ALBERT

Description: Background Immune Thrombocytopenia (ITP) is an autoimmune disorder caused by immunologic destruction of otherwise normal platelets, most commonly occurring in response to an unknown stimulus. ITP is diagnosed after excluding other possible causes of disease, and symptoms can present across varying severities and treatments. The poor understanding of the symptoms and cause can result in both misdiagnosis and complex treatment patterns, which may significantly affect health related quality of life (HRQoL) in this patient population. There is currently no disease specific prospective tool in routine clinical practice to capture HRQoL in the adult ITP population. To help assess the impact of this condition on HRQOL, the ITP Life Quality Index (ILQI), a 10-item patient-reported outcome (PRO) measure was developed as a tool for clinical practice to aid discussions between patients and physicians about disease experience so to inform patient-centric treatment decisions. The ILQI was originally developed by clinical experts in the field of ITP and content validity was confirmed by conducting qualitative interviews with 15 adult patients with ITP. The ILQI was then cognitively debriefed patients with ITP and items refined following qualitative analysis and additional clinical input. The ILQI was included in the ITP World Impact Survey (I-WISh), a global observational study which collected data on the impact of ITP on patients' HRQoL. This large study provided an opportunity to assess the psychometric properties of the ILQI and confirm the scoring cut-offs. Methods The I-WISh survey resulted in data from 1,507 patients with ITP across 12 countries worldwide and was used to assess the structure, reliability and validity of the ILQI. The structure of the ILQI, how the items fit into total scores and subscales, was assessed by splitting the data into two datasets. One dataset was used for identifying the structure using exploratory factor analysis and one was used for checking the structure using confirmatory factor analysis. Validity, the ability of the ILQI to measure the correct construct, was assessed through known groups and convergent validity methods. Reliability, the consistency of the ILQI items and their ability to create reproducible scores, was assessed via internal consistency methods. To understand whether each ILQI item measured ITP in a similar way across countries, differential item functioning (DIF) was assessed using Cochran-Mantel-Haenszel test and Logistic regression. Finally, existing score cut-offs (20-"significantly impaired QoL"; 30 - "severely impaired QoL") were assessed using receiver operating characteristic (ROC) curves and a simulation study was conducted to develop rules for missing data. Results Results indicated that the ILQI has an essentially unidimensional structure, supporting the creation of a total score including all 10 items. The ILQI items work together to create a reproducible total score, usable for making judgements on an individual patient basis (Omega total and Cronbach's alpha coefficients ≥ 0.90). Known groups methods showed that ILQI monotonically increased with ITP severity (linear trends p's

Description: Background: Polycythemia vera (PV), essential thrombocythemia (ET) and myelofibrosis (MF) are clonal hematological disorders collectively named as myeloproliferative neoplasms (MPN). Discovery of JAK2 mutation in 2005, altered WHO classification for MPN diagnosis in 2008 and availability of new treatment of MPN may have substantial effect on epidemiology of MPN. Published data on epidemiology of MPN after the discovery of JAK2 mutation and the introduction of 2008 WHO classifications for MPN, in particular on the prevalence of MPN, are scarce. We aimed to study the epidemiology of MPN in Norway and to explore the impact of JAK-2 mutation and new guidelines on the incidence of MPN using data from the Norwegian cancer registry. Method: We identified 2344 persons diagnosed with MPN from the Norwegian Cancer Registry diagnosed between 1995 and 2012. Registration of cancer in the Norwegian Cancer Registry is mandatory according to the law. We report age-adjusted incidence, prevalence and relative survival of MPN. Age adjusted incidence was reported for 2 years periods from 1995 to 2012. The prevalence was calculated according to the Norwegian population per 31.12.2011. Results: A total of 945 cases of PV was identified with a median age at diagnosis of 70 years; 471 males (50%) and 474 females (50%). The overall age-adjusted incidence rate both genders was 0.4/10⁵ in 1995-1997, 0.5/10⁵ in 1998-2000, 0.7/10⁵ in 2001-2003, 0.8/10⁵ in 2004-2007, 2008-2009 and 0.7/10⁵ in 2010-12. We identified a total of 762 cases of ET with a median age at diagnosis of 65 years, 297 males (39%) and 465 females (61%). The overall age adjusted incidence rate both genders being 0.3/10⁵ in 1995-1997 and 1998-2000, 0.5/10⁵ in 2001-2003 and 2004-2006, 0.9/10⁵ in 2007-2009 and 2010-2012. A total of 418 cases of MF was identified with a median age at diagnosis of 71 years; 243 males (58%) and 175 females (42%). Age adjusted incidence rates of both genders were 0.2/10⁵ from 1995-2006, 0.3/10⁵ in 2007-2009 and 0.5/10⁵ in 2010-2012. There were a total of 219 persons with unclassified MPN both genders,119 males (54%) and 100 females (46%) and age adjusted incidence rate varied from 0.1-0.2 to 0.1/10⁵ 1995-2012. Per 31.12.2011 the prevalence of PV, ET and MF was 9.2, 8.6 and 3.0 per 10⁵ inhabitants respectively. The survival curves for males and females for the three conditions are shown in the figure. Conclusions: This population-based study shows that the incidence of ET and MF almost doubled during the years 2007-2012 as compared to 1995-2006 as shown in the table. This increment in the incidence may possibly be related to improved diagnostics including the JAK2 mutation and the introduction of 2008 WHO-guidelines for MPN. Surprisingly, the discovery of JAK2 does not seem to have had impact on the incidence of PV as indicated by steady incidence rates since 2001. The relative survival was only slightly reduced for PV and ET, but substantially reduced for MF. Only 50% of patients with MF survive for more than 5 years. Table Incidence of MPN per 105 inhabitants during the period 1995 to 2012 in Norway 1995-97 1998-2000 2001-03 2004-06 2007-09 2010-12 PV 0.4 0.5 0.7 0.8 0.8 0.7 ET 0.3 0.3 0.5 0.5 0.9 0.9 MF 0.2 0.2 0.2 0.2 0.3 0.5 Figure showing the relative survival of PV, ET and MF Figure. showing the relative survival of PV, ET and MF Disclosures Roaldsnes: Novartis Norge AS: Research Funding. Ghanima:Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding.

Description: Background α-interferon (IFN) is an effective treatment in polycythemia vera (PV) and essential thrombocythemia (ET). IFN can induce cytogenetic remissions in PV pts. However, 21–25 % of pts discontinue therapy due to side effects. Pegylated IFN has been suggested to have less side effects. Study design and patients: The Nordic MPD Study Group performed an open label phase II feasibility study of PegIntron® treatment in 42 MPD pts, 20 females and 22 males, median age 54 yr (range 29–77). Inclusion criteria were a platelet count of 〉 400x109/L in pts with symptoms or previous thrombosis (n=26) or 〉1000x109/L in asymptomatic pts without previous thrombosis (n=16). 22 pts had PV, 20 ET. 15 pts had previously received cytoreductive therapy; anagrelide (7), hydroxyurea (6), busulfan (1), P32 (1). Previous IFN therapy was not allowed. Median time from diagnosis to study start was 0.74 ys (0.01–30.2). Initial dose was 0.5 μg/kg once weekly. The treatment goal was a platelet count 〈 400x109/L in symptomatic patients and 〈 600 in asymptomatic patients (CR). If CR was not achieved within 12 weeks PegIntron was increased to 1 μg/kg/week. The dose was subsequently decreased to the lowest dose maintaining CR. Pts QoL was investigated using QLQ-C30 and HAD forms. Neutrophil PRV-1 mRNA expression was analyzed by quantitative RT-PCR prior to and after therapy. Results: Response to treatment was evaluated at 1,2,3,6,9 and 12 months. At 6 months, 29/42 pts (69 %) had achieved CR, after a median time of 83 days. The median dose at time of CR was 0.5 μg/kg/week. CR rate was not related to diagnosis or gender. 4/42 pts (10%) discontinued therapy early due to side effects, another 9 pts were taken off study at 6 months due to side effects or insufficient response. Only 4/14 responding PV pts required phlebotomy therapy, compared to 20/22 before PegIntron. 20/29 patients with CR at 6 months maintained CR at 12 months, whereas 9 pts had gone off study due to side effects. The mean platelet count was 875x109/L prior to therapy, and 512, 448, 362x109/L at 3,6 and 12 months in pts on therapy. No thrombotic or bleeding complications were observed. Side effects were common, the majority WHO grades I-II; fatigue (76 %), injection site reaction (64 %), flu-like symptoms (64 %), headache (56 %), muscle pain (51 %), depression (35 %), insomnia (24 %), hair loss (20 %). Mild elevations were noted of serum ALAT, creatinine and TSH in 19, 3 and 3 pts respectively. QoL data will be presented. Changes in PRV-1 expression were determined in 13 PRV-1 positive pts (9 PV, 4 ET) after 6 months and in 2 PV pts after 24 months. PRV-1 expression was normalized in 3/4 ET pts at 6 months and in 1/2 PV pts after 24 months treatment. Conclusion: We found PegIntron to be effective in reducing the platelet count in 29/42 pts (69 %) and 20 pts (48 %) remained in CR at 1 year. 22 pts (52 %) had discontinued therapy at 12 months, mainly due to side effects, a higher rate than in previous trials. Although analyzed in a small number of pts, reversal of PRV-1 positivity nonetheless suggests that PRV-1 quantification may be useful as a molecular marker of therapeutic efficacy.

Description: Management of immune thrombocytopenia (ITP) during pregnancy can be challenging since treatment choices are limited. Thrombopoietin receptor agonists (Tpo-RAs), which likely cross the placenta, are not recommended during pregnancy. To better assess safety and efficacy of off-label use of Tpo-RA during pregnancy, a multicenter observational and retrospective study was set up. Results from 15 pregnant women with ITP (17 pregnancies and 18 neonates) treated with either eltrombopag (N=8) or romiplostim (n=7) during pregnancy, including 2 patients with secondary ITP, were analyzed. Median time of Tpo-RA exposure during pregnancy was 4.4 weeks [range: 1-39 weeks]; the indication for starting Tpo-RA was preparation for delivery in 10/17 (58%) pregnancies whereas 4 had chronic refractory symptomatic ITP and 3 were on eltrombopag when the pregnancy started. Regarding safety, neither thromboembolic events among mothers nor Tpo-RA-related fetal or neonatal complications were observed except for one case of neonatal thrombocytosis. Response to Tpo-RA was achieved in 77% of cases, mostly in combination (70% of responders) with concomitant ITP therapy. Based on these preliminary findings, temporary off-label use of a Tpo-RA for severe and/or refractory ITP during pregnancy seems safe for both mother and neonate and likely to be helpful especially prior to delivery.

Description: Abstract LBA-1 Background: Following acute deep vein thrombosis (DVT) of the lower limb, approximately 1 in 4 patients treated with anticoagulation (AC) and elastic compression stockings (ECS) in accordance with current guidelines, are still at risk for developing a chronically reduced functional outcome, i.e., the post-thrombotic syndrome (PTS). Additional treatment with catheter-directed thrombolysis (CDT) enhances clot removal and is suggested to favor venous competence and patency, thereby reducing the risk for PTS. This interventional therapy is expensive, associated with life-threatening bleeding, and converts an outpatient disease to an inpatient disease. However, it has become standard care in some centers despite a complete lack of evidence from randomized, controlled trials (RCT). The CaVenT study, representing the first RCT in this area, aimed to evaluate whether additional CDT with alteplase improved the functional outcome by reducing PTS development following acute iliofemoral DVT. Methods: The CaVenT study was an open, multicenter RCT that recruited patients from 20 hospitals in the Norwegian south-eastern health region. Patients of age 18–75 years with a first-time objectively verified acute iliofemoral DVT above mid-thigh level and symptoms for up to 21 days were eligible for recruitment. Study patients were randomly assigned with a 1:1 ratio to standard (control) treatment with AC and ECS grade II (30 mmHg) or to CDT with alteplase in addition to standard treatment. The present report concerns the primary clinical end-point; the frequency of PTS after 24 months follow-up. The study was designed to detect a reduction in PTS from 25% to 10% with a 5% significance level and with 80% power. Follow-up visits were conducted at 6 months ± 2 weeks and 24 months ± 4 weeks and included evaluation of PTS by the Villalta scale as recommended by the International Society on Thrombosis and Haemostasis. Iliofemoral patency was assessed with ultrasonography and air-plethysmography. A two-sided uncorrected Chi-square test was used for comparing dichotomous variables in the two treatment groups. Results: 209 patients with acute iliofemoral DVT were randomized during 2006–2009; 101 patients were allocated the CDT arm and 108 the control arm. At the completion of 24 months follow-up, data on clinical status were available and included in the intention to treat analyzes for 90 patients in the CDT arm and 99 control patients. Mean age was 51.5 years (SD 15.8), 36% were female, and mean duration of symptoms was 6.6 days (SD 4.6). 80/90 patients receiving CDT had successful lysis. At 24 months follow-up 37 (41.1%, 95% CI 31.5–51.4%) allocated additional CDT presented with PTS compared to 55 (55.6%, 95% CI 45.7–65.0%) in the control group (p=0.047), including one control with severe PTS. The difference in PTS corresponds to an absolute risk reduction of 14.4% (95% CI 0.2–27.9), and the number needed to treat was 7 (95% CI 4–502). No patients presented with venous ulcer. In total 20 bleeding complications were reported; 3 were classified as major and 5 as clinically relevant. The majority of bleedings were related to the puncture site. The major bleedings included 1 abdominal wall hematoma requiring blood transfusion, 1 compartment syndrome of the calf requiring surgery, and 1 inguinal puncture site hematoma. No bleeding led to a permanently reduced outcome, and there were no deaths, pulmonary embolism or cerebral hemorrhage related to CDT. Patients who had regained venous patency after 6 months, developed PTS in 38/103 (36.9%, 95% CI 28.2–46.5%) as compared to 49/80 (61.3%, 95% CI 50.3–71.2%) of patients with insufficient recanalization (p

Description: Background: Several strategies for managing patients with suspected PE have been validated. However, most of these strategies are complicated, involving multiple rounds of tests, and are thus, time consuming, costly and difficult to apply in clinical practice. Aiming to introduce a simple, fast and cost-effective strategy, we adopted a new diagnostic approach combining clinical probability assessment, D-Dimer and multi-slice spiral CT (MSSCT) scanning. Aims: The aim of this study was to assess the safety and efficacy of this management strategy by a prospective outcome study with 3-month follow-up. Methods: 495 consecutive patients referred to the Emergency Department at Østfold Hospital, Fredrikstad, Norway, for suspected PE, between Feb 2002 and Dec 2003, were considered for inclusion. 63 (12.7%) patients were excluded and the final cohort consisted of 432 patients. Patients were managed by serial non-invasive testing starting with D-Dimer test. Normal plasma D-Dimer (Liatest, latex agglutination assay, Stago-France, cut-off

Description: Background: ITP is a rare autoimmune disorder characterized by low platelet counts, resulting in an increased risk for bleeding. Paradoxically, patients with cITP may also have an increased incidence of thrombotic or thromboembolic events, but population-based data on this are limited. We estimated the incidence of these events in a prospective cohort study of incident cITP patients in three Nordic countries. Methods: Based on National Health Registry Systems (NHRSs) and medical records in Denmark, Norway, and Sweden, the study cohort included all adults diagnosed with cITP from January 1, 2008 to December 31, 2012 (n=1,821). Arterial thrombotic events (myocardial infarction [MI] and stroke) and venous thromboembolic events (pulmonary embolism and deep vein thrombosis) were identified in the NHRSs. Patients were followed from the latest of cITP diagnosis or April 1, 2009, until the earliest date of the first occurrence of the event of interest, death, emigration, or December 31, 2012. Incidence rates (events per 1,000 person-weeks [PW]) were computed for the entire cohort and stratified by gender, age, splenectomy status, and comorbidity burden. Results: Nearly 60% of the cITP cohort was 〉50 years in age, and 56% were female. Overall, the incidence of arterial thrombotic events was 0.31 per 1,000 PW (95% confidence interval [CI]: 0.25-0.39) and the incidence of venous thromboembolic events was 0.18 per 1,000 PW (95% CI: 0.13-0.24). For arterial thrombotic events, the risk was higher in males (compared with females), and the risk for both event types increased with increasing age and comorbid burden. Given the small number of patients who underwent splenectomy (n=101), it was difficult to detect any differences in the risk of thrombotic events by splenectomy status, but the incidence of venous thromboembolic events was higher in splenectomized versus non-splenectomized patients (0.27 per 1,000 PW [95% CI: 0.15-0.50] versus 0.16 [95% CI: 0.11-0.23]). Conclusions: Among patients with cITP, the risk of arterial thrombotic events is higher in males than females and increases with increasing age and level of comorbid burden. The risk of venous thromboembolic events is heightened in cITP patients who have undergone splenectomy. Abstract 4845. TableArterial thrombotic eventsVenous thromboembolic eventsN / PWIncidence rate per 1,000 PW (95% CI)N / PWIncidence rate per 1,000 PW (95% CI)Overall (n=1,821)68 / 218,3910.31 (0.25-0.39)39 / 220,8650.18 (0.13-0.24)GenderMale (n=794)39 / 90,5310.43 (0.31-0.59)17 / 92,4080.18 (0.11-0.30)Female (n=1,027)29 / 127,8600.23 (0.16-0.33)22 / 128,4570.17 (0.11-0.26)Age18-50 years (n=751)5 / 96,1190.05 (0.02-0.12)5 / 95,9630.05 (0.02-0.13)51-70 years (n=544)13 / 65,6520.20 (0.11-0.34)13 / 65,5560.20 (0.12-0.34)〉70 years (n=526)50 / 56,6210.88 (0.67-1.17)21 / 59,3450.35 (0.23-0.54)Comorbid burdenLow (n=1,243)25 / 154,1810.16 (0.11-0.24)18 / 155,0580.12 (0.07-0.18)Moderate (n=435)29 / 48,3070.60 (0.42-0.86)14 / 49,6940.28 (0.17-0.48)High (n=143)14 / 15,9030.88 (0.52-1.49)7 / 16,1130.43 (0.21-0.91)Splenectomy statusSplenectomized (n=101)10 / 37,1210.27 (0.14-0.50)10 / 36,9570.27 (0.15-0.50)Non-splenectomized (n=1,720)58 / 181,2700.32 (0.25-0.41)29 / 183,9080.16 (0.11-0.23) Disclosures Cetin: Amgen: Employment. Ghanima:Roche: Consultancy, Research Funding; Amgen: Consultancy, Research Funding; GlaxoSmithKline: Consultancy. Stryker:Amgen Inc.: Employment, Equity Ownership.

Description: Background In myeloproliferative neoplasms (MPN), Interferon-α (IFN-α) has been shown effective in inducing hematologic and molecular responses and in reducing vascular events. In clinical practice its use is mainly limited by intolerance due to side effects. Aim We sought to evaluate the tolerability of IFN-α therapy, the thromboembolic incidence and the causes of termination of therapy in a cohort of MPN, treated outside of clinical trials. Methods One hundred patients (M/F 41/59, median age 48 years, range 15-73) with a diagnosis of polycythemia vera (PV, n=47), essential thrombocythemia (ET, n=43) and myelofibrosis (MF, n=10) according to the WHO 2008 criteria, on current or previous treatment with IFN-α (IFN-α-2b, Peg-IFN-α-2b, Peg-IFN-α-2a) were included. The patients, diagnosed 1987-2012, were recruited from 9 centers in Sweden and Norway, and retrospectively analyzed. Hematologic response in PV and ET was assessed according to ELN criteria from 2009. Response to treatment in MF was defined as platelets ² 400x109 /L, white blood counts ² 10x109/L and transfusion independency. Results IFN-α treatment characteristics are displayed in Table 1. The median treatment duration for IFN-α was 34 months. Treatment prior to IFN-α had been received by 44 pts including hydroxyurea (n=34), anagrelide (n=19), busulphan (n=2), radioactive phosphorus (n=1), 10 pts having received more than one cytoreductive agent. Complete hematologic response (CR) was observed in 58 pts (PV=28/47, ET=30/43) and partial hematologic response (PR) in 15 pts (PV=2, ET=13). In MF, hematologic response was noted in 8 out of 10 patients. IFN-α related adverse events (AE) were recorded in 76 pts (76/100, 76%) with similar rates between genders (M 30/41, 73%, F 45/59, 76%). AE were generally of low grade. Twenty pts experienced multiple (³ 3) side effects (M/F 6/14), females reporting a total of 96 AE compared to 53 in males. Hematologic toxicity was low with 4 pts presenting with anemia, 4 with leukopenia and 3 with thrombocytopenia. Most common non-hematologic toxicities were fatigue in 30 pts (M/F 11/19), myalgia in 28 (M/F 11/17) and depression in 21 (M/F 4/17), followed by liver function test elevation (n=9), headache (n=9), alopecia (n=8) and skin reaction (n=7). In two pts with autoimmune co-morbidities (rheumatoid arthritis, psoriasis), flare-up of symptoms related to autoimmune activity were seen, leading to discontinuation of therapy. Only one vascular event occurred in a 64 year old woman with PV, in CR since 92 months, who developed a myocardial infarction after 94 months of IFN-α-2b treatment. A total of 43 pts (M 16/41, 39%, F 27/59, 46%) discontinued therapy, of whom 34 (M 13/41, 32%, F 21/59, 36%) due to side effects. The most common cause of discontinuation of therapy due to side effects was depression (15/21), followed by fatigue (12/30) and myalgia (9/28). Nineteen (19/34, 58%) of the pts who discontinued therapy due to side effects were in CR. Discontinuation due to other reasons than side effects were lack of efficacy/progression of disease (n=5), co-morbidities (n=2), CR including molecular response (n=2) and pregnancy (n=1). Out of the 53 pts with ET and MF, 25 were JAK2V617F mutated and 14 had a CALR-mutation. No significant differences between these two groups were seen regarding side effects or discontinuation rate. Out of the 57 pts remaining on IFN-α, 19 still received IFN-α-2b (19/35, 54%), 7 PegIFN-α-2b (7/12, 58%) and 31 PegIFN-α-2a (31/53, 58%). Conclusion In this retrospective cohort study, the treatment discontinuation rate due to side effects was higher than in previous reports. This may be explained by the relatively long median duration of treatment in this cohort, reflecting a poor tolerance of low-grade toxicity over time. Depression was frequent and the most common reported side effect when therapy was discontinued. Gender difference, with females reporting a higher incidence of depression and a larger total burden of AE, was noted. The frequency of thromboembolic events was very low in this IFN-α treated cohort. Table 1. IFN-α treatment characteristics. Type of Interferon Patiens (n) Dose per week–median (range) Treatment time-median IFN α-2b 35 9 MIE (1,2-20) 58 PegIFN α-2b 12 40 μg (30-80) 46 PegIFN α-2a 53 90 μg (30-135) 15 Disclosures No relevant conflicts of interest to declare.

Description: Treatment strategies for immune thrombocytopenia (ITP) aim to achieve a platelet count associated with adequate hemostasis while maximizing patient (pt) quality of life (QoL) and minimizing treatment-emergent toxicities. However, physicians' (MDs) and pts' treatment goals have not been studied and compared. I-WISh is a cross-sectional survey of 1507 ITP pts and 472 MDs across 13 countries studying burden of ITP and pt QoL using a global pt and MD sampling frame; pts were recruited via MDs and pt advocacy groups (PAGs). ITP expert MDs and PAG representatives designed and endorsed the survey. We report treatment goals that MDs and pts perceive as being most important and how these vary among countries and different healthcare systems. Pt-reported use of ITP treatments at time of survey and any prior time are also described. Top-ranked treatment goals for MDs were reduction in spontaneous bleeds (72%), better QoL (64%), and healthy blood counts (51%). The top treatment goals for pts were healthy blood counts (64%), preventing episodes of ITP worsening (44%), and increasing energy levels (41%). More MDs than pts selected reduction in spontaneous bleeds (72% vs 38%) and QoL (64% vs 38%) as top priorities; fewer MDs vs pts selected healthy blood counts (51% vs 64%) and reduction of fatigue (15% vs 41%). MD and pt rankings of their top 3 treatment goals by country are shown in Fig.1 A&B and differences in MD vs pt perceptions in Fig. 1C. The largest differences in the percentage of MDs and pts who ranked reduction in spontaneous bleeds as a top goal were observed in France, India, Canada, UK, and USA; in each country, MDs ranked this treatment goal more often in their top 3 than pts. In contrast, lighter menstrual periods had a 〈 10% difference between pts and MDs in all but 2/13 countries. In certain countries, responses appeared to be balanced across similar goals, i.e. ~40% difference in ranking of reduction in spontaneous bleeds by MDs vs pts and healthy blood counts by pts vs MDs in the UK. Similarly, an 11% difference in MD vs pt ranking of improved QoL and pt vs MD ranking of increasing energy levels were observed. The largest differences in the percentages of MDs and pts who ranked healthy blood counts in their top 3 goals were observed in UK and Colombia. The largest differences between MDs and pts in their perception of reduction of fatigue as a top 3 goal were in Canada, Egypt, USA, and UK; MDs generally ranked it less important than pts, except in India. Corticosteroids (CSs) were prescribed in 79% of pts for treatment of ITP, with 26% of pts on CSs at time of survey. Countries with the highest CS use at any time were Colombia, France, and USA; Colombia, India, and Egypt had the highest CS use at time of survey. Lowest CS use at any time prior to the survey was reported by pts in Japan, Turkey, and Germany. Thrombopoietin receptor-agonists (TPO-RAs) were prescribed in 27% of pts, with 17% of pts on TPO-RA at time of survey. Countries with highest TPO-RA use at any time were Turkey, UK, Egypt, and USA. At the time of survey, TPO-RAs were most frequently used by pts in Egypt, with the lowest use in Japan. Anti-CD20 was prescribed in 29% of pts, with 5% on anti-CD20 at the time of survey. Countries with highest anti-CD20 use at any time were USA, France, and Canada. At the time of survey, anti-CD20s were most frequently used by pts in India, with no use in Turkey or Egypt. CSs were the most prevalent ITP treatments prior to and at time of survey. While this was to be expected, the high use of steroids in the USA and France compared with other countries was unanticipated. TPO-RAs and anti-CD20 have changed the landscape of second-line ITP therapies, but their utilization varies greatly among countries. Access to advanced/costly therapies is one factor that differs between countries and may influence treatment trends, but others are also important. The I-WISh survey showed that certain specific treatment goals are perceived differently by MDs and pts. Interpretation of the data is limited by the similarity of answer choice (eg, healthy blood counts and absence of spontaneous bleeds; fatigue, a key component of QoL; and QoL), which may have led to differences in responses despite similar understanding. Nonetheless, an overall agreement was generally observed, which may have been facilitated by increased MD and pt access to information through the internet and other sources, eg, medical education activities. Figure 1 Disclosures Bussel: Tranquil: Honoraria, Membership on an entity's Board of Directors or advisory committees; Amgen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; argenx: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Kezar Life Sciences: Consultancy, Membership on an entity's Board of Directors or advisory committees; UCB: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; RallyBio: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Physician Education Resource: Speakers Bureau; 3S Bio: Speakers Bureau; Rigel: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; GSK: Honoraria, Membership on an entity's Board of Directors or advisory committees; Dova Pharmaceuticals: Consultancy, Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Momenta Pharmaceuticals: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Regeneron: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees. Ghanima:Amgen: Consultancy, Honoraria; Bayer: Honoraria, Research Funding; Pfizer/BMS: Research Funding; Novartis: Consultancy, Honoraria, Research Funding. Tomiyama:Chugai: Honoraria; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Kyowa-Kirin: Honoraria. Arnold:Rigel: Consultancy, Research Funding; Novartis: Honoraria, Research Funding; Bristol-Myers Squibb: Research Funding; Principia: Consultancy. Provan:Rigel ONO: Consultancy, Research Funding; ONO Pharmaceutical: Consultancy; Amgen: Consultancy, Honoraria, Research Funding; MedImunne: Consultancy; UCB: Consultancy; Novartis: Consultancy, Honoraria, Research Funding. Santoro:Grifols: Membership on an entity's Board of Directors or advisory committees; Novo Nordisk: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Shire: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; CSL: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Sobi: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Amgen: Speakers Bureau; GSK: Speakers Bureau; Bayer: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Pfizer: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Gilead: Membership on an entity's Board of Directors or advisory committees. Kruse:UCB: Honoraria; Amgen: Research Funding; Argenx: Research Funding; Dova: Research Funding; Novartis: Research Funding; Momenta: Research Funding; Principia: Research Funding; Octapharma: Research Funding; CSL Behring: Research Funding; UCB: Research Funding; Novartis: Consultancy. Waller:Adelphi Real World: Employment; Novartis: Consultancy. Haenig:Novartis: Employment. Cooper:Principia: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Amgen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Rigel: Consultancy, Membership on an entity's Board of Directors or advisory committees.

Description: Introduction Romiplostim was approved in 2009 in Europe for treatment of adult chronic immune thrombocytopenia (cITP) in splenectomized patients, refractory to other ITP treatments, and as second-line treatment for adult non-splenectomized patients. In 2016 the indication was extended to second-line treatment in all cITP patients. Few data exist on how romiplostim is used in routine clinical care. Methods To examine romiplostim treatment patterns we used the Nordic Country Patient Registry for romiplostim (NCPRR). NCPRR was established in 2009 and includes all adult (≥18 years) patients in Denmark, Sweden and Norway with a confirmed cITP (ITP lasting 〉6 months) diagnosis requiring hospital contact. The cohort is based on data from national health registries and enriched by medical record review. All patients diagnosed with cITP from April 1, 2009 to December 31, 2016 (data cut-off) are included. We describe age, comorbidity, previous treatment, platelet level, and both romiplostim dose and duration in patients who started romiplostim after date of their cITP diagnosis by line of treatment. Results Among 2895 patients diagnosed with cITP: 103 patients started romiplostim treatment before the data cut-off. Of these, 40% were aged 18- 50 years old, 30% were 35-70 years old, and 30% were ≥71 years. A total of 76% had no recorded comorbidity. Romiplostim was first-line treatment in 8 cITP patients of whom one had been splenectomized. In the month before romiplostim start, six of these patients (75%) had platelet count 50x109/L and 15 (34%) had a highest platelet count of 50 to 150x109/L. In the remaining 31 romiplostim-treated patients, it was used as fourth or later treatment line: median latest platelet count in the month preceding initiation was 14 x109/L (IQR: 5-27), with 7 (22.6%) patients experiencing clinically important bleeding during this time. Duration of the previous treatment in these patients had been ≤1 month in 16 (52%) and 1- 3 months in 12 (39%) patients. During their previous line of treatment 14 (45%) patients did not reach a platelet count of 〉50x109/L and an additional 9 (29%) had a highest platelet count of 50 to 150x109/L. In patients initiating romiplostim at first-line, median maximum platelet count while on therapy was 147x109/L (IQR: 109-237): this value was 299x109/L (IQR: 187,752), 295x109/L (IQR: 107,454), and 132x109/L (IQR: 52-305) for second, third, and fourth-or-later lines respectively. Median duration of romiplostim therapy was shortest at first-line (37 days, IQR: 21-180), and longest at second-line (91 days, IQR: 21, 169). Two patients on second-line, and 8 patients on third-line eltrombopag, switched to romiplostim. Conclusion Approximately 4% of cITP patients were treated with romiplostim, predominantly at third or later treatment lines: median platelet counts were seen to improve from 100x109/L while on therapy across all lines. Romiplostim treatment had a relatively short duration. However, romiplostim-treated patients were characterized by a short duration on their previous non-romiplostim treatment line, and a high proportion had low platelet counts during this prior treatment. These data indicate that romiplostim is effective at increasing platelet counts in cITP patients with varying clinical history. Larger studies are needed to confirm these results and investigate drivers at different lines of therapy. Disclosures Bahmanyar: Amgen: Research Funding. Ghanima:GlaxoSmithKline and Pfizer: Other: Personal Fees; Roche, Amgen, Novartis, Bayer, BMS: Other: Personal Fees, Research Funding. Alam:Amgen: Employment, Equity Ownership. Christiansen:Amgen: Research Funding.