Cutaneous Manifestations in Patients with Dermatomyositis, Are They Only Skin Deep?

Background: Dermatomyositis (DM) is a rare and severely debilitating autoimmune disease that can affect children and adults; however, there is little understanding of the patient-reported experience and uncertainty around validated clinical outcomes assessments (COAs) that could measure changes in the condition during clinical trials of new treatments.

Objectives: The aim of this study was to understand the patient experience of DM, with a focus on its cutaneous manifestations, to describe the patient experience and determine the suitability of existing COA measures.

Methods: Adult (≥ 18 years) patients (N = 28) with severe active cutaneous manifestations of DM were interviewed.

Development of the Cutaneous Dermatomyositis Investigator Global Assessment (CDM-IGA): A De Novo IGA of Cutaneous Manifestations of Dermatomyositis.

Introduction: Dermatomyositis (DM) is a rare systemic autoimmune disease characterized by a distinctive debilitating skin rash and skeletal muscle weakness. It is unclear if existing clinical outcome assessment (COA) measures include the concepts of priority to patients and those necessary to fully capture improvements in the active cutaneous manifestations of DM. This study aimed to develop the Cutaneous Dermatomyositis Investigator Global Assessment (CDM-IGA), a de novo IGA, for use in clinical trials of adult DM.

Development of a Conceptual Model for the Patient Experience of Immunoglobulin A Nephropathy (IgAN): A Qualitative Literature Review.

Introduction: Immunoglobulin A nephropathy (IgAN) is a kidney disorder that can lead to progressive kidney disease. Currently, there lacks a comprehensive overview of the symptoms and impacts experienced by those living with IgAN that would help inform the selection or development of fit-for-purpose clinical outcome assessments (COA) to be used in clinical trials. The aim of this study was to develop a conceptual model of the adult and pediatric patient experience of IgAN, including disease signs and symptoms, treatment side effects, and impact on functioning and well-being.

Development of a Conceptual Model for the Patient Experience of Focal Segmental Glomerulosclerosis (FSGS): A Qualitative Targeted Literature Review.

Introduction: Focal segmental glomerulosclerosis (FSGS) is a leading cause of kidney disease and can progress to end stage kidney disease (ESKD). An overview of symptoms and impacts of the disease experienced will help inform the selection or development of fit-for-purpose clinical outcome assessments (COA) to be used in FSGS clinical trials. This study aimed to develop a conceptual model (CM) of the adult and pediatric patient experience of FSGS including disease signs/symptoms, treatment side-effects, and impact on functioning and wellbeing.

Efficacy and Safety of the TYK2/JAK1 Inhibitor Brepocitinib for Active Psoriatic Arthritis: A Phase IIb Randomized Controlled Trial.

Objective: Brepocitinib is a TYK2/JAK1 inhibitor in development for the treatment of several immunologic diseases. The efficacy and safety of oral brepocitinib were assessed in participants with moderately-to-severely active psoriatic arthritis (PsA) for up to 52 weeks.

Methods: In this placebo-controlled, dose-ranging, phase IIb study, participants were randomized to receive 10 mg, 30 mg, or 60 mg of brepocitinib once daily or placebo, advancing to 30 mg or 60 mg of brepocitinib once daily at week 16.

A Review of Labeling Based on Patient-Reported Outcome Endpoints for New Oncology Drugs Approved by the European Medicines Agency (2017-2021).

Objective: A review of new oncology indications approved by the European Medicines Agency (EMA) for 2012-2016 showed that 33% of new drugs had labeling based on patient-reported outcomes (PROs). We reviewed labeling text based on PRO endpoints for new oncology indications approved during 2017-2021.

Methods: New oncology drugs approved by EMA to treat indications of cancers during 2017-2021 were identified from the EMA website.

Validation of the Alopecia Areata Patient Priority Outcomes (AAPPO) Questionnaire in Adults and Adolescents with Alopecia Areata.

Introduction: Individuals with alopecia areata (AA) may experience significant impacts on their health-related quality of life. The novel Alopecia Areata Patient Priority Outcomes (AAPPO) questionnaire has been developed to assess hair loss signs, emotional symptoms, and activity limitations associated with AA. The objective of this study was to evaluate psychometric properties and establish scoring of the AAPPO in adults and adolescents with AA.

Voice Analysis of Cancer Experiences Among Patients With Breast Cancer: VOICE-BC.

Patient experience literature in early-stage breast cancer (eBC) is limited. This study used a mixed-methods approach to examine patient conversations from public online forums to identify and evaluate eBC-related themes. Among 60,000 eBC-related posts published September 2014-2019, text from a random subset of 15,000 posts was extracted and grouped into linguistically similar, mutually exclusive clusters using an advanced natural language processing (NLP) algorithm.

Patient Perspectives of the Social, Emotional and Functional Impact of Alopecia Areata: A Systematic Literature Review.

Introduction: Alopecia areata (AA) is a chronic, autoimmune disease of hair loss, which can significantly affect the emotional and psychological well-being of patients. A systematic literature review was conducted to better understand the burden of AA from the patient perspective.

Methods: Embase, MEDLINE and Cochrane databases were searched for published studies (2008-2018) reporting on assessments of health-related quality of life (HRQoL) for patients with AA.

Development of the Alopecia Areata Patient Priority Outcomes Instrument: A Qualitative Study.

Introduction: Although alopecia areata (AA) profoundly impacts patients' physical appearance, emotional state, and daily activities, no treatment approved for AA currently exists. Patient-reported outcome (PRO) instruments currently used to capture patients' AA experiences do not meet the requirements to support claims of treatment benefit as described in the US Food and Drug Administration's 2009 PRO guidance. Our objective was to explore the consequences and priority treatment outcomes among individuals with AA and develop a PRO measure consistent with regulatory requirements that assesses these priorities and represents clinical benefit from the AA patient perspective.

The Patient Experience with Soft Tissue Sarcoma: A Systematic Review of the Literature.

Background: Soft tissue sarcomas (STS) are a heterogenous group of rare tumors that involve the connective tissue in the body (e.g. muscle, tendons).

Patient-Reported Outcomes in Latin America: Implementation in Research and Role in Emerging HTA Systems.

Background: Patient-reported outcomes (PROs) are increasingly used to demonstrate the value of interventions and support health technology assessment (HTA).

Objective: The objective of this work was to analyze trends regarding PROs in Latin America (LatAm), highlight challenges in the application of PROs in this region, and suggest solutions.

Methods: A team of researchers with expertise in PROs conducted a nonsystematic PubMed literature search pertaining to the use of PROs in LatAm.

Proposing a standardized method for evaluating patient report of the intensity of dyspnea during exercise testing in COPD.

Background: Measuring dyspnea intensity associated with exercise provides insights into dyspnea-limited exercise capacity, and has been used to evaluate treatment outcomes for chronic obstructive pulmonary disease (COPD). Three patient-reported outcome scales commonly cited for rating dyspnea during exercise are the modified Borg scale (MBS), numerical rating scale for dyspnea (NRS-D), and visual analogue scale for dyspnea (VAS-D). Various versions of each scale were found.

Capturing patients' perspectives of treatment in clinical trials/drug development.

The patient's perspective of treatment outcomes is increasingly important to consumers and providers of healthcare. Recent studies have shown that traditional clinical endpoints may not accurately reflect the patient experience with treatment. Often patients' experience of their disease and associated treatment differs from the perspective of their physicians.

Development of the EXAcerbations of Chronic Obstructive Pulmonary Disease Tool (EXACT): a patient-reported outcome (PRO) measure.

Background: This article describes the qualitative methods used to develop the EXAcerbation of Chronic Pulmonary Disease Tool (EXACT), a new patient-reported outcome (PRO) instrument for evaluating frequency, severity, and duration of exacerbations of chronic obstructive pulmonary disease (COPD).

Methods: Focus groups and interviews were conducted in the United States with COPD patients treated for exacerbations during the past 6 months. Participants were asked to describe exacerbation attributes, care-seeking cues, and indications of progression and recovery.