Articles

Patient reported outcomes – experiences with implementation in a University Health Care setting

Patient-reported outcomes (PROs) have traditionally been implemented through a manual process of paper and pencil with little standardization throughout a Healthcare System. Each practice has asked patients sp...

Authors: Joshua Biber, Dominik Ose, Jenny Reese, Anna Gardiner, Julio Facelli, Joshua Spuhl, Darrel Brodke, Vivian S. Lee, Rachel Hess and Howard Weeks

Patients’ experience of recurrent/metastatic head and neck squamous cell carcinoma and their perspective on the EORTC QLQ-C30 and QLQ-H&N35 questionnaires: a qualitative study

Head and neck squamous cell carcinoma (HNSCC) and its associated treatments may affect all aspects of patients’ health-related quality of life (HRQoL). Although the EORTC QLQ-H&N35 is regularly administered to...

Authors: Arnold Degboe, Sarah L. Knight, Katarina Halling, Andrew Trigg, Tamara Al-Zubeidi, Natalie Aldhouse, Helen Kitchen, Lori Wirth and Simon N. Rogers

The feasibility of implementing the ICHOM Standard Set for Hip and Knee Osteoarthritis: a mixed-methods evaluation in public and private hospital settings

There is growing international momentum for standardising patient outcome assessment and using patient-reported outcome measures (PROMs) to capture outcomes that matter to patients. The International Consortiu...

Authors: Ilana N. Ackerman, Bernarda Cavka, Jacob Lippa and Andrew Bucknill

Burden of renal angiomyolipomas associated with tuberous sclerosis complex: results of a patient and caregiver survey

Tuberous sclerosis complex (TSC) is a rare genetic disorder characterized by benign tumors in multiple organs, including non-cancerous kidney lesions known as renal angiomyolipomas. This study’s objective is t...

Authors: Anne M. Rentz, Anne M. Skalicky, Zhimei Liu, David W. Dunn, Michael D. Frost, Jo Anne Nakagawa, Judith Prestifilippo, Qayyim Said and James W. Wheless

Measurement properties of the German version of the IKDC subjective knee form (IKDC-SKF)

To examine the measurement properties of the German International Knee Documentation Committee Subjective Knee Form (IKDC-SKF) in knee disorder patients.

Authors: Danica Kümmel, Stefan Preiss, Laurent P. Harder, Michael Leunig and Franco M. Impellizzeri

The relationship between comorbidity medication adherence and health related quality of life among patients with cancer

Studies have demonstrated that comorbidities compound the adverse influence of cancer on health-related quality of life (HRQoL). Comorbidities adversely impact adherence to cancer treatment. Additionally, adhe...

Authors: Dana Drzayich Antol, Adrianne Waldman Casebeer, Raya Khoury, Todd Michael, Andrew Renda, Sari Hopson, Aparna Parikh, Alisha Stein, Mary Costantino, Stephen Stemkowski and Mikele Bunce

Health-related quality of life burden of nonalcoholic steatohepatitis: a robust pragmatic literature review

Nonalcoholic steatohepatitis (NASH) is a form of chronic liver disease (CLD): patients have an increased risk of developing cirrhosis, liver failure, and complications (e.g. hepatocellular carcinoma). NASH has...

Authors: Tessa Kennedy-Martin, Jay P. Bae, Rosirene Paczkowski and Emily Freeman

Patient-reported outcomes for patients with metastatic castration-resistant prostate cancer receiving docetaxel and Atrasentan versus docetaxel and placebo in a randomized phase III clinical trial (SWOG S0421)

SWOG S0421 was a large randomized trial comparing docetaxel/prednisone plus placebo (DPP) to docetaxel/prednisone plus atrasentan over 12 cycles for patients with metastatic castration-resistant prostate cance...

Authors: Joseph M. Unger, Katherine Griffin, Gary W. Donaldson, Karen M. Baranowski, Margorie J. Good, Eunicia Reburiano, Maha Hussain, Paul J. Monk, Peter J. Van Veldhuizen, Michael A. Carducci, Celestia S. Higano, Primo N. Lara, Catherine M. Tangen, David I. Quinn, James L. Wade, III…

Development and validation of the Ulcerative Colitis patient-reported outcomes signs and symptoms (UC-pro/SS) diary

The clinical course of ulcerative colitis (UC) and the effects of treatment are assessed through patient-reported signs and symptoms (S&S), and endoscopic evidence of inflammation. The Ulcerative Colitis Patie...

Authors: Peter D. R. Higgins, Gale Harding, Dennis A. Revicki, Gary Globe, Donald L. Patrick, Kristina Fitzgerald, Hema Viswanathan, Sarah M. Donelson, Brian G. Ortmeier, Wen Hung Chen, Nancy K. Leidy and Kendra DeBusk

Rasch analysis in the development of the NutriQoL® questionnaire, a specific health-related quality of life instrument for home enteral nutrition

Home enteral nutrition (HEN) is a therapeutic method used in patients who are unable to ingest the required amounts of nutrients but retain a functional gastrointestinal tract. The objective of this study was ...

Authors: Antonio Apezetxea, Lourdes Carrillo, Felipe Casanueva, Cristina de la Cuerda, Federico Cuesta, Jose Antonio Irles, Maria Nuria Virgili, Miquel Layola and Luis Lizán

Development and validation of the Crohn’s disease patient-reported outcomes signs and symptoms (CD-PRO/SS) diary

The clinical course of Crohn’s disease (CD) and the effect of its treatment are monitored through patient-reported signs and symptoms (S&S), and endoscopic evidence of inflammation. The Crohn’s Disease Patient...

Authors: Peter D. R. Higgins, Gale Harding, Nancy K. Leidy, Kendra DeBusk, Donald L. Patrick, Hema N. Viswanathan, Kristina Fitzgerald, Sarah M. Donelson, Marcoli Cyrille, Brian G. Ortmeier, Hilary Wilson, Dennis A. Revicki and Gary Globe

Care-related Quality of Life of informal caregivers of the elderly after a hip fracture

Reforms in the Dutch healthcare system in combination with the aging of the population will lead to a strong increase in the demand for informal care in the Netherlands. A hip fracture is one of the most impor...

Authors: Cornelis L. P. van de Ree, Kari Ploegsma, Tim A. Kanters, Jan A. Roukema, Mariska A. C. De Jongh and Taco Gosens

Health-related quality of life in colorectal cancer survivors: are there differences between sporadic and hereditary patients?

To compare health-related quality of life (HRQoL) in colorectal cancer (CRC) survivors with sporadic CRC to those with hereditary cancer, specifically Lynch syndrome (LS).

Authors: Allison M. Burton-Chase, Wendy M. Parker, Kirsten M. Donato, Shannon McCormick, Ellen R. Gritz, Christopher I. Amos, Karen H. Lu, Patrick M. Lynch, Miguel A. Rodriguez-Bigas, Y. Nancy You and Susan K. Peterson

Changes in health-related quality of life and work-related outcomes for patients with mild-to-moderate ulcerative colitis receiving short-term and long-term treatment with multimatrix mesalamine: a prospective, open-label study

Ulcerative colitis (UC) is associated with lower health-related quality of life (HRQoL), and with disease activity predicting lower HRQoL and worse work-related outcomes. The current study examined the burden ...

Authors: Mary Kaye Willian, Geert D’Haens, Aaron Yarlas and Ashish V. Joshi

A utility valuation study assessing the impact of postprandial glucose control on quality of life of individuals with type 1 or type 2 diabetes

Consideration of health-related quality of life (HRQOL) in diabetes has been associated with long-term and short-term complications such as hypoglycaemia, but not with short-term glucose control. This study ai...

Authors: J. Jendle, A. Sandberg, S. Buchs, P. Swinburn, M. Hadi and L. Å. Levin

Understanding and measuring symptoms and health status in asthma COPD overlap: content validity of the EXACT and SGRQ

Asthma-chronic obstructive pulmonary disease overlap (ACO) differs from asthma and chronic obstructive pulmonary disease (COPD) in demographics, phenotypic characteristics and outcomes, yet the patient experie...

Authors: Linda Nelsen, Adam Gater, Charlotte Panter, Chloe Tolley, Laurie Lee and Steven Pascoe

Towards the use of mixed methods inquiry as best practice in health outcomes research

Mixed methods research (MMR) has found an increased interest in the field of health outcomes research. Consideration for both qualitative and quantitative perspectives has become key to contextualising patient...

Authors: Antoine Regnault, Tom Willgoss and Skye Barbic

Development of the Weight-Related Sign and Symptom Measure

Overweight and obesity have been associated with physical and emotional signs & symptoms. Research has shown that modest weight loss can mitigate some symptoms in individuals with overweight or obesity. This s...

Authors: Meryl Brod, Lise Højbjerre, Kathryn M. Pfeiffer, Robyn Sayner, Henrik H. Meincke and Donald L. Patrick

Content validity and ePRO usability of the BPI-sf and “worst pain” item with pleural and peritoneal mesothelioma

The Brief Pain Inventory-short form (BPI-sf) is widely used in self-reported pain assessment, incorporates pain numeric rating scales (NRS) and is commonly utilized in electronic format in clinical trials, how...

Authors: Heather L. Gelhorn, Sonya Eremenco, Anne M. Skalicky, Zaneta Balantac, Tricia Cimms, Katarina Halling and Chris Sexton

Development of the Impact of Juvenile Metachromatic Leukodystrophy on Physical Activities scale

Metachromatic leukodystrophy (MLD) is a rare disease with three forms based on the age at onset of signs and symptoms. The objective of this study was to develop a caregiver-reported clinical outcome assessmen...

Authors: T. Michelle Brown, Susan Martin, Sheri E. Fehnel and Linda S. Deal

Measurement of alienation among adolescents: construct validity of three scales on powerlessness, meaninglessness and social isolation

Psychological alienation is an important concept in the study of adolescents’ health and behavior but no gold standard for measuring alienation among adolescents exists. There is a need for new scales with hig...

Authors: Signe Boe Rayce, Svend Kreiner, Mogens Trab Damsgaard, Tine Nielsen and Bjørn Evald Holstein

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

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 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

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

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

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

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

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

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

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

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

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

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

Journal of Patient-Reported Outcomes: aims and scope

Authors: Dennis Revicki and David Feeny

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