Dataset Information

Off-label use of targeted therapies in osteosarcomas: data from the French registry OUTC'S (Observatoire de l'Utilisation des Therapies Ciblees dans les Sarcomes).

ABSTRACT: The objective of this study is to explore the off-label use of targeted therapies (TTs) for patients with osteosarcoma registered within the French Sarcoma Group--Bone Tumor Study Group (GSF-GETO) national registry.All patients with an osteosarcoma, registered between January 1, 2009 and July 15, 2013 were analyzed.Twenty-nine patients with refractory relapsed osteosarcomas received 33 treatment lines of TTs. The median age at the beginning of treatment was 19 years (range 9-72). The median number of previous lines of chemotherapy was 3 (range 1-8). Before inclusion, 3 patients were in second complete remission, 26 were in progression for metastatic relapse. Twenty-three patients received sirolimus (in combination with cyclophosphamide for 18); 5, sunitinib; 4, sorafenib; and one, pazopanib. Stable disease was observed for 45.5% of patients (95% Confidence Interval (CI) [20-52.8]). The median Progression-Free Survival (PFS) was 3 months (95% CI [2-5.4]) for patients treated by sirolimus and 1.8 months (95% CI [1.3-2.8]) for patients receiving multi-targeted tyrosine kinase inhibitors; 6-month PFS 15%. The median Overall Survival (OS) was 6.8 months (95% CI [4.7-12.1]), and one-year OS was 24%. In a multivariate analysis, PFS was superior for patients receiving sirolimus compared to other TTs (Hazard Ratio (HR)?= 2.7, 95% CI [1.05-7.1]). No toxic death was reported. Grade 3 and 4 toxicities were observed in 27 and 6% of cases respectively.Off-label TTs, especially sirolimus, reported benefit in the treatment of refractory osteosarcomas with an acceptable toxicity profile, including in pediatric population.

SUBMITTER: Penel-Page M

PROVIDER: S-EPMC4635968 | biostudies-other | 2015 Nov

REPOSITORIES: biostudies-other

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Off-label use of targeted therapies in osteosarcomas: data from the French registry OUTC'S (Observatoire de l'Utilisation des Thérapies Ciblées dans les Sarcomes).

Penel-Page Mathilde M Ray-Coquard Isabelle I Larcade Julie J Girodet Magali M Bouclier Laure L Rogasik Muriel M Corradini Nadège N Entz-Werle Natacha N Brugieres Laurence L Domont Julien J Lervat Cyril C Piperno-Neumann Sophie S Pacquement Helène H Bay Jacques-Olivier JO Gentet Jean-Claude JC Thyss Antoine A Chaigneau Loic L Narciso Bérangère B Cornille Helène H Blay Jean-Yves JY Marec-Bérard Perrine P

BMC cancer 20151105

<h4>Background</h4>The objective of this study is to explore the off-label use of targeted therapies (TTs) for patients with osteosarcoma registered within the French Sarcoma Group--Bone Tumor Study Group (GSF-GETO) national registry.<h4>Methods</h4>All patients with an osteosarcoma, registered between January 1, 2009 and July 15, 2013 were analyzed.<h4>Results</h4>Twenty-nine patients with refractory relapsed osteosarcomas received 33 treatment lines of TTs. The median age at the beginning of t ...[more]

PMID: 26541413

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Project description:Introduction L’épidémie COVID-19 a contraint enseignants et étudiants à s’adapter rapidement à des modes d’enseignements « distanciels ». Le format « visioconférence » s’est généralisé à tous les enseignements, de la formation initiale jusqu’aux congrès virtuels. Ces roues de secours, que nous pensions transitoires, ont modifié nos pratiques pendant plus d’un an : que faut-il en retenir pour notre enseignement ? Notre objectif était d’évaluer auprès des enseignants et des internes de dermatologie, leur expérience numérique au cours de l’épidémie. Matériel et méthodes Entre le 10 et le 21/05/2021, une enquête par questionnaires, destinée aux enseignants et aux internes, a porté sur les thématiques suivantes: expériences en enseignement « visio »; aspects techniques; ressenti pédagogique. Les questionnaires ont été envoyés par un lien GoogleForm aux mailing-listes « enseignants » et « internes » du CEDEF; les réponses étaient anonymes. Résultats Les taux de participation étaient de 50 répondants/106 enseignants (47 %) et de 120 répondants/453 internes (26 %). La majorité des enseignants avait participé à de multiples enseignements en « visio » (cours DFASM 96%, congrès 84 %, réunion DES 70 %, cours DES 58 %, DU/DIU 56 %, EPU 60 %) et était autonome pour la “visio” (cours DFASM et DES 78 %, réunion DES 58 %, EPU 56 %). Le logiciel Zoom était utilisé par la majorité (90 %) des enseignants et des internes. La connexion restait problématique au CHU pour plus de 80 % des répondants. La « simplicité d’utilisation » était la qualité prioritaire d’un logiciel « visio ». Une majorité des enseignants estimait que les cours de DES (54 %) et les réunions de DES (52 %) pourraient être pérennisés en distanciel, tandis que les internes estimaient que les DU/DIU (56 %), les cours de DES (57 %), les réunions de DES (56 %) et les séminaires du CEDEF (61 %) pourraient être maintenues « en visio » après la pandémie. Seuls 3% des internes estimaient qu’aucun cours ne devrait être maintenu en “visio” contre 14% des enseignants (p = 0,03). Le format « hybride » des futurs congrès était plébiscité par la majorité (> 70 %) des répondants. Discussion L’expérience COVID a servi de catalyseur à un nouveau mode d’enseignement, pour lequel les enseignants ont acquis une expérience diversifiée et une certaine autonomie. Les enseignants sont plus réticents que les étudiants au maintien de formats « visio » en post-COVID, ces derniers privilégiant le format « visio » pour de nombreuses formes d’enseignements. Ainsi, la digitalisation de certains séminaires du CEDEF permet une accessibilité à un plus grand nombre et un suivi personnalisé du parcours de l’étudiant. Le présentiel reste privilégié pour les formations post-universitaires, les solutions « hybrides » permettant de combiner les avantages des deux formats.

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Project description:BACKGROUND: Postmarket surveillance of the comparative safety and efficacy of orphan therapeutics is challenging, particularly when multiple therapeutics are licensed for the same orphan indication. To make best use of product-specific registry data collected to fulfill regulatory requirements, we propose the creation of a distributed electronic health data network among registries. Such a network could support sequential statistical analyses designed to detect early warnings of excess risks. We use a simulated example to explore the circumstances under which a distributed network may prove advantageous. METHODS: We perform sample size calculations for sequential and non-sequential statistical studies aimed at comparing the incidence of hepatotoxicity following initiation of two newly licensed therapies for homozygous familial hypercholesterolemia. We calculate the sample size savings ratio, or the proportion of sample size saved if one conducted a sequential study as compared to a non-sequential study. Then, using models to describe the adoption and utilization of these therapies, we simulate when these sample sizes are attainable in calendar years. We then calculate the analytic calendar time savings ratio, analogous to the sample size savings ratio. We repeat these analyses for numerous scenarios. KEY RESULTS: Sequential analyses detect effect sizes earlier or at the same time as non-sequential analyses. The most substantial potential savings occur when the market share is more imbalanced (i.e., 90% for therapy A) and the effect size is closest to the null hypothesis. However, due to low exposure prevalence, these savings are difficult to realize within the 30-year time frame of this simulation for scenarios in which the outcome of interest occurs at or more frequently than one event/100 person-years. CONCLUSIONS: We illustrate a process to assess whether sequential statistical analyses of registry data performed via distributed networks may prove a worthwhile infrastructure investment for pharmacovigilance.

Dataset Information

Off-label use of targeted therapies in osteosarcomas: data from the French registry OUTC'S (Observatoire de l'Utilisation des Therapies Ciblees dans les Sarcomes).

Publications

Off-label use of targeted therapies in osteosarcomas: data from the French registry OUTC'S (Observatoire de l'Utilisation des Thérapies Ciblées dans les Sarcomes).

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