Search

In prior work, Friends of Cancer Research convened multiple data partners to establish standardized definitions for oncology real-world end points derived from electronic health records (EHRs) and claims data. Here, we assessed the performance of real-world overall survival (rwOS) from data sets sourced from EHRs by evaluating the ability of the end point to reflect expected differences from a previous randomized controlled trial across five data sources, after applying inclusion/exclusion criteria. The KEYNOTE-189 clinical trial protocol of platinum doublet chemotherapy (chemotherapy) vs. programmed cell death protein 1 (PD-1) in combination with platinum doublet chemotherapy (PD-1 combination) in first-line nonsquamous metastatic non-small cell lung cancer guided retrospective cohort selection. The Kaplan-Meier product limit estimator was used to calculate 12-month rwOS with 95% confidence intervals (CIs) in each data source. Cox proportional hazards models estimated hazard ratios (HRs) and associated 95% CIs, controlled for prognostic factors. Once the inclusion/exclusion criteria were applied, the five resulting data sets included 155 to 1,501 patients in the chemotherapy cohort and 36 to 405 patients in the PD-1 combination cohort. Twelve-month rwOS ranged from 45% to 58% in the chemotherapy cohort and 44% to 68% in the PD-1 combination cohort. The adjusted HR for death ranged from 0.80 (95% CI: 0.69, 0.93) to 1.15 (95% CI: 0.71, 1.85), controlling for age, gender, performance status, and smoking status. This study yielded insights regarding data capture, including ability of real-world data to precisely identify patient populations and the impact of criteria on end points. Sensitivity analyses could elucidate data set-specific factors that drive results.

INTRODUCTION AND OBJECTIVE:Primary treatment options for patients with high-risk non-metastatic prostate cancer (CaP) include radical prostatectomy or radiation therapy. The optimal treatment appro...

The purpose of this study was to evaluate the potential collective opportunities and challenges of transforming real-world data (RWD) to real-world evidence (RWE) for clinical effectiveness by focusing on aligning analytic definitions of oncology endpoints. Patients treated with a qualifying therapy for advanced non-small cell lung cancer (aNSCLC) in the frontline setting meeting broad eligibility criteria were included to reflect the real-world population. While a trend toward improved outcomes in patients receiving PD-(L)1 therapy over standard chemotherapy was observed in RWD analyses, the magnitude and consistency of treatment effect was more heterogeneous than previously observed in controlled clinical trials. The study design and analysis process highlighted the identification of pertinent methodological issues and potential innovative approaches that could inform the development of high-quality RWD studies.

Background: Outpatients with cancer commonly have nonmedical opioid use (NMOU) behaviors and use opioids to dull emotional and existential suffering. Buprenorphine is often used for cancer pain due to less reported euphoria when compared to other opioids. Methods: A retrospective review was done in patients who were prescribed buprenorphine for cancer pain. Pain scores were reported on a Likert pain scale of 1 to 10. Nonmedical opioid use was defined as patients taking opioids for emotional pain at or above the maximum prescribed amount. Results: For 16 patients, the mean pain score prior to buprenorphine (pain pre) was 8.3 (Standard deviation (Std) 1.6), and the mean pain score on follow-up post-buprenorphine (pain post) was 6.1 (Std 2.3) with a reduction in mean pain score (pain change) of −2.0 (Std 2.9, P = .059). Those patients without NMOU had a pain prescore of 9.5 (Std 1.0) and pain post of 4.3 (Std 2.5) with a mean pain change of −5.0 (Std 1.7, P = .20). The mean pain change in those with chemical coping (−1.3/Std 2.7), illicit drug use (−2.8/Std 1.0), or psychiatric comorbidity (−2.4/Std 2.7) were reduced after buprenorphine, however, not statistically significant. Outpatient rotation to buprenorphine was well tolerated. Conclusions: The pain score in those patients without NMOU was significantly lower after rotation to buprenorphine than those with NMOU. We deduce that in those with NMOU, it is more challenging to achieve pain relief with buprenorphine. Overall, for all patients, rotation to buprenorphine resulted in a marginally significantly reduced pain score.

In recent years, the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Adult Cancer Pain have undergone substantial revisions focusing on the appropriate and safe prescription of opioid analgesics, optimization of nonopioid analgesics and adjuvant medications, and integration of nonpharmacologic methods of cancer pain management. This selection highlights some of these changes, covering topics on management of adult cancer pain including pharmacologic interventions, nonpharmacologic interventions, and treatment of specific cancer pain syndromes. The complete version of the NCCN Guidelines for Adult Cancer Pain addresses additional aspects of this topic, including pathophysiologic classification of cancer pain syndromes, comprehensive pain assessment, management of pain crisis, ongoing care for cancer pain, pain in cancer survivors, and specialty consultations.

In the era of effective antiretroviral therapy, persons living with HIV/AIDS (PLWHA) are living longer, transforming HIV from a universally fatal disease to a serious chronic illness, warranting discussions between patients and their loved ones about advance care planning (ACP). Evidence is needed on factors associated with patients' likelihood to discuss ACP with loved ones.To further characterize factors associated with successful ACP in PLWHAs with their loved ones, we examined associations between patients having ACP discussions with the need for assistance with personal care, chronic pain, life satisfaction, prior family disagreements over health care decisions, sex, age, and interference in daily routines due to memory problems.Data were from the Affirm Care study (N = 370), which examined social and environmental factors associated with health outcomes among PLWHAs and their informal caregivers.Slightly more than half of respondents discussed ACP with loved ones (57%). In adjusted analysis, higher levels of chronic pain (odds ratio [OR] = 2.09, P = 0.045), needing assistance with personal care (OR = 1.63, P = 0.023), greater life satisfaction (OR = 1.02, P = 0.002), prior family arguments over health care decisions (OR = 2.80, P 0.001), and female sex (OR = 2.22, P = 0.001) were associated with higher odds of discussing ACP with loved ones, whereas age, drug use, education level, depression, and memory problems were nonsignificant.These results suggest that interventions to increase ACP among PLWHAs and their loved ones should target males. The findings also suggest PLWHAs with chronic pain, the need for assistance with personal care, and those with a history of prior family arguments over health care decisions may be primed for ACP.

Purpose To describe a patient with an unusual presentation of iris metastasis from breast cancer and her response to systemic therapy. Methods Retrospective chart review of one patient. Results A 57-year-old woman presented with a superonasal translucent vascularized iris stromal mass with fish egg-like structures budding from the surface. High frequency anterior segment ultrasonography demonstrated a solid iris stromal mass measuring 6.0 x 3.3 x 1.9 mm. On optical coherence tomography, the egg-like structures appeared as hyperreflective spheres, some of which were detached from the main iris stromal tumor. Oncologic evaluation revealed metastatic breast cancer involving the brain and lung. She was treated with oral abemaciclib and letrozole, as well as external beam radiotherapy to the brain. The iris mass had completely regressed within 4 months and remained undetectable through 8 months follow-up. The other metastatic lesions responded well to therapy. Conclusion We report a case of iris metastasis as the presenting sign of cancer dissemination that was successfully treated with targeted systemic therapy without ocular radiotherapy.

Background African Americans (AA) are disproportionately affected by multiple myeloma (MM) with over double the incidence and mortality rates as compared to white patients. Although there have been significant therapeutic advances for the treatment of MM over the last 20 years, research has suggested that these benefits may not be uniform across racial groups. We sought to explore racial disparities in multiple myeloma using contemporary real-world data (RWD). Methods A total of 1790 patients meeting the study criteria were identified in the COTA real-world database, a de-identified database of RWD derived from the electronic health records of healthcare providers in the United States. Study eligibility criteria included active MM diagnosis after Jan. 1, 2015, and white (n=1494) or AA (n=296) race. Patient characteristics and treatment patterns were summarized by line of therapy (LOT). LOT was assigned programmatically on the retrospective RWD using an algorithm based on IMWG criteria and clinical guidance. Time to next treatment (TTNT) was calculated using the Kaplan-Meier method. Results In comparison to white patients, the AA cohort consisted of younger (median age 61.0 vs 66.0 yrs, p Conclusions These findings suggest that frontline treatment patterns were similar by race in a contemporary real-world cohort treated predominately in the academic setting. Despite delays in 1L therapy initiation among African American patients, TTNT from 1L to 2L were similar. Additional disparities were observed in testing rates for 1p deletion and tp53. Future analyses will investigate treatment patterns by practice setting and expand into later LOTs to further elucidate if academic practices and uniform treatment patterns drive similar outcomes by race.

Immune checkpoint inhibitors (ICIs) are the standard of care for the treatment of several cancers. While these immunotherapies have improved patient outcomes in many clinical settings, they bring accompanying risks of toxicity, specifically immune-related adverse events (irAEs). There is a need for clear, effective guidelines for the management of irAEs during ICI treatment, motivating the Society for Immunotherapy of Cancer (SITC) to convene an expert panel to develop a clinical practice guideline. The panel discussed the recognition and management of single and combination ICI irAEs and ultimately developed evidence- and consensus-based recommendations to assist medical professionals in clinical decision-making and to improve outcomes for patients.

Background The COVID-19 pandemic remains a significant global threat. However, despite urgent need, there remains uncertainty surrounding best practices for pharmaceutical interventions to treat COVID-19. In particular, conflicting evidence has emerged surrounding the use of hydroxychloroquine and azithromycin, alone or in combination, for COVID-19. The COVID-19 Evidence Accelerator convened by the Reagan-Udall Foundation for the FDA, in collaboration with Friends of Cancer Research, assembled experts from the health systems research, regulatory science, data science, and epidemiology to participate in a large parallel analysis of different data sets to further explore the effectiveness of these treatments. Methods Electronic health record (EHR) and claims data were extracted from seven separate databases. Parallel analyses were undertaken on data extracted from each source. Each analysis examined time to mortality in hospitalized patients treated with hydroxychloroquine, azithromycin, and the two in combination as compared to patients not treated with either drug. Cox proportional hazards models were used, and propensity score methods were undertaken to adjust for confounding. Frequencies of adverse events in each treatment group were also examined. Results Neither hydroxychloroquine nor azithromycin, alone or in combination, were significantly associated with time to mortality among hospitalized COVID-19 patients. No treatment groups appeared to have an elevated risk of adverse events. Conclusion Administration of hydroxychloroquine, azithromycin, and their combination appeared to have no effect on time to mortality in hospitalized COVID-19 patients. Continued research is needed to clarify best practices surrounding treatment of COVID-19.

Context: Malignant bowel obstruction (MBO) is a complication of advanced malignancy. For inoperable patients, symptoms are often treated using analgesics, anticholinergics, and anti-emetics. There are, however, few published guidelines for the medical management of MBO. Objective: To measure the effect of the combination of dexamethasone, octreotide, and metoclopramide (“triple therapy”) in patients with MBO, compared to patients who received none of the 3 medications (“no drug therapy”). Methods: A retrospective cohort study of patients with MBO admitted in a single-center comprehensive cancer center. Patients who received dexamethasone, octreotide, and metoclopramide during their hospitalization for treatment of inoperable MBO were selected for analysis. Patients were excluded if they received a venting gastric tube. Rate of de-obstruction as well as time to de-obstruction were measured. Results: There were 20 patients identified who received all 3 drugs of interest, and 29 patients identified who received none of the 3 medications. There was no statistically significant difference in rates of de-obstruction between the 2 groups, though there was a non-significant trend toward patients who received triple therapy were more likely to reach de-obstruction, compared to patients who had no drug therapy (95% vs. 83%, p = 0.379); there was no significant difference in adjusted analysis. Conclusion: In patients with inoperable MBO, there was no statistically significant difference in rates of de-obstruction with triple drug therapy compared to patients who received none of the 3 drugs, though the study may not have been powered to detect a difference and further investigation is warranted.

Summary Background Tocilizumab, a monoclonal antibody directed against the interleukin-6 receptor, has been proposed to mitigate the cytokine storm syndrome associated with severe COVID-19. We aimed to investigate the association between tocilizumab exposure and hospital-related mortality among patients requiring intensive care unit (ICU) support for COVID-19. Methods We did a retrospective observational cohort study at 13 hospitals within the Hackensack Meridian Health network (NJ, USA). We included patients (aged ≥18 years) with laboratory-confirmed COVID-19 who needed support in the ICU. We obtained data from a prospective observational database and compared outcomes in patients who received tocilizumab with those who did not. We applied a multivariable Cox model with propensity score matching to reduce confounding effects. The primary endpoint was hospital-related mortality. The prospective observational database is registered on ClinicalTrials.gov , NCT04347993 . Findings Between March 1 and April 22, 2020, 764 patients with COVID-19 required support in the ICU, of whom 210 (27%) received tocilizumab. Factors associated with receiving tocilizumab were patients' age, gender, renal function, and treatment location. 630 patients were included in the propensity score-matched population, of whom 210 received tocilizumab and 420 did not receive tocilizumab. 358 (57%) of 630 patients died, 102 (49%) who received tocilizumab and 256 (61%) who did not receive tocilizumab. Overall median survival from time of admission was not reached (95% CI 23 days–not reached) among patients receiving tocilizumab and was 19 days (16–26) for those who did not receive tocilizumab (hazard ratio [HR] 0·71, 95% CI 0·56–0·89; p=0·0027). In the primary multivariable Cox regression analysis with propensity matching, an association was noted between receiving tocilizumab and decreased hospital-related mortality (HR 0·64, 95% CI 0·47–0·87; p=0·0040). Similar associations with tocilizumab were noted among subgroups requiring mechanical ventilatory support and with baseline C-reactive protein of 15 mg/dL or higher. Interpretation In this observational study, patients with COVID-19 requiring ICU support who received tocilizumab had reduced mortality. Results of ongoing randomised controlled trials are awaited. Funding None.

BackgroundHydroxychloroquine has not been associated with improved survival among hospitalized COVID-19 patients in the majority of observational studies and similarly was not identified as an effective prophylaxis following exposure in a prospective randomized trial. We aimed to explore the role of hydroxychloroquine therapy in mildly symptomatic patients diagnosed in the outpatient setting.MethodsWe examined the association between outpatient hydroxychloroquine exposure and the subsequent progression of disease among mildly symptomatic non-hospitalized patients with documented SARS-CoV-2 infection. The primary outcome assessed was requirement of hospitalization. Data was obtained from a retrospective review of electronic health records within a New Jersey USA multi-hospital network. We compared outcomes in patients who received hydroxychloroquine with those who did not applying a multivariable logistic model with propensity matching.ResultsAmong 1274 outpatients with documented SARS-CoV-2 infection 7.6% were prescribed hydroxychloroquine. In a 1067 patient propensity matched cohort, 21.6% with outpatient exposure to hydroxychloroquine were hospitalized, and 31.4% without exposure were hospitalized. In the primary multivariable logistic regression analysis with propensity matching there was an association between exposure to hydroxychloroquine and a decreased rate of hospitalization from COVID-19 (OR 0.53; 95% CI, 0.29, 0.95). Sensitivity analyses revealed similar associations. QTc prolongation events occurred in 2% of patients prescribed hydroxychloroquine with no reported arrhythmia events among those with data available.ConclusionsIn this retrospective observational study of SARS-CoV-2 infected non-hospitalized patients hydroxychloroquine exposure was associated with a decreased rate of subsequent hospitalization. Additional exploration of hydroxychloroquine in this mildly symptomatic outpatient population is warranted.Lay SummaryIn this observational study of 1,274 COVID-19 patients, hydroxychloroquine given as an outpatient treatment was associated with a 47% reduction in the hazard of hospitalization. Adverse events were not increased (2% QTc prolongation events, 0% arrhythmias). Further validation is required. Use of hydroxychloroquine to treat COVID-19 in the outpatient setting should be reserved for a clinical trial or after discussion with a physician regarding risks and benefits.

IntroductionCOVID-19 disproportionately affects those with comorbidities and the elderly. Hypertension is the most common pre-existing condition amongst COVID-19 patients. Upregulation of the renin-angiotensin-aldosterone system (RAAS) is common in hypertensive patients and may promote inflammation and ensuing cytokine storm in COVID-19. It is unknown whether RAAS inhibition with ACE1 inhibitors or angiotensin-receptor blockers (ARB) can be harmful or beneficial.MethodsWithin Hackensack Meridian Health network, the largest healthcare provider in New Jersey, we performed a retrospective, multicenter, convenience sampling study of hospitalized COVID-19 patients. Demographics, clinical characteristics, treatments, and outcomes were manually abstracted. Fishers exact tests, and logistic regression were performed.ResultsAmong 3017 hospitalized COVID-19 patients, 1584 (52.5%) carried a diagnosis of hypertension. In the discharged or deceased cohort, the overall mortality was significantly increased at 35% vs 13% among COVID-19 patients with hypertension. However, when adjusted for age, the effect of hypertension on mortality was greatly diminished, with a reduction in odds-ratio by over half; and completely disappeared when adjusted for other major covariates. The mortality rates were lower for hypertensive patients prescribed ACE1 (27%, p=0.001) or ARBs (33%, p=0.12) compared to other anti-hypertensive agents (39%) in the unadjusted analyses. RAAS inhibitor therapy appeared protective compared to other anti-hypertensive agents (p=0.001).ConclusionsWhile our results are limited by the retrospective nature of our study and by potential confounders, our data argue against a harmful effect of RAAS inhibition and support the HFSA/AHA/ACC joint statement recommending continuing ACE1 and ARB therapy in hypertensive COVID-19 patients.

As palliative care (PC) continues its rapid growth, an emerging body of evidence is demonstrating that its approach of interdisciplinary supportive care benefits many patient populations, including in the oncology setting. As studies and data proliferate, however, questions persist about who, what, why, when, and how PC as well as the ideal time for a PC consult and length of involvement. When comparing outcomes from chemotherapy trials, it is important to consider the dosing regimens used in the various studies. In the same way, it is important to account for the “dose” of the PC interventions utilized across studies, and apples to apples comparisons are needed in order to draw accurate conclusions about PC’s benefits. Studies which include a true interdisciplinary PC intervention consistently show improvements in patient quality of life, as well as cost savings, with further study needed for other outcomes. These benefits cannot be extrapolated to care which may be labeled “palliative care,” but which does not meet the standard of true interdisciplinary PC. The ultimate question is: Does PC indeed improve outcomes?

BackgroundHydroxychloroquine has been touted as a COVID-19 treatment. Tocilizumab, an inhibitor of IL-6, has been proposed as a treatment of critically ill patients.ObjectiveTo describe the association between mortality and hydroxychloroquine or tocilizumab therapy among hospitalized COVID-19 patients.DesignRetrospective observational cohort study of electronic health records Setting: 13-hospital network spanning the state of New Jersey.ParticipantsPatients hospitalized between March 1, 2020 and April 22, 2020 with positive polymerase chain reaction results for SARS-CoV-2. Follow up was through May 5, 2020.Main OutcomesThe primary outcome was death.ResultsAmong 2512 hospitalized patients with COVID-19 there have been 547 deaths (22%), 1539 (61%) discharges and 426 (17%) remain hospitalized. 1914 (76%) received at least one dose of hydroxychloroquine and 1473 (59%) received hydroxychloroquine with azithromycin. After adjusting for imbalances via propensity modeling, compared to receiving neither drug, there were no significant differences in associated mortality for patients receiving any hydroxychloroquine during the hospitalization (HR, 0.99 [95% CI, 0.80-1.22]), hydroxychloroquine alone (HR, 1.02 [95% CI, 0.83-1.27]), or hydroxychloroquine with azithromycin (HR, 0.98 [95% CI, 0.75-1.28]). The 30-day unadjusted mortality for patients receiving hydroxychloroquine alone, azithromycin alone, the combination or neither drug was 25%, 20%, 18%, and 20%, respectively. Among 547 evaluable ICU patients, including 134 receiving tocilizumab in the ICU, an exploratory analysis found a trend towards an improved survival association with tocilizumab treatment (adjusted HR, 0.76 [95% CI, 0.57-1.00]), with 30 day unadjusted mortality with and without tocilizumab of 46% versus 56%.ConclusionsThis observational cohort study suggests hydroxychloroquine, either alone or in combination with azithromycin, was not associated with a survival benefit among hospitalized COVID-19 patients. Tocilizumab demonstrated a trend association towards reduced mortality among ICU patients. Our findings are limited to hospitalized patients and must be interpreted with caution while awaiting results of randomized trials.Trial RegistrationClinicaltrials.gov Identifier: NCT04347993

Immune checkpoint inhibitors (ICIs) have opened an exciting chapter in the treatment of patients with advanced cancer. For the palliative care clinician, however, ICIs present several new challenges, including new ways to define treatment success, as well as treatment-related toxicities that differ in nature and timing from traditional chemotherapy. In this article, we review the mechanism of action of ICIs, as well as selected published data supporting the efficacy of ICIs in patients with advanced cancer. In addition, we summarize existing data of ICI toxicity prevalence, patterns of severity, and timing of onset. Finally, we briefly review key principles from published guidelines on the management of ICI toxicities.

Background: Multiple myeloma (MM) risk stratification schemata such as the International Staging System (ISS) and Revised-ISS (R-ISS) were derived from clinical trial subjects made up predominately of younger White individuals with adequate renal function. It is unknown whether these prognostic indices are applicable to all patients with newly diagnosed (ND) MM, especially among Black individuals, older adults, and those with renal dysfunction. The R-ISS expanded on the ISS by including and serum lactate dehydrogenase (LDH) and high-risk cytogenetic abnormalities (HRCA) identified by fluorescence in-situ hybridization (FISH), but HRCA may not translate into poor prognosis for older adults and for Black individuals. We sought to create an inclusive risk prognostic index for NDMM using real-world data derived from electronic health records. Methods: De-identified NDMM patient-level data in the real-world setting was provided by COTA, Inc. 3000 patients were identified who met the inclusion criteria of NDMM between 2005 and 2020. Baseline diagnostic parameters available within 60 days before or after diagnosis were included. Progression free survival (PFS) was defined as the time from diagnosis to disease progression or death of any cause. Overall survival (OS) was defined as the time from diagnosis to death of any cause. Proportional hazards models were used to calculate hazard ratios (HR) and 95% confidence intervals for all-cause mortality. Age-adjusted univariate analyses identified variables significantly associated with OS, and continuous variables were dichotomized based on accepted cutoffs. Multivariate Cox models to identify the variables with the strongest association with OS were performed adjusting for age, sex, Black race, receipt of proteasome inhibitor and immunomodulatory imide during induction, autologous stem cell transplant within 1 year of diagnosis, ECOG performance status, and creatinine. An additive risk score was created with one point given to each significant variable. The risk score was then validated for PFS using the Multiple Myeloma Research Foundation's (MMRF) CoMMpass database (version IA15). Results: 3000 NDMM pts from the COTA, Inc. real-world database were initially evaluated, and a total of 689 NDMM pts had sufficient level of data to be included. The median follow-up time was 49.9 months (interquartile range (IQR) 29.1-76.2 months). Median age was 64 (IQR 32-86), including 44% age 65+. Of the 607 with reported race, 474 (78%) were White, 86 (14%) Black, 17 (3%) Asian, and 30 (5%) other. Of the 676 pts with reported serum creatinine (mg/dL), the median was 1 mg/dL (IQR 0.8-1.3) with 85 (13%) measuring >2 mg/dL. Examined peripheral blood variables were: calcium (corrected for albumin), albumin3.5 mcg/mL, LDH >250 U/L, hemoglobin 3 g/dL, and IgA isotype. Variables with significance using multivariate analysis at p250 U/L, B2M >3.5 mcg/mL, hemoglobin Validation of the inclusive risk prognostic index (IRPI) using the MMRF CoMMpass database in 938 patients with all four criteria showed median PFS was 44 (std) vs 33 (int) vs 20.5 months (high). In comparison, median PFS by ISS was 45.9 (I) vs 31.5 (II) vs 20.5 (III) months. Median PFS by R-ISS was 50.1 (I) vs 32.7 (II) vs 19.1 (III) months. Conclusions: Employing real-world datasets that incorporate a more diverse patient population led to the generation of an inclusive risk prognostic index incorporating beta2-microgloublin, LDH, hemoglobin, and IgA isotype. This IRPI does not require bone marrow sampling, performs similarly to ISS and R-ISS in predicting PFS, and with datasets with longer follow-up may prove to predict OS. Figure 1 Figure 1. Disclosures Derman: Sanofi: Membership on an entity's Board of Directors or advisory committees. Belli: COTA, Inc.: Current Employment, Other: Equity ownership. Wang: COTA, Inc.: Current Employment, Other: Equity ownership. Hansen: COTA, Inc.: Current Employment. Jakubowiak: Amgen: Membership on an entity's Board of Directors or advisory committees; Karyopharm: Membership on an entity's Board of Directors or advisory committees; Gracell: Membership on an entity's Board of Directors or advisory committees; BMS: Membership on an entity's Board of Directors or advisory committees; GSK: Membership on an entity's Board of Directors or advisory committees; Abbvie: Membership on an entity's Board of Directors or advisory committees; Sanofi: Membership on an entity's Board of Directors or advisory committees; Janssen: Membership on an entity's Board of Directors or advisory committees; Celgene: Membership on an entity's Board of Directors or advisory committees.

Introduction: Acute myeloid leukemia (AML) is a disease of the older population, with a median age at diagnosis of 68 years. Outcomes remain poor for patients (pts) older than 60 years of age who are unfit to receive intensive chemotherapy. Ven based combination therapy has become the standard of care based on the results of the Viale-A and Viale-C in combination with hypomethylating agents (HMA) or low dose cytarabine (LDAC), respectively. This combination therapy is myelosuppressive leading to multiple dose adjustments in the real-world setting, which may impact the validity of the trial responses and overall survival (OS) in a community-based practice. We therefore sought to investigate the outcomes of pts treated with ven based combination therapy in the real-world setting to better understand efficacy and patterns of adverse events. Methods: Pts 65 years of age or older, diagnosed with AML on or after January 1, 2019, and who received frontline ven based combination therapy with azacitidine (aza), decitabine (dac) or LDAC were identified in the COTA real-world database. The COTA database is a USA-based dataset comprised of longitudinal, Health Insurance Portability and Accountability Act (HIPAA)-compliant data on the diagnosis, clinical management, and outcomes of pts with cancer. Clinical outcomes, including real-world overall response rate (rwORR), OS and adverse events were calculated by treatment group. Responses were defined per treating physician as complete response (CR) or partial (PR) or progressive disease (PD). Categorical variables and rwORR were compared using Fisher's exact test. Survival outcomes were calculated using the Kaplan-Meier method and compared amongst groups using log-rank test. Results: A cohort of 112 pts were treated with ven based combination: ven/aza (n=54), ven/dac (n=52) and ven/LDAC (n=6). Across all treatment groups, the majority (91.1%) of pts were treated in the community setting. The median age at diagnosis was 77 yrs (IQR: 71-81 yrs). Per ELN risk, 6.2% had favorable, 19.6% had intermediate and 62.5% had high-risk AML. Five of the 6 pts receiving ven/LDAC had secondary AML (Table 1). The most common molecular abnormalities across the cohort were TP53 (38.4%), TET2 (26.8%), ASXL1 (27.7%), and DNMT3A (20.5%). The rwORR by treatment was 57.7% for ven/dac, 55.6% for ven/aza and 33.3% for ven/LDAC (ven/dac vs. ven/aza, p=0.85). Median OS (mOS) was similar for ven/dac with a longest mOS of 13.9 mo, followed by ven/aza with a mOS of 11.3 mo, and ven/LDAC mOS of 6.5 mo (ven/dac vs. ven/aza, p=0.77). The most common reasons for treatment discontinuation were toxicity (32.1%) followed by progression/inadequate response (10.7%). The most common toxicities were related to myelosuppression/cytopenias (Table 3). In this cohort, only 6 pts underwent an allogeneic stem cell transplant in first remission and no pts underwent an allogeneic transplant in second remission. Conclusions: To our knowledge, this dataset represents the largest cohort of pts treated with ven based combination therapy in the real world setting across multiple community practices. As less intense regimens are used to treat AML, more pts are receiving induction in the community setting, making these findings relevant in clinical practice. Although ven/dac combination had the longest OS, this was not significantly different from ven/aza. The ven/HMA response rates were lower and survival outcomes were shorter than those reported in the Viale-A trial, which raises concerns about early discontinuation of therapy in the real world setting due to myelosuppression. Ven/LDAC combination had the shortest OS, though most of these patients had sAML. Despite this enriched population for targeted therapies, the outcomes remained poor overall. There are drawbacks to interpreting retrospective outcomes data. First, there were no treatment related AML cases in this dataset, which would be highly unusual in an elderly population of 112 cases of AML. It is more plausible that the diagnosis of treatment related AML was not utilized in the patient's chart. Other data points that were not adequately captured were cause of death and International Working Group response criteria. Mutational panel testing remains underutilized for AML patients in a community-based practice setting. However, this dataset highlights the need for larger datasets to compare the outcomes of AML therapy by practice setting. Figure 1 Figure 1. Disclosures Madanat: Blue Print Pharmaceutical: Honoraria; Stem line pharmaceutical: Honoraria; Onc Live: Honoraria; Geron Pharmaceutical: Consultancy. Belli: COTA, Inc.: Current Employment, Other: Equity ownership. Hansen: COTA, Inc.: Current Employment. Foss: COTA, Inc.: Current Employment. Schulte: COTA, Inc.: Current Employment. Wang: COTA, Inc.: Current Employment, Other: Equity ownership.

Background: There have been significant advancements in the treatment of multiple myeloma (MM) over the last 20 years including an influx of recently approved novel therapies. However, with these advances in treatment, the optimal combination and sequence of agents remain largely unknown especially in relapsed/refractory MM. A major unmet need in MM is patients with high-risk disease. Revised International Staging System is used to identify patients with high-risk MM, however a better definition of high risk includes "functional high risk" (i.e. patients relapsing within 18 months from diagnosis). As it has been previously shown that clinical trial populations are not universally representative of those in the routine practice setting, real-world data (RWD) can provide valuable insight into this rapidly evolving treatment landscape and high-risk population. Here we explore treatment patterns and outcomes of functional high-risk MM, patients who relapse within 18 months of initial diagnosis. Methods: This retrospective study utilized the COTA real-world database, a de-identified database of RWD derived from the electronic health records of partnered healthcare providers in the United States. A total of 958 patients were identified as having been diagnosed with active MM between Jan. 1, 2015 and Jan. 1, 2020 and experienced early relapse (defined as relapse within 18 months of initial active MM diagnosis and treatment). Practice setting distribution of this cohort was 84% academic and 16% community. Line of therapy was assigned programmatically utilizing IMWG definitions and guidelines. Patient characteristics and treatment patterns across the first (1L) and second lines (2L) of treatment were assessed using descriptive statistics. Time to next treatment (TTNT) was calculated overall and within treatment subgroups of interest as a surrogate for progression-free survival. Results: Among this functional high-risk patient population, the mean age was 64 yrs. (SD ±11.3) and the patients were predominantly white (72.6%). The most common cytogenetic abnormalities at diagnosis were del(13) (46.0% positive), 1q (36.4%), and t(11;14) (25.4%). In 1L, the majority of patients received a triplet regimen (75.4%), most commonly proteosome inhibitor (PI) + immunomodulator (IMiD) + steroids (45.0%). A total of 56 patients (5.9%) received stem-cell transplant (SCT) in 1L. Overall, 16.3% of the population did not receive 2L therapy due to death. Among the patients who received 2L therapy after early relapse (N=869), 50.5% received an SCT. TTNT was significantly longer for patients receiving SCT in 2L as compared to those who did not (34.8 vs. 5.8 months, respectively). Among patients who did not receive SCT (N=430), triplet therapy was most common (61.2%) with PI + IMiD + steroids representing the largest regimen group (30.2%). Table 1 shows the distribution of PI drugs within the 2L PI + IMID + steroids group and their associated median TTNT. No significant differences were observed when comparing median TTNT of 2L PI + IMID + steroids to daratumumab-based regimens (5.3 vs. 5.6 months, respectively). Conclusions: In our real-world population, median TTNT for functional high-risk patients was 17.8 months. 50.5% of these functional high-risk patients received SCT in 2L with the most common induction regimen containing cyclophosphamide + etoposide + dexamethasone (CED). For patients not receiving SCT in 2L, the most common regimen type included PI + IMID + Steroids. In comparing antibody-based therapy vs. PI + IMID + dexamethasone-based therapy, daratumumab-based combinations showed no significant difference in unadjusted analysis. Our study highlights some very important observations in functional high-risk patients. First, if patients are SCT eligible and it is not performed as part of first line treatment, SCT still provides the best outcomes in regard to TTNT in the second line setting. For patients not receiving SCT, our RWD demonstrates that 30.2% receive PI + IMID + dexamethasone as 2L treatment with only 13% receiving carfilzomib-based combination. Our study highlights the poor outcome of functional high-risk patients and provides insight into treatment patterns in 2L therapy. Further research is needed to explore patient and disease characteristics of functional high-risk patients and study novel treatment strategies like CAR T cell therapy or T-cell engagers in these patient population. Disclosures Belli: COTA, Inc.: Current Employment, Current equity holder in private company. Hansen:COTA, Inc.: Current Employment, Current equity holder in private company. Kansagra:Alnylam Pharmaceuticals, Bristol Myers Squibb /Celgene, GlaxoSmithKline, Janssen, Pharmacyclics, Takeda Pharmaceuticals, Pfizer, Karyopharm Therpeutics: Other: Advisory Board. Dilwali:COTA, Inc.: Current Employment, Current equity holder in private company. Wang:COTA, Inc.: Current Employment, Current equity holder in private company.

e21156 Background: The identification of driver mutations and development of targeted therapies have significantly improved outcomes of patients with advanced non-small cell lung cancer (aNSCLC). It has been shown in clinical trials that immunotherapy (IO) has limited effectiveness in patients with aNSCLC and driver mutations, but as there is a lag in the widespread use of targeted agents for newer driver oncogenes it is important to define the efficacy of non-targeted treatments. We sought to explore the real-world treatment patterns and outcomes of patients with aNSCLC and driver mutations treated with first-line (1L) chemotherapy (chemo) as compared to chemo +/- IO. Methods: This retrospective study utilized the COTA real-world database, a de-identified database derived from the electronic health records of healthcare providers in the United States. Patients included in the study had diagnosed aNSCLC and at least one positive test result for BRAF v600, HER2 mutation, EGFR, MET, RET or ROS1 prior to the initiation of 1L therapy. Additionally, patients must have received frontline chemotherapy, IO monotherapy, or IO+chemo combination in the advanced setting. Outcomes to first-line treatment (real-world progression-free survival (rwPFS) and real-world time to next treatment (rwTTNT)) were calculated using the Kaplan-Meier method. Results: A total of 170 qualified patients were identified as having at least one positive mutation and having received frontline chemotherapy (n=119), IO monotherapy (n=28), or chemo+IO combination (n=23). Mean age was 65 yrs and patients were predominantly white (71.2%), treated in the academic setting (74.1%), and denovo metastatic (67.1%). Outcomes to 1L treatment are reported in Table. Patients treated with 1L chemo+IO combination had longer rwPFS and rwTTNT as compared to patients treated with chemo alone or IO monotherapy, though outcomes did not reach statistical significance. Conclusions: Our study showed that in a real-world population of patients with aNSCLC and driver mutations, patients treated with chemo+IO combination had longer rwPFS and rwTTNT as compared to those treated with chemo alone or IO monotherapy. The overall short duration of benefit supports the model of using targeted treatment as optimal first line therapy, and the overall lack of benefit from IO. [Table: see text]

e18749 Background: Friends of Cancer Research and five data partners used electronic health record-sourced datasets to conduct parallel analyses in real-world data (RWD). The study assessed estimates of rwOS among patients taking platinum doublet chemotherapy (chemo) or pembrolizumab in combination with chemo (IO+chemo) in first-line treatment for metastatic non-small cell lung cancer (mNSCLC). Methods: The KEYNOTE-189 clinical trial was selected as a reference for cohort selection in the RWD. We applied select available KEYNOTE-189 eligibility criteria to compare rwOS in a homogenous real-world population. Analyses were conducted following a mutually developed statistical analysis plan that was harmonized across data partners. Twelve-month rwOS, and unadjusted and adjusted hazard ratios (HR) for death were calculated using time-to-event methods with patients censored at last structured activity prior to the data cutoff. Results: The five datasets included patients treated with chemo (range of n=155 to 1501) and IO+chemo patients (n=36 to 405, Table). Twelve-month rwOS estimates ranged from 45% to 58% in the chemo arm and 44% to 68% in the IO +chemo arm. The HR for death comparing IO+chemo to chemo varied across groups, ranging from 0.79 (95% CI: 0.68, 0.92) to 1.10 (95% CI: 0.70, 1.72). The HR for death adjusted for age, gender, performance status, and smoking status did not significantly alter results, ranging from 0.80 (95% CI: 0.69, 0.93) to 1.15 (96% CI: 0.71, 1.85). Conclusions: The systematic evaluation of reproducibility and performance of rwOS across five real-world cohorts and the impact of implementing consistent eligibility criteria and methods was useful to understand the consistency of results in real-world populations and relationship between treatments and outcomes in the absence of randomization. A well-articulated research question and a pre-specified analytic plan that includes covariate definitions are necessary preliminary steps to guide data harmonization, standardization, and analysis. A range of observed results highlights the complexity of real-world data, including differences in sample size, underlying patient variability across datasets, and missing covariate data. The harmonization process and observed results underscore the importance of a well-articulated research question and a pre-specified analytic plan to guide data harmonization, standardization, and analysis. [Table: see text]

e16249 Background: The difference between the FOLFIRINOX and gemcitabine/nab-paclitaxel (GnP) regimens’ clinical trial designs limit the ability to generate cross-study comparisons. Therefore, there is a significant need to understand the impact of various demographic and clinical characteristics on the effectiveness of these systemic therapies in the real-world treatment setting. This study seeks to compare the real-world outcomes of patients with metastatic pancreatic cancer treated with frontline FOLFIRINOX or GnP. Methods: Patients with primary metastatic pancreatic cancer who received first-line (1L) FOLFIRINOX or GnP were identified in the COTA real-world database. The COTA database is a de-identified database of real-world data (RWD) derived from the electronic health records of healthcare providers in the United States. Real-world overall response rate (rwORR) was calculated as the proportion of patients achieving complete response (CR) or partial response (PR). Overall survival (OS) was calculated using the Kaplan-Meier method and multivariate analyses utilized Cox proportional hazards. Results: The overall qualified cohort (n=236) was stratified by 1L FOLFIRINOX (n=109) or GnP (n=127). Select patient characteristics are shown in table. Patients treated with 1L FOLFIRINOX showed greater rwORR as compared to those treated with GnP (68.8% vs. 55.9%, p=0.04). Additionally, patients treated with 1L FOLFIRINOX had longer median OS (14.4 vs 11.4 mos, respectively). In univariate analysis, patients treated with GnP had a greater chance of mortality (HR: 1.3, 95% CI: 1.0, 1.8, p=0.05). This relationship strengthened in multivariate analysis (GnP treated HR: 1.6, 95% CI: 1.1, 2.1, p=0.01). Conclusions: Due to lack of enrollment of representative patients in clinical trials and in the absence of a comparative clinical trial, real-world experience with chemotherapy regimens provide critical insights on the outcome of treatments. In our cohort, patients treated with frontline GnP had a significantly greater chance of mortality as compared to patients treated with frontline FOLFIRINOX. The FOLFIRINOX cohort also showed greater rwORR. Future research will continue to expand on treatment patterns in subsequent lines of therapy, as well as emerging therapy types, in order to better understand the optimal treatment sequence in metastatic pancreatic cancer.[Table: see text]

e19008 Background: Based upon interesting recent published findings, we characterize disparities in clinical characteristics, treatment patterns, and outcomes for African American (AA) patients with multiple myeloma (MM) using real-world (RW) data. Methods: A random sample of patients diagnosed with MM were selected from the COTA RW database for the retrospective analysis to be stratified by race, AA (n=395) and other (n=396). To adjust for differences in age and gender, case-control matching was performed. Patient characteristics and treatment patterns were summarized. A Cox proportional hazards model was used to evaluate the relationship between patient characteristics and time to stem cell transplant (SCT). Overall Survival (OS) was calculated using the Kaplan-Meier method. Results: Median time from diagnosis of active MM to start of first-line (1L) treatment was longer among AA as compared to the other cohort (23 vs. 17 days respectively, p=0.01); VRD was the most common 1L therapy (17.39% vs. 18.58% respectively). The percent of patients receiving SCT was similar across racial cohorts (64.56% vs. 66.16%), but AA patients had a longer time to first SCT (234 vs. 204.50 days, p=0.01). Among all patients, Charlson Comorbidity index (CCI) score of 0 (HR: 2.85, CI: 1.77-4.58) and CCI score of 1-2 (HR: 2.79, CI: 1.73-4.49) were significantly associated with an increased likelihood of receiving a SCT. MM diagnosis at an older age was associated with a lower likelihood of receiving a SCT (HR: 0.97, CI: 0.96-0.98). AA cohort exhibited a higher incidence of prior history of MGUS (10.63% vs 8.08%). Cytogenetic profile of the AA and other cohorts are shown in the Table below. Median OS was similar between the AA and other cohorts (145.61 vs. 146.50 months, respectively, p=0.88). Conclusions: AA patients had a longer duration of time to 1L therapy and 1st SCT in our RW study. Despite these differences in time to treatment, OS was similar across racial groups. Future studies are needed to explore the significance of our RW findings and other potentially important clinical characteristics. [Table: see text]

e19311 Background: Leveraging data from a collaboration with 9 data partners, Friends of Cancer Research convened the Real-world Evidence Pilot 2.0, to examine trends and real world (rw) data endpoints in immunotherapy (IO) use for the front line treatment of aNSCLC. Methods: This study leveraged parallel analyses of rw data elements across heterogenous data sources (EHR, administrative claims, and registry) to: a) describe trends in uptake and use of novel IO frontline therapy after advanced diagnosis in NSCLC patients treated in usual care settings and b) examine associations between treatment and rw outcomes at one-year follow-up. The proportion of patients treated on each regimen (IO single agent, chemo, or IO + chemo) from 2011 through 2017 were calculated. Analysis included proportion of patients across treatment regimens stratified by year to describe post approval uptake of IO. Kaplan-Meier survival estimates were reported to adjust for follow-up time and stratified by PD-L1 status and stage. Results: Seven datasets identified a range of 999 to 4617 patients per dataset for this analysis. Across datasets, 2508, 3446, and 4176 patients initiated treatment in 2015, 2016, and 2017, respectively. No patients received IO or IO + chemo regimens prior to 2015. Initial approvals for IO use in aNSCLC occurred in October 2015 and for first line in metastatic NSCLC in October 2016. When examining survival at 1 year, overall, OS in PD-(L)1 + patients appeared longer than those with a PD-(L)1 - status. Conclusions: RWE analyses may reveal important trends in clinical cancer patient care including patterns of off-label use. The heterogeneity in the timing of IO uptake across datasets ranged from immediately after approval to ~12 months post-approval. [Table: see text]

124 Background: The use of medical cannabis (MC) in cancer symptom treatment has been increasing. Since its legalization is limited to select states, there are few clinical trials that have studied the effectiveness and safety of MC, and even fewer studies in the elderly patient population. Given this, we aimed to evaluate the effects of MC on nausea, appetite, and body mass index (BMI) of elderly cancer patients. Methods: We conducted a retrospective chart review of patients age 65 and older prescribed MC in the year 2018 in an outpatient palliative care clinic at a comprehensive cancer center. Nausea and appetite were measured by numerical rating (0-10) and BMI was recorded with data collected at consecutive clinic visits before and after MC use. Results: Eight-three patients aged 65 and over were included in our analysis. Half of patients were age 65-70, while 12% were age 76 or older. More than half were male (58%) and Caucasian (92%). For patients with anorexia or nausea, 58% had previously used cannabis. For nausea, 58% were prescribed ondansetron, 53% were prescribed prochlorperazine or metoclopramide, and 20% were prescribed olanzapine. For anorexia, 24% were prescribed mirtazapine, 6% were prescribed dronabinol, and 1% were prescribed megestrol. The majority of patients used oil (64%), with one-third using vape (33%) and fewer using pill (17%) and powder (5%). Patients primarily used high THC (50%) or equal THC:CBD (45%) formulations initially, with only 7% using high CBD products. The median nausea and anorexia trended towards improvement, though neither was significant (delta nausea = 0.1, p = 0.81) nor anorexia score (delta anorexia = 0.7, p = 0.69). BMI worsened despite MC use (delta BMI 1.9, p < 0.001). Conclusions: In this study of elderly patients with cancer prescribed MC, more than half had previously used cannabis. Two-thirds of patients with anorexia were using MC first-line for appetite stimulation. The majority of patients used oil, with vape next most commonly used, and the vast majority of patients using high THC or equal THC:CBD initially. Use of MC was not associated with a significant improvement in nausea nor anorexia, and BMI significantly decreased despite MC use.

13 Background: Use of medical cannabis (MC) for cancer-related symptoms is growing, yet many prior studies used formulations not available in the United States (US). Clinical data on formulation, dosing and efficacy for formulations available in the US is limited. Furthermore, though interest is growing in the use of MC for elderly patients, data on safety and efficacy in this population is limited. We aimed to assess the clinical characteristics of elderly people using MC, formulations prescribed, and efficacy for cancer-related pain, insomnia and anxiety. Methods: We conducted a retrospective chart review of patients age 65 and older prescribed MC in the year 2018 in an outpatient palliative care clinic at a tertiary comprehensive cancer center. Pain intensity and anxiety were measured by numerical rating (0-10), and insomnia was assessed by self-reported hours of sleep per night, with data collected at consecutive clinic visits before and after MC use. Results: Eighty-three patients aged 65 and over were included in our analysis. Half of patients were age 65-70, while 12% were age 76 or older. Slightly more than half were male (58%), while the majority were Caucasian (92%). For patients with pain, two thirds were already prescribed opioids; 28% were using gabapentin/pregabalin, 22% duloxetine, and 7% nortriptyline/amitriptyline. Two-thirds (65%) reported never using cannabis previously. The most common MC product used was oil (35%), followed by vape (13%), pill (6%) and powder (3%). The most common initial concentrations were equal THC/CBD (41%) and high THC (43%); 8% of patients used high CBD, and 8% used a combination. The median delta pain score did not change with use of MC (delta pain = 0.0, p = 0.72), nor did anxiety (delta anxiety = -0.2, p = 1.00) nor insomnia (delta hours sleep = -0.1, p = 1.000). Conclusions: In this study of elderly patients prescribed MC, the majority of whom were cannabis naïve and already prescribed opioids, most were initially prescribed high THC or 1:1 THC/CBD, with oil and vape the most common formulations used. Use of MC was not associated with changes in pain, anxiety nor insomnia scores.

125 Background: Malignant bowel obstruction (MBO) is a complication of advanced malignancy. For inoperable patients, symptoms are often treated using analgesics, anticholinergics, and anti-emetics. There are, however, few published guidelines or algorithms for the medical management of MBO. Therefore, the objective was to measure the effect of the combination of dexamethasone, octreotide, and metoclopramide (“triple therapy”) in patients with MBO, compared to patients who received none of the three medications (“no drug therapy”). Methods: A retrospective cohort study was done of patients with malignant bowel obstruction admitted between 1/1/2015 to 12/31/2018. The outcome measures were a patient having de-obstruction (defined as toleration of oral intake and resolution of nausea and vomiting), as well as time to de-obstruction. Results: Medical staff identified 34 patients who received triple therapy and 34 patients who received no drug therapy. Patients who received triple therapy were more likely to reach de-obstruction, compared to patients who had no drug therapy (OR: 9.02 [1.43, 56.99], p=0.0194), after adjusting for related covariates (i.e. length of stay and percutaneous endoscopic gastrostomy [PEG] placement). Patients who reached de-obstruction in the triple therapy arm, however, took longer to reach de-obstruction than those in the no drug therapy arm (5.4 days versus 3.4 days, p=0.045). Conclusions: Triple drug therapy with dexamethasone, octreotide, and metoclopramide leads to higher rates of de-obstruction in patients with inoperable MBO, compared to patients who received none of the three drugs, though time to de-obstruction is longer.

e20706 Background: Despite advances in therapy, recent observational data shows that aNSCLC pts with RDP continue to have a poor prognosis. This retrospective, observational study characterizes the demographic, molecular, & treatment profile of pts with RDP. Methods: Adult aNSCLC pts receiving first-line (1L) platinum-based (Pt) therapy between 01/2014 - 12/2018 were identified in the COTA Real-World Evidence database and assigned to RDP (n = 280) & non-RDP (n = 1,212) cohorts based on time to progression during 1L Pt therapy (≤ 12 and > 12 weeks, respectively). Results: Of 1,492 eligible pts, the incidence of RDP was 19%. Mean age (±SD) was 64.6 (10.9) and 66.1 (10.2) in the RDP and non-RDP group, respectively (p = 0.04). Data showed RDP patients had higher percentage of stage IV disease at diagnosis (77 v 72, p < 0.01), higher histologic grade G3/G4 (37 v 29, p = 0.01), and PD-L1 negative (< 1% expression) status (p = 0.01). Table shows molecular profiling across cohorts. No notable difference in treatment patterns across 1L and 2L was observed. Conclusions: This study identifies stage IV disease at diagnosis, higher grade, & PD-L1 negative ( < 1% expression) as potential risk factors for RDP. A better understanding of this poor prognosis cohort may offer an opportunity to better optimize therapies & outcomes. [Table: see text]

e19531 Background: There is uncertainty about the prognostic and predictive significance of t(11;14), long considered a standard risk cytogenetic abnormality in multiple myeloma (MM). This translocation is associated with elevated BCL-2 expression which may explain responses to venetoclax that have been reported previously. In a real-world database derived from EMR data, we sought to characterize real-world treatment patterns and outcomes from this unique MM cohort. Methods: Records for MM patients with t(11;14) diagnosed between 2000 and 2017 were identified in the COTA real-world database. Descriptive statistics were used to summarize the data. Results: 399 MM patients with t(11;14) were identified. Patient characteristics are summarized in the Table. The most frequent first-line treatments were bortezomib + dexamethasone + lenalidomide (134, 33.6%), bortezomib + cyclophosphamide + dexamethasone (69, 17.3%), and bortezomib + dexamethasone (60, 15.0%). Six (1.5%) patients received venetoclax. Response and progression-free survival data are being analyzed and will be presented at the meeting. Median overall survival was 14.3 (95% CI 10.4 – not yet reached) years. Conclusions: Real-world databases are useful in describing treatment patterns and outcomes in narrowly defined cohorts such as MM with t(11;14). The OS result reported here is unexpectedly long and will be fully explored prior to presentation. [Table: see text]

Major hindrances to controlling the current childhood obesity epidemic include access to prevention and/or treatment programs that are affordable, provide minimal barriers for participation, and are available to the general public. Moreover, successful childhood obesity prevention efforts will require coordinated partnerships in multiple sectors such as government, health care, school/afterschool, and the community but very few documented sustainable programs currently exist. Effective, community-based health and wellness programs with a focus on maintaining healthy weight via physical activity and healthy eating have the potential to be a powerful referral resource for pediatricians and other healthcare professionals who have young patients who are overweight/obese. The Miami Dade County Department of Parks, Recreation and Open Spaces in partnership with the University of Miami UHealth Systems have created a "Park Prescription Program (Parks Rx 4Health(TM))" that formally coordinates pediatricians, families, parents, caregivers, and child/adolescents to provide daily obesity-prevention activities. This Parks Rx 4Health(TM) program that we describe here allows UHealth pediatricians to seamlessly refer their overweight and obese patients to Fit2Play(TM), an evidence-based, park-based afterschool health and wellness program. Measurable outcomes that include body mass index, blood pressure, fitness, and nutrition knowledge are being collected at baseline and at 3-and 6-mo after referral to document patient progress. Results are then shared with the referring physician so they can follow up with the patient if necessary. Identifying successful models that integrate primary care, public health, and community-based efforts is important to accelerating progress in preventing childhood obesity. Effective, community-based health and wellness programs with a focus on physical activity and nutrition education could be a powerful referral resource for pediatricians who have obese patients.

Primary vitreoretinal lymphoma (PVRL) is a rare and aggressive lymphoma. Most PVRL are of the diffuse large B-cell lymphoma cell type that arise either in the retina or the vitreous representing ophthalmic variant of primary central nervous system lymphoma (PCNSL-O)

Retinal astrocytic hamartoma ((RAH) also known as retinal astrocytoma or phakoma) is a benign, minimally progressive, neoplastic lesion of the retinal nerve fiber layer caused by proliferation of glial cells.

200 Background: The management of cancer-related pain is challenging in the age of the opioid epidemic. Patients who struggle with substance misuse or chemical coping use these substances to dull emotional and or existential suffering. Buprenorphine is often used for cancer pain due to less reported euphoria when compared to other opioids. Methods: We conducted a retrospective review of patients in an outpatient palliative care clinic at a tertiary comprehensive cancer center who were prescribed buprenorphine for cancer pain. Total oral morphine equivalent (OME), as well as use of adjuvant non-opioid neuropathic pain medication, were assessed prior to transition to buprenorphine. Chemical coping behavior was defined as patients taking opioids for emotional pain in excess of the maximum prescribed amount. Pain scores were reported before and after buprenorphine administration using a Likert pain scale (0-10). Patients were also assessed for the presence of withdrawal symptoms after transition to buprenorphine. Results: Patients were predominantly female (75%), slightly more than half had a psychiatric diagnosis (56%), a majority were on non-opioid neuropathic pain medication (93%), and the majority were identified as chemical copers (75%). For all patients, the mean pain score prior to buprenorphine (pain pre) was 8.3 and the mean pain score on follow up post buprenorphine (pain post) was 6.1, with a reduction in mean pain score (pain change) of -2.0 (p = 0.059). Patients without chemical coping behaviors reported a significantly greater reduction in pain scores compared to patients with chemical coping (pain change -5.0 vs -1.3, p = 0.02). Illicit drug use and psychiatric co-morbidity were not associated with differences in reported pain scores. Only a small number of patients reported withdrawal symptoms (6%) during buprenorphine transition. Conclusions: Rotation to buprenorphine led to a trend of reduction in pain score, though not statistically significant. Patients without chemical coping experienced greater reductions in pain score than those with chemical coping behavior. Illicit drug use and psychiatric co-morbidity were not associated with differences in reported pain.

Purpose To review outcomes with intensity-modulated radiation therapy (IMRT) in the community setting for the treatment of nasopharyngeal and oropharyngeal cancer. Methods and Materials Between April 2003 and April 2007, 69 patients with histologically confirmed cancer of the nasopharynx and oropharynx underwent IMRT in our practice. The primary sites included nasopharynx (11), base of tongue (18), and tonsil (40). The disease stage distribution was as follows: 2 Stage I, 11 Stage II, 16 Stage III, and 40 Stage IV. All were treated with a simultaneous integrated boost IMRT technique. The median prescribed doses were 70 Gy to the planning target volume, 59.4 Gy to the high-risk subclinical volume, and 54 Gy to the low-risk subclinical volume. Forty-five patients (65%) received concurrent chemotherapy. Toxicity was graded according to the Radiation Therapy Oncology Group toxicity criteria. Progression-free and overall survival rates were estimated with the Kaplan-Meier product-limit method. Results Median duration of follow-up was 18 months. The estimated 2-year local control, regional control, distant control, and overall survival rates were 98%, 100%, 98%, and 90%, respectively. The most common acute toxicities were dermatitis (32 Grade 1, 32 Grade 2, 5 Grade 3), mucositis (8 Grade 1, 33 Grade 2, 28 Grade 3), and xerostomia (0 Grade 1, 29 Grade 2, 40 Grade 3). Conclusions Intensity-modulated radiotherapy in the community setting can be accomplished safely and effectively. Systematic internal review systems are recommended for quality control until sufficient experience develops.

BACKGROUND AND OBJECTIVE: To clinically correlate and evaluate the screening and treatment patterns of retinopathy of prematurity by a single physician at a tertiary care center between 2002 and 2012. PATIENTS AND METHODS: A group of 10,924 newborns screened for ROP between 2002 and 2012 were retrospectively analyzed in a consecutive case series. Records of the neonatal intensive care unit (NICU) at Jackson Memorial Hospital/Bascom Palmer Eye Institute were reviewed to identify patients clinically diagnosed with retinopathy of prematurity who underwent treatment. RESULTS: Laser photocoagulation was used to treat 231 of 10,924 (2.1%). Of these 231 patients, 176 (76.2%) were included in the study (55 infants were excluded because they were referred from outside institutions for advanced ROP). Of the 176 treated patients, 89 (50.6%) were male and 87 (49.4%) were female. The average birth weight was 687.3 g. The mean gestational age was 24.8 weeks. The mean time between birth and treatment was 3 months. Of the 176 patients, 31 required re-treatment with laser photocoagulation. The rates of treatment and retreatment significantly declined with the experience of the treating physician ( P < .01). Retinal detachment occurred in two of 176 patients (1.1%). CONCLUSION: Experience is essential in delivering optimal care in a complex disease such as ROP. Between 2002 and 2012, the rate of treatment and retreatment significantly decreased. Possible reasons include improvements in neonatal care, but it is more likely that the rate of treatment and re-treatment is inversely proportional to the ophthalmologist’s experience with and comfort in managing ROP. [ Ophthalmic Surg Lasers Imaging Retina. 2015;46:752–754.]

Purpose: The aim of this study was to document adequacy of training, career plans after residency, use of the in-service examination, and motivation for choice of radiation oncology as a specialty. Methods and Materials: In 2004, the Association of Residents in Radiation Oncology (ARRO) conducted a nationwide survey of all radiation oncology residents in the United States. Results: The survey was returned by 297 residents (response rate, 54%). Of the respondents, 29% were female and 71% male. The most popular career choice was joining an established private practice (38%), followed by a permanent academic career (29%). Residents for whom a permanent academic career was not their first choice were asked whether improvements in certain areas would have led them to be more likely to pursue an academic career. The most commonly chosen factors that would have had a strong or moderate influence included higher salary (81%), choice of geographic location (76%), faculty encouragement (68%), and less time commitment (68%). Of respondents in the first 3 years of training, 78% believed that they had received adequate training to proceed to the next level of training. Of those in their fourth year of training, 75% believed that they had received adequate training to enter practice. Conclusions: Multiple factors affect the educational environment of physicians in training. Data describing concerns unique to resident physicians in radiation oncology are limited. The current survey was designed to explore a variety of issues confronting radiation oncology residents. Training programs and the Residency Review Committee should consider these results when developing new policies to improve the educational experiences of residents in radiation oncology.

Purpose: This report describes a new image-guided radiotherapy (IGRT) technique using megavoltage cone-beam computed tomography (MV-CBCT) to treat paraspinous tumors in the presence of orthopedic hardware. Methods and Materials: A patient with a resected paraspinous high-grade sarcoma was treated to 59.4 Gy with an IMRT plan. Daily MV-CBCT imaging was used to ensure accurate positioning. The displacement between MV-CBCT and planning CT images were determined daily and applied remotely to the treatment couch. The dose–volume histograms of the original and a hypothetical IMRT plan (shifted by the average daily setup errors) were compared to estimate the impact on dosimetry. Results: The mean setup corrections in the lateral, longitudinal, and vertical directions were 3.6 mm (95% CI, 2.6–4.6 mm), 4.1 mm (95% CI, 3.2–5.0 mm), and 1.0 mm (95% CI, 0.6–1.3 mm), respectively. Without corrected positioning, the dose to 0.1 cc of the spinal cord increased by 9.4 Gy, and the doses to 95% of clinical target volumes 1 and 2 were reduced by 4 Gy and 4.8 Gy, respectively. Conclusions: Megavoltage-CBCT provides a new alternative image-guided radiotherapy approach for treatment of paraspinous tumors in the presence of orthopedic hardware by providing 3D anatomic information in the treatment position, with clear imaging of metallic objects and without compromising soft-tissue information. © 2006 Elsevier Inc.

Objectives/Hypothesis: There are many treatments available for advanced oropharyngeal cancer. Organ-sparing protocols reserve surgery for salvage and are thought to provide adequate rehabilitation. Surgical resection with free tissue transfer may also provide adequate functional rehabilitation. The objective was to describe swallowing status and time to decannulation in a series of patients treated with combined-modality therapy that included free flap reconstruction. Study Design: Retrospective chart review. Methods: Patient data were obtained from medical records of 20 patients with stage III or IV oropharyngeal carcinoma, who were consecutively treated with surgical tumor extirpation, free flap reconstruction, and postoperative irradiation at a tertiary academic center from 1985 to 2002. The following variables were identified: patient and tumor characteristics, free flap type, irradiation data, and airway and swallowing status before and after treatment. Results: One patient underwent total laryngopharyngectomy, and the remaining 19 patients underwent tracheotomy at the time of definitive surgery. Free flap reconstructions included 1 ulnar and 15 radial forearm fasciocutaneous flaps and 4 fibula osteocutaneous flaps. Postoperatively, all 19 tracheotomized patients had successful decannulation. Average time to decannulation was 15 days (range, 3-42 d). After surgery and before irradiation, 13 patients initiated oral intake, on average, at 19.5 days (range, 7-28 d); 6 patients required no additional supplementation. By 4 months after surgery, having completed radiation therapy, 10 patients were consuming all nutrition orally; the other 10 patients still required tube-feed supplementation, although 6 of these patients were also eating by mouth. Conclusion: Combined-modality treatment that includes free flap reconstruction for advanced-stage oropharyngeal cancer may provide reasonable functional rehabilitation with respect to postoperative airway and swallowing.

e19030 Background: The peripheral blood absolute lymphocyte-to-monocyte ratio (ALC/AMC) is prognostic of overall survival (OS) in Hodgkin Lymphoma, Diffuse Large B-cell Lymphoma, and several solid tumors. Lymphocyte and monocytes have been suggested to be surrogate biomarkers of immune homeostasis and tumor microenvironment, respectively. We sought to determine if the post-induction therapy ALC/AMC is prognostic in mantle cell lymphoma. Methods: A retrospective review was conducted of 96 consecutive mantle cell lymphoma patients (pts) with available data treated at the John Theurer Cancer Center (n=77) and 4 Regional Cancer Care Associate practices (n=19) by 24 physicians between Aug 2005 and Dec 2015 (90% cases after 2009). Cases were identified via the COTA database which extracts and organizes relevant data from the electronic health records. Peripheral blood counts (to calculate the ALC/AMC) were determined approximately 30 days following completion of initial therapy or immediately prior to stem cell mobilization in those pts undergoing first line transplant. All analyses were performed using the R statistical language. Results: 67 pts had ALC/AMC less than 2 and 29 pts had ALC/AMC greater than or equal to 2. The cohorts (2) had similar median ages (64 vs 68; p=0.18), ethnicities (p=0.38), stage distributions (including 87% vs 79% stage IV disease; p=0.51), elevated beta-2-microglobulin (p=1), elevated LDH (p=1) and MIPI scores (including 19% vs 41% high risk; p=0.13). ALC/AMC was 2, following induction therapy, is associated with improved overall survival in MCL. Novel maintenance programs, including targeting the microenvironment or immune response, might be appropriate among pts with low ratios.

Introduction: The outcome of MCL patients (pts) has improved over the last three decades, although this is debated outside clinical trials (Chandran, Leuk Lymphoma Aug 2012; Smith, Br J Cancer. April 2015). The Mantle Cell International Prognostic Index (MIPI) (Hoster, Blood Jan 2008) is based on 4 variables which predict survival: age (host factor), PS (tumor/host), LDH (tumor burden) and WBC (leukemic phase). The additional value of including co-morbidities into risk stratification has not been fully explored. Methods: Using the COTA database we retrospectively analyzed MCL cases treated at John Theurer Cancer Center and the affiliated practices of Regional Cancer Care Associates from 2004 to 2016. Clinical and treatment characteristics, including calculation of the CCI index (Charlson J Chronic Diseases 1987) were captured via the COTA platform by extracting data from the electronic health records. Results: 490 pts with MCL were evaluated and full longitudinal data from diagnosis is currently reported on 195 subjects. Pts characteristics included: male (66.15%), med age (65, range 34-94), stage IV (87%), LDH (elevated 26%; median 197, range 112-7950), MIPI (low 38%, interm 32%, high 30%), Ki-67 (≥30%: 51%, 86 NA), blastoid variant (16%, 35 NA), SOX-11 positive (87%, 143 NA), 17p abnormalities (p53 del or overexpression/mutation (24%, 88 NA) and b-2 microglobulin (b-2m) > 3 mg/L (52%). Frontline therapy consisted of R-Hyper-CVAD (with or without bortezomib on study) (36%), R-HyperCVAD or R-CHOP followed by high-dose therapy followed by autologous stem cell transplantation (ASCT) (9%), BR alone (8%), BR+ maintenance (8%), R-CHOP alone (4%), Rituximab (3%), R-BAC (3%), BR+ Ibrutinib vs placebo (2%), radiation (2%), R-Lenalidomide (1%), R-CHOP + maintenance (1%), other treatments (10%) while 10% of patients were treated expectantly Seventeen pts underwent ASCT consolidation (15 auto vs 2 allo (del17p/blastoid at presentation). Overall and progression free survival was computed using Kaplan Meier curves and significance tested using log-rank tests. The 5y OS for this entire cohort was 81. Overall, dose-intensive strategies (with or without ASCT) approach was associated with a 23 mo difference in PFS (median 74 mo, range 0-111 mo (intensive) vs median 51 mo, range 2-57 mo for the non-intensive group (p=0.37). The median OS was not reached for either group, with a 5y OS of 88% in intensive vs. 71% non-intensive regimens (p=0.14). Using MIPI as stratification, low/intermediate risk pts had similar outcome in intensive and non-intensive therapy groups (5y OS 88% vs 71%; p=0.13). Pts with high MIPI had a 5 year OS of 80% in the intensive therapy group vs 46% for non-intensive group (p=0.376). The CCI scores for the whole cohort were 0 in 16 pts (8%), 1-3 in 81 pts (41%), and ≥ 4 in 98 pts (50%). Baseline CCI score (pre-treatment) was highly predictive of outcome with a 5y OS of 90% in CCI 0-3 vs 62% in CCI 3+ (p=0.001) (Figure 1). CCI did not predict complete response rate (CR) to induction therapy (CCI 0-3 94% vs CCI 3+ 80%). The median MIPI scores were 5.7 for CCI 0-3 and 6.3 for CCI 3+. Age is a component of both indexes but more heavily weighted in the CCI. Adding CCI to MIPI defined a subset of pts among the high MIPI group who did better than expected with a 5y OS of 88% in combined high MIPI / CCI 0-3 vs 31% for high MIPI / CCI 4+/ (p=0.03). b-2m (cut-off 3mg/L) correlated with 5y OS 93% vs 80%; (p=0.04) as previously reported but did not add to MIPI or CCI risk stratification. Ki-67 (30% cut-off) was marginally associated with OS: 5-y 89% vs 77% (p=0.06). Conclusions: Our cohort is consistent with the improvement of MCL outcome comparing to historical controls and illustrates the importance of comorbidities captured at baseline. A combined CCI-MIPI approach might help identify pts who can still benefit from current therapy approaches in spite of age. Among the high MIPI score group, CCI further refines cohorts with significantly different outcomes. Figure 1 Figure 1. FIgure 2 FIgure 2. Disclosures Goy: Acerta: Consultancy, Membership on an entity's Board of Directors or advisory committees; Genentech: Other: Research funding for clinical trials through institution; Takeda: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; COTA: Membership on an entity's Board of Directors or advisory committees; Janssen/Pharmacyclics: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Research funding for clinical trials through institution, Speakers Bureau; Celgene: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Research funding for clinical trials through institution. Wu:COTA: Employment. Hansen:COTA: Employment. Arunajadai:COTA: Employment. Protomastro:COTA: Employment. Valentinetti:COTA: Employment. Murphy:COTA: Employment. Smith:COTA: Employment. Pe Benito:COTA: Employment. Hasan:COTA: Employment. Suryadevara:COTA: Employment. Feldman:Seattle Genetics: Consultancy, Speakers Bureau; Abbvie: Consultancy, Speakers Bureau; Pharmacyclics: Speakers Bureau; Celgene: Speakers Bureau. Skarbnik:Pharmacyclics: Consultancy; Genentech: Speakers Bureau; Seattle Genetics: Speakers Bureau; Gilead Sciences: Speakers Bureau; Abbvie: Consultancy. Leslie:Celgene: Speakers Bureau; Seattle Genetics: Speakers Bureau. Pecora:COTA: Employment, Equity Ownership. Goldberg:Bristol Myers Squibb, Novartis: Speakers Bureau; Neostem: Equity Ownership; Novartis: Consultancy; COTA Inc: Employment; Pfizer: Honoraria.

Many patients with head-and-neck (HN) cancer have tumor shrinkage and/or weight loss during the course of radiotherapy. We conducted this retrospective study to determine the dosimetric effects of repeat computed tomography (CT) imaging and replanning during the course of intensity-modulated radiotherapy (IMRT) on both normal tissues and target volumes.A retrospective chart review identified 13 patients with HN cancer treated with IMRT who had repeat CT imaging and replanning during the course of radiotherapy. The first IMRT plan for each patient was generated based on the original planning CT scan acquired before the start of treatment. Because of tumor shrinkage or weight loss during radiotherapy, a second CT scan was acquired, and a new plan was generated and used to complete the course of IMRT. CT-CT fusion was used to correct patient positioning differences between the scans. By using a commercial inverse IMRT planning system, a hybrid IMRT plan was generated for each patient by applying the beam configurations of the first IMRT plan (including the intensity profile of each beam) to the anatomy of the second CT scan. The dose-volume histograms of the actual and hybrid IMRT plans were compared using analysis of variance methods for repeated measures.All patients had locally advanced, nonmetastatic Stage III or IV disease, including 6 nasopharynx, 6 oropharynx, and 1 unknown primary site. All patients were treated with concurrent platinum-based chemotherapy. When replanning vs. not replanning was compared, the hybrid IMRT plans (without replanning) demonstrated reduced doses to target volumes and increased doses to critical structures. The doses to 95% (D95) of the planning target volumes of the gross tumor volume (PTVGTV) and the clinical target volume (PTVCTV) were reduced in 92% of patients, by 0.8-6.3 Gy (p=0.02) and 0.2-7.4 Gy (p=0.003), respectively. The maximum dose (Dmax) to the spinal cord increased in all patients (range, 0.2-15.4 Gy; p=0.003) and the brainstem Dmax increased in 85% of patients without replanning (range, 0.6-8.1 Gy; p=0.007).Repeat CT imaging and replanning during the course of IMRT for selected patients with HN cancer is essential to identify dosimetric changes and to ensure adequate doses to target volumes and safe doses to normal tissues. Future prospective studies with larger sample sizes will help to determine criteria for repeat CT imaging and IMRT replanning for HN cancer patients undergoing radiotherapy.

This paper describes the socioeconomic conditions under which the 3 to 5 million migrant and seasonal farmworkers in the United States live. Health consequences resulting from occupational hazards and from poverty, substandard living conditions, migrancy, language and cultural barriers, and impaired access to health care are described. Specific problems include infectious diseases, chemical- and pesticide-related ill- nesses, dermatitis, heat stress, respiratory conditions, musculoskeletal disorders and traumatic injuries, reproductive health problems, dental diseases, cancer, poor child health, inadequate preventive care, and social and mental health problems. By increasing awareness among health care professionals of the plight of migrant and seasonal farmworkers, the authors hope to encourage development of a stronger public health infra- structure and to improve the health status of these individuals.

Between 1985 and 1999, 43 patients with locally-advanced, resectable oropharyngeal cancer were treated with combined surgery and post-operative radiation therapy (RT) at Oregon Health and Science University. Five patients (12 per cent) had Stage III disease and 38 patients (88 per cent) had Stage IV disease. All patients had gross total resections of the primary tumour. Thirty-seven patients had neck dissections for regional disease. RT consisted of a mean tumour-bed dose of 63.0 Gy delivered in 1.8–2.0 Gy fractions over a mean of 49 days. At three- and five-years, the actuarial local control was 96 per cent and the actuarial local/regional control was 80 per cent. The three- and five-year actuarial rates of distant metastases were 41 per cent and 46 per cent, respectively. The actuarial overall survival at three- and five-years was 41 per cent and 34 per cent, respectively. The actuarial rates of progression-free survival were 49 per cent at three-years and 45 per cent at five years. Combined surgery and post-operative RT for advanced-stage oropharyngeal cancer results in excellent local/regional control. This particular group of patients experienced a high-rate of developing distant metastases.

Foot and ankle operations are being performed with greater frequency as outpatient procedures. Although the surgical procedure remains the same whether the operation is done in an inpatient or an oupatient setting, the anesthesia and postoperative analgesia are greatly affected when patients must be discharged soon after their operation. We have evaluated a regional anesthetic technique which blocks the sciatic nerve in the popliteal fossa and the saphenous nerve block at the knee. This was the sole anesthetic technique for both the operation and the immediate postoperative period. This technique appears to have several advantages: 1) Excellent anesthesia during the operation and for about ten hours postoperatively; 2) Use of a proximal calf tourniquet, and 3) Absence of systemic or local complications as might be seen with general, spinal or epidural anesthesia.