134 Background: Understanding and gaining familiarity with the adverse event (AE) and drug-drug interaction (DDI) potential of novel agents can be challenging, cumbersome, and time consuming for clinicians. A comprehensive and secure AE management/DDI mobile application (CT Scholar [Novartis Pharmaceuticals Corp]) was developed to ease the management of clinical trial participants with hormone receptor–positive, human epidermal growth factor receptor 2–negative advanced breast cancer receiving ribociclib. Methods: CT Scholar was designed specifically for the ribociclib clinical program to provide easy access to information on AE and DDI management. The AE management module characterizes and quickly informs drug-specific AE management based on protocol guidelines and supportive care guidance. The DDI module includes a database of > 100 drugs searchable by both trade and generic name. Number of application visits, unique users, and screen views were monitored from Jan to Sept 2016. Results: There were 1055 visits to CT Scholar (117/month) by 145 unique users (28/month), with 5764 screen views (640/month) and 5.5 screens viewed/visit. Among the most-viewed modules were hematologic AE management (1132 views), gastrointestinal AE management (771), and DDI (734). Diarrhea, neutropenia, and nausea were the most viewed submodules (308, 230, and 205 views, respectively). The information on CT Scholar was consistently shown to be accessed more quickly than information obtained from printed clinical protocols. No patients discontinued from the ribociclib studies due to AEs following the introduction of CT Scholar. Conclusions: CT Scholar was widely used by clinicians, and it responded to their needs by providing essential information, especially on prevalent AEs, which could be easily updated and rapidly accessed. Additionally, CT Scholar provides a platform showing how technology can assist clinicians and patients in managing care during a trial, which can inform the development of future supportive materials.

Introduction: Immune Thrombocytopenia (ITP) is a disease of immune-mediated destruction of platelets and suppression of platelet production. ITP has been historically treated with corticosteroids and/or immune globulins as first-line agents. There are several second-line treatments available, should patients fail to respond to initial therapy or relapse after it is tapered. These include eltrombopag, romiplostim, rituximab and splenectomy. This study utilized a national electronic health record (EHR) database to begin to explore the real world treatment patterns of the aforementioned second-line (index) therapies. Methods: Utilizing the Optum EHR database, we identified patients who initiated their first second-line treatment (i.e. the index treatment) with eltrombopag, romiplostim, rituximab or splenectomy from Jan. 1, 2009 to Sep. 30, 2016 for primary or unspecified ITP. Patients included in the analysis had the following characteristics: 18 years or older; previous treatment with corticosteroids and/or immune globulin products; active in the database for at least 6 months prior to and 12 months post initiation of the index treatment. Outcomes that were evaluated after initiation of the index treatment included: (1) Duration of therapy for eltrombopag and romiplostim; (2) Proportion of patients who started a subsequent line of treatment after their index treatment; (3) Treatment free duration between the end of the index treatment and start of a subsequent line of treatment; and (4) Proportion of patients using a first-line medication (corticosteroids and/or immune globulin) during treatment with eltrombopag and romiplostim. Chi-square and t-tests were used for statistical analysis. Results: 2,047 patients met the inclusion criteria and used an index treatment as follows: eltrombopag, N=110 (5.4%); romiplostim, N=189 (9.2%); rituximab, N=1488 (72.7%); splenectomy, N=260 (12.7%). The mean age was 60.8 years (standard deviation [SD]: 17.4), with 52.4% female and mean Charlson comorbidity score of 2.1 (SD: 2.1). Treatment duration was 481 days for eltrombopag versus 346 days for romiplostim (p=0.033). The proportion of patients who started a subsequent line of treatment after their index treatment ranged from 41% for rituximab to 49% for splenectomy (p=0.071). Treatment free duration between the end of the index treatment and start of a subsequent treatment ranged from a mean of 248 days for romiplostim to 575 days for splenectomy (p<0.001). The proportion of patients who did not use first-line medications during treatment with eltrombopag and romiplostim were similar (24% vs. 17%, p=0.157). See Table 1 below for details. Conclusions: In this dataset, rituximab was the predominant second-line treatment. Patients receiving eltrombopag had a greater treatment duration compared to romiplostim. As expected, a greater treatment free duration was observed with splenectomy and rituximab, though mean treatment free duration after treatment with romiplostim and eltrombopag was surprisingly long (248-270 days). Despite the longer duration off treatment following splenectomy and rituximab, a similar percentage of patients across all index treatments ultimately required a subsequent line of therapy. Further research is required to better understand the differences in real world treatment patterns among these cohorts. Disclosures Said: Novartis: Employment. Lal:Optum: Employment. Nezami:Novartis Pharmaceuticals: Employment. Andrade:Optum: Employment. Graves:Novartis: Employment. Roy:Novartis: Employment. Cuker:Spark Therapeutics: Research Funding; Kedrion: Membership on an entity's Board of Directors or advisory committees; Synergy: Consultancy; Genzyme: Consultancy.

INTRODUCTION: Immune Thrombocytopenia (ITP) is a disease of immune-mediated destruction of platelets and suppression of platelet production. ITP has been historically treated with corticosteroids and/or immune globulins as first-line agents. There are several second-line treatments available, should patients fail to respond to initial treatment or relapse after it is tapered. This study utilizes a national electronic health record (EHR) database to understand clinical outcomes with use of second-line treatments including: thrombopoietin receptor agonists (specifically, eltrombopag and romiplostim), rituximab, and splenectomy. METHODS: Utilizing the Optum EHR database, we identified patients who initiated a second-line treatment from Jan. 1, 2009 to Sep. 30, 2016 for primary or unspecified ITP. Additionally, patients included in the analysis had the following characteristics: 18 years or older; previous treatment with corticosteroids or immune globulin products; and active in the database for at least 6 months prior to and 12 months post initiation of a second-line treatment. Evaluated outcomes included: (1) platelet counts; (2) bleeding related episodes (BREs); and (3) thrombotic events (TEs), including stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis, and pulmonary embolism. All outcomes were evaluated over the 12-month period following the initiation of second-line treatment. BREs and TEs were defined using ICD codes, collapsed to first three characters, indicating a form of bleeding or thrombotic events. Chi-square test and t-test were used to evaluate differences among the four treatment cohorts (eltrombopag, romiplostim, rituximab, and splenectomy). RESULTS: 2,047 patients met the inclusion criteria and used the second-line treatments as follows: eltrombopag, N=110 (5.4%); romiplostim, N=189 (9.2%); rituximab, N=1,488 (72.7%); splenectomy, N=260 (12.7%). The mean age was 60.8 years (standard deviation [SD]: 17.4), with 52.4% female and mean Charlson comorbidity score of 2.1 (SD: 2.1). Compared with platelet counts at the initiation of second-line treatments (collected within +/- 14 days of initiation of second-line treatments), mean platelet counts achieved during the 12 months post initiation of a second-line treatment increased in all treatment cohorts, ranging from an increase of 63,000/μL for rituximab to an increase of 157,000/μL for splenectomy. Mean platelet counts achieved during the 12 months post initiation of a second-line treatment differed significantly across treatment cohorts, ranging from 102,000/μL for eltrombopag to 240,000/μL for splenectomy (p<0.001) (Table 1). The proportion of patients who experienced BREs differed across the treatment cohorts, ranging from 25.5% for eltrombopag to 36.5% for romiplostim (p=0.038) (Table 2). Most common BREs observed included acute posthemorrhagic anemia, GI hemorrhage, hematuria, hemoptysis and hemorrhage, and rectal hemorrhage. TEs were observed in all treatment cohorts ranging from 11.6% for eltrombopag to 15.7% for splenectomy (p=0.744). (Table 2). CONCLUSIONS: This retrospective real world evidence study compares mean platelet response and burden of both BREs and TEs in ITP patients treated with different second-line treatments. Although there were significant differences in mean platelet counts achieved with the second-line treatments, TEs were observed with similar incidence across all treatments. Patients who received splenectomy had the highest mean platelet counts and also the highest proportion of patients who experienced TEs, though differences in TEs did not reach statistical significance. Incidence proportion of patients with BREs was lower in patients treated with eltrombopag than in patients treated with other second-line treatments. Such differences in outcomes may be useful when selecting treatment in the second-line setting. Disclosures Said: Novartis Pharmaceuticals Corporation: Employment. Lal:Optum: Employment. Andrade:Optum: Employment. Nezami:Novartis Pharmaceuticals: Employment. Graves:Novartis: Employment. Roy:Novartis: Employment. Cuker:Kedrion: Membership on an entity's Board of Directors or advisory committees; Synergy: Consultancy; Genzyme: Consultancy; Spark Therapeutics: Research Funding.

Introduction: Chronic immune thrombocytopenia (cITP) is an autoimmune disorder defined by low platelet count (<100 x 109/L) lasting ≥12 months in the absence of other causes of thrombocytopenia. Splenectomy is an option for patients with cITP who fail to respond to oral corticosteroids and/or intravenous immunoglobulin or relapse after treatment is discontinued. A systematic literature review (SLR) conducted in 2004 (Kojouri et al) identified articles describing outcomes associated with splenectomy in patients with cITP. The objective of this study was to update this SLR with a focus on contemporary data on long-term outcomes (≥12 months of follow-up). Methods: MEDLINE, Embase, Cochrane CENTRAL and recent congresses were searched in June 2018. Results were screened against predefined criteria by two independent researchers. Included studies assessed patients with cITP (N≥15) who underwent splenectomy; studies of patients with secondary ITP, newly diagnosed ITP, and/or persistent ITP were excluded unless separate outcomes were reported for cITP subgroups. Outcomes of interest were clinical efficacy (response and relapse rates), safety (rates of complications), mortality, and health-related quality of life (HRQoL). Prospective or retrospective clinical studies or real-world study types were included. English-language studies published during or after 2000 were included, with no geographic restrictions. Results: The literature search identified 3140 records for title-abstract screening. Of these, 159 full-text studies were evaluated and 108 were included in the analysis. Most studies (93) were retrospective. Fifteen prospective studies (9 interventional, 6 observational) but no randomized controlled trials were identified. Nine studies were comparative (all retrospective): splenectomy vs rituximab (3), splenectomy vs rituximab vs romiplostim (1), and splenectomy vs non-splenectomy (5). Reports of the long-term efficacy of splenectomy varied widely, with multiple definitions of response and remission across the heterogeneous study types. Among 40 studies, the mean complete response (CR) rate within 12 months of surgery was 77% (median: 81%; range: 26-97%). Relapse rates varied widely, ranging from 0-94% among 47 studies with ≥12 months of follow up. Five of 7 studies reporting remission rates at multiple time points at ≥1 year noted a decrease in clinical remission over time. Mortality generally increased with length of follow up: in studies with ≤1 month of follow-up (28 studies) the mean mortality rate was 1% (range: 0-5%), while in studies with 1-5 years of follow-up (20 studies) and ≥5 years of follow-up (15 studies), the mean mortality rate was 2% (range: 0-17%) and 11% (range: 0-30%), respectively. Four studies reported that long-term response rates were higher with splenectomy than rituximab; all other efficacy comparisons were inconclusive. Although 11 of 15 prospective studies and 61 of 93 retrospective studies reported some safety information, there were very limited data on the long-term safety of splenectomy. Commonly reported complications were bleeding (mean: 14%; median: 12% range: 0-50%; 22 studies), infections (mean: 8%; median: 4% range: 0-33%; 38 studies), venous thromboembolism (VTE) (mean: 5%; median: 3% range: 0-21%; 27 studies) and sepsis/septic shock (mean: 2%; median: 0%; range: 0-11%; 18 studies). Rates of postoperative complications (≤30 days) ranged from 3-50% (mean: 13%; 31 studies), and 2 studies suggested that older age may be associated with higher rates of postoperative complications. HRQoL data were rarely reported (3 studies). Rates of remission, relapse, and infections for studies reporting at least 1 of these outcomes at 1 or more discrete time points are shown in Figure 1. Conclusions: Although more than 100 studies reported long-term outcomes for patients with cITP treated with splenectomy, available evidence on the durability of response and long term safety are limited. In general, most measures of efficacy declined over time, while complications (infections, bleeding, VTE) and mortality increased over time. The extent to which the outcomes for splenectomy differ from currently available treatments is unclear. Additional data are needed to understand the long-term benefits and risks of splenectomy in patients with cITP. Disclosures Nellesen: Analysis Group, Inc.: Employment; Novartis Pharmaceuticals Corporation: Consultancy. Said:Novartis: Employment. Shak:Analysis Group, Inc.: Employment; Novartis Pharmaceuticals Corporation: Consultancy. Patton:Novartis Pharmaceuticals Corporation: Consultancy; Analysis Group, Inc.: Employment. Lucas:Novartis Pharmaceuticals Corporation: Consultancy; Analysis Group, Inc.: Employment. Graves:Novartis: Employment. Nezami:Novartis Pharmaceuticals: Employment. Cuker:Kedrion: Membership on an entity's Board of Directors or advisory committees; Spark Therapeutics: Research Funding; Synergy: Consultancy; Genzyme: Consultancy.