul 22;: Authors: Micallef J, Soeiro T, Jonville-Béra AP PMID: 32839015 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - July 21, 2020 Category: Psychiatry & Psychology Authors: Micallef J, Soeiro T, Jonville-Béra AP Tags: Therapie Source Type: research

No arguments for extra risk from ibuprofen in SARS-COV2 infection.
PMID: 32829912 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - July 10, 2020 Category: Psychiatry & Psychology Authors: Moore N Tags: Therapie Source Type: research

Drug prescriptions in French pregnant women between 2015 and 2016: A study in the EGB database.
CONCLUSION: This descriptive study demonstrates that numerous drugs are prescribed and dispensed to pregnant women in France. These include drugs with a proven teratogenic or fetotoxic effect and many drugs that have not yet been well evaluated in pregnancy. PMID: 32736872 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - July 9, 2020 Category: Psychiatry & Psychology Authors: Araujo M, Hurault-Delarue C, Sommet A, Damase-Michel C, Benevent J, Lacroix I Tags: Therapie Source Type: research

[Evaluation of direct oral anticoagulants (DOACs) prescriptions in geriatric hospital over 3 years].
CONCLUSION: The prescription of DOACs in elderly patients has increased considerably in recent years. However, dosing errors remain frequent (overdoses as underdoses), associated with hemorrhagic or thrombotic events. Similarly, drug interactions should be known as they are a risk factor for avoidable under- or overdose. Improving prescriptions of DOACs in geriatrics needs to respect the rules of good practice, to regularly reassess renal function, and to transmit observations of adverse effects to pharmacovigilance centers. PMID: 32736873 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - July 9, 2020 Category: Psychiatry & Psychology Authors: Nahornyj E, Goutelle S, Bourguignon L, de la Gastine B Tags: Therapie Source Type: research

The cardiovascular health of prisoners who use cannabis: An exploratory study among hospitalised prisoners.
CONCLUSION: This description of serious cardiovascular outcomes in prisoners who use cannabis provides insights into the clinical features possibly observed in this vulnerable population The findings indicate that 7.5% of hospitalizations of prisoners for cardiovascular disorders are potentially linked to cannabis used in prison. PMID: 32718583 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - July 8, 2020 Category: Psychiatry & Psychology Authors: Mongiatti M, Bayle P, Lagarrigue A, Fabre D, Telmon N, Lapeyre-Mestre M, Jouanjus E Tags: Therapie Source Type: research

Long-term use of proton pump inhibitors as a risk factor for various adverse manifestations.
Abstract The long-term use of proton pump inhibitors (PPIs) can lead to increased gastric pH, hypochlorhydria and in some cases to achlorhydria when compared to other acid-suppressing agents like histamine-2 (H2) receptor blockers and antacids. These consequences by the use of long-term PPIs may lead to significant vitamin (B12 and C) and mineral (iron, calcium and magnesium) deficiencies which needs gastric acid for their absorption and bioavailability. Long-term use of PPIs by the pregnant patients may impose a potential risk of congenital malformations. Various studies have recommended the life style modificati...
Source: Therapie - July 8, 2020 Category: Psychiatry & Psychology Authors: Koyyada A Tags: Therapie Source Type: research

[Reaction during dalbavancin infusion: About one case].
enneville E PMID: 32723578 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - July 7, 2020 Category: Psychiatry & Psychology Authors: Valentin B, Dartus J, Trouillez T, Gaboriau L, Loiez C, Dezèque H, Odou P, Décaudin B, Migaud H, Genay S, Senneville E Tags: Therapie Source Type: research

Evolution of clotting factor concentrates prescriptions and impact of recommendations of prophylaxis in children with haemophilia.
The objective of this 16-year study (2001 to 2016) was to describe the clotting factor concentrates (CFCs) use in haemophiac outpatients. This is a retrospective monocentric study. Pharmaceutical and clinical data were captured using medical records. Main outcome measures are CFCs use and clinical data in paediatrics. Eighty haemophiliacs A or B under 12 years old with a factor level less than 2% were included (74% of HA), from pharmaceutical outpatient data. Global use of CFCs followed the evolution of the patients' number and regimen type introduced: increase of prophylaxis and decrease of on demand regimen. The average ...
Source: Therapie - July 2, 2020 Category: Psychiatry & Psychology Authors: Bel F, Meunier S, Lienhart A, Revy A, Chamouard V Tags: Therapie Source Type: research

Efficacy and safety of neural stem cell therapy for spinal cord injury: A systematic literature review.
Abstract AIMS: To summarize the evidence on the efficacy and safety of neural stem cell therapy (NSCT) for the treatment of spinal cord injury (SCI). METHODS: A systematic literature review of Medline®, EMBASE® and Cochrane library was performed to identify studies reporting efficacy and safety of NSCT in SCI. Articles were included if they reported efficacy and safety data of SCI patients who received NSCT. RESULTS: Overall, four studies of the 277 records met all the study eligibility criteria. Over the 1-year follow-up period, motor scores were significantly higher among patients who received ...
Source: Therapie - June 26, 2020 Category: Psychiatry & Psychology Authors: Tiwari S, Khan S, Kumar SV, Rajak R, Sultana A, Pasha SA, Gauba D, Ghosh P, Khurana T, Kulkarni A, Reddy YP, Khan AA, Sharma VK Tags: Therapie Source Type: research

Challenges of autoimmune rheumatic disease treatment during the COVID-19 pandemic: A review.
acute;rapeutique (SFPT) Abstract Since December 2019, the COVID-19 pandemic has become a major public health problem. To date, there is no evidence of a higher incidence of COVID in patients with autoimmune rheumatic diseases and we support the approach of maintaining chronic rheumatological treatments. However, once infected there is a small but significant increased risk of mortality. Among the different treatments, NSAIDs are associated with higher rates of complications, but data for other drugs are conflicting or incomplete. The use of certain drugs for autoimmune inflammatory rheumatisms appears to be a pote...
Source: Therapie - June 26, 2020 Category: Psychiatry & Psychology Authors: Grange L, Guilpain P, Truchetet ME, Cracowski JL, Société française de pharmacologie et de thérapeutique (SFPT) Tags: Therapie Source Type: research

NSAIDs may increase the risk of thrombosis and acute renal failure in patients with COVID-19 infection.
PMID: 32636031 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - June 26, 2020 Category: Psychiatry & Psychology Authors: Cumhur Cure M, Kucuk A, Cure E Tags: Therapie Source Type: research

Are β-lactam concentrations adequate in severe sepsis and septic shock in children?
Are β-lactam concentrations adequate in severe sepsis and septic shock in children? Therapie. 2020 Jun 13;: Authors: Chosidow A, Benaboud S, Beranger A, Zheng Y, Moulin F, Dupic L, Renolleau S, Treluyer JM, Oualha M Abstract AIM OF THE STUDY: Early administration of appropriate antibiotic therapy with adequate concentration is the cornerstone of the severe sepsis and septic shock's treatment. We aim to describe the plasma concentration of the most used β-lactams in critically ill children, to describe the rate of patients with suboptimal exposure, and associated clinical and biological facto...
Source: Therapie - June 12, 2020 Category: Psychiatry & Psychology Authors: Chosidow A, Benaboud S, Beranger A, Zheng Y, Moulin F, Dupic L, Renolleau S, Treluyer JM, Oualha M Tags: Therapie Source Type: research

[Potentially inappropriate medication: Adaptation of EU(7)PIM criteria to the French medical practice].
CONCLUSION: This work provides an adaptation of the EU(7)PIM to the French medical practice with the guidance of the HAS recommendations. This list is intended to be easy to use for the identification of PIMs by French physicians. PMID: 32593421 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - June 5, 2020 Category: Psychiatry & Psychology Authors: Zacarin A, Strumia M, Piau A, Bagheri H Tags: Therapie Source Type: research

Ultrafast response of the French Society of Pharmacology and Therapeutics to the COVID-19 pandemic.
PMID: 32553502 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - June 5, 2020 Category: Psychiatry & Psychology Authors: Cracowski JL, Molimard M, Richard V Tags: Therapie Source Type: research

The French pharmacovigilance surveys: A French distinctiveness, a real input.
CONCLUSION: The current process allows a collegiate, clinical and pharmacological discussion with integration of the regulatory framework. A significant number of new signals are detected and/or validated. This efficient tool which is unique in Europe and is allowed by the decentralized pharmacovigilance which leads to a great reactivity of decision-making. It has to be reassessed and to be adapted continuously to the new challenges of the growing multiplication of signals. PMID: 32553501 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - May 23, 2020 Category: Psychiatry & Psychology Authors: Abou Taam M, Jacquot B, Ferard C, Thery AC, Mounier C, Grandvuillemin A, Jonville-Béra AP, Perault-Pochat MC, Réseau français des centres régionaux de pharmacovigilance Tags: Therapie Source Type: research

Is the drug a scientific, social or political object?
PMID: 32482295 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - May 22, 2020 Category: Psychiatry & Psychology Authors: Bordet R Tags: Therapie Source Type: research

Chloroquine and hydroxychloroquine in the management of COVID-19: Much kerfuffle but little evidence.
Abstract Chloroquine and hydroxychloroquine are drugs that have shown in vitro activity on the replication of certain coronaviruses. In the context of the SARS-Cov-2 epidemic, the virus responsible for the novel coronavirus disease (COVID-19), these two drugs have been proposed as possible treatments. The results of the first clinical studies evaluating the effect of hydroxychloroquine do not support any efficacy of this drug in patients with COVID-19, due to major methodological weaknesses. Yet, these preliminary studies have aroused considerable media interest, raising fears of massive and uncontrolled use. In t...
Source: Therapie - May 22, 2020 Category: Psychiatry & Psychology Authors: Roustit M, Guilhaumou R, Molimard M, Drici MD, Laporte S, Montastruc JL, French Society of Pharmacology and Therapeutics (SFPT) Tags: Therapie Source Type: research

Diabetes and COVID-19.
Abstract According to previous reports, diabetes seems to be a risk factor which worsens the serious clinical events caused by COVID-19. But is diabetes per se a risk factor that increases the probability of getting the virus? This paper will discuss this point. There are not many research data on antidiabetic drugs in this context. The potential influence of glucose-lowering agents on the severity of COVID-19 has not been described yet. Dipeptidylpeptidase-4 (DPP-4) is a cell surface protein ubiquitously expressed in many tissues and it is also a soluble molecule found in serum/plasma fluids. DPP-4 is involved in...
Source: Therapie - May 14, 2020 Category: Psychiatry & Psychology Authors: Bouhanick B, Cracowski JL, Faillie JL, French Society of Pharmacology, Therapeutics (SFPT) Tags: Therapie Source Type: research

Diabetes and COVID-19.
PMID: 32425249 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - May 14, 2020 Category: Psychiatry & Psychology Authors: Bouhanick B, Cracowski JL, Faillie JL, French Society of Pharmacology and Therapeutics (SFPT) Tags: Therapie Source Type: research

Pharmacological characteristics of patients infected with SARS-Cov-2 admitted to Intensive Care Unit in South of France.
PMID: 32425250 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - May 14, 2020 Category: Psychiatry & Psychology Authors: Montastruc F, Romano C, Montastruc JL, Silva S, Seguin T, Minville V, Georges B, Riu-Poulenc B, Fourcade O Tags: Therapie Source Type: research

Incretin-based drugs and intestinal obstruction: A pharmacovigilance study.
CONCLUSIONS: We identified a pharmacovigilance signal that suggests a risk of potentially serious intestinal obstruction associated with incretin-based drugs, as a class and with a greater signal for DPP4-i. Other studies are needed to confirm and better understand the potential risk of intestinal obstruction associated with incretin-based drugs. PMID: 32418731 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - May 10, 2020 Category: Psychiatry & Psychology Authors: Gudin B, Ladhari C, Robin P, Laroche ML, Babai S, Hillaire-Buys D, Faillie JL Tags: Therapie Source Type: research

Chloroquine and hydroxychloroquine during pregnancy: What do we know?
se-Michel C PMID: 32418732 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - May 10, 2020 Category: Psychiatry & Psychology Authors: Lacroix I, Bénévent J, Damase-Michel C Tags: Therapie Source Type: research

Refractory non-variceal upper gastrointestinal bleeding in a liver-transplant patient: Everolimus withdrawal should be considered.
PMID: 32418733 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - May 7, 2020 Category: Psychiatry & Psychology Authors: Morel G, Morandeau V, Grunebaum L, Tebacher M, Faitot F, Schneider F Tags: Therapie Source Type: research

Non-steroidal anti-inflammatory drugs, pharmacology, and COVID-19 infection.
SFPT) Abstract Non-steroidal anti-inflammatory drugs (NSAIDs) have an optional prescription status that has resulted in frequent use, in particular for the symptomatic treatment of fever and non-rheumatic pain. In 2019, a multi-source analysis of complementary pharmacological data showed that using NSAIDs in these indications (potentially indicative of an underlying infection) increases the risk of a severe bacterial complication, in particular in the case of lung infections. First, the clinical observations of the French Pharmacovigilance Network showed that severe bacterial infections can occur even after a shor...
Source: Therapie - May 6, 2020 Category: Psychiatry & Psychology Authors: Micallef J, Soeiro T, Jonville-Béra AP, French Society of Pharmacology, Therapeutics (SFPT) Tags: Therapie Source Type: research

Meningiomas after cyproterone acetate exposure: Case reports in twin sisters.
PMID: 32418729 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - May 6, 2020 Category: Psychiatry & Psychology Authors: de Germay S, Lafaurie M, Dupuy M, de Germay B Tags: Therapie Source Type: research

"Off-label" use of hydroxychloroquine, azithromycin, lopinavir-ritonavir and chloroquine in COVID-19: A survey of cardiac adverse drug reactions by the French Network of Pharmacovigilance Centers.
CONCLUSION: "Off-label" use of treatments in COVID-19 increases the risk of cardiac ADRs, some of them avoidable. Even if these drugs are perceived as familiar, they are used in patients with added risk factors caused by infection. Precautions should be taken to mitigate the risk, even if they will be proven efficacious. PMID: 32418730 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - May 6, 2020 Category: Psychiatry & Psychology Authors: Gérard A, Romani S, Fresse A, Viard D, Parassol N, Granvuillemin A, Chouchana L, Rocher F, Drici MD, French Network of Pharmacovigilance Centers Tags: Therapie Source Type: research

Adverse drug reactions of hydroxychloroquine: Analysis of French pre-pandemic SARS-CoV2 pharmacovigilance data.
PMID: 32402475 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - May 4, 2020 Category: Psychiatry & Psychology Authors: Grandvuillemin A, Fresse A, Cholle C, Yamani S, Dautriche A, French network of regional pharmacovigilance centres Tags: Therapie Source Type: research

Interest of a general practitioner pharmacovigilance network to provide drug information: A comparative study in France.
CONCLUSION: This is the first study about GPs' drug-related questions asked to a PV center. We found that an organization, such as a CRA-GP "face to face" visit, increased the number of drug questions. This kind of organization should be developed in order to improve independent drug information outreach. PMID: 32354462 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - April 21, 2020 Category: Psychiatry & Psychology Authors: Jacquot J, Rousseau V, de Canecaude C, Montastruc JL, Durrieu G Tags: Therapie Source Type: research

Olmesartan-associated enteropathy: Belgian survey.
PMID: 32284216 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - March 28, 2020 Category: Psychiatry & Psychology Authors: Allaoui A, Marcoux D, Vokaer B, Couturier B Tags: Therapie Source Type: research

The 2019 Global Initiative for Asthma report: Evidence-based or evidence-biased medicine?
PMID: 32199637 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - February 25, 2020 Category: Psychiatry & Psychology Authors: Girodet PO, Molimard M Tags: Therapie Source Type: research

Severe serotonin syndrome caused by an interaction between an antidepressant and a cough syrup.
, Rigaud JP PMID: 32192714 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - February 25, 2020 Category: Psychiatry & Psychology Authors: Declercq PL, Eraldi JP, Beuzelin M, Gélinotte S, Marchalot A, Bougerol F, Allorge D, Loriot MA, Paret N, Mégarbane B, Rigaud JP Tags: Therapie Source Type: research

Uses of pharmacovigilance databases: An overview.
Abstract Over the past decades, assessment of drug safety and of their benefits harms balance has been profoundly modified by the availability of large databases and computerized automated statistical approaches. Improvement of digital data storage capacity has been applied to pharmacovigilance reports. VigiBase, the international pharmacovigilance database, is now aggregating over 21 million individual case safety reports in 2020. Identification and investigation of drug safety signals - concerning notably rare and unknown adverse drug reactions - is one of the major tasks in pharmacovigilance that can ...
Source: Therapie - February 25, 2020 Category: Psychiatry & Psychology Authors: Bihan K, Lebrun-Vignes B, Funck-Brentano C, Salem JE Tags: Therapie Source Type: research

[Capecitabine aggravating lentiginosis].
PMID: 32169288 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - February 20, 2020 Category: Psychiatry & Psychology Authors: Idoudi S, Boussofara L, Belkahla M, Ghariani Fetoui N, Ghariani N, Belajouza C, Denguezli M Tags: Therapie Source Type: research

The challenges of pharmacoepidemiology of orphan drugs in rare diseases.
Abstract Large electronic health records database available at a national level offer great opportunity for research in rare diseases and orphan drugs. Methods and data used in pharmacoepidemiology present a great potential for epidemiology, drug utilization studies, drug safety, drug effectiveness and pharmacoeconomics. This review presents the different sources of data in Europe, with a special focus on the French situation, with the recent implementation of SNDS (système national des données de santé [French national health data wharehouse]). Some examples are given. Development of rigorous...
Source: Therapie - February 12, 2020 Category: Psychiatry & Psychology Authors: Lapeyre-Mestre M Tags: Therapie Source Type: research

Drug repositioning for rare diseases: Knowledge-based success stories.
Abstract While more than 7000 rare diseases have been identified, only about 5 percent benefit from a licensed treatment. As the majority of these diseases is life threatening, these facts underscore the need for new drugs. Drug repositioning is an alternative strategy in drug development, which represents an attractive opportunity for rare diseases. Drug repositioning (also called drug repurposing, drug reprofiling or drug re-tasking) consists in identifying for an already approved or investigational drug a new use outside the scope of the original medical indication. Drug repositioning is considered in the field...
Source: Therapie - February 12, 2020 Category: Psychiatry & Psychology Authors: Scherman D, Fetro C Tags: Therapie Source Type: research

A methodological framework for drug development in rare diseases. The CRESim program: Epilogue and perspectives.
Abstract Based on the 'European Child-Rare-Euro-Simulation' (CRESim) project, this article proposes a generalizable strategy utilizing datasets analysis in combination with modeling and simulation, in order to optimize the clinical drug development applied in the field of rare diseases. The global process includes: (i) the simulation of a realistic virtual population of patients (modeled from a real dataset of patients), (ii) the modeling of disease pathophysiological components and of pharmacokinetic-pharmacodynamic relations of the drug(s) of interest, (iii) the modeling of several randomized controlled clinical...
Source: Therapie - February 12, 2020 Category: Psychiatry & Psychology Authors: Nony P, Kassai B, Cornu C, CRESim, Epi-CRESim research groups Tags: Therapie Source Type: research

Orphan drug designation in Europe: A booster for the research and development of drugs in rare diseases.
Abstract The research and drug development process in rare diseases is challenging in addition to those for common diseases. To stimulate its development, the orphan drug designation (ODD) was introduced in in European Union in 2000. In the present paper, we describe the main characteristics of ODD in European Union in particular the requested criteria for ODD, the overview of the general procedure and the main incentives for Sponsors and finally the predicted factors related to successful development and marketing approval of orphan drugs after designation. In accordance with regulation, an application for ODD mu...
Source: Therapie - February 12, 2020 Category: Psychiatry & Psychology Authors: Micallef J, Blin O Tags: Therapie Source Type: research

An old drug for a rare disease: The example of celiprolol for vascular Ehlers-Danlos syndrome.
Abstract Vascular Ehlers-Danlos syndrome (OMIM 130050, 1/150,000 birth) is caused by mutations in collagen 3A1 gene. It is associated with severe phenotype associating early arterial dissection and rupture, digestive and uterine perforations, and skin and joints fragility. Until recently, no treatment was available. Celiprolol, a beta1 antagonist with beta2 partial antagonist properties betablocker was tested in a randomized, controlled trial. We could show that this compound was associated with a 3-fold decrease in major events related to the disease. This effect was similar in molecular-prove...
Source: Therapie - February 12, 2020 Category: Psychiatry & Psychology Authors: Boutouyrie P Tags: Therapie Source Type: research

Patients' organizations in rare diseases and involvement in drug information: Illustrations with LMC France, the French Association of Chronic Myeloid leukemia.
Abstract There are few areas in medicine in which patient-parent advocacy groups play such a central and prominent role as in the field of rare diseases. As illustrated by the French Association of Chronic Myeloid Leukemia (LMC France), its contribution is not only focused on its role as participants or key informant related to clinical studies but also on drug information and drug safety. The discovery of tyrosine kinase inhibitors against the BCR-ABL1 oncogenic fusion protein has revolutionized the management of CML, becoming a chronic illness rather than a life-threatening disease. Because ensuring ongoing well...
Source: Therapie - February 12, 2020 Category: Psychiatry & Psychology Authors: Daban M, Lacroix C, Micallef J Tags: Therapie Source Type: research

Pharmacovigilance and drug-induced rare diseases: Strengths of the French Network of Regional Pharmacovigilance Centres.
Abstract The French-style organization in the field of rare diseases allows a close contact between reference centres and regional pharmacovigilance centres thanks to their implementation within the French university hospital. This collaboration leads to highlight more and more drug-induced rare diseases. Through several historical examples (eosinophilia-myalgia syndrome due to L-tryptophan, type 1 narcolepsy with H1N1 pandemic influenza vaccine, capillary leak syndrome, acquired von Willebrand syndrome), it remains clear that pharmacovigilance is the cornerstone of the alert system. Clinicians from the rare disea...
Source: Therapie - February 12, 2020 Category: Psychiatry & Psychology Authors: Lacroix C, Mallaret M, Jonville-Bera AP Tags: Therapie Source Type: research

Update on bradykinin-mediated angioedema in 2020.
Abstract Bradykinin-mediated angioedema is a rare disease, due to vasodilation and increased vascular permeability resulting from bradykinin. This kind of angioedema affects abdominal and/or upper airways. It differs clinically from histamine-mediated angioedema by the absence of urticaria or skin rash. Antihistamines and corticosteroids are not effective. Delayed diagnosis can lead to inadequate and potentially fatal management by asphyxiation. Bradykinin-mediated angioedema results from either overproduction of bradykinin or inhibition of its degradation. Etiology can be hereditary or acquired. Deficiency of C1 ...
Source: Therapie - February 12, 2020 Category: Psychiatry & Psychology Authors: Lepelley M, Bernardeau C, Defendi F, Crochet J, Mallaret M, Bouillet L Tags: Therapie Source Type: research

[Satisfaction survey in the clinical investigations center (CIC-1427) in Saint-Louis hospital Paris, France].
PMID: 32113690 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - February 7, 2020 Category: Psychiatry & Psychology Authors: Dupont A, Chevret S, Montlahuc C, Resche-Rigon M, Fontaine Y, Allaoua S, Kiladjian JJ, Ghrieb Z Tags: Therapie Source Type: research

Warning on increased serious health complications related to non-medical use of nitrous oxide.
let P PMID: 32063400 [PubMed - as supplied by publisher] (Source: Therapie)
Source: Therapie - January 16, 2020 Category: Psychiatry & Psychology Authors: French Association of Addictovigilance Centres, Micallef J, Mallaret M, Lapeyre-Mestre M, Daveluy A, Victorri-Vigneau C, Peyrière H, Debruyne D, Deheul S, Bordet R, Chevallier C, Perault-Pochat MC, Le Boisselier R, Libert F, Chaouachi L, Gillet P Tags: Therapie Source Type: research