Review
Cardiac & Cardiovascular Systems
Ruth Masterson Creber, Cristiano Spadaccio, Arnaldo Dimagli, Annie Myers, Brittany Taylor, Stephen Fremes
Summary: Patient-reported outcomes (PROs) offer a global perspective of a person's health status and are categorized into global, mental, physical, and social health domains. Challenges in using PROs in cardiac clinical trials include long-term measurement, inconsistency in outcome measures, and lack of measurement across multiple domains. Opportunities for advancement include utilizing consumer health informatics tools to measure PROs and quantifying minimal clinically important differences.
CANADIAN JOURNAL OF CARDIOLOGY
(2021)
Editorial Material
Biochemistry & Molecular Biology
Ameeta Retzer, Olalekan Lee Aiyegbusi, Anna Rowe, Philip N. Newsome, Jessica Douglas-Pugh, Sheeba Khan, Saloni Mittal, Roger Wilson, Daniel O'Connor, Lisa Campbell, Sandra A. Mitchell, Melanie Calvert
Summary: Patient-reported outcomes can offer preliminary evidence of efficacy and tolerability from the patient's perspective, and they can also support regulatory review.
Article
Rheumatology
Vibeke Strand, Namita Tundia, Alvin Wells, Maya H. Buch, Sebastiao C. Radominski, Heidi S. Camp, Alan Friedman, Jessica L. Suboticki, Kendall Dunlap, Debbie Goldschmidt, Martin Bergman
Summary: The study evaluated the impact of UPA monotherapy compared to MTX on patient-reported outcomes in RA patients who were MTX-naive or had an inadequate response to MTX. The results showed that UPA monotherapy at 15 or 30mg for 12/14 weeks resulted in statistically significant and clinically meaningful improvements in pain, physical function, morning stiffness, HRQOL, and WPAI compared to MTX alone.
Article
Dermatology
Alexander A. Navarini, Joerg C. Prinz, Akimichi Morita, Tsen-Fang Tsai, Manuelle-Anne Viguier, Ling Li, Christian Thoma, Mogana Sivalingam, Mark G. Lebwohl
Summary: In the Effisayil (TM) 1 study, GPP patients treated with spesolimab showed significant improvements in patient-reported outcomes (PROs) and achieved the minimal clinically important differences (MCIDs) as early as 1 week, with sustained improvements over 12 weeks. Patients in the placebo arm also experienced improvements in PROs and achieved MCIDs after receiving open-label spesolimab at Week 1.
JOURNAL OF THE EUROPEAN ACADEMY OF DERMATOLOGY AND VENEREOLOGY
(2023)
Editorial Material
Oncology
Eleonora Ghisoni, Matteo Morotti, Sara Colomer-Lahiguera, Manuela Eicher, George Coukos, Lionel Trueb, Massimo Di Maio
Summary: Adoptive cell therapies show promise in treating cancer, yet little is known about patients' experiences. Patient-reported outcomes (PROs) and patient-reported outcome measures (PROMs) are tools increasingly used in oncology, but their use in adoptive cell therapies remains limited.
JOURNAL FOR IMMUNOTHERAPY OF CANCER
(2022)
Article
Rheumatology
Vibeke Strand, Namita Tundia, Martin Bergman, Andrew Ostor, Patrick Durez, In-Ho Song, Jeffrey Enejosa, Casey Schlacher, Yan Song, Roy Fleischmann
Summary: In MTX-IR patients with RA, treatment with upadacitinib resulted in statistically significant and clinically meaningful improvements in PROs equivalent to or greater than with adalimumab.
Article
Oncology
Mallorie H. Fiero, Jessica K. Roydhouse, Vishal Bhatnagar, Ting-Yu Chen, Bellinda L. King-Kallimanis, Shenghui Tang, Paul G. Kluetz
Summary: Time-to-event endpoints for patient-reported outcomes, such as time to deterioration of symptoms or function, have unique considerations and challenges in cancer clinical trials. Analytical decisions using the estimand framework can help answer specific clinical questions, while the complexity and burdens associated with measuring time to deterioration make it problematic for widespread use. Instead, analyzing at pre-specified relevant time-points might be a better approach for trials focusing on comparative symptom or functional benefits.
Review
Medicine, General & Internal
Antonia Morga, Silvia Dibenedetto, Rocco Adiutori, Jun Su
Summary: This targeted literature review aimed to determine if PRO instruments psychometrically validated in a phase 3 trial setting could support label claims from the same phase 3 study. The results suggest that quantitative validation of novel PRO instruments, and existing PROs for new indications, can occur within the context of phase 3 trials, and these PROs can also support label claims.
CURRENT MEDICAL RESEARCH AND OPINION
(2023)
Editorial Material
Oncology
Cristiane Decat Bergerot, Sumanta K. Pal, Abhishek Tripathi
Summary: This commentary discusses the potential relevance and barriers to including patient-reported outcomes in early phase trials.
Article
Health Care Sciences & Services
Jinxiang Hu, Jeffrey Thompson, Dinesh Pal Mudaranthakam, Lynn Chollet Hinton, David Streeter, Michele Park, Berend Terluin, Byron Gajewski
Summary: This study aimed to estimate power in a two-arm clinical trial with PROMIS measures as endpoints using IRT model. Results indicated that sample size, effect size, and number of items are important indicators for IRT based power estimation. When effect size is small and sample size is limited, IRT model provides higher power.
JOURNAL OF CLINICAL EPIDEMIOLOGY
(2022)
Review
Medicine, General & Internal
Alexander Fabian, Justus Domschikowski, Anne Letsch, Claudia Schmalz, Sandra Freitag-Wolf, Juergen Dunst, David Krug
Summary: This systematic review examined the use and reporting of patient-reported outcomes (PROs) in clinical trials of palliative radiotherapy. Among the 225 eligible trials, 20% used a PRO as a primary endpoint and 31% used a PRO as a secondary endpoint. The most common PRO measures were the Numeric Rating Scale/Visual Analogue Scale, European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire C30, and trial-specific unvalidated measures. Adherence to CONSORT-PRO reporting guidelines was generally poor or moderate. The study suggests the need for improved use and reporting of PROs in future trials, particularly as primary endpoints.
Article
Rheumatology
Donald Stull, Sean O'Quinn, Betsy Williams, Stephanie Bean, Erik Schwetje, Gabriel Abreu, Raj Tummala
Summary: Anifrolumab treatment improves disease activity and health-related quality of life in SLE patients by reducing pain and enhancing mood and physical function.
Article
Endocrinology & Metabolism
Suzanne M. Jan de Beur, Tricia Cimms, Annabel Nixon, Christina Theodore-Oklota, Diana Luca, Mary Scott Roberts, Shayna Egan, Christina A. Graham, Elizabeth Hribal, Christopher J. Evans, Sue Wood, Angela Williams
Summary: This study examines the patients' perception of treatment effects in tumor-induced osteomalacia (TIO). It finds that improvements in pain and fatigue are the most important aspects for patients in treatment. Additionally, the study shows meaningful improvements in health-related quality of life and physical function after treatment.
JOURNAL OF BONE AND MINERAL RESEARCH
(2023)
Article
Medical Laboratory Technology
Marta Jimenez-Barragan, Manuel Rodriguez-Oliva, Catalina Sanchez-Mora, Carmen Navarro-Bustos, Sandra Fuentes-Cantero, Salomon Martin-Perez, Jose M. Garrido-Castilla, Luisa Undabeytia-Lopez, Antonio Luque-Cid, Juan de Miguel-Melendez, Antonio Leon-Justel
Summary: The study showed that a new patient flow based on Point-of-Care Testing (POCT) can significantly reduce Emergency Department length of stay, time to disposition decision, and laboratory turnaround time. This strategy did not lead to an increase in readmission rates, indicating it as a promising approach to optimize patient flow in the Emergency Department.
CLINICA CHIMICA ACTA
(2021)
Article
Oncology
Andrew Rosowicz, Venu G. Bangla, James W. Johnson, Laura Olson, Jenny Chen, Celia M. Divino
Summary: In this study, the effect of surgical resection on quality of life in patients with intestinal and pancreatic neuroendocrine tumors (NETs) was examined. The results showed significant improvements in both physical and mental health after surgery, as well as alleviation of specific carcinoid syndrome symptoms.
ANNALS OF SURGICAL ONCOLOGY
(2023)
Article
Gastroenterology & Hepatology
Rohit Loomba, Eric Lawitz, Parvez S. Mantry, Saumya Jayakumar, Stephen H. Caldwell, Hays Arnold, Anna Mae Diehl, C. Stephen Djedjos, Ling Han, Robert P. Myers, G. Mani Subramanian, John G. McHutchison, Zachary D. Goodman, Nezam H. Afdhal, Michael R. Charlton
Article
Gastroenterology & Hepatology
S. L. Flamm, B. Bacon, M. P. Curry, S. Milligan, C. U. Nwankwo, N. Tsai, Z. Younossi, N. Afdhal
ALIMENTARY PHARMACOLOGY & THERAPEUTICS
(2018)
Article
Microbiology
Darius Babusis, Michael P. Curry, Brian Kirby, Yeojin Park, Eisuke Murakami, Ting Wang, Anita Mathias, Nezam Afdhal, John G. McHutchison, Adrian S. Ray
ANTIMICROBIAL AGENTS AND CHEMOTHERAPY
(2018)
Letter
Gastroenterology & Hepatology
Z. Gordon Jiang, Bynvant Sandhu, Linda Feldbruegge, Eric U. Yee, Eva Csizmadia, Shuji Mitsuhashi, Jinhe Huang, Nezam H. Afdhal, Simon C. Robson, Michelle Lai
CLINICAL GASTROENTEROLOGY AND HEPATOLOGY
(2018)
Article
Gastroenterology & Hepatology
Omar El-Sherif, Z. Gordon Jiang, Elliot B. Tapper, K. C. Huang, Alex Zhong, Anu Osinusi, Michael Charlton, Michael Manns, Nezam H. Afdhal, Kenneth Mukamal, John McHutchison, Diana M. Brainard, Norah Terrault, Michael P. Curry
Article
Gastroenterology & Hepatology
Stephen A. Harrison, Manal F. Abdelmalek, Stephen Caldwell, Mitchell L. Shiffman, Anna Mae Diehl, Reem Ghalib, Eric J. Lawitz, Don C. Rockey, Raul Aguilar Schall, Catherine Jia, Bryan J. McColgan, John G. McHutchison, G. Mani Subramanian, Robert P. Myers, Zobair Younossi, Vlad Ratziu, Andrew J. Muir, Nezam H. Afdhal, Zachary Goodman, Jaime Bosch, Arun J. Sanyal
Article
Mathematical & Computational Biology
Joy Leahy, Aisling O'Leary, Nezam Afdhal, Emma Gray, Scott Milligan, Malte H. Wehmeyer, Cathal Walsh
RESEARCH SYNTHESIS METHODS
(2018)
Article
Immunology
Jason Grebely, Jordan J. Feld, David Wyles, Mark Sulkowski, Liyun Ni, Joe Llewellyn, Heshaam M. Mir, Nika Sajed, Luisa M. Stamm, Robert H. Hyland, John McNally, Diana M. Brainard, Ira Jacobson, Stefan Zeuzem, Marc Bourliere, Graham Foster, Nezam Afdhal, Gregory J. Dore
OPEN FORUM INFECTIOUS DISEASES
(2018)
Article
Gastroenterology & Hepatology
Andrew J. Muir, Cynthia Levy, Harry L. A. Janssen, Aldo J. Montano-Loza, Mitchell L. Shiffman, Stephen Caldwell, Velimir Luketic, Dora Ding, Catherine Jia, Bryan J. McColgan, John G. McHutchison, G. Mani Subramanian, Robert P. Myers, Michael Manns, Roger Chapman, Nezam H. Afdhal, Zachary Goodman, Bertus Eksteen, Christopher L. Bowlus
Article
Gastroenterology & Hepatology
Zobair Younossi, Maria Stepanova, Arun J. Sanyal, Stephen A. Harrison, Vlad Ratziu, Manal F. Abdelmalek, Anna Mae Diehl, Stephen Caldwell, Mitchell L. Shiffman, Raul Aguilar Schall, Bryan McColgan, G. Mani Subramanian, Robert P. Myers, Andrew Muir, Nezam H. Afdhal, Jaime Bosch, Zachary Goodman
JOURNAL OF HEPATOLOGY
(2018)
Article
Gastroenterology & Hepatology
Saumya Jayakumar, Michael S. Middleton, Eric J. Lawitz, Parvez S. Mantry, Stephen H. Caldwell, Hays Arnold, Anna Mae Diehl, Reem Ghalib, Magdy Elkhashab, Manal F. Abdelmalek, Kris V. Kowdley, C. Stephen Djedjos, Ren Xu, Ling Han, G. Mani Subramanian, Robert P. Myers, Zachary D. Goodman, Nezam H. Afdhal, Michael R. Charlton, Claude B. Sirlin, Rohit Loomba
JOURNAL OF HEPATOLOGY
(2019)
Article
Medicine, Research & Experimental
Rohit Loomba, Yevgeniy Gindin, Zhaoshi Jiang, Eric Lawitz, Stephen Caldwell, C. Stephen Djedjos, Ren Xu, Chuhan Chung, Robert P. Myers, G. Mani Subramanian, Zachary Goodman, Michael Charlton, Nezam H. Afdhal, Anna Mae Diehl
Article
Gastroenterology & Hepatology
Sentia Iriana, Michael P. Curry, Nezam H. Afdhal
CLINICS IN LIVER DISEASE
(2017)
Article
Gastroenterology & Hepatology
Ira M. Jacobson, Mary K. Washington, Maria Buti, Alexander Thompson, Nezam Afdhal, Robert Flisiak, Ulus Salih Akarca, Konstantin G. Tchernev, John F. Flaherty, Raul Aguilar Schall, Robert P. Myers, G. Mani Subramanian, John G. McHutchison, Zobair Younossi, Patrick Marcellin, Keyur Patel
CLINICAL GASTROENTEROLOGY AND HEPATOLOGY
(2017)
Article
Gastroenterology & Hepatology
Christopher J. Danford, Margery A. Connelly, Irina Shalaurova, Misung Kim, Mark A. Herman, Imad Nasser, James D. Otvos, Nezam H. Afdhal, Z. Gordon Jiang, Michelle Lai
HEPATOLOGY COMMUNICATIONS
(2018)