Fr518 LONG-TERM SAFETY OF TOFACITINIB: REAL WORLD DATA FROM A TERTIARY CENTER

Abstract Background Hypothetical concept of disproportionate secondary mitral regurgitation (SMR) has been recently introduced to facilitate patient's selection for mitral valve intervention. However, real world data validating this concept are unavailable. Purpose To investigate long-term effects of minimally invasive mitral valve annuloplasty (MVA) in patients with disproportionate (dSMR) versus proportionate SMR. Methods The study population consisted of 44 consecutive patients (age 67±9,5 years; 64% males) on guidelines-directed therapy with advanced heart failure (HF), reduced LV ejection fraction (EF) (32±9,7%) and SMR undergoing isolated mini-invasive MVA. Patients with organic mitral regurgitation or concomitant myocardial revascularization were excluded. To assess SMR disproportionality, the PISA-derived effective regurgitant orifice area (EROA) and regurgitant volume (RV) were compared to the estimated EROA and RV by using Gorlin formula and pooled real world data. Results According to EROA, a total of 20 (46%) and 24 (54%) patients, respectively, had dSMR and proportionate SMR (pSMR). According to RV, a total of 17 (39%) had dSMR and 27 (61%) had pSMR. Patients with dSMR showed significantly lower prevalence of male gender and higher prevalence of diabetes mellitus than patients with pSMR (p<0,001). Moreover, we observed smaller LV end-diastolic volume, larger EROA and RV (both p<0,01) and higher LV EF (p=0,02) in the dSMR versus the pSMR group. Other baseline characteristics were similar. During median follow up of 4.39 y (IQR 2,2–9,96y), a total of 25 (56%) patients died from any cause while 21 (47%) individuals were readmitted for worsening HF. Patients with dSMR versus pSMR according to both EROA and RV showed significantly lower rate of HF readmissions (both p<0.05) (Figure 1, 2). In Cox regression analysis combining clinical and imaging parameters, dSMR was the only independent predictor of HF readmissions (HR 0.20, 95% CI 0.07–0.60, p=0.004). In contrast, mortality was similar between dSMR and pSMR (NS) with age as the only independent predictor (HR 1,10; 95% CI 1,03–1,18, p=0,003). Conclusions Minimally invasive MVA is associated with significant reduction of HF readmissions in patients with dSMR versus pSMR while the mortality is similar. This suggests the importance of other parameters, i.e. age and degree of LV remodeling, to guide clinical management in SMR. Funding Acknowledgement Type of funding source: None

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Modern management of iron overload in thalassemia major patients guided by MRI techniques: real-world data from a long-term cohort study

Annals of Hematology ◽

10.1007/s00277-021-04748-w ◽

2022 ◽

Author(s):

Selen Bayraktaroglu ◽

Nihal Karadas ◽

Sebnem Onen ◽

Deniz Yılmaz Karapinar ◽

Yesim Aydinok

Keyword(s):

Cohort Study ◽

Iron Overload ◽

Real World ◽

Thalassemia Major ◽

Real World Data ◽

World Data ◽

Modern Management

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VP08 Real-World Data Use In Health Technology Assessments: A Comparison Of Five Health Technology Assessment Agencies

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462317003051 ◽

2017 ◽

Vol 33 (S1) ◽

pp. 149-150

Author(s):

Amr Makady ◽

Ard van Veelen ◽

Anthonius de Boer ◽

Hans Hillege ◽

Olaf Klunger ◽

...

Keyword(s):

Health Technology Assessment ◽

Technology Assessment ◽

Real World ◽

Relative Effectiveness ◽

Internal Validity ◽

Health Technology ◽

Real World Data ◽

World Data ◽

The Impact

INTRODUCTION:Reimbursement decisions are usually based on evidence from randomized controlled trials (RCT) with high internal validity but lower external validity. Real-World Data (RWD) may provide complimentary evidence for relative effectiveness assessments (REA's) and cost-effectiveness assessments (CEA's) of treatments. This study explores to which extent RWD is incorporated in REA's and CEA's of drugs used to treat metastatic melanoma (MM) by five Health Technology Assessment (HTA) agencies.METHODS:Dossiers for MM drugs published between 1 January 2011 and 31 December 2016 were retrieved for HTA agencies in five countries: the United Kingdom (NICE), Scotland (SMC), France (HAS), Germany (IQWiG) and the Netherlands (ZIN). A standardized data-extraction form was used to extract data on RWD mentioned in the assessment and its impact on appraisal (for example, positive, negative, neutral or unknown) for both REA and CEA.RESULTS:In total, fourty-nine dossiers were retrieved: NICE = 10, SMC = 13, IQWiG = 16, HAS = 8 and ZIN = 2. Nine dossiers (18.4 percent) included RWD in REA's for several parameters: to describe effectiveness (n = 5) and/or the safety (n = 2) of the drug, and/or the prevalence of MM (n = 4). CEA's were included in 25/49 dossiers (IQWiG and HAS did not perform CEA's). Of the twenty-five CEA's, twenty (80 percent) included RWD to extrapolate long-term effectiveness (n = 19), and/or identify costs associated with treatments (n = 7). When RWD was included in REA's (n = 9), its impact on the appraisal was negative (n = 4), neutral (n = 2), unknown (n = 1) or was not discussed in the appraisal (n = 2). When RWD was included in CEA's (n = 11), its impact on the appraisal varied between positive (n = 2), negative (n = 5) and unknown (n = 4).CONCLUSIONS:Generally, RWD is more often included in CEA's than REA's (80 percent versus 18.4 percent, respectively). When included, RWD was mostly used to describe the effectiveness of the drug (REA) or to predict long-term effectiveness (CEA). The impact of RWD on the appraisal varied greatly within both REA's and CEA's.

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