Academic literature on the topic 'Robert Kimmel'

Background:Comparing drug effectiveness in observational settings is hampered by several major threats, among them confounding and attrition bias bias (patients who stop treatment no longer contribute information, which may overestimate true drug effectiveness).Objectives:To present points to consider (PtC) when analysing and reporting comparative effectiveness with observational data in rheumatology (EULAR-funded taskforce).Methods:The task force comprises 18 experts: epidemiologists, statisticians, rheumatologists, patients, and health professionals.Results:A systematic literature review of methods currently used for comparative effectiveness research in rheumatology and a statistical simulation study were used to inform the PtC (table). Overarching principles focused on defining treatment effectiveness and promoting robust and transparent epidemiological and statistical methods increase the trustworthiness of the results.Points to considerReporting of comparative effectiveness observational studies must follow the STROBE guidelinesAuthors should prepare a statistical analysis plan in advanceTo provide a more complete picture of effectiveness, several outcomes across multiple health domains should be comparedLost to follow-up from the study sample must be reported by the exposure of interestThe proportion of patients who stop and/or change therapy over time, as well as the reasons for treatment discontinuation must be reportedCovariates should be chosen based on subject matter knowledge and model selection should be justifiedThe study baseline should be at treatment initiation and a description of how covariate measurements relate to baseline should be includedThe analysis should be based on all patients starting a treatment and not limited to patients remaining on treatment at a certain time pointWhen treatment discontinuation occurs before the time of outcome assessment, this attrition should be taken into account in the analysis.Sensitivity analyses should be undertaken to explore the influence of assumptions related to missingness, particularly in case of attritionConclusion:The increased use of real-world comparative effectiveness studies makes it imperative to reduce divergent or contradictory results due to biases. Having clear recommendations for the analysis and reporting of these studies should promote agreement of observational studies, and improve studies’ trustworthiness, which may also facilitate meta-analysis of observational data.Disclosure of Interests:Delphine Courvoisier: None declared, Kim Lauper: None declared, Sytske Anne Bergstra: None declared, Maarten de Wit Grant/research support from: Dr. de Wit reports personal fees from Ely Lilly, 2019, personal fees from Celgene, 2019, personal fees from Pfizer, 2019, personal fees from Janssen-Cilag, 2017, outside the submitted work., Consultant of: Dr. de Wit reports personal fees from Ely Lilly, 2019, personal fees from Celgene, 2019, personal fees from Pfizer, 2019, personal fees from Janssen-Cilag, 2017, outside the submitted work., Speakers bureau: Dr. de Wit reports personal fees from Ely Lilly, 2019, personal fees from Celgene, 2019, personal fees from Pfizer, 2019, personal fees from Janssen-Cilag, 2017, outside the submitted work., Bruno Fautrel Grant/research support from: AbbVie, Lilly, MSD, Pfizer, Consultant of: AbbVie, Biogen, BMS, Boehringer Ingelheim, Celgene, Lilly, Janssen, Medac MSD France, Nordic Pharma, Novartis, Pfizer, Roche, Sanofi Aventis, SOBI and UCB, Thomas Frisell: None declared, Kimme Hyrich Grant/research support from: Pfizer, UCB, BMS, Speakers bureau: Abbvie, Florenzo Iannone Consultant of: Speaker and consulting fees from AbbVie, Eli Lilly, Novartis, Pfizer, Roche, Sanofi, UCB, MSD, Speakers bureau: Speaker and consulting fees from AbbVie, Eli Lilly, Novartis, Pfizer, Roche, Sanofi, UCB, MSD, Joanna KEDRA: None declared, Pedro M Machado Consultant of: PMM: Abbvie, Celgene, Janssen, Lilly, MSD, Novartis, Pfizer, Roche and UCB, Speakers bureau: PMM: Abbvie, BMS, Lilly, MSD, Novartis, Pfizer, Roche and UCB, Lykke Midtbøll Ørnbjerg Grant/research support from: Novartis, Ziga Rotar Consultant of: Speaker and consulting fees from Abbvie, Amgen, Biogen, Eli Lilly, Medis, MSD, Novartis, Pfizer, Roche, Sanofi., Speakers bureau: Speaker and consulting fees from Abbvie, Amgen, Biogen, Eli Lilly, Medis, MSD, Novartis, Pfizer, Roche, Sanofi., Maria Jose Santos Speakers bureau: Novartis and Pfizer, Tanja Stamm Grant/research support from: AbbVie, Roche, Consultant of: AbbVie, Sanofi Genzyme, Speakers bureau: AbbVie, Roche, Sanofi, Simon Stones Consultant of: I have been a paid consultant for Envision Pharma Group and Parexel. This does not relate to this abstract., Speakers bureau: I have been a paid speaker for Actelion and Janssen. These do not relate to this abstract., Anja Strangfeld Speakers bureau: AbbVie, BMS, Pfizer, Roche, Sanofi-Aventis, Robert B.M. Landewé Consultant of: AbbVie; AstraZeneca; Bristol-Myers Squibb; Eli Lilly & Co.; Galapagos NV; Novartis; Pfizer; UCB Pharma, Axel Finckh Grant/research support from: Pfizer: Unrestricted research grant, Eli-Lilly: Unrestricted research grant, Consultant of: Sanofi, AB2BIO, Abbvie, Pfizer, MSD, Speakers bureau: Sanofi, Pfizer, Roche, Thermo Fisher Scientific

Background:Comparative effectiveness studies using observational data are increasingly used. Despite their high potential for bias, there are no detailed recommendations on how these studies should best be analysed and reported in rheumatology.Objectives:To conduct a systematic literature review of comparative effectiveness research in rheumatology to inform the EULAR task force developing points to consider when analysing and reporting comparative effectiveness research with observational data.Methods:All original articles comparing drug effectiveness in longitudinal observational studies of ≥100 patients published in key rheumatology journals (Scientific Citation Index > 2) between 1.01.2008 and 25.03.2019 available in Ovid MEDLINE® were included. Titles and abstracts were screened by two reviewers for the first 1000 abstracts and independently checked to ensure sufficient agreement has been reached. The main information extracted included the types of outcomes used to assess effectiveness, and the types of analyses performed, focusing particularly on confounding and attrition.Results:9969 abstracts were screened, with 218 articles proceeding to full-text extraction (Figure 1), representing a number of rheumatic and musculoskeletal diseases. Agreement between the two reviewers for the first 1000 abstracts was 92.7% with a kappa of 0.6. The majority of the studies used several outcomes to evaluate effectiveness (Figure 2A). Most of the studies did not explain how they addressed missing data on the covariates (70%) (Figure 2B). When addressed (30%), 44% used complete case analysis and 10% last observation carried forward (LOCF). 25% of studies did not adjust for confounding factors and there was no clear correlation between the number of factors used to adjust and the number of participants in the studies. An important number of studies selected covariates using bivariate screening and/or stepwise selection. 86% of the studies did not acknowledge attrition (Figure 2C). When trying to correct for attrition (14%), 38% used non-responder (NR) imputation, 24% used LUNDEX1, a form of NR imputation, and 21% LOCF.Conclusion:Most of studies used multiple outcomes. However, the vast majority did not acknowledge missing data and attrition, and a quarter did not adjust for any confounding factors. Moreover, when attempting to account for attrition, several studies used methods which potentially increase bias (LOCF, complete case analysis, bivariate screening…). This systematic review confirms the need for the development of recommendations for the assessment and reporting of comparative drug effectiveness in observational data in rheumatology.References:[1]Kristensen et al. A&R. 2006 Feb;54(2):600-6.Acknowledgments:Support of the Standing Committee on Epidemiology and Health Services ResearchDisclosure of Interests:Kim Lauper: None declared, Joanna KEDRA: None declared, Maarten de Wit Grant/research support from: Dr. de Wit reports personal fees from Ely Lilly, 2019, personal fees from Celgene, 2019, personal fees from Pfizer, 2019, personal fees from Janssen-Cilag, 2017, outside the submitted work., Consultant of: Dr. de Wit reports personal fees from Ely Lilly, 2019, personal fees from Celgene, 2019, personal fees from Pfizer, 2019, personal fees from Janssen-Cilag, 2017, outside the submitted work., Speakers bureau: Dr. de Wit reports personal fees from Ely Lilly, 2019, personal fees from Celgene, 2019, personal fees from Pfizer, 2019, personal fees from Janssen-Cilag, 2017, outside the submitted work., Bruno Fautrel Grant/research support from: AbbVie, Lilly, MSD, Pfizer, Consultant of: AbbVie, Biogen, BMS, Boehringer Ingelheim, Celgene, Lilly, Janssen, Medac MSD France, Nordic Pharma, Novartis, Pfizer, Roche, Sanofi Aventis, SOBI and UCB, Thomas Frisell: None declared, Kimme Hyrich Grant/research support from: Pfizer, UCB, BMS, Speakers bureau: Abbvie, Florenzo Iannone Consultant of: Speaker and consulting fees from AbbVie, Eli Lilly, Novartis, Pfizer, Roche, Sanofi, UCB, MSD, Speakers bureau: Speaker and consulting fees from AbbVie, Eli Lilly, Novartis, Pfizer, Roche, Sanofi, UCB, MSD, Pedro M Machado Consultant of: PMM: Abbvie, Celgene, Janssen, Lilly, MSD, Novartis, Pfizer, Roche and UCB, Speakers bureau: PMM: Abbvie, BMS, Lilly, MSD, Novartis, Pfizer, Roche and UCB, Lykke Midtbøll Ørnbjerg Grant/research support from: Novartis, Ziga Rotar Consultant of: Speaker and consulting fees from Abbvie, Amgen, Biogen, Eli Lilly, Medis, MSD, Novartis, Pfizer, Roche, Sanofi., Speakers bureau: Speaker and consulting fees from Abbvie, Amgen, Biogen, Eli Lilly, Medis, MSD, Novartis, Pfizer, Roche, Sanofi., Maria Jose Santos Speakers bureau: Novartis and Pfizer, Tanja Stamm Grant/research support from: AbbVie, Roche, Consultant of: AbbVie, Sanofi Genzyme, Speakers bureau: AbbVie, Roche, Sanofi, Simon Stones Consultant of: I have been a paid consultant for Envision Pharma Group and Parexel. This does not relate to this abstract., Speakers bureau: I have been a paid speaker for Actelion and Janssen. These do not relate to this abstract., Anja Strangfeld Speakers bureau: AbbVie, BMS, Pfizer, Roche, Sanofi-Aventis, Robert B.M. Landewé Consultant of: AbbVie; AstraZeneca; Bristol-Myers Squibb; Eli Lilly & Co.; Galapagos NV; Novartis; Pfizer; UCB Pharma, Axel Finckh Grant/research support from: Pfizer: Unrestricted research grant, Eli-Lilly: Unrestricted research grant, Consultant of: Sanofi, AB2BIO, Abbvie, Pfizer, MSD, Speakers bureau: Sanofi, Pfizer, Roche, Thermo Fisher Scientific, Sytske Anne Bergstra: None declared, Delphine Courvoisier: None declared

Abstract The Society gratefully acknowledges the time and effort of the following individuals who served as reviewers of abstracts for this meeting: ASH ABSTRACTS COORDINATING REVIEWERS Blanche P. Alter Stephen M. Ansell Ralph B. Arlinghaus Scott Armstrong Asad Bashey Philip Bierman Neil Blumberg Chiara Bonini Dominique Bonnet Jacqueline Boultwood Rena Buckstein John C. Byrd Marc Carrier Lucio H. Castilla Selina Chen-Kiang Nicholas Chiorazzi Jorge Cortes-Franco Claire E. Dearden Mary C. Dinauer Harry Paul Erba Carolyn A. Felix Pierre Fenaux Debra L. Friedman Irene M. Ghobrial Jason R. Gotlib Brandon Hayes-Lattin Cheryl A. Hillery Achille Iolascon Jean-Pierre J. Issa Sundar Jagannath Diane F. Jelinek H. Phillip Koeffler John Koreth Robert J. Kreitman Robert B. Levy David Lillicrap Richard Lottenberg John D. McMannis Mark D. Minden Charles G. Mullighan Arnon Nagler Peter J. Newman Robert Z. Orlowski Antonio Palumbo Julie A. Panepinto Warren S. Pear Sibrand Poppema Barbara Pro Ching-Hon Pui A. Koneti Rao Aaron P. Rapoport Pieter H. Reitsma Douglas D. Ross J. Eric Russell Barbara Savoldo Kirk R. Schultz Radek C. Skoda Marilyn L. Slovak Susan Smyth Hugo ten Cate Herve Tilly John M. Timmerman Ivo Touw Amy J. Wagers Russell E. Ware Catherine J. Wu Virginia M. Zaleskas ASH ABSTRACTS REVIEWERS Camille Abboud Omar Abdel-Wahab Jeremy Abramson Suneet Agarwal Sikander Ailawadhi Onder Alpdogan Andrew Aprikyan Mary Armanios Aneel Ashrani Norio Asou Aglaia Athanassiadou Eyal Attar Mohammad Azam Maria Baer Jorg Baesecke Sarah Ball Karen Ballen Frederic Baron Shannon Bates Minoo Battiwalla Marie Bene Charles Bennett James Berenson Steven Bernstein Francesco Bertoni Monica Bessler Wolfgang Bethge Kapil Bhalla Deepa Bhojwani James Bieker Bruce R. Blazar Annemarie Block David Bodine Catherine Bollard Antonio Bonati Eric Bouhassira Benjamin Braun Christopher Bredeson Patrick Brown Ross Brown Jan Burger Dario Campana Jose Cancelas Paul Carpenter Andrew Carroll James Casella Rebecca Chan Roy Chemaly Benny Chen Jerry Cheng Linzhao Cheng Bruce Cheson Mark Chiang Athar Chishti Hearn Cho Magdalena Chrzanowska-Wodnicka Richard E. Clark Joseph Connors Kenneth Cooke Miguel Cruz Adam Cuker Sandeep Dave Janice Davis Sproul Lucia De Franceschi Philip De Groot Rodney DeKoter Richard Delarue Stephen Devereux Steven Devine Paola Jorge Di Don Diamond Meletios Dimopoulos John DiPersio Angela Dispenzieri Benjamin Djulbegovic Jing-fei Dong James Downing William Drobyski Rafael Duarte Charles Dumontet Kieron Dunleavy Brian Durie Dimitar Efremov Elizabeth Eklund Jonas Emsley Patricia Ernst Andrew Evens Chris Fegan Andrew Feldman Giuliana Ferrari Willem Fibbe Adele Fielding Thoas Fioretos Robert Flaumenhaft Rafael Fonseca James Foran Joseph Frank Janet Franklin Paul Frenette Alan Friedman Terry Fry Saghi Gaffari Naomi Galili Patrick Gallagher Anne Galy David Garcia Randy Gascoyne Cristina Gasparetto Norbert Gattermann Tobias Gedde-Dahl Alan Gewirtz Francis Giles Robert Godal Lucy Godley Ivana Gojo Norbert Gorin Andre Goy Eric Grabowski Steven Grant Timothy Graubert Elizabeth Griffiths H. Leighton Grimes Claudia Haferlach Corinne Haioun Parameswaran Hari Christine Harrison Robert Hasserjian Nyla Heerema Shelly Heimfeld Roland Herzog Elizabeth Hexner Teru Hideshima William H. Hildebrand Gerhard Hildebrandt Devendra Hiwase Karin Hoffmeister Donna Hogge Scott Howard Brian Huntly Hiroto Inaba Baba Inusa Shai Izraeli Suresh Jhanwar Amy Johnson Craig Jordan Joseph Jurcic Nina Kadan-Lottick Lawrence Kaplan Jonathan Kaufman Neil Kay Michelle Kelliher Craig Kessler H. 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Meijerink Junia Melo Thomas Mercher Bradley Messmer Marco Mielcarek Ken Mills Shin Mineishi Arturo Molina Silvia Montoto Marie Joelle Mozziconacci Auayporn Nademanee Vesna Najfeld Eneida Nemecek Ellis Neufeld Peter Newburger Heyu Ni Charlotte Marie Niemeyer Yago Nieto Anne Novak Paul O\'Donnell Vivian Oehler Fritz Offner Johannes Oldenburg Rebecca Olin Richard J. 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Wijermans John Wingard Anne Woolfrey Mingjiang Xu Qing Yi Anas Younes Ryan Zarychanski Arthur Zelent Clive Zent Dong-Er Zhang Xianzheng Zhou James Zimring