@article{9d0ac2953f99444c8afa3ab25877101b,
title = "Development of a distributed international patient data registry for hairy cell leukemia",
abstract = "Hairy cell leukemia (HCL) is a rare lymphoproliferative disorder, comprising only 2% of all leukemias. The Hairy Cell Leukemia Foundation (HCLF) has developed a patient data registry to enable investigators to better study the clinical features, treatment outcomes, and complications of patients with HCL. This system utilizes a centralized registry architecture. Patients are enrolled at HCL Centers of Excellence (COE) or via a web-based portal. All data are de-identified, which reduces regulatory burden and increases opportunities for data access and re-use. To date, 579 patients have been enrolled in the registry. Efforts are underway to engage additional COE{\textquoteright}s to expand access to patients across the globe. This international PDR will enable researchers to study outcomes in HCL in ways not previously possible due to the rarity of the disease and will serve as a platform for future prospective research.",
keywords = "HCL, outcomes, registry",
author = "Andritsos, {Leslie A.} and Mirela Anghelina and Jasmine Neal and Blachly, {James S.} and Puneet Mathur and Omkar Lele and Claire Dearden and Sunil Iyengar and Matthew Cross and Zent, {Clive S.} and Rogers, {Kerry A.} and Narendranath Epperla and Gerard Lozanski and Oakes, {Christopher C.} and Eric Kraut and Ruppert, {Amy S.} and Qiuhong Zhao and Bhat, {Seema A.} and Francesco Forconi and Versha Banerji and Sasanka Handunnetti and Tam, {Constantine S.} and Seymour, {John F.} and Monica Else and Kreitman, {Robert J.} and Alan Saven and Timothy Call and Parikh, {Sameer A.} and Farhad Ravandi and Johnston, {James B.} and Enrico Tiacci and Xavier Troussard and Tallman, {Martin S.} and Sascha Dietrich and Tamar Tadmor and Alessandro Gozzetti and Zinzani, {Pier Luigi} and Tadeusz Robak and Graeme Quest and Judit Demeter and Kanti Rai and Fernandez, {Soledad A.} and Michael Grever",
note = "Funding Information: The Ohio State University (OSU) and Signet Accel., LLC (SA) had a financial conflict of interest in that SA provided technical support and maintained the database infrastructure for the Hairy Cell Leukemia Patient Data Registry, a project initiated and developed at OSU. OSU had a financial interest in SA as part of a licensing agreement. Dr. Phillip Payne had a financial interest in SA. SA is no longer providing database support, and these functions are now accessed through the Department of Research Information Technology at The Ohio State University. LA previously provided consultation services for Innate Pharma and AstraZeneca. The other authors declare no competing interests. MA has consulted for AstraZeneca in 2019; received travel funding from Hairy Cell Leukemia Foundation. JSB was on Advisory Board and has received consulting fees from: AbbVie, AstraZeneca, INNATE Pharma, KITE Pharma; has received research funding from MingSight Pharmaceuticals; has patents and intellectual property on a leukemia diagnostic device (patent pending). OL is currently employed by Bristol-Myers Squibb Company. His contribution to the work described in this manuscript was during his employment with The Ohio State University which ended as of June 2018. CD has been an advisor for Medimmune/Innate Pharma (moxetumomab); consulting/Advisory Board for Abbie and Jansen. SI: Advisory and speaker fees: Gilead and Takeda; Advisory: Beigene; Speaker fees: Janssen, Takeda and Gilead. CSZ: funding through the University of Rochester for laboratory research, from Acerta/AstraZeneca and TG Therapeutics, and from the Hairy Cell Leukemia Foundation. KAR receives research funding from Genentech, AbbVie, Novartis, and Janssen, has consulted for Acerta Pharma, Genentech, AbbVie, Pharmacyclics, Innate Pharma, and AstraZeneca, and received travel funding from AstraZeneca; NE is on the Speakers Bureau: Incyte; Ad Board: TG therapeutics, Beigene, Pharmacyclics; Consultancy: Novartis; GL has received travel funding from Hairy Cell Leukemia Foundation. ASR is currently employed by Eli Lily and Company, though contributions to the work described in this manuscript occurred during employment with The Ohio State University which ended as of January 2022; served on an independent DSMB for Telios Pharma; SAB served on Advisory board for Pharmacyclics and Janssen, Beigene and AstraZeneca; received honorarium from OncLive; received travel grant from Arqule; VB serves on the Advisory Boards of Janssen, AstraZeneca, and Abbvie and has had research funding from CIHR, LLSC, CCMF, Roche, Janssen, Abbvie, and Lundbeck. VB also received fees from BIOGEN for patented compounds unrelated to this study; SH: Abbvie (Travel Grant), Novartis (Travel Grant), Roche (Honoraria), Gilead (Honoraria); CST: Honoraria from Janssen, AbbVie, and Beigene, and his hospital receives research funding from Janssen, AbbVie, and Beigene; JFS: AbbVie, Advisory board, speakers{\textquoteright} bureau, research funding; Astra Zeneca, Advisory board; Celgene, Advisory board, speakers{\textquoteright} bureau, research funding, expert testimony; Genentech, Advisory board; Gilead, Advisory Board; Janssen, Advisory board, research funding; Mei Pharma, Advisory board; Morphosys, Advisory board; Roche, Advisory board, speakers{\textquoteright} bureau, research funding, expert testimony; Sunesis, Advisory board; Takeda, Advisory boardME; RJK: Employment: National Institutes of Health and Regional Cancer Care Associates; Honoraria: PlatformQ, OncLive, Cure; Research Funding: Innate, AstraZeneca, Novartis, Genetech, Pfizer, Teva, Hairy Cell Leukemia Foundation; Patents: Coinventor for NIH patent for Moxetumomab Pasudotox; AS: Consultant and Advisory Boards: AstraZeneca and Innate Pharmaceuticals; Speakers Bureau: AbbVie and Pharmacyclics; SAP: Research funding has been provided to the institution from Pharmacyclics, Janssen, AstraZeneca, TG Therapeutics, Merck, AbbVie, and Ascentage Pharma for clinical studies in which Sameer A. Parikh is a principal investigator. SAP has also participated in Advisory Board meetings of Pharmacyclics, AstraZeneca, Genentech, GlaxoSmithKline, Innate Pharma, Adaptive Biotechnologies, and AbbVie (he was not personally compensated for his participation); FR: Consultancy and Honoraria – Celgene, BMS, Amgen, Astellas, Xencor, Agios, AstraZeneca, Orsenix, Innate Pharma, Syros, Taiho, Novartis; Research Funding – BMS, Amgen, Xencor, Macrogenics, Orsenix, Abbvie, Taiho, Prelude, Astex; ET: Consultant for Innate Pharma. Research funding: Roche. Travel cost: Shire. Holder of a patent on the use of mutant BRAF as HCL biomarker. Our research work in HCL is funded by the Hairy Cell Leukemia Foundation, the Leukemia and Lymphoma Society and the Associazione Italiana Ricerca sul Cancro (AIRC); XT: Consultant for Innate Pharma, AstraZeneca; Advisor: Abbvie; MST: Research funding: AbbVie, Amgen, Biosight, Glycomimetics, Orsenix, Rafael; Advisory boards: Amgen, Daiichi-Sankyo, Delta Fly Pharma, Innate pharmaceuticals, Jazz, Kahr, Kura, Novartis, Orsenix, Roche, Syros; Royalties: UpToDate; AG: Advisory board: Amgen, Takeda. Research funding: Jansenn and Cilag SPA. Honoraria: Abbvie, Jansenn Cilag, Celgene, Amgen, Takeda; PLZ: Speakers Bureau: Verastem, Celltrion, Gilead, Janssen-Cilag, BMS, Servier, MSD, TG Therap., Takeda, Roche, Eusapharma, Kyowa Kirin; Advisory Board: Verastem, Celltrion, Gilead, Janssen-Cilag, BMS, Servier, Sandoz, MSD, TG Therap., Takeda, Roche, Eusapharma, Kyowa Kirin, ADC Therap.; Consultant: Verastem, MSD, Eusapharma, Sanofi; TR: Research funding: AstraZeneca, Medimmune, Roche, Janssen, Abbvie, Advisory board; AstraZeneca, Jassen, Abbvie; Travel grant: Roche, Janssen; Honoraria: Abbvie, AstraZeneca, Janssen; TT: Consultant Abbvie, BMS, Roche, Janssen, Novartis, Pfizer, Takeda, Astra Zeneca, Gilead; JD: Has participated in advisory committees in Hungary for Novartis, Bristol Myers Squibb, Amicus, Angelini, Pfizer, Amgen, and Roche; MG has been consultant for AstraZeneca, Pharmacyclics, Ascerta, Axio, Inc.; EMD Serono (Merck) Research and Development Institute; has received research funding and travel expenses from Hairy Cell Leukemia Foundation; Scientific Board Chair: Hairy Cell Leukemia Foundation Scientific Board (no reimbursement); Scientific Honorarium: University of Pittsburgh. The other authors declare no competing interests. Funding Information: Research reported in this publication was supported by Hairy Cell Leukemia Foundation. OSU services utilized include the College of Medicine Research Information Technology (National Center For Advancing Translational Sciences Grant: UL1TR002733, OSU Comprehensive Cancer Center Support Grant: P30CA016058, and National Center for Advancing Translational Sciences, Grant UL1TR002733). We are grateful to Joshua W. Sadvari from OSU University Libraries, for his help with mapping the registry patients, as well as to the following developers whose contribution has been instrumental in making the registry software platform: Wei-Chao Chuang; John Strickler; John Thompson. We also acknowledge Philip Payne for his contribution to the initial design of the federated registry platform. Publisher Copyright: {\textcopyright} 2022 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group.",
year = "2022",
doi = "10.1080/10428194.2022.2109157",
language = "English (US)",
volume = "63",
pages = "3021--3031",
journal = "Leukemia and Lymphoma",
issn = "1042-8194",
publisher = "Informa Healthcare",
number = "13",
}
