Reported Quality Of Life Outcomes Research Articles

Quality of life outcomes comparing primary Transoral Robotic Surgery (TORS) with primary radiotherapy for early-stage oropharyngeal squamous cell carcinoma: A systematic review and meta-analysis

BackgroundTransoral Robotic Surgery (TORS) and radiotherapy are considered oncologically equivalent primary treatment options for early-stage HPV-positive oropharyngeal squamous cell carcinoma (OPSCC). Quality of Life (QoL) and Patient Reported Outcome Measures (PROMs) are therefore imperative in supporting clinical decision-making and optimising patient-centred care. The aim of this article is to evaluate how these primary treatment modalities compare in terms of QoL. Materials and methodsSystematic review and meta-analysis of studies comparing primary TORS and primary radiotherapy for OPSCC using validated QoL tools. Swallowing and global QoL were the primary endpoints with secondary endpoints including all other QoL domains. An inverse variance random-effects model was employed to calculate the weighted estimate of the treatment effects across trials. ResultsA total of six studies collectively reporting on 555 patients were included (n = 236 TORS and n = 319 radiotherapy). Meta-analysis showed no significant difference for swallowing (mean difference = −0.24, p = 0.89) and global QoL (mean difference = 4.55, p = 0.14). For the remaining QoL domains (neck/shoulder impairment, neurotoxicity, voice, xerostomia, speech, and distress), the scarcity of data did not permit meta-analysis. However, the existing data showed no significant difference for any except for xerostomia where TORS appears favourable in the sole study reporting on this. ConclusionsTORS and radiotherapy appear to be comparable primary treatment options for early stage OPSCC when it comes to QoL. However, a substantial proportion of patients in the TORS group received adjuvant (chemo)radiotherapy rendering it difficult to establish the ‘true’ QoL outcomes following surgery alone. There are also minimal studies reporting QoL outcomes beyond swallowing and global QoL. Further research is therefore needed, including more randomised trials adequately powered to detect differences in QoL outcomes.

Long-term quality of life outcomes in patients undergoing microsurgical resection of vestibular schwannoma

BackgroundWhile previous studies have assessed patient reported quality of life (QOL) of various vestibular schwannoma (VS) treatment modalities, few studies have assessed QOL as related to the amount of residual tumor and need for retreatment in a large series of patients. Objective: To assess patient reported QOL outcomes following VS resection with a focus on extent of resection and retreatment. MethodsA retrospective chart review was performed using single-center institutional data of adult patients who underwent VS resection by the senior authors between 1989-2018 at Loyola University Medical Center. The Penn Acoustic Neuroma Quality of Life (PANQOL) survey was sent to all patients via postal mail. ResultsFifty-five percent of 367 total patients were female with a mean age of 61.6 years (SD 12.63). The mean period between surgery and PANQOL response was 11.4 years (IQR: 4.74-7.37). The median tumor size was 2 cm (IQR: 1.5-2.8). The mean total PANQOL score was 70 (SD 19). Patients who required retreatment reported lower overall scores (μdiff = -10.11, 95% CI: -19.48 to -0.74; p = 0.03) and face domain scores (μdiff = -20.34, 95% CI: -29.78 to -10.91; p < .001). There was no association between extent of resection and PANQOL scores in any domain. ConclusionIn an analysis of 367 patients who underwent microsurgical resection of VS, extent of resection did not affect PANQOL scores in contrast to previous reports in the literature, while the need for retreatment and facial function had a significant impact on patient-reported outcomes.

A systematic review of oncosurgical and quality of life outcomes following pelvic exenteration for locally advanced and recurrent rectal cancer.

Pelvic exenteration (PE) is now the standard of care for locally advanced (LARC) and locally recurrent (LRRC) rectal cancer. Reports of the significant short-term morbidity and survival advantage conferred by R0 resection are well established. However, longer-term outcomes are rarely addressed. This systematic review focuses on long-term oncosurgical and quality of life (QoL) outcomes following PE for rectal cancer. A systematic review of the PubMed®, Cochrane Library, MEDLINE® and Embase® databases was conducted, in accordance with the PRISMA (Preferred Reporting Items for Systematic reviews and Meta-Analyses) guidelines. Studies were included if they reported long-term outcomes following PE for LARC or LRRC. Studies with fewer than 20 patients were excluded. A total of 25 papers reported outcomes for 5,489 patients. Of these, 4,744 underwent PE for LARC (57.5%) or LRRC (42.5%). R0 resection rates ranged from 23.2% to 98.4% and from 14.9% to 77.8% respectively. The overall morbidity rates were 17.8-87.0%. The median survival ranged from 12.5 to 140.0 months. None of these studies reported functional outcomes and only four studies reported QoL outcomes. Numerous different metrics and timepoints were utilised, with QoL scores frequently returning to baseline by 12 months. This review demonstrates that PE is safe, with a good prospect of R0 resection and acceptable mortality rates in selected patients. Morbidity rates remain high, highlighting the importance of shared decision making with patients. Longer-term oncological outcomes as well as QoL and functional outcomes need to be addressed in future studies. Development of a core outcomes set would facilitate better reporting in this complex and challenging patient group.

Comparison of Quality of Life Outcomes for Percutaneous Versus Transcutaneous Implantable Hearing Devices: A Systematic Review and Meta-analysis.

To compare quality of life (QOL) outcomes of percutaneous and transcutaneous bone conduction devices (pBCD and tBCD, respectively). Pubmed, Scopus, CINAHL. A systematic review was performed searching for English language articles from inception to March 15, 2023. Studies reporting QOL outcomes measured using a validated tool following implantation of either pBCDs or tBCDs were considered for inclusion. QOL outcomes included scores for Glasgow Benefit Inventory, Glasgow Children's Benefit Inventory, Abbreviated Profile of Hearing Aid Benefit, and the Speech, Spatial, and Qualities of Hearing Scale. A meta-analysis of continuous measures was performed. A total of 52 articles with 1,469 patients were included. Six hundred eighty-nine patients were implanted with pBCDs, and the remaining 780 were implanted with tBCDs. Average Glasgow Benefit Inventory scores for the tBCD group (33.0, 95% confidence interval [22.7-43.3]) were significantly higher than the pBCD group (30.9 [25.2-36.6]) (Δ2.1 [1.4-2.8], p < 0.0001). Mean Glasgow Children's Benefit Inventory scores (Δ3.9 [2.0-5.8], p = 0.0001) and mean gain in Abbreviated Profile of Hearing Aid Benefit scores (Δ5.6 [4.8-6.4], p < 0.0001) were significantly higher among patients implanted with tBCDs than those implanted with pBCDs. Patients implanted with tBCDs also had significantly higher gains on the Speech (Δ1.1 [0.9-1.3], p < 0.0001), Spatial (Δ0.8 [0.7-0.9], p < 0.0001), and Qualities of Hearing (Δ1.2 [1.1-1.3], p < 0.0001) portions of the Speech, Spatial, and Qualities of Hearing Scale than those implanted with pBCDs. Patients implanted with transcutaneous devices had better QOL outcomes than those implanted with percutaneous devices.

The effect of health behavior interventions to manage Type 2 diabetes on the quality of life in low-and middle-income countries: A systematic review and meta-analysis.

Behavioral interventions targeted at managing Type 2 diabetes mellitus (T2DM) may have a positive effect on quality of life (QOL). Limited reviews have synthesized this effect in low- and middle-income countries (LMICs). This review and meta-analysis synthesised available evidence on the effect of behavioral interventions to manage T2DM on the QOL of people with T2DM in LMICs. Electronic databases PUBMED/MEDLINE, SCOPUS, CINAHL, Embase, Web of Science and PsycINFO were searched from May to June 2022. Studies published between January 2000 and May 2022, conducted in LMICs using randomized controlled trial design, using a health behavior intervention for T2DM management, and reporting QOL outcomes were included. Difference in QOL change scores between the intervention and control group was calculated as the standardized mean difference (SMD) of QOL scores observed between the intervention and control groups. Random-effects model was used for meta-analysis. Of 6122 studies identified initially, 45 studies met the inclusion criteria (n = 8336). Of them, 31 involved diabetes self-management education and 14 included dietary and/or physical activity intervention. There was moderate quality evidence from the meta-analysis of mean QOL (n = 25) that health behavior intervention improved the QOL of people with T2DM (SMD = 1.62, 95%CI = 0.65-2.60 I2 = 0.96, p = 0.001). However, no significant improvements were found for studies (n = 7) separately assessing the physical component summary (SMD = 0.76, 95%CI = -0.03-1.56 I2 = 0.94, p = 0.060) and mental component summary (SMD = 0.43, 95%CI = -0.30-1.16 I2 = 0.94, p = 0.249) scores. High heterogeneity and imprecise results across studies resulted in low to moderate quality of evidence. The findings suggest that health behavior interventions to manage T2DM may substantially improve the QOL of individuals with T2DM over short term. However, due to low to moderate quality of evidence, further research is required to corroborate our findings. Results of this review may guide future research and have policy implications for T2DM management in LMICs.

Dynamic Change in Patient Reported Quality of Life is a Predictor for Survival in Localized Prostate Cancer: Exploratory Analysis from a Phase III Randomized Controlled Trial

<h3>Purpose/Objective(s)</h3> Patient reported quality of life (QOL) outcomes are pivotal endpoints to determine treatment efficacy and safety. However, it remains unknown if treatment induced change in patient reported QOL outcomes correlate with survival in localized prostate cancer (LPCa). We performed an exploratory analysis of a phase III randomized controlled study to determine the association of treatment induced dynamic change in patient reported global QOL with long-term overall survival (OS) and disease-free survival (DFS) in LPCa treated with radiotherapy (RT) and androgen deprivation therapy (ADT). <h3>Materials/Methods</h3> LPCa patients with Gleason score ≤7, clinical stage T1b-T3a, and PSA <30 ng/mL were randomized to neoadjuvant and concurrent ADT for 6 mos starting 4 mos before prostate RT or concomitant and adjuvant ADT for 6 mos starting concurrently with RT. Patient reported QOL outcomes were collected using EORTC QOL questionnaires. We used the global QOL (G-QOL) score from C30 questionnaire. Increase in G-QOL score indicated improvement in global QOL. To capture the association between the dynamic change in the G-QOL of each patient and the hazard of outcome, we utilized the framework of multivariate joint modeling for longitudinal and survival outcomes. For the time-to-event submodel, a Cox proportional hazard regression model was built with covariables including treatment arm, baseline PSA, tumor stage, Gleason score, and age. For the longitudinal submodel, a linear mixed effect model was built with an interaction term for treatment arm and time of evaluation in addition to fixed covariables - treatment arm, time of evaluation, baseline G-QOL score and age. Time of assessment was included as a random slope while patients were included as random intercepts in the mixed models. The two submodels were linked through a random effect. Baseline hazard was modeled using penalized splines. A two-sided p-value <0.025 was chosen to be the threshold for statistical significance. <h3>Results</h3> A total of 388 patients were eligible for this study – 191 belonged to the neoadjuvant arm while 197 to the adjuvant arm. At a median follow-up of 148 mos, a total of 133 deaths were recorded. On joint modelling, an increase in G-QOL score by 10 units (the threshold for clinically meaningful change) was associated with a 11.2% decrease in the hazard of death (hazard ratio [HR] per 1 unit increase G-QOL: 0.988, 95% CI: 0.980-0.996; p=0.007). An increase in G-QOL score by 10 units was associated with 10% decrease in the hazard of progression or death (HR per 1 unit increase in G-QOL: 0.990; 95% CI: 0.984-0.999; p=0.023). <h3>Conclusion</h3> Treatment induced improvement in global QOL is a predictor for improved DFS and OS in LPCa. This suggests a significant interplay of patient-reported quality of life and quantity of life in LPCa which could mainly be driven by the non-cancer specific mortality in this patient population. Judicious use of these QOL metrics could guide risk stratification and potentially inform treatment decision in these patients

Concept: A randomised multicentre trial of first line chemotherapy comparing three weekly cabazitaxel versus weekly paclitaxel in HER2 negative metastatic breast cancer

BackgroundPaclitaxel is commonly used as first-line chemotherapy for HER2-negative metastatic breast cancer (MBC) patients. However, with response rates of 21.5–53.7% and significant risk of peripheral neuropathy, there is need for better chemotherapy. Patients and methodsThis open-label phase II/III trial randomised HER2-negative MBC patients 1:1 to either 6 cycles of three-weekly cabazitaxel (25 mg/m2), or, weekly paclitaxel (80 mg/m2) over 18 weeks.The primary endpoint was progression free survival (PFS). Secondary endpoints included objective response rate (ORR), time to response (TTR), overall survival (OS), safety and tolerability and quality of life (QoL). Results158 patients were recruited. Comparing cabazitaxel to paclitaxel, median PFS was 6.7 vs 5.8 months (HR 0.87; 80%CI 0.70–1.08, P = 0.4). There was no difference in median OS (20.6 vs 18.2 months, HR 1.00; 95%CI 0.69–1.45, P = 0.99), ORR (41.8% vs 36.7%) or TTR (HR 1.09; 95%CI 0.68–1.75, P = 0.7).Grade ≥3 adverse events occurred in 41.8% on cabazitaxel and 46.8% on paclitaxel; the most common being neutropenia (16.5%) and febrile neutropenia (12.7%) cabazitaxel and neutropenia (8.9%) and lung infection (7.6%) paclitaxel. Peripheral neuropathy of any grade occurred in 54.5% paclitaxel vs 16.5% cabazitaxel.Mean EQ-5D-5L single index utility score (+0.05; 95%CI 0.004–0.09, P = 0.03) and visual analogue scale score (+7.7; 95%CI 3.1–12.3, P = 0.001) were higher in cabazitaxel vs paclitaxel. ConclusionsThree-weekly cabazitaxel in HER2-negative MBC does not significantly improve PFS compared to weekly paclitaxel, although it has a lower risk of peripheral neuropathy with better patient reported QoL outcomes. It is well tolerated and requires fewer hospital visits.

Quantitative Cross-Sectional Analyses of Quality-of-Life Outcomes Related to Race, Sex, Age, and Clinical Stage Among Multiple Myeloma Patients at an Urban Center

Introduction. Multiple myeloma (MM) patients suffer from disease symptoms and impact on quality of life (QoL) - a patient's sense of enjoyment, well-being, and ability to carry out activities of daily living. Reports on QoL in MM have focused on clinical trials that usually involve younger patients with adequate performance status (PS), mostly Caucasians. The median age of MM onset is 69 years; advanced age is associated with multiple comorbidities and deteriorating PS. Blacks have twice the incidence of MM compared to Caucasians; they also have a higher risk of dying due to poor healthcare utilization. Here, we explored the multifactorial nature of race, sex, age, and clinical stage in patient reported QoL outcomes using quantitative cross-sectional analyses from a single center in urban setting.Methods. Participants with a diagnosis of plasma cell disorders at various stages: MGUS, smoldering myeloma (SMM), or MM (newly diagnosed, maintenance, relapsed/refractory) were sequentially enrolled for a one-time measurement of QOL outcomes. Consent and study questionnaire were conducted verbally. Participants simply responded ‘yes’ or ‘no’ to nine question prompts outlined in Table 1, followed by verbal reasoning which was transcribed by the interviewer. The questionnaire included three main domains of QoL assessment based on existing literature review and feedback with our patient population. The first is physical symptoms and function (disease-related symptoms, treatment-related adverse events, sexual health and satisfaction with care), the second is mental health (depression, spirituality), and the third is social impact (financial burden, family support). Data were analyzed using chi-square test of independence. Participants will be re-approached for a second interview to determine changes to impact across the same QoL variables over time. The questionnaires validated within the current study will be used for a longitudinal cohort study to investigate whether a greater degradation is observed in one or more QoL domains during a specific time-point of MM disease course.Results. A total of 100 patients (males n=52) were enrolled. Median age was 64 years (IQR 55 - 68); 59 were Caucasian, 34 were Black, and 7 from other racial groups. At the time of interview: 7 were newly diagnosed or receiving induction therapy, 25 were undergoing stem cell transplant (SCT), 22 were on maintenance therapy, 33 were relapsed on salvage therapy, and 13 on no therapy (MGUS/SMM). Regardless of race, age, or sex, patients on therapy felt their QOL was impacted by fatigue (67%), and myeloma symptoms (65%). Black participants reported more fatigue as an impact on their QoL compared to other racial groups, p = .009. Women reported more impact from MM on sexual intimacy, p = .04, and impact from financial burden than male participants, p = .03. Younger patients <60 years old (n=38) reported more impact from treatment-related adverse events, p = .004, disease-related symptoms, p = .02, and dissatisfaction with information received about their disease and/or treatment, p = .004, compared to patients aged >60 years. Not surprisingly, patients with active MM (n=87); newly diagnosed on therapy, those undergoing SCT, or on maintenance, and relapsed disease) reported more MM symptoms p = <.001, and suffered from more fatigue, p = <.02, with higher impact on QoL compared to those with early-stage plasma cell disorders (MGUS, SMM). However, early-stage patients felt more dissatisfaction with information received and uncertainty about what to expect, p = <.001, compared to MM patients receiving therapy. Interestingly, there were no age, sex, race, or clinical stage dependent effects observed from SCT experience, spirituality or religion, and dissatisfaction with cancer care.Conclusions. Patients <60 years were impacted more by disease-related symptoms, treatment-related adverse events, in comparison to patients >60 years. Women felt their QoL was impacted by MM's affect on sexual intimacy and resulting financial burden in comparison to men. Black participants felt greater impact from fatigue in comparison to Caucasian and other races. Active MM patients on treatment were more impacted by disease-related symptoms and fatigue compared to early-stage patients. MGUS/SMM patients not on therapy were more impacted by dissatisfaction of information received compared to patients receiving treatment at time of data collection. [Display omitted] DisclosuresBadros: Janssen: Research Funding; J&J: Research Funding; BMS: Research Funding; GlaxoSmithKline: Research Funding.

General Patient-Reported Outcome Measure Tools Non-Reflective of AL Amyloidosis Quality of Life (QoL): A Systematic Literature Review

Background: Systemic AL Amyloidosis is characterized by deposition by amyloid fibrils of light chains produced by clonal plasma cells. New combination therapies have substantially prolonged the life expectancy of patients with AL Amyloidosis . Still the prognosis of the disease in the majority of the patients is dismal and Quality of Life (QoL) issues should be included in clinicians' primary objectives. Nowadays, patient-reported outcome measures (PROMs) are considered one of the most responsive tools that can guide personalized interventions to optimize QoL in parallel with therapeutic interventions. The aim of this metanalysis was to establish which PROMs have been utilized in studies on AL Amyloidosis and evaluate their validity.Methods: Two independent investigators (S.B.; C.K.) systemically reviewed PubMed, Medline and EMBASE databases for publications up to May 2021 on PROMs employed to report QoL outcomes in AL Amyloidosis. The identified PROMs were subsequently assessed for their validity in this context against COSMIN (Consensus-based Standards for the selection of health Measurement Instruments) quality criteria.Results:Of the 246 publications originally retrieved, only 57 were further analysed as 92 were duplicates, 11 were irrelevant to AL Amyloidosis, 56 did nor refer to QOL and 13 reported QOL without using QOLQs, 13 were case-reports and 4 were review articles. These included 47 observational studies and 10 prospective clinical trials. In these, thirteen different PROMs were used on occasion to report QOL outcomes (SF-36; EQ-5D-3L; FACT-G; PROMIS-GH); HPRSS; DT; EORTC QLQ-C30; KCCQ-12; GAF; SWLS; STAI; CESD; MDASI) with SF-36 being the most popular (35/ 58 publications). All of them are traditionally validated in similar to AL Amyloidosis diseases (e.g. Multiple Myeloma) or in its complications (e.g. Congestive Heart Failure). In the absence of face-validity studies, the content validity of these PROMs was assessed against QOL aspects identified in literature by open questionnaires (Table 1). The outcome of the intensive analysis of these studies showed that the QoLQ fail to cover the broad spectrum of disease symptoms and current therapy-related toxicity. Furthermore, there is limited if any evidence for the validation of these tools in this context (Table 2). COSMIN criteria were met only for SF-36 and PROMIS-GH as regards internal consistency (Cronbach's a>70).Conclusions: This literature review reveals that commonly applied PROMs in studies on AL Amyloidosis do not represent the impact of this complex disease and its treatment on QoL issues. Thus, there is a need to develop a new, well-validated, disease-specific PROM that can facilitate the approval of new treatments and the adjustment of therapy-intensity according to its toxicity and QoL. [Display omitted] DisclosuresNo relevant conflicts of interest to declare.

Quality of life outcomes in cryoablation of atrial fibrillation-A literature review.

Cryoballoon ablation (CRYO) for pulmonary vein isolation (PVI) in atrial fibrillation (AF) has become an established treatment option as alternative for radiofrequency catheter ablation (RFCA). As symptom relief is still the main indication for PVI, quality of life (QoL) is a key outcome parameter. This review summarizes the evidence about the evolution of QoL after CRYO. A search for clinical studies reporting QoL outcomes after CRYO was performed on PUBMED and COCHRANE. A total of 506 publications were screened and 10 studies met the in- and exclusion criteria. All studies considered QoL as a secondary endpoint and reported significant improvement in QoL between baseline and 12 months follow-up, independent of the QoL instruments used. The effect size of CRYO on QoL was comparable between studies and present in both paroxysmal and persistent AF. Direct comparison between CRYO and RFCA was limited to two studies, there was no difference between ablation modalities after 12 months FU. Two studies in paroxysmal AF reported outcome beyond 12 months follow-up and QoL improvement was maintained up to 36 months after ablation. There were no long-term data available for persistent AF. CRYO of AF significantly improves QoL. The scarce amount of data with direct comparison between subgroups limits further exploration. Assessment of QoL should be considered a primary outcome parameter in future trials with long-term follow-up.

Prevalence vs impact: a mixed methods study of survivorship issues in colorectal cancer

PurposeThis study aims to explore the prevalence of CRC survivorship issues and their impact on survivors’ quality of life (QoL).MethodsThis study utilised a mixed methods sequential explanatory design. Adult CRC survivors between 6- and 60-months post-diagnosis (n = 304) were purposively recruited from three hospitals and twenty-one cancer support centres in Ireland. QoL was evaluated using the EuroQol and FACT-C questionnaires and results compared to population norms. 22 survey participants took part in semi-structured interviews exploring the impact of survivorship issues on their daily lives.ResultsWhile CRC survivors reported QoL outcomes comparable to or better than normative populations, 54% were dissatisfied with their QoL. The most common survivorship issues reported included negative body image (74%), fatigue (68%), sexual dysfunction (66%) and sleep disturbance (59%). Thematic analysis of the qualitative data illustrated survivors’ attempts to live with the impact of cancer and its treatment (loss, fear, impact) and striving to contextualise, reframe and understand the consequences of cancer and its treatment (control, vigilance, benefit). Within these themes, the cross-domain impact of less prevalent symptoms including bowel dysfunction (28–57%) and peripheral neuropathy (47%) were widely discussed.ConclusionsAlthough cancer survivors report positive QoL outcomes, many experience distressing physical, psychological and social effects. The findings suggest less common and difficult to manage symptoms are the greatest source of distress and unmet need. Support and information must be tailored to address survivors’ individual needs and preferences for support, informed by holistic person-centred assessment.

Informative censoring due to missing data in quality of life was inadequately assessed in most oncology randomized controlled trials

Objective We aimed to systematically characterize reporting missing quality of life (QoL) data in oncology randomized controlled trials (RCTs) and to estimate prevalence of adequate reporting according to existing guidelines.Study Design and Setting This cross-sectional analysis includes all articles on anti-cancer drugs tested in RCTs in six high impact medical/oncology journals, published between January 2015 and May 2020, that reported QoL outcomes. From 1942 identified articles, 215 (11%) met inclusion criteria. Data abstracted included whether compliance for QoL assessment were reported, whether results from a missing data statistical analysis were reported, whether articles met current recommendations for reporting missing data in QoL assessments.Results The results from a missing data statistical analysis were available in 22 trials (10.2%). Overall, 16 trials (7.4%) met current recommendations for reporting missing data in QoL assessments. Articles specifically reporting on QoL or patient reported outcomes were more likely to meet recommendations than other reports (P < 0.0001).Conclusion This systematic cross-sectional study found that most oncology RCTs reporting on QoL do not report adequately on missing data in QoL, with only 7.4% of trials meeting current reporting guidelines. The possibility of informative censoring, therefore, cannot be assessed in most of trials.

A structured review of quality of life in advanced and high-risk cutaneous squamous cell carcinoma shows the need for more studies and better measures.

BackgroundCutaneous squamous cell carcinoma (cSCC) accounts for nearly a quarter of non‐melanoma skin cancers. Studies reporting Quality of Life (QoL) in this group focus on early stage disease. A small proportion of cSCC patients have high‐risk or advanced disease, with potentially significant QoL impacts, yet are largely overlooked.AimsThis structured review appraises measures and published QoL outcomes in this group.Materials & MethodsWe conducted searches in MEDLINE, EMBASE, CINAHLplus and PsycInfo in June 2020 (updated in October) to identify publications specifically reporting QoL outcomes in this cohort. Returns were reviewed against a strict set of eligibility criteria.ResultsWe identified seven publications for inclusion; three relating to high‐risk cSCC, three to metastatic disease and one to unresectable disease. Publications were appraised for quality using the Mixed Methods Appraisal Tool. Only one fulfilled more than two of the five quality criteria. Studies employed a range of patient reported outcome measures to assess QoL, both generic and disease specific.DiscussionAll studies with multiple time‐points reported stable or improving QoL, however extrapolation of these findings to the cSCC population is not warranted due to study limitations including mixed populations, incomplete data sets or single measurements. We set out to review the QoL literature for high‐risk and advanced cSCC and found a small and disparate body of evidence. Studies varied significantly in terms of study population, design and quality. While the identified studies suggested stable or improving QoL, we question the choice of measures used and highlight the need for further work in this area.ConclusionWhile there are some published reports about quality of life for patients with early stage cutaneous squamous cell carcinoma, these impacts for the high‐risk or advanced cohort are largely unexplored. We conducted a structured review of published measures and outcomes used in this cohort and found a demonstrable need for further, targeted, exploration of patient needs in this area.

PIH2 Systematic Literature Review of Clinical Outcomes, Economic Burden, and Quality of Life Burden of Anemia in Elderly Patients

This study aimed to synthesize the relationship between chronic anemia associated with various diseases and the clinical, economic, and quality of life (QoL) outcomes in elderly patients. Three systematic literature reviews (SLRs) were conducted in February 2019. The clinical SLR results were summarized through meta-analysis, whereas the economic and QoL SLRs were summarized qualitatively. The meta-analysis of clinical outcomes included 32 studies. Anemia was associated with a significantly increased risk of mortality (hazard ratio [HR] 1.63, 95% confidence interval [CI] 1.51–1.77), cardiac-related mortality (HR 1.47, 95% CI 1.29–1.68), and hospitalization (HR 1.53, 95% CI 1.24–1.88) compared with non-anemic elderly patients. Many studies were conducted among nursing home residents, whereas others were conducted in patients with pre-existing conditions (e.g. heart failure); findings were consistent across these studies. Sixteen studies reported QoL outcomes; the Activities of Daily Living (ADL) score was reported most frequently (n=8), along with the 36-item short form (n=5), the Mini-Mental State Examination (n=5), and the Geriatric Depression Score (n=4). More than half of the studies suggested that anemia worsened ADL, whereas 2 studies reported comparable ADL. Some studies suggested that anemia was associated with depression; however, the results varied depending on the scale used. Of the 4 studies with economic outcomes, 2 reported on direct costs and 3 reported on resource utilization. The largest driver of cost differences between anemia and non-anemia groups was hospitalization-related costs. Among those with comorbidities, the length of hospital stay was greater for anemic versus non-anemic patients. In the elderly, anemia was associated with worsened QoL and increased mortality, hospitalizations, and costs. These findings were consistent across disease areas. Limitations include inconsistencies in the QoL literature owing to differences in QoL measurement tools, and a scarce economic evidence base.

The clinical and humanistic burden of cutaneous T-cell lymphomas and response to conventional and novel therapies: results of a systematic review

ABSTRACTObjectives: To summarize the impact of relapsed/refractory primary cutaneous T-cell lymphomas (CTCL) on quality of life (QoL) and the efficacy of available treatments in two systematic reviews (SRs).Methods: Searches were performed on 16 January 2018 and 23 January 2018, respectively, in Medline, Medline in process, the Cochrane database, and EconLit. Studies reporting QoL outcomes in adults with CTCL or treatment efficacy in relapsed/refractory CTCL were included.Results: Based on 15 QoL studies, CTCL symptoms/complications negatively affect patients’ physical, emotional, and social functioning. Skin problems pose considerable symptom burden, while advanced disease stage is associated with poorer QoL. CTCL negatively affects caregivers, primarily through family dynamics and relationships. The clinical efficacy SR included 72 publications covering 23 therapies. Overall response rate (ORR) ranged from 14% (belinostat) to 95% (total skin electron beam therapy). ORRs >50% were reported for several therapies including brentuximab vedotin (50–78%) and bexarotene (39–86%). Over half (13 of 23 therapies) had ORRs <30%. Median progression-free survival varied between treatments (3.5–116.4 months) and was >20 months for brentuximab vedotin and alemtuzumab.Conclusion: CTCL negatively affects patients’ and caregivers’ QoL. A considerable proportion of patients have no response or no sustainable response to current treatments.

A Systematic Literature Review of the Burden of Ischemic Priapism in Patients with Sickle Cell Disease

INTRODUCTION: Ischemic or low-flow priapism is a serious condition characterized by a persistent, painful erection that lasts at least 4 hours or is unrelated to sexual stimulation. Both stuttering (repeated short episodes) and fulminant (long-lasting) priapism are prevalent in men and boys with sickle cell disease (SCD). Approximately 40% of patients with SCD will experience priapism during their lifetime. The objective of this study was to conduct a systematic literature review (SLR) to identify published studies reporting the burden associated with priapism in patients with SCD. METHODS: MEDLINE, Embase, Cochrane CENTRAL/CDSR and 14 congresses were searched from May 9-16, 2019. Results were screened against predefined criteria by two independent researchers. Studies assessing the clinical, humanistic, or economic burden of human patients with SCD and priapism (N≥15) were included. Outcomes of interest included priapism-related symptoms, treatment-related complications, quality of life (QoL), and economic burden. RESULTS: The literature search identified 1236 articles; 62 full-text studies were reviewed in detail and 34 studies were included in the analysis. Most studies were retrospective observational (21), others were prospective (7 observational, 6 interventional). Study size varied widely (range: 15-10,788 patients). Of 27 studies that reported mean or median patient age, the age statistic for patients with SCD and priapism ranged from 10 to 32. Overall, 19 studies reported at least one aspect of clinical burden. Thirteen reported the rate of erectile dysfunction or impotence, ranging from 0% to 56%. Eight reported treatment-related complications, including perceived penile scarring/deformity (19.4%) and small intrapenile hematoma (13.3%). Nineteen studies reported QoL outcomes. Eight reported physical outcomes, including a negative impact of SCD-related priapism on sleep quality and worse scores on periodic limb movement and apnea hypopnea indices. Seven reported measures of sexual function, including low rates of satisfactory sexual intercourse. One study reported a diminished Sexual Health Inventory for Men score in patients with active priapism, and another found a statistically significant relationship between stuttering priapism and premature ejaculation. Three studies reported measures of mental function, one of which outlined increased cognitive fatigue and impaired emotional functioning among children with priapism and another reported that 36% of patients had substantial to very extreme worry regarding episodes of priapism. Use of validated measures for QoL were limited: 3 studies assessed patients with the International Index of Erectile Function (IIEF) and 1 with the Priapism Impact Profile (PIP). No studies measuring the burden to caregivers of pediatric patients with priapism were identified. Eight studies reported the economic burden of priapism in patients with SCD, including rates of emergency department visits (4 studies: 56.5% sought hospital treatment - 26/46; 49.8% admission to the hospital out of emergency department visits - 5371/10,788; 50.4% admitted at the emergency department - 930/1844; 26% vs. 11% readmission vs no readmission - p &amp;lt;0.001) and hospital length of stay (4 studies: mean 3.38 days; mean 3.8 days; median 2 days; median 5 days in patients who received an exchange transfusion as a treatment for priapism.). Three studies reported direct treatment costs, ranging from a mean of $4496 (2015 USD) for patients who did not undergo penile operations to a mean of $19,670 (2013 USD) total adjusted hospital costs for patients receiving both transfusions and urologic procedures. CONCLUSIONS: This SLR identified 34 studies reporting the burden of priapism in patients with SCD. No RCTs were identified. The use of comprehensive or validated measures of humanistic burden was limited. Manifestations of priapism are wide-ranging, including painful treatment-related complications, impaired sleep, and diminished physical, mental and sexual function. Patients with priapism and SCD are more likely to visit the emergency department and experience longer hospital stays compared to SCD patients without priapism. Together, the evidence identified in this SLR indicates that priapism not only can result in erectile dysfunction, but can impair multiple dimensions of a patient's physical and mental health. Disclosures Nellesen: Analysis Group, Inc.: Consultancy. Lucas:Analysis Group, Inc.: Consultancy. Liu:Analysis Group, Inc.: Consultancy. Bhor:Novartis: Employment, Equity Ownership. Paulose:Novartis Pharmaceuticals Corporation: Employment.

Trajectory of HRQOL scores in pediatric patients receiving proton therapy: results from the Pediatric Proton Consortium Registry (PPCR)

Health-related quality of life (QOL) outcomes are an important metric in understanding the perceived health and emotional well-being of pediatric proton radiotherapy (RT) patients. Here we compare reported QOL outcomes at two time points during radiotherapy to determine the effect RT has on the holistic health of a patient. Pediatric patients (<22 years) treated with RT across multiple institutions were prospectively enrolled on an optional QOL study through the PPCR. Child and proxy answers to the PedsQL v.4.0 are evaluated to generate a total core score (QOL score) ranging from 0-100 (best). QOL scores captured during the first (early) and last (late) week of RT were evaluated. Paired t-tests and Wilcoxon sign-ranked tests were used to assess differences in early and late-RT QOL score within groups, independent t-tests and the Kruskal-Wallis test were used to assess differences between groups. One hundred twenty patients (n=69 (57.5%) CNS, n= 51 (42.5%) non-CNS) enrolled between 9/2015 and 1/2018 comprised the study cohort. Among non-CNS patients, 22 (43.1%) had a head/neck primary tumor. Median age was 10.5 years (<1 to 21.8). Median RT total dose was 54 Gy (21.0-76.0). Mean RT QOL score did not change significantly during RT (early: 74.6; late: 74.8, p=0.87). Children with CNS tumors noted a 3-point increase in QOL scores during RT (p=0.03), whereas QOL among children with non-CNS tumors did not significantly change (p=0.13). Non-CNS primary tumor site had no significant effect on change in QOL score (p=0.31). Patients with KPS=100 at the beginning of RT showed a non-significant decline of 5.0-points, but started higher than patients with a KPS of <100 who remained stable during RT. QOL scores did not differ by anesthesia use for RT (p=0.85), age (p=0.09), or by exposure to concurrent chemotherapy (p=0.34). During RT, children with CNS tumors reported an increase in the QOL score which differed from patients with non-CNS primaries who reported a non-significant decrease in scores. Anesthesia use, age, and concurrent chemotherapy did not significantly effect QOL scores. The PedsQL can be used to monitor the QOL of patients receiving proton radiotherapy. Future efforts to determine if QOL monitoring can lead to improvements in patient’s care and thus treatment experience are warranted.Tabled 1Abstract TU_41_3730; Table 1N(%)Early-RT Score Mean (sd)Late-RT Score Mean (sd)P within groupsP between groups PTumor TypeCNS69 (57.5)76.3 (16.1)79.3 (13.8)0.030.003Non-CNS51 (42.5)72.3 (16.4)68.7 (17.9)0.13AnesthesiaYes47(39.2)72.7 (14.8)72.6 (17.1)0.860.85No70 (58.3)76.0(17.1)76.3 (16.2)0.78Age (years)<10.56175.2 (14.8)73.5 (16.1)0.350.09>10.55974.0 (17.9)76.1 (16.9)0.11ChemotherapyYes79 (65.8)72.2 (16.9)71.3 (17.4)0.960.35No38 (31.7)80.7 (13.1)82.0 (12.0)0.51KPS1003080.8 (15.3)75.8 (15.4)0.070.02<1005870.9 (15.7)72.7 (15.7)0.24 Open table in a new tab

P2.01-03 Quality of Life Outcomes for FDA-Approved Agents in Advanced Non-Small Cell Lung Cancer

Symptom control and quality of life (QoL) improvement are among the most important goals of systemic therapy in advanced NSCLC, but they are not required for regulatory approval. We reviewed published QoL data for agents approved for use in advanced NSCLC by the United States Food and Drug Administration (FDA) and explore their potential uses in the era of precision medicine. Through systematic review, we identified 19 agents approved by the FDA for the treatment of advanced NSCLC between 1999 and 2017. Clinical trials associated with regulatory approval of these agents were identified. QoL results were abstracted from study publications, in addition to trial characteristics (phase, sponsor). 46 trials were identified supporting the FDA approval of the 19 agents. Of these, 33 (72%) were phase 3 randomized trials (including 3 cooperative group studies) and 13 (28%) were early phase (I/II) trials. QoL data were reported for 27 trials (59%), including all cooperative group trials. Another 5 (11%) measured QoL but results were not published. The remaining 13 (24%) trials did not measure QoL, 8/13 are phase III, 3/13 phase II and 2 phase I. In trials that reported QoL outcomes, better global QoL and cancer-related symptoms were identified in 17/33 (51%) and 18/33 (55%), respectively. Time to symptom deterioration was reported in 18 (55%) studies, with all but one showing benefit for the approved agent. The remaining studies reported no significant difference in QoL between study treatment arms. All studies reporting improved QoL parameters also reported improved response rates. In trials demonstrating OS and PFS improvement, 3/14 and 10/21 reported better QoL outcomes, respectively. ORR was higher in 26 trials, and it was associated with improvement in QoL in 12/26. Of 12 early phase trials leading to drug approval based on response rate and duration, 6 reported improved QoL outcomes. Three have not yet reported QoL data and 3 did not measure QoL. QoL is clearly associated with response rate but improved QoL or symptom control was only demonstrated in17/33 (51%) and 18/33 (55%) of studies supporting regulatory approval. QoL may be a clinically relevant endpoint for drug approval based on early phase trials of agents with high response rates and should be given further consideration by pharmaceutical sponsors and regulators in this era of precision medicine.

Incobotulinumtoxina treatment improves quality of life of patients with upper- and lower-limb spasticity

The safety and efficacy of botulinum neurotoxin treatment for limb spasticity are well established. However, reports on the effect of treatment on quality of life (QoL) are inconclusive. Here we report QoL outcomes following incobotulinumtoxinA treatment of upper- and lower-limb spasticity in the TOWER study ( NCT01603459 ). This open-label study assessed the safety and efficacy of escalating incobotulinumtoxin A doses (400 U, 600 U and 600–800 U) in patients (18–80 years) with upper- and lower-limb spasticity on the same body side due to stroke or other cerebral causes. QoL was assessed using the EuroQoL-5 dimensions (EQ-5D) questionnaire, which includes five dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), and a visual analogue scale (VAS) on which patients rate their current state of health from 0 (worst) to 100 (best). In total, 155 patients were enrolled and received incobotulinumtoxin A. For all EQ-5D dimensions, the proportion of patients with improvements from the study baseline to the end of injection cycle (IC) 3 was higher than the proportion with worsening: Mobility, 11.4% vs. 2.9%; Self-care, 21.4% vs. 7.1%; Usual activities, 24.3% vs. 5.0%; Pain/discomfort, 30.7% vs. 9.3%; Anxiety/depression, 25.0% vs. 9.3%. The mean (SD) change in EQ-5D VAS score from the study baseline to 4 weeks post-treatment was 6.7 (14.1) points, 9.6 (16.3) points and 8.6 (17.0) points in IC1, IC2 and IC3, respectively ( P < 0.0001 for all, t -test). The mean (SD) EQ-5D VAS score improved from 59.9 (18.9) at baseline to 70.5 (16.7) at the end of IC3, corresponding to a mean (SD) change of 10.5 (17.5) points ( P < 0.0001, t -test). Treatment of upper- and lower-limb spasticity with increasing incobotulinumtoxin A doses (up to 800 U) improved QoL over the study duration (three treatments). Data encourage the further evaluation of the impact of botulinum neurotoxin A treatment on QoL in patients with spasticity.

Systematic Literature Review of the Impact of Endocrine Monotherapy and in Combination with Targeted Therapy on Quality of Life of Postmenopausal Women with HR+/HER2- Advanced Breast Cancer.

A major treatment goal for advanced breast cancer (ABC) is to maintain or ideally improve patient quality of life (QoL). Given the changing disease landscape, this systematic literature review (SLR) aims to assess the impact of endocrine therapies (ET), including ET monotherapy (ET mono) and ET combined with targeted therapy (ET+TT), on QoL of women with HR+/HER2- ABC. A SLR was conducted to identify randomized controlled trials (RCTs) meeting the following criteria: (1) included ET mono or ET+TT, (2) reported QoL outcomes, (3) focused on women with HR+/HER2- ABC, and (4) published after 2007 (when standardized HER2 testing became available). The databases searched included MEDLINE, EMBASE, Cochrane Library, and key conference proceedings from 2013 to 2016. QoL outcomes for ET mono, ET+TT, and comparisons between the two were summarized from the identified trials. A total of 11 studies (representing 6 RCTs) were identified. The study populations included first-line (5 studies) and ET-failure settings (6 studies). Across settings, global health status (GHS) maintained or deteriorated slightly on these treatments during the trial period. Time to deterioration (TTD) in QoL measured by GHS was analyzed in 6 studies and 4 RCTs. In the first-line setting, reported median TTD in GHS was similar between ET mono and ET+TT (7.2-13.8months in ET mono; 11.1months in ET+TT). In the ET-failure setting, ET+TT showed significantly longer TTD vs. ET mono in GHS (median 5.6-8.4months in ET mono and 8.3-11.7months in ET+TT) and some additional domains. ET+TT users experienced similar QoL in the first-line and ET-failure setting relative to patients on ET mono. Moreover, ET+TT users experienced better QoL outcomes in some domains in the ET-failure setting relative to ET mono users. Novartis.