Browsing by Subject "Patient reported outcomes"

Now showing 1 - 5 of 5
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    Comorbidity, Physical Function, and Quality of Life in Older Adults with Acute Myeloid Leukemia
    (Springer, 2017-12) Storey, Susan; Gray, Tamryn Fowler; Bryant, Ashley Leak; School of Nursing
    Purpose of review: To describe the pathology, impact of comorbidities, functional limitations, symptoms, and quality of life (QOL) related to treatment of acute myeloid leukemia (AML) in older adults. Recent findings: AML is a rare aggressive hematologic disease that occurs most often in older adults. The prognosis for older patients with AML is markedly worse due to genetic mutations and patient characteristics such as comorbidities and functional limitations. Patient characteristics may influence treatment decisions, as well as impact symptoms, functional ability, health-related outcomes and (QOL). Summary: As the population continues to age, the number of people diagnosed with AML is expected to increase. Better management of comorbidities is imperative to improving QOL and other treatment related outcomes. Prospective, longitudinal and multi-site studies are warranted to further understand the interaction between these characteristics on symptoms, outcomes and QOL.
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    Development of the Adult PedsQL™ Neurofibromatosis Type 1 Module: Initial Feasibility, Reliability and Validity
    (Springer Nature, 2013-02-21) Nutakki, Kavitha; Hingtgen, Cynthia M.; Monahan, Patrick; Varni, James W.; Swigonski, Nancy L.; Pediatrics, School of Medicine
    Background: Neurofibromatosis type 1 (NF1) is a common autosomal dominant genetic disorder with significant impact on health-related quality of life (HRQOL). Research in understanding the pathogenetic mechanisms of neurofibroma development has led to the use of new clinical trials for the treatment of NF1. One of the most important outcomes of a trial is improvement in quality of life, however, no condition specific HRQOL instrument for NF1 exists. The objective of this study was to develop an NF1 HRQOL instrument as a module of PedsQL™ and to test for its initial feasibility, internal consistency reliability and validity in adults with NF1. Methods: The NF1 specific HRQOL instrument was developed using a standard method of PedsQL™ module development - literature review, focus group/semi-structured interviews, cognitive interviews and experts' review of initial draft, pilot testing and field testing. Field testing involved 134 adults with NF1. Feasibility was measured by the percentage of missing responses, internal consistency reliability was measured with Cronbach's alpha and validity was measured by the known-groups method. Results: Feasibility, measured by the percentage of missing responses was 4.8% for all subscales on the adult version of the NF1-specific instrument. Internal consistency reliability for the Total Score (alpha =0.97) and subscale reliabilities ranging from 0.72 to 0.96 were acceptable for group comparisons. The PedsQL™ NF1 module distinguished between NF1 adults with excellent to very good, good, and fair to poor health status. Conclusions: The results demonstrate the initial feasibility, reliability and validity of the PedsQL™ NF1 module in adult patients. The PedsQL™ NF1 Module can be used to understand the multidimensional nature of NF1 on the HRQOL patients with this disorder.
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    Health-related Quality of Life in a Prospective Study of Ultrasound to Detect Cystic Fibrosis-related Liver Disease in Children
    (Wiley, 2022) Schwarzenberg, Sarah Jane; Palermo, Joseph J.; Ye, Wen; Huang, Suiyuan; Magee, John C.; Alazraki, Adina; Freeman, A. Jay; Harned, Roger; Karmazyn, Boaz; Karnsakul, Wikrom; Leung, Daniel H.; Ling, Simon C.; Masand, Prakash; Molleston, Jean P.; Murray, Karen F.; Navarro, Oscar M.; Nicholas, Jennifer L.; Otto, Randolph K.; Paranjape, Shruti M.; Siegel, Marilyn J.; Stoll, Janis; Towbin, Alexander J.; Narkewicz, Michael R.; Alonso, Estella M.; CFLD NET; Pediatrics, School of Medicine
    Objectives: Cystic fibrosis liver disease (CFLD) begins early in life. Symptoms may be vague, mild, or nonexistent. Progressive liver injury may be associated with decrements in patient health before liver disease is clinically apparent. We examined Health-Related Quality of Life (HRQOL) in children enrolled in a multi-center study of CFLD to determine the impact of early CFLD on general and disease-specific QOL. Methods: Ultrasound (US) patterns of normal (NL), heterogeneous (HTG), homogeneous (HMG), or nodular (NOD) were assigned in a prospective manner to predict those at risk for advanced CFLD. Parents were informed of results. We assessed parent/child-reported (age ≥5 years) HRQOL by PedsQL 4.0 Generic Core and CF Questionnaire-revised (CFQ-R) prior to US and annually. HRQOL scores were compared by US pattern at baseline (prior to US), between baseline and 1 year and at 5 years. Multivariate analysis of variance (MANOVA) with Hotelling-Lawley trace tested for differences among US groups. Results: Prior to US, among 515 participants and their parents there was no evidence that HTG or NOD US was associated with reduced PedsQL/CFQ-R at baseline. Parents of NOD reported no change in PedsQL/CFQ-R over the next year. Child-report PedsQL/CFQ-R (95 NL, 20 NOD) showed improvement between baseline and year 5 for many scales, including Physical Function. Parents of HMG children reported improved CFQ-R scores related to weight. Conclusions: Early undiagnosed or pre-symptomatic liver disease had no impact on generic or disease-specific HRQoL, and HRQoL was remarkably stable in children with CF regardless of liver involvement.
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    Pain in adults with congenital heart disease - An international perspective
    (Elsevier, 2021) Leibold, Allison; Eichlera, Erin; Chung, Sukyung; Moons, Philip; Kovacs, Adrienne H.; Luyckx, Koen; Apers, Silke; Thomet, Corina; Budts, Werner; Enomoto, Junko; Sluman, Maayke A.; Wang, Jou-Kou; Jackson, Jamie L.; Khairy, Paul; Cook, Stephen C.; Chidambarathanu, Shanthi; Alday, Luis; Oechslin, Erwin; Eriksen, Katrine; Dellborg, Mikael; Berghammer, Malin; Johansson, Bengt; Mackie, Andrew S.; Menahem, Samuel; Caruana, Maryanne; Veldtman, Gruschen; Soufi, Alexandra; White, Kamila; Callus, Edward; Kutty, Shelby; Fernandes, Susan M.; APPROACH-IS consortium; International Society for Adult Congenital Heart Disease (ISACHD); Medicine, School of Medicine
    Background: Patients with adult congenital heart disease (ACHD) have many risk factors for chronic pain such as prior cardiac interventions and adult comorbidities. However, the prevalence of chronic pain has not been well described in this population. We sought to determine the prevalence of pain in a large international cohort of patients with ACHD. Methods: Data from the APPROACH-IS dataset was utilized for this study which includes 4028 patients with ACHD from 15 different countries. The prevalence of pain was assessed under the health status patient reported outcome domain utilizing the EuroQol-5D 3 level version tool. Multivariable logistic regression was used to assess differences across countries in pain, taking into account country-level random effects for clustering across observations within each country. Results: A total of 3832 patients with ACHD met the study criteria, median age 32 years [IQR 25, 42], 52.6% females. The prevalence of at least moderate pain was reported by 28.9% (95% CO 27.5 ​= ​30.3%) of participants. Pain was associated with country of origin, age, gender, background, education and marital status as well as several clinical variables including disease complexity, cardiac device presence, history of heart failure, psychiatric conditions and presence of other medical conditions. Those with pain had lower levels of perceived health and a lower quality of life score. Conclusion: Pain in patients with ACHD is common, impacting nearly one-third of patients. Given the far reaching implications of pain in patients with ACHD, further study of pain characteristics and treatment management appear warranted.
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    Treatment satisfaction and time in range after 16 weeks of treatment with iGlarLixi in insulin-naive adults with suboptimally controlled type 2 diabetes
    (Wiley, 2025) Shah, Viral N.; Dex, Terry; Meneghini, Luigi; Rodrigues, Amélie; Polonsky, William H.; Medicine, School of Medicine
    Aims: In Soli-CGM, treatment with iGlarLixi (insulin glargine 100 U/mL and lixisenatide 33 μg/mL) in insulin-naive adults with suboptimally controlled type 2 diabetes (T2D; haemoglobin A1c 9%-13% on ≥2 oral antihyperglycaemic agents (OADs) ± glucagon-like peptide-1 receptor agonist (GLP-1 RA) therapy) increased time in range (TIR; primary endpoint) from 26.4% at baseline to 52.7% at Week 16. This exploratory analysis examined the impact of treatment with iGlarLixi on patient-reported treatment satisfaction. Materials and methods: Soli-CGM was a single-arm, 16-week, multicentre, interventional, open-label, phase 4 study using blinded continuous glucose monitoring (CGM; FreeStyle Libre Pro) to assess glycaemic metrics (N = 124). CGM data were collected for a 2-week period before initiation of iGlarLixi, and after treatment with iGlarLixi (Weeks 14-16). Treatment satisfaction was assessed using the Diabetes Medication Treatment Satisfaction Tool (DM-SAT, which comprises four domains: well-being, medical control, lifestyle and convenience), at baseline and end-of-treatment. Association of TIR and overall satisfaction (sum of all items) was also assessed. Results: Overall, 118 (95.9%) and 107 (87.0%) participants completed the DM-SAT at baseline and Week 16, respectively. Mean overall score increased by 0.18, from 0.59 (baseline) to 0.78 (Week 16). A trend in improvement in score was observed in all domains. Improvement in TIR had a positive, but weak, trend of association with improvement in overall treatment satisfaction (mean r = 0.14). Conclusions: In people with T2D suboptimally controlled on ≥2 OADs ± GLP-1 RA, 16 weeks' treatment with iGlarLixi resulted in a trend of improvement in treatment satisfaction.