scholarly journals Effectiveness of First-Line Treatment With Recombinant Interleukin-1 Receptor Antagonist in Steroid-Naive Patients With New-Onset Systemic Juvenile Idiopathic Arthritis: Results of a Prospective Cohort Study

2014 ◽  
Vol 66 (4) ◽  
pp. 1034-1043 ◽  
Author(s):  
Sebastiaan J. Vastert ◽  
Wilco de Jager ◽  
Bo Jan Noordman ◽  
Dirk Holzinger ◽  
Wietse Kuis ◽  
...  
Keyword(s):  
Cohort Study ◽  
Juvenile Idiopathic Arthritis ◽  
Receptor Antagonist ◽  
Prospective Cohort ◽  
Interleukin 1 ◽  
Systemic Juvenile Idiopathic Arthritis ◽  
Line Treatment ◽  
First Line ◽  
First Line Treatment ◽  
New Onset

scholarly journals Bortezomib-based therapy in non-transplant multiple myeloma patients: a retrospective cohort study from the FABIO project

2021 ◽  
Vol 12 ◽  
pp. 204062072199648
Author(s):  
Matteo Franchi ◽  
Claudia Vener ◽  
Donatella Garau ◽  
Ursula Kirchmayer ◽  
Mirko Di Martino ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Cohort Study ◽  
Cost Effectiveness ◽  
Line Treatment ◽  
First Line ◽  
Conventional Chemotherapy ◽  
Risk Of Death ◽  
Model Average ◽  
Italian Regions ◽  
First Line Treatment

Introduction: Randomized clinical trials showed that bortezomib, in addition to conventional chemotherapy, improves survival and disease progression in multiple myeloma (MM) patients not eligible for stem cell transplantation. The aim of this retrospective population-based cohort study is the evaluation of both clinical and economic profile of bortezomib-based versus conventional chemotherapy in daily clinical practice. Methods: Healthcare utilization databases of six Italian regions were used to identify adult patients with non-transplant MM, who started a first-line therapy with bortezomib-based or conventional chemotherapy. Patients were matched by propensity score and were followed from treatment start until death, lost to follow-up or study end-point. Overall survival (OS) and restricted mean survival time (RMST) were estimated using the Kaplan–Meier method. Association between first-line treatment and risk of death was estimated by a conditional Cox proportional regression model. Average mean cumulative costs were estimated and compared between groups. Results: In the period 2010–2016, 3509 non-transplant MM patients met the inclusion criteria, of which 1157 treated with bortezomib-based therapy were matched to 1826 treated with conventional chemotherapy. Median OS and RMST were 33.9 and 27.9 months, and 42.9 and 38.4 months, respectively, in the two treatment arms. Overall, these values corresponded to a HR of death of 0.79 (95% CI 0.71–0.89) over a time horizon of 84 months. Average cumulative cost were 83,839 € and 54,499 €, respectively, corresponding to an incremental cost-effectiveness ratio of 54,333 € per year of life gained, a cost coherent with the willingness-to-pay thresholds frequently adopted from Western countries. Conclusions: These data suggested that, in a large cohort of non-transplant MM patients treated outside the experimental setting, first-line treatment with bortezomib-based therapy was associated with a favourable effectiveness and cost-effectiveness profile.

scholarly journals Effect of first-line biologic initiation on glucocorticoid exposure in children hospitalized with new-onset systemic juvenile idiopathic arthritis: emulation of a pragmatic trial using observational data

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Rosemary G. Peterson ◽  
Rui Xiao ◽  
Hannah Katcoff ◽  
Brian T. Fisher ◽  
Pamela F. Weiss
Keyword(s):  
Juvenile Idiopathic Arthritis ◽  
Biologic Therapy ◽  
Tertiary Care ◽  
Pragmatic Trial ◽  
Systemic Juvenile Idiopathic Arthritis ◽  
Source Population ◽  
First Line ◽  
Eligibility Criteria ◽  
Over Time ◽  
New Onset

Abstract Background Glucocorticoid exposure is a significant driver of morbidity in children with systemic juvenile idiopathic arthritis (sJIA). We determined the effect of early initiation of biologic therapy (IL-1 or IL-6 inhibition) on glucocorticoid exposure in hospitalized patients with new-onset sJIA. Methods We emulated a pragmatic sequence of trials (“pseudo-trials”) of biologic initiation in children (≤ 18 years) hospitalized with new-onset sJIA utilizing retrospective data from an administrative database from 52 tertiary care children’s hospitals from 2008 to 2019. Eligibility window, treatment assignment and start of follow-up between biologic and non-biologic study arms were aligned for each pseudo-trial. Patients in the source population could meet eligibility criteria at several timepoints. Mixed-effects logistic regression was used to determine the effect of biologic initiation on in-hospital glucocorticoid exposure. Results Four hundred sixty-eight children met eligibility criteria, of which 19% received biologic therapy without preceding or concomitant initiation of immunomodulatory medications. This proportion significantly increased over time during the study period (p <  0.01). 1451 trial subjects were included across 4 pseudo-trials with 71 assigned to the biologic arm and 1380 assigned to the non-biologic arm. After adjustment, there was a trend toward decreased odds of glucocorticoid initiation in the biologic arm compared to the non-biologic arm (OR 0.39, 95% CI [0.13, 1.15]). Conclusion Biologic initiation in children hospitalized with new-onset sJIA significantly increased over time and may be associated with reduced glucocorticoid exposure. The increasing use of first-line biologic therapy may lead to clinically relevant reductions in treatment-related adverse effects of glucocorticoid-reliant therapeutic approaches.

scholarly journals Erratum on “Non-indolent mantle cell lymphoma at a single public hospital in Brazil: real world first-line treatment cohort study data”

2020 ◽  
Vol 42 (2) ◽  
pp. 194
Author(s):  
Sergio Augusto Buzian Brasil ◽  
Carolina Colaço ◽  
Tomas Barrese ◽  
Roberto P. Paes ◽  
Cristina Bortolheiro ◽  
...  
Keyword(s):  
Cohort Study ◽  
Mantle Cell Lymphoma ◽  
Real World ◽  
Public Hospital ◽  
Cell Lymphoma ◽  
Study Data ◽  
Line Treatment ◽  
First Line ◽  
Mantle Cell ◽  
First Line Treatment
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