Zhang, Nan

Objective: To determine factors contributing to disease etiology and treatment efficacy. Study Design: Original Report. Setting: Tertiary academic center. Methods: IRB approved prospective study of 20 patients with reported dysphagia who exhibited normal oropharyngeal and esophageal swallowing function as evidenced by videofluoroscopic swallow study, esophagogastroduodenoscopy, high-resolution esophageal manometry with stationary impedance, and Bravo pH probe off proton pump inhibitor. Patients underwent speech-language pathology intervention. Results: Atypical laryngeal muscle tension was present in 100% of patients. Forty percent of patients had diagnosed positive gastroesophageal reflux disease. Sixty-five percent of patients showed signs of non-specific laryngeal inflammation and laryngeal hyperresponsiveness during strobolaryngoscopy. All patients reported a mean of 90% recovery by the completion of voice therapy directed toward unloading muscle tension. Conclusion: The study results suggest an association between laryngeal muscle tension and these patients’ dysphagia symptoms regardless of associated conditions. Speech-language pathology intervention showed high treatment efficacy. Level of Evidence: 2c- Outcomes research.

70 Background: The prevalence of health insurance literacy (HIL) and financial literacy (FL) in cancer patients is not well described. Further, a better understanding of the association between these constructs and the domains of financial hardship (FH) can guide potential interventions. Methods: We examined the prevalence of HIL and FL and FH in cancer patients on active treatment at our institution. HIL and FL were assessed using Health Insurance Literacy Measure (HILM) and 5 questions from the National Financial Capability Study respectively. COST FACIT and National Health Interview Survey (NHIS) questions were used to examine overall FH and its 3 domains (material, behavioral and psychological hardship). Pearson correlation coefficient was used to describe the relation between the COST score and FH domains assessed by NHIS questions. Logistic regression analysis was performed to determine the associations of overall FH (measured by COST FACIT) with FL and HIL. Results: Among 256 patients approached, 202 completed questionnaires. Median age was 67 (range 22 to 91) years. 51% were male, 93% Whites and 5% were Hispanic. 69% had a solid tumor with 37% stage IV. Median number of lines of treatment was 4 (range 1 to 18). 21% were currently employed, 59% had ≥college degree, 41% had private insurance and 42% had monthly income≥$7000. 50% participants answered at least 4/5 FL questions correctly, indicating high FL. Being male, White, having insurance, higher education and income ≥$7000 were associated with higher FL. 72% of participants had high HIL, and they were more likely to be Non-Hispanic, married and have received more lines of treatment. FL significantly correlated with HIL (r = 0.29, p < .001). 65% patients endorsed FH based on NHIS questions (10% behavioral, 20% material, and 62% psychological hardship). 48% patients had FH based on a COST score < 28 (Median score), which correlated with material (r = -0.59, p < .001), psychological (r = -0.55, p < .001) and behavioral FH (r = -0.42, p < .001). The association between FH and HIL was not significant (OR 0.78; p = 0.08), though higher FL was associated with lower FH (OR 0.69; p = 0.02). Conclusions: Despite high FL and HIL (as compared to national standards), the prevalence of FH in our relatively affluent patient cohort was high, driven mainly by psychological hardship. We did not find strong association between the HIL and FH unlike in the reported literature. Our findings behoove us to develop tailored interventions to address unique domains of the FH in a diverse population.

Background: Financial distress from medical treatment is an increasing concern. Healthcare organizations may have different levels of organizational commitment, existing programs, and expected outcomes of screening and management of patient financial distress. Patients and Methods: In November 2018, representatives from 17 (63%) of the 27 existing NCCN Member Institutions completed an online survey. The survey focused on screening and management practices for patient financial distress, perceived barriers in implementation, and leadership attitudes about such practices. Due to the lack of a validated questionnaire in this area, survey questions were generated after a comprehensive literature search and discussions among the study team, including NCCN Best Practices Committee representatives. Results: Responses showed that 76% of centers routinely screened for financial distress, mostly with social worker assessment (94%), and that 56% screened patients multiple times. All centers offered programs to help with drug costs, meal or gas vouchers, and payment plans. Charity care was provided by 100% of the large centers (≥10,000 unique annual patients) but none of the small centers that responded (<10,000 unique annual patients; P =.008). Metrics to evaluate the impact of financial advocacy services included number of patients assisted, bad debt/charity write-offs, or patient satisfaction surveys. The effectiveness of institutional practices for screening and management of financial distress was reported as poor/very poor by 6% of respondents. Inadequate staffing and resources, limited budget, and lack of reimbursement were potential barriers in the provision of these services. A total of 94% agreed with the need for better integration of financial advocacy into oncology practice. Conclusions: Three-fourths of NCCN Member Institutions reported screening and management programs for financial distress, although the actual practices and range of services vary. Information from this study can help centers benchmark their performance relative to similar programs and identify best practices in this area.

11615 Background: Deficiencies and barriers exist to delivering comprehensive and affordable cancer care. Understanding the variation in organizational commitment, existing programs, and expected outcomes for screening and management of financial distress is needed. Methods: Representatives from 17 of 27 NCCN Member Institutions (63%) completed an online survey in November 2018 conducted by the NCCN Best Practices Committee. Centers were classified based on number of unique patients seen per year, as large ( > 10,000) (76%), or small ( < 10,000) (34%). The survey focused on institutions’ screening and management practices for patient financial distress, perceived barriers in implementation, and leadership attitudes. Results: Routine screening for financial distress was reported by 77% of centers, and most used social worker assessments (94%). 56% screened patients throughout the cancer journey. Help with drug costs, meal or gas vouchers and payment plans were offered by 100% of centers. Formal pre-authorization programs and assistance with claims and denials was offered by 81%. Charity care for medical costs was provided by 100% of the large centers compared to only 33% of small centers (p = 0.03). Median number of social workers (24 vs. 3; p = 0.01) and pharmacy representatives (6 vs. 2; p = 0.02) was also different between large and small centers. 76% evaluated the impact of financial advocacy services through number of patients assisted (85%), bad debt and charity write-offs (85%) or patient satisfaction surveys (54%). 6% and 12% reported overall effectiveness of institutional practice for screening and management of financial distress as poor/ very poor respectively. Inadequate staffing and real time resources (69%), limited institutional budget (50%), lack of reimbursement (50%), and clinical time constraints (50%) were reported as potential barriers in provision of these services. 94% agreed about stronger integration of financial advocacy services into oncology practice and 84% felt that success of these services should be a quality metric. 31% of large centers vs. 100% of small centers plan to increase staffing in this area in the next 5 years. Conclusions: Majority of NCCN Member Institutions report screening and management programs for financial distress, though the actual practices and range of services vary widely. Information from this study can help centers benchmark their performance relative to similar cancer programs and identify best practices in this area.

Spontaneous portosystemic shunts (SPSSs) have been associated with worse clinical outcomes in the pre–liver transplantation (LT) setting, but little is known about their post‐LT impacts. Our aim was to compare LT candidates with and without SPSSs and assess the impact of SPSSs on patient mortality and graft survival in the post‐LT setting. Patients 18 years or older with abdominal imaging done prior to LT were included. Exclusion criteria were the presence of pre‐LT surgical shunts, LT indications other than cirrhosis, and combined solid organ transplantations. SPSSs were classified as absent, small, or large according to their maximum diameter (8 mm). Multiple variables that could influence the post‐LT course were extracted for analysis. Patient and graft survival were estimated using the Kaplan‐Meier method and were compared between groups using a log‐rank test. The project received institutional review board approval. We extracted data from 326 patients. After comparing patients without SPSS or with small or large SPSSs, no statistical difference was found for overall patient survival: no SPSS (n = 8/63), reference; small SPSS (n = 18/150), hazard ratio (HR), 1.05 (95% confidence interval [CI], 0.45‐2.46); and large SPSS (n = 6/113), HR, 0.60 (95% CI, 0.20‐1.78); P = 0.20. Also, no difference was found for graft survival: no SPSS (n = 11/63), reference; small SPSS (n = 21/150), HR, 0.80 (95% CI, 0.38‐1.70); large SPSS (n = 11/113), HR, 0.59 (95% CI, 0.25‐1.40); P = 0.48. Similarly, no statistical significance was found for these variables when comparing if the graft used was procured from a donation after circulatory death donor versus a donation after brain death donor. In conclusion, the previously described association between SPSSs and worse clinical outcomes in pre‐LT patients seems not to persist once patients undergo LT. This study suggests that no steps to correct SPSS intraoperatively are necessary.

Abstract Introduction: The impact of genomic alterations, such as mutations in ASXL1, on the risk of disease progression and leukemic transformation in patients with myelofibrosis (MF) is well established. Further, emerging data suggests that the number and type of mutations may impact response to therapies such as ruxolitinib or imetelstat. Allogeneic hematopoietic stem cell transplant (allo-HSCT) remains the only potentially curative treatment for MF patients. However, the impact of somatic mutations on overall survival (OS) and relapse-free survival (RFS) is poorly understood. Using next-generation sequencing of pre-transplant blood and bone marrow samples from a well clinically-annotated cohort of MF patients who underwent allo-HSCT, we sought to determine the impact of mutational burden on outcomes. Methods: A multicenter retrospective analysis of a cohort of 84 patients was carried out. This included 52 patients treated on the MPD-RC 101 prospective study (NCT00572897), 18 patients treated at Prince Margaret Hospital, and 14 patients treated at Memorial Sloan Kettering Cancer Center. Patient and transplant characteristics are displayed in Table 1. DNA was extracted from pre-transplant bone marrow aspirate samples or peripheral blood samples. High-throughput sequencing of a panel of genes was performed. Average coverage of 829x (standard deviation of ±130) was obtained. Mutect was utilized to call single point variants (comparing our samples to a pool of normal samples) and PINDEL was used to call short insertions and deletions. We excluded all mutations present in at least one database of known non-somatic variants (DBSNP and 1000 genomes) and absent from COSMIC. Univariate Cox regression and Kaplan-Meier graphics were used to investigate the association of patient, transplant, and disease characteristics with OS and RFS. Results: JAK2V617F was the most frequent mutation detected in 41(48.8%) patients (Table 2). Eighteen patients (21.4%) had triple negative disease (negative for JAK2, MPL, and CALR mutations). Univariate analysis included the following: patient characteristics (age, gender), transplant characteristics (related vs. unrelated donor, matched vs. mismatched donor and myeloablative vs. reduced intensity conditioning) and disease characteristics (DIPSS and presence of mutations). Decreased OS was associated with unrelated donor status (HR 2.09, 95% CI: 1.03-4.23, p=0.04), reduced intensity conditioning (HR 4.21, 95% CI: 1.01-17.59, p=0.049), triple negative disease (HR 2.09, 95% CI: 1.02-4.30, p=0.04), and presence of U2AF1 (HR 2.53, 95% CI: 1.10-5.81, p=0.03) or SUZ12 mutations (HR 3.92, 95% CI: 1.19-12.21, p=0.02). Decreased RFS was associated with unrelated donor status (HR 2.27, 95% CI: 1.16-4.45, p=0.02), and the presence of SUZ12 mutation (HR 6.97, 95% CI: 2.37-20.49, p<0.001). A descriptive decrease in RFS in patients with U2AF1 (HR 2.15, 95% CI: 0.94-4.88, p=0.07) was observed but did not reach statistical significance. Importantly, mutations previously reported to be associated with reduced OS and RFS in the non-transplant setting, such as ASXL1, EZH2, IDH1/2, and SRSF2, were not associated with poorer outcomes in this analysis in transplanted patients. In an exploratory multivariate analysis including donor type (related vs. unrelated) and presence of U2AF1 and SUZ12 mutations, there was a significantly reduced OS and RFS in patients who harbor these mutations regardless of donor type (OS: HR 5.30, 95% CI: 2.08-13.47, p<0.001; RFS: HR 5.49, 95% CI: 2.27-13.30, p<0.001). In patients without the above mutations, having an unrelated donor was associated with worse OS (HR 2.55, 95% CI: 1.09-5.96, p=0.03) and RFS (HR 2.61, 95% CI: 1.17-5.83, p=0.02, Figure 1). Conclusions: Our analysis demonstrates that mutations previously associated with poor prognosis in MF, such as ASXL1, do not appear to confer a worsened prognosis in patients undergoing allo-HSCT, suggesting transplant may be able to overcome the impact of these mutations. However, mutations in SUZ12 and U2AF1 are associated with reduced OS in univariate and multivariate analysis (together with donor type). Further studies with larger cohorts of patients are indicated to validate these findings, and to elucidate the impact of these mutations on disease biology. Disclosures Rampal: Incye and CTI: Consultancy. Mascarenhas:Janssen: Research Funding; CTi Biopharma: Research Funding; Promedior: Research Funding; Merk: Research Funding; Incyte: Research Funding. Mesa:Galena: Consultancy; Gilead: Research Funding; Promedior: Research Funding; Incyte: Research Funding; CTI Biopharma: Research Funding; Celgene: Research Funding; Ariad: Consultancy; Novartis: Consultancy. Gupta:Novartis: Consultancy, Honoraria, Research Funding; Incyte Corporation: Consultancy, Research Funding.

Abstract Background: Myeloproliferative neoplasms (MPN) including polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF) are clonal hemopathies characterized by burdensome symptom profiles and impaired quality of life. Few studies have evaluated patient-reported outcomes during treatment with non-experimental pharmacological regimens. Aims: The Myeloproliferative Neoplasm Quality of Life (MPN-QOL) Study Group aims to objectively quantify MPN symptom severity, frequency and quality of life at baseline and throughout treatment with non-experimental therapies utilizing the Myeloproliferative Neoplasm Symptom Assessment Form - Total Symptom Score (MPN-SAF TSS; JCO 2012). In this abstract, we provide updated results for the prospective international cohort trial currently in active enrollment: the MPN Experimental Assessment of Symptoms by Utilizing Repetitive Evaluation (MEASURE) trial. Methods: This study aims to recruit 180 international ET, PV, and MF (including primary MF and post-ET or post-PV MF) patients receiving non-experimental medical therapy and/or phlebotomy. Patients complete the MPN-SAF for seven consecutive days at enrollment and repeat the survey for an additional seven consecutive days between 90 days and six months. Patients also complete the European Organisation for Research and Treatment of Cancer (EORTC) and M.D. Anderson Symptom Inventory (MDASI) instruments at enrollment and on the first day of the second assessment. At visits, physicians acquired demographic, laboratory, physical examination, and radiographic data. Descriptive statistics were used to summarize data. Results: Clinical Data The MEASURE trial opened for enrollment in 2012 and remains in recruitment phase with 15 participating international sites. To date, 39 patients have been enrolled and 25 have completed both study visits. Participants include ET (28%), PV (24%), and MF (48%; 50% primary MF, 8% post-ET, 42% post-PV) patients. The majority of patients are male (64%) and of expected age (mean 69.3, range 39-89) for the disorders. Seventeen percent had prior thrombosis, 9% required red blood cell transfusion, and none reported prior splenectomy or hemorrhage. Mean hematologic measures included hemoglobin 13.2 g/dL, WBC count 11.4 x109/L, ANC 8.5 x109/L, and platelets 514 x109/L. Therapies received prior to enrollment included aspirin (n=16), hydroxyurea (n=11), phlebotomy (n=8), warfarin/clopidogrel/anticoagulation (n=8), erythropoietin (n=2), and interferon (n=1). The most common current MPN therapies were hydroxyurea (n=9), aspirin (n=9), interferon (n=4), and phlebotomy (n=2). Symptom Assessment In comparing MPN-SAF TSS mean symptom scores, all symptoms except bony pain improved between the first and second visits, including fatigue, early satiety, abdominal discomfort, inactivity, concentration, night sweats, itching, fever, weight loss, and overall quality of life (Figure1). Total MPN-SAF TSS scores improved from a mean of 32.3 to 25.9. On the EORTC, mean scores for physical, role, emotional, and social functioning improved from the first to the second visit (Figure 2). Cognitive functioning showed a slight decline. Global health status measure improved from 60.2 to 72.9. On the MDASI, symptom severity scores decreased from 3.6 to 2.8 from the first to second visit (Figure 3). Symptom distress measure decreased from 4.1 to 3.0. Discussion: Interim results from the MEASURE trial demonstrate that standard, non-experimental treatment regimens offer improvement in quality of life-related symptoms on multiple patient-reported survey instruments including the MPN-SAF TSS, EORTC QLQ-C30, and MDASI. Updated data including symptom correlations and mutational analysis to be presented at the 2016 ASH conference. Disclosures Ross: Novartis Pharmaceuticals: Honoraria, Research Funding; BMS: Honoraria. Radia:Novartis: Honoraria; Pfizer: Honoraria. McMullin:Novartis: Honoraria, Speakers Bureau. Cargo:Novartis: Honoraria; Celgene: Honoraria, Research Funding. Sekhar:Novartis: Research Funding. Mesa:Gilead: Research Funding; CTI Biopharma: Research Funding; Galena: Consultancy; Ariad: Consultancy; Incyte: Research Funding; Novartis: Consultancy; Celgene: Research Funding; Promedior: Research Funding.

INTRODUCTION: Prometheus IBD sgi Diagnostic (SGI) is an Inflammatory Bowel Disease (IBD) advanced diagnostic test developed by Prometheus Laboratories. It employs a diagnostic algorithm combining serologic, genetic, and inflammatory markers to help with clinical decision making in determining the accuracy of a diagnosis of IBD in patients suspected to have the disease. Moreover, among those it identifies as having IBD, it also seeks to assist in distinguishing those with Crohn's vs. those with ulcerative colitis (UC). In this observational clinical decision making study, we sought to examine the real world application of the SGI at a single tertiary care IBD practice especially in correctly identifying those with Crohn's vs. those with UC. METHODS: We included all adult IBD patients seen at a single tertiary IBD center practice from 2014 to 2017 that had baseline SGI done and during care at the practice, had a diagnosis of IBD (Crohn's or UC) established. We calculated sensitivity, specificity, positive predictive value (PPV), negative predictive value (NPV), accuracy, and area under the curve based on results of SGI at baseline visit and the eventual established diagnosis of UC or CD. RESULTS: Of a total of 275 patients, there were 96 (34.9%) male patients and 179 (65.1%) female patients. The median age of these patients was 45.8. There were a total of 201 patients with biopsy proven CD. Of these, 104 were identified as having CD based on the baseline SGI and 97 who were identified as not having CD. Based on these data, the sensitivity of the SGI for CD at the practice was 52%, specificity was 88%, PPV was 92%, NPV was 40.1%, diagnostic accuracy was 61.5%, and Area under ROC was 70%. There were 66 patients with biopsy proven UC. Of these, 44 were identified as having UC based on the baseline SGI and 22 were identified as not having UC. Based on these data, the sensitivity of the SGI for UC at the practice was 67%, specificity was 85%, PPV was 58%, NPV was 89%, accuracy was 80%, and Area under ROC was 76%. CONCLUSION: Based on the results of this study, we conclude that the SGI is not a robust initial test in identifying those with Crohn's or those with UC. However, in a cohort of IBD patients seen at a tertiary care IBD practice, it is reasonably specific and accurate when it comes to correctly confirming Crohn's and even more so UC in patients with a caveat that the results should be interpreted in the proper clinical context and with the backing of other clinical indicators.

INTRODUCTION: Crohn's Disease (CD) is a chronic inflammatory condition, primarily of the gastrointestinal tract with an increasing incidence and prevalence worldwide. In clinical practice, gastroenterologists are often faced with challenges in evaluating disease activity, severity, and prognosis to guide appropriate management of CD. We studied the association between serum albumin level and the rate of active clinical disease in CD patients seen at a tertiary care Inflammatory Bowel Disease (IBD) referral center. METHODS: We designed a retrospective cohort study using adult patients (>18 years) with CD followed for at least one year at University of Alabama at Birmingham (UAB) IBD center by a single trained IBD specialist between 2014-2018. Serum albumin levels were divided into: low mean serum albumin (≤3.2 mg/dL) vs. appropriate mean serum albumin (>3.2 mg/dL). Clinical disease activity was measured using the Harvey-Bradshaw index (HBI). Active disease was defined as HBI score of 8 or higher. Poisson Regression Models (GPR) for Rate Data were used to estimate partially adjusted and fully adjusted incidence rate ratios (IRR) of active clinical disease among CD patients. We also examined IRRs for serum albumin level as a continuous variable. RESULTS: In this single IBD practice at UAB, 269 patients with CD had albumin drawn at initial visit and serially at each subsequent visit. Poisson regression model adjusted for age, gender, race, duration of disease, steroid use, smoking and Crohns location demonstrated that CD patients with a low mean serum albumin during clinical observation were approximately 1.5 times more likely to have active clinical disease during observation (IRR 1.49, 95% CI 1.04-2.15, P = 0.032) compared to those with a serum albumin level of >3.2 mg/dL. The adjusted model with serum albumin as a continuous variable demonstrated that with every unit (0.1 mg/dL) reduction in mean serum albumin, there was 30% higher likelihood of active Crohns in clinic (IRR 1.30, 95% CI 1.02-1.66, P = 0.037). CONCLUSION: Lower mean serum albumin during clinical observation is associated with a higher likelihood of active clinical disease among CD patients. Whether interventions designed to correct or increase serum albumin stores/levels in these patients would be beneficial remains to be examined.