Quality Of Patient-reported Outcomes Research Articles

EHealth Solutions for Therapy Management in Oncology

In an outpatient setting, some challenges of cancer treatment include continuous patient–physician communication, lack of adherence, potential side effects and their impact on quality of life and other patient-reported outcomes. These challenges in the support of disease management can be overcome by the introduction of eHealth applications. Though the market of eHealth applications is fast growing, many applications lack evidence regarding their effectiveness, safety and utility. Only few prospective randomized trials have been conducted, so far. Results of these studies univocally show a gain in health-related quality of life, in the examined eHealth applications. It remains unclear if procedural and cost efficacy are affected by eHealth applications. The upcoming PreCycle study will be the largest randomized eHealth study in oncology.

A Systematic Review of Outcome Measures Assessing Disability Following Upper Extremity Trauma.

Objectives:To define upper extremity outcome measures focusing on trauma and level of initial psychometric evaluation and to assess methodological quality of relevant patient-reported outcome (PRO) measures.Data Sources:A broad search strategy using PubMed, OVID, CINAHL, and PsycINFO was deployed and reported using PRISMA (PROSPERO: CRD42016046243).Study Selection, Extraction, Synthesis:PRO measures involving orthopedic trauma in their original development were selected and original publications assessed, including psychometric evaluations. Extraction, synthesis, and quality assessment were performed using COSMIN.Results:Of 144 upper extremity outcome measures, the majority were designed for the shoulder, wrist, and hand; 20% (n = 29/144) involved trauma conditions in their initial development, PRO measurements, and psychometric evaluation on introduction. Methodological quality was highly variable.Conclusion:A few PRO measures were originally designed for use in upper extremity trauma. Methodological quality and psychometric evaluation need to improve. This review aims to highlight strengths and weaknesses and guide decision making in this field.

Evaluation of the geriatric co-management for patients with fragility fractures of the proximal femur (Geriatric Fracture Centre (GFC) concept): protocol for a prospective multicentre cohort study

IntroductionTreatment of fractures in the elderly population is a clinical challenge due partly to the presence of comorbidities. In a Geriatric Fracture Centre (GFC), patients are co-managed by a geriatrician...

Routine inclusion of long-term functional and patient-reported outcomes into trauma registries: The FORTE project.

The National Academies of Sciences, Engineering, and Medicine (formerly the Institute of Medicine) recently recommended inclusion of postdischarge health-related quality of life (HRQoL) and patient-reported outcomes (PROs) metrics to benchmark the quality of trauma care. Currently, these measures are not routinely collected at most trauma centers. We sought to determine the feasibility and value of adding such long-term outcome measures to trauma registries. As part of the FORTE (Functional Outcomes and Recovery after Trauma Emergencies) project, we included patients with an Injury Severity Score of 9 or greater, admitted to the Brigham and Women's Hospital in Boston, MA, who were identified retrospectively using the institutional trauma registry and contacted 6 or 12 months after injury to participate in a telephone survey evaluating HRQoL (Short Form 12 [SF-12]), PROs (Trauma Quality of Life), posttraumatic stress disorder, return to work, residential status, and health care utilization. Data were collected for 171 of 394 eligible patients: 85/189 (45%) at 6 months and 86/205 (42%) at 12 months; 25%/29% (6/12 months) patients could not be contacted, 15%/16% (6/12 months) declined to participate, and 15%/13% (6/12 months) were interested in participating at another time but were not reached again. Approximately 20% patients screened positive for posttraumatic stress disorder, and half had not yet returned to work. There were significant reductions in SF-12 physical composite scores relative to population norms (mean, 50 [SD, 10]) at 6 months (mean, 44; 95% confidence interval [CI], 41-47) and 12 months (45; 95% CI, 42-47); no difference was noted in the SF-12 mental composite scores (6 months: 51 [95% CI, 48-54]; 12 months: 50 [95% CI, 46-53]). Trauma patients reported considerable impairment 6 and 12 months after injury. Routine collection of PROs and HRQoL provides important data regarding trauma outcomes beyond mortality and will enable the development of quality improvement metrics that better reflect patients' postinjury experiences. Improved and alternate methods for collection of these data need to be developed to enhance response rates before widespread adoption across trauma centers in the United States. Prognostic/epidemiologic, level II; Therapeutic, level III.

What are patients’ biggest concerns? A patient reported outcome case-management system.

6572 Background: Cancer patients (pts) receive complex care that may cause physical, emotional, & financial sequelae. Current practice limits clinician time to address patient concerns. The Patient Reported Outcome Quality Of Life (PROQOL) system was developed as a self-reported electronic questionnaire to assess symptoms, QOL and provide data about bothersome issues. Our aim was to determine if the PROQOL system improves QOL without negatively impacting routine clinic workflow. Methods: Eligible pts had multiple myeloma, amyloidosis, head & neck or gynecologic cancer seen in Hematology/Oncology clinics at Mayo Clinic, and were stratified by stem cell transplant and active treatment status. Pts were randomized 2:1 to PROQOL system or usual care. PROQOL system was offered prior to every visit. Pts select from various categories about their single biggest concern, and receive a printed list of actionable resources based on selected concern. Clinicians also receive the PROQOL results to review with pts. Providers and pts randomized to PROQOL completed a “was it worth it survey” (WIWI). An 8 item Linear Analogue Self-Assessment was used to assess QOL. The study was powered to detect a 0.5 standard deviation difference in QOL between groups. Herein we report the planned results of first 6 months. Results: Among the first 118 pts accrued, 55% were female, median age was 63 (32-86), & 93% were on therapy. Median time from diagnosis to PROQOL was 26 months. The PROQOL system took 3.6 minutes to complete. Baseline median QOL (range) was 7 (3-10) for both groups. The most common PROQOL issue selected was “cancer & diagnosis” (36.5%) followed by “physical health” (35.3%). Specific concerns were related to treatment plan, prognosis, fatigue, sleep, and neuropathy. WIWI showed: 71% of pts thought it was worthwhile, 83% would participate again, 80% would recommend it, & 65% used resources provided. 80% of providers reported the PROQOL did not interfere with care & 75% believed the pt wellbeing may improve. Conclusions: Notably treatment plan & prognosis remain pts' greatest concern despite being over 2 year from diagnosis.The PROQOL system demonstrates integrated PRO reporting in clinic is quick & worthwhile to pts & providers.

Stigma perceptions in patients with eosinophilic gastrointestinal disorders.

This study aims to evaluate the presence of perceived stigma in people diagnosed (self-reported) with an eosinophilic gastrointestinal disorder and examine the relationship to the patient's health-related quality of life and additional psychosocial patient-reported outcomes. One hundred forty-nine patients diagnosed for a minimum of 6 months participated in the study. Eligible participants completed questionnaires to assess perceived stigma, psychological functioning, and health-related quality of life. Perceived stigma was moderately associated with a decrease in total health-related quality of life and perceived treatment efficacy. Additionally, greater perceived stigma was positively correlated with an increase in anxiety, depression, and healthcare utilization. The study demonstrates the influence of perceived stigma on several aspects of health-related quality of life in patients with these illnesses. As such, it is important for health professionals to be aware of stigma in patients diagnosed with an eosinophilic gastrointestinal disorder.

Prospective assessment of oral mucositis and its impact on quality of life and patient-reported outcomes during radiotherapy for head and neck cancer.

Oral mucositis (OM) is a common acute side effect during radiotherapy treatments for head and neck cancer (HNC), with a potential impact on patient's compliance to therapy, quality of life (QoL) and clinical outcomes. Its timely and appropriate management is of paramount importance. Several quantitative scoring scales are available to properly assess OM and its influence on patient-reported outcomes (PROs) and QoL. We prospectively assessed OM in a cohort of HNC patients submitted to radiation using the Oral Mucositis Assessment Scale (OMAS), while its impact on PROs and QoL was evaluated employing the Oral Mucositis Weekly Questionnaire-Head and Neck Cancer (OMWQ-HN) and the Functional Assessment of Cancer Therapy-Head and Neck Cancer (FACT-HN). Evaluation of OMAS scores highlighted a progressive increase in OM during treatment and a partial recovery after the end of radiation. These trends were correlated to PROs and QoL as evaluated with OMWQ-HN and FACT-HN questionnaires. In the present study, we provided a quantitative assessment of OM, PROs and QoL in HNC patient undergoing radiotherapy, potentially useful for future comparison.

Key issues affecting quality of life and patient-reported outcomes in prostate cancer: an analysis conducted in 2128 patients with initial psychometric assessment of the prostate cancer symptom scale (PCSS)

ObjectiveEvidence-based quality of life (QL) questionnaires require the identification of issues of importance to patients. The primary aim of this study was to inform providers on patient-expressed issues while enhancing...

Feasibility of a Telehealth Educational Program on Self-Management of Pain and Fatigue in Adult Cancer Patients

ContextPain and fatigue are common symptoms among cancer patients and often lead to substantial distress. Innovative self-management programs for pain and fatigue are needed. ObjectivesThe primary objective was to assess the feasibility of a telehealth pain and fatigue self-management program among adult cancer patients. Secondary objectives included assessment of differences in patient characteristics, recruitment, and retention of patients based on two screening strategies: 1) navigator-collected, patient-reported pain or fatigue and 2) in-clinic, physician-identified pain or fatigue. MethodsThis prospective, nonrandomized, pre-post evaluation assessed feasibility, which was defined as 50% of eligible patients choosing to participate and completing the intervention. Patient demographics and patient-reported outcomes (patient activation, distress, symptoms, and quality of life) were collected at baseline and study completion. Differences in baseline characteristics were compared between cohorts and for patients who did vs. did not graduate from the program. ResultsThe program did not meet feasibility requirements because of only 34% of eligible patients choosing to participate. However, 50% of patients starting the program graduated. Differences in baseline characteristics and retention rates were noted by recruitment strategy. At baseline, 27.3% of navigated patients were at the highest activation level compared with 7.1% in the physician-referred, non-navigated patients (P = 0.17); more than 15% of non-completers were at the lowest activation level compared with 9% of completers (P = 0.85). ConclusionTelehealth self-management program for pain and fatigue may be better accepted among selected segments of cancer patients. Larger scale studies are needed to assess the efficacy of this program in a more selective activated population.

Research Priorities for Optimal Use of Patient-reported Outcomes in Quality and Outcome Improvement for Total Knee Arthroplasty.

The national Functional and Outcomes Research for Comparative Effectiveness in Total Joint Replacement program routinely collects pre- and postoperative patient-reported outcomes for clinicians to use when making individual patient treatment decisions and monitoring aggregate quality of care and outcomes. When the pre-post gains in pain or function at one site vary from the national norms, the next question is, "how do we improve?" This paper will use the traditional quality management's framework of inputs (patients), processes (clinical care), and outcomes to outline priority research questions to learn how clinicians, hospital managers, and patients can interpret patient-reported outcomes to improve total knee arthroplasty care and outcomes. In summary, research should identify best practices to minimize variation in a patient's health status before surgery, tailor peri-total knee arthroplasty care pathways to match individual patient risks to optimize safe care, and implement patient-reported measures to document optimal outcomes.

Patient-Reported Outcomes of Periacetabular Osteotomy from the Prospective ANCHOR Cohort Study.

Current literature describing the periacetabular osteotomy (PAO) is mostly limited to retrospective case series. Larger, prospective cohort studies are needed to provide better clinical evidence regarding this procedure. The goals of the current study were to (1) report minimum 2-year patient-reported outcomes (pain, hip function, activity, overall health, and quality of life), (2) investigate preoperative clinical and disease characteristics as predictors of clinical outcomes, and (3) report the rate of early failures and reoperations in patients undergoing contemporary PAO surgery. A large, prospective, multicenter cohort of PAO procedures was established, and outcomes at a minimum of 2 years were analyzed. A total of 391 hips were included for analysis (79% of the patients were female, and the average patient age was 25.4 years). Patient-reported outcomes, conversion to total hip replacement, reoperations, and major complications were documented. Variables with a p value of ≤0.10 in the univariate linear regressions were included in the multivariate linear regression. The backward stepwise selection method was used to determine the final risk factors of clinical outcomes. Clinical outcome analysis demonstrated major clinically important improvements in pain, function, quality of life, overall health, and activity level. Increasing age and a body mass index status of overweight or obese were predictive of improved results for certain outcome metrics. Male sex and mild acetabular dysplasia were predictive of lesser improvements in certain outcome measures. Three (0.8%) of the hips underwent early conversion to total hip arthroplasty, 12 (3%) required reoperation, and 26 (7%) experienced a major complication. This large, prospective cohort study demonstrated the clinical success of contemporary PAO surgery for the treatment of symptomatic acetabular dysplasia. Patient and disease characteristics demonstrated predictive value that should be considered in surgical decision-making. Therapeutic Level IV. See Instructions for Authors for a complete description of levels of evidence.

The Prognostic Value for Survival of Patient-Reported Outcomes in Hematological Malignancies: A Systematic Review

Introduction: Patient-reported outcomes (PROs) are now critical to more comprehensively evaluate treatment outcomes in oncology. There is convincing evidence indicating that, similarly to traditional biomarkers, PROs may also predict survival outcomes. This systematic review examined state of the art literature on the prognostic value of PROs for survival in hematological malignancies.Methods: We searched PubMed and Google Scholar from 2000-2016 for studies assessing the predictive relationship between PROs and overall survival (OS) in Acute Myelogenous Leukemia (AML), Chronic Lymphocytic Lymphoma (CLL), Diffuse Large B-Cell Lymphoma (DLBCL), indolent Non-Hodgkin's Lymphoma (iNHL), Follicular Lymphoma (FL), marginal Zone Lymphoma (mZL), Myelodysplastic Syndrome (MDS), and Multiple Myeloma (MM). Search terms included but were not limited to: quality of life, physical functioning, predictive/prognostic, survival, patient-reported outcomes, and fatigue. Studies were excluded if they did not use multivariate analyses, did not examine OS as an endpoint, were case reports, did not report results for PROs, were reported in an abstract only, or used only clinician-reported outcomes as a predictor. A pre-defined data extraction form (DEF) was used to assess the following data from each article: disease, sample size, demographics, type of PRO measures included, clinical parameters included as predictors, type of analysis, and outcomes assessed.Results: 223 articles were reviewed of which 13 met the inclusion criteria (6 AML/MDS, 3 MM, 2 aggressive lymphomas, 1 mixed heme diagnoses, and 1 DLBCL). PROs in these studies were captured with the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire - Core30 (EORTC QLQ-C30) or the EORTC myeloma module 24 item (MY24), a health-related quality of life questionnaire for patients with myelodysplastic syndromes (QOL-E), Functional Assessment of Cancer Treatment (FACT) or Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F), and the MD Anderson Symptom Inventory (MDASI). PROs that were independently associated with OS in multivariate analyses from the different studies were: physical function, fatigue, pain, difficulty with strenuous activity, and impaired physical performance in AML patients; physical function and fatigue in MM; fatigue in MDS; global/total QOL and functional well-being in aggressive lymphomas; drowsiness in a mixed group of heme diagnoses; and global QOL, function, and symptoms/signs in DLBCL (see Table). Baseline PROs were obtained following diagnosis of de novo or relapsed disease in 3 studies, within 6 months of registration in 1 study, and prior to any treatment in 6 studies.Conclusions: PROs related to physical functioning, fatigue, and performance were the most common predictors of OS in hematological malignancies and remained significant indicators even after accounting for clinical variables. This review highlights the prognostic value of PROs in both newly diagnosed and relapsed patients, and argues for their use in clinical decision-making and risk assessment at the time of diagnosis and pre-treatment. It also raises the question of how baseline PROs should be used in assessing new treatments within clinical trials in order to identify patients who may respond better to treatments or be in need of pre-treatment adjuvant therapy. [Display omitted] DisclosuresCampinha-Bacote:Genentech, Inc.: Employment. Trask:Genentech, Inc.: Employment, Equity Ownership. Efficace:Lundbeck: Research Funding; Seattle Genetics: Consultancy; Bristol Myers Squibb: Consultancy; TEVA: Consultancy, Research Funding.

An evaluation of health-related quality of life in patients with systemic lupus erythematosus using PROMIS and Neuro-QoL.

Systemic lupus erythematosus (SLE) is a multi-organ chronic autoimmune disease that can negatively affect patients' health-related quality of life (HRQOL). This study evaluated HRQOL of SLE patients using questionnaires from the Patient-Reported Outcomes Measurement Information System (PROMIS) and Quality of Life in Neurological Disorders (Neuro-QoL). Individuals with SLE completed an online survey consisting of the PROMIS-29 health profile, PROMIS Psychosocial Illness Impact-Negative, and Neuro-QoL Applied Cognition. PROMIS and Neuro-QoL scores have a mean of 50 in the US general population. Patients self-rated SLE disease severity as negligible, mild, moderate, or severe. Of the 333 participants (mean age 45years; 92% female; 26% Black; mean SLE disease duration 12years, 56% with SLE disease severity as moderate or severe), mean HRQOL scores were worse than those of the general population by ≥0.5 SD with the greatest deficits observed in the domains of fatigue, applied cognition, psychosocial illness impact-negative, pain interference, and physical function. Greater SLE disease severity was associated with worse mean HRQOL scores (all p<0.05). Pain severity was also associated with worse HRQOL scores on all domains (p<0.05) except for satisfaction with social role. Test-retest reliability exceeded 0.70 for all PROMIS and Neuro-QoL scores. PROMIS-29 and Neuro-QoL are valid tools to assess HRQOL in patients with SLE. These patients reported substantial deficits that correlated with their SLE disease severity, with pain being an important independent contributor. These deficits should be monitored in SLE patients during their routine clinical care and evaluated when investigating new therapies.

Living well after breast cancer randomized controlled trial protocol: evaluating a telephone-delivered weight loss intervention versus usual care in women following treatment for breast cancer.

BackgroundObesity, physical inactivity and poor diet quality have been associated with increased risk of breast cancer-specific and all-cause mortality as well as treatment-related side-effects in breast cancer survivors. Weight loss intervention trials in breast cancer survivors have shown that weight loss is safe and achievable; however, few studies have examined the benefits of such interventions on a broad range of outcomes and few have examined factors important to translation (e.g. feasible delivery method for scaling up, assessment of sustained changes, cost-effectiveness). The Living Well after Breast Cancer randomized controlled trial aims to evaluate a 12-month telephone-delivered weight loss intervention (versus usual care) on weight change and a range of secondary outcomes including cost-effectiveness.Methods/designWomen (18–75 years; body mass index 25–45 kg/m2) diagnosed with stage I-III breast cancer in the previous 2 years are recruited from public and private hospitals and through the state-based cancer registry (target n = 156). Following baseline assessment, participants are randomized 1:1 to either a 12-month telephone-delivered weight loss intervention (targeting diet and physical activity) or usual care. Data are collected at baseline, 6-months (mid-intervention), 12-months (end-of-intervention) and 18-months (maintenance). The primary outcome is change in weight at 12-months. Secondary outcomes are changes in body composition, bone mineral density, cardio-metabolic and cancer-related biomarkers, metabolic health and chronic disease risk, physical function, patient-reported outcomes (quality of life, fatigue, menopausal symptoms, body image, fear of cancer recurrence) and behaviors (dietary intake, physical activity, sitting time). Data collected at 18-months will be used to assess whether outcomes achieved at end-of-intervention are sustained six months after intervention completion. Cost-effectiveness will be assessed, as will mediators and moderators of intervention effects.DiscussionThis trial will provide evidence needed to inform the wide-scale provision of weight loss, physical activity and dietary interventions as part of routine survivorship care for breast cancer survivors.Trial registrationAustralian and New Zealand Clinical Trial Registry (ANZCTR) - ACTRN12612000997853 (Registered 18 September 2012).Electronic supplementary materialThe online version of this article (doi:10.1186/s12885-016-2858-0) contains supplementary material, which is available to authorized users.

Efficacy and safety of the glycine transporter type-1 inhibitor AMG 747 for the treatment of negative symptoms associated with schizophrenia

ObjectiveTo determine the safety and efficacy of AMG 747, an oral inhibitor of glycine transporter type-1 (GlyT1), as an add-on to antipsychotic therapy in clinically stable people with schizophrenia with enduring negative symptoms. MethodAnalysis of pooled data from two phase 2 studies. Adults diagnosed with schizophrenia stabilized on antipsychotic medication randomized (2:2:2:3) to orally receive daily AMG 747 (5mg, 15mg, or 40mg) or placebo. Primary endpoint was Negative Symptom Assessment (NSA)-16 total score change from baseline to week 12. ResultsStudies were terminated early after a report of Stevens-Johnson Syndrome/Toxic Epidermal Necrolysis (SJS/TEN) in one participant (40-mg AMG 747). At termination, 232 participants had enrolled and 153 completed 12weeks of treatment. At week 12, change from baseline NSA-16 total score showed no differences between groups. Mean decrease in Positive and Negative Syndrome Scale (PANSS) Negative Symptom Factor Score (NSFS) and NSA-16 global score were greater with 15-mg AMG 747 than placebo (p<0.05). Changes in PANSS-Positive Symptom Factor Scale were not significantly different for any group. Changes in patient-reported outcomes (Sheehan Disability Scale and Quality of Life Enjoyment and Satisfaction Questionnaire) showed trends consistent with greater efficacy of 15-mg AMG 747 compared with placebo (p≤0.1). Adverse event rates were similar among all groups, with no clear differences observed. ConclusionsSignificant treatment effects of 15-mg AMG 747, but not higher or lower doses, were observed on secondary endpoints but not on the primary outcome. These results replicate previous reports of an inverted-U dose response curve and suggest further evaluation of GlyT1 inhibitors in schizophrenia negative symptoms is warranted. Trial registrationClinicaltrials.govNCT01568216 (https://clinicaltrials.gov/ct2/show/NCT01568216) and NCT01568229 (https://clinicaltrials.gov/ct2/show/NCT01568229?term=NCT01568229&rank=1); EudraCT number 2011-004844-23 and 2011-004845-42

1506TiP - Quality of life, efficacy, and patient-reported outcome with NEPA as antiemetic prophylaxis in patients receiving highly or moderately emetogenic chemotherapy

1506TiP - Quality of life, efficacy, and patient-reported outcome with NEPA as antiemetic prophylaxis in patients receiving highly or moderately emetogenic chemotherapy

Quality of care received and patient-reported regret in prostate cancer: Analysis of a population-based prospective cohort.

Meeting quality of care standards in oncology is recognized as important by physicians, professional organizations, and payers. Data from a population-based cohort of patients with prostate cancer were used to examine whether receipt of care was consistent with published consensus metrics and whether receiving high-quality care was associated with less patient-reported treatment decisional regret. Patients with incident prostate cancer were enrolled in collaboration with the North Carolina Central Cancer Registry, with an oversampling of minority patients. Medical record abstraction was used to determine whether participants received high-quality care based on 5 standards: 1) discussion of all treatment options; 2) complete workup (prostate-specific antigen, Gleason grade, and clinical stage); 3) low-risk participants did not undergo a bone scan; 4) high-risk participants treated with radiotherapy (RT) received androgen deprivation therapy; and 5) participants treated with RT received conformal or intensity-modulated RT. Treatment decisional regret was assessed using a validated instrument. A total of 804 participants were analyzed. Overall, 66% of African American and 73% of white participants received care that met all standards (P = .03); this racial difference was confirmed by multivariable analysis. Care that included "discussion of all treatment options" was found to be associated with less patient-reported regret on univariable analysis (P = .03) and multivariable analysis (odds ratio, 0.59; 95% confidence interval, 0.37-0.95). The majority of participants received high-quality care, but racial disparity existed. Participants who discussed all treatment options appeared to have less treatment decisional regret. To the authors' knowledge, this is the first study to demonstrate an association between a quality of care metric and patient-reported outcome. Cancer 2017;138-143. © 2016 American Cancer Society.

The International development of PROQOL-HCV: An instrument to assess the health-related quality of life of patients treated for Hepatitis C virus.

BackgroundHepatitis C virus (HCV) compromises Health-related Quality of Life (HRQL) with detriments to Physical, Mental and Social health domains. Treatment with interferon and ribavirin is associated with side effects which further impair HRQL. New treatments appear potent, effective and tolerable. However, Patient Reported Outcomes instruments that capture the impact on HRQL for people with hepatitis C are largely non-specific and will be needed in the new treatment era. Therefore, we developed a conceptually valid multidimensional model of HCV-specific quality of life and pilot survey instrument, the Patient Reported Outcome Quality of Life survey for HCV (PROQOL-HCV).MethodsHCV patients from France (n = 30), Brazil (n = 20) and Australia (n = 20) were interviewed to investigate HCV-HRQL issues raised in the scientific literature and by treatment specialists. Interviews were recorded, transcribed and translated into English and French.ResultsFifteen content dimensions were derived from the qualitative analysis, refined and fitted to four domains: (1) Physical Health included: fatigue, pain, sleep, sexual impairment and physical activity; (2) Mental Health: psychological distress, psychosocial impact, and cognition; (3) Social Health: support, stigma, social activity, substance use; (4) Treatment: management, side effects, and fear of treatment failure. The impact of some dimensions extended beyond their primary domain including: physical activity, cognition, sleep, sexual impairment, and the three treatment dimensions. A bank of 300 items was constructed to reflect patient reports and, following expert review, reduced to a 72-item pilot questionnaire.ConclusionWe present a conceptually valid multidimensional model of HCV-specific quality of life and the pilot survey instrument, PROQOL-HCV. The model is widely inclusive of the experience of hepatitis C and the first to include the treatment dimension.

Health-related quality of life and patient-reported outcomes of ofatumumab plus chlorambucil versus chlorambucil monotherapy in the COMPLEMENT 1 trial of patients with previously untreated CLL

Background: Patients diagnosed with chronic lymphocytic leukemia (CLL) are usually elderly and frequently have a number of comorbidities. Health-related quality of life (HRQoL) for these patients is of utmost importance and should be taken into consideration when assessing new treatment options. The combination of ofatumumab with chlorambucil has shown longer progression-free survival compared with chlorambucil alone. In this study, we aim to assess how this treatment combination affects patients’ health-related quality of life and patient-reported symptoms.Material and methods: In this open-label phase III trial, patients with previously untreated CLL for whom fludarabine-based treatment was contra-indicated, were randomized 1:1 to receive oral chlorambucil (10 mg/m2) on Days 1–7 of a 28-day treatment cycle or to receive chlorambucil by this schedule plus intravenous ofatumumab (Cycle 1: 300 mg on Day 1 and 1000 mg on Day 8; subsequent cycles: 1000 mg Day 1) for 3–12 cycles. The EORTC QLQ-C30 and QLQ-CLL16 questionnaires were administered to patients before and during treatment, in follow-up and at the time of disease progression. The primary specified patient-reported outcomes were HRQoL and fatigue.Results: Patient-reported improvements from baseline in Global Health Status (GHS)/HRQoL scores and fatigue scores were recorded during treatment with both chlorambucil monotherapy and ofatumumab in combination with chlorambucil. There were no significant differences between the two treatment arms for GHS/HRQoL (p = 0.667) or fatigue (p = 0.103). Following treatment, numerical improvements to GHS/HRQoL and fatigue scores were reported, with no significant differences between the two treatment arms.Conclusion: Small but detectable improvements in patients’ quality of life were reported as a result of treatment. The addition of ofatumumab to chlorambucil did not negatively impact HRQoL. Quality of life was maintained in the months following treatment.

The fast route to evidence development for value in healthcare

Over the past 30 years, our greatest accomplishments in advancing understanding about the safety, effectiveness and value of healthcare interventions have been achieved through the availability and accessibility of health insurance claims for large populations and, more recently, access to pockets of electronic health records that can be linked to information on expenditures, outcomes and patient experience. Although useful, this data lacks clinical detail and patient relevance. Going forward, we need qualified data networks that understand their data, have a research administration in place to evaluate requests, obtain institutional review and transfer data – all on a timely basis. The mecca of health information will be networks with comprehensive electronic health records that are able to collect and link supplementary data collected from clinicians, tissue banks, etc., and/or directly from patients, including information on quality of life and other patient-reported outcomes. A combination of data collected from clinicians, patients and health systems will provide details about treatment use, patient characteristics and health outcomes that are not consistently available in a patient’s medical record, including whether prescriptions were actually filled, and if patients were hospitalized or had other costly healthcare that may not have been reported to their network doctors. We need a simple process to serve as a gateway to identify qualified research partners, the types of data each has access to, and how to work with them. The best partners will have established processes for contracting, prompt and informed institutional review, and data integration including supplementation from patients, clinicians and others, as needed, to support evidence generation that is more nuanced than simply choosing treatments based on how the average patient would respond.