Articles

Value of transfusion independence in severe aplastic anemia from patients’ perspectives – a discrete choice experiment

Aplastic anemia is a rare, serious blood disorder due to bone marrow failure to produce blood cells. Transfusions are used to reduce risk of bleeding, infection and relieve anemia symptoms. In severe patients,...

Authors: A. Simon Pickard, Lynn Huynh, Jasmina I. Ivanova, Todor Totev, Sophia Graham, Axel C. Mühlbacher, Anuja Roy and Mei Sheng Duh

Qualitative methods in the development of a parent survey of children’s oral health status

Parents’ perceptions of their 8–17-year-old children’s oral health status were assessed using a sample from diverse dental clinics in Greater Los Angeles County to identify constructs for a survey instrument.

Authors: Carl A. Maida, Marvin Marcus, Ron D. Hays, Ian D. Coulter, Francisco Ramos-Gomez, Steve Y. Lee, Patricia S. McClory, Laura V. Van, Yan Wang, Jie Shen, Bryant Lau, Vladimir W. Spolsky, James J. Crall and Honghu Liu

Patient-Reported Outcome (PRO) Consortium translation process: consensus development of updated best practices

This paper describes the rationale and goals of the Patient-Reported Outcome (PRO) Consortium’s instrument translation process. The PRO Consortium has developed a number of novel PRO measures which are in the ...

Authors: Sonya Eremenco, Sheryl Pease, Sarah Mann and Pamela Berry

Patient experience in systemic lupus erythematosus: development of novel patient-reported symptom and patient-reported impact measures

Comprehensive assessment of systemic lupus erythematosus (SLE) and its treatment requires patient-reported outcome (PRO) measures to capture impacts and fluctuating symptoms. The objective of this study was to...

Authors: S. D. Mathias, P. Berry, J. De Vries, K. Pascoe, H. H. Colwell, D. J. Chang and A. D. Askanase

Psychometric evaluation of a caregiver diary for the assessment of symptoms of respiratory syncytial virus

There are no clinical outcome assessment (COA) tools developed in accordance with Food and Drug Administration (FDA) guidance suitable for the evaluation of symptoms associated with respiratory syncytial virus...

Authors: Valerie Williams, Carla DeMuro, Sandy Lewis, Nicole Williams, Todd Wolynn, Paul Wisman, Stan L. Block, Shelly Senders, Seth Toback and Jason W. Chien

Development of a novel observer-reported outcome measure for the assessment of Respiratory Syncytial Virus (RSV) infection symptoms in pediatric clinical trials

Respiratory syncytial virus (RSV) is a seasonal infection affecting most children by 2 years of age and the leading cause of lower respiratory tract infection requiring hospitalization in infants. Novel antivi...

Authors: Sandy Lewis, Carla DeMuro, Stan L. Block, Shelly Senders, Paul Wisman, Seth Toback, Jason W. Chien and Valerie Williams

Emerging good practices for Translatability Assessment (TA) of Patient-Reported Outcome (PRO) measures

This paper presents emerging Good Practices for Translatability Assessment (TA) of Patient-Reported Outcome (PRO) Measures. The ISOQOL Translation and Cultural Adaptation Special Interest Group (TCA-SIG) under...

Authors: Catherine Acquadro, Donald L. Patrick, Sonya Eremenco, Mona L. Martin, Dagmara Kuliś, Helena Correia and Katrin Conway

Development and psychometric validation of the Nausea/Vomiting Symptom Assessment patient-reported outcome (PRO) instrument for adults with secondary hyperparathyroidism

We developed the Nausea/Vomiting Symptom Assessment (NVSA^©) patient-reported outcome (PRO) instrument to capture patients’ experience with nausea and vomiting while on calcimimetic therapy to treat secondary hype...

Authors: Colleen A. McHorney, Mark E. Bensink, Laurie B. Burke, Vasily Belozeroff and Chad Gwaltney

Design and development of the MacTSQ measure of satisfaction with treatment for macular conditions used within the IVAN trial

The purpose of the study was to design a measure of patient satisfaction with treatment for macular disease, the MacTSQ, and to carry out psychometric evaluation of the measure. The measure was designed along ...

Authors: Jan Mitchell and Clare Bradley

The Swedish RAND-36 Health Survey - reliability and responsiveness assessed in patient populations using Svensson’s method for paired ordinal data

The Short Form 36-Item Survey is one of the most commonly used instruments for assessing health-related quality of life. Two identical versions of the original instrument are currently available: the public do...

Authors: Lotti Orwelius, Mats Nilsson, Evalill Nilsson, Marika Wenemark, Ulla Walfridsson, Mats Lundström, Charles Taft, Bo Palaszewski and Margareta Kristenson

Validation of the French version of the Functional, Communicative and Critical Health Literacy scale (FCCHL)

Health literacy is a key asset, defined as the capacity to acquire, understand and use information in ways which promote and maintain good health.

Authors: Youssoufa M. Ousseine, Alexandra Rouquette, Anne-Déborah Bouhnik, Laurent Rigal, Virginie Ringa, Allan ‘Ben’ Smith and Julien Mancini

The Japanese version of the National Cancer Institute’s patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE): psychometric validation and discordance between clinician and patient assessments of adverse events

The Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) was developed by the National Cancer Institute as an adverse event assessment system to evaluate patients...

Authors: Takashi Kawaguchi, Kanako Azuma, Motohiko Sano, Soan Kim, Yosuke Kawahara, Yoko Sano, Tomohide Shimodaira, Keiichiro Ishibashi, Tempei Miyaji, Ethan Basch and Takuhiro Yamaguchi

Adding patient-reported outcomes to a multisite registry to quantify quality of life and experiences of disease and treatment for youth with juvenile idiopathic arthritis

Children with Juvenile Idiopathic Arthritis (JIA) often have poor health-related quality of life (HRQOL) despite advances in treatment. Patient-centered research may shed light on how patient experiences of tr...

Authors: Elissa R. Weitzman, Lauren E. Wisk, Parissa K. Salimian, Kara M. Magane, Fatma Dedeoglu, Aimee O. Hersh, Yukiko Kimura, Kenneth D. Mandl, Sarah Ringold and Marc Natter

Barnhöft: a hip specific 6-item questionnaire for children

Health-related quality of life instruments, both general and more disease specific, would ideally be included in the evaluation of outcome in paediatric orthopaedics. The aim of this study was to translate and...

Authors: Bengt Herngren, Margaretha Stenmarker and Karin Enskär

A comparison of three methods to generate a conceptual understanding of a disease based on the patients’ perspective

The Food and Drug Administration patient-reported outcome (PRO) guidance provides standards for PRO development, but these standards bring scientific and logistical challenges which can result in a lengthy and...

Authors: Louise Humphrey, Thomas Willgoss, Andrew Trigg, Stephanie Meysner, Mary Kane, Sally Dickinson and Helen Kitchen

The development of a patient-reported outcome measure for patients with obstructive sleep apnea: the Patient-Reported Apnea Questionnaire (PRAQ)

Obstructive sleep apnea (OSA) is a chronic condition that can have a wide range of consequences for a patient’s health-related quality of life. Monitoring aspects of quality of life in clinical practice has th...

Authors: Inger L. Abma, Maroeska Rovers, Marijke IJff, Bernard Hol, Gert P. Westert and Philip J. van der Wees

Content validation of the SF-36v2® health survey with AL amyloidosis patients

This study examined the content validity of the SF-36v2® Health Survey (SF-36v2) in patients with AL amyloidosis using qualitative interviews with physicians and patients. The study included three distinct pha...

Authors: Michelle K. White, Martha S. Bayliss, Spencer D. Guthrie, Kimberly P. Raymond, Avery A. Rizio and Kristen L. McCausland

Japanese translation and linguistic validation of the US National Cancer Institute’s Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE)

The US National Cancer Institute (NCI) has developed the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) to capture patients’ self-reported symptomatic adver...

Authors: Tempei Miyaji, Yukiko Iioka, Yujiro Kuroda, Daigo Yamamoto, Satoru Iwase, Yasushi Goto, Masahiro Tsuboi, Hiroki Odagiri, Yu Tsubota, Takashi Kawaguchi, Naoko Sakata, Ethan Basch and Takuhiro Yamaguchi

The responsiveness of the PROMIS instruments and the qDASH in an upper extremity population

This study evaluated the responsiveness of several PROMIS patient-reported outcome measures in patients with hand and upper extremity disorders and provided comparisons with the qDASH instrument.

Authors: Man Hung, Charles L. Saltzman, Tom Greene, Maren W. Voss, Jerry Bounsanga, Yushan Gu, Angela A. Wang, Douglas Hutchinson and Andrew R. Tyser

Leveraging pediatric PROMIS item banks to assess physical functioning in children at risk for severe functional loss

Pediatric neuromuscular illnesses often result in decreased health-related quality of life (HRQL), notably in physical functioning. Generic HRQL measures have been developed for use in general populations, but...

Authors: Angie Mae Rodday, Robert J. Graham, Ruth Ann Weidner, Nan E. Rothrock, Darren A. Dewalt and Susan K. Parsons

Methodological considerations in using patient reported measures in dialysis clinics

Patient reported measures (PRMs), including patient-reported outcomes, play a critical role in dialysis care. The usage of PRMs is extensive in dialysis clinics. While there are excellent PRMs to choose from, ...

Authors: John D. Peipert and Ron D. Hays

Cognitive testing of the Colon Cancer Screening Behaviours Survey with South Asian immigrants in Canada

The purpose of this study was to cognitively test the Urdu and English language versions of a survey to assess colon cancer screening behaviours among South Asian immigrants in Canada.

Authors: Joanne Crawford, Farah Ahmad, Arlene S. Bierman and Dorcas Beaton

A systematic evaluation of compliance and reporting of patient-reported outcome endpoints in ovarian cancer randomised controlled trials: implications for generalisability and clinical practice

This study aimed to evaluate the patient-reported outcome (PRO) content of ovarian cancer randomised-controlled trial (RCT) publications, describe PRO compliance, and explore potential relationships among thes...

Authors: Rebecca Mercieca-Bebber, Michael Friedlander, Melanie Calvert, Martin Stockler, Derek Kyte, Peey-Sei Kok and Madeleine T. King

Exploring the quality of life issues in people with retinal diseases: a qualitative study

The lack of an appropriate retina-specific patient-reported outcome instrument restricts the understanding of the full impact of hereditary retinal diseases and other less common but potentially blinding acqui...

Authors: Mallika Prem Senthil, Jyoti Khadka, Jagjit Singh Gilhotra, Sumu Simon and Konrad Pesudovs

Journal of Patient-Reported Outcomes: aims and scope

Authors: Dennis Revicki and David Feeny

The content validity of the PSS in patients with plaque psoriasis

The primary objective of this study was to evaluate the content validity of the Psoriasis Symptom Scale (PSS), with a specific focus on understanding of the content of the PRO measure by conducting one-on-one ...

Authors: A. M. Rentz, A. M. Skalicky, K. Burslem, K. Becker, D. Kaschinski, D. Esser and D. A. Revicki

Psychometric validation of the Dysphagia Symptom Questionnaire in patients with eosinophilic esophagitis treated with budesonide oral suspension

Eosinophilic esophagitis (EoE) is characterized by high levels of eosinophils in the esophageal mucosa. Patients with the disease present with a range of symptoms, including dysphagia (difficulty swallowing). ...

Authors: Stacie Hudgens, Christopher Evans, Elaine Phillips and Malcolm Hill

Two-item PROMIS® global physical and mental health scales

Self-reports of health provide useful information about function and well-being that can improve communication between patients and clinicians. Global health items provide summary information that are predicti...

Authors: Ron D. Hays, Benjamin D. Schalet, Karen L. Spritzer and David Cella

Feasibility and acceptability of electronic symptom surveillance with clinician feedback using the Patient-Reported Outcomes version of Common Terminology Criteria for Adverse Events (PRO-CTCAE) in Danish prostate cancer patients

The aim was to examine the feasibility, acceptability and clinical utility of electronic symptom surveillance with clinician feedback using a subset of items drawn from the Patient-Reported Outcomes version of...

Authors: Christina Baeksted, Helle Pappot, Aase Nissen, Niels Henrik Hjollund, Sandra A. Mitchell, Ethan Basch, Pernille Envold Bidstrup, Susanne Oksbjerg Dalton and Christoffer Johansen