Prognostic implications of DPP-4 inhibitor vs. sulfonylurea use on top of metformin in a real world setting - results of the 1 year follow-up of the prospective DiaRegis registry

2013 ◽  
Vol 67 (10) ◽  
pp. 1005-1014 ◽  
Author(s):  
A. K. Gitt ◽  
P. Bramlage ◽  
C. Binz ◽  
M. Krekler ◽  
E. Deeg ◽  
...  
Keyword(s):  
Real World ◽  
Real World Setting ◽  
Prognostic Implications

scholarly journals Real-World Clinical Outcomes Associated with Canagliflozin in Patients with Type 2 Diabetes Mellitus in Spain: The Real-Wecan Study

2020 ◽  
Vol 9 (7) ◽  
pp. 2275
Author(s):  
Juan J. Gorgojo-Martínez ◽  
Manuel A. Gargallo-Fernández ◽  
Alba Galdón Sanz-Pastor ◽  
Teresa Antón-Bravo ◽  
Miguel Brito-Sanfiel ◽  
...  
Keyword(s):  
Diabetes Mellitus ◽  
Real World ◽  
Primary Outcome ◽  
Baseline Hba1c ◽  
Real World Setting ◽  
Antihyperglycemic Therapy ◽  
The Mean ◽  
Hba1c Levels

The aims of this multicentric retrospective study were to assess in a real-world setting the effectiveness and safety of canagliflozin 100 mg/d (CANA100) as an add-on to the background antihyperglycemic therapy, and to evaluate the intensification of prior sodium–glucose co-transporter type 2 inhibitor (SGLT-2i) therapy by switching to canagliflozin 300 mg/d (CANA300) in patients with T2DM. One cohort of SGLT2i-naïve patients with T2DM who were initiated on CANA100 and a second cohort of patients with prior background SGLT-2i therapy who switched to CANA300 were included in the study. The primary outcome of the study was the mean change in HbA1c over the follow-up time. In total, 583 patients were included—279 in the cohort of CANA100 (HbA1c 8.05%, weight 94.9 kg) and 304 in the cohort of CANA300 (HbA1c 7.51%, weight 92.0 kg). Median follow-up periods in both cohorts were 9.1 and 15.4 months respectively. CANA100 was associated to significant reductions in HbA1c (−0.90%) and weight (−4.1 kg) at the end of the follow-up. In those patients with baseline HbA1c > 8% (mean 9.25%), CANA100 lowered HbA1c levels by 1.51%. In the second cohort, patients switching to CANA300 experienced a significant decrease in HbA1c (−0.35%) and weight (−2.1 kg). In those patients with baseline HbA1c > 8% (mean 8.94%), CANA300 lowered HbA1c levels by 1.12%. There were significant improvements in blood pressure in both cohorts. No unexpected adverse events were reported. In summary, CANA100 (as an add-on therapy) and CANA300 (switching from prior SGLT-2i therapy) significantly improved several cardiometabolic parameters in patients with T2DM.

Long-term follow-up of patients undergoing decompressive hemicraniectomy for malignant stroke: Quality of life and caregiver’s burden in a real-world setting

2020 ◽  
Vol 197 ◽  
pp. 106168
Author(s):  
Pedro Tadao Hamamoto Filho ◽  
Lucas Braz Gonçalves ◽  
Nicholas Falcomer Koetz ◽  
Aline Maria Lara Silvestrin ◽  
Aderaldo Costa Alves Júnior ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Real World ◽  
Decompressive Hemicraniectomy ◽  
Real World Setting ◽  
Long Term Follow Up

scholarly journals TCT-350 Safety and Performance of the Resorbable Magnesium Scaffold, Magmaris in a Real World Setting - First 400 Subjects at 12-month Follow-up of the BIOSOLVE-IV Registry

2018 ◽  
Vol 72 (13) ◽  
pp. B142-B143
Author(s):  
Michael Lee ◽  
Adrian Wlodarczak ◽  
Johan Bennett ◽  
Jan Torzewski ◽  
Michael Haude ◽  
...  
Keyword(s):  
Real World ◽  
Real World Setting ◽  
And Performance

scholarly journals 087 Endovascular clot retrieval (ECR) in the elderly. For better or worse in the real world?

2019 ◽  
Vol 90 (e7) ◽  
pp. A28.1-A28
Author(s):  
Stacey K Jankelowitz ◽  
Kylie Tastula ◽  
Nicola Mitchell ◽  
Patrick Tang ◽  
Tim Ang ◽  
...  
Keyword(s):  
Real World ◽  
Stroke Risk ◽  
The Elderly ◽  
Early Improvement ◽  
Transcatheter Aortic Valve ◽  
Real World Setting ◽  
Multicentre Trials ◽  
Valve Implantation ◽  
Lost To Follow Up

IntroductionAcross multicentre trials ECR is safe and effective in octogenarians. Despite RCT evidence elderly patients may be denied ECR due to perceived poor risk-benefit. We examine impact of age on ECR outcomes and outcomes in transcatheter aortic valve implantation (TAVI) cases (where stroke risk is high), in a real world setting.MethodsWe analysed 311 consecutive ECR cases between 2016 and 2019 in 10 year age bands for ECR outcomes including 90 day mRS and mortality. Impact of premorbid function (mRS), NIHSS, recorded co-morbidities, and aetiology was assessed. TAVI case outcomes were examined.ResultsThirty one percent of ECR outcome cases were over 79 years of age; 90 day mortality was 34%; 25% had a 90 day mRS 0–2. Early NIHSS improvement was 5. Ninety-day mortality and mRS 0–2 for 10–19 (n=3), 20–29 (n=2), 30–39 (n=4), 40–49 (n=23), 50–59 (n=27), 60–69 (n=69), 70–79 (n=84),80–89 (n=62) and 90–99 (n=11) years were 0 and 100%, 0 and 100%, 33 and 67%, 4 and 78%, 15 and 52%, 13 and 49%, 17 and 33%, 24 and 2% and 55 and 18%, respectively. There was 9% lost to follow-up.Six TAVI cases had a NIHSS of 8–20 and pre-morbid mRS<3, four with mRS 0. Mean 24 hour NIHSS improvement was 8.ConclusionWithout age exclusions older patients had worse unadjusted outcomes. However, patients over 79 years had clinically important early improvement in NIHSS score and ninety day outcomes were comparable to favourable RCT data and TAVI patients also had early improvement.

Impact of Treatment on Overall Survival (OS) in Higher-Risk Myelodysplastic Syndromes (MDS): A Report from the Erasme Study

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 5240-5240
Author(s):  
Montserrat Arnan ◽  
Rosa Coll ◽  
Mar Tormo ◽  
José María Bastida ◽  
María Calbacho ◽  
...  
Keyword(s):  
Prospective Study ◽  
Board Of Directors ◽  
Real World ◽  
Who Classification ◽  
Refractory Anemia ◽  
Advisory Committees ◽  
Prior Therapy ◽  
Real World Setting ◽  
Very High

Abstract Introduction: Treatment of MDS has undergone a dramatic change in recent years with the emergence of demethylating agents; however, there are limited data on the impact of these agents on OS in the "real-world" setting. The aim of this study is to evaluate the use and outcomes of different therapies in patients (pts) with International Prognostic Scoring System (IPSS)-defined Intermediate-2- and High-risk (higher-risk) MDS. Methods: The ERASME study (CEL-SMD-2012-01) is an observational, prospective study of pts with either MDS or chronic myelomonocytic leukemia (CMML); pts were defined using the 2008 World Health Organization (WHO) classification system. Initial pt management strategy was classified into 3 groups: active therapy (AT), such as chemotherapy or treatment with azacitidine (AZA), lenalidomide (LEN), etc.; allogeneic hematopoietic cell transplantation (HCT), which included those pts receiving other therapies before transplantation; and observation and support (OB&SP), which included red blood cell and platelet transfusions, and growth factors. OS rates of higher-risk pts are presented using the Kaplan-Meier method. Results: A total of 160 pts with higher-risk MDS were recruited between Jan 2013 and Feb 2015. Median follow-up was 8.6 months (interquartile range [IQR] 4.0-11.8). Pt characteristics are described in the Table. AT was the first therapeutic decision in 83 (52%) pts, HCT in 43 (27%), and OB&SP in 34 (21%). Within the AT group, 75 (90%) pts received AZA alone, 5 (6%) AZA plus chemotherapy, and 3 (4%) received other options; most pts (n = 78; 94%) were treated with a 7-day regimen. Among the 43 potential HCT candidates, 40 (93%) had received prior therapy: 25 (58%) with AZA alone, 8 (19%) with chemotherapy, 6 (14%) with AZA plus chemotherapy, and 1 (2%) with LEN; 3 (7%) had not received prior therapy. Of the potential HCT candidates, 20 had undergone transplantation within a median of 8 months (IQR 4.1-13.3); 7 (16%) pts died before transplantation and 16 (37%) are still awaiting transplantation. The main reasons for OB&SP being the initial pt strategy were disease risk (100%), age (95%), comorbidities (60%), and symptomatology (44%). At last follow-up, 70 of 160 pts (44%) had died after a median of 6 months (IQR 2.9-11.8): 34 (41%), 14 (32%), and 22 (65%) pts undergoing AT, HCT, and OB&SP, respectively (log-rank 6.9; P = 0.032). Median OS in pts who received AT, HCT, and OB&SP was 18.60 months (95% confidence interval [CI] 13.1-21.8), 14.92 months (95% CI 11.6-not reached), and 8.44 months (95% CI 4.3-13.4), respectively. Median OS was significantly longer in the AT group compared with the OB&SP group (hazard ratio [HR] 1.9; 95% CI 1.1-3.3; P = 0.0197). No statistically significant difference was observed in OS between pts treated with AT and those considered potential HCT candidates (HR 0.9; 95% CI 0.5-17.7; P = 0.8). Conclusions: The preliminary data from this observational, prospective study indicate that AT significantly prolongs OS compared with OB&SP when treating pts with higher-risk MDS in a real-world setting. AZA was the most common treatment in the AT group. Abstract presented on behalf of the ERASME Study Investigators Group. Table. Baseline characteristics of the MDS population (N = 160) Characteristic Age, median (IQR), years 73 (64.5-79.0) Male, n (%) 86 (53.8) Bone marrow blast count, median (IQR), % 12 (7.5-16.3) Hemoglobin level, median (IQR), g/dL 9.55 (8.0-10.6) Platelet count, median (IQR), × 109/L 64.75 (41.0-121.0) ANC, median (IQR), × 109/L 1 (0.47-2.20) Cytogenetic risk (by IPSS-R), n (%) Low 64 (40.0) Intermediate 9 (5.6) High 27 (16.9) Very High 39 (24.4) Missing data 21 (13.1) WHO classification, n (%) RCMD 17 (10.6) RAEB type 1 28 (17.5) RAEB type 2 94 (58.8) Unclassified 2 (1.3) Missing 1 (0.6) AML 20-30% blasts 18 (11.3) IPSS risk classification, n (%) Intermediate-2 86 (53.8) High 71 (44.4) Missing 3 (1.9) IPSS-R risk classification, n (%) Very Low 4 (2.5) Low 28 (17.5) Intermediate 20 (12.5) High 40 (25.0) Very High 46 (28.8) Missing 22 (13.8) AML, acute myeloid leukemia; ANC, absolute neutrophil count; FAB, French-American-British; IPSS-R, Revised-IPSS; RAEB, refractory anemia with excess blasts; RCMD, refractory anemia with multilineage dysplasia. Disclosures Off Label Use: Azacitidine was used to treat patients who are potential hematopoietic stem cell transplant candidates. Ramos:GlaxoSmithKline: Honoraria; Janssen-Cilag: Honoraria, Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Honoraria; Celgene Corporation: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Amgen: Consultancy, Honoraria. Rafel:Celgene Corporation: Employment. Valcárcel:Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Amgen: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Celgene Corporation: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; GlaxoSmithKline: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau.

Single-Agent Ibrutinib Vs Standard of Care for Patients with Relapsed/Refractory (R/R) and Treatment-Naive (TN) Chronic Lymphocytic Leukemia (CLL): An Adjusted Comparison of RESONATETM and RESONATE-2TM with the French Lyon-Sud Database

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 2039-2039
Author(s):  
Gilles A. Salles ◽  
Lucile Baseggio ◽  
Emmanuel Bachy ◽  
Clementine Sarkozy ◽  
Herve Ghesquieres ◽  
...  
Keyword(s):  
Real World ◽  
Research Funding ◽  
Disease Stage ◽  
Treatment Regimens ◽  
Real World Setting ◽  
Level Data ◽  
Advanced Disease Stage ◽  
Independent Risk Factors ◽  
Male Sex

Abstract INTRODUCTION: The benefits of ibrutinib (ibr) have been demonstrated by the phase 3 RESONATE and RESONATE-2 trials for patients (pts) with R/R and TN (≥ 65 years) CLL, respectively. In these studies, ibr showed significantly improved progression-free survival (PFS) and overall survival (OS) vs approved comparators (ofatumumab [ofa] in RESONATE and chlorambucil [clb] in RESONATE-2). However, a number of other treatment regimens are widely used in clinical practice for CLL based on individual patient and disease characteristics; it is currently unknown if ibr results in better survival outcomes when compared directly with each of these other treatments. AIM: In the absence of a head-to-head comparison, we investigated the relative efficacy of ibr vs physician's choice (PC) in pts with R/R or TN CLL by comparing pt-level data from RESONATE and RESONATE-2 with data from pts in a real-world setting (the Lyon-Sud database). This database holds electronic medical records for 390 pts with CLL from the academic Lyon-Sud Hospital in France; nearly all patients were diagnosed between 1990 and 2014. This is the first analysis of ibr vs PC in a real-world setting for pts with TN CLL. METHODS: Pt-level data from RESONATE (ibr, n = 195; ofa, n = 196) were compared with data on pts with CLL from Lyon-Sud who received 2nd(n = 107) or later-line treatment (3rd [n = 62], 4th [n = 43], and subsequent [n = 51] lines). Similarly, RESONATE-2 (ibr, n = 136; clb, n = 133) pt-level data were compared with those of pts aged ≥ 65 years who received 1st-line treatment (n = 131). In order to account for non-comparability due to lack of randomization, a multivariate Cox proportional hazards model was used to compare PFS and OS between treatments, including line of therapy, age, sex, disease stage (based on Binet/Rai), and deletion 17p or 11q mutations as covariates. For the definition of PFS, missing data for date of disease progression for pts in Lyon-Sud who initiated subsequent therapy were replaced by the conservative proxy of date of initiation of next treatment. Predicted PFS and OS curves for the Lyon-Sud cohort were derived from the multivariate model. RESULTS: For R/R pts in Lyon-Sud, across all treatment lines the most frequent regimens used were rituximab (R) + chemotherapy (n = 46), bendamustine + R (BR; n = 28), fludarabine + cyclophosphamide + R (FCR) (n = 27), clb + R (n = 19), R monotherapy (n = 21), and R-CHOP-based (n = 19); the remaining percentages comprised various other therapies, each used in < 5% of pts. Median age at treatment initiation for Lyon-Sud and RESONATE was 69 and 67 years, respectively; median number of prior therapies was 2 and 3, and median follow-up was 30.0 and 21.9 months. More lines of prior therapy, older age, male sex, advanced disease stage (Binet C/Rai III/IV), and presence of deletion 17p/11q were independent risk factors for worse PFS and OS outcome. After adjustment for differences in baseline characteristics, PFS and OS hazard ratios (HRs) for ibr vs PC were 0.18 (95% confidence interval [CI], 0.13-0.26) and 0.28 (0.17-0.46), respectively. Adjusted HRs for ibr vs the most frequent treatments ranged from 0.09 (R monotherapy) to 0.38 (FCR) for PFS and 0.21 (R monotherapy) to 0.33 (FCR) for OS; all were statistically significant. For TN pts in Lyon-Sud, the most frequent treatment regimens used were FCR (n = 40), clb + R (n = 21), clb monotherapy (n = 17), R-(mini)CHOP (n = 11), and BR (n = 11). Median age at treatment initiation was 73 years for both Lyon-Sud and RESONATE-2; median follow-up was 36.9 and 28.1 months, respectively. Older age, male sex, and advanced disease stage were independent risk factors for worse PFS and OS outcome. After adjustment for baseline differences, the PFS and OS HRs for ibr vs PC were 0.25 (0.14-0.47) and 0.41 (0.17-0.99). Predicted PFS and OS curves for both R/R and TN patients are presented in Figure 1. CONCLUSIONS: Investigation of survival outcomes suggests that ibr administered to pts in RESONATE and RESONATE-2 was more effective than PC used in a real-world cohort of French pts with R/R or TN CLL, suggesting a 4.8-fold improvement in PFS and a 3.4-fold improvement in OS in R/R pts and 4-fold and 2.4-fold improvements in TN pts, respectively. These results further support the growing evidence that ibr significantly improves both PFS and OS vs commonly used regimens in the R/R and TN settings, and could have important implications for improving treatment of CLL in clinical practice. Disclosures Salles: Janssen: Consultancy, Honoraria; Gilead: Honoraria, Research Funding; Mundipharma: Honoraria; Amgen: Consultancy, Honoraria; Novartis: Consultancy, Honoraria; Celgene: Consultancy, Honoraria; Roche/Genentech: Consultancy, Honoraria, Research Funding. Sarkozy:Sandoz: Research Funding; Janssen Research & Development: Honoraria; Gilead: Honoraria; Takeda: Research Funding. Ghesquieres:Mundipharma: Consultancy; Celgene: Consultancy, Membership on an entity's Board of Directors or advisory committees; Roche France: Research Funding. Diels:Johnson & Johnson: Employment, Equity Ownership. Besson:Janssen: Employment. MacDougall:Janssen Research & Development: Research Funding; IMS Health: Employment. Hermans:Janssen Research & Development: Research Funding; IMS Health: Employment. Healy:Janssen Research & Development: Employment. Garside:Janssen Research & Development: Employment. Iraqi:Janssen Research & Development: Employment.

Treatment patterns among patients with higher-risk myelodysplastic syndromes in a real-world setting: Electronic medical record (EMR)-based data.

2017 ◽  
Vol 35 (8_suppl) ◽  
pp. 247-247
Author(s):  
Bruce Jeffrey Dezube ◽  
Jill Bell ◽  
Aaron Galaznik ◽  
Eileen Farrelly ◽  
Marlo Blazer ◽  
...  
Keyword(s):  
Real World ◽  
Index Date ◽  
Treatment Guidelines ◽  
Specific Treatment ◽  
Cell Transplant ◽  
Newly Diagnosed ◽  
Limited Information ◽  
Nccn Guidelines ◽  
Real World Setting

247 Background: Treatment decisions in MDS are based on a prognostic scoring system that divides pts into five distinct risk categories (NCCN 2016). Treatment guidelines for HR MDS pts include hypomethylating agents (HMAs) alone (azacitidine & decitabine), high-intensity induction chemotherapy (IC), & stem cell transplant (SCT) alone or after HMAs. Limited information is available on how these recommendations are applied in practice. This study evaluated the real-world treatment of HR MDS pts. Methods: Newly diagnosed HR MDS pts who were ≥18 years old & initiated first-line therapy (1LT) were retrospectively identified from a large United States EMR database between 1/1/2008 & 7/31/2015. As complete cytogenetics were not available in the database, HR was based on: ICD coding: ≥1 inpatient claim with an HR MDS ICD-9/10 code (ICD-9 code: 238.73; ICD-10 codes: D46.20, D46.21, D46.22), or ≥2 outpatient claims with an MDS ICD-9/10 code, or an adapted HR MDS algorithm (NCCN Guidelines in Oncology for MDS v.1.2016; Greenberg, et al. Blood. 2012;120:2454-65; Schanz et al. J Clin Oncol. 2012;30:820-9). The first MDS claim served as the index date. 1LT was defined as an MDS-specific treatment initiated on or after the index date. Pts were followed until death, progression to acute myeloid leukemia (AML), loss to follow-up, or end of study (9/30/2015). Results: 720 newly diagnosed HR MDS pts were identified; of these, 229 (32%) pts received MDS-specific treatment. Median time to treatment was 22 days (interquartile range [IQR]: 10, 74). HMAs were the most common agents in the 1LT with 60% (n= 138) & 24% (n=54) receiving azacitidine & decitabine, respectively. Lenalidomide was used in 7.4% of pts (n=17), 4.8% received SCT alone (n=11), & 3.9% (n=9) received IC. At median follow-up of 9.4 months (IQR: 4.3, 18.4), 38% (n=86) died & 28% (n=63) progressed to AML. Conclusions: Despite guidelines, most HR MDS pts in a real-world setting were not treated with MDS-specific treatment. Among treated pts, 1LT with HMAs & azacitidine predominated. Subsequent research is needed to understand reasons for lack of treatment.

Examination of PD-1/PD-L1 inhibitor use and adverse events in a real-world setting.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e19294-e19294
Author(s):  
Debra E. Irwin ◽  
Brenna L. Brady
Keyword(s):  
Adverse Events ◽  
Real World ◽  
Metastatic Cancer ◽  
Immune Checkpoint Blockade ◽  
Administrative Claims ◽  
Treatment Results ◽  
Real World Setting ◽  
Common Diagnosis ◽  
History Of

e19294 Background: Immune checkpoint blockade through PD-1 and PD-L1 inhibition is an effective treatment for multiple cancers. This study used administrative claims to examine potential adverse events (AEs) associated with real-world PD-1 or PD-L1 inhibitor use. Methods: Adult patients newly initiating a PD-1 (pembrolizumab or nivolumab) or PD-L1 (atezolizumab, avelumab, or durvalumab) inhibitor from September 1, 2016 to August 30, 2018 were selected in the MarketScan Commercial and Medicare Supplemental Database. Patients were grouped by use of PD-1 or PD-L1 inhibitor; the study period consisted of 90 days baseline and 60 days follow-up around drug initiation. Patients who used both PD-1 and PD-L1 inhibitors during follow-up were excluded. Clinical characteristics were examined during baseline, while AEs were investigated over follow-up. Results: A total of 6,430 patients qualified for the analysis. The majority of the sample (N = 5,956; 93%) received PD-1 inhibitors. Compared to the PD-1 cohort, the PD-L1 cohort was older (64±10 vs. 61±12 yrs) and more likely to be male (61% vs. 56%), p < 0.05. PD-L1 patients were significantly more likely to have history of chronic pulmonary disease (28% vs. 23%) or myocardial infarction (4% vs. 3%) but less likely to have liver disease (2% vs. 0.6%) compared to PD-1 patients, p < 0.05. Lung cancer was the most common diagnosis in both groups (PD-1: 47%; PD-L1: 62%, p < 0.001). The PD-L1 cohort was more likely to have evidence of bladder cancer (36% vs. 5%), while the PD-1 cohort was more likely to have a melanoma (19% vs. 0.8%) or renal cell carcinoma diagnosis (10% vs. 7%), p < 0.05. Over half of the PD-1 (65%) and PD-L1 (61%) patients had metastatic cancer diagnosis during the study period. Incident AEs occurring in > 5% of the sample included dyspnea (PD-1: 13%; PD-L1: 14%), nausea/vomiting (PD-1: 11%; PD-L1: 8%, p < 0.05), anemia (PD-1: 11%; PD-L1: 12%), fatigue (PD-1: 10%; PD-L1: 12%), abdominal pain (PD-1: 7%; PD-L1: 7%), cough (PD-1: 7%; PD-L1: 10%, p < 0.05), back pain (PD-1: 7%; PD-L1: 10%, p < 0.05), constipation (PD-1: 6%; PD-L1: 8%), arthralgia (PD-1: 6%; PD-L1: 6%), pyrexia (PD-1: 5%; PD-L1: 8%, p < 0.01), and edema (PD-1: 6%; PD-L1: 5%). Conclusions: This study assessed real-world AEs associated with PD-1/PD-L1 inhibitor use in the 60 days following first treatment. Results showed AEs are common soon after starting therapy. Although, PD-1 and PD-L1 inhibitors target the same pathway, slightly different AE profiles exist for the two classes. More longitudinal analyses of real-world AEs are needed to better understand potential impacts of prolonged therapy.

scholarly journals Straightforward Transition to Bay 81-8973 Prophylaxis in Patients with Hemophilia A: Prospective Real-World Data from the Taurus Non-Interventional Study Show That Number of Infusions per Week Is Maintained or Reduced

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 5045-5045
Author(s):  
Michael Wang ◽  
Beng Fuh ◽  
Philip Maes ◽  
Maria Eva Mingot-Castellano ◽  
Rubén Berrueco ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Real World ◽  
Hemophilia A ◽  
Weekly Dose ◽  
Real World Data ◽  
World Data ◽  
Real World Setting ◽  
Patient Reported ◽  
Dosing Frequency

Abstract BACKGROUND: BAY 81-8973 (Kovaltry®, Bayer) is an unmodified full-length recombinant FVIII indicated for prophylaxis and treatment of bleeds in patients with hemophilia A; BAY 81-8973 was launched in 2016 and has since accumulated 6765 patient-years of exposure. The TAURUS study (NCT02830477) was established to investigate BAY 81-8973 prophylaxis dosing regimens chosen in clinical practice and confirm the established safety and efficacy results from the LEOPOLD clinical trials in a real world setting. OBJECTIVES: To analyse the proportion of patients on specific BAY 81-8973 prophylaxis regimens, bleeds, and patient-reported outcomes at baseline and most recent follow-up. METHODS: TAURUS is an international, open label, prospective, non-interventional, single arm study with a target recruitment of 350 previously treated patients with hemophilia A of all ages with moderate or severe hemophilia A (≤ 5% FVIII:C) with ≥ 50 exposure days to any FVIII product who have been switched to prophylaxis with BAY 81-8973. At baseline, physicians document clinical information including age, BMI, severity of hemophilia, number of target joints, prior treatment regimen, bleed history, inhibitor history, and reason for choosing a specific prophylaxis regimen. Patients/caregivers reported bleeds in ongoing patient diaries, and completed questionnaires on treatment satisfaction (HEMOSAT) and adherence (VERITAS-PRO) at baseline and follow-up. A scheduled interim analysis (30% of patients recruited) was conducted with data collected up to 2 July 2018. RESULTS: At the cut-off, 160 enrolled patients were included in the baseline analysis set, of whom 89 had ≥ 6 months of follow-up data available (median observation period 201 days), 33% of whom had completed one year of the study. Median (range) patient age was 22 (2‒69) years, time since diagnosis was 15 (0.5‒64) years, and most patients (76/89, 85%) had baseline FVIII level of <1%. Treatment assignments are shown in the table. All patients had received pre-study prophylaxis, for a median of 15 years, with 66% of patients using rFVIII-FS as their most recent FVIII treatment prior to BAY 81-8973. Most (91%) had been treated with BAY 81-8973 for <3 months prior to study entry. Pre-study, 72% of patients were treated ≥3 times per week (xW). At baseline, most patients (59%) were assigned treatment ≥3xW (every day, 1%; every other day, 16%; 3xW, 41%). The majority remained on their previous regimen (78% on ≤2xW and 97% on ≥3xW); any changes were mainly a reduction in frequency on BAY 81-8973 vs previous treatment (22%), with only 2% increasing frequency on BAY 81-8973. At last follow up, most patients remained on the same regimen: 60% on ≥3xW (≥ every other day, 17%; 3xW, 42%). Most patients (92%) did not alter their dosing frequency. Of the 8% who changed dosing frequency, the majority (6 patients) changed from ≥3xW to ≤2xW; 1 patient changed from ≤2xW to ≥3xW. The median prescribed weekly dose was 52 IU/kg (64 IU/kg for ≥3xW and 43 IU/kg for ≤2xW) on study, slightly lower than those with previous product: 56 IU/Kg overall, 64 IU/kg for ≥3xW and 50 IU/kg for ≤2xW. Median (Q1; Q3) patient diary-reported annualized joint bleed rates were 1.5 (0.0; 5.3), 1.2 (0.0; 5.3) and 1.4 (0.0; 6.1); for ≤2xW, ≥3xW, and all patients, respectively. HEMO-SAT and VERITAS-PRO data will be presented in the poster. No recruited patients developed inhibitors with BAY 81-8973. CONCLUSIONS: These real-world data from 89 patients show that the range of dosing options available for BAY 81-8973 allowed the majority of patients to become established quickly on this treatment upon switching. In the few instances where patients changed dosing frequency either upon switching to BAY 81-8973 or once established on treatment, most moved to less frequent treatment. Joint bleeding rates confirm and extend findings from the clinical trials and speak to effective bleeding prophylaxis with BAY 81-8973 in a real-world setting. Therefore, BAY 81-8973 treatment may be successfully individualized according to patient need and disease characteristics. Disclosures Wang: Terumo BCT: Other: CPC Clinical Research; CSL Behring: Consultancy; Bayer, Bioverative, Novo Nordisk, Octapharma, Shire, Genentech, Biomarain, Pfizer, CSL Behring, HEMA Biologics, Daiichi Sankyo: Research Funding; Bayer: Consultancy; Bayer, Novo Nordisk, Octapharma, Genentech, HEMA Biologics, Shire, CSL Behring: Honoraria; Novo Nordisk: Consultancy. Maes:Bayer: Honoraria. Rauchensteiner:Bayer: Employment.

scholarly journals The OPEN WATER trial: Aquablation in a real-world setting 1 year follow-up

2020 ◽  
Vol 19 ◽  
pp. e379
Author(s):  
N. Barber ◽  
P. Gilling ◽  
A. El Hajj ◽  
P. Anderson ◽  
T. Bach
Keyword(s):  
Real World ◽  
Open Water ◽  
Real World Setting
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