Real-world Single-center Experience Research Articles

Role of the T2Dx magnetic resonance assay in patients with suspected bloodstream infection: a single-centre real-world experience

BackgroundT2Dx was approved by the US Food and Drug Administration for the rapid detection of a modified panel of ESKAPE bacterial species or Candida spp. causing bloodstream infection (BSI).Patients and methodsWe performed a retrospective, observational study from January 1, 2018 to December 31, 2019 of all hospitalised patients with suspected BSI who underwent assessment using T2Dx in addition to standard blood culture (BC). T2-positive patients (cases) were compared to a matched group of patients with BSI documented only by BC (1:2 ratio) to investigate the possible impact of T2Dx on the appropriateness of empirical antimicrobial therapy and 21-day mortality.ResultsIn total, 78 T2Dx-analysed samples (49 patients) were analysed. The T2Dx assay result was positive for18 patients and negative for 31 patients. The concordance rates of the T2Bacteria Panel and T2Candida Panel results with those of standard BC were 74.4% and 91.4%, respectively. In the matched analysis, inappropriate empiric antimicrobial therapy administration was significantly less frequent in cases than in comparators (5.5% vs. 38.8%). The 21-day mortality rate was twofold lower in cases than in comparators (22.2% vs. 44.4%), although the difference was not significant. No other analysed variables were significantly different between the two groups.ConclusionsThis study illustrated that T2Dx might be associated with an increase in the appropriateness of empiric antimicrobial therapy in patients with BSI. Further studies are needed to evaluate whether the T2Dx assay can improve patient outcomes.

Activation of PPCI team in the octogenarian and nonagenarians population: real-world single-centre experience.

ObjectiveAdvancement in healthcare provision has led to increasing octogenarian ST elevation myocardial infarction (STEMI) presentation to hospital for early revascularisation therapies. Limited literature to date exists to suggest octogenarian STEMI population; with majority of trials excluding these age group patients. Due to an ageing population, we expect increasing rates of STEMI in the octogenarian and nonagenarian population in the future. This study seeks to identify the outcomes of patients over the age of 80 presenting with STEMI and determine the factors associated with better or worse outcome.Patients and methodsThis study is a single-centre retrospective observational study involving patients’ age 80 or older presenting with STEMI between January 2014 and December 2019. Patient data were collected by chart review and analysis of the local STEMI database. Standard Bayesian statistics were employed for analysis.Results1301 patients presented with STEMI during this period. 159/1301 (12.2%) were 80 years or older that fulfilled STEMI criteria, 35/159 (22.1%) were medically managed. 107/124 (86.29%) had angiographic evidence of acute total or partial thrombotic occlusion, and 97/107 were treated with primary percutaneous coronary intervention (PPCI). The activation ECG most commonly exhibited an anterior STEMI, while inferior STEMI ECGs had the strongest positive predictive value. PPCI group had a 30-day mortality rate of 20% (p=0.07) and 1-year mortality was 22.4%. Highest mortality was observed with cardiogenic shock, low ejection fraction, higher high sensitivity cardiac troponin T and creatinine at presentation. Conservatively managed patients had significant higher mortality rate (48% vs 22.4%, p=0.005) at 1 year.ConclusionPatients over the age of 80 who present with STEMI and undergo PPCI have a significantly lower mortality rate at 1 year. These patients have a 77.6% survival at 1 year, with 92.4% likelihood of discharge to home (without need for long-term nursing home care). Cardiogenic shock in this group was associated with a 1-year mortality of 87.5%. Despite the advanced age, we suggest favourable outcomes described in the absence of patients presenting with cardiogenic shock.

A 10-Year Single-Center Experience With the GORE TAG Conformable Thoracic Stent Graft in the Treatment of Thoracic Aortic Disease

Objective: The aim of this study was to report 10-year real-world single-center experience with the GORE TAG conformable thoracic aortic graft (CTAG), focusing on rupture-free survival, aortic-related reintervention, and device-related complications during midterm and long-term follow-up (FU). Methods: This retrospective study analyzes results of thoracic endovascular aortic repair (TEVAR) performed between January 2009 and December 2018. Out of 419 TEVAR procedures within this period, 194 patients (male 57.2%, 111/194), with a mean age of 65 ± 13 years, were treated with the CTAG device. Indication for TEVAR was a thoracic aortic aneurysm in 24.7% (48/194), type B aortic dissection in 32.5% (63/194), penetrating aortic ulcer 15.5% (30/194), and miscellaneous 27.3% (53/194). Emergently were operated 43.8% (85/194) patients. Median follow-up (FU) including computed tomography imaging was 43.5 months (Q1-Q3: 8.6–67.0) and was completed in 91.2% (177/194) of patients. Results: Overall survival rates were 75.8% (95% confidence interval [CI] = [0.76–0.70]) and 56.6% (95% CI = [0.57–0.50]) at 12 and 60 months, respectively. Cumulative incidence for aortic rupture was 11.9% (95% CI = [0.07–0.17]) at 60 and 90 months, respectively. Cumulative incidence for aortic-related reintervention was 27.5% (95% CI = [0.21–0.34]) at 60 and 90 months. Cumulative incidence for migration was 2.8% (95% CI = [0.004–0.05]) and 3.9% (95% CI = [0.007–0.07]) at 60 and 90 months, respectively. New endograft infections or material fatigue were not observed. Conclusions: The herein reported 10-year real-world single-center experience with the CTAG observed favorable long-term outcome. Thus, the device demonstrates appropriate persistent safety, efficacy, and clinical durability up to long-term FU in the treatment of diverse thoracic aortic pathologies.

P-194: Pomalidomide-based therapy in Relapsed/Refractory Multiple Myeloma: a real-world single-center experience

P-194: Pomalidomide-based therapy in Relapsed/Refractory Multiple Myeloma: a real-world single-center experience

Direct-acting antiviral regimens in Egyptian patients with chronic hepatitis C virus infection: A real-world single-center experience

Background and study aims:Chronic hepatitis C virus (HCV) infection has always been identified as a major health threat and a potential cause of liver cirrhosis, portal hypertension, and other associated problems. The introduction of direct-acting antiviral agents (DAAs) has represented a paradigm shift in HCV management. In this study, we aim to observe the rate of sustained virologic response (SVR12) in a large scale of patients at a single center as well as record the post-treatment changes in the hematologic, hepatic, and renal biochemical profiles. Patients and methodsIn total, 1933 chronic HCV genotype 4 mono-infected non-HCC patients who completed the treatment with six different DAA regimens in the Faculty of Medicine, Ain Shams University Research Institute (MASRI), were retrospectively enrolled in this study. The rate of sustained virologic response after 12 weeks off-therapy (SVR12) was assessed. The baseline characteristics to predict the SVR12 were then analyzed. The post-treatment changes in many profiles were recorded and analyzed. ResultsThe overall SVR12 rate was 96.2% (after excluding 84 cases who were lost to follow-up). It was achieved in 346/375 patients (92.3%), 466/477 patients (97.7%), 60/62 patients (96.8%), 11/11 patients (100%), 532/545 patients (97.6%), and 445/463 patients (96.1%) who received sofosbuvir/daclatasvir (SOF/DCV), sofosbuvir/daclatasvir/ribavirin (SOF/DCV/RBV), sofosbuvir/ledipasvir (SOF/LDV), sofosbuvir/ledipasvir/ribavirin (SOF/LDV/RBV), sofosbuvir/simeprevir (SOF/SMV), and ombitasvir/paritaprevir/ritonavir/ribavirin (OBV/PTV/r + RBV), respectively. In total, 73 patients (3.8%) failed to achieve SVR12. The baseline aspartate aminotransferase (AST), cirrhotic status, and treatment regimen were determined to have a significant impact on SVR12. In the overall treated population, the levels of serum AST, alanine aminotransferase, albumin, creatinine, bilirubin, and hemoglobin and platelet count improved significantly after treatment. Furthermore, sustained virologic response was strongly related to cirrhosis and its degree. ConclusionThe interferon-free DAA regimens offered high SVR12 rates in Egyptian patients with chronic HCV infection. They were associated with a significant improvement in the hematologic, hepatic, and renal biochemical profiles. The baseline AST, liver cirrhosis, and treatment regimen might have an impact on achieving SVR.

Risk factors affecting the outcomes in pediatric liver transplantation: A real-world single-center experience

Despite liver transplantation (LT) being the standard treatment for pediatric end-stage liver disease, complications often persist and can adversely affect the post-transplant outcomes. This study aimed to identify the risk factors affecting the outcomes in pediatric LT patients. Data from pediatric patients who underwent primary LT from March 1988 to December 2018 were retrospectively analyzed. Chronic liver disease was defined as an explanted liver showing fibrosis regardless of grade, cirrhosis, or any other underlying disease that may cause progressive liver injury leading to fibrosis or cirrhosis. A total of 255 pediatric patients underwent LT during the study period. Their 1-, 5-, and 10-year overall survival rates were 90.5%, 88.4%, and 87.8%. According to multivariate analysis, while liver disease without underlying chronic liver disease (p = 0.024) and a pediatric end-stage liver disease (PELD) score ≥ 30 (p = 0.036) were the only factors associated with worse survival, body weight < 6 kg (p = 0.050), whole-liver DDLT compared to LDLT (p = 0.001), fulminant liver failure (p = 0.008), and postoperative hepatic artery complications (p < 0.001) were associated with worse graft survival. Liver disease without underlying chronic liver disease was the only factor independently associated with hepatic artery complications (p = 0.003). Greater caution is recommended in pediatric patients with liver disease unaccompanied by underlying chronic liver disease, high PELD score, or low body weight to improve survival after LT. Hepatic artery complication was the only surgical complication affecting the graft survival outcome, especially in patients having liver disease without underlying chronic liver disease.

Tolerability and Efficacy of Post Transplant Lenalidomide Maintenance Therapy in Multiple Myeloma: A Real World Single Centre Experience

Tolerability and Efficacy of Post Transplant Lenalidomide Maintenance Therapy in Multiple Myeloma: A Real World Single Centre Experience

Use of drug-eluting stents in patients with critical limb ischemia and infrapopliteal arterial disease: a real-world single-center experience

BackgroundAlthough no drug-eluting stent (DES) has been approved by the Food and Drug Administration to treat infrapopliteal arterial disease, several industry-sponsored trials have reported the outcomes with the use of paclitaxel or sirolimus DESs. To the best of our knowledge, only one study to date has reported on the use of everolimus DESs for infrapopliteal arterial disease. In the present study, we analyzed the clinical outcomes with everolimus DESs in our real-world, single-center experience. MethodsA total of 107 limbs with critical limb threatening ischemia (98 patients; 118 lesions) treated with DESs (Xience; Abbott Vascular, Santa Clara, Calif) were analyzed. The postoperative early outcomes, major adverse limb events (above the ankle limb amputation or major intervention at 1 year), and major adverse events (death, amputation, target lesion thrombosis or reintervention) were analyzed. Kaplan-Meier analysis was used to estimate the primary patency rates (using duplex ultrasound), amputation-free rates, and amputation-free survival rates. ResultsOf the 118 lesions treated, 33% were in the anterior tibial artery, 28% were in the tibioperoneal (TP) artery, 21% were in the posterior tibial artery, 8% were in the peroneal artery, 5% were in the TP/posterior tibial artery, 4% were in the TP artery/PA, and 1% were in the TP/anterior tibial artery. The mean lesion length was 41 mm, and 59% were totally occluded (41% stenotic). The mean follow-up was 18.5 months (range, 1-70 months). The overall postoperative complication rate was 11% (2% major amputations), with 2% mortality. Late symptom improvement of one or more Rutherford category was obtained in 71%. The major adverse events rate at 30 days and 1 year was 12% and 45%, respectively. The major adverse limb events rate at 1 year was 15%. The overall primary patency rate was 42%. The primary patency rate at 1, 2, and 3 years was 57%, 45%, and 33%, respectively. The major amputation-free and overall amputation-free survival rates were 87%, 80%, and 77% and 76%, 65%, and 61% at 1, 2, and 3 years, respectively. ConclusionsThe clinical outcomes after DES (Xience; Abbott Vascular) for infrapopliteal lesions were somewhat satisfactory at 1 year but inferior to the previously reported outcomes, especially at 3 years. Further data with long-term follow-up are needed.

Adverse Events Induced by PD-1/PD-L1 Inhibitors: A Real-World Single-Centre Experience with a Management-Based Approach.

AimTo assess the efficacy and tolerance of programmed death-1 (PD-1) and PD-ligand 1 (PD-L1) inhibitors and the impact of a standardised management-based protocol in a real-world setting.Patients and MethodsData from patients who had received anti-PD-(L)1 were collected from our pharmacy database. Clinical response and toxicity were assessed using RECIST criteria and CTCAE version 5.0, respectively. Overall survival (OS) and progression-free survival (PFS) were estimated with the Kaplan–Meier method. Potential prognostic factors were identified using Cox’s model.ResultsA total of 196 patients and 201 lines of treatment were included (median age: 66 (range: 38–89) years). Types of cancer included non-small cell lung cancer (73%), transitional cell carcinoma (10%), renal cell carcinoma (6%), small cell lung cancer (5%), head and neck squamous cell carcinoma (4%) and classical Hodgkin’s lymphoma (1%). Twenty-five (12%) patients had pre-existing autoimmune conditions. Our standardised management-based protocol included 129 (64%) patients. Objective response rate was 29%, median OS was 10 months (IQR: 7–15) and median PFS was 5 months (IQR: 1–22). Patients with an abnormal baseline complete blood count had a worse OS (HR=2.48 [95% CI: 1.24–4.96]; p=0.0103). Thirty-three (16%) patients experienced severe (grade 3 or 4) immune-related adverse event (irAE). There were three (1%) irAE-related deaths. AEs resolved faster when patients were assessed by an internist before anti-PD-(L)1 initiation (p=0.0205).ConclusionPD-1 and PD-L1 inhibitors are effective and safe in a real-world setting. Implementation of a standardised management-based protocol with internal medicine specialists is an effective way to optimise irAE management.

Risk Factors Affecting Outcomes in Pediatric Liver Transplantation: A Real-World Single-Center Experience.

BackgroundDespite liver transplantation (LT) being the standard treatment for pediatric end-stage liver disease, complications often persist and can adversely affect the post-transplant outcomes. This study aimed to identify the risk factors affecting the outcomes in pediatric LT patients.Material/MethodsData from pediatric patients who underwent primary LT from March 1988 to December 2018 were retrospectively analyzed. Chronic liver disease was defined as an explanted liver showing fibrosis regardless of grade, cirrhosis, or any other underlying disease that may cause progressive liver injury leading to fibrosis or cirrhosis.ResultsA total of 255 pediatric patients underwent LT during the study period. Their 1-, 5-, and 10-year overall survival rates were 90.5%, 88.4%, and 87.8%, respectively. According to multivariate analysis, while liver disease without underlying chronic liver disease (P=0.024) and a pediatric end-stage liver disease (PELD) score ≥30 (P=0.036) were the only factors associated with worse survival, body weight <6 kg (P=0.050), whole-liver DDLT compared to LDLT (P=0.001), fulminant liver failure (P=0.008), and postoperative hepatic artery complications (P<0.001) were associated with worse graft survival. Liver disease without underlying chronic liver disease was the only factor independently associated with hepatic artery complications (P=0.003).ConclusionsGreater caution is recommended in pediatric patients with liver disease unaccompanied by underlying chronic liver disease, high PELD score, or low body weight to improve survival after LT. Hepatic artery complication was the only surgical complication affecting the graft survival outcome, especially in patients having liver disease without underlying chronic liver disease.

Abstract No. 11 Single-center real-world experience of the novel 355 nm Auryon atherectomy device

Peripheral artery disease (PAD) affects more than 200 million adults in the world and is frequently associated with intermittent claudication. Open surgical approaches have been used in the past, but endovascular approaches have evolved as the first line of treatment. The present study details a single-center real-world case series assessing the safety and efficacy of the Auryon atherectomy system in patients with infrainguinal PAD.

Ocrelizumab treatment in patients with progressive multiple sclerosis: a single-center real-world experience (2504)

Ocrelizumab treatment in patients with progressive multiple sclerosis: a single-center real-world experience (2504)

Multiple Myeloma in Young Adults: A Single Centre Real World Experience.

Multiple Myeloma accounts for 1% of all cancers. In India myeloma accounts for 1.23% of all cancers. The median age at diagnosis varies from 65 to 70 and only a small proportion of MMs are diagnosed at younger age, before 40 (approximately 2%). The present study is designed to analyse the clinical, haematological profile and outcomes in young adults with multiple myeloma. Data of all patients with age ≤ 40years, diagnosed with multiple myeloma from 2013 to 2018 was analysed. A total of 258 patients were diagnosed with multiple myeloma between 2013 and 2018, of which 22 (8.5%) were aged ≤ 40years. The median age was 33.5years(range,18-40). Male to female ratio was 1.75:1. Bone pain (59%) and fatigue (45.4%) were the most common symptoms at presentation. Majority of patients were ISS stage 3(63.6%). Cytogenetic data was available only in seven patients and IgH translocation and del 13q were the most common abnormalities, seen in four and three patients respectively. Of 22 patients, 17 patients had at least one response evaluation and were evaluated for outcomes. Eleven patients (64.7%) had responses greater than VGPR. Six (41.6%) patients underwent Auto HSCT. Four patients received second line therapy and only two received further lines of therapy. At a median follow up of 18months, 2-year EFS was 76.5% and 2-year OS was 94.1%. Patients younger than 40years, constitute higher proportion of patients in Indian sub-continent. Renal failure was more common in young myeloma patients. Light chain myeloma was more common in young adults. Most patients were ISS stage 3 at presentation and survival seems to be better in young adults when compared to elderly patients.

Effect of discontinuation of ticagrelor and switching-over to other P2Y12 agents in patients with acute coronary syndrome: a single-center real-world experience from India

BackgroundDual antiplatelet therapy is the current standard of care after acute coronary syndrome (ACS) and percutaneous coronary intervention (PCI). We intended to study the pattern of use of ticagrelor in patients with acute coronary syndrome undergoing PCI and the effect of switching over to other P2Y12 receptor inhibition on clinical outcomes.ResultsAll patients aged > 18 years who had been admitted with acute coronary syndrome and had been provided ticagrelor as the second antiplatelet agent were included as study participants. The primary outcome of the study was the composite outcome of death, recurrent myocardial infarctions, re-intervention, and major bleeding. We studied 321 patients (54 female patients, 16.82%). The mean age of the patients was 56.65 ± 11.01 years. Ticagrelor was stopped in 76.7% on follow-up. It was stopped in 6.3%, 13.5%, 13.1%, 21.9%, and 45.1% of patients during the first month but after discharge, between first and third months, between 3 and 6 months, between 6 and 12 months, and after 12 months, respectively. In the majority of patients, ticagrelor was replaced by clopidogrel (97.9%). It was stopped according to the physician’s discretion in 79.3% of patients, whereas it was the cost of the drug that made the patient to get swapped to another agent in 18.6%. No difference in the primary composite outcome was observed between the groups where ticagrelor was continued post 12 months and ticagrelor was continued and ticagrelor was switched-over to another agent. Similarly, no difference in death, recurrent myocardial infarctions, re-interventions, or major bleeding manifestations was observed between the two groups.ConclusionIn patients with acute coronary syndrome who undergo PCI, we observed that early discontinuation of ticagrelor and switching over to other P2Y12 inhibitors after discharge did not affect clinical outcomes.

15260 Dupilumab treatment in adult patients with moderate to severe atopic dermatitis: A real-world single-center experience from Turkey

Background: Dupilumab has been shown to be an effective and safe treatment alternative for recalcitrant atopic dermatitis (AD) in adults.

Management of Patients with Aggressive B Cell Non-Hodgkin Lymphoma after Relapse from Axicabtagene Ciloleucel: Single Center Real-World Experience

Background: Chimeric Antigen Receptor (CAR) T-cell therapy has changed the treatment landscape for patients with Non-Hodgkin Lymphoma (NHL). Despite the excellent responses in relapsed or refractory (R/R) aggressive NHL (aNHL), the outcome of patients (pts) that fail CAR T-cell therapy remains poor, and there is not a clear path for management of their disease. Methods: We conducted a retrospective analysis of aNHL pts treated with axicabtagene ciloleucel (axi-cel) at the Mayo Clinic campuses in Arizona and Florida between June 2018 and August 2020. We evaluated the predisposing factors, management, toxicities, and response after CAR T-cell therapy failure. Statistical calculations using parametric tests were performed, and survival curves were estimated using the Kaplan-Meier method and compared statistically using the log-rank test and Pearson's correlation. Results: Thirty-four pts with aNHL received axi-cel. The median age was 53 years [IQR 42-63], and 62% were male. All pts received inpatient axi-cel infusions and the median length of hospital stay was 14 days (IQR: 11-17). Cytokine Release Syndrome (CRS) was observed in 91% of pts (3% grade ≥3), while Immune Effector Cell Associated Neurotoxicity Syndrome (ICANS) was observed in 41% (24% grade ≥3). At day 30 response assessment, 16 pts (47%) had complete responses (CR), 9 (26%) had a partial response (PR), 4 (12%) had stable disease (SD), 4 (12%) showed progression with primary refractory disease (PD) and 1 (3%) died before assessment due to grade 5 ICANS (Table 1). After a median follow-up of 178 days, we observed PD in 12 (35%) pts. The median time-to-progression was 72 days (IQR 58-93) and most of the pts (83%) progressed during the first 3 months. None of the patients with more than 5 months of sustained response developed progression of disease. The likelihood of progression during the first 6 months after axi-cel infusion was 19%, 57%, 50% for pts that initially achieved a CR, PR, SD, respectively. Expression of CD19 was observed in 66% (4/6) of pts with available biopsies after axi-cel suggesting a failure mechanism other than antigen escape. The mortality rate of the R/R aNHL group was 58% with a median survival time of 83 days (IQR: 50-109). There was no association between age, stage, number of previous therapies, time from previous therapy to axi-cel infusion, time from apheresis to infusion, use of tocilizumab, or steroids with progression of disease. Of note, no correlation between CRS or ICANS with progression of disease was found (2-way ANOVA test F (1, 4) = 3.802, p=0.1230). Maintaining a response to axi-cel treatment (CR, PR, or SD) for ≥ 3 months was a strong predictor of durable response with an HR of 0.05 (p= &amp;lt;0.0001). Eleven R/R pts received subsequent therapies with a median time to retreatment of 76 days. Those treatments included: Radiotherapy (n=7), pembrolizumab (n=3), polatuzumab-rituximab with (n=3) and without (n=1) bendamustine, obinutuzumab with (n=1) or without (n=1) lenalidomide, Hyper-CVAD (n=2), R-GemOx (n=1), rituximab with lenalidomide (n=1) and intrathecal methotrexate (n=1). Only 2 (17%) patients have responded to salvage therapy achieving PR (one patient treated with radiotherapy and the other with rituximab-lenalidomide after two other salvage therapies). Conclusion: Our experience demonstrates the majority of aNHL patients respond to axi-cel. If patients maintain their response for more than 3 months, the likelihood of progression is very low - 15%. Similar to what has been previously reported in the literature, our series showed that 35% of patients progressed after axi-cel, and subsequently have a poor prognosis with median survival after a relapse of only 83 days (IQR: 50-109). Therapy options following axi-cel were limited due to severe cytopenias, only 2 of 11 patients have responded to salvage therapy, suggesting that conventional treatments are probably not effective/safe in this high-risk group of patients. Interestingly, the majority of R/R pts with available biopsies showed persistent CD19 expression suggesting that CAR T-cell exhaustion, poor in vivo expansion, and inhibitory signals of the tumor microenvironment may contribute to resistance. Additional strategies for monitoring of axi-cel persistence and its immunophenotypic profile could be helpful for prognosis and management of CAR T-cell pts receiving axi-cel. Disclosures Kharfan-Dabaja: Daiichi Sankyo: Consultancy; Pharmacyclics: Consultancy. Castro:Fate Therapeutics: Research Funding; Kite Pharma: Research Funding; Pharmacyclics: Research Funding.

Outcomes of FLT3-Mutated Acute Myeloid Leukemia Patients: A Single Center Real-World Experience

Background:Internal tandem duplications (ITDs) within the FMS-like tyrosine kinase 3 (FLT3) gene (FLT3-ITD) is a common driver mutation and confers a poor prognosis in patients (pts) with acute myeloid leukemia (AML) due to proliferative presentation, short remission durations and early relapse. The addition of midostaurin to induction therapy has become standard therapy in newly diagnosedFLT3-mutated (FLT3-mu) AML. Next-generation FLT3 inhibitors (FLT3i) as gilteritinib and quizartinib are more specific, more potent, and may have fewer toxicities associated with off-target therapies but are only available in clinical trials or for salvage therapy. Real-world experience in smaller academic institutions is scanty. Methods:We reviewedFLT3-ITD mu AML pts at our institution from October 2016-December 2019. FLT3i were given as a single agent or in combination with cytotoxic chemotherapy (CCT) or low intensity therapy (LIT) (hypomethylating agents). AllFLT3-ITD DNA was subjected to PCR amplification using primers targeting the juxtamembrane domain of FLT3, and products were analyzed by capillary electrophoresis. We analyzed the characteristics of these pts, responses to therapy, and outcomes. Results:Sixteen pts with newly diagnosedFLT3-ITD mu AML were identified. The median age was 47 years [range, 17-76 years] and the median follow-up was 9.3 months [range, 1.2-29.5 months]. The median time from AML diagnoses to FLT3 detection was 4 days [range, 3-5 days]. Patient characteristics are shown in Table 1. The most common co-occurring mutations wereNPM1(44%) andNRAS(31%); 2 pts (12%) had both mutations. Five pts (31%) received CCT and 11 pts (69%) received CCT+FLT3i. The most frequently used FLT3i was midostaurin 25 mg twice daily on days 8 through 21 (14 days) (10 pts, 63% of all pts); 1 pt received sorafenib (6% of all pts). The complete remission rate (CR) and the overall response rate (ORR) (CR+CR with incomplete hematologic recovery [CRi]) were 31% and 88%, respectively. The two pts who did not achieve a remission were older than 65 years. Among the pts who received CCT+FLT3i, CR and OOR were 50% and 100%, respectively. The median number of cycles to remission was 1 [range, 1-2 cycles], and the median number of consolidation cycles was 0 [range, 0-3 cycles]. Overall, the median duration of response in frontlineFLT3-ITD pts who did not receive allogeneic hematopoietic stem cell transplantation (Allo-HSCT) was 5 months [range, 1-9 months] (all of them received CCT+FLT3i). There was no statistically significant difference in response in pts with NPM1 mutation compared with pts with NRAS mutation (P=0.7), or with no additional mutations. Seven pts (44%) received an Allo-HSCT (6 after CCT+FT3i, and 1 after CCT alone): 2 (28%) from matched unrelated donor, 1 (14%) from a haploidentical transplant, 1 (14%) from dual cord transplant, and the remaining (44%) from matched related donors. Two patients relapsed after Allo-HSCT (28%). Both of these patients had received FLT3i before Allo-HSCT; one of the two relapsed pts received ivosidenib and azacitidine (IDH1 mutation) after Allo-HSCT, achieved remission then received the 2nd Allo-HSCT with subsequent remission. Two pts (29%) received maintenance therapy after Allo-HSCT, one with midostaurin, and the other with azacitidine + gilteritinib; both pts are still on remission (24 and 6 months after CR, respectively). All 7FLT3-ITD pts who did not receive Allo-HSCT relapsed. Gilteritinib was used as a single agent (n=3) or in combination with LIT (n=1) in these pts after relapse. CR rate was 25% and CRI 50%; one pt relapsed (33%). All pts who responded are still receiving gilteritinib. Conclusion:Combination of FLT3i based therapy with CCT is effective therapy inFLT3-ITD frontline pts in real-world setting. Duration of response is short in the absence of Allo-HSCT. Disclosures Cortes: Arog:Research Funding;Pfizer:Consultancy, Research Funding;Amphivena Therapeutics:Research Funding;Daiichi Sankyo:Consultancy, Research Funding;Astellas:Research Funding;Telios:Research Funding;Novartis:Consultancy, Research Funding;BioPath Holdings:Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding;Merus:Research Funding;Bristol-Myers Squibb:Research Funding;Takeda:Consultancy, Research Funding;Sun Pharma:Research Funding;BiolineRx:Consultancy, Research Funding;Immunogen:Research Funding;Jazz Pharmaceuticals:Consultancy, Research Funding.

Prognostic Value of PET/CT Performed Day +100 Post Autologous Stem Cell Transplantation in Multiple Myeloma: Real-World Single Center Experience

Background: Evaluation of minimal residual disease (MRD) in multiple myeloma (MM) has become a standard procedure and has been incorporated to International Myeloma Working Group updated response criteria in 2016. Its value as an important prognostic factor with impact on survival as well as a relevant end-point in clinical trials has been established by many studies. Assessment of MRD inside the bone marrow is performed basically by two standardized techniques: i) next generation flow cytometry (NGF) and ii) next generation sequencing (NGS). Positron emission tomography and computed tomography (PET/CT) is currently considered as a best tool to evaluate MRD outside of the bone marrow. The standard timepoint to assess the MRD by PET/CT in transplant eligible newly diagnosed multiple myeloma (TE NDMM) patients is considered Day +100 post-autologous stem cell transplantation (ASCT) according to largest CASSIOPET study (Moreau et al., Blood, 2019). Nevertheless, this kind of data from real-world outside the clinical trials setting remains limited. Aim: To evaluate the prognostic impact of PET/CT performed on Day +100 post-ASCT in consecutive cohort of real-world TE NDMM patients from a single center in the Czech Republic. Methods: 18 TE NDMM patients with median age of 59 years (range 42 - 71 years), ISS stage III 17% (3/18) diagnosed between September 2017 and June 2019 received proteasome inhibitor plus immunomodulatory drug containing induction. Patients who reached at least very good partial response (VGPR) after ASCT were indicated for MRD evaluation using NGF technique according to standardized EuroFlow protocol with sensitivity to 10e-6 and for PET/CT evaluation - both at Day +100 post-ASCT. For the evaluation of PET/CT scans complex evaluation by nuclear medicine specialist based on Italian Myeloma criteria for PET Use (IMPeTUs) criteria were used (IMPeTUs) criteria were used (Nanni et al., Eur. J. Nucl. Med. Mol. Imaging. 2018). Results: Of 18 evaluated TE NDMM patients (17 in complete remission (CR), 1 in VGPR), there were 78% (14/18) considered PET/CT negative and 22% (4/18) were considered PET/CT positive at Day +100 post-ASCT. Patients who were PET/CT positive had significantly shorter median PFS (mPFS 29 days) versus those who were PET/CT negative (mPFS not reached); p value &amp;lt; 0.001. Calculated median follow-up for the whole cohort was 245 days. Interestingly, one patient who was NGF MRD negative within the bone marrow was considered PET/CT positive and has early relapse after ASCT. Conclusion: We demonstrated on limited number of real-world TE NDMM patients that those who are PET/CT positive at Day +100 post-ASCT have significantly shorter mPFS compared to those who are PET/CT negative. The aim of myeloma community should lead to the implementation of simultaneous assessment of MRD inside the bone marrow in combination with imaging technique (PET/CT) outside the bone marrow, as this combined evaluation seems to be the most important prognostic factor. Moreover, great effort should be make to establish standardized evaluation protocols for nuclear medicine specialists as well as the most appropriate time-points to perform PET/CT, such as Day +100 post-ASCT in TE NDMM. Figure Disclosures No relevant conflicts of interest to declare.

Efficacy and Safety of Direct-Acting Antiviral Therapy in Patients With Chronic Hepatitis C Virus Infection: A Real-World Single-Center Experience in Tianjin, China.

ObjectiveToward the limited real-world data concerning the treatment response to brand direct-acting antiviral agents (DAAs) therapy, we proposed to evaluate the efficacy and safety of DAAs for the treatment of chronic hepatitis C virus (HCV) in mainland China.MethodsIn this retrospective, single-center, cohort study, all HCV-infected adult patients treated with brand DAA drugs covered by Tianjin local health insurance (Apr 2018–Sept 2019) and responding to other specific inclusion criteria were recruited. The five available DAA regimens included sofosbuvir + ribavirin (SOF + RBV), elbasvir/grazoprevir (EBR/GZR), ombitasvir/paritaprevir/ritonavir/dasabuvir (OBV/PTV/r/DSV) ± RBV, daclatasvir + asunaprevir (DCV + ASV), and SOF + DCV ± RBV. Demographic, virologic, clinical, and adverse effects data obtained during and after DAAs treatment were collected. We evaluated the rate of sustained virological response at 12 weeks post-treatment (SVR12), the incidence of adverse effects, and assessed the factors associated with SVR12.ResultsFour hundred ninety-four patients finished the treatment and completed the 12-week post-treatment follow-up. The overall SVR12 rate was estimated at 96.96%. SVR rates greater than 95% were achieved in most of the HCV genotypes with the exception of GT1a (0%), GT3a (93.33%), and GT3b (88.24%). SVR12 for patients treated with DCV + ASV, EBR/GZR, OBV/PTV/r/DSV ± RBV, SOF + DCV ± RBV, and SOF + RBV for 12 or 24 weeks was 86.67%, 100%, 98.11%, 97.56%, and 95.06%, respectively. Subjects with compensated cirrhosis (92.73%) and prior treatment experience (77.78%) had significantly lower SVR rates when compared to chronic hepatitis C (98.15%) and treatment-naive (97.69%) groups. In Tianjin, the available DAA regimens were generally well-tolerated, and not a single serious adverse event was reported.ConclusionIn this large real-life single-center HCV cohort from China, oral DAAs were highly effective and well-tolerated. Further and larger-scale studies are needed to evaluate their clinical safety and efficacy.

Cardiovascular Toxicity in Cancer Patients Treated with Tyrosine Kinase Inhibitors: A Real-World Single-Center Experience

Background: Target therapy can cause various cardiovascular complications. The aim of this study was to evaluate the burden of cardiovascular complications related to treatment with anti-BCR-ABL tyrosine kinase inhibitors (TKIs) and to determine if there are differences between the latest- and first-generation TKIs. Methods: A retrospective observational study was carried out on 55 patients (39 men, 16 women; mean age ± SD: 58 ± 11 years) treated with TKIs targeting Bcr-Abl for a median period of 3.5 years. Patients were divided in two groups according to the type of treatment. Group A included patients treated with latest-generation TKI (nilotinib, dasatinib, and ponatinib), while group B included patients treated with first-generation TKI (imatinib). Cardiological evaluation included electrocardiogram, echocardiogram with global longitudinal strain of left ventricle (GLS), and carotid ultrasound scan with arterial stiffness measurement (pulse wave velocity, PWV). Adverse cardiovascular events were recorded in both groups. Results: Statistical analysis showed that cardiovascular adverse events (myocardial ischemia, peripheral artery disease, deep vein thrombosis, and pleural effusion) were significantly more frequent in group A than group B (p value = 0.044). Moreover, there was a significant reduction in GLS and PWV in group A when compared to group B (respectively, p = 0.03 and p = 0.004). Conclusions: Our study confirms that imatinib is a relatively safe drug, while it reveals that the latest-generation TKIs may cause a burden of cardiovascular complications. GLS and PWV allow detection of early signs of cardiac and vascular toxicity in oncohematologic patients treated with TKI, and their use is advisable.