Browsing by Subject "Patient-reported outcome"

Now showing 1 - 4 of 4
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    Impaired insight in schizophrenia: impact on patient-reported and physician-reported outcome measures in a randomized controlled trial
    (BMC, 2022-08-28) Lysaker, Paul H.; Weiden, Peter J.; Sun, Xiaowu; O’Sullivan, Amy K.; McEvoy, Joseph P.; Psychiatry, School of Medicine
    Background: Impaired insight poses a challenge in the treatment of patients with schizophrenia because of its potential to jeopardize therapeutic engagement and medication adherence. This study explored how insight impairment, graded from none to extreme, is related to patient-reported mental health status, depression, and neurocognition in schizophrenia. Methods: In a post hoc analysis of the Clinical Antipsychotic Trials of Intervention Effectiveness (CATIE) study (NCT00014001), insight was measured using the Positive and Negative Syndrome Scale (PANSS) Item G12 (lack of insight). Additional assessments for this analysis included the 12-Item Short-Form Health Survey (SF-12) Mental Component Summary (MCS), physician- and patient-reported Clinical Global Impression-Severity (CGI-S), MATRICS Consensus Cognitive Battery, and Calgary Depression Scale for Schizophrenia. Relationships between patient-reported outcomes and PANSS total and Item G12 ratings were evaluated. Results: Among 1431 CATIE study participants in this analysis, increasingly impaired insight at baseline was significantly associated with better patient-reported quality of life (QoL), lower baseline depression, and greater divergence between physician- and patient-reported illness severity. Patients with more severely impaired insight reported milder illness compared with physician reports, particularly those with moderate-severe to extreme impairment (PANSS Item G12 rating ≥ 5), approximately 10% (138/1431) of CATIE participants. For the 90% of patients with PANSS Item G12 ratings < 5, patient-reported QoL decreased with increasing symptoms. SF-12 MCS scores were linearly related to baseline PANSS total score only in patients with PANSS total score < 90 (moderately ill or better), and better symptom scores were associated with higher QoL. No significant relationship between insight and neurocognition was observed. Conclusions: In the small subgroup (10%) of CATIE study patients with schizophrenia and PANSS Item G12 ratings ≥5, moderate-severe-severe/extreme insight impairment was associated with significantly more positive perception of QoL and illness severity by the patient versus the treating physician. This was not observed in the remaining 90% of patients with normal to moderately impaired insight, suggesting that poor insight as a threat to the validity of self-report is uncommon.
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    Linguistic, Content and Face Validity of the Swedish Version of a Quality-of-Life Assessment for Children, Teenagers and Adults with Spina Bifida
    (MDPI, 2024-05-15) Dellenmark-Blom, Michaela; Andersson, Marie; Szymanski, Konrad M.; Andréasson, Charlotta Levén; Arnell, Magdalena Vu Minh; Sjöström, Sofia; Abrahamsson, Kate; Pediatrics, School of Medicine
    Spina bifida includes a spectrum of different neural tube defects. Myelomeningocele is the most serious type and is associated with a risk of paralysis and sensory dysfunction below the affected level, bladder/bowel dysfunction, brain dysmorphology, and impaired health-related quality of life (HRQoL). The aim of this study was to describe the establishment of linguistic, content and face validity of the Swedish version of a Quality-of-Life Assessment for children (QUALAS-C, n = 10 items), teenagers (QUALAS-T, n = 10 items) and adults with spina bifida (QUALAS-A, n = 15 items) based on the original US English versions. The process included close collaboration with the original instrument developer and complied with international standards on patient-reported outcome measurements. The procedure includes forward translation, expert and patient/parent review and reconciliation, back translation, back translation review and cognitive debriefing interviews with 16 people with spina bifida aged 8 to 33, providing them with the possibility of evaluating the clarity, adequacy, and comprehensiveness of QUALAS-C, QUALAS-T and QUALAS-A, respectively. The interviews lasted a median of 15 min (range 8–16) for QUALAS-C, 10 min (range 9–15) for QUALAS-T and 24 min (range 9–38) for QUALAS-A. Four main issues/topics needed attention and discussion after both the forward and back translation. Following the back translation review, all issues were resolved. The patient feedback revealed recognition of the HRQoL issues included in QUALAS, and also difficulties in understanding some questions. After the patients’ evaluation, four items were reworded for clarity. No study participant reported a wish to add to or remove questions from QUALAS. Hence, the Swedish versions of QUALAS became conceptually equivalent to the original US English versions and achieved linguistic, content and face validity. While empowering the voices of people with spina bifida, these results also enable their HRQoL to be properly assessed in research and clinical care in Sweden and in international studies.
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    The Patient Mania Questionnaire (PMQ-9): a Brief Scale for Assessing and Monitoring Manic Symptoms
    (Springer, 2022) Cerimele, Joseph M.; Russo, Joan; Bauer, Amy M.; Hawrilenko, Matt; Pyne, Jeffrey M.; Dalack, Gregory W.; Kroenke, Kurt; Unützer, Jürgen; Fortney, John C.; Medicine, School of Medicine
    Background: Measurement-based care is an effective clinical strategy underutilized for bipolar disorder partly due to lacking a widely adopted patient-reported manic symptom measure. Objective: To report development and psychometric properties of a brief patient-reported manic symptom measure. Design: Secondary analysis of data collected in a randomized effectiveness trial comparing two treatments for 1004 primary care patients screening positive for bipolar disorder and/or PTSD. Participants: Two analytic samples included 114 participants with varied diagnoses and test-retest data, and 179 participants with psychiatrist-diagnosed bipolar disorder who had two or more assessments with the nine-item Patient Mania Questionnaire-9 [PMQ-9]). Main measures: Internal and test-retest reliability, concurrent validity, and sensitivity to change were assessed. Minimally important difference (MID) was estimated by standard error of measurement (SEM) and by standard deviation (SD) effect sizes. Key results: The PMQ-9 had high internal reliability (Cronbach's alpha = 0.88) and test-retest reliability (0.85). Concurrent validity correlation with manic symptom measures was high for the Internal State Scale-Activation Subscale (0.70; p<0.0001), and lower for the Altman Mania Rating Scale (0.26; p=0.007). Longitudinally, PMQ-9 was completed at 1511 clinical encounters in 179 patients with bipolar disorder. Mean PMQ-9 score at first and last encounters was 14.5 (SD 6.5) and 10.1 (SD 7.0), a 27% decrease in mean score during treatment, suggesting sensitivity to change. A point estimate of the MID was approximately 3 points (range of 2-4). Conclusions: The PMQ-9 demonstrated excellent test-retest reliability, concurrent validity, internal consistency, and sensitivity to change and was widely used and acceptable to patients and clinicians in a pragmatic clinical trial. Combined with the Patient Health Questionnaire-9 (PHQ-9) measure of depressive symptoms this brief measure could inform measurement-based care for individuals with bipolar disorder in primary care and mental health care settings given its ease of administration and familiar self-report response format.
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    Tolerability of bevacizumab and chemotherapy in a phase 3 clinical trial with human epidermal growth factor receptor 2-negative breast cancer: A trajectory analysis of adverse events
    (Wiley, 2021) Ip, Edward H.; Saldana, Santiago; Miller, Kathy D.; Carlos, Ruth C.; Gareen, Ilana F.; Sparano, Joseph A.; Graham, Noah; Zhao, Fengmin; Lee, Ju-Whei; O’Connell, Nathaniel S.; Cella, David; Peipert, John D.; Gray, Robert J.; Wagner, Lynne I.; Medicine, School of Medicine
    Background: E5103 was a study designed to evaluate the efficacy and safety of bevacizumab. It was a negative trial for the end points of invasive disease-free survival and overall survival. The current work examines the tolerability of bevacizumab and other medication exposures with respect to clinical outcomes and patient-reported outcomes (PROs). Methods: Adverse events (AEs) collected from the Common Terminology Criteria for Adverse Events were summarized to form an AE profile at each treatment cycle. All-grade and high-grade events were separately analyzed. The change in the AE profile over the treatment cycle was delineated as distinct AE trajectory clusters. AE-related and any-reason early treatment discontinuations were treated as clinical outcome measures. PROs were measured with the Functional Assessment of Cancer Therapy-Breast + Lymphedema. The relationships between the AE trajectory and early treatment discontinuation as well as PROs were analyzed. Results: More than half of all AEs (57.5%) were low-grade. A cluster of patients with broad and mixed AE (all-grade) trajectory grades was significantly associated with any-reason early treatment discontinuation (odds ratio [OR], 2.87; P = .01) as well as AE-related discontinuation (OR, 4.14; P = .001). This cluster had the highest count of all-grade AEs per cycle in comparison with other clusters. Another cluster of patients with primary neuropathic AEs in their trajectories had poorer physical well-being in comparison with a trajectory of no or few AEs (P < .01). A high-grade AE trajectory did not predict discontinuations. Conclusions: A sustained and cumulative burden of across-the-board toxicities, which were not necessarily all recognized as high-grade AEs, contributed to early treatment discontinuation. Patients with neuropathic all-grade AEs may require additional attention for preventing deterioration in their physical well-being.