The development and initial validation of the PROMIS®+HF-27 and PROMIS+HF-10 profiles.

Aims: Heart failure (HF) is a common and morbid condition impacting multiple health domains. We previously reported the development of the PROMIS®-Plus-HF (PROMIS+HF) profile measure, including universal and HF-specific items. To facilitate use, we developed shorter, PROMIS+HF profiles intended for research and clinical use.

Optimizing brief, focused assessment of priority symptoms and concerns in recurrent and/or metastatic squamous cell carcinoma of the head and neck: Content validation of the Functional Assessment of Cancer Therapy/National Comprehensive Cancer Network Head and Neck Symptom Index-10 (FHNSI-10).

Background And Aims: Patients with recurrent and/or metastatic (R/M) squamous cell carcinoma of the head and neck (SCCHN) experience vast disease and treatment burdens. Brief, focused instruments are needed to assess patient-reported priority symptoms and concerns as targeted outcome assessments for use in clinical research. Although the instrument was developed based on expert and patient input and is psychometrically valid, the Functional Assessment of Cancer Therapy (FACT)/National Comprehensive Cancer Network (NCCN) Head and Neck Symptom Index-10 (FHNSI-10) has yet to undergo content validation from the perspective of R/M SCCHN patients to evaluate its use as a brief symptom-focused targeted endpoint assessment for use in clinical research.

The PROMIS-Plus-Osteoarthritis of the Knee (OAK) profile measure integrates generic and condition-specific content to enhance relevance and efficiency.

Objective: The Patient-Reported Outcomes Measurement Information System (PROMIS)-Plus-Osteoarthritis of the Knee (OAK) profile integrates universal PROMIS items with knee-specific items across 13 domains. We evaluated the psychometric properties of a subset of six domains associated with quality of life in people with OAK.

Study Design And Setting: In a cross-sectional study of OAK patients (n=600), we estimated reliability using Pearson and Spearman correlations with Knee Injury and Osteoarthritis Outcome Score (KOOS) subscores and known-groups validity with PROMIS Global Health.

Development of the Functional Assessment of Cancer Therapy-Carcinoid Syndrome Symptom Index.

Objective: To develop a symptom-focused index to evaluate representative symptoms, treatment side effects, and emotional and functional well-being of patients with carcinoid syndrome (CS).

Methods: The development of the Functional Assessment of Cancer Therapy-Carcinoid Syndrome Symptom Index (FACT-CSI) followed US Food and Drug Administration guidelines for the development of patient-reported outcome (PRO) measures and involved the following: (a) literature review; (b) interviews with 14 CS patients; (c) interviews with 9 clinicians; and (d) instrument development involving input from a range of PRO measure development and CS experts. The resulting draft instrument underwent cognitive interviews with 7 CS patients.

Validation of the Functional Assessment of Cancer Therapy-Leukemia instrument in patients with acute myeloid leukemia who are not candidates for intensive therapy.

Background: The objective of this study was to validate the Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu) in patients with acute myeloid leukemia (AML) who are not candidates for intensive therapy.

Methods: A sample of 317 patients with AML who were not eligible for intensive chemotherapy completed the FACT-Leu and EuroQol 5-Dimension (EQ-5D) measures (Utility Index and Visual Analogue Scale) every 28 days until the end of treatment. Internal consistency reliability was estimated with Cronbach's α.

Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ).

Purpose: To develop a patient preference questionnaire (PPQ) assessing eculizumab and ravulizumab treatment for paroxysmal nocturnal hemoglobinuria (PNH).

Patients And Methods: The development of the PNH-PPQ was consistent with Food and Drug Administration guidelines for patient-reported outcome measure development, and included 1) a targeted literature review; 2) PNH expert clinician input on treatment preferences; 3) review of existing qualitative data on the PNH treatment and disease experience; 4) concept elicitation interviews with 8 PNH patients who received eculizumab and/or ravulizumab; 5) translatability review; and 6) cognitive debriefing with 5 patients. Interview participants were recruited through a United Kingdom PNH patient advocacy group and a Canadian clinical site involved in clinical trial ALXN1210-PNH-302.

Financial burden and quality of life among thyroid cancer survivors.

Background: Survivors of cancer in the United States are often financially encumbered by expenses and lost wages from cancer treatment. The majority of patients with thyroid cancer are diagnosed before age 65, when they are not eligible for Medicare. We hypothesized that financial distress would be common among thyroid cancer survivors and would be associated with poor health-related quality of life.

Responsiveness of PROMIS® to change in chronic obstructive pulmonary disease.

Background: Chronic obstructive pulmonary disease (COPD) is a progressive chronic disease characterized by airflow obstruction that leads to shortness of breath and substantial negative impacts on health-related quality of life (HRQL). The course of COPD includes periodic acute exacerbations that require changes in treatment and/or hospitalizations. This study was designed to examine the responsiveness of Patient-Reported Outcomes Measurement Information System® (PROMIS®) measures to changes associated with COPD exacerbation recovery.

Establishing clinically-relevant terms and severity thresholds for Patient-Reported Outcomes Measurement Information System (PROMIS) measures of physical function, cognitive function, and sleep disturbance in people with cancer using standard setting.

Purpose: Patient-Reported Outcomes Measurement Information System (PROMIS) physical function, cognitive function, and sleep disturbance measures are increasingly used in cancer care. However, there is limited guidance for interpreting the clinical meaning of scores. This study aimed to apply bookmarking, a standard setting methodology, to identify PROMIS score thresholds in the context of cancer care.

Development and Initial Validation of the PROMIS®-Plus-HF Profile Measure.

Background Bringing together generic and heart failure (HF)-specific items in a publicly available, patient-reported outcome measure may facilitate routine health status assessment for improving clinical care and shared decision-making, assessing quality of care, evaluating new interventions, and comparing groups with different conditions. Methods and Results We performed a mixed-methods study to develop and validate the PROMIS®-Plus-HF (Patient-Reported Outcomes Measurement Information System®-Plus-Heart Failure) profile measure-a HF-specific instrument based on the generic PROMIS. We conducted 8 focus groups with 61 patients with HF and phone interviews with 10 HF clinicians.

PROMIS®: Standardizing the patient voice in health psychology research and practice.

The Patient-Reported Outcomes Measurement Information System® (PROMIS®) is a collection of person-centered measures of physical, mental, and social health in children and adults. Developed using state-of-the-art psychometric methods, these measures are offered as fixed-length questionnaires and computerized adaptive tests, with translations available in Spanish and other languages. This special issue presents articles that illustrate the use of PROMIS® measures to advance health behavior research across a wide range of health studies.

Post-thyroidectomy neck appearance and impact on quality of life in thyroid cancer survivors.

Background: There is a paucity of patient-reported data on thyroidectomy scar perception. The effect of neck scarring on quality of life is not known. We hypothesized that worse perception of neck appearance would be related to worse health-related quality of life and that perception improves with time.

Clinical factors associated with worse quality-of-life scores in United States thyroid cancer survivors.

Introduction: Thyroid cancer survivors are a rapidly growing population in the United States. The factors that drive health-related quality of life (HRQOL) in this population have not been well characterized. We hypothesized that more aggressive treatments and greater treatment-related adverse effects would be associated with worse HRQOL scores in thyroid cancer survivors.

Benchmarking health-related quality of life in thyroid cancer versus other cancers and United States normative data.

Background: Thyroid cancer survivors may experience long-lasting physical, psychosocial, and financial challenges. No previous studies have compared health-related quality of life in thyroid cancer survivors to United States normative data and patient-reported outcomes from other types of cancers. We hypothesized that thyroid cancer survivors would report health-related quality of life poorer than the general United States population but similar to individuals with other cancers.

Quality of life in multiple endocrine neoplasia type 2A compared with normative and disease populations.

Background: Patient-reported outcomes are measured in chronic disease states to inform intervention and management decisions while minimizing negative outcomes. We hypothesized that health-related quality of life in patients with multiple endocrine neoplasia type 2A would be worse than the general US population but similar to other chronic diseases.

Methods: Adults ≥18 years with multiple endocrine neoplasia type 2A were recruited to complete the Patient-Reported Outcomes Measurement Information System 29-item questionnaire (n = 45).

Psychological Functioning in Patients With Chronic Obstructive Pulmonary Disease: A Preliminary Study of Relations With Smoking Status and Disease Impact.

Introduction: Chronic obstructive pulmonary disease (COPD) is a tobacco-related disease associated with several comorbid conditions, including elevated rates of depression and anxiety. Psychological factors that commonly underlie nicotine dependence, depression, and anxiety may represent novel treatment targets, but have not yet been examined among patientswith COPD. We assessed three psychological factors-anxiety sensitivity (AS; fear of anxiety-related sensations), distress intolerance (DI; inability to withstand distressing states), and anhedonia (Anh; diminished sense of pleasure or interest)-in relation to smoking status, COPD symptom impact, and negative response to COPD symptoms.

Health-related quality of life in MEN1 patients compared with other chronic conditions and the United States general population.

Background: Health-related quality of life (HRQOL) in multiple endocrine neoplasia type-1 (MEN-1) is poorly described. HRQOL in MEN-1 was compared with other chronic conditions and the US general population.

Methods: Adults aged ≥18 years recruited from an MEN-1 support group (n=153) completed the Patient-Reported Outcomes Measurement Information System (PROMIS) 29-item profile.

Financial burden is associated with worse health-related quality of life in adults with multiple endocrine neoplasia type 1.

Background: Health-related quality of life and financial burden among patients with multiple endocrine neoplasia type 1 is poorly described. It is not known how financial burden influences health-related quality of life in this population. We hypothesized that the financial burden attributable to multiple endocrine neoplasia type 1 is associated with worse health-related quality of life.

Disease and treatment factors associated with lower quality of life scores in adults with multiple endocrine neoplasia type I.

Background: Physical and psychosocial morbidity of multiple endocrine neoplasia type-1 is ill-defined. How disease and treatment-related factors relate to patient-reported outcomes including health-related quality of life is unknown. We hypothesized that disease and treatment burden negatively impacts health-related quality of life in adults with multiple endocrine neoplasia type-1.

A method to create a standardized generic and condition-specific patient-reported outcome measure for patient care and healthcare improvement.

Purpose: Patient-reported outcome measures (PROMs), which are generic or condition-specific, are used for a number of reasons, including clinical care, clinical trials, and in national-level efforts to monitor the quality of health care delivery. Creating PROMs that meet different purposes without overburdening patients, healthcare systems, providers, and data systems is paramount. The objective of this study was to test a generalizable method to incorporate condition-specific issues into generic PROM measures as a first step to producing PROMs that efficiently provide a standardized score.

Health-Related Quality of Life in Patients with Idiopathic Pulmonary Fibrosis.

Purpose: Idiopathic pulmonary fibrosis (IPF) produces symptoms and activity limitations that impair health-related quality of life (HRQOL). The Patient-Reported Outcomes Measurement Information System(®) (PROMIS(®)) includes measures of self-reported health and HRQOL for a range of conditions. This study evaluated the HRQOL of individuals with IPF using PROMIS measures and examined associations between HRQOL and key symptoms or supplemental oxygen need.

Development and validation of the functional assessment of cancer therapy-antiangiogenesis subscale.

The Functional Assessment of Cancer Therapy (FACT)-Antiangiogenesis (AntiA) Subscale was developed and validated to enhance treatment decision-making and side effect management for patients receiving anti-angiogenesis therapies. Side effects related to anti-angiogenesis therapies were identified from the literature, clinician input, and patient input. Fifty-nine possible patient expressions of side effects were generated.

Correlation of PROMIS scales and clinical measures among chronic obstructive pulmonary disease patients with and without exacerbations.

Purpose: The Patient-Reported Outcomes Measurement Information System (PROMIS(®)) initiative was developed to advance the methodology of PROs applicable to chronic diseases. Chronic obstructive pulmonary disease (COPD) is a progressive chronic disease associated with poor health. This study was designed to examine the correlation of PROMIS health-related quality of life (HRQOL) scales and clinical measures among COPD patients.

A qualitative study of unmet healthcare needs in chronic obstructive pulmonary disease. A potential role for specialist palliative care?

Rationale: Patients with chronic obstructive pulmonary disease (COPD) have high symptom burdens and poor health-related quality of life. The American Thoracic Society issued a consensus statement outlining the need for palliative care for patients with chronic respiratory diseases. A better understanding of the unmet healthcare needs among patients with COPD may help determine which aspects of palliative care are most beneficial.