Citations

Background and Purpose— The American Heart Association and the American Stroke Association guidelines for early management of patients with ischemic stroke offer guidance to physicians involved in acute stroke care and clarify endovascular treatment indications. The purpose of this study was to assess concordance of physicians’ endovascular treatment decision-making with current American Heart Association and the American Stroke Association stroke treatment guidelines using a survey-approach and to explore how decision-making in the absence of guideline recommendations is approached. Methods— In an international cross-sectional survey (UNMASK-EVT), physicians were randomly assigned 10 of 22 case scenarios (8 constructed with level 1A and 11 with level 2B evidence for endovascular treatment and 3 scenarios without guideline coverage) and asked to declare their treatment approach (1) under their current local resources and (2) assuming there were no external constraints. The proportion of physicians offering endovascular therapy (EVT) was calculated. Subgroup analysis was performed for different specialties, geographic regions, with regard to physicians’ age, endovascular, and general stroke treatment experience. Results— When facing level 1A evidence, participants decided in favor of EVT in 86.8% under current local resources and in 90.6% under assumed ideal conditions, that is, 9.4% decided against EVT even under assumed ideal conditions. In case scenarios with level 2B evidence, 66.3% decided to proceed with EVT under current local resources and 69.7% under assumed ideal conditions. Conclusions— There is potential for improving thinking around the decision to offer endovascular treatment, since physicians did not offer EVT even under assumed ideal conditions in 9.4% despite facing level 1A evidence. A majority of physicians would offer EVT even for level 2B evidence cases.

Abstract Background: American Indians and Alaska Natives (AI/AN) currently represent approximately 2% of the US population. However, population-based cancer studies are generally limited. Utilizing the Surveillance, Epidemiology and End Results Program (SEER), we investigated the incidence, major subtype distribution, and survival of non-Hodgkin lymphomas (NHL) in the AI/NA population. Methods: We queried the SEER 2000-2012 database for AI/AN patients with NHL using ICD-O-3 codes 9590-9823. We calculated their incidence rate (case/1,000,000) and trend as well as risk ratios compared to other races, age-adjusted to the US 2000 standard population. Patient level data were extracted to perform summary statistics and calculate overall survival (OS) using STATA/IC 12.1. Results: We identified 1,135 AI/NA patients with NHL. The average age was 59.6 years (range, 20-85) and 53% were males. The distribution of the major NHL subtypes resembles that seen among the rest of the population and is as follows: diffuse large B-cell (DLBCL; 28%), marginal zone (MZL; 19%), follicular (FL; 13%), small lymphocytic/chronic lymphocytic leukemia (SLL/CLL; 10%), NHL not-otherwise specified (NOS, 4%), mantle cell (MCL; 2%), anaplastic large cell (ALCL; 2%), lymphoplasmacytic (LPL; 1%), and peripheral T-cell (PTCL; 1%). The overall incidence of NHL is 26.4 and did not significantly change over the 12-year study period (Annual Percent Change 0.0, 95% CI [-0.3, 0.3]). Compared to other races, AI/NA have lower incidence of NHL including most of the major subtypes (Table). The median OS for all types of NHL combined is 4.16 years. AI/AN OS is statistically inferior compared to Whites(Figure). Conclusions: The NHLincidence in AI/NA is lowest among the major race groups and has remained stable in the past 12 years. However their OS is inferior when compared to Whites. The distribution of major NHL subtypes and OS are similar to the rest of the population. Figure 1. Figure 1. Disclosures No relevant conflicts of interest to declare.

Abstract Introduction Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an extremely rare hematologic malignancy that was grouped with myeloid malignancies in the 2008 WHO classification. The current available literature is limited to small case series and single-institution experiences. This population study using the Surveillance, Epidemiology, and End Results Program (SEER) and the National Cancer Database (NCDB) is the largest to explore epidemiologic and outcome data in BPDCN. SEER collects cancer incidence and survival data from cancer registries covering approximately 26% of the US population. NCDB is a nationwide, facility-based, comprehensive, clinical-surveillance resource for oncology data coming from more than 1500 Commission of Cancer (CoC)-accredited programs. Methods Using data from the SEER and NCDB registries, this study respectively identified 379 (years 2000-2013) and 216 (years 2004-2012) adult patients over the age of 20 diagnosed with BPDCN (ICDO-3 coding 9727). We used the SEER registry to estimate incidence rates, ratios and trends and the NCDB registry to study clinical characteristics, survival and outcomes. The JMP statistical software and the SEER*STAT 8.3.2 were used to conduct statistical analysis. We analyzed survival using the Kaplan-Meier method and used log-rank tests to compare survival distributions. The prognostic effect of pertinent clinical variables was studied using multivariate Cox proportional hazards models. All tests of statistical significance were two-sided and P < 0.05 was considered significant. Results The median age at diagnosis for BPDCN was 52 years (20-93). The incidence of BPDCN was found to be 0.45/1,000,000 age-adjusted to the 2000 US standard population with similar trends over the years. No racial disparities were identified. The male:female ratio was 2 (P<0.00001). The bone marrow was the most common primary site of involvement (42%) followed by the skin (20%) and the heart, mediastinum and pleura (13%). The median overall survival for the entire cohort was found to be 23 months (95% CI; 17-34). Survival rates at 1 year, 2 years and 5 years were found to be 66%, 49% and 38% respectively. 20 patients (10%) underwent a hematopoietic stem cell transplantation (HSCT) as part of their initial therapy and the median overall survival for these patients was not reached compared to a median survival of 19 months for patients who did not undergo upfront HSCT (95% CI; 15-30, P=0.006). A univariate and multivariate analysis was performed including age, race, sex, HSCT, insurance status, income, treatment facility (academic versus non-academic) and disease primary site. Young age and HSCT were predictors of better outcomes. 60 years and older was found to be the age cut-off associated with significantly inferior survival based on receiver operating characteristic (ROC) curve analysis. Conclusion BPDCN is a rare hematologic malignancy associated with a poor median survival of less than two years. Males are twice more likely to be affected than females. The bone marrow is the primary site of involvement in most cases. Younger age (<60 years) and HSCT were predictors of significantly better outcomes. Kaplan-Meier survival curve for the entire cohort Kaplan-Meier survival curves in patients Age <60 versus > 60 Kaplan-Meier survival curves in patients who underwent HSCT versus those who did not Figure 1 Figure 1. Figure 2 Figure 2. Figure 3 Figure 3. Disclosures Al-Kali: Celgene: Research Funding; Onconova Therapeutics, Inc.: Research Funding.

INTRODUCTION: Treatment options for patients with moderate-severe ulcerative colitis (UC) are increasing. The appropriateness of their use in mild-moderate UC is less clear, as is the role of surgical intervention. Our aim was to define the appropriateness of therapeutic options in a range of clinical scenarios in mild-moderate UC METHODS: We applied the RAND/UCLA Appropriateness Method to rate appropriateness of treatment choices in patients with mild-moderate UC; severe disease (likely to need hospitalization or surgery within 2 weeks) was excluded. A literature review was presented to the BRIDGe group, a panel of 13 IBD specialists, as well as 1 external IBD expert and 2 IBD surgical experts. 12 chapters were constructed including failed 5-ASA, steroid or thiopurine therapy, and primary/secondary non-response to biologics or tofacitinib. Scenarios were also divided by disease extent (proctitis vs more extensive) and activity (mild vs moderate) creating 260 scenarios in all. Panellists used a modified Delphi method to anonymously rate the appropriateness of a therapy on a 1-9 scale (1-3 not appropriate, 4-6 uncertain, 7-9 appropriate). Disagreement was assessed using a validated index (DI) and was defined as a DI > 1. The panellists then convened in person to discuss areas of disagreement, followed by a second round of rating. Scenarios rated a median of 7 or higher were deemed appropriate and those rated 3 or lower were deemed inappropriate. Scenarios rated 4-6 were considered uncertain, as were any with a DI > 1 RESULTS: Appropriateness was agreed in 131 and inappropriateness in 47 scenarios. Greatest disagreement was around the use of thiopurines, but no scenario had a DI > 1. There were 82 scenarios were rated uncertain. Biologics and tofacitinib were more likely to be rated appropriate for treatment resistance and greater disease activity; disease extent less likely to influence decision making. Surgery was felt to be appropriate in 8 scenarios, generally in more extensive and active disease that had failed to respond to medical therapy. Surgery was felt to be inappropriate in 16 scenarios and was uncertain in 24 CONCLUSION: Although there was little disagreement amongst an expert panel of IBD physicians and surgeons, there remained uncertainty about the use of medical and surgical therapy in nearly one third of situations in patients with mild-moderate UC. These results could be used to guide the design of clinical trials for these patient groups in order to guide best practice.

Abstract Background : Sex is recognized as a significant determinant of outcome among glioblastoma patients, but the relative prognostic importance of glioblastoma features has not been thoroughly explored for sex differences. Methods : Combining multi-modal MR images, biomathematical models, and patient clinical information, this investigation assesses which pretreatment variables have a sex-specific impact on the survival of glioblastoma patients (299 males and 195 females). Results : Among males, tumor (T1Gd) radius was a predictor of overall survival (HR=1.027, p=0.044). Among females, higher tumor cell net invasion rate was a significant detriment to overall survival (HR=1.011, p<0.001). Female extreme survivors had significantly smaller tumors (T1Gd) (p=0.010 t-test), but tumor size was not correlated with female overall survival (p=0.955 CPH). Both male and female extreme survivors had significantly lower tumor cell net proliferation rates than other patients (M p=0.004, F p=0.001, t-test). Additionally, extent of resection, tumor laterality, and IDH1 mutation status were also found to have sex-specific effects on overall survival. Conclusion : Despite similar distributions of the MR imaging parameters between males and females, there was a sex-specific difference in how these parameters related to outcomes.

Background and purpose Differences in the treatment practice of female and male physicians have been shown in several medical subspecialties. It is currently not known whether this also applies to endovascular stroke treatment. The purpose of this study was to explore whether there are differences in endovascular treatment decisions made by female and male stroke physicians and neurointerventionalists. Methods In an international survey, stroke physicians and neurointerventionalists were randomly assigned 10 case scenarios and asked how they would treat the patient: (A) assuming there were no external constraints and (B) given their local working conditions. Descriptive statistics were used to describe baseline demographics, and the adjusted OR for physician gender as a predictor of endovascular treatment decision was calculated using logistic regression. Results 607 physicians (97 women, 508 men, 2 who did not wish to declare) participated in this survey. Physician gender was neither a significant predictor for endovascular treatment decision under assumed ideal conditions (endovascular therapy was favored by 77.0% of female and 79.3% of male physicians, adjusted OR 1.03, P=0.806) nor under current local resources (endovascular therapy was favored by 69.1% of female and 76.9% of male physicians, adjusted OR 1.03, P=0.814). Conclusion Endovascular therapy decision making between male and female physicians did not differ under assumed ideal conditions or under current local resources.

Background and Purpose— Little is known about the real-life factors that clinicians use in selection of patients that would receive endovascular treatment (EVT) in the real world. We sought to determine patient, practitioner, and health system factors associated with therapeutic decisions around endovascular treatment. Methods— We conducted a multinational cross-sectional web-based study comprising of 607 clinicians and interventionalists from 38 countries who are directly involved in acute stroke care. Participants were randomly allocated to 10 from a pool of 22 acute stroke case scenarios. Each case was classified as either Class I, Class II, or unknown evidence according to the current guidelines. We used logistic regression analysis applying weight of evidence approach. Main outcome measures were multilevel factors associated with EVT, adherence to current EVT guidelines, and practice gaps between current and ideal practice settings. Results— Of the 1330 invited participants, 607 (45.6%) participants completed the study (53.7% neurologists, 28.5% neurointerventional radiologists, 17.8% other clinicians). The weighed evidence approach revealed that National Institutes of Health Stroke Scale (34.9%), level of evidence (30.2%), ASPECTS (Alberta Stroke Program Early CT Score) or ischemic core volume (22.4%), patient’s age (21.6%), and clinicians’ experience in EVT use (19.3%) are the most important factors for EVT decision. Of 2208 responses that met Class I evidence for EVT, 1917 (86.8%) were in favor of EVT. In case scenarios with no available guidelines, 1070 of 1380 (77.5%) responses favored EVT. Comparison between current and ideal practice settings revealed a small practice gap (941 of 6070 responses, 15.5%). Conclusions— In this large multinational survey, stroke severity, guideline-based level of evidence, baseline brain imaging, patients’ age and physicians’ experience were the most relevant factors for EVT decision-making. The high agreement between responses and Class I guideline recommendations and high EVT use even when guidelines were not available reflect the real-world acceptance of EVT as standard of care in patients with disabling acute ischemic stroke.