Listing 1 - 10 of 33 | << page >> |
Sort by
|
Choose an application
Complex vascular malformations can induce pain, cosmetic and functional troubles, being therefore responsible for important morbidity.Despite these complications, we lack guidelines for the treatment of these pathologies (Hammill et al., 2011). As conventional treatments such as interventional radiology and/or surgery are seldomly curative, there is a need for new therapeutic tools. Recent pre-clinical studies showed the importance of the PBKinase/AKT/mTOR pathway in the growth and organisation of the vascular and lymphatic network, and suggested a possible therapeutic efficacy by inhibition of this pathway in patients with complex vascular malformations (Hammill et al., 2011).Purpose: We realized a prospective phase II clinical trial to evaluate efficacy and security of Rapamycin, mTOR inhibitor, in children and adults presenting microcystic lymphatic malformations, extensive venous malformations and/or complex vascular malformations, for which conventional treatments are inefficient. Method: Eighteen patients aged from 3 to 64 years, symptomatic despite conventional treatments, were enrolled in the study. Rapamycin was administrated as tablets for adults and oral solution for children. Efficacy was evaluated on a monthly basis by anamnesis of symptoms (functional, cosmetic and psychological), pain evolution (frequency and intensity), quality of life questionnaire, clinical parameters, photographies of the visible clinical lesions and blood sampling. Yolumetric MRI and naso-pharyngeal fibroscopy (if required) were performed after 12 months of treatment. Side effects were evaluated according to CTCAE version 3.Results: Fifteen patients out of eighteen reached 12 months of follow-up. Most of them had a beneficial effect of Rapamycin. A significant response was observed in ail patients with lymphatic malformations, six venous malformations out of seven and ail patients with complex vascular anomalies. Clinical improvement observed in our patients was con-elated with the action mechanism of Rapamycin on the PI3K/Akt/mTOR pathway which intervenes in angiogenesis.Conclusion: Our study suggests that Rapamycin could become an alternative treatment for patients presenting microcystic lymphatic malformations, extensive venous and/or complex vascular anomalies not responding to conventional treatments. Les malformations vasculaires complexes sont à l'origine de douleurs. de troubles esthétiques et fonctionnels pouvant entraîner une morbidité importante. Outre ces complications, nous manquons d"arbres décisionnels concernant le traitement de ces pathologies (Hammill et al.. 2011). Les traitements conventionnels tels que la radiologie interventionnelle et/ou la chirurgie étant rarement curatifs, nous avons besoin de nouvelles options thérapeutiques.Des études précliniques récentes ont montré l'importance de la voie Pl3Kinase/AKT/mTOR dans la croissance et l'organisation vascula ire et lymphatique. et suggéré une possible efficacité thérapeutique par l ïnhi bition de cette voie pour les patients présentant des malfonnations vasculaires complexes (Hammill et al., 2011).But : Nous avons réalisé une étude prospective de phase II afin d"évaluer l'efficacité et la sécurité de la Rapamycine, inhibiteur mTOR, chez les enfants et les adultes présentant des malfonnati ons lymphatiques de type microkystiques, des malformations veineuses étendues et/ou des malfonnations vasculaires complexes, toutes réfractaires aux traitements conventionnels.Méthode : Dix-huit patients âgés de 3 à 64 ans, symptomatique s malgré les traitements conventionnels, ont été enrôlés dans l"étude. La Rapamycine a été administrée sous forme de comprimés chez les adultes et de solution orale chez les enfants. L'efficacité a été évaluée mensuellement par une anamnèse de la symptomatologie (fonctionnelle, esthétique et psychologique). l'évolution de la douleur (fréquence et intensité), un questionnaire de qualité de vie, un examen clinique, des photographies des lésions cliniques visibles et une prise de sang. Une IRM volumétrique et une fibroscopie naso-pharyngée (si nécessaire) ont été réalisées après 12 mois de traitement. Les effets secondaires ont été évalués selon le CTCAE version 3.Résultats : Quinze patients sur dix-huit ont atteint 12 mois de suivi. La majorité d'entre eux a montré un effet bénéfique de la Rapamycine. Une réponse significative a été observée chez tous les patients présentant des malformations lymphatiques, six patients sur sept avec des malformations veineuses et chez tous les patients avec des malfonnation s vasculaires complexes. L'amélioration clinique observée chez nos patients est corrélée au mécanisme d'action de la Rapamycine sur la voie Pl3K/AKT/mTOR qui intervient dans l'angiogenèse. Conclusion : Notre étude montre que la Rapamycine pourrait devenir un traitement alternatif pour les patients présentant des malfonnations lymphatiques microkystiques, veineuses étendues et/ou vasculaires complexes ne répondant pas aux traitements conventionnels.
Choose an application
Heart --- Cardiovascular Abnormalities --- Cardiovascular System --- Abnormalities
Choose an application
Fetal Heart --- Heart Diseases --- Cardiovascular Abnormalities --- Prenatal Diagnosis
Choose an application
Cardiovascular system --- Cardiovascular Abnormalities. --- Diseases --- Diseases. --- Cardiovascular diseases --- Abnormalities, Cardiovascular --- Abnormality, Cardiovascular --- Cardiovascular Abnormality --- Cardiovascular System --- abnormalities --- Cardiovascular Abnormalities
Choose an application
Choose an application
Migraine Disorders --- Migraine Disorders --- Cardiovascular Abnormalities --- Arteriovenous Malformations --- etiology --- prevention & control --- complications --- therapy
Choose an application
Cardiac Imaging Techniques --- Cardiovascular Abnormalities --- Cardiovascular system --- Child. --- Infant. --- Magnetic Resonance Imaging --- Methods. --- Radiography. --- Magnetic resonance imaging. --- Methods.
Choose an application
Cardiovascular Diseases --- Cardiovascular Abnormalities --- Infant --- Child --- Fetal Heart --- Pediatric cardiology --- Congenital heart disease in children --- Cardiologie pédiatrique --- Cardiopathie congénitale chez l'enfant --- Handbooks, manuals, etc. --- Guides, manuels, etc. --- Cardiology --- Fetus --- Heart Diseases --- Appareil cardiovasculaire --- Heart Defects, Congenital --- Maladies --- Chez l'enfant --- Cardiologie pédiatrique --- Cardiopathie congénitale chez l'enfant --- Cardiovascular Diseases. --- Heart Defects, Congenital. --- Infant. --- Fetus. --- Cardiovascular Abnormalities. --- Child. --- Fetal Heart. --- Cardiologie pédiatrique. --- Chez l'enfant.
Choose an application
Heart Defects, Congenital --- Adult --- Adults --- Abnormality, Heart --- Congenital Heart Defect --- Congenital Heart Defects --- Defects, Congenital Heart --- Heart Defect, Congenital --- Heart, Malformation Of --- Heart Abnormalities --- Abnormalities, Heart --- Defect, Congenital Heart --- Heart Abnormality --- Heart --- Myocardium --- abnormalities --- Cardiovascular Abnormalities --- Congenital Heart Disease --- Congenital Heart Diseases --- Disease, Congenital Heart --- Heart Disease, Congenital --- Malformation Of Heart --- Malformation Of Hearts
Choose an application
This invaluable book is a comprehensive review of congenital heart defects in the major mammalian groups. It begins with a comparison of normal cardiac anatomy between these groups. The analysis of cardiac malformations is simplified and presented in a didactic manner. The anatomy of the common malformations is presented with examples. A comment on the common anomalies seen in each group, together with an extensive reference list, helps the reader to access the source of information easily.
Congenital heart disease in animals. --- Mammals -- Diseases. --- Veterinary cardiology. --- Cardiovascular Abnormalities --- Heart Diseases --- Vertebrates --- Cardiovascular Diseases --- Chordata --- Congenital Abnormalities --- Diseases --- Animals --- Congenital, Hereditary, and Neonatal Diseases and Abnormalities --- Eukaryota --- Organisms --- Mammals --- Heart Defects, Congenital --- Health & Biological Sciences --- Veterinary Medicine --- Diseases. --- Eutheria --- Mammalia --- Mammalians --- Prototheria --- Theria --- Cardiology
Listing 1 - 10 of 33 | << page >> |
Sort by
|