Elise Hoover1, Ronald D Perrone2, Chris Rusconi1, Beverly Benson1, Neera K Dahl3, Berenice Gitomer4, Amy Manelli1, Michal Mrug5, Meyeon Park6, Stephen L Seliger7, Milind A Phadnis8, Nadeesha Thewarapperuma8, Terry J Watnick7. 1. PKD Foundation, Kansas City, Missouri. 2. Department of Medicine, Division of Nephrology, Tufts Medical Center, Boston, Massachusetts. 3. Section of Nephrology, Yale University School of Medicine, New Haven, Connecticut. 4. Division of Renal Diseases and Hypertension, University of Colorado Anschutz Medical Campus, Aurora, Colorado. 5. Division of Nephrology, Department of Veterans Affairs Medical Center and University of Alabama at Birmingham, Birmingham, Alabama. 6. Division of Nephrology, Department of Medicine, University of California San Francisco, San Francisco, California. 7. Department of Medicine, Division of Nephrology, University of Maryland School of Medicine, Baltimore, Maryland. 8. Department of Biostatistics. University of Kansas Medical Center, Kansas City, Kansas.
Abstract
Background: Autosomal dominant polycystic kidney disease (ADPKD) is the most common form of inherited kidney disease worldwide. Over the past five years, the therapeutic pipeline for ADPKD has expanded, leading to a growing need for patient enrollment in clinical trials and improved understanding of patient-centered outcomes that can be used in trial design. To advance these goals, the Polycystic Kidney Disease Foundation (PKDF) established a national web-based ADPKD Registry. Methods: The ADPKD Registry is hosted on a secure, HIPAA-compliant, online platform (IQVIA, oc-meridian.com/pkdcure). Participants are consented through the online system and complete a series of modules. The Core Questionnaire includes patient-reported diagnosis, latest creatinine values, and comorbidities. Additional modules include surveys of family history, diet, quality of life, extrarenal manifestations, and attitudes surrounding research participation. Results: As of October 2021, 1563 ADPKD patients across the United States have registered and completed the Core Questionnaire. Participants have a median age of 44 years and are 72% women, 93% White, with 4% self-identifying as Hispanic/Latino and 2% as Black. All CKD stages are present, including post kidney transplant. To date, seven clinical studies have used the Registry as a recruitment tool. Additionally, quality-of-life burden scores revealed a correlation with disease stage as determined by kidney function. Conclusions: The Registry described here is the only one of its kind and is a valuable longitudinal research tool encompassing all stages of ADPKD. The registry will allow investigators to pursue a range of research questions related to the management of ADPKD, including definition of health-related quality of life (HRQoL) outcomes and recruitment for a variety of observational and therapeutic clinical protocols.
Background: Autosomal dominant polycystic kidney disease (ADPKD) is the most common form of inherited kidney disease worldwide. Over the past five years, the therapeutic pipeline for ADPKD has expanded, leading to a growing need for patient enrollment in clinical trials and improved understanding of patient-centered outcomes that can be used in trial design. To advance these goals, the Polycystic Kidney Disease Foundation (PKDF) established a national web-based ADPKD Registry. Methods: The ADPKD Registry is hosted on a secure, HIPAA-compliant, online platform (IQVIA, oc-meridian.com/pkdcure). Participants are consented through the online system and complete a series of modules. The Core Questionnaire includes patient-reported diagnosis, latest creatinine values, and comorbidities. Additional modules include surveys of family history, diet, quality of life, extrarenal manifestations, and attitudes surrounding research participation. Results: As of October 2021, 1563 ADPKD patients across the United States have registered and completed the Core Questionnaire. Participants have a median age of 44 years and are 72% women, 93% White, with 4% self-identifying as Hispanic/Latino and 2% as Black. All CKD stages are present, including post kidney transplant. To date, seven clinical studies have used the Registry as a recruitment tool. Additionally, quality-of-life burden scores revealed a correlation with disease stage as determined by kidney function. Conclusions: The Registry described here is the only one of its kind and is a valuable longitudinal research tool encompassing all stages of ADPKD. The registry will allow investigators to pursue a range of research questions related to the management of ADPKD, including definition of health-related quality of life (HRQoL) outcomes and recruitment for a variety of observational and therapeutic clinical protocols.
Authors: Dorothee Oberdhan; Jason C Cole; Holly B Krasa; Rebecca Cheng; Frank S Czerwiec; Ron D Hays; Arlene B Chapman; Ronald D Perrone Journal: Am J Kidney Dis Date: 2017-11-14 Impact factor: 8.860
Authors: Dana C Miskulin; Kaleab Z Abebe; Arlene B Chapman; Ronald D Perrone; Theodore I Steinman; Vicente E Torres; K Ty Bae; William Braun; Franz T Winklhofer; Marie C Hogan; Fred Rahbari-Oskoui; Charity G Moore; Michael F Flessner; Robert W Schrier Journal: Am J Kidney Dis Date: 2013-11-01 Impact factor: 8.860
Authors: Stephen L Seliger; Terry Watnick; Andrew D Althouse; Ronald D Perrone; Kaleab Z Abebe; Kenneth R Hallows; Dana C Miskulin; Kyongtae T Bae Journal: Kidney360 Date: 2020-12-31
Authors: Daniel Eriksson; Linda Karlsson; Oskar Eklund; Hans Dieperink; Eero Honkanen; Jan Melin; Kristian Selvig; Johan Lundberg Journal: Nephrol Dial Transplant Date: 2017-12-01 Impact factor: 5.992
Authors: Marijke C Jansen-van der Weide; Charlotte M W Gaasterland; Kit C B Roes; Caridad Pontes; Roser Vives; Arantxa Sancho; Stavros Nikolakopoulos; Eric Vermeulen; Johanna H van der Lee Journal: Orphanet J Rare Dis Date: 2018-09-05 Impact factor: 4.123