Life Expectancy in Duchenne Muscular Dystrophy: Reproduced Individual Patient Data Meta-analysis

Objective:Duchenne Muscular Dystrophy (DMD) is a rare progressive disease, which is often diagnosed in early childhood, and leads to considerably reduced life-expectancy; due to its rarity, research literature and patient numbers are limited. To fully characterise the natural history, it is crucial to obtain appropriate estimates of the life-expectancy and mortality rates of patients with DMD.Methods:A systematic review of the published literature on mortality in DMD up until July 2020 was undertaken, specifically focusing on publications in which Kaplan-Meier (KM) survival curves with age as a time-scale were presented. These were digitised and individual patient data (IPD) reconstructed. The pooled IPD were analysed using the Kaplan-Meier estimator and parametric survival analysis models. Estimates were also stratified by birth cohort.Results:Of 1177 articles identified, 14 publications met the inclusion criteria and provided data on 2283 patients, of whom 1049 had died. Median life-expectancy was 22.0 years (95% CI: 21.2, 22.4). Analyses stratifying by three time-periods in which patients were born showed markedly increased life-expectancy in more recent patient populations; patients born after 1990 have a median life-expectancy of 28.1 years (95% CI 25.1, 30.3).Conclusions:This paper presents a full overview of mortality across the lifetime of a patient with DMD, and highlights recent improvements in survival. In the absence of large-scale prospective cohort studies or trials reporting mortality data for patients with DMD, extraction of IPD from the literature provides a viable alternative to estimating life-expectancy for this patient population.

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Life Expectancy in Duchenne Muscular Dystrophy: A Systematic Review and Reconstructed Individual Patient Data Meta-Analysis

SSRN Electronic Journal ◽

10.2139/ssrn.3795309 ◽

2021 ◽

Author(s):

Jonathan Broomfield ◽

Micki Hill ◽

Michael J. Crowther ◽

Michela Guglieri ◽

Keith R. Abrams

Keyword(s):

Systematic Review ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Life Expectancy ◽

Individual Patient Data ◽

Meta Analysis ◽

Patient Data

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Life expectancy at birth in Duchenne muscular dystrophy: a systematic review and meta-analysis

European Journal of Epidemiology ◽

10.1007/s10654-020-00613-8 ◽

2020 ◽

Vol 35 (7) ◽

pp. 643-653 ◽

Cited By ~ 5

Author(s):

Erik Landfeldt ◽

Rachel Thompson ◽

Thomas Sejersen ◽

Hugh J. McMillan ◽

Janbernd Kirschner ◽

...

Keyword(s):

Systematic Review ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Life Expectancy ◽

Meta Analysis ◽

Life Expectancy At Birth

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Mortality in trials on transcatheter aortic valve implantation versus surgical aortic valve replacement: a pooled meta-analysis of Kaplan–Meier-derived individual patient data

European Journal of Cardio-Thoracic Surgery ◽

10.1093/ejcts/ezaa087 ◽

2020 ◽

Vol 58 (2) ◽

pp. 221-229 ◽

Cited By ~ 6

Author(s):

Fabio Barili ◽

Nicholas Freemantle ◽

Alberto Pilozzi Casado ◽

Mauro Rinaldi ◽

Thierry Folliguet ◽

...

Keyword(s):

Aortic Valve ◽

Individual Patient Data ◽

Meta Analysis ◽

Patient Data ◽

Time Varying ◽

Time To Event ◽

Surgical Aortic Valve Replacement ◽

Transcatheter Aortic Valve ◽

Kaplan Meier ◽

Valve Implantation

Abstract OBJECTIVES This meta-analysis of Kaplan–Meier-estimated individual patient data was designed to evaluate the effects of transcatheter aortic valve implantation (TAVI) and surgical aortic valve replacement (SAVR) on the long-term all-cause mortality rate, to examine the potential time-varying effect and to model their hazard ratios (HRs) over time. Moreover, we sought to compare traditional meta-analytic tools and estimated individual patient data meta-analyses. METHODS Trials comparing TAVI versus SAVR were identified through Medline, Embase, Cochrane databases and specialist websites. The primary outcome was death from any cause at follow-up. Enhanced secondary analyses of survival curves were performed estimating individual patient time-to-event data from published Kaplan–Meier curves. Treatments were compared with the random effect Cox model in a landmark framework and fully parametric models. RESULTS We identified 6 eligible trials that included 6367 participants, randomly assigned to undergo TAVI (3252) or SAVR (3115). According to the landmark analysis, the incidence of death in the first year after implantation was significantly lower in the TAVI group [risk-profile stratified HR 0.85, 95% confidence interval (CI) 0.73–0.99; P = 0.04], whereas there was a reversal of the HR after 40 months (risk-profile stratified HR 1.31, 95% CI 1.01–1.68; P = 0.04) favouring SAVR over TAVI. This time-varying trend of HRs was also confirmed by a fully parametric time-to-event model. Traditional meta-analytic tools were shown to be biased because they did not intercept heterogeneity and the time-varying effect. CONCLUSIONS The mortality rates in trials of TAVI versus SAVR are affected by treatments with a time-varying effect. TAVI is related to better survival in the first months after implantation whereas, after 40 months, it is a risk factor for all-cause mortality.

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Prosthesis–patient mismatch after mitral valve replacement: A pooled meta‐analysis of Kaplan–Meier‐derived individual patient data

Journal of Cardiac Surgery ◽

10.1111/jocs.15108 ◽

2020 ◽

Vol 35 (12) ◽

pp. 3477-3485

Author(s):

Anton Tomšič ◽

Bardia Arabkhani ◽

Jan W. Schoones ◽

Jonathan R. G. Etnel ◽

Nina A. Marsan ◽

...

Keyword(s):

Mitral Valve ◽

Valve Replacement ◽

Mitral Valve Replacement ◽

Individual Patient Data ◽

Meta Analysis ◽

Patient Data ◽

Kaplan Meier

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Systematic Review and Individual Patient Data Meta-Analysis Of Treatment-Related Risk Of Secondary Malignant Neoplasms After Hodgkin Lymphoma

Blood ◽

10.1182/blood.v122.21.1753.1753 ◽

2013 ◽

Vol 122 (21) ◽

pp. 1753-1753

Author(s):

Jeremy Franklin ◽

Dennis A. Eichenauer ◽

Ingrid Becker ◽

Andreas Engert

Keyword(s):

Systematic Review ◽

Hodgkin Lymphoma ◽

Large Scale ◽

Individual Patient Data ◽

Conflicts Of Interest ◽

Meta Analysis ◽

Treatment Strategies ◽

Progression Free Survival ◽

Patient Data ◽

Malignant Neoplasms

Abstract Introduction Secondary malignant neoplasms (SMN) are a major late effect of treatment for Hodgkin lymphoma (HL). Single trials have inadequate power to detect differences in SMN rates, while the many large-scale cohort-based studies of SMN after HL suffer from non-randomised, potentially biased comparisons of treatment strategies. The consequences of choice of first-line treatment for SMN risk remain unclear. We performed a Cochrane systematic review addressing this question based on our previous such review in 2000-2004. Material and methods Individual patient data (IPD) were collected from randomised controlled trials testing 5 currently relevant experimental strategies: avoidance of additional radiotherapy (RT) after chemotherapy (CT); reduction of RT field; reduction of RT dose; use of fewer CT cycles; intensification of CT regimen. All trials employed modern ABVD-like regimens and limited radiation fields, and recruited at least 50 patients per treatment group. Incidence of SMN, overall survival (OS) and progression-free survival (PFS) were analysed. Due to small numbers of events, SMN was analysed using Peto’s method. Results and Discussion Data from 16 of the 21 eligible trials were obtained, including 9498 patients. These trials recruited between 1984 and 2007 and randomised between 100 and 1351 patients each. Standard chemotherapy was predominantly ABVD, followed by COPP/ABVD and MOPP/ABV. Most frequent intensified chemotherapies were escalated BEACOPP and Stanford V. For each study question, key information and resulting odds ratios are shown in the table. Avoidance of additional RT significantly reduced the SMN risk (Peto odds ratio 0.433, 95% confidence interval (0.28; 0.82), p=0.010). Intensified chemotherapy regimens were associated with a consistent slightly higher risk but the effect was not significant. Other study questions showed no marked effects on SMN risk. Regarding solid tumors, follow-up is still too short: an update after 5 to 10 years is necessary. Disclosures: No relevant conflicts of interest to declare.

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