Real-world Clinical Effectiveness Research Articles

Real-world clinical effectiveness of rituximab rescue therapy in patients with progressive rheumatoid arthritis-related interstitial lung disease

ObjectiveTo assess the real-world, long-term effectiveness of rituximab (RTX) as a rescue therapy in patients with progressive rheumatoid arthritis-related interstitial lung disease (RA-ILD) in whom more conventional therapy has failed. MethodsLongitudinal retrospective observational study of a cohort of patients with RA-ILD that started treatment with RTX due to ongoing progressive ILD despite treatment with glucocorticoids and csDMARDs or immunosuppressants (IS). All patients were treated with two or more cycles of RTX and evaluated for at least 12 months. Ongoing therapy with csDMARDs or IS remained unchanged. ResultsThirty-one patients were analyzed. Before initiation of RTX the mean decline (delta) in %pFVC and %pDLCO from the ILD diagnosis (median 21 months) was -16.5% and -19.7%, respectively. After 1 year of treatment, RTX was able to reverse the decline of pulmonary function test (PFTs) parameters: ∆%pFVC +8.06% compared to baseline (95% CI: -10.9 to -5.2; p<0.001) and ∆%pDLCO +12.7% (95% CI: -16.3 to -9.1; p<0.001). In addition, there was a significant reduction in the median dose of prednisone, and it could be suspended in 26% of cases. Dividing the population into UIP and non-UIP patterns, we observed a significant increase in PFTs parameters in both groups. In the 25 patients (80.6%) that completed 2 years of treatment, the statistically significant amelioration in PFTs parameters observed at one year was maintained: ∆%pFVC +11.2% (95% CI: -15.6 to -6.8; p<0.001) and ∆%pDLCO +14.8% (95% CI: -19.3 to -10.3; p<0.001). At the end of the follow-up period (median 32 months; IQR 25th–75th 26–64), only 23 of the 31 patients (74.2%) were still undergoing treatment with RTX: in 3 cases (10%) it was stopped due to adverse events, in another 3 (10%) treatment failed ultimately requiring a lung transplant, and 2 patients (6%) died due to progression of the ILD and infectious complications. The frequency of adverse events reached 32% of cases. ConclusionBased on our results, RTX appears to be effective as rescue therapy in a considerable proportion of patients with progressive RA-ILD unresponsive to conventional treatment.

Real-world clinical effectiveness and safety of vedolizumab and anti-tumor necrosis factor alpha treatment in ulcerative colitis and Crohn\u2019s disease patients: a German retrospective chart review

BackgroundReal-world comparisons of biologic treatment outcomes for ulcerative colitis (UC) or Crohn’s disease (CD) patients are limited. We sought to evaluate the real-world effectiveness of vedolizumab (VDZ) and anti-tumor necrosis factor alpha (anti-TNFα) in UC and CD patients in Germany.MethodsA retrospective chart review (15 sites) investigated UC and CD patients who were biologic-treatment naïve (biologic-naïve) or had received no more than one prior anti-TNFα before initiating treatment with VDZ or anti-TNFα between 15 July 2014 and 20 October 2015. Kaplan-Meier analyses assessed time to first chart-documented clinical remission (CR) and symptom resolution (UC: rectal bleeding [RB], stool frequency [SF]; CD: abdominal pain [AP], liquid stools [LS]) and outcome duration.ResultsA total of 133 UC (76 VDZ; 57 anti-TNFα) and 174 CD (69 VDZ; 105 anti-TNFα) patients were included. By Week 26, estimated cumulative rates of patients achieving CR or symptom resolution with VDZ vs anti-TNFα treatment were for UC: CR, 53.7% vs 31.7%; RB, 66.8% vs 55.8%; and SF, 59.8% vs 50.7%, respectively; and for CD: CR, 14.4% vs 32.8%; AP, 62.5% vs 56.0%; and LS, 29.9% vs 50.3%, respectively. Outcomes were sustained similarly between treatments, except RB (VDZ vs anti-TNFα: median 38.1 vs 15.1 weeks, P = 0.03). Treatment-related adverse events occurred in 5.3% vs 7.0% (UC) and 8.7% vs 19.0% (CD) of VDZ vs anti-TNFα patients, respectively.ConclusionsAlthough there were differences in CR, symptom resolution, and safety profiles, real-world data support both VDZ and anti-TNFα as effective treatment options in UC and CD.

721 - Real-world clinical effectiveness of second-line sunitinib following immuno-oncology therapy in patients with metastatic renal cell carcinoma

721 - Real-world clinical effectiveness of second-line sunitinib following immuno-oncology therapy in patients with metastatic renal cell carcinoma

One-year real-world clinical effectiveness, safety, and laboratory safety of dupilumab in Japanese adult patients with atopic dermatitis: A single-center retrospective study

One-year real-world clinical effectiveness, safety, and laboratory safety of dupilumab in Japanese adult patients with atopic dermatitis: A single-center retrospective study

Real-world clinical effectiveness and safety of olaparib monotherapy in HER2-negative gBRCA-mutated metastatic breast cancer: Phase IIIb LUCY interim analysis.

1087 Background: OlympiAD (NCT02000622) demonstrated the benefit of olaparib over standard of care in patients (pts) with HER2-negative (HER2-) metastatic breast cancer (MBC) and germline BRCA mutations (gBRCAm). LUCY (NCT03286842) aimed to provide additional data on the real-world effectiveness and safety of olaparib monotherapy in this setting. Methods: This Phase IIIb, open-label, single-arm study of olaparib 300 mg twice-daily, enrolled pts with HER2- gBRCAm MBC who had received a taxane and/or anthracycline in the (neo)adjuvant/metastatic setting, and ≤2 lines of chemotherapy for MBC. Hormone receptor-positive (HR+) pts had progressed on prior endocrine therapy (ET), and further ET was considered unsuitable. Primary endpoint: investigator-defined progression-free survival (PFS). Secondary endpoints: overall survival, time to first subsequent therapy or death (TFST), and investigator-assessed clinical response rate (CRR). The interim analysis was planned after 160 PFS events. Results: From Oct 2018-Sept 2019, 252 pts were enrolled (160 sites, 15 countries; mean age 46.2 [range 22-75] years; 73.4% ECOG PS 0). Median total treatment duration: 7.9 months (mo; range 0.2-20.0). Median PFS: 8.1 mo (95% confidence interval [CI] 6.9, 8.7; 166 events [65.9%]). Clinical trial information: NCT03286842 . Median TFST: 9.7 mo (95% CI 8.7, 11.1). CRR: 48.6% (95% CI 42.2, 55.0). Adverse events (AEs) in &gt;20% of pts (all grades): nausea, anemia, asthenia, vomiting, and fatigue. 24.6% of pts reported a grade ≥3 AE, including anemia (n=33 [13.1%]). 4.4% of pts had an AE leading to treatment discontinuation. Conclusions: Interim results in this real-world population of pts with HER2- gBRCAm MBC were consistent with the OlympiAD study, and support olaparib as a chemotherapy-free alternative treatment for pts with gBRCAm advanced BC. [Table: see text]

Improved Clinical Outcomes with Dulaglutide as Add-on Medication to Oral Antidiabetic Drugs with or Without Insulin in Overweight Indian Patients with Type 2 Diabetes Mellitus: Retrospective Study in a Real-World Setting.

Glucagon-like peptide-1 receptor agonists (GLP-1 RAs) have demonstrated several extra-pancreatic benefits in addition to glycemic control. This study retrospectively evaluates the realworld clinical effectiveness of dulaglutide as add-on therapy in overweight patients with inadequately controlled type 2 diabetes mellitus (T2DM). This single-center study included overweight adult patients (N, 85; women, 45) with inadequately controlled T2DM (mean glycated hemoglobin [HbA1c] (standard deviation [SD]), 7.55 [0.43] %; and body mass index [BMI] [SD], 29.01 [2.30] kg/m2) treated with dulaglutide (1.5 mg) once weekly as an add-on therapy. Follow-up improvements in outcomes were analyzed using the paired t-test. Subgroup analysis was performed for selected outcomes. Safety parameters were also evaluated. At the 20-week follow-up, dulaglutide based therapy demonstrated a significant reduction (P<.001) in HbA1c, body weight and BMI, with a mean reduction (MR [SD]) of 0.45 [0.38] %, 5.06 [2.33] kg, and 1.82 [0.81] kg/m2, respectively, in the overall population. Similarly, reduction in urine albumin/creatinine ratio [U-ACR] (6.04 [15.53] mg/g), cholesterol (3.24 [4.14] mg/dL), triglycerides (16.60 [12.39] mg/dL), very-low-density lipoprotein [VLDL] (3.31 [2.48] mg/dL), serum glutamicoxaloacetic transaminase (1.80 [2.92] U/L) and glutamic-pyruvic transaminase (8.00 [5.64] U/L) was also significant (P<.05). Target HbA1c of <7% was achieved in 40% of patients. Reduction in HbA1c and body weight was significant across all subgroups analyzed. Predominantly, gastrointestinal adverse events were reported. Dulaglutide as an add-on therapy was well tolerated with significant improvement in HbA1c, body weight, BMI, U-ACR, lipid fractions and serum transaminases in overweight Indian patients with T2DM.

DOP69 Tofacitinib in ulcerative colitis: Early ‘real-world’ experience from four UK tertiary centres

Abstract Background Tofacitinib is a partially selective Janus kinase inhibitor that was approved for the treatment of refractory moderate to severe ulcerative colitis (UC) in 2018. We report the real-world clinical effectiveness and adverse effects of tofacitinib in UC. Methods We conducted a retrospective observational cohort study of tofacitinib-treated patients with UC between October 2018 to October 2019 from 4 UK centres. Disease activity was assessed using the Simple Clinical Colitis Activity Index (SCCAI) or Partial Mayo Score (PMS) depending on the study site. Response and remission were defined at week 8 and 26 as a reduction in SCCAI or PMS of ≥3, and SCCAI &amp;lt;3 or PMS &amp;lt;2, respectively. Corticosteroid-free remission was defined as remission with no corticosteroid use at the time of assessment irrespective of baseline corticosteroid status. Results We included 140 patients (65% M; median age 37y [range 16–81]) with a median disease duration of 5.5y (IQR 2.2–11.8). Forty-six per cent (65/140) were receiving corticosteroids at baseline and 83% (116/140) had previously received at least one biologic (62 anti-TNF, 4 vedolizumab, 50 both). Median (IQR) serum CRP and faecal calprotectin levels at baseline were 4 mg/l (1.6–15) and 540 µg/g (316–1175). Response and remission rates were 73% (81/111) and 56% (62/111) respectively at week 8 (median ΔSCCAI of −3 [IQR -6 to -1], median ΔPMS −4 [−6 to −1]) and 48% (39/82) and 39% (32/82) respectively at week 26 (median ΔSCCAI −3 [IQR −7 to –1], median ΔPMS −4 [−6 to –1]). Steroid-free remission was seen in 47% (52/111) and 37% (30/82) patients at week 8 and 26. Patients with response or remission had a significantly lower CRP (p = 0.02) but not calprotectin (p = 0.38) levels at baseline. Response and remission rates were no different stratified by prior biologic use (p = 0.56). Treatment was discontinued after a median of 3 months (IQR 2–4) in 43 patients: 32 with primary non-response, 9 loss of response and in 1 each because of an adverse drug reaction (headache) and patient choice. 7/17 patients had a clinical response to dose re-escalation following a loss of response on dose reduction. The median time to dose de-escalation was 67 (IQR 25–240) days. Seven patients were hospitalised and 5 underwent colectomy. Six serious infections were noted including 2 herpes zoster infections but there were no venous thromboembolic events. Median total cholesterol, low-density lipoprotein and high-density lipoprotein increased from 4.4 mmol/l (IQR 3.7–5.2), 2.47 (1.9–2.9) and 1.5 (1.1–1.9) to 4.8 mmol/l (4.1–6.0), 2.8 (2.13.5) and 1.7 (1.4–1.9) respectively after 8 weeks of tofacitinib. Conclusion Clinical effectiveness and side-effect profile of tofacitinib in UC in this multi-centre real-world cohort were similar to that reported in the pivotal OCTAVE clinical trials.

A Multicentre, Retrospective Observational Study to Evaluate the Clinical Outcomes of Patients with Chronic Lymphocytic Leukaemia (CLL) Treated with Idelalisib and Rituximab in the UK (RETRO-idel) - a Cohort of 110 Patients

Background: B-cell receptor signalling pathway plays a key role in CLL pathogenesis. Overexpression of phosphatidylinositol 3-kinase delta isoform (PI3Kδ) is a characteristic of malignant lymphoid cells {Zhang 2014}. Idelalisib is an oral, selective PI3Kδ inhibitor approved for the treatment of CLL {Brown 2014}. Since its approval there has been a paucity of real world clinical effectiveness and safety data on idelalisib-rituximab (IDELA-R). A large U.S.-based national, retrospective series of 682 CLL patients (pts) explored the optimal sequence of targeted agents in this setting, but included only 62 (9%) pts treated with IDELA-R {Mato 2017}. Aims: To evaluate the effectiveness of IDELA-R in pts with CLL in routine clinical practice in the UK through a protocol-led, retrospective cohort study; RETRO-idel (NCT03582098). Methods: Secondary or tertiary care centres (sites) across the UK were invited to participate. Data were collected retrospectively from sites by study personnel from source data-verified medical records using electronic case report forms (eCRFs). Eligible pts included those who initiated IDELA-R as part of routine clinical management of CLL from Sep 2014 (following European approval), up to and including 31 Dec 2017. Primary endpoint was overall response rate (ORR) defined as the proportion of pts who achieved an objective clinical response after the initiation of IDELA-R treatment. Secondary endpoints included overall survival (OS), progression free survival (PFS), time to next treatment (TTNT), duration of response (DOR), and safety (including serious adverse events [SAE] and AEs of special interest [AESI]). Results: A total of 110 pts were included in the study from 16 UK sites, of whom 74 (67.3%) were males and the median age at IDELA-R initiation was 72 years (range 48 - 90) (Fig 1a). Median number of prior therapies was 1 (range 0 - 8; 23.6% 1st line). Seven (6.4%) pts had received prior ibrutinib. Median time since diagnosis was 7 years (range: 0 - 23), and 54 (49.1%) pts tested had TP53 mutated / del (17p) CLL. Median duration of follow-up was 39.1 months (range 0.1 - 51.9), with 65 (59.1%) pts followed &gt;2 years. Ninety-two (83.6%) pts received pneumocystis jirovecii pneumonia (PJP) prophylaxis and 8 (7.3%) pts had received a stem-cell transplant (SCT) [(before IDELA-R; 2 (1.8%) alloSCT; 2 (1.8%) autoSCT); after IDELA-R; 4 (3.6%) alloSCT)]. The median duration of therapy with IDELA was 12 months (range 0 - 41). The ORR was 88.2% (95% CI: 82.2 - 94.2) and median DOR 32.7 months (95% CI 19.7 - NR). Seven (6.4%) pts had no documented response and response data were missing for 6 (5.4%) pts. Median OS was not reached (95% CI: 31.5 - NR) and median PFS was 29.5 months (95% CI: 22.1, NR) (Fig 1b, c). Median TTNT was 29.2 months 95% CI; 25.3 - 42.8). Overall, 108 (98.2%) pts experienced an SAE or AESI. Diarrhoea was reported in 34 (30.9%) pts, followed by lower respiratory tract infection [LRTI] in 24 (21.8%), neutropenia in 23 (20.9%), rash in 18 (16.4%) and pneumonia in 18 (16.4%). Ten (9.1%) pts had colitis, of which Gr 3/4 colitis occurred in 6 (5.5%). A total of 71 (64.5%) pts experienced a Gr 3/4 AEs including pneumonia in 14 (12.7%), LRTI in 10 (9.1%), neutropenia in 7 (6.4%), diarrhoea in 7 (6.4%) and neutropenic sepsis in 7 (6.4%). There were 85 (77.2%) discontinuations; 54 (56.3%) due to toxicity, 18 (18.8%) due to PD, 11 (11.5%) by investigator discretion, and 2 (2.1%) by subject decision. There were 11 (11.5%) deaths reported whilst pts were receiving IDELA-R. Richter's transformation was documented in 1 (0.9%) pt. At the time of analysis, 46 (41.8 %) pts had died. Conclusions: We report to our knowledge the largest cohort of CLL pts treated with IDELA-R outside of the clinical trial setting. Our findings corroborate those from interventional studies of IDELA {Furman 2014, Sharman 2019}. Longer PFS observed in our study versus prospective trials probably reflects the relatively fewer median number of prior therapies. Median TTNT was slightly shorter than median PFS possibly due to certain pts discontinuing IDELA-R without PD and continuing to a subsequent therapy. Safety profile of IDELA-R was consistent with trial experience, and no new safety signals were identified. IDELA-R remains an effective treatment option for CLL pts. Analyses assessing a) response duration and TTNT in pts stopping therapy due toxicity and b) predictors of PFS (univariable and multivariable analysis) are ongoing and will be presented. Disclosures Eyre: Janssen: Honoraria; Roche: Honoraria; Gilead: Consultancy, Honoraria, Other: commercial research support; Abbvie: Honoraria. Islam:Gilead: Honoraria, Other: Conference attendance sponosrship. Nicholson:Pfizer: Other: Conference attendance sponosrship; Takeda: Other: Conference attendance sponsorship. Fegan:Gilead: Honoraria; Roche: Honoraria; Janssen: Honoraria; Abbvie: Consultancy, Other: Conference attendance sponsorship. Smith:Gilead: Employment, Equity Ownership. Cursley:Gilead: Employment. Ramroth:Gilead Sciences: Employment. Rajakumaraswamy:Gilead Sciences, Inc.: Employment.

Real-world Clinical Effectiveness of Whole Genome and Transcriptome Analysis to Guide Advanced Cancer Care

Real-world Clinical Effectiveness of Whole Genome and Transcriptome Analysis to Guide Advanced Cancer Care

Real-World Clinical Effectiveness and Tolerability of Hydroxychloroquine 400 Mg in Uncontrolled Type 2 Diabetes Subjects who are not Willing to Initiate Insulin Therapy (HYQ-Real-World Study).

The epidemic of T2DM is rising across the globe. Systemic inflammation plays a pivotal role in the pathogenesis and complications of T2DM. Combination of two or more oral hypoglycemic agents (OHA) is widely prescribed in patients with T2DM, however many patients have poor glycemic control despite receiving combination therapy. The new antidiabetic drugs are relatively costly or many patients have anxiety over the use of injectable insulin. The objective of this observational study was to investigate the effectiveness and tolerability of hydroxychloroquine (HCQ) in T2DM patients uncontrolled on multiple OHA and despite high sugar level not willing to initiate insulin therapy in a real-world clinical setting. A prospective, investigator-initiated, observational, single-centred study was conducted where 250 patients (18-65 years) with T2DM for more than 5 years, with uncontrolled glycemia despite on a combination of multiple OHA, HbA1c between ≥7% and <10.5%, FPG >130 mg/dL or PPG >180 mg/dL and BMI between >25 and <39 kg/m2, were prescribed hydroxychloroquine sulphate 400 mg once daily for 48 weeks. Percentage of drugs used at the baseline were as follows: metformin 2000 mg (100%), glimepiride 4 mg (100%), pioglitazone 30 mg (100%), sitagliptin 100 mg (100%), canagliflozin 300 mg (52.4%), empagliflozin 25 mg (22.8%), dapagliflozin 10 mg (17.6%) and voglibose 0.3 mg (62%). Mean change in HbA1c, blood glucose and hs-CRP at baseline, week 12, 24 and 48 were assessed using the paired t-test. After 48 weeks of add-on treatment with HCQ, almost all SGLT-2 inhibitors were withdrawn; metformin dose was reduced to 1000 mg, glimepiride reduced to 1 mg and sitagliptin reduced to 50 mg OD. Patients continued to have good glycemic control. HbA1c was reduced from 8.83% to 6.44%. Reduction in FPG was 40.78% (baseline 177.30 mg/dL) and PPG was reduced by 58.95% (baseline 329.86 mg/dL). Change in mean body weight was -4.66 Kg. The reduction in glycemic parameters and mean body weight was significant (p < 0.0001). Hs-CRP was significantly reduced from 2.70±1.98 mg/L to 0.71±0.30 mg/L 9 (p < 0.0001). More reduction in glycemic parameters and body weight was observed among the patients with higher hs-CRP (> 3 mg/L) as compared to patients with baseline hs- CRP ≤ 3 mg/L. Most common adverse events reported with the drug therapy were GI irritation (3.6%) and hypoglycemia (2%). None of the patients required medical assistance for hypoglycemia. Add-on treatment of HCQ effectively improved glycemic control in T2DM patients uncontrolled on multiple antidiabetic drugs. By virtue of its antidiabetic and anti-inflammatory properties, it may emerge as a valuable therapeutic intervention for the patients with T2DM.

338P - Real-world effectiveness of first-line palbociclib + letrozole for metastatic breast cancer 4 years post approval in the US

BackgroundIn Feb 2015, palbociclib plus letrozole (P+L) became the first cyclin-dependent-kinase 4/6 inhibitor approved for mBC/aBC in the US. Until now, long-term outcomes studies have included a limited number of patients receiving P+L. The objective of this study is to describe real-world patient characteristics and clinical effectiveness associated with P+L as initial endocrine-based therapy use 4 years post approval. MethodsAdult postmenopausal women diagnosed with metastatic HR+/HER2- breast cancer who initiated P+L as first-line therapy on or after 2/3/2015 were identified by providers in the Cardinal Health Oncology Provider Extended Network, including 1800 oncologists/hematologists in the US. A subset of providers abstracted data related to patient characteristics and clinical outcomes including disease response and disease progression into an electronic case report from (eCRF). Data were abstracted from the time of initiation of P+L through Feb 2019 (or end of follow up/death). Providers were shown response classification schema corresponding to RECIST v1.1 and asked to indicate the patient’s best response if recorded. The objective response rates (ORR – incl. complete response [CR] and partial response [PR]), clinical benefit rates (CBR – incl. CR+PR+stable disease [SD] >24 wks), and progression-free survival (PFS) rate at 12-month were reported. Each eCRF was reviewed by independent clinical staff; source data was verified on 10% of the patients. Results31 providers submitted 202 eCRFs, of which, 193 were eligible. Median follow-up from 1L P+L initiation was 15.4 months, 45.6% of patients had discontinued P+L at data cut-off. Table shows key patient characteristics and ORR, CBR, and PFS rate at 12-month, each showing benefit of 1L P+L in the real-world. ConclusionsThese real-world data demonstrate the benefit of 1L P+L 4 years post approval in the US and support the clinical benefit of 1L P+L reported in randomized clinical trials.Table338PTableN=193AllMean age at diagnosis, yrs62.7Non-Hispanic white, %74.6Black/African-American, %17.1ECOG-PS 0/1, %89.6N0, %22.8Visceral mets, %51.3Bone only mets, %25.4ORR, %65.8CR9.8PR56.0CBR†, %88.0CR12.7PR61.3SD†14.012-month PFS, %74.5†Excluding 43 patients with duration of treatment <24 wks Legal entity responsible for the studyPfizer Inc. FundingPfizer Inc. DisclosureJ. Kish: Full / Part-time employment: Cardinal Health; Advisory / Consultancy, Paid consultant in connection with the development of this abstract: Pfizer Inc. J. Trocio: Shareholder / Stockholder / Stock options, Full / Part-time employment: Pfizer Inc. T. Miller: Full / Part-time employment: Cardinal Health; Advisory / Consultancy, Paid consultant in connection with the development of this abstract: Pfizer Inc. D. Nero: Full / Part-time employment: Cardinal Health; Advisory / Consultancy, Paid consultant in connection with the development of this abstract: Pfizer Inc. D. Liassou: Full / Part-time employment: Cardinal Health; Advisory / Consultancy, Paid consultant in connection with the development of this abstract: Pfizer Inc. X. Liu: Shareholder / Stockholder / Stock options, Full / Part-time employment: Pfizer Inc. L. McRoy: Shareholder / Stockholder / Stock options, Full / Part-time employment: Pfizer Inc. B. Feinberg: Full / Part-time employment: Cardinal Health; Advisory / Consultancy, Paid consultant in connection with the development of this abstract: Pfizer Inc.

Real-world effectiveness of infection prevention interventions for reducing procedure-related cardiac device infections: Insights from the veterans affairs clinical assessment reporting and tracking program.

To measure the association between receipt of specific infection prevention interventions and procedure-related cardiac implantable electronic device (CIED) infections. Retrospective cohort with manually reviewed infection status. Setting: National, multicenter Veterans Health Administration (VA) cohort. Sampling of procedures entered into the VA Clinical Assessment Reporting and Tracking-Electrophysiology (CART-EP) database from fiscal years 2008 through 2015. A sample of procedures entered into the CART-EP database underwent manual review for occurrence of CIED infection and other clinical/procedural variables. The primary outcome was 6-month incidence of CIED infection. Measures of association were calculated using multivariable generalized estimating equations logistic regression. We identified 101 procedure-related CIED infections among 2,098 procedures (4.8% of reviewed sample). Factors associated with increased odds of infections included (1) wound complications (adjusted odds ratio [aOR], 8.74; 95% confidence interval [CI], 3.16-24.20), (2) revisions including generator changes (aOR, 2.4; 95% CI, 1.59-3.63), (3) an elevated international normalized ratio (INR) &gt;1.5 (aOR, 1.56; 95% CI, 1.12-2.18), and (4) methicillin-resistant Staphylococcus colonization (aOR, 9.56; 95% CI, 1.55-27.77). Clinically effective prevention interventions included preprocedural skin cleaning with chlorhexidine versus other topical agents (aOR, 0.41; 95% CI, 0.22-0.76) and receipt of β-lactam antimicrobial prophylaxis versus vancomycin (aOR, 0.60; 95% CI, 0.37-0.96). The use of mesh pockets and continuation of antimicrobial prophylaxis after skin closure were not associated with reduced infection risk. These findings regarding the real-world clinical effectiveness of different prevention strategies can be applied to the development of evidence-based protocols and infection prevention guidelines specific to the electrophysiology laboratory.

PF314 REAL WORLD CLINICAL EFFECTIVENESS AND SAFETY OF CT-P10 IN PATIENTS WITH DIFFUSE LARGE B-CELL LYMPHOMA: INTERIM RESULTS FROM A EUROPEAN NON-INTERVENTIONAL POST-AUTHORISATION SAFETY STUDY

Background: In February 2017, CT-P10 became the first biosimilar of rituximab to be approved in Europe for treatment of rheumatic diseases and specific blood cancers including non-Hodgkin's lymphoma (NHL). Diffuse large B-cell lymphoma (DLBCL) is the commonest type of NHL, representing an estimated 40–58% of cases (Sant et al, 2010). To date, no studies have investigated the effectiveness or safety of CT-P10 in patients with DLBCL in the real world clinical setting. Aims: To evaluate the real world clinical effectiveness and safety of CT-P10 in patients with DLBCL in Europe. Methods: This non-interventional post-authorisation safety study is in progress in five European countries (France, Germany, Italy, Spain and the United Kingdom [UK]), and involves collection of data from the medical records of consenting adult patients with DLBCL who were treated with CT-P10 as part of their routine clinical care. Effectiveness and safety data are being collected for an observation period of 30 months after the index date (date of CT-P10 initiation) or until death, if sooner. The primary objective is to describe the effectiveness of CT-P10 (overall survival, progression free survival, CT-P10 responses), with secondary objectives to describe the safety profile and CT-P10 treatment pathways. Response to CT-P10 is as documented by the local investigator. Here we report interim results (patients’ baseline characteristics and preliminary response/safety data), based on a data cut on 18 January 2019. Where n is less than the total number of patients, data were missing. Results: The interim analysis includes 151 patients enrolled from sites in the UK and Spain, with a median 8.9 months (interquartile range [IQR] 6.1–12.6) post-index observation at the cut-off. Patients are receiving CT-P10 as a first-line (n = 138, 91%), second-line (n = 9, 6%) or third-line (n = 4, 3%) DLBCL treatment. Ninety-five (63%) patients are male. Other patient characteristics at index: median age 70.9 years (IQR 61.5–76.0) (n = 149); median disease duration 20 days (IQR 10.5–41.0) (n = 147); Eastern Cooperative Oncology Group performance status (n = 98): 0 (n = 45, 46%), 1 (n = 36, 37%), 2 (n = 11, 11%), 3 (n = 4, 4%) or 4 (n = 2, 2%); Ann Arbor stage (n = 77): I (n = 8, 10%), II (n = 9, 11%), III (n = 18, 21%), IV (n = 40, 48%) or other (n = 2, 2%; recorded as ‘at least stage II’). Best responses to CT-P10 (evaluable in n = 128) are shown in Fig. 1. Safety of CT-P10 was evaluable in 128 patients, of whom 118 (92%) experienced adverse events (AEs) during the observation period (732 AEs recorded in total, including infusion-related reactions), 77 (60%) had AEs of grade 3 and above and 11 (9%) discontinued CT-P10 due to AEs.Summary/Conclusion: Early results from this ongoing study suggest that a high proportion of patients with DLBCL treated with CT-P10 in the real world achieve complete or partial responses, similar to the response rates reported previously for reference rituximab (Horvat et al, 2017). Continued recruitment and extended follow-up will enable more comprehensive analyses of CT-P10 effectiveness and safety.

Real-World Clinical Effectiveness of Liraglutide 3.0 mg for Weight Management in Canada.

ObjectiveReal‐world clinical effectiveness of liraglutide 3.0 mg, in combination with diet and exercise, was investigated 4 and 6 months post initiation. Changes in absolute and percent body weight were examined from baseline.MethodsA cohort of liraglutide 3.0 mg initiators in 2015 and 2016 was identified from six Canadian weight‐management clinics. Post initiation values at 4 and 6 months were compared with baseline values using a paired t test.ResultsThe full cohort consisted of 311 participants, with 210 in the ≥ 4‐month persistence group and 167 in the ≥ 6‐month persistence group. Average baseline BMI was 40.7 kg/m2, and weight was 114.8 kg. There was a significant change in body weight 6 and 4 months after initiation of treatment in persistent subjects (≥ 6‐month: −8.0 kg, P < 0.001; ≥ 4‐month: −7.0 kg, P < 0.001) and All Subjects, regardless of persistence (−7.3 kg; P < 0.001). Percentage change in body weight from baseline was −7.1% in the ≥ 6‐month group and −6.3% in the ≥ 4‐month group, and All Subjects lost 6.5% body weight. Of participants in the ≥ 6‐month group, 64.10% and 34.5% lost ≥ 5% and > 10% body weight, respectively.ConclusionsIn a real‐world setting, liraglutide 3.0 mg, when combined with diet and exercise, was associated with clinically meaningful weight loss.

Real-world clinical effectiveness of liraglutide 3.0 mg for weight management in Canada

Real-world clinical effectiveness of liraglutide 3.0 mg for weight management in Canada

SAT-LB031 Empagliflozin as an Add-On Medication to Subjects Uncontrolled on DPP4 Inhibitors and Metformin: Real World Data Supporting the Use of Empagliflozin as a Rational Second Add-On in Indian Type 2 Diabetic Patients

A huge burden of uncontrolled type 2 diabetes mellitus (T2D) prevails in the Indian settings with an estimated prevalence of patients with >7 % HbA1c at 63 %1 and glycaemic control tends to decline over time with monotherapy2. Newer guidelines supported the use of DPP4 inhibitors along with SGLT2 inhibitors as sequential therapy in patients with type 2 diabetes mellitus. This study evaluated the real-world clinical effectiveness of Empagliflozin as second add-on therapy in overweight subjects with inadequately controlled type 2 diabetes mellitus (T2DM). This single-centre study included overweight adult subjects with poorly controlled T2DM on optimal dose of metformin and DPP4 inhibitors, (N, 110) (mean glycated haemoglobin [HbA1c] (standard deviation [SD]), 7.65 [± 0.34] %; and BMI [SD], 28.39 [± 1.87] kg/m2) treated with Empagliflozin (25 mg) as add-on therapy. Follow-up improvements in outcomes were analysed using the paired t-test. Safety parameters were also evaluated. The mean age and weight at baseline were 42 ± 9.6 years and 81.23 ± 5.54 kgs respectively, with a male to female ratio of 1:1. An established cardiovascular disease was present in 18.2 % patients. At the 24-week follow-up, Empagliflozin as add-on therapy demonstrated a significant reduction (P< 0.0001) in HbA1c, body weight and BMI, with a mean reduction (MR [SD]) of 0.46 [0.44-0.48] %, 5.22 [4.92-5.51] kg, and 1.82 [1.72-1.92] kg/m2, respectively, in the overall population. Reduction in urine albumin/creatinine ratio [U-ACR] 14.7 [10.61-18.79] mg/g), SBP of 2.89 mmHg [2.24-3.54], and serum glutamic-pyruvic transaminase (10.95 [9.86-12.03] U/L) were also significant (P<.0.0001). The reduction in serum glutamic-oxaloacetic transaminase (0.76 [0.33-1.2] U/L) too was significant (P=0.0001). The target HbA1c of <7% (53 mmol/mol) was achieved in more subjects (31.8%) with the addition of Empagliflozin. Genital mycotic infection was reported in 9 patients. Empagliflozin as an add-on therapy was well tolerated with significant improvement in HbA1c, body weight, BMI, U-ACR, systolic blood pressure and serum transaminases levels in overweight Indian patients with T2DM. Not received any institutional grant. Saydah SH, Fradkin J, Cowie CC. Poor Control of Risk Factors for Vascular Disease Among Adults With Previously Diagnosed Diabetes. JAMA. 2004;291(3):335-342. doi:10.1001/jama.291.3.335 Turner RC, Cull CA, Frighi V, Holman RR, for the UK Prospective Diabetes Study (UKPDS) Group. Glycemic Control With Diet, Sulfonylurea, Metformin, or Insulin in Patients With Type 2 Diabetes Mellitus: Progressive Requirement for Multiple Therapies (UKPDS 49). JAMA. 1999;281(21):2005-2012. doi:10.1001/jama.281.21.2005 Unless otherwise noted, all abstracts presented at ENDO are embargoed until the date and time of presentation. For oral presentations, the abstracts are embargoed until the session begins. s presented at a news conference are embargoed until the date and time of the news conference. The Endocrine Society reserves the right to lift the embargo on specific abstracts that are selected for promotion prior to or during ENDO.

Abstract #364 Real-World Clinical Effectiveness and Tolerability of Hydroxychloroquine 400mg in T2dm

Abstract #364 Real-World Clinical Effectiveness and Tolerability of Hydroxychloroquine 400mg in T2dm

P491 INSPIRE: design and implementation aspects of a registry of complex perianal fistulas in Crohn’s disease patients treated with darvadstrocel

Perianal fistulas (PAF) are a common presentation of Crohn’s disease (CD), the majority being complex (CPAF).1–4 Existing medical and surgical therapies for CPAF have low long-term success rates.4,5 Darvadstrocel (DVS) is a mesenchymal stem cell (MSC) therapy that demonstrated efficacy and tolerability in patients with CPAF over 52 weeks,4 and is the first MSC therapy centrally approved in Europe for the treatment of CPAF.6 However, real-world and longer-term effectiveness data for DVS are lacking. This abstract describes the development of a DVS registry aiming to establish a framework to capture real-world clinical effectiveness and safety data. (EU PAS Register Number: EUPAS24267). The INSPIRE registry aims to establish a data collection framework to capture real-world data on all patients treated with DVS for CPAF over 36 months. Data on patient selection, concomitant treatment, surgical technique, fistula response, remission and complications will be captured and associations between practice parameters and outcome will be assessed. An observational, multi-national, open-enrolment registry was set up to record all patients treated with DVS for CPAF. Ensuring enrolment of almost 100% of patients treated is required for sufficient longer-term evaluation of DVS and to support value-based reimbursement requests in some countries. The made to order manufacturing process and traceability lends itself to more complete data capture. The goal of this registry is to improve understanding of disease presentation, patient characteristics, treatment patterns and clinical outcomes. The primary outcomes of INSPIRE are clinical response (a reduction in ≥50% of draining PAF) and clinical remission (closure of all treated PAF on physical examination).1,7 Health-related quality of life, patient-reported outcomes and MRI images will be collected. INSPIRE will attempt to enrol ≥1600 patients, establishing the largest CPAF database. Patient engagement will be facilitated using digital solutions where possible. Data will be reviewed by the Steering Committee who will act in an independent advisory capacity to provide scientific oversight and to evaluate the effectiveness and safety of DVS. The first patient was treated with DVS on 21 June 2018. The first site initiation visit was conducted on 31 October 2018. INSPIRE will be the first registry to collect data assessing the effectiveness and safety of DVS in patients with CPAF. A comprehensive set of parameters will be collected and correlated, to better understand appropriate patient selection and surgical approach. Patient-centric features of the DVS registry are continuing to be explored.

Real-World Clinical Effectiveness and Cost Savings of Liraglutide Versus Sitagliptin in Treating Type 2 Diabetes for 1 and 2Years.

IntroductionThis study compared the clinical and economic outcomes of long-term use of liraglutide versus sitagliptin for the treatment of type 2 diabetes (T2DM) in real-world practice in the USA.MethodsWe identified adult patients (≥ 18 years old) with T2DM who initiated liraglutide or sitagliptin in 2010–2014 using a large claims database. Quarterly glycemic control measures and annual healthcare costs were assessed during the 1st and 2nd years of persistent medication use. Their associations with medication use (liraglutide or sitagliptin) were estimated using multivariable regression models adjusted for patient demographic and clinical characteristics.ResultsA total of 3113 patients persistently used liraglutide (N = 493) or sitagliptin (N = 2620) for ≥ 1 year [mean age (standard deviation, SD): 53 (8.5) vs. 56 (9.7) years; 48.3% vs. 62.3% males; both p < 0.05]; 911 (including 113 liraglutide users) were persistent users for ≥ 2 years. During the 1st-year follow-up, liraglutide users (versus sitagliptin users, after adjustment) experienced larger glycated hemoglobin (HbA1c) reductions from baseline (ranging from 0.34%-point in quarter 1 to 0.21%-point in quarter 4); higher likelihoods of obtaining HbA1c reductions of ≥ 1%-points or ≥ 2%-points [odds ratios (ORs) range 1.47–2.04]; and higher likelihoods of reaching HbA1c goals of < 6.5% or < 7% (ORs range 1.51–2.12) (all p < 0.05). Liraglutide users also experienced HbA1c reductions from baseline in the 2nd-year follow-up (0.53–0.33%-point, all p < 0.05). Although liraglutide users incurred higher healthcare costs than sitagliptin users during the 1st-year follow-up, they had $2674 (per patient) lower all-cause medical costs (adjusted cost ratio: 0.67, p < 0.05) and similar total costs (all-cause and diabetes-related) in the 2nd year.ConclusionLong-term use of liraglutide for 1 or 2 years was associated with better glycemic control than using sitagliptin. Savings in medical costs were realized for liraglutide users during the 2nd year of persistent treatment, which offset differences in pharmacy costs.FundingNovo Nordisk Inc.Electronic supplementary materialThe online version of this article (10.1007/s13300-018-0432-2) contains supplementary material, which is available to authorized users.

PSY10 - Real-World Clinical Effectiveness of Liraglutide 3.0 mg for Weight Management in Canada

Real-world clinical effectiveness of liraglutide 3.0 mg, in combination with diet and exercise, was investigated at 4 and 6 months post-initiation. Changes in absolute and percent body weight, and in cardiometabolic markers, were examined from baseline. Using a database of de-identified electronic medical records from 6 Canadian weight management clinics, a cohort of liraglutide 3.0 mg initiators during 2015-2016 was identified. Post-initiation values at 4 and 6 months were compared to respective baseline values using a paired t-test. The full cohort consisted of 311 subjects, with 210 subjects in the ≥4months and 167 subjects in the ≥6months persistence groups. For all subjects, average age was 49.7 and subjects were predominantly white (77.5%) and female (83.0%). Average BMI was 40.7 kg/m2, and weight was 114.8 kg. At baseline, 74.9%, 19.9% and 5.1% of subjects had normoglycemia, prediabetes, and diabetes, respectively. Average baseline values for HbA1c and blood pressure were 5.8% and 127/77 mmHg. There was a significant change in body weight 6 months after initiation of treatment in persistent subjects (≥6months: -8.1 kg, p<0.001). Weight loss was also significant for subjects persistent on treatment for ≥4months (-6.9 kg, p<0.001) and in all subjects, regardless of persistence (-7.5 kg, p-value<0.001). Percentage change in body weight from baseline for the ≥6months group was -7.1%, with 63.4% and 35.2% of subjects having lost ≥5% and >10% body weight, respectively. Overall percentage change in body weight was also observed in the ≥4months group (-6.2%) and in all subjects (-6.6%). For the ≥6months treatment group, there was a statistically significant change in HbA1c (-0.35%, p<0.001) and SBP (-3.0 mmHg, p<0.01), but not DBP (0.1 mmHg, p=0.90). In a real-world setting, liraglutide 3.0 mg, when combined with diet and exercise, was associated with clinically meaningful weight loss and with improvements in cardiometabolic markers.