Fat abnormality was prevalent in close to half of children and adolescents, who had accumulated long treatment durations. Risk of fat abnormality was associated with specific drugs, including stavudine and ritonavir, and other variables. Our results underline the importance of continued surveillance of children treated with antiretroviral therapy.

Background: Allogeneic HCT is a potentially curative procedure for many hematologic malignancies, but is associated with several complications. Acute GvHD is a condition that affects 35-50% of allogeneic HCT recipients, typically occurs within 100 days after transplant, and can be life-threatening. Existing treatments are poorly tolerated and frequently ineffective. While the clinical consequences of acute GvHD are understood, the economic burden of the condition has not been well characterized. Methods: Using a large US healthcare claims database (Truven MarketScan® Commercial Claims and Encounters Database), patients aged ≥2 years old were identified who underwent allogeneic HCT between October 2009 and March 2013. Patients without continuous health plan enrollment or evidence of hematologic malignancy in the 6-month period prior to the "Index Admission" (hospital admission during which HCT was performed) were excluded. Patients were followed from the first day of the Index Admission until death, plan disenrollment, or one year; whichever occurred first ("Follow-Up"). Patients were classified into two cohorts: "Acute GvHD" based on ICD-9 CM diagnosis codes within first 100 days of Follow Up, or "No Acute GvHD". Total healthcare costs (inpatient care, outpatient care, outpatient pharmacy) and hospital length of stay (LOS) at discharge from Index Admission, the 100th day of Follow-Up, and the 365th day of Follow-Up were determined. Reimbursed amounts (plan payment plus patient liability) were used as a proxy for healthcare costs. Total healthcare costs and LOS between the two cohorts were compared for each of the three evaluation periods using Student's t-tests (unadjusted analyses), and analyses of covariance (ANCOVA; adjusting for differences in age, sex, plan type, geography, year of Index Admission, type of malignancy, Charlson Comorbidity Index score, and pre-admission healthcare cost). Results: 1,635 patients underwent HCT and met all selection criteria (mean age was 48 years, 56% were men, acute myeloid leukemia was the most common malignancy); 42% met the criteria for inclusion in the Acute GvHD cohort. Univariate mean total healthcare costs for the Acute GvHD cohort (vs No Acute GvHD) were $36,651 greater during Index Admission, $83,322 greater at the 100th day of Follow-Up, and $123,220 greater at the end of the 1-year Follow-Up (all p<0.01); additional mean LOS (days) were 5.7 during Index Admission, 14.0 at the 100th day of Follow-Up, and 17.4 at the end of the 1-year Follow-Up (all p<0.01) [Table 1]. Multivariate-adjusted mean total healthcare costs were $24,292 greater during Index Admission (p=0.02), $73,622 greater at the 100th day of Follow-Up (p<0.01), and $114,698 greater at the end of the 1-year Follow-Up (p<0.01); additional LOS was 5.2 during the Index Admission, 14.1 at the end of the 100th day of Follow-Up, and 17.9 at the end of the 1-year Follow-Up (all p<0.01) [Table 2]. Conclusions: Over the one-year period following allogeneic HCT, patients who develop Acute GvHD experience over $100,000 more in total healthcare costs-and nearly three additional weeks in hospital-relative to those who do not. While healthcare claims appear to represent a good source with which to assess the impact of complications of HCT, confirmation with prospective clinical studies is recommended. Given that nearly one-half of patients develop Acute GvHD, our findings suggest that therapeutic strategies that prevent this complication may confer substantial savings to the healthcare system. Disclosures Grubb: Fate Therapeutics: Employment, Equity Ownership. Huse:Evidera: Employment; Fate Therapeutics: Consultancy. Alam:Evidera: Employment; Fate Therapeutics: Consultancy. Dychter:Fate Therapeutics: Employment, Equity Ownership. Wingard:Merck: Consultancy; Ansun: Consultancy; Fate Therapeutics: Consultancy; Gilead: Consultancy; Astellas: Consultancy. Berger:Evidera: Employment; Fate Therapeutics: Consultancy.

Background Prior to regulatory approval of blinatumomab in pediatric patients with relapsed/refractory Philadelphia chromosome‐negative B‐cell precursor acute lymphoblastic leukemia (R/R Ph− BCP‐ALL), blinatumomab was made available via an expanded access program (EAP). Procedure This retrospective observational study included patients receiving blinatumomab in the EAP between January 1, 2014 and June 30, 2017 who were followed until death, entry into a clinical trial, end of follow‐up, or end of the study period (December 31, 2017), whichever occurred first. Results Among 113 children enrolled, 72 were diagnosed with R/R Ph− BCP‐ALL and 41 were minimal residual disease positive (MRD+, either Ph− or Ph+). In the R/R group, 38 (53%) patients achieved hematological response within two cycles. Of these, 19 (50%) proceeded to hematopoietic stem cell transplantation (HSCT) without bridging myelosuppressive therapy. Of 36 patients in the R/R group evaluable for MRD, 30 (83%) had an MRD response. In the R/R group, median relapse‐free survival was 5.4 months and median overall survival (OS) was 8.2 months. Of 36 patients in the MRD+ group who were evaluable for MRD after two cycles, 27 (75%) had an MRD response. Overall, 24 (59%) of the MRD+ patients proceeded to HSCT without other bridging therapy. Median disease‐free survival was 13.6 months; median OS was not reached. Conclusions In this real‐world pediatric cohort, blinatumomab was effective within two cycles. Over half of patients with R/R Ph− BCP‐ALL achieved hematological response and most achieved MRD response in the MRD+ group, confirming the efficacy of blinatumomab in pediatric trials.

Introduction: Romiplostim has been approved in Europe since 2009 to treat patients with chronic primary immune thrombocytopenia (ITP). Using real-world data from seven European countries, we measured the effectiveness and safety outcomes within 24 weeks following romiplostim initiation by duration of ITP: less than 3 months (''newly diagnosed''), 3--12 months (''persistent''), and more than 12 months (''chronic''). Methods: Adults with ITP and C 1 romiplostim administration between 2009 and 2012 were included. Endpoints included durable platelet response, median platelet count, rescue therapy, bleeding and adverse events. We used inverse probability of censoring weighted esti-Supplementary Information The online version contains supplementary material available at

Background: Blinatumomab is approved in Europe for adult and pediatric patients (pts) with relapsed and/or refractory Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (R/R Ph- BCP-ALL), and for adult pts with minimal residual disease (MRD)-positive Ph- BCP-ALL. Prior to country-specific reimbursement, blinatumomab was made available to pts who met pre-specified criteria via an expanded access program: this included both adult and pediatric pts with a diagnosis of R/R Ph- BCP-ALL, R/R Ph+ BCP-ALL, or MRD-positive Ph-/Ph+ ALL. Here, we describe adults with R/R Ph- BCP-ALL enrolled in this retrospective observational study (NEUF) in specific European countries, with reference to their characteristics, blinatumomab usage and effectiveness. Methods: Eligible pts initiated blinatumomab in the expanded access setting between 1 Jan 2014 and 31 Dec 2016. Data were extracted from medical notes. Pts were followed from blinatumomab initiation until death, entry into a clinical trial, end of follow-up, or the end of the study period (30 June 2017), whichever occurred first. Adverse events were reported separately, according to local regulations. Calculation of percentages excluded patients with missing data, unless otherwise indicated. Results: In total, 253 adult pts were enrolled (113 in Italy, 45 in Russia, 53 in Spain, 33 in France, and 9 in the UK): prior to blinatumomab initiation 106 (43%) had a diagnosis of R/R Ph- BCP-ALL, 32 (13%) had R/R Ph+ BCP-ALL, 109 (44%) had MRD positive ALL (either Ph- or Ph+), and 6 (2%) had diagnosis data missing. Among R/R Ph- BCP-ALL pts (n=106), 47% were female and median age was 36.5 years (interquartile range [IQR]: 24.0, 52.0). Forty-one percent (n=43) had prior allogeneic hematopoietic stem cell transplant ((HSCT). The median number of prior salvage therapies was 1.0 (range: 0.0, 2.0). At blinatumomab initiation, 64 (60%) experienced a relapse and 42 (40%) were refractory. At least half (53%, n=54) of pts were treated with pre-phase and 89% (n=93) with pre-medication with dexamethasone. Within two cycles of blinatumomab, 54 (51%) pts achieved complete remission (CR) with full/partial/incomplete recovery of peripheral blood counts. Among patients achieving CR and who had evaluable MRD (n=33), 85% (n=28) had MRD response (16 with non-detectable MRD and 12 with MRD <10-4). Following blinatumomab, 41% (n=43) pts proceeded to HSCT, among whom 77% (n=33) achieved CR prior to transplant. Median time from CR to HSCT was 4.6 months (range: 0.2, 7.4). Median relapse-free survival in R/R Ph- BCP-ALL was 11.0 months (range: 0.0, 15.4). Among the 22 adults who experienced blinatumomab relapse and were also tested for CD19 expression and 95% (n=21) were positive. At 24 months following blinatumomab initiation, the Kaplan-Meier (KM) median estimate of overall survival (OS) among 102 evaluable pts was 40% (95% confidence interval [CI]: 29, 51) (Figure); when censoring for HSCT, the OS probability (KM median estimate) was 36% (95% CI: 19, 53): median follow-up time in these analyses was 17.3 months (IQR: 10.8, 23.6) and 9.5 months (IQR: 4.4, 24.2), respectively. The 3-month non-relapse mortality following HSCT post-blinatumomab was 11% (95% CI: 4, 29). Conclusions: This is the largest documented cohort of R/R Ph- BCP-ALL patients treated with blinatumomab in real-world clinical practice. A high proportion of pts achieved CR, and over one-third could proceed to HSCT. Over one-third of pts were still alive 24 months after blinatumomab initiation. The results are widely consistent with published results from clinical trials and they confirm the effectiveness of blinatumomab in this real-world setting. Disclosures Boissel: NOVARTIS: Consultancy. Chiaretti:Incyte: Membership on an entity's Board of Directors or advisory committees; Pfizer: Membership on an entity's Board of Directors or advisory committees; Amgen: Membership on an entity's Board of Directors or advisory committees; Shire: Membership on an entity's Board of Directors or advisory committees. Rambaldi:Novartis: Membership on an entity's Board of Directors or advisory committees, Other: travel support, Speakers Bureau; Jazz: 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, Other: travel support, Speakers Bureau; Roche: Membership on an entity's Board of Directors or advisory committees, Other: travel support, Research Funding, Speakers Bureau; Celgene: Membership on an entity's Board of Directors or advisory committees, Other: travel support, Speakers Bureau; Amgen: Membership on an entity's Board of Directors or advisory committees, Other: travel support, Research Funding, Speakers Bureau; Pfizer: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Omeros: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Italfarmaco: Membership on an entity's Board of Directors or advisory committees, Other: travel support, Research Funding, Speakers Bureau. Bassan:Pfizer: Honoraria; Incyte: Honoraria; Amgen Inc.: Honoraria; Shire: Honoraria. Papayannidis:Amgen: Honoraria; Incyte: Honoraria; Shire: Honoraria; Pfizer: Honoraria; Novartis: Honoraria; Teva: Honoraria. Alam:Amgen: Employment, Equity Ownership. Brescianini:Amgen: Employment, Equity Ownership. Pezzani:Amgen: Employment, Equity Ownership. Kreuzbauer:Amgen: Employment, Equity Ownership. Foà:Incyte: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Abbvie: Consultancy, Speakers Bureau; Celltrion: Membership on an entity's Board of Directors or advisory committees; Roche: Consultancy, Speakers Bureau; Abbvie: Consultancy, Speakers Bureau; Roche: Consultancy, Speakers Bureau; Shire: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Amgen Inc.: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Amgen Inc.: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Janssen: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Celltrion: Membership on an entity's Board of Directors or advisory committees; Janssen: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Shire: Consultancy, 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; Pfizer: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Incyte: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau.