Changes In QoL Research Articles

Health-related quality of life in patients with RAS wild-type metastatic colorectal cancer treated with panitumumab-based first-line treatment strategy: A pre-specified secondary analysis of the Valentino study

BackgroundQuality of life (QoL) patient-reported outcomes (PROs) data from pivotal first-line trials in metastatic colorectal cancer (mCRC) are poor. The Valentino study showed that de-escalation to single-agent panitumumab after 4-month induction with panitumumab-FOLFOX is inferior to panitumumab-5-FU/LV in patients with RAS wild-type mCRC, although slightly reducing toxicity. We report QoL, a secondary end-point. MethodsPROs were assessed by European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire – Core 30 (QLQ-C30), EORTC QLQ-CR29, EuroQol EQ-5D questionnaires, at baseline and every 8 weeks until disease progression. First two evaluations correspond to induction treatment (identical in both arms), while subsequent to maintenance. To describe QoL changes over time, mean changes from baseline at each time point were calculated in overall population. To compare maintenance between two arms, mean changes and proportion of improved/stable/worse patients versus baseline were compared for each item. ResultsIn arm A/B, 91.5%/92.0% of enrolled patients completed questionnaires at baseline. No significant differences in the two arms were reported in compliance, baseline scores and mean changes versus baseline for the three questionnaires during maintenance (24/32/40 weeks). Overall, mean changes versus baseline showed an early deterioration during induction with partial recovering during maintenance for global QoL, functional scales and several symptoms/items of QLQ-C30 (fatigue, nausea/vomiting, appetite loss, diarrhoea) and QLQ-CR29 (body image, dry mouth, hair loss, taste, faecal incontinence, sore skin), and EQ-5D Visual Analogue Scale (VAS) score. ConclusionIn patients with RAS wild-type mCRC, induction with oxaliplatin-containing chemotherapy plus anti-EGFRs induces a transient significant QoL deterioration. After induction phase, treatment deintensification determines an overall recovery of health-related QoL, besides the expected prevention of oxaliplatin-related neurotoxicity.

Trifluridine/tipiracil plus bevacizumab in patients with untreated metastatic colorectal cancer ineligible for intensive therapy: the randomized TASCO1 study

We designed an open-label, noncomparative phase II study to assess the safety and efficacy of first-line treatment with trifluridine/tipiracil plus bevacizumab (TT-B) and capecitabine plus bevacizumab (C-B) in untreated patients with unresectable metastatic colorectal cancer (mCRC) who were not candidates for combination with cytotoxic chemotherapies. From 29 April 2016 to 29 March 2017, 153 patients were randomly assigned (1:1) to either TT-B (N= 77) or C-B (N= 76). The primary end point was progression-free survival (PFS). The primary PFS analysis was performed after 100 events (radiological progression or death) were observed. Secondary end points included overall survival (OS), quality of life (QoL; QLQ-C30 and QLQ-CR29 questionnaires), and safety. Median (range) duration of treatment was 7.8 (6.0-9.7) months and 6.2 (4.1-9.1) months in the TT-B and C-B groups, respectively. Median (range) PFS was 9.2 (7.6-11.6) and 7.8 (5.5-10.1) months, respectively. Median (range) OS was 18 (15.2 to NA) and 16.2 (12.5 to NA) months, respectively. QoL questionnaires showed no relevant changes over time for either treatment. Therapies were well tolerated. Patients receiving TT-B had more grade ≥3 neutropenia (47% versus 5% with C-B). Patients receiving C-B had more grade ≥3 hand-foot syndrome (12% versus 0% with TT-B) and grade ≥3 diarrhea (8% versus 1% with TT-B), consistent with the known safety profiles of these agents. TT-B treatment showed promising clinical activity in untreated patients with unresectable mCRC ineligible for intensive therapy, with an acceptable safety profile and no clinically relevant changes in QoL. NCT02743221 (ClinicalTrials.gov).

Efficacy of Maitland joint mobilization technique on pain intensity, mouth opening, functional limitation, kinesiophobia, sleep quality and quality of life in temporomandibular joint dysfunction following bilateral cervicofacial burns

BackgroundBilateral cervicofacial burn contracture initially affects facial expressions and later affects tempero mandibular joint (TMJ) function of the patient. It further tightens the ligaments and capsules around the TMJ and leads to tempero mandibular dysfunction. The consequences of temporomandibular joint dysfunction will be pain, limitation of mouth opening, functional disability and poor quality of life (Qol). However, studies examining the efficacy of Maitland joint mobilization technique in this population are lacking. ObjectivesTo find the short term effects of Maitland joint mobilization technique on pain, mouth opening, functional limitation, kinesiophobia, sleep quality and Quality of life in tempero mandibular joint dysfunction following bilateral cervicofacial burns. MethodsThirty subjects with temporomandibular joint dysfunction following cervicofacial burn (N=30) were allocated to Maitland joint mobilization group (n=15) and home based training group (n=15) randomly to receive Maitland joint mobilization technique and home based training for 4 weeks. All the participants received ultrasound therapy as common treatment. Primary (Numeric Pain Rating Scale – NPRS, maximal mouth opening – MMO & Tempero mandibular disability index – TDI) and secondary (Tampa Scale of Kinesiophobia – TSK-17, Sleep quality questionnaire – SSQ & Global Rating of Change – GRC) outcome measures were measured at baseline, after four weeks and three months follow up. Repeated measures of ANOVA and independent t test were performed for analyzing the dependent variables in both groups. ResultsBaseline demographic characters and study variables show homogenous distribution between the groups (p>0.05). Four weeks following different training protocols Maitland joint mobilization group shows more significant changes in pain intensity, mouth opening, functional limitation, kinesiophobia, sleep quality and Qol than home based training group (p≤0.05). Also Maitland joint mobilization group show significant improvement in study variables than home based training group (p≤0.05) at three months follow up. ConclusionBoth groups improved over time, however, differences between the groups were noticed small. However, physiotherapy management which includes Maitland joint mobilization technique and therapeutic exercise program has an effective strategy in the treatment of tempero mandibular joint dysfunction following cervicofacial burns.

Quality of life improved for patients after starting dialysis but is impaired, initially, for their partners: a multi-centre, longitudinal study

BackgroundQuality of life (QOL) is important to patients with end stage renal disease and their partners. Despite the first 12 weeks being a critical time in the treatment pathway, limited research exists which examines how the transition onto dialysis impacts QOL. In this study we measured QOL in patients and their partners at pre-dialysis and over the first 12 weeks on dialysis to investigate QOL during this crucial period.MethodsPatients and their partners, recruited from 10 renal units in England, completed questionnaires at pre-dialysis (n = 166 participants, 83 couples), 6 weeks (n = 90 participants, 45 couples) and 12 weeks (n = 78, 39 couples) after starting dialysis. On each occasion participants completed a QOL questionnaire (WHOQOL-BREF). Multilevel modelling accommodated the nested structure of couples with repeated measures within participants. Three-level random intercept models estimated changes in WHOQOL general QOL and its four domains (Physical, Psychological, Social and Environment). Two-level random intercept models assessed the relationship between baseline clinical and socio-demographic variables with changes in general QOL.ResultsPatients reported positive changes in general QOL from pre-dialysis to 6 weeks (β = 0.42, p < 0.001, 95% CI 0.19, 0.65) and from pre-dialysis to 12 weeks (β = 0.47, p < 0.001, 95% CI 0.24, 0.71). Partners’ general QOL decreased significantly from pre-dialysis to 6 weeks (β = − 0.24, p = 0.04, 95% CI -0.47, − 0.01) but returned to its original level at 12 weeks. Patients reported improvements in the physical domain between pre-dialysis and 12 weeks (β = 6.56, p < 0.004, 95% CI 2.10, 11.03). No other domains changed significantly in patients or partners. Only in patients were there significant associations between moderator variables and general QOL. High comorbidity risk level and diabetes were associated with poorer QOL at pre-dialysis whereas being female and having an arteriovenous fistula were linked with improvements in general QOL.ConclusionsPatients reported significant improvements in their general and physical QOL after starting dialysis. Partners’ general QOL worsened after patients started dialysis but improved by 12 weeks. Both patients and partners may benefit from additional educational and counselling services in the lead up to, and immediately after starting dialysis, which could facilitate the transition onto dialysis and improve QOL in both.Study registrationThis study was adopted on the NIHR Clinical Research Network (UK). The details of this study are registered on the Research Registry website (www.researchregistry.com). The identifier for this study is researchregistry2574.

Patient-Reported Outcome Measures and Health-Related Quality-of-Life Scores of Patients Undergoing Anatomic Total Shoulder Arthroplasty.

Health-related quality-of-life (HRQoL) scores are required for cost-effectiveness and health-care value analysis. We evaluated HRQoL scores and patient-reported outcome measures (PROMs) in patients with advanced glenohumeral osteoarthritis treated with anatomic total shoulder arthroplasty to establish values of HRQoL scores that can be used for cost-effectiveness and value analysis and to assess relationships between HRQoL scores and shoulder and upper-extremity PROMs. We analyzed 143 patients (143 shoulders) with glenohumeral osteoarthritis treated with anatomic total shoulder arthroplasty; 92 patients had 1-year follow-up. Preoperative and postoperative functional outcomes were assessed with the Disabilities of the Arm, Shoulder and Hand (DASH) score, the American Shoulder and Elbow Surgeons (ASES) score, the Simple Shoulder Test (SST), and a visual analog scale (VAS) for shoulder pain and function. Health utility was assessed with the EuroQol-5 Dimensions (EQ-5D), Short Form-6 Dimensions (SF-6D), and VAS Quality of Life (VAS QoL). HRQoL score validity was determined through correlations between the PROMs and HRQoL scores. The responsiveness of HRQoL scores was measured through the effect size and the standardized response mean. There were significant improvements in all PROMs and HRQoL scores (p < 0.001) at 1 year after the surgical procedure. The changes in VAS QoL (very weak to moderate), EQ-5D (weak), and SF-6D (weak) were significantly correlated (p < 0.05) with the changes in PROMs, demonstrating comparably acceptable validity. There were large effect sizes in the VAS QoL (1.843), EQ-5D (1.186), and SF-6D (1.084) and large standardized response mean values in the VAS QoL (1.622), EQ-5D (1.230), and SF-6D (1.083), demonstrating responsiveness. The effect sizes of all PROMs were larger than those of the HRQoL scores. PROMs and HRQoL scores are not interchangeable, and studies of the cost-effectiveness and value of shoulder arthroplasty should incorporate both shoulder and upper-extremity PROMs and HRQoL scores. The findings of this study provide data on HRQoL scores that are specific to the treatment of advanced glenohumeral osteoarthritis with anatomic total shoulder arthroplasty and can be used for future cost-effectiveness and value analysis studies. Therapeutic Level IV. See Instructions for Authors for a complete description of levels of evidence.

The KOOS-12 shortform shows no ceiling effect, good responsiveness and construct validity compared to standard outcome measures after total knee arthroplasty.

To investigate the validity, responsiveness and ceiling effect of the recently introduced KOOS-12 and compare its performance to the KOOS, OKS, WOMAC and UCLA activity scales. Patients from an independent multicentre study examining a medially stabilized knee system prospectively completed the KOOS, OKS, WOMAC and UCLA preoperatively and at 1year postoperatively. KOOS-12 scores were calculated from the full length KOOS data. Construct validity was assessed using Spearman's correlation analysis. The ceiling effect was evaluated by calculating the percentage of patients with a maximum score. If the percentage exceeded 15%, a ceiling effect was considered to be present. Responsiveness was evaluated by performing paired t tests on the changes in measures and calculation of Cohen's d. A ceiling effect was present for the KOOS Pain, ADL and QoL subscales and the KOOS-JR at 1year postoperatively. No ceiling effect was observed for the KOOS-12. Correlation of the KOOS-12 was low (0.3 < r < 0.5) with the UCLA, moderate (0.5 < r < 0.7) with the KOOS symptoms, sports and WOMAC stiffness subscales and high (r > 0.7) with all other scores and subscales. Effect size of the UCLA activity scale was moderate (Cohen's d 0.2-0.8) whereas effect sizes of all other outcome measures were large (d > 0.8). The KOOS-12 does not exhibit a ceiling effect, has good convergent construct validity and is responsive to changes in pain, function, QoL and knee impact between preoperatively and 1year postoperatively. Diagnostic level III.

Abstract P5-12-09: Patient-reported outcomes (PROs) during one year of adjuvant enzalutamide for the treatment of early stage androgen receptor positive (AR+) triple negative breast cancer

Abstract BACKGROUND: A subset of TNBC expresses a luminal signature and demonstrates AR-dependence. Enzalutamide (ENZA), an AR-antagonist, has activity with metastatic AR+ TNBC, with a clinical benefit rate of 33% (Traina et al, JCO 2018). We recently demonstrated that one year (y) of adjuvant ENZA is feasible in early stage, AR+ TNBC, with no new safety signals observed (Traina ASCO 2019). Fatigue was the most common, treatment (tx)-related, adverse event of any grade according to investigator assessment (24%). Here we report per-protocol planned secondary analyses of PROs during one year of ENZA including quality of life (QoL), fatigue, and self-reported physical activity (NCT02750358). METHODS: Eligible patients have Stage I-III, ER/PR &amp;lt;1%, HER2(-), AR ≥1% breast cancer and completed all planned standard of care surgery, chemotherapy and radiation within 6 months of study start. Tx = ENZA 160mg orally daily for 1y with the option to extend tx to 2y as per patient preference. Clinician-reported toxicities (NCI CTCAEv4) were assessed every 4 weeks for 12 weeks, then every 3 months. Total study N=50 patients. PROs were assessed every 12 weeks. We examined serial changes in overall QoL (functional assessment of cancer therapy – breast (FACT-B), fatigue (FACIT-fatigue) and exercise behavior (total exercise minutes/week over past 7 days) from pre-treatment and every 3 months thereafter until month 12. All analyses are exploratory and consist of descriptive summaries. RESULTS: Between 5/2016 and 6/2018, 50 patients were enrolled. As of 6/27/19, 34 patients completed 1y of ENZA; 15 patients are off tx: recurrence (3), toxicity (5), nonadherence (4), and consent withdrawal (3). Patients evaluable for PRO (N): baseline (50), 12 weeks (43) and 52 weeks (34). Median scores on FACT-B and fatigue scales remained stable over the course of adjuvant ENZA in the 1st year of therapy (Table). Self-reported exercise increased during this period. Table. Median scores (range) for PROs during 1 year of adjuvant ENZATime PointBaselineWeek 12Week 52FACT-G88.0 (53.5-105.0)82.0 (36.4-105.0)89.5 (58.0-105.0)Breast Cancer Subscale28.5 (15.6-39.0)29.0 (9.0-40.0)29.0 (19.0-40.0)Total118.0 (77.0-141.0)111.0 (45.4-145.0)121.0 (83.0-145.0)FACT-B trial outcome index73.0 (43.0-92.0)73.0 (25.7-96.0)76.9 (51.0-96.0)FACIT43.0 (17.0-52.0)39.0 (10.0-52.0)43.0 (11.0-52.0)Total Exercise (min/week)120 (0-1260)152 (0-3120)180 (0-2580) CONCLUSIONS: Patient reported outcomes of health related QOL remain stable when ENZA is administered for 1 year in the adjuvant setting. Self-reported time spent engaged in physical exercise increased numerically during 1y of therapy. During presentation, additional analyses will be reported including QOL at additional time points, relationship of FACT-B score with FACIT fatigue score and exercise, and QOL changes in relation to relative dose intensity of ENZA, toxicity and off study reason. Citation Format: Tiffany A Traina, Lee W Jones, Victoria Blinder, Jessica Scott, Leigh Ann Boyle, Artavazd Arumov, Tina Alano, Sujata Patil, Patricia DeFusco, Nicholas Lamparella, Mark Robson, Ayca Gucalp. Patient-reported outcomes (PROs) during one year of adjuvant enzalutamide for the treatment of early stage androgen receptor positive (AR+) triple negative breast cancer [abstract]. In: Proceedings of the 2019 San Antonio Breast Cancer Symposium; 2019 Dec 10-14; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2020;80(4 Suppl):Abstract nr P5-12-09.

Phase II study of nanoliposomal irinotecan (nal-IRI) with 5-fluorouracil (5-FU)/folinic acid (FA) in refractory advanced high-grade neuroendocrine cancer (HG-NEC) of gastroenteropancreatic (GEP) or unknown origin.

TPS636 Background: The incidence of GEP HG-NEC is increasing and the prognosis is poor. Etoposide–platinum (EP) combination is considered the standard (std) therapy (Rx), but survival is less than 1 year. There is an unmet need for an optimal 2nd line Rx in these patients (pts). Nal-IRI has shown efficacy in small cell lung cancer (pathologically similar to HG-NEC) and in 2nd line pancreatic adenocarcinoma. In a retrospective study, HG-NEC pts with progression on 1st line EP when treated with 2nd line FOLFIRI (FA+5-FU+ IRI) had a good response and a tolerable safety profile (Hentic et al, 2012). Based on this data and the fact that no std 2nd line Rx option exists in HG-NEC, we are evaluating the efficacy of nal-IRI+ 5-FU/FA in a prospective single-arm multicenter study. Methods: Advanced GEP or unknown origin HG-NEC pts with progressive disease/intolerance to 1st line Rx with EP or temozolomide/capecitabine; ECOG PS 0-2 would receive nal-IRI 70 mg/m2, FA 400 mg/m2 followed by 5-FU 2400 mg/m2 on D1 &amp; 15 of 28-days cycle. Rx to continue till disease progression or unacceptable toxicity. Tumor assessment by CT/MRI per RECIST 1.1 q6 wks. Primary end point is to measure ORR (CR+PR). Secondary endpoints include PFS, TTP, OS, safety and QOL changes. Assuming a historic ORR of 15% with std Rx, 37 pts (upto 41 pts considering non-evaluable pts) are required to show an ORR of 30% at a power of 80% at 1-sided significance of α = 0.1. In stage 1, n1= 18 pts will be enrolled. If 3 or more responses are seen, an additional n2= 19 pts to be enrolled. If 8 or more responses are observed of the n = 37 evaluable pts, the Rx would be considered promising for further study. Genomic mutational profiling to be conducted on pre-study tumor samples and correlated with circulating tumor DNA (CT DNA). CT DNA level, assessed serially, will be correlated with Rx response and disease recurrence. We plan to accrue over 3 years at 3 sites. As of August 2019, 1-patient has been enrolled. The trial is funded by Ipsen Pharmaceuticals and the North American Neuroendocrine Tumor Society through the Clinical Investigator Scholarship. Clinical trial information: NCT03736720.

NCOG-01. NEUROCOGNITIVE FUNCTION (NCF) AND QUALITY OF LIFE (QOL) RESULTS FROM A PHASE II STUDY OF TEMOZOLOMIDE-BASED CHEMORADIOTHERAPY REGIMEN FOR HIGH RISK LOW-GRADE GLIOMAS

Abstract BACKGROUND RTOG 0424 reported a 73.5% 3-year overall survival (OS) rate. This secondary analysis describes changes in NCF and QOL after therapy. METHODS Patients with HR-LGG were treated with radiation and concurrent and adjuvant temozolomide. Standardized NCF tests were performed at baseline, 6 and 12 months (mos). Rates of NCF decline were examined using the reliable change index on Hopkins Verbal Learning Test (HVLT), Trail Making Test (TMT), and Controlled Oral Word Association. Relationships between NCF and subjective cognitive concerns (MOS-Cognitive Function [MOS-CF] scale) were evaluated with Wilcoxon Rank Sum Test. QOL was assessed using FACT-Brain. Longitudinal modeling using maximum likelihood estimation evaluated predictors of change in QOL. Cox models assessed the association of baseline NCF with OS after adjusting for age, anticonvulsants, number of high risk factors, EORTC OS risk group, and tumor crossing the midline. RESULTS From 1/2005 to 8/2009, 129 evaluable patients were accrued, and 93 (72%) completed at least one NCF/QOL measurement with completers having better neurologic function than noncompleters (p=0.04). Compliance across measures was 55–59% and 54–57% at 6 and 12mos. Deterioration occurred in 50%/40% at 6mos/12mos, respectively, and was most frequent on HVLT (21%/20%), TMTA (29%/20%), and TMTB (22%/14%). Patients with HVLT deterioration at 12mos, compared to patients without deterioration, had significantly greater decrease in MOS-CF (p=0.01). No NCF test at baseline was independently associated with OS. FACT Emotional (p=0.02) and Functional Well-Being (p=0.004) subscales improved over time. EORTC OS high-risk group was associated with worse QOL on all subscales (p&lt; 0.05 to 0.001) except Emotional Well-Being, tumor crossing the midline was associated with worse Emotional Well-Being (p=0.04), and unmethylated MGMT status was associated with better Physical Well-Being (p=0.05). CONCLUSIONS Approximately half of patients with HR-LGG experience NCF decline in the first year after treatment with temozolomide-based concurrent chemoradiation. QOL remained stable or improved.

2382. Recurrent Clostridioides difficile Infection (CDI) Worsens Anxiety-Related Patient-Reported Quality of Life

BackgroundThe Health-Related Quality of life (HR-QOL) instrument, the Cdiff32, allows studies on QOL changes associated with recurrent CDI. An ongoing real-world study of bezlotoxumab (BEZ) provided a unique opportunity to study anxiety-related HR-QOL in patients at high risk for recurrent CDI using the anxiety sub-domain of Cdiff32. The aims of this study were to assess baseline anxiety-related HR-QOL based on the number of prior episodes of CDI and to evaluate changes in patients with or without recurrence.MethodsPatients at high risk for recurrent CDI given BEZ were administered the anxiety sub-domain questions of the Cdiff32 prior to infusion and at approximately 90 days after administration (0 = worst anxiety; 100 = no anxiety). The number of prior episodes of CDI were collected, along with demographics and co-morbid conditions. Patients were followed for 90 days for CDI recurrence, which was defined as new onset of diarrhea requiring CDI-active antibiotics.ResultsThere were 107 patients evaluated, aged 68 ± 14 years (mean ± SD) with multiple co-morbid conditions (mean Charlson: 4 ± 3) and multiple previous CDI episodes (3 ± 1 episodes). Fourteen patients (13%) experienced a further CDI recurrence within 90 days following BEZ. Overall, baseline anxiety HR-QOL was 29 ± 22. Risk factors for lower baseline anxiety-related HR-QOL included immunocompromised conditions (P < 0.046) and receipt of a proton pump inhibitor (P < 0.018). Compared with patients with primary CDI disease (Score: 35 ± 20), baseline anxiety HR-QOL was worse with subsequent prior recurrences (Score: 26 ± 23) for CDI episodes 2–4, and then improved for subsequent episodes (Score: 38 ± 22). Anxiety-related HR-QOL improved by a mean of 32 ± 25 points compared with patients that experienced a further recurrence where HR-QOL declined (P < 0.0001). Results were confirmed in a multivariate model controlling for Charlson score and chronic renal failure.ConclusionPoor anxiety-related HR-QOL was observed at baseline in all patients regardless of number of prior episodes. QOL improved 90 days after BEZ infusion in patients without further recurrences of CDIs and worsened in patients with a subsequent recurrence.Disclosures All authors: No reported disclosures.

Patient-Reported Outcome Measures and Health-Related Quality-of-Life Scores of Patients Undergoing Anatomic Total Shoulder Arthroplasty.

Health-related quality-of-life (HRQoL) scores are required for cost-effectiveness and health-care value analysis. We evaluated HRQoL scores and patient-reported outcome measures (PROMs) in patients with advanced glenohumeral osteoarthritis treated with anatomic total shoulder arthroplasty to establish values of HRQoL scores that can be used for cost-effectiveness and value analysis and to assess relationships between HRQoL scores and shoulder and upper-extremity PROMs. We analyzed 145 patients (145 shoulders) with glenohumeral osteoarthritis treated with anatomic total shoulder arthroplasty; 93 patients had 1-year follow-up. Preoperative and postoperative functional outcomes were assessed with the Disabilities of the Arm, Shoulder and Hand (DASH) score, the American Shoulder and Elbow Surgeons (ASES) score, the Simple Shoulder Test (SST), and a visual analog scale (VAS) for shoulder pain and function. Health utility was assessed with the EuroQol-5 Dimensions (EQ-5D), Short Form-6 Dimensions (SF-6D), and VAS Quality of Life (VAS QoL). HRQoL score validity was determined through correlations between the PROMs and HRQoL scores. The responsiveness of HRQoL scores was measured through the effect size and the standardized response mean. There were significant improvements in all PROMs and HRQoL scores (p < 0.001) at 1 year after the surgical procedure. The changes in VAS QoL and EQ-5D were significantly correlated (weak to moderate) with the changes in all PROMs except the SST, demonstrating comparably acceptable validity. The VAS QoL had a large effect size (1.833) and standardized response mean (1.603), and the EQ-5D also had a large effect size (1.163) and standardized response mean (1.228), demonstrating responsiveness. The effect sizes of all PROMs were larger than those of the HRQoL scores. The change in SF-6D had only a moderate effect size and standardized response mean and was not significantly correlated with the change in any of the PROMs. PROMs and HRQoL scores are not interchangeable, and studies of the cost-effectiveness and value of shoulder arthroplasty should incorporate both shoulder and upper-extremity PROMs and HRQoL scores. The findings of this study provide data on HRQoL scores that are specific to the treatment of advanced glenohumeral osteoarthritis with anatomic total shoulder replacement and can be used for future cost-effectiveness and value analysis studies. Therapeutic Level IV. See Instructions for Authors for a complete description of levels of evidence.

Quality of life assessment in dose escalating head and neck radiation therapy.

e17511 Background: Both Dose escalation and acceleration can achieve higher tumor control rates with uncertain role of addition of concurrent chemotherapy. We aimed at developing a protocol for patients unable to receive the standard CCRT with little toxicity profile. Toxicity and Quality of life assessment were our primary end points in this pilot phase II trial. It is known from literature that pre-treatment QOL affects survival. Also, changes in QOL after treatment predict survival in cancer patients. Methods: 63 locally advanced HNC patients were randomized to receive (Arm A) 70Gy/35fr/7wks at 2Gy/fx concurrently with weekly Cisplatin 40mg/m2 or to receive (Arm B) 74Gy/33fx/6.5wks at 2.24Gy/fx with calculation of the Biological equivalent doses. CTCEA v4.3 was used to assess toxicity and Functional Assessment Cancer Therapy-Head &amp; Neck (FACT-H&amp;N) questionnaire (39-item) was used to assess QOL. The Questionnaires were collected and their scorings were calculated to get the FACT-H&amp;N Trial outcome index (TOI), FACT-G and FACT-H&amp;N total score pre-radiotherapy, 3 and 6 months post-radiotherapy for each patient. Results: There were 33 patients in arm A vs. 30 patients in arm B with median follow up 24.2 months. Our results showed statistical significant decrease of chemotherapy related toxicities (nausea, vomiting, neutropenia and anemia), acute (mucositis, oral pain, voice alteration, fatigue and acute dysphagia) and some late toxicities (late xerostomia) in the investigational arm vs. the CCRT arm. Regarding the FACT QOL assessment and scorings of the different parameters, there was no statistical difference between both arms in all FACT-H&amp;N TOI, FACT-G and FACT-H&amp;N total scores at pre-treatment, 3 and 6 months post treatment. Also when analyzing the mean change with time across the two groups pre-radiotherapy, 3 and 6 months post radiotherapy, it was not statistically significant. Our results did not demonstrate any significant differences in 2-year disease local control, PFS and OS between both arms, with comparable patterns of relapse. Conclusions: Slightly dose escalated hypofractionated regimen could be an option in LA-HNC treatment without affecting QOL, which needs confirmation in prospective large multi-centric trials. Clinical trial information: NCT03699969.

Analysis of symptoms and functional HRQoL scales in TAGS, a phase III trial of trifluridine/tipiracil (FTD/TPI) in metastatic gastric cancer (mGC).

4043 Background: The phase 3, randomized, double-blind, placebo-controlled study (TAGS) evaluated the efficacy and safety of FTD/TPI (35 mg/m² given orally twice a day on days 1–5 and 8–12 of a 28-day cycle) in mGC patients who had previously received≥2 prior regimens for advanced disease and demonstrated a clinically relevant and statistically significant benefit in OS and PFS with a predictable and manageable safety profile. HRQoL data and association between QoL and time to ECOG status deterioration (2 or more) are reported here. Methods: HRQoL was evaluated using EORTC QLQ-C30 and the gastric-specific module (QLQ-STO22) questionnaires at baseline and at every 4 weeks thereafter until treatment discontinuation. Prespecified key HRQoL were changes from baseline and time to deterioration. Changes ≥10 points were deemed clinically relevant. A time-dependent Cox-regression analysis was performed to evaluate the association of 10-point Global Health Status deterioration with worsening ECOG status. Results: Of 507 patients randomized, 332/337 (98.5%) of FTD/TPI and 164/170 (96.5%) of placebo had baseline QoL data. Overall compliance was 84% for both questionnaires. Demographic and disease were generally balanced between the two groups; QoL scores were also similar between groups. HRQoL was largely maintained during treatment in both arms for most items; mean changes from baseline remained under the 10-point threshold. Clinically relevant changes from baseline were observed only for pain relief at cycle 2 (favouring FTD/TPI); and improved role functioning at cycle 3 (favouring placebo). In a sensitivity analysis including death or progression as an event, FTD/TPI was associated with a positive trend suggesting a reduced risk of QoL deterioration across all scales compared to placebo (HRs ranged from 0.57 to 0.74. A 10-point Global Health Status deterioration was associated with a worsening ECOG status (HR, 95% CI, 1.5, 1.2 to 1.86). Conclusions: During the treatment period, HRQoL remained stable for most functional and symptom scales in both arms, suggesting that HRQoL is largely maintained with FTD/TPI. Treatment with FTD/TPI was associated with a positive trend toward a lower risk of QoL deterioration than placebo across all scales. Changes in QoL were informative for patients ‘expected ECOG status. Clinical trial information: NCT02500043.

Reliability and validity of a smart quality of life scale for patients with tuberculosis

This study was conducted to evaluate the internal consistency and validity of a 6-item quality of life scale (QOL-6) among tuberculosis (TB) patients in China and to explore its associations with demographic and psychosocial factors. In this cross-sectional study, data were collected from 1342 patients using a structured questionnaire. Information about demographic and psychosocial characteristics was recorded, and QOL was evaluated by the QOL-6 scale. The internal consistency reliability and construct validity of the QOL-6 scale were examined. Moreover, linear regression analysis was used to assess the associations between demographic and psychosocial factors and QOL. The overall Cronbach’s alpha was 0.81. A one-factor model demonstrated excellent model fit (CFI = 0.99; TLI = 0.99; RMSEA = 0.02), suggesting that the QOL-6 has good construct validity. Linear regression analysis indicated that lower QOL scores among patients with TB was associated with older age, lower education level, lower monthly income, lack of TB cure confidence, stigma, and depressive symptoms. The QOL-6 is a reliable, simple, and easy-to-administer tool for routinely monitoring QOL changes among patients with TB. Our study provides some evidence for the reduced QOL among patients with TB in China, and it is necessary to improve QOL using multifactorial approaches.

在宅重症心身障害児（者）の母親の養育経験における主観的QOLの変化と関連要因

在宅重症心身障害児（者）の母親の養育経験における主観的QOLの変化と関連要因

Feasibility of using a novel non-invasive ambulatory tibial nerve stimulation device for the home-based treatment of overactive bladder symptoms.

BackgroundTo evaluate safety, acceptability and pilot efficacy of transcutaneous low-frequency tibial nerve stimulation (TNS) using a novel device as home-based neuromodulation.MethodsIn this single-centre pilot study, 48 patients with overactive bladder (OAB) (24 with neurogenic and 24 with idiopathic OAB) were randomized to use a self-applicating ambulatory skin-adhering device stimulating transcutaneously the tibial nerve at 1 Hz for 30 minutes, either once daily or once weekly, for 12 weeks. Changes in OAB symptoms and QoL were measured at baseline, weeks 4, 8, and 12 using validated scoring instruments (ICIQ-OAB and ICIQ-LUTSqol), 3-day bladder diary and a Global Response Assessment (GRA) at week 12.ResultsThirty-four patients completed the study (idiopathic n=15, neurogenic n=19). No significant adverse effects were noted. Patients found the device acceptable. Eighteen patients (53%) reported a moderate or marked improvement in symptoms from the GRA. Between baseline and week-12, ICIQ-OAB part A sub-scores improved from mean (SD) 9.3 (2.5) to 7.5 (3.1), and from 9.1 (1.9) to 5.9 (1.7) in the daily and the weekly arms, respectively. ICIQ-LUTSqol part A sub-scores improved from mean (SD) 51 (12.8) to 44.2 (13.1) and 44.9 (9.0) to 35.9 (8.8) in the daily and the weekly arms, respectively. Bladder diary mean 24-hour frequency episodes improved from 11.5 to 8.8 at week 12 for both arms.ConclusionsThis novel ambulatory transcutaneous TNS (TTNS) device is safe and acceptable for use in patients reporting OAB symptoms as a form of home-based neuromodulation. A larger study however is required to confirm clinical efficacy.

Health Related Quality of Life Remains Stable over Time in Myelodysplastic Syndrome: An MDS-CAN Prospective Study

Health Related Quality of Life Remains Stable over Time in Myelodysplastic Syndrome: An MDS-CAN Prospective Study

A17995 Value-based assessment of blood pressure telemonitoring in hypertensive patients

Objectives: To investigate whether BPTM fits all three principles of value-based approach: clinical benefit, cost-effectiveness and improved patient reported outcome/experience measures (PROMs/PREMs). Methods: Study included 190 ambulatory patients (137 males, mean age 51 ± 17 years) with uncontrolled HTN assigned to BPTM group (110 patients) and usual care group (UC, 80 patients). Both groups had baseline and 3-months follow-up clinic visits. Between visits BPTM patients used simple, secure web-platform allowing physician-patient communication, storage and blood pressure (BP) and medication data exchange. Clinical efficacy endpoints included BP changes and BP target rate. Economic evaluation settled in Euro currency consisted of cost of illness (COI), incremental cost-effectiveness ratio (ICER); cost-utility analysis (CUA) was performed using generic PROM (SF-36). PREMs collection via PEQ chart. Results: BPTM group characterized by significantly larger decrease in BP (−16 ± 6 mmHg SBP,p = 0,005; − 8,4 ± 3,4 mmHg DBP,p = 0,02) while the number of antihypertensive drugs remained unchanged (+0,3 drugs in BPTM group; p = 0,15). COI was x2.36 higher in BPTM (196.9 € vs 83.5 €, p < 0.005) and ICER was 8.45 €/mmHg/patient (95% CI €4,9–12). BPTM improved PROMs (+2.9 for PH-SF-36,p = 0.04) with 0,06 quality adjusted life years (QALYs) gained. Resultant CUA for BPTM was 2698 €/QALY/patient at a willingness-to-pay threshold of 20383 €/QALY. PREMs improved (+10 points,p = 0,01). UC showed minor QoL changes (+1,3 in PH-SF-36,p = 0,56; +6 PEQ, p = 0,03). No association between BP and QoL results (P = 0,5 for all). Conclusion: BPTM being cost-effective and cost-saving tool incorporates both clinical benefits with the patient-perceived value.

The effects of bone marrow mononuclear cell transplantation on the quality of life of children with cerebral palsy

BackgroundQuality of life (QOL) is an important factor in evaluating the effectiveness of treatment in children with cerebral palsy (CP). The aim of this study was to evaluate the effects of autologous bone marrow mononuclear cells (BM MNCs) on the QOL of children with CP.MethodsFrom December 2015 to December 2016, 30 children with CP aged from 2 to 15 years received two intrathecal infusions of BM MNCs, one at baseline and the other 3 months later, at Vinmec International Hospital. The motor function and muscle tone of the patients were evaluated using the Gross Motor Function Measure (GMFM)-88 and Modified Ashworth Score, respectively. Their QOL was assessed at baseline and 6 months after the first BM MNC transplant using the Vietnamese version of the Cerebral Palsy Quality of Life Questionnaire for children (CP QOL-Child)–the parental proxy report, which comprises seven domains. Nineteen mothers (mean age: 32.9±4.9 years) and 11 fathers (mean age: 36.1±6.8 years) were invited to complete the CP QOL-Child assessment before and after the transplantations, Paired t-tests and multivariate regression analyses were used to evaluate the changes in QOL and GMFM scores and to identify the key factors correlated with the QOL score.ResultsSignificant changes were observed in the children’s gross motor function and muscle spasticity, as evidenced by the GMFM-88 total score, scores for each of its domains, the GMFM-66 percentile and the muscle tone (P < 0.001). Six months after the transplantations, the QOL scores of children with CP were markedly increased (P < 0.001) for all the domains, except for the domain of access to services. In the multivariate regression analysis, significant associations were found between higher age of children and higher QOL except for feeling about functioning and pain and impact of disability domains. Gross Motor Function Classification System (GMFCS) level was negatively correlated with the score of pain and impact of disability domain, while the GMFM-88 scores were positively correlated with the QOL in terms of feelings about functioning and family health domain (P < 0.05).ConclusionThe QOL of the children with CP was noticeably improved 6 months after BM MNC transplantation and was accompanied by improvements in gross motor function and muscle tone.Trial registrationClinicalTrials.gov Identifier: NCT02574923. Registered on October 14, 2015.

EORTC QLQ-C30 summary score reliably detects changes in QoL three months after anatomic lung resection for Non-Small Cell Lung Cancer (NSCLC)

IntroductionWe tested the European Organization for Research and Treatment of Cancer Quality of Life (EORTC QLQ-C30) summary score (SumSC) to detect changes in the HRQOL after Non-small-cell lung cancer (NSCLC) surgery and compared its performance to the traditional scales. MethodEORTC QLQ-C30 data was obtained from 326 consecutive pre-operative patients submitted for anatomical lung resections for NSCLC.66 patients completed post-operative assessments 3 months after surgery. The data was analysed to evaluate the ability of the SumSC compared to the traditional scales to [1] preoperatively differentiate between clinical groups [2]; detect post-op changes and to [3] compare pre and post-op changes in clinically different groups.The importance of perioperative changes was measured by calculating the effect size (ES). ResultsOf the 326 patients, those older than 70 years, with higher DLCO value and Performance Status (PS) ≤1 had a significantly better preoperative SumScore.Physical function (PF) showed a large and significant decline (ES 0.91). Role and social function also showed a significant and medium decline (ES 0.62 and 0.41).Postoperatively some symptoms scales showed significant increases in the values, implying worse symptoms with the largest increase in dyspnoea (ES -0.88). The change in General Health score (GH) was not significant after surgery (ES 0.26, p = 0.062).The SumSc, decreased significantly postoperatively. In particular, medium or large postoperative declines of SumSc were observed in both males and females, in patients with lower FEV1, lower performance score, and in those older than 70 years. Interestingly the decline of SumSc was observed irrespective of the preoperative DLCO level. DiscussionThe Summary Score was more sensitive to changes in subjects' HRQOL, than the GH score. The SumSc can be used as a parsimonious and easy to interpreted patient-reported-outcome measure in multi-institutional database and future clinical trials.