Quality Of Life Metrics Research Articles

Postural Stability and Dynamic Balance in Adult Spinal Deformity: Prospective Pilot Study

We sought to evaluate dynamic balance and postural stability in patients with adult spinal deformity (ASD) compared with published age-matched normative data. Eleven patients with ASD were prospectively enrolled. Postural stability was tested using static and dynamic posturography; patients stood on a movable platform with an integrated force plate and performed standardized sensory organization testing (SOT), evaluating the influence of sensory processing on postural stability under 6 conditions, and motor control testing, assessing reflexive postural reactions to an external perturbation. Patient performance was compared with that of published age-matched controls. Quality of life metrics included scores on the Scoliosis Research Society-22 questionnaire, SF-36, and Morse Fall Scale. Correlations between postural stability and radiographic measurements were performed. ASD patients demonstrated significantly lower SOT scores (P ≤ 0.03) in 5 of 6 conditions tested and greater latency of limb movement during backward translation (P= 0.04) compared with controls. Lower SOT scores were associated with a history of falls. ASD patients who self-reported falling in the previous 6 months, when compared with nonfallers, demonstrated significantly lower SOT scores (P= 0.04) and significantly lower Scoliosis Research Society-22 self-image subscores (P= 0.003). Thoracic kyphosis and mediolateral sway (predictor of falls) were positively correlated in the eyes-open and eyes-closed conditions (P ≤ 0.04). ASD patients demonstrated impaired postural stability, diminished sensory integration, and delayed response to external perturbations compared with normal control data. Postural stability and quality of life metrics correlated with self-reported falls. These findings suggest that ASD patients have abnormal postural stability and may be at elevated risk of falls.

PROSPER: Phase III randomized study comparing perioperative nivolumab versus observation in patients with renal cell carcinoma (RCC) undergoing nephrectomy (ECOG-ACRIN EA8143).

TPS5101 Background: There is no standard adjuvant systemic therapy that increases overall survival (OS) over surgery alone for non-metastatic RCC. Anti-PD-1 nivolumab (nivo) improves OS in metastatic RCC and is well tolerated. In mouse models, priming the immune system prior to surgery with anti-PD-1 results in superior OS compared to adjuvant dosing. Remarkable pathologic responses have been seen with neoadjuvant PD-1 in multiple ph 2 studies in bladder, lung and breast cancers. Phase 2 neoadjuvant RCC trials of nivo show preliminary feasibility and safety with no surgical delays. PROSPER RCC seeks to improve clinical outcomes by priming the immune system with neoadjuvant nivo prior to nephrectomy followed by continued immune system engagement with adjuvant blockade in patients (pts) with high risk RCC compared to standard of care surgery alone. Methods: This global, unblinded, phase 3 National Clinical Trials Network study is accruing pts with clinical stage ≥T2 or TanyN+ RCC of any histology planned for radical or partial nephrectomy. Select oligometastatic disease is permitted if the pt can be rendered ‘no evidence of disease’ within 12 weeks of nephrectomy (≤3 metastases; no brain, bone or liver). In the investigational arm, nivo is administered 480mg IV q4 weeks with 1 dose prior to surgery followed by 9 adjuvant doses. The control arm is nephrectomy followed by standard of care surveillance. There is no placebo. Baseline tumor biopsy is required only in the nivo arm but encouraged in both. Randomized pts are stratified by clinical T stage, node positivity, and M stage. 805 pts provide 84.2% power to detect a 14.4% absolute benefit in recurrence-free survival at 5 years assuming the ASSURE historical control of ~56% to 70% (HR = 0.70). The study is powered to evaluate a significant increase in OS (HR 0.67). Critical perioperative therapy considerations such as safety, feasibility, and quality of life metrics are integrated. PROSPER RCC embeds a wealth of translational studies to examine the contribution of the baseline immune milieu and neoadjuvant priming with anti-PD-1 on clinical outcomes. As of February 2020, 396 patients have been enrolled. Clinical trial information: NCT03055013 .

An analysis of trends in prostate cancer treatment from a CMS database.

e19288 Background: The Centers for Medicare and Medicaid services (CMS) has announced a new payment model for radiotherapy services, a Radiation Oncology Alternative Payment Model (ROAPM), for 17 malignancies. The data used to calculate the base payments were released with the program announcement. The purpose of this study was to analyze trends in treatment as well as payments for each treatment modality in an attempt to provide a baseline for future comparison once the ROAPM has been completed. Methods: The database, CY2015-2017, contains payment, site of service, anatomic site and limited patient data for 517,988 patients, 91,551 having a diagnosis of prostate cancer. Only CPT codes for radiotherapy were included in the calculation of payment and only codes registered 90 days after a treatment planning charge were included. Stata 15m College Station, Texas, was used to perform all statistical analysis. Chi squared test was used to determine associations between variables. The Kruskal-Wallis H test was used to determine the relationship of total payment to clinical variables. Results: Treatment included IMRT (66,663 patients[pts], conventional therapy[ceb] (8,942 pts), brachy therapy (7,156 pts), stereotactic radiotherapy (srs) (5,120 pts) and proton beam therapy [pbt] (3,661). A slight majority of patients, 51%, were treated in the outpatient setting. Patients treated in a freestanding center were significantly more likely to have more than 41 fractions (54%) as compared to patients treated in an outpatient setting (32%), P < 0.001. Patients receiving pbt were significantly younger when compared to patients receiving other therapies, p < 0.001. Pbt was significantly costlier, mean $45,606.85 when compared to IMRT, $26,33.14, ceb, $10,242.27, srs, $16,514.98, or brachytherapy, $15,615.44, p < 0.001. In addition, patients treated at an outpatient center with IMRT experienced higher cost compared to patients treated at a freestanding setting, $25,448.48 vs $22,934.69 p = 0.001. Conclusions: These data gives baseline treatment practice information prior to the implementation of a ROAPM. These data, however, are limited and do not give any indication as to the outcome, both disease specific metrics, and quality of life metrics. Post ROAPM analysis will be needed to determine the effect of a new payment paradigm on treatment practices of this malignancy.

A randomized, controlled trial of structured palliative care versus standard supportive care for patients enrolled on phase I clinical trials.

3634 Background: Phase 1 clinical trials are the first step in developing new cancer therapeutics. Patients enrolled in these studies have typically exhausted standard therapies and are at a point in their disease trajectory where they often are choosing between a phase 1 clinical trial and hospice care. These patients may have significant symptom burden, which can result in early trial discontinuation and confound phase 1 trial outcomes, including toxicity profiles, which may influence further drug development. This study aimed to determine the palliative care needs of patients enrolled on phase 1 clinical trials and their caregivers (CGs), along with differences in study duration, adverse event (AE) and symptom profiles, and quality of life (QOL) between those receiving structured palliative care vs usual supportive care. Methods: 68 patients enrolled on phase 1 clinical trials were randomly assigned to receive structured palliative care or usual supportive care. 39 of their CGs were enrolled and assigned to the same arm as the patient. Quality of life metrics were obtained monthly: the Functional Assessment of Cancer Therapy-General and Memorial Symptom Assessment Scale -Short Form for patients, and The Quality of Life in Life Threatening Illness - Family Carer Version and Caregiver Reaction Assessment for CGs. Palliative care resources utilized were assessed for those in the palliative care arm, and referrals to supportive care services assessed in those in the usual care arm. AEs recorded on the Phase 1 trials were evaluated & compared between arms. Results: Mean duration on phase 1 study was 132 days in the palliative care arm vs 114 days in the usual care arm (p = 0.55). Total weighted AE rate (# of AE [x] AE grade per month) was 26.9 in the palliative care arm vs 34.0 in the usual care arm (p = 0.53). Patients in the palliative care arm experienced better QOL and lower symptom burden, as did their CGs, compared to those in the usual care arm. While the differences in outcomes were not statistically significant, all results favored structured palliative care. Conclusions: Phase 1 patients and their CGs have physical and psychosocial needs which warrant palliative care services. Preliminary results suggest structured palliative care is associated with increased duration on study (by nearly 3 weeks), improved patient and CG QOL, and reduced patient symptom and CG burden. A larger study is warranted to confirm these results and further develop the ideal palliative care intervention in these populations. Clinical trial information: NCT02543541 .

Efficacy and toxicity of extending bone modifying agents beyond two years for bone metastases in breast or castrate-resistant prostate cancer patients: A systematic review.

e24083 Background: Randomized clinical trials evaluated bone modifying agents (BMAs) such as bisphosphonates and denosumab for bone metastases for 1-2 years in duration even though BMA therapy is commonly administered for much longer. A systematic review on the risk-benefit of continuing BMAs for longer than 2 years in breast cancer or castrate-resistant prostate cancer was conducted. Methods: Medline, Embase and Cochrane Register of Controlled Trials were searched (1970 - February 2019) for randomized studies, observational studies, and case series reporting on BMA efficacy and toxicity beyond 2 years. Efficacy was assessed by skeletal-related event (SRE) rates and quality of life metrics. Toxicity was assessed by rates of osteonecrosis of the jaw (ONJ), renal impairment, hypocalcemia, and atypical femoral fractures (AFF). Results: Of 2107 citations, 64 studies were identified. A total of 3 prospective and 9 retrospective studies were eligible. Data beyond 2 years was limited to subgroup analyses in all studies. Three studies [zoledronate (ZOL) (n = 481), pamidronate (PAM) (n = 87), PAM-ZOL (n = 43) ibandronate (n = 30)] reported on SRE-related endpoints. Only one study reported SRE rates based on duration of BMA exposure and showed reduced SRE rates from 27.6% (0-24 months) to 15.5% ( > 24 months) over time. None reported on quality of life. All 12 studies [denosumab (n = 948), ZOL (n = 1036), PAM (n = 163), PAM-ZOL (n = 522), ibandronate (n = 118)] reported on at least one toxicity outcome. Data from 7 bisphosphonate studies (n = 1077) and 1 denosumab study (n = 948) reported on ONJ incidence. In 3 studies (n = 67, n = 221, n = 948), ONJ incidence ranged from 1-4% in the first 2 years and from 3.8-18% beyond 2 years. In another study (n = 252), the cumulative hazard of ONJ was 0% (1% ZOL), 3% (7% ZOL), 7% (21% ZOL) and 11% (21% ZOL) at 12, 24, 36, and 48 months, respectively (p = 0.005); 4 remaining studies reported 1 (n = 181), 3 (n = 57), 7 (n = 99) and 2 (n = 200) cases of ONJ, all after 24 months. Incidence of clinically significant hypocalcemia ranged from 1-2%. Incidence of severe renal function decline was ≤ 3%. AFF occurred in two subjects in one of two studies after 37 and 79 months of BMA exposure. Conclusions: Although the evidence informing the use of BMA is heterogeneous and based on retrospective analysis, BMA exposure beyond 2 years is associated with reduced SRE risk and increased ONJ incidence. Prospective randomized studies addressing the use of BMA for more than 2 years is needed with greater emphasis on quality of life.

Light therapy for multiple sclerosis-associated fatigue: a randomized, controlled phase II trial

Bright white light therapy (LT) can improve fatigue in several disease states but has not been studied in multiple sclerosis (MS). To determine whether controlled home-based LT is feasible, tolerable, and well-adhered to in MS-associated fatigue. A randomized, controlled trial of twice-daily 1-h bright white LT (BWLT) (10,000lx, active arm) versus dim red LT (DRLT) (< 300lx, control arm) was performed. Adults with MS-associated fatigue were enrolled for 10weeks: 2-week baseline, 4-week intervention, 4-week washout. 41 participants were enrolled; 35 were randomized (average age 42years, 80% female; BWLT n = 20; DRLT n = 15). 31 were in the intention to treat analysis. The average duration of LT sessions was similar between groups (BWLT 60.9min, DRLT 61.5min, p = 0.70). The most commonly reported adverse event was headache. There were no events that led to discontinuation. Baseline fatigue was severe in both arms (each 53/63 points on the Fatigue Severity Scale (FSS), p = 0.92). FSS was lower following BWLT (FSS 45.8 post-LT, p = 0.04; 44.9 post-washout, p = 0.02 intra-group compared to baseline FSS) and DRLT (FSS 46.7 post-LT, p = 0.03; 43.9 post-washout, p = 0.002 intragroup compared to baseline FSS). There was no difference between BWLT and DRLT groups in the magnitude of reduction of FSS scores (p = 0.81 after LT; p = 0.77 after washout for between group comparisons). Similarly, MS quality of life metrics improved in both arms but were not significantly different between groups after LT (p = 0.22) or washout. LT is safe, feasible, and well-tolerated in people with MS-associated fatigue. Improvement in both light spectra likely indicates a strong placebo effect for the DRLT group.

Abstract 200: Gender Affirming Female To Male Top Surgery: An Effective Treatment For Gender Dysphoria?

Purpose: Hormonal therapy followed by gender affirming surgery (GAS) are seen as definitive treatments for gender dysphoria. Although there is a paucity of long-term follow-up data on GAS patients, recent literature suggests that transgender (TG) individuals may not experience anticipated improvement in quality of life (QoL) measures with dysphoria resolution. This study aims to determine GAS’s impact on resolution of gender dysphoria as measured by QoL metrics and validated measures of suicidal ideation (SI)/ suicide attempts (SA) in female-to-male (FTM) TG individuals undergoing chest surgery as part of multidisciplinary team (NOLA Transgender) care for TG patients. Methods: Thirty-one FTM patients presenting for GAS masculinizing top surgery completed a preoperative (n=14) and postoperative (n=17) survey related to phase of gender transition, demographics, sexual orientation, gender identity, QoL and SI/SA. The survey included validated questions from multiple sources. All surgeries were performed according to World Professional Association for Transgender Health (WPATH) standards. IRB approval at LSUHSC is currently pending. Results: 25 patients identified as sexually oriented to men and four as non-binary. Ages ranged from 19-50 years (mean 29). Patients reported onset of gender dysphoria at 3-36 years (mean 10). There was no significant difference between pre-operative and post-operative patients with regard to chest dysphoria, Body Uneasiness Test-A, UCLA loneliness scale, SI/SA, daily pain or emotional distress (WPATH), or BREAST-Q forms. However, significant difference existed in the UGDS-F gender dysphoria scale with mean score of 56.1 and 52.3 in pre-operative and post-operative patients respectively (p-value =0.03). Conclusion: For patients, goals of GAS FTM top surgery are highly individualized, both physically and psychologically. Consistent with prior research, QoL measures looking at pain, emotional distress, SI and loneliness in our study showed no difference between pre-operative and post-operative patients. Our sample reported mostly positive QoL measures, so these groups may be artificially equated. It is possible that TG individuals seeking GAS shared confounding characteristic(s) that improved overall QoL as compared to TG patients not seeing chest GAS. Postoperative patients experienced significantly improved overall dysphoria on UGDS-F evaluation, however this cross-sectional study did not track longitudinal improvement. As our study only looked at FTM patients undergoing top surgery, these results cannot be generalized to bottom surgery or male-to-female GAS. Despite these study limitations, our data highlight significant positive gender dysphoria outcomes following FTM GAS surgery.

Outcomes of Left Ventricular Assist Device Implantation with Mitral Regurgitation with and without Concomitant Mitral Operation

The clinical impact of mitral valve procedures (MVP) at time of left ventricular assist device (LVAD) is not well understood. We examined the association of MVP, according to severity of MR, with long term survival and quality of life metrics. Continuous-flow LVAD recipients with MR from the 2008-2017 INTERMACS registry were included in the analysis. Patients with concomitant MVP were compared to those without intervention, stratified by severity of MR. Descriptive analyses, hazard modeling, and Kaplan-Meier analyses were used to examine associations between intervention and clinical outcomes. 6360 (40.0%) mild, 5592 (35.2%) moderate, and 3936 (24.8%) severe MR cases were included. MVP was not associated with improved survival for mild (median: 80.7 months for MVP vs. 55.0 months for non-MVP, P=0.49, HR 0.65 [95%:0.34-1.23]), moderate (58.8 vs. 49.3 months, P=0.31, HR 0.90 [0.57-1.42]), or severe MR (76.8 vs 49.7 months, P=0.15, HR 1.08 [0.73-1.59]). MVP was not associated with differential risk for device failure, bleeding, arrhythmia, thrombotic, right heart failure, or renal failure. There were no differences in Kansas City Cardiomyopathy Questionnaire with or without MVP for mild, moderate, or severe MR at 1- or 2-years after implantation. Concomitant MVP with LVAD implantation was not associated with mortality, adverse events, or objective quality of life metrics. The role of concomitant MVP with LVAD implantation must be carefully considered.

Questionable Value of Concomitant Tricuspid Valve Procedure with Left Ventricular Assist Device Implantation

Tricuspid regurgitation (TR) is common in end stage heart failure patients receiving left ventricular assist devices (LVADs), but the value of intervention is uncertain. This study examined the impact of tricuspid valve procedures (TVP) at time of LVAD implantation on clinical outcomes and quality of life metrics. We included patients in the INTERMACS database with TR who received continuous-flow LVADs from the 2008 to 2017. TR was stratified by severity. Patients receiving concomitant TVP were compared to those without intervention in a stratified analysis. Survival analyses and Andersen-Gill hazard models were used to examine associations with clinical outcomes. Our analysis included 8263 (53.1%) mild, 4252 (33.3%) moderate, and 2100 (13.5%) severe TR cases. TVP rate increased with severity: 13.0% of mild cases underwent TVP versus 55.2% of severe cases. TVP was not associated with survival difference for mild (median 57.8 months for TVP vs. 55.6 months for non-TVP, P=0.69, HR 1.10 [95%: 0.87-1.50]), moderate (47.5 vs. 51.1 months, P=0.23, HR 1.20 [0.99-1.50]), or severe TR (43.3 vs 46.3 months, P=0.09, HR 1.30 [0.99-1.60]). TVP was associated with increased risk of bleeding, arrhythmia, stroke, renal failure, and respiratory failure (P<0.01 for each) and not with Kansas City Cardiomyopathy Questionnaire (KCCQ) differences at 12- or 24-months follow-up. TVP at time of LVAD implantation was not associated with differences in survival or KCCQ scores. However, there were associations with increased risk of adverse events. Further studies are needed to understand appropriateness criteria for TVP with LVAD implantation.

Clinical Implications of Concomitant Surgical Intervention for Aortic Insufficiency with Left Ventricular Assist Device Implantation

Aortic insufficiency (AI) is a common problem in advanced heart failure and progression after left ventricular assist device implantation is well described. Patterns of care for AVP at the time of LVAD implantation and the associated outcomes remain poorly described. We included all patients in the INTERMACS (Interagency Registry for Mechanically Assisted Circulatory Support) who received a continuous-flow LVADs and at least mild AI from 2008 to 2017. AI was stratified by mild, moderate, or severe disease. Patients undergoing any AVP were compared to those of identical severity without intervention. Descriptive, Cox regression, and Kaplan-Meier analysis were performed to identify associations between intervention and clinical outcomes. Of 6,492 patients identified with any degree of AI, 5,457 (84.1%) had mild, 814 (12.5%) had moderate, and 221 (3.4%) had severe insufficiency. AVP rate increased with AI severity, from 3.7% of mild to 55.2% of severe insufficiency. AVP was not associated with a survival difference for moderate or severe AI. In mild AI, AVP was associated with decreased survival compared to mild without AVP (median 57.8 vs 58.9 months, log-rank test: P=0.0089, Hazard Ratio 1.5 [95% confidence, 1.1-2.2]). AVP was not associated with decreased adverse event rates, nor was AVP associated with improved quality of life metrics at 24 months follow-up for any degree of AI severity. AVP at time of LVAD implantation was not associated with improved survival for end-stage heart failure patients with mild, moderate, or severe AI and was not associated with improved quality of life. Additional evidence to define a role for AVP among patients receiving LVAD is needed.

PROMIS is superior to established outcome measures in capturing disability resulting from sagittal malalignment in patients with back pain.

Patient reported outcomes measurement information system (PROMIS) is a quality of life metric that has gained increased popularity due to computer adaptive testing. Previous studies have shown that PROMIS correlates with Oswestry Disability Index (ODI) in patients with back pain and takes significantly less time to complete. However, the ability of PROMIS to capture disability from spinal malalignment relative to established metrics is unknown. The aim of the present study is to validate the correlation between ODI and PROMIS in patients with back pain, analyze correlations of PROMIS and legacy metrics to sagittal alignment, and identify major drivers of PROMIS scores and ODI in patients with back pain. A retrospective review was conducted of a prospectively collected outcome measures database (PROMIS, ODI, VAS Back, VAS Leg, VAS Neck, and VAS Arm) of spine patients > 18years. Inclusion criteria for the present study was a chief complaint of back pain and full length weight bearing X-rays within 30days of health related quality of life (HRQL) completion. Demographic information, radiographic alignment, psychiatric diagnoses, and comorbidities were recorded. PROMIS metrics were correlated to legacy metrics (ODI and VAS). Next, outcome metrics were correlated with sagittal alignment variables T1 Pelvic Angle (TPA), SVA, PT, and PI-LL. Patients were grouped based on the presence of spinal deformity (defined radiographically as any one of SVA > 4cm, PI-LL > 10°, PT > 20°) and mean HRQL scores were investigated for the adult spinal deformity (ASD) and non-ASD groups. Finally, drivers of PROMIS PF scores and ODI scores were determined using multiple stepwise regression. 150 patients met inclusion criteria including 60 patients with ASD and 90 patients without. For the whole cohort, PROMIS PF correlated with ODI (r = -0.651, p < 0.001), VAS Back (r = -0.260, p = 0.014) and Charleson Comorbidity Index (r = -0.336, p < 0.001). PROMIS PF had higher correlations than ODI for each sagittal alignment parameter tested, including TPA, SVA, PT, and PI-LL. When patients with ASD were compared to non-ASD patients, the PROMIS score was different between the groups but the ODI and VAS scores were not. Finally, stepwise linear regression showed that SVA, PI-LL, and VAS Leg were significant drivers of PROMIS PF (r2 = 0.406, p < 0.001). VAS Leg and VAS Back were significant contributors to ODI (r2 = 0.376, p < 0.001). In a cohort of 150 patients with back pain, PROMIS correlated strongly with legacy outcome metrics, including VAS and ODI. PROMIS PF correlated more strongly with sagittal malalignment than ODI. Additionally, patients with spinal deformity had significantly worse PROMIS PF scores but similar ODI scores as patients without ASD. Finally, sagittal alignment was found to be a significant driver of PROMIS PF scores but not ODI scores. PROMIS PF should be utilized as a disability assessment tool in patients with spinal deformity due to ease of use, strong correlations with legacy metrics, and ability to capture disability resulting from sagittal alignment.

Relationship of Pain, Function and Quality of Life with Disease Grading among Patients with Knee Osteoarthritis

Knee Osteoarthritis (KOA) is a global public health problem. The prevalence of KOA is on the rise leading to increased disease burden. Female and older adults have a higher risk of developing KOA. The diagnosis of KOA is usually based on specific clinical signs and symptoms or based on structural changes evident on radiographs. The clinical decision for management in these patients is based on X-ray grading. Recently there is a discussion on concordance between clinical and radiological findings in KAO patients. Therefore, it is necessary to identify a relationship between patient-reported findings and disease grading based on standard radiographs. This cross-sectional study recruited KAO patients from rural and urban parts of Phagwara and Jalandhar, Punjab, India. Participants were identified during medical and physiotherapy camps. Participants were included based on the American College of Rheumatology Criteria (ACR). Demographic characteristics, pain intensity, functional status, and Quality of Life (QOL) were assessed and documented. Disease grading was done based on presenting X-rays using grading proposed by Kellgren and Lawrence. The association between these variables was analyzed using R software and reported. We also predicted disease grading using QOL metrics. 120 Participants’ data were included in the final analysis. Findings suggest an association between demographic (age, height, weight and BMI) variables and disease grading. Pain, functional status, and QOL were found to be associated with disease grading. SF-Physical functioning and SF-General Health QOL metrics significantly predicted disease grading. The study’s findings suggest that disease grading based on X-rays shows an association with the participants’ clinical presentation.

PROSPER: Phase III randomized study comparing perioperative nivolumab versus observation in patients with renal cell carcinoma (RCC) undergoing nephrectomy (ECOG-ACRIN EA8143).

TPS765 Background: There is no standard adjuvant systemic therapy that increases overall survival (OS) over surgery alone for non-metastatic RCC. Anti-PD-1 nivolumab (nivo) improves OS in metastatic RCC and is well tolerated. In mouse models, priming the immune system prior to surgery with anti-PD-1 results in superior OS compared to adjuvant dosing. Remarkable pathologic responses have been seen with neoadjuvant PD-1 in multiple ph 2 studies in bladder, lung and breast cancers. Phase 2 neoadjuvant RCC trials of nivo show preliminary feasibility and safety with no surgical delays. PROSPER RCC seeks to improve clinical outcomes by priming the immune system with neoadjuvant nivo prior to nephrectomy followed by continued immune system engagement with adjuvant blockade in patients (pts) with high risk RCC compared to standard of care surgery alone. Methods: This global, unblinded, phase 3 National Clinical Trials Network study is accruing pts with clinical stage ≥T2 or TanyN+ RCC of any histology planned for radical or partial nephrectomy. Select oligometastatic disease is permitted if the pt can be rendered ‘no evidence of disease’ within 12 weeks of nephrectomy (≤3 metastases; no brain, bone or liver). In the investigational arm, nivo is administered 480mg IV q4 weeks with 1 dose prior to surgery followed by 9 adjuvant doses. The control arm is nephrectomy followed by standard of care surveillance. There is no placebo. Baseline tumor biopsy is required only in the nivo arm but encouraged in both. Randomized pts are stratified by clinical T stage, node positivity, and M stage. 805 pts provide 84.2% power to detect a 14.4% absolute benefit in recurrence-free survival at 5 years assuming the ASSURE historical control of ~56% to 70% (HR = 0.70). The study is powered to evaluate a significant increase in OS (HR 0.67). Critical perioperative therapy considerations such as safety, feasibility, and quality of life metrics are integrated. PROSPER RCC embeds a wealth of translational studies to examine the contribution of the baseline immune milieu and neoadjuvant priming with anti-PD-1 on clinical outcomes. As of October 18, 2019, 317 patients have been enrolled. Clinical trial information: NCT03055013.

Impact of palliative care in patients with metastatic esophageal cancer declining chemotherapy.

315 Background: Palliative care has been associated with improved overall survival (OS), but limited data exist in metastatic esophageal cancer (mEC). We investigated the impact of palliative care in patients with mEC who declined chemotherapy (CTX). Methods: The National Cancer Database was used to identify patients between 2004-2015. Patients with M1 disease who declined CTX and had known palliative care status (surgery, radiotherapy [RT], pain management, or any combination of) were included. Cases with unknown CTX, RT, or nonprimary surgery status were excluded. Kaplan-Meier estimates of OS were calculated. Univariable and multivariable Cox regressions were performed. Results: Among 140,234 EC cases, we identified 1,493 patients who declined CTX and had complete data. Median age was 70 years, most (66.3%) had a Charlson Comorbidity Index (CCI) of 0, and 37.1% were treated at an academic center. Most (72.7%) did not receive palliative care. Median OS was 2.53 months (mos), with no statistically significant difference in median OS between those receiving palliative care (2.83 mos, 95% confidence interval [CI] 2.53-3.12) vs. no palliative care (2.37 mos, 2.2-2.56; p = 0.288). On univariable analysis, treatment at an academic center (hazard ratio [HR] 0.90, 0.80-1.00) and CCI ≥2 (HR 1.20, 1.00-1.42) were predictive of OS (p &lt; 0.05). On multivariable analysis, male sex (HR 1.23, 1.08-1.40), South geographic region (HR 1.23, 1.04-1.46), CCI of 1 (HR 1.17, 1.03-1.32), higher grade (HR 1.21, 1.07-1.38), and higher T stage (HR 1.39, 1.12-1.73) were associated with poor OS (p &lt; 0.05). Conclusions: Palliative care conferred a numerically higher, but not statistically significant difference in OS among patients with mEC declining CTX. Quality of life metrics, inpatient status, and subgroup analyses are important for examining the role of palliative care in mEC and future studies are warranted.

Examining the Relationship Between Two Pediatric Asthma-Related Quality of Life Metrics

The Childhood Asthma Control Test (c-ACT) and the Pediatric Asthma Impact Scale (PAIS) are tools used to evaluate asthma control and asthma-related quality-of-life, respectively, in pediatric populations. The c-ACT is the gold-standard for asthma outcome measurements, whereas the PAIS is a newer tool that offers the benefit of being a patient-reported outcome measure (PROM). We aim to determine if the PAIS and the c-ACT measure similar outcomes, and could be used as proxies for each other. 39 pediatric patients (mean age 10, range 7 to 16) used the ASTHMAXcel mobile application to learn about asthma management through videos and games. At baseline, 4-month, and 6-month clinic visits, patients were administered the PAIS and the c-ACT. Scores on the tests were standardized to a 100-point scale, and the relationships of the scores at each visit and between visits were analyzed using the Pearson correlation. Significant correlations were found between scores on the PAIS and the c-ACT at the baseline, 4-month, and 6-month visits (r=0.05, p<0.01; r=0.52, p<0.01; r=0.55, p<0.01, respectively). Significant correlations were once again found when comparing changes between baseline and 4-month visits, baseline and 6-month visits, and 4-month and 6-month visits (r=0.57, p<0.01; r=0.47, p<0.01; r=0.40, p<0.01, respectively). A strong correlation exists between the outcome measurements of the PAIS and the c-ACT. These results indicate that there is potential to use the newer PAIS to measure similar outcomes as the c-ACT, particularly given its advantage as a PROM.

Effects of Transcatheter Aortic Valve Implantation on Frailty and Quality of Life

BackgroundTranscatheter aortic valve implantation (TAVI) is an effective alternative to surgical valve replacement in high-risk patients with severe aortic stenosis. Although measures of frailty have been used to attempt to predict outcomes in this population, few studies have demonstrated changes in these measures. MethodsWe performed a prospective, observational study of 171 patients undergoing TAVI, of whom 44 had maximal follow-up of 1 month and 50 had maximal follow-up of 1 year. Quality of life was assessed using the Minnesota Living With Heart Failure Questionnaire, Katz Index of Independence in Activities of Daily Living questionnaire, and patient perception of overall well-being. Frailty was measured using the 10-m walk test and handgrip strength testing. ResultsIn the overall cohort, participants demonstrated improvements in quality of life metrics, but deterioration in 10-m walk test and handgrip at 1 month. These trends continued at 1 year. However, patients in the lowest quintile of handgrip and 10-m walk test demonstrated a trend of improvements in these metrics during follow-up. ConclusionsDespite improvements in quality of life after TAVI, no improvements in frailty were observed in patients at 1 year.

Quality of Life After Radiotherapy for Rectal and Anal Cancer.

With advances in radiation therapy (RT) techniques for rectal and anal cancers allowing for the modulation of critical normal tissues, there has been an increased emphasis on improving the quality of life (QOL) of cancer survivors. Herein, we review the literature to examine the impact of RT on QOL and patient reported outcomes (PROs) to better inform providers about the challenges of survivorship. Large systematic reviews, recent studies and long-term follow-up of pivotal clinical trials have shown that RT impacts QOL, particularly fecal continence and sexual function. Modern preoperative RT techniques allow for decreased dose to organs-at-risk will likely improve QOL. RT, though critical in the treatment of rectal and anal cancer, has a profound impact on QOL for some patients. Recent studies have included PROs and validated QOL metrics to better inform providers and patients.

10 kHz spinal cord stimulation for the treatment of chronic back and/or leg pain: Summary of clinical studies.

Chronic pain has a major impact on sufferers and their families. The associated health care costs are substantial. In the context of increasing prevalence, effective treatment options are ever more important. 10 kHz spinal cord stimulation has been shown to effectively provide pain relief, aid in opioid reduction, and improve quality of life in patients with chronic intractable pain. The present review aims to summarize the clinical evidence related to the use of 10 kHz SCS in chronic back and/or leg pain. We searched the PubMed database between 2009 and 2 June 2020 for articles reporting clinical studies that included at least 10 human subjects permanently treated with a 10 kHz SCS system (Senza® system) for chronic back and/or leg pain for a minimum of 3 months. A randomized controlled trial (SENZA-RCT), as well as several prospective and retrospective studies, reported clinical outcomes in subjects with chronic back and leg pain treated with 10 kHz SCS. A high proportion of subjects (60%–80%) reported long-term response to therapy. Pain relief was provided without paresthesia. Other studies showed promising pain relief outcomes in subjects with back pain ineligible for spinal surgery, neuropathic limb pain, and in those with previously failed traditional low-frequency SCS. Most studies reported improved quality of life metrics and/or reduced opioid intake. Level 1 evidence has already been established for the use of 10 kHz SCS in treating chronic back and leg pain, corroborated by real-world, clinical experience. Exploratory studies also show the potential of the therapy in other refractory pain syndromes, although larger studies are desired to validate their findings. Overall, the literature suggests that 10 kHz SCS provides long-term pain relief in a high proportion of patients, along with improved quality of life and reduced opioid consumption.

Two-Year Functional Outcomes of Operative vs. Non-Operative Treatment of Completely Displaced Midshaft Clavicle Fractures in Adolescents: Results from a Prospective, Multicenter, Level 2 Study

Purpose: To investigate the two-year functional outcomes and complications following operative versus non-operative treatment of completely displaced midshaft clavicle fractures in adolescents.&#x0D; Methods: All patients 10-18 years old treated for a midshaft clavicle fracture between August, 2013 and August, 2018 at one of 8 geographically diverse, high-volume, tertiary-care pediatric centers were enrolled, with independent treatment decisions determined by individual providers. The sub-population of patients with completely displaced fractures was prospectively followed for over 2 years. Clinical course, complications, validated patient-reported outcome measures (PROs), quality of life metrics, and satisfaction scores were analyzed. To address the ceiling effect of the PRO/satisfaction data following clavicle injuries, a priori thresholds for ‘suboptimal’ scores were established (ASES scores &lt;90, QuickDASH scores &gt;10, EQ-5D &lt;0.80). According to ‘intention to treat’ statistical principles, one post-operative complication (and a subsequent secondary operation) was analyzed within the non-operative cohort, given that the patient represented a ‘crossover’ from the non-operative to the operative treatment group.&#x0D; Results: Of the 909 patients enrolled in the prospective study, 417 patients (45.9%) demonstrated completely displaced fractures and maintained enrollment over the study period, 277 (66%) of whom had reached two year follow up, and 151 of whom provided adequate PRO data, representing a 55% response rate. Of these patients, 55 (36%) underwent operative treatment, while 96 (64%) were treated non-operatively. Those treated surgically showed no difference in gender distribution (76% males,p=0.43), athletic participation (p=0.76), or fracture pattern (p=0.18), but were older (mean age 15.3 vs. 13.5 years, p&lt;0.001) and had greater shortening (p&lt;0.001) than those treated non-operatively. Within the subset with adequate complication data, listed in Table 1, complications were less common in non-surgical than surgical patients (p=0.0003), but this difference did not reach significance when sensory deficits were excluded (p=0.17). There was no difference in secondary surgeries (p=0.43). While greater percentages of operative than non-operative patients reported suboptimal PRO/satisfaction scores (ASES: 15% vs. 5%, QuickDASH 11% vs. 5%, satisfaction 11% vs. 5%), these differences did not reach significance (p=0.07, 0.20, 0.06, respectively).&#x0D; Conclusion: At eight large pediatric centers with many surgeons making independent treatment decisions, non-operative treatment of adolescent clavicle fractures demonstrated lower complication rates and similar satisfaction and functional outcomes.&#x0D; Significance: These data establish a comprehensive functional assessment of adolescents treated for clavicle fractures, which represents the epidemiological sub-population most affected by this condition. Unlike several adult studies demonstrating superiority in operative treatment, this adolescent study demonstrates equivalent function and fewer complications associated with non-operative treatment.

Validation of a Quality of Life Metric "Prolac-10"

Validation of a Quality of Life Metric "Prolac-10"