Patient-reported Quality Of Life Research Articles

Physical exercise during neoadjuvant chemotherapy for breast cancer as a mean to increase pathological complete response rates: Trial protocol of the randomized Neo-ACT trial.

IntroductionIn early breast cancer, neoadjuvant chemotherapy (NACT) is increasingly used. The proof of efficacy is pathologically complete response (pCR), i.e. the absence of invasive tumour in breast and lymph nodes at surgery. Today, pCR is a common endpoint in pharmaceutical trials since it is significantly associated with survival especially in triple-negative and HER2-positive subtypes. Apart from the mitigation of treatment-related toxicity and symptoms, physical exercise mediates anti-tumoral systemic effects associated with tumour regression in preclinical and clinical models. The aim of Neo-ACT is to test the hypothesis that physical exercise can improve pCR rates in breast cancer patients receiving NACT.MethodThe Neo-ACT trial is a prospective clinical trial, randomising T1-3N0-2 breast cancer patients planned for NACT to either a home-based physical exercise intervention supported by a mobile application or routine care. The primary endpoint is pCR; secondary endpoints are patient-reported quality of life, toxicity-related outcomes, and oncological outcomes such as Residual Cancer Burden, objective radiological tumour response, as well as overall, breast cancer-specific and disease-free survival at 2, 5 and 10 years. The intervention consists of a combination of high-intensity interval and resistance training of progressing intensity, and includes at least 150 min of moderate to vigorous physical activity per week, inclusive of two weekly 60-min exercise sessions. In order to show an improvement in pCR of 10%, a total of 712 participants need to be included in the analysis. The Neo-ACT has been registered at clinicaltrials.gov on January 11, 2022 (NCT05184582).Expected resultsIf Neo-ACT can prove the oncological efficacy of physical exercise, implementation of training programmes into NACT schedules will be pursued. The use of a digitally led exercise intervention aims to test the potential of such a strategy for use in rural areas and areas of limited resources.

Chronic gvhd dictionary—eurograft cost action initiative consensus report

Chronic graft versus host disease (cGVHD) affects patients after allogeneic hematopoietic stem cell transplantation (alloHSCT). This orphan disease poses a challenge for clinicians and researchers. The purpose of the cGVHD Dictionary is to provide a standardized structure for cGVHD databases on an international level, reconciling differences in data retrieval and facilitate database merging. It is derived from several consensus meetings of the EUROGRAFT consortium (European Cooperation in Science and Technology—COST Action CA17138) followed by a consensus process involving European Society for Blood and Marrow Transplantation (EBMT), US GvHD consortium and Center for International Bone Marrow Transplant Registry (CIBMTR). Databases used for the dictionary were: the National Institutes of Health (NIH) database, the Center for International Blood and Marrow Transplant Research, Applying Biomarkers to Minimize Long Term Effects of Childhood/Adolescent Cancer Treatment - Pediatric Blood and Marrow Transplant Consortium database, EBMT registry, the German-Austrian-Swiss GvHD registry, Italian Blood and Marrow Transplantation Society registry and Regensburg-Göttingen-Newcastle HSCT dataset. A four-part cGVHD Dictionary was formed based on the databases, consensus, and evidence in the literature. The Dictionary is divided into: (1) Patient characteristics, (2) Transplant characteristics, (3) cGVHD characteristics and (4) patient-reported quality of life, symptom burden and functional indicators.

Patient-Reported Quality of Life, Depression, Anxiety, and Physical Activity in Patients Receiving an Implantable Cardioverter-Defibrillator for Primary versus Secondary Prevention: A Single-Centre, Prospective, Observational Cohort Study.

Study objectives: The aim of the study was to evaluate of the quality of life, depression, anxiety levels, and physical activity in the groups after the implantation of an ICD or CRT-D. Methods: All subjects (111 CHF patients) underwent tests to assess the quality of life (NHP), the level of physical activity (IPAQ), the level of perceived stress (PSS), and the incidence of depression (BDI). Results: After the implantation, physical activity (PA) of the patients from the primary prevention (PP) group remains unchanged, whereas in the secondary prevention (SP) group, it decreases noticeably. Physical activity is lower in the SP group in comparison with the PP group. There are no statistically significant differences in the level of depression. The scores in the second part of the NHP questionnaire indicate that the SP group significantly more often declare problems with housework and with social life than the PP group. Conclusions: 1. The type of prevention does not have an influence on the level of anxiety, stress, or depression. 2. The patients after implantation as SP are physically less active; lower PA is associated additionally with the higher NYHA class and chronic kidney disease. 3. The quality of life of the patients from SP group is at a lower level than patients from PP group.

Comparative efficacy and acceptability of non-pharmacological interventions in fibromyalgia: Protocol for a network meta-analysis.

IntroductionAlthough several non-pharmacological interventions have been tested in the management of Fibromyalgia (FM), there is little consensus regarding the best options for the treatment of this health condition. The purpose of this network meta-analysis (NMA) is to investigate the comparative efficacy and acceptability of non-pharmacological interventions for FM, in order to assist clinical decision making through a ranking of interventions in relation to the most important clinical outcomes in these patients.Methods and analysisWe will perform a systematic search to identify randomised controlled trials of non-pharmacological interventions endorsed in guidelines and systematic reviews. Information sources searched will include major bibliographic databases without language or date restrictions (MEDLINE, Cochrane Library, EMBASE, AMED, PsycINFO and PEDro). Our primary outcomes will be pain intensity, patient-reported quality of life (QoL), and acceptability of treatment will be our secondary outcome. Risk of bias of the included trials will be assessed using the Cochrane risk of bias tool (RoB2). For each pairwise comparison between the different interventions, we will present mean differences (MDs) for pain intensity and QoL outcomes and Relative Risks (RRs) for acceptability, both with respective 95% confidence intervals (CIs). Initially, standard pairwise meta-analyses will be performed using a DerSimonian-Laird random effects model for all comparisons with at least two trials and then we will perform a frequentist NMA using the methodology of multivariate meta-analysis assuming a common heterogeneity parameter, using the mvmeta command and network suite in STATA. In the NMA, two different types of control group, such as placebo/sham and no intervention/waiting list will be combined as one node called “Control”. The competing interventions will be ranked using the P-score, which is the frequentist analogue of surface under the cumulative ranking curve (SUCRA) for the outcomes of interest at immediate- (intervention duration of up to 2 weeks), short- (over 2 weeks up to 12 weeks) and long-terms (over 12 weeks). The confidence in the results from NMA will be assessed using the Confidence in Network Meta‐analysis (CINeMA) framework.Ethics and disseminationThis work synthesises evidence from previously published studies and does not require ethics review or approval. A manuscript describing the findings will be submitted for publication in a peer-reviewed scientific journal.RegistrationOSF (DOI: 10.17605/OSF.IO/7MS25) and registered in the PROSPERO database (CRD42020216374).

Quality of life (QoL) in patients with heart failure with preserved ejection fraction (HFpEF): a systematic review

Abstract Introduction Heart failure with preserved ejection fraction (HFpEF) is associated with significant clinical unmet need as mortality and morbidity rates are high despite current treatments. Patient-reported quality of life (QoL) is an important and clinically relevant endpoint in patients with HF so understanding the impact of HFpEF on QoL is essential. Purpose The aim of this systematic review was to identify and summarize data on QoL in HFpEF. Methods Systematic searches of Medline, Medline Epub Ahead of Print (In-Process & Other Non-Indexed Citations), Embase, and EBM Reviews were conducted in October 2021. Congress proceedings from the past 3 years and reference lists of included publications were also searched. Publications were screened against eligibility criteria by title/abstract and then by full text. Eligibility criteria included clinical studies of any design reporting baseline QoL in adults with HFpEF published from 2016 onwards. Results The electronic database search identified 6,403 articles on HFpEF. After screening, 35 full publications reporting HFpEF and QoL were included. Study designs included observational cohort (n=17), cross-sectional (n=3), and post-hoc analyses of interventional studies (n=15). Tools used to measure QoL included KCCQ (n=17), MLHFQ (n=12), EQ-5D-VAS (n=8), SF-12 (n=2), SF-36 (n=2), and EHFSQ-1 (n=1). Health-state utility values (HSUVs) were captured using EQ-5D in four studies. HSUVs in HFpEF ranged from 0.67–0.74, indicating a substantial QoL burden. Eleven studies using various instruments compared QoL in patients with HFpEF vs non-HFpEF (Table 1). Of these, five reported statistically significantly poorer QOL in HFpEF vs non-HFpEF, one reported statistically significantly poorer QOL in non-HFpEF vs HFpEF and five found no statistically significant difference between HF subtypes. Four studies compared QoL in men vs women with three reporting statistically significantly worse QoL in women. Three studies examined the impact of comorbidities, and all reported statistically significantly poorer QoL in patients with comorbid atrial fibrillation, diabetes, or metabolic syndrome. Two studies examined the effect of age; one reported that, vs elderly patients (age ≥85 years), younger patients (age ≤55 years) with HFpEF had statistically significantly worse QoL despite having fewer comorbidities; in contrast, the second study reported that younger patients (age ≤55 years) had statistically significantly better QoL than elderly patients (age ≥75 years). Two studies reported on hospitalizations, with one reporting no difference in QoL between patients who had previously been hospitalized and those who had not and the other reporting that readmission for HF correlated with QoL. Conclusions QoL burden among HFpEF patients is substantial. QoL may be affected by age, comorbidities, gender, and prior hospitalization, and may be associated with poorer outcomes. QoL in HFpEF is generally poorer than in non-HFpEF. Funding Acknowledgement Type of funding sources: Private company. Main funding source(s): AstraZeneca

Patient-reported hope, quality of life, symptom burden, coping, and financial toxicity in early-phase clinical trial participants.

275 Background: Early phase clinical trials (EP-CTs) investigate novel treatment options in oncology, with recent advances in personalized therapy leading to improved outcomes and offering hope to patients with cancer. However, little research has sought to understand associations of patient-reported hope with quality of life (QOL), symptom burden, coping, and financial toxicity in EP-CT participants. Methods: We prospectively enrolled consecutive adults with cancer participating in EP-CTs at Massachusetts General Hospital from 04/2021-05/2022. Participants completed baseline surveys prior to treatment initiation that assessed hope (Herth Hope Index [HHI], higher scores indicate greater hope), QOL (Functional Assessment of Cancer Therapy-General), symptom burden (physical: Edmonton Symptom Assessment System [ESAS]; psychological: Patient Health Questionaire-4 [PHQ4]), coping (Brief COPE: self-blame, acceptance, denial, emotional support, active, behavioral disengagement), and financial toxicity (COST tool, higher scores indicate greater financial wellbeing). We used regression models to determine associations of hope scores with patient-reported QOL, symptom burden, coping, and financial toxicity. Results: Of 157 eligible patients, we enrolled 129 (enrollment rate 82.2%, median age = 62.5 years [range 33.0-83.0], 53.9% female, and 96.0% metastatic cancer). Most common cancer types were gastrointestinal (37.5%), breast (20.3%), lung (8.6%), and head and neck (7.8%). Patients had an average HHI score of 27.5 (range 15.3 – 36.0), with 30.5% reporting high levels of hope. We found associations of higher hope scores with better QOL (B = 0.24, p < 0.001) and lower symptom burden (ESAS-physical: B = -0.14, p < 0.001; PHQ4-depression: B = -2.07, p < 0.001; PHQ4-anxiety: B = -0.93, p = 0.001). We also found that hope scores were associated with patients’ coping (self-blame [B = -1.44, p < 0.001]; acceptance [B = 1.40, p < 0.001], denial [B = -1.12, p = 0.004], emotional support [B = 0.99, p < 0.001], active [B = 1.02. p = 0.001], behavioral disengagement [B = -2.52, p < 0.001]). Lastly, we found that higher hope scores were associated with greater financial wellbeing (B = 0.11, p = 0.026). Conclusions: In this prospective cohort study, we demonstrated a substantial proportion of EP-CT participants had high baseline hope and identified associations of hope scores with other important patient-reported outcomes. Specifically, we found novel associations of higher hope scores with better QOL, lower symptom burden, more adaptive coping mechanisms, and greater financial wellbeing, underscoring the importance of targeting these patient-reported outcomes when seeking to enhance the care experience of EP-CT participants.

MM-253 Efficacy, Safety, and Quality of Life (QoL) in Patients With Newly Diagnosed Multiple Myeloma (NDMM) After In-Class Transition (iCT) From Parenteral Bortezomib-Based Induction Therapy to All-Oral Ixazomib-Lenalidomide-Dexamethasone (IRd): Data From the Phase 4, Community-Based US MM-6 Study (Fully Accrued Cohort)

Achieving prolonged therapy with parenteral proteasome inhibitors (PIs) in clinical practice can be challenging. To increase the duration of PI-based therapy, improve outcomes, and maintain QoL. Prospective study (NCT03173092) evaluating iCT from parenteral bortezomib-based induction to all-oral IRd. IRd was received for up to 39 cycles or until progression/toxicity (Manda CLML 2020). Interim data (N=101) were previously reported (Rifkin ASH 2021). We report results from the fully accrued study cohort (enrollment completed, May 2021; N=141). US community sites. Transplant-ineligible/delayed-transplant (≥24 months) NDMM patients with ≥stable disease after 3 cycles of bortezomib-based induction. Primary endpoint: 2-year progression-free survival (PFS) rate. Other endpoints included: key secondary, overall response rate (ORR; partial response [PR]+very good PR [VPGR]+complete response [CR]) and duration of treatment (DOT); secondary, overall survival (OS), safety, and QoL. At Feb-28-2022, 140 patients (median age, 72.5 years [range, 48-90]; 79% ≥65 years; 42% ≥75 years) had received IRd; 94% had ≥1 comorbidity at IRd initiation. Median follow-up was 20.0 months. 2-year PFS was 69% (95% confidence interval [CI]: 59-77%). ORR was 62% (CR, 8%; VGPR, 24%; PR, 30%) after bortezomib-based induction, increasing to 78% (CR, 33%; VGPR, 27%; PR, 18%) after iCT. At data cut-off, 28 patients (20%) were ongoing on IRd, median DOT with IRd was 10.0 months, and median duration of all PI-based treatment was 12.7 months. 2-year OS was 85% (95% CI: 76-90%). 66% of patients had grade ≥3 treatment-emergent adverse events (TEAEs) (treatment-related, 36%), 44% had serious TEAEs (treatment-related, 14%), and 6 on-study deaths had occurred. Most common TEAEs (any-grade/grade ≥3) were diarrhea (48%/9%), peripheral neuropathies not elsewhere classified (39%/4%), and fatigue (34%/4%). Modification/discontinuation of ixazomib, lenalidomide, or dexamethasone due to TEAEs occurred in 59%/15% of patients. Patient-reported QoL (EORTC QLQ-C30 Global Health Status/QoL score) and treatment satisfaction (TSQM-9 Global Satisfaction, Effectiveness, and Convenience scores) were maintained on IRd. Improved responses and promising PFS and OS were seen following iCT to IRd in these mostly elderly, community-treated patients with NDMM and comorbidities, with no adverse impact on QoL/treatment satisfaction. Safety results were consistent with previous reports.

Differential changes in self-reported quality of life in elderly populations after diagnosis of a genitourinary malignancy.

The time of cancer diagnosis is a major event during which quality of life (QOL) can be affected and represents a crucial time to identify patients at high risk of decline. We sought to compare the differential effects of the diagnosis of 3 major urologic malignancies on QOL. The Surveillance, Epidemiology, and End Results-Medicare Health Outcomes Survey database was queried for patients who completed a QOL questionnaire (SF-36 or VR-12) before and after a diagnosis of bladder, kidney, or prostate cancer. Primary outcome measures were the mental component summary (MCS), and physical component summary (PCS) scores. Mixed effects linear regression was performed with cancer diagnosis as the primary variable of interest, with race and cardiovascular comorbidity status included as potentially confounding independent variables. There were 3,258 patients with urologic cancers. Both MCS and PCS scores dropped after diagnosis in all disease states. Bladder and kidney cancer patients demonstrated the greatest decline in MCS score (-1.762 points, 95% CI-2.571 to -0.952, P < 0.001) and PCS score (-3.769 points, 95% CI-5.042 to -2.496, P < 0.001), respectively, after adjustment for potential confounders. By contrast, prostate cancer patients demonstrated the smallest decline in both domains. Race and cardiovascular comorbidity status were independently associated with QOL, with an association 2 to 3 times greater than that of cancer diagnosis. Diagnosis of a urologic cancer was associated with a decline in patient-reported QOL, particularly in those with bladder or kidney cancer. Changes in physical health were more prominent than in mental health. Race and cardiovascular comorbidity status influenced QOL domains to a greater extent than specific urologic cancer diagnosis.

Minimally invasivE versus open total GAstrectomy (MEGA): study protocol for a multicentre randomised controlled trial (DRKS00025765)

IntroductionThe only curative treatment for most gastric cancer is radical gastrectomy with D2 lymphadenectomy (LAD). Minimally invasive total gastrectomy (MIG) aims to reduce postoperative morbidity, but its use has not...

A single-arm feasibility phase II study of EMF (erlotinib + methotrexate + 5-fluorouracil) regimen in platinum-refractory recurrent/metastatic head and neck squamous cell carcinoma (R/M HNSCC).

Head and neck squamous cell carcinoma (HNSCC) is a huge burden in India with the majority of patients presenting in advanced unresectable stages. Innovative, low-cost but efficacious regimens that can be easily administered in the outpatient setting are the need of the hour. We envisaged assessing whether a readily available triplet therapy of erlotinib + methotrexate + 5-fluorouracil (EMF) is efficacious in terms of extending life and maintaining the quality of life in such patients. This was a single-arm, phase II, investigator-initiated interventional study. Thirty-five platinum-resistant/refractory patients of HNSCC were treated with a combination of erlotinib 150 mg, methotrexate 40 mg/m2 (d1, d8) and 5-fluorouracil 500 mg/m2 (d1, d8) every 28 days till progression or unacceptable toxicities. The primary endpoint was overall response rates (ORRs) at 3 months; additional endpoints were disease control rate (DCR) at 3 months, overall survival (OS) and progression-free survival (PFS), safety and patient-reported quality of life. The ORR and DCR at 3 months were 45.7% and 68.5%, respectively. The PFS was 5 months (95% confidence interval (95% CI): 3.9-6 months) and the OS was 9 months (95% CI: 7.4-10.5 months). The 3- and 6-month PFS rates were 86% ± 6% and 45% ± 9%, respectively, while the OS rates at 3 and 6 months were 91% ± 5% and 68% ± 8%, respectively. Rash, mucositis and fatigue were common adverse events occurring in 23 (65%), 14 (40%) and 9 (25.7%), respectively. The most common grade 3 events seen were rash in 5 (14.2%) and diarrhoea in 2 (5.7%). Clinically significant improvement from baseline was seen in many domains of Quality of Life Core Questionnaire and Quality of Life Head and Neck Module. The triplet regimen of EMF is a feasible and safe therapeutic option in patients with platinum-resistant/refractory HNSCC. It has demonstrated favourable response rates and improvement in quality of life; however, a randomised phase III study would add more robust value (NCT: CTRI/2020/02/023378).

Karnofsky performance status and visual analogue scale scores are simple indicators for quality of life in long-term AYA survivors who received allogeneic hematopoietic stem cells transplantation in childhood

The purpose of this study was to investigate Karnofsky performance status (KPS) scores and visual analogue scale (VAS) scores to explain which domains in the standardized self-reported quality of life (QOL) are instrumental for long-term hematopoietic stem cell transplantation (HSCT) survivors. We conducted a nationwide cross-sectional questionnaire study on 221 survivors with allogeneic-HSCT in 28 pediatric centers. Patient-reported QOL was assessed at a single time point using the 36-item Short-Form Survey (SF-36), the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT), and VAS scores. KPS scores were significantly correlated with both physical and role component summary scores of the SF-36, while the VAS provided by the patient (VASpt) was significantly correlated with the mental component summary score of the SF-36 and many subscales of the FACT-BMT. The VAS provided by the participants' attending physician (VASdoc) was correlated well with KPS scores. A VASpt score more than 40% lower than KPS scores suggested mental health problems. In conclusion, KPS scores might be considered as an indicator for physical and role/social components and VASpt score as an indicator for mental components and HSCT-specific QOL.

The Role of Parkinson Nurses for Personalizing Care in Parkinson's Disease: A Systematic Review and Meta-Analysis.

Background:Quality of life (QoL) of persons with Parkinson’s disease (PD) is diminished by (non-)motor symptoms, that require personalized care. Parkinson Nurses (PN) may be pivotal promoting tailored care offerings. This systematic review and meta-analysis investigates PD care models and aims at furnishing current concepts of PN to offer personalized care.Objective:The purpose of this study is to identify the various roles and functions that PN may hold for personalized PD care.Methods:We performed a systematic literature review, utilizing: PubMed, Web of Science, The Cochrane Library, and PsycINFO. The review qualitatively evaluated articles, which described personalized care models involving PNs and was guided by the personalized care management model. A meta-analysis compared patient-reported QoL (quantified using the 39-item Parkinson’s Disease Questionnaire) between personalized care interventions involving PN versus standard care with.Results:Twenty-seven publications were identified, including six randomized, controlled trials ascertaining with health related QoL (n = 1830 PwPs). The qualitative evaluation revealed that PN contribute to all aspects of personalized care. The meta-analysis showed no improved QoL in personalized care models compared to standard care, thought a great heterogeneity among study design and interventions was outlined (Standardized Mean Difference = –0.8935; 95% Confidence Interval, –2.1177 to 0.3307; z = –1.43, p = 0.1526).Conclusion:PN fulfil important functions in personalized PD care. For the future, a clear role definition will be necessary to adjust training for PN across healthcare systems and care settings but especially to realize their full potential for PD care.

EP04.02-004 The Patient Impact of Liquid Biopsy - Health-Related Quality of Life in Patients Undergoing Liquid Biopsy for Advanced Non-Small Cell Lung Cancer

Liquid biopsy (LB) with plasma cell-free DNA (cfDNA) next-generation sequencing (NGS) leads to more complete molecular profiling with faster turnaround time than tumor tissue (TT) profiling alone and may be cost-effective. The impact of comprehensive LB testing at diagnosis or progression on patient’s quality of life (QOL) in advanced NSCLC has not been previously reported. In this prospective multicentre Canadian study using LB to guide treatment, we tested the hypothesis that by identifying more candidates for targeted therapy, patient-reported QOL outcomes would be improved.

Impact of ixazomib-lenalidomide-dexamethasone therapy on overall survival in multiple myeloma patients: Analysis of the emerging-markets subgroup of the TOURMALINE-MM1 trial.

Ixazomib-lenalidomide-dexamethasone (ixazomib-Rd) showed clinical efficacy over placebo-Rd in patients with relapsed/refractory multiple myeloma (MM) in the TOURMALINE-MM1 trial. Over a median follow-up of ∼85months, as patients showed disease progression, they received subsequent novel therapies that confounded the overall survival (OS) benefit. Here, we conducted a post hoc analysis in 148 patients from seven countries defined as emerging markets, with limited access to novel therapies for MM during the trial period, to describe the impact of these therapies on OS. Patients were randomised to ixazomib-Rd (n = 71) or placebo-Rd (n = 77). The median progression-free survival (PFS) was 18.7 versus 10.2months, with ixazomib-Rd versus placebo-Rd (hazard ratio [HR], 0.504; p = 0.008) demonstrating a statistically significant improvement as observed in the primary trial. The median OS improved by 32.6months with ixazomib-Rd over placebo-Rd (63.5 vs. 30.9months; HR, 0.794; p = 0.261); however, the statistically significant benefit seen in PFS was not observed for OS. Improvement with ixazomib-Rd over placebo-Rd was observed in overall response (81.7% vs. 64.9%; odds ratio [OR], 2.38; p = 0.019) and complete response (22.5% vs. 3.9%; OR, 7.57; p < 0.001). Patient-reported quality of life and use of subsequent therapies were similar across treatment groups. No new safety concerns were identified. Compared with the main cohort, median OS was 10months longer with ixazomib-Rd and 21months shorter with placebo-Rd in this subgroup, indicating a clinically meaningful survival benefit of ixazomib-Rd treatment in this patient population with limited access to subsequent novel therapies.

Outcomes and toxicities after proton partial breast radiotherapy for early stage, hormone receptor positive breast cancer: 3-Year results of a phase II multi-center trial

PurposeProton therapy (PT) for partial breast irradiation (PBI) in early-stage breast cancer can decrease morbidity versus photon PBI with superior organs-at-risk sparing. We report 3-year outcomes of the first prospective, multicenter, phase II trial of proton PBI. Methods and MaterialsThis Proton Collaborative Group phase II trial (PCG BRE007-12) recruited women ≥ 50 years with node-negative, estrogen receptor (ER)-positive, ≤3cm, invasive ductal carcinoma (IDC) or ductal carcinoma in situ undergoing breast conserving surgery followed by proton PBI (40 Gy(RBE), 10 daily fractions). Primary endpoint was freedom from ipsilateral breast cancer recurrence. Adverse events were prospectively graded using CTCAEv4.0. Breast Cancer Treatment Outcome Scale (BCTOS) assessed patient-reported quality of life (PRQOL). ResultsThirty-eight evaluable patients enrolled between 2/2013–11/2016. Median age was 67 years (range 50–79); 55 % had left-sided disease, and median tumor size was 0.9 cm. Treatment was delivered in ≥ 2 fields predominantly with uniform scanning PT (n = 37). At 35-month median follow-up (12–62), all patients were alive, and none had local, regional or distant disease progression. One patient developed an ER-negative contralateral IDC. Seven grade 2 adverse events occurred; no radiotherapy-related grade ≥ 3 toxicities occurred. Changes in BCTOS subdomain mean scores were maximum 0.36, indicating no meaningful change in PRQOL. Median heart volume receiving 5 Gy (V5Gy), lung V20Gy, and lung V10Gy were 0 %, 0 % and 0.19 %, respectively. ConclusionAt 3 years, proton PBI provided 100 % cancer control for early-stage, ER-positive breast cancer. Toxicities are minimal, and PRQOL remains acceptable with continued follow-up. These findings support PT as a safe and effective PBI delivery option.

Impaired insight in schizophrenia: impact on patient-reported and physician-reported outcome measures in a randomized controlled trial

BackgroundImpaired insight poses a challenge in the treatment of patients with schizophrenia because of its potential to jeopardize therapeutic engagement and medication adherence. This study explored how insight impairment, graded from none to extreme, is related to patient-reported mental health status, depression, and neurocognition in schizophrenia.MethodsIn a post hoc analysis of the Clinical Antipsychotic Trials of Intervention Effectiveness (CATIE) study (NCT00014001), insight was measured using the Positive and Negative Syndrome Scale (PANSS) Item G12 (lack of insight). Additional assessments for this analysis included the 12-Item Short-Form Health Survey (SF-12) Mental Component Summary (MCS), physician- and patient-reported Clinical Global Impression–Severity (CGI-S), MATRICS Consensus Cognitive Battery, and Calgary Depression Scale for Schizophrenia. Relationships between patient-reported outcomes and PANSS total and Item G12 ratings were evaluated.ResultsAmong 1431 CATIE study participants in this analysis, increasingly impaired insight at baseline was significantly associated with better patient-reported quality of life (QoL), lower baseline depression, and greater divergence between physician- and patient-reported illness severity. Patients with more severely impaired insight reported milder illness compared with physician reports, particularly those with moderate-severe to extreme impairment (PANSS Item G12 rating ≥ 5), approximately 10% (138/1431) of CATIE participants. For the 90% of patients with PANSS Item G12 ratings < 5, patient-reported QoL decreased with increasing symptoms. SF-12 MCS scores were linearly related to baseline PANSS total score only in patients with PANSS total score < 90 (moderately ill or better), and better symptom scores were associated with higher QoL. No significant relationship between insight and neurocognition was observed.ConclusionsIn the small subgroup (10%) of CATIE study patients with schizophrenia and PANSS Item G12 ratings ≥5, moderate-severe–severe/extreme insight impairment was associated with significantly more positive perception of QoL and illness severity by the patient versus the treating physician. This was not observed in the remaining 90% of patients with normal to moderately impaired insight, suggesting that poor insight as a threat to the validity of self-report is uncommon.

Effects and Processes of an mHealth Intervention for the Management of Chronic Diseases: Prospective Observational Study.

BackgroundMobile health (mHealth) interventions for self-management are a promising way to meet the needs of patients with chronic diseases in primary care practices. Therefore, an mHealth intervention, TelePraCMan, was developed and evaluated for patients with type 2 diabetes mellitus, chronic obstructive pulmonary disease, high blood pressure, or heart failure in a German primary care setting. TelePraCMan entails a symptom diary, an appointment manager, a manager to document goals, and a warning system. The app should foster the self-management of participating patients.ObjectiveWe aimed to examine the effects of TelePraCMan on patient activation and quality of life and explored the underlying contextual factors, impacts, and degree of implementation.MethodsIn a prospective observational study design, we collected data by using interviews and written questionnaires from participating patients (intervention and control groups) and primary care workers (physicians and practice assistants). The primary outcomes of interest were patient-reported quality of life (12-Item Short Form Survey) and patient activation (patient activation measure). The quantitative analysis focused on differences between patients in the intervention and control groups, as well as before (T0) and after (T1) the intervention. Interviews were analyzed by using qualitative content analysis via MAXQDA (VERBI GmbH).ResultsAt baseline, 25 patients and 24 primary care workers completed the questionnaire, and 18 patients and 21 primary care workers completed the follow-up survey. The patients were predominantly male and, on average, aged 64 (SD 11) years (T0). The primary care workers were mostly female (62%) and, on average, aged 47 (SD 10) years (T0). No differences were observed in the outcomes before and after the intervention or between the intervention and control groups. In the additional interviews, 4 patients and 11 primary care workers were included. The interviewees perceived that the intervention was useful for some patients. However, contextual factors and problems with implementation activities negatively affected the use of the app with patients. The main reasons for the low participation were the COVID-19 pandemic and the target group, which seemed to have less interest in mHealth; the interviewees attributed this to the older age of patients. However, the respondents felt that the app would be better accepted in 5 or 10 years.ConclusionsAlthough the TelePraCMan app was rated as very good and important by the participants, few patients used it. The digital intervention was hardly implemented and had limited impact in the current setting of German primary care.Trial RegistrationGerman Clinical Trials Register DRKS00017320; https://tinyurl.com/4uwrzu85

Quality-of-Life Outcomes and Toxicity Profile Among Patients With Localized Prostate Cancer After Radical Prostatectomy Treated With Stereotactic Body Radiation: The SCIMITAR Multicenter Phase 2 Trial

Postoperative radiation therapy (RT) is an underused standard-of-care intervention for patients with prostate cancer and recurrence/adverse pathologic features after radical prostatectomy. Although stereotactic body RT (SBRT) is a well-studied and convenient option for definitive treatment, data on the postprostatectomy setting are extremely limited. The purpose of this study was to evaluate short-term physician-scored genitourinary (GU) and gastrointestinal (GI) toxicities and patient-reported outcomes after postprostatectomy SBRT. The SCIMITAR trial was a phase 2, dual-center, open-label, single-arm trial that enrolled patients with postoperative prostate-specific antigen >0.03 ng/mL or adverse pathologic features. Coprimary endpoints were 4-year biochemical recurrence-free survival, physician-scored acute and late GU and GI toxicities by the Common Terminology Criteria for Adverse Events (version 4.03) scale, and patient-reported quality-of-life (QOL) outcomes, as represented by the Expanded Prostate Cancer Index-26 and the International Prostate Symptom Score. Patients received SBRT 30 to 34 Gy/5 fractions to the prostate bed ± bed boost ± pelvic nodes with computed tomography (CTgRT) or magnetic resonance imaging guidance (MRgRT) in a nonrandomized fashion. Physician-scored toxicities and patient-reported QOL outcomes were collected at baseline and at 1, 3, and 6 months of follow-up. Univariable and multivariable analyses were performed to evaluate predictors of toxicities and QOL outcomes. One hundred participants were enrolled (CTgRT, n=69; MRgRT, n=31). The median follow-up was 29.5 months (CTgRT: 33.3 months, MRgRT: 22.6 months). The median (range) prostate bed dose was 32 (30-34) Gy. Acute and late grade 2 GU toxicities were both 9% while acute and late grade 2 GI toxicities were 5% and 0%, respectively. Three patients had grade 3 toxicity (n=1 GU, n=2 GI). No patient receiving MRgRT had grade 3 GU or grade ≥2 GI toxicity. Compared with CTgRT, MRgRT was associated with a 30.5% (95% confidence interval, 11.6%-49.5%) reduction in any-grade acute GI toxicity (P=.006). MRgRT was independently associated with improved any-grade GI toxicity and improved bowel QOL. Postprostatectomy SBRT was well tolerated at short-term follow-up. MRgRT may decrease GI toxicity. Longer toxicity and/or efficacy follow-up and randomized studies are needed.

Two-Year Functional Outcomes of Operative vs Nonoperative Treatment of Completely Displaced Midshaft Clavicle Fractures in Adolescents: Results From the Prospective Multicenter FACTS Study Group

Background: The optimal treatment of midshaft clavicle fractures is controversial. Few previous comparative functional outcome studies have investigated these fractures in adolescents, the most commonly affected epidemiologic subpopulation. Purpose/Hypothesis: The purpose was to prospectively compare the outcomes of operative versus nonoperative treatment in adolescents with completely displaced midshaft clavicle fractures. The study hypothesis was that surgery would yield superior outcomes. Study Design: Cohort study; Level of evidence, 2. Methods: Patients aged 10 to 18 years treated for a midshaft clavicle fracture over a 5-year period at 1 of 8 pediatric centers were prospectively screened, with independent treatment decisions determined by individual musculoskeletal professionals. Demographics, radiographic clinical features, complications, and patient-reported outcomes (PROs) were prospectively recorded for 2 years. Regression and matching techniques were utilized to adjust for potential age- and fracture severity–based confounders for creation of comparable subgroups for analysis. Results: Of 416 adolescents with completely displaced midshaft clavicle fractures, 282 (68) provided 2-year PRO data. Operative patients (n = 88; 31%) demonstrated no difference in sex (78% male) or athletic participation but were older (mean age, 15.2 vs 13.5 years; P < .001), had more comminuted fractures (49.4% vs 26.3%; P < .001), and had greater fracture shortening (25.5 vs 20.7 mm; P < .001) than nonoperative patients (n = 194; 69%). There was no difference in mean PRO scores or rates of “suboptimal” scores (based on threshold values established a priori) between the operative and nonoperative treatment groups (American Shoulder and Elbow Surgeons, 96.8 vs 98.4; shortened version of the Disabilities of the Arm, Shoulder and Hand, 3.0 vs 1.6; EuroQol [EQ] visual analog scale, 93.0 vs 93.9; EQ–5 Dimensions index, 0.96 vs 0.98), even after regression and matching techniques adjusted for confounders. Operative patients had more unexpected subsequent surgery (10.4% vs 1.4%; P = .004) and clinically significant complications (20.8% vs 5.2%; P = .001). Overall, nonunion (0.4%), delayed union (1.9%), symptomatic malunion (0.4%), and refracture (2.6%) were exceedingly rare, with no difference between treatment groups. Conclusion: Surgery demonstrated no benefit in patient-reported quality of life, satisfaction, shoulder-specific function, or prevention of complications after completely displaced clavicle shaft fractures in adolescents at 2 years after injury. Registration: NCT04250415 (ClinicalTrials.gov identifier).

Stereotactic Ablative Radiation for Systemic Therapy–naïve Oligometastatic Kidney Cancer

BackgroundEvidence-based guidelines for the management of systemic therapy–naïve oligometastatic renal cell carcinoma (RCC) are lacking. ObjectiveTo evaluate the potential of stereotactic ablative radiotherapy (SAbR) to provide longitudinal disease control while preserving quality of life (QOL) in patients with systemic therapy–naïve oligometastatic RCC. Design, setting, and participantsRCC patients with three or fewer extracranial metastases were eligible. SAbR was administered longitudinally to all upfront and, as applicable, subsequent metastases. Outcome measurements and statistical analysisThis prospective phase II single-arm trial was powered to achieve a primary objective of freedom from systemic therapy for >1 yr in >60% of patients (using the Clopper and Pearson methodology). Secondary endpoints included progression-free survival (PFS), defined as the time from first SAbR to progression not amenable to SAbR (local failure at SAbR-treated sites, new metastases not amenable to SAbR, more than three new metastases, or brain metastases); patient-reported QOL metrics; local control (LC) rates; toxicity; cancer-specific survival (CSS); and overall survival (OS). Results and limitationsTwenty-three patients received SAbR to 33 initial and 57 total sites. The median follow-up was 21.7 mo (interquartile range 16.3–30.3). Exceeding the prespecified 60% benchmark, freedom from systemic therapy at 1 yr was 91.3% (95% confidence interval [CI]: 69.5, 97.8). One-year PFS was 82.6% (95% CI: 60.1, 93.1). QOL was largely unaffected. LC was 100%. There were no grade 3/4 toxicities, but there was one death due to immune-related colitis 3 mo after SAbR while on subsequent checkpoint inhibitor therapy, where a SAbR contribution could not be excluded. One-year OS was 95.7% (95% CI: 72.9, 99.4); one-year CSS was 100%. ConclusionsSAbR for oligometastatic RCC was associated with meaningful longitudinal disease control while preserving QOL. These data support further evaluation of SAbR for systemic therapy–naïve oligometastatic RCC. Patient summarySequential stereotactic radiation therapy can safely and effectively control metastatic kidney cancer with limited spread for over a year without compromising patients’ quality of life.