Analysis of patient-reported experiences up to 2 years after receiving idecabtagene vicleucel (ide-cel, bb2121) for relapsed or refractory multiple myeloma: Longitudinal findings from the phase 2 KarMMa trial.

OBJECTIVE: To understand the long-term experience of patients receiving ide-cel chimeric antigen receptor T (CAR T) cell therapy for relapsed or refractory multiple myeloma in the pivotal phase 2 KarMMa trial. METHODS: This qualitative study analyzed semi-structured patient interviews 6-24 months after ide-cel infusion. Thematic analysis with quantitative and longitudinal analyses explored patient perceptions of ide-cel treatment experience, advantages and disadvantages, and long-term health-related quality of life impact. Patient journeys were developed from narrative analysis of perceived treatment benefits with known remission length. RESULTS: Interviews with 45 patients 6-24 months postinfusion were analyzed; all reported ≥ 1 ide-cel treatment advantage, most often related to efficacy (n = 42/45, 93%), few or no side effects (n = 35/45, 78%), and avoidance of other treatments (n = 34/45, 76%). Patients generally reported 6-month improvements in physical health, functioning, emotional well-being, social life, and outlook on the future; these improvements mostly remained "stable" through 18 and 24 months. The most common patient journeys comprised physical, functioning, or emotional benefit with remission < 2 years. CONCLUSIONS: Longitudinal analysis of patient experiences showed sustained benefits and preference for ide-cel up to 24 months after treatment. Trial Registration Number and Date: NCT03361748. December 5, 2017.

Patient experience before and after treatment with idecabtagene vicleucel (ide-cel, bb2121): qualitative analysis of patient interviews in the KarMMa trial.

OBJECTIVE: To understand the experience of patients with relapsed and refractory multiple myeloma (RRMM) receiving idecabtagene vicleucel (ide-cel), a B-cell maturation antigen-directed chimeric antigen receptor T cell therapy, in the pivotal, phase 2 KarMMa trial. METHODS: Optional semi-structured interviews before leukapheresis (pre-treatment) captured expectations and after ide-cel infusion (1, 2, and 3 months post-treatment), assessed treatment experience, ide-cel advantages/disadvantages, and health and well-being. In a mixed-method analysis, treatment experiences were categorized by clinical response status, health and well-being, and self-reported recovery after infusion. RESULTS: Pre-treatment interviews indicated unmet treatment needs. In post-treatment interviews, most patients reported the positives of ide-cel outweighed negatives (69%, n = 27/39). Most common advantages of ide-cel were efficacy (18-64%), favorable side-effect profile (46-68%), and recovery time (13-18%); most common disadvantages were related to side effects (13-20%). When analyzed by clinical response, patients most often had stringent complete or very good partial response and improved health and well-being with mild or severe recovery from the infusion (27/58, 47%). Most patients with minimal clinical response reported mild infusion recovery (5/6, 83%). CONCLUSIONS: Patient interviews before ide-cel treatment showed unmet needs in triple-class exposed RRMM. Post-treatment experiences generally favored ide-cel versus previously received treatments.

The Worst Itch Numeric Rating Scale for patients with moderate to severe plaque psoriasis or psoriatic arthritis.

Plaque psoriasis (PP) and psoriatic arthritis (PsA) are autoinflammatory chronic conditions associated with skin involvement. Pruritus, or itching, is a prevalent and bothersome symptom in patients with PP and is associated with reduced health-related quality of life. The Worst Itch Numeric Rating Scale (WI-NRS) has been developed as a simple, single item with which to assess the patient-reported severity of this symptom at its most intense during the previous 24-hour period. Qualitative research was undertaken to assess the content validity of the WI-NRS. Patients with moderate to severe PP and patients with PsA were recruited from clinical sites in the USA. The qualitative research entailed two-part interviews, which began with concept elicitation to gain understanding of patients' experiences of itching, followed by cognitive debriefing of the WI-NRS to assess the instrument's understandability, clarity, and degree of appropriateness from the patient's perspective. Twelve patients with PP and 22 with PsA participated in the study. Patients reported that itching was an important and relevant symptom of their psoriatic disease. The WI-NRS was reported to be complete and easy to understand; the recall period was considered appropriate, the response scale was familiar, and, overall, the instrument was found to be appropriate for assessing itching severity. Patient responses support the content validity of the WI-NRS. The psychometric properties of the tool will be evaluated in future studies.

Barriers to mesalamine adherence in patients with inflammatory bowel disease: a qualitative analysis.

BACKGROUND: The causes for nonadherence to mesalamine in patients with inflammatory bowel disease (IBD) have been characterized using mostly indirect methods. Patient-reported barriers are lacking in this population. OBJECTIVE: To identify patient-reported barriers to mesalamine adherence through direct interviews. METHODS: Focus groups and one-on-one interviews were undertaken in adult patients with IBD. Transcripts from the focus groups and interviews were analyzed to identify themes and links between these themes, assisted by qualitative data software MaxQDA. RESULTS: Of 27 patients participating, 21 (78%) had ulcerative colitis, and 6 (22%) had Crohn's disease. Their self-reported adherence ranged from complete adherence (n = 3) to intermittent nonadherence (n = 24). Patients frequently indicated that they were resistant to taking medications for their condition. The barriers to adherence that emerged from interviews could be categorized under a number of themes: competing priorities, social stigma, refill inconvenience, costs, efficacy values, side effects, and pill characteristics. Efficacy values reported to influence adherence included doubts about efficacy, consequences of missed doses, and doubts about need for maintenance medication. Pill characteristics reported as barriers included pill size and pill frequency. Despite use of electronic prescribing, obtaining refills was reported as an obstacle to adherence in this cohort. Decanting of pills to multiple containers to increase accessibility was also reported. CONCLUSIONS: Patients with both ulcerative colitis and Crohn's disease report a number of common barriers to mesalamine adherence. Factors in medication-taking behavior and beliefs were reported in this study that may have implications for strategies to improve adherence by health care providers.

The burden of inflammatory bowel disease: a patient-reported qualitative analysis and development of a conceptual model.

BACKGROUND: The aim of this study was to describe the impacts of inflammatory bowel disease (IBD) from the patients' perspective and to inform the development of a conceptual model. METHODS: Focus groups and one-on-one interviews were undertaken in adult patients with IBD. Transcripts from the focus groups and interviews were analyzed to identify themes and links between themes, assisted by qualitative data software MaxQDA. Themes from the qualitative research were supplemented with those reported in the literature and concepts included in IBD-specific patient-reported outcome measures. RESULTS: Twenty-seven patients participated. Key physical symptoms included pain, bowel-related symptoms such as frequency, urgency, incontinence, diarrhea, passing blood, and systemic symptoms such as weight loss and fatigue. Participants described continuing and variable symptom experiences. IBD symptoms caused immediate disruption of activities but also had ongoing impacts on daily activities, including dietary restrictions, lifestyle changes, and maintaining close proximity to a toilet. More distal impacts included interference with work, school, parenting, social and leisure activities, relationships, and psychological well-being. The inconvenience of rectal medications, refrigerated biologics, and medication refills emerged as novel burdens not identified in existing patient-reported outcome measures. CONCLUSIONS: IBD symptoms cause immediate disruption in activities, but patients may continue to experience some symptoms on a chronic basis. The conceptual model presented here may be useful for identifying target concepts for measurement in future studies in IBD.

Patient Preferences for Chronic Myeloid Leukemia Medication Regimen Attributes and their Potential Impact on Adherence: Results from a Multi-national Conjoint Study.

Background: Tyrosine kinase inhibitors (TKIs) have significantly improved survival for patients with chronic myeloid leukemia (CML) but require long-term administration and non-adherence due to regimen requirements has been reported. Objectives: This study sought to identify how much patients value more convenient regimens and the potential impact that regimen may have on medication adherence. Methods: This cross-sectional, six-country study utilized a web-based discrete conjoint experiment (DCE) survey in which participants selected between hypothetical treatments that differed on three attributes: meal requirements/restrictions, frequency of administration, and monthly co-pay, to quantify willingness to pay. Attribute percent importance ratings were derived from a multinomial logit model, and utilities were summed for each product profile to determine the most preferred regimen profile. Additional survey questions asked about attributes perceived to affect adherence and the ease and convenience of participants' current regimen. Results: A total of 318 patients completed the survey; median age 53 years (range 18-87); 43.7% male. Four participants were excluded from the conjoint analysis due to illogical responses. The most important regimen attribute driving preferences was the meal requirement/restriction, which was almost twice as important as dose frequency. The majority of participants preferred the profile of a once a day dosing taken with or without a meal, and estimates of willingness-to-pay helped to quantify this preference. In terms of adherence, the majority of participants perceived that having to fast before and after taking medication would be the most likely reason for missing a dose. Conclusions: The results suggest that patients value the convenience of CML treatments and perceive certain regimen characteristics, particularly meal requirements or restrictions, as likely to affect adherence. It is important for healthcare providers to be aware of the potential impact of treatment convenience on non-adherence and communicate closely with patients to decrease this potential.

The patient experience with fatigue and content validity of a measure to assess fatigue severity: qualitative research in patients with ankylosing spondylitis (AS).

BACKGROUND: Ankylosing spondylitis (AS) is an autoimmune disorder characterized by inflammation of the spine and large joints. Fatigue is a common symptom that many AS patients find significantly impacts their health-related quality of life. The Worst Fatigue - Numeric Rating Scale (WF-NRS) assesses the severity of this symptom during the previous 24-hour period. The objective of this study was to perform qualitative research to support the development and content validity of the WF-NRS. METHODS: Patients with AS were recruited from clinical sites in the U.S. for a qualitative study which first entailed concept elicitation interviews to gain understanding of the patients' experience with AS and fatigue. Subsequently, cognitive debriefing interviews were undertaken to assess the understandability, clarity, and appropriateness from the patient's perspective, of the content of a measure of fatigue severity. RESULTS: Thirteen patients with AS participated in concept elicitation interviews and cognitive debriefing of the Brief Fatigue Inventory (BFI) fatigue severity subscale. The WF-NRS was developed from the worst fatigue item of the BFI as patients generally reported it to be understandable and covered an important concept, the completion instructions were modified, but the response scale remained as it was familiar and readily completed, and the recall period was appropriate. CONCLUSIONS: Patient responses resulted in the development of and supported the content validity of the WF-NRS. Further quantitative evaluation of the WF-NRS is warranted in order to assess its psychometric properties and confirm its usefulness as a clinical trial tool.

Demonstrating measurement equivalence of the electronic and paper formats of the Urticaria Patient Daily Diary in patients with chronic idiopathic urticaria.

BACKGROUND: The Urticaria Patient Daily Diary (UPDD), originally developed on paper, is a measure of key symptoms of chronic idiopathic urticaria (CIU). The development of the electronic version (eUPDD) involved moderate modifications to the appearance of the paper version. OBJECTIVE: This study assessed the measurement equivalence of the electronic and paper versions of the UPDD in a sample of patients with CIU. METHODS: This was a cross-over study of patients with moderate-severe CIU refractory to H1 antihistamines. Patients were randomized to either the eUPDD followed by the paper UPDD or vice versa. The UPDD includes morning and evening questions; both sets were administered together in this study. An hour-long filler task was given between paper and electronic administrations. Patients with stable symptoms between the two assessments were included in the analyses. Cohen's kappa coefficients and intraclass correlation coefficients (ICC) were computed as applicable to assess equivalence. RESULTS: A total of 91 patients participated (mean age 43 years, 79.1 % female). Symptoms were stable between assessments for 67-74 (74-81 %) patients (varied by symptom). Kappa coefficients ranged from 0.82 to 1.00 for the individual UPDD items. For the Urticaria Activity Score (the sum of the 'itch severity' and 'number of hives' item scores) the ICC was 0.90 for the morning (Wilcoxon p = 0.331) and 0.95 for the evening (Wilcoxon p = 0.836). CONCLUSIONS: All test-retest statistics in this study were well above the accepted threshold, indicating excellent agreement between the two administration methods. Findings support the measurement equivalence of the electronic and paper versions of the UPDD to measure CIU symptoms.

Validation of the Cardiovascular Limitations and Symptoms Profile (CLASP) in chronic stable angina.

PURPOSE: This study aimed to establish the reliability, validity, and sensitivity of the Cardiovascular Limitations and Symptoms Profile (CLASP) in a group of patients with chronic stable angina. METHODS: After 226 patients with angina had been recruited, they were randomly allocated to one of three groups: a 10-week hospital-based angina management program (n = 75; men = 56; age = 60 +/- 8 years), routine care (n = 74; men = 52; age = 61 +/- 7 years), and exercise therapy (n = 77; men = 60; age = 60 +/- 7 years). All the patients were assessed with CLASP on two occasions: at baseline and at 10 weeks. The Sickness Impact Profile (SIP), the Hospital Anxiety and Depression Scale (HADS), and the Sleep Problems Questionnaire (SPQ) also were administered at the same time. RESULTS: Significant positive correlations between the actual number of angina episodes and the CLASP angina subscale scores (r =.60, P <.001) were observed. The CLASP subscale scores for shortness of breath (r = -.36; P <.001) and ankle swelling (r = -.24; P <.001) were significantly correlated with the total treadmill time. The CLASP tiredness subscale score showed a significant positive correlation with the SPQ score (r =.48; P <.001). The CLASP subscale scores were significantly correlated with their corresponding SIP subscale scores: the tiredness score with the sleep and rest score (r =.49; P <.001), the social and leisure score with the recreation and pastimes score (r =.41; P <.001), the home score with the home management score (r =.45; P <.001), and the mobility score with the mobility (r =.37; P <.001) and total treadmill time scores (r = -.49; P <.001). CONCLUSIONS: The findings show CLASP to be a reliable, valid, sensitive measure of health-related quality of life in patients with chronic stable angina. Before it can be recommended for all patients with heart disorders, similar data will be required from other diagnostic groups such as patients with heart failure or those who have sustained an acute myocardial infarction.