Biomarkers of disease progression in adolescents and adults with 5q spinal muscular atrophy: a systematic review and meta-analysis

2022 ◽  
Author(s):  
Maria Gavriilaki ◽  
Maria Moschou ◽  
Vasileios Papaliagkas ◽  
Konstantinos Notas ◽  
Evangelia Chatzikyriakou ◽  
...  
Keyword(s):  
Systematic Review ◽  
Spinal Muscular Atrophy ◽  
Disease Progression ◽  
Muscular Atrophy ◽  
Meta Analysis ◽  
Adolescents And Adults

Efficacy and Safety of Valproic Acid for Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis

CNS Drugs ◽  
2019 ◽  
Vol 33 (3) ◽  
pp. 239-250
Author(s):  
Abdelrahman Elshafay ◽  
Truong Hong Hieu ◽  
Mohamed Fahmy Doheim ◽  
Mahmoud Attia Mohamed Kassem ◽  
Mohammed Fathi ELdoadoa ◽  
...  
Keyword(s):  
Systematic Review ◽  
Valproic Acid ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Meta Analysis ◽  
Efficacy And Safety

Efficacy and security of onasemnogene abeparvovec-XIOI in spinal muscular atrophy: a systematic review and meta-analysis

2021 ◽  
Vol 429 ◽  
pp. 118887
Author(s):  
Aaron Rodriguez-Calienes ◽  
Sandra Chavez-Malpartida ◽  
Giancarlo Alvarado-Gamarra ◽  
Niels Pacheco-Barrios ◽  
Peggy Martinez-Esteban ◽  
...  
Keyword(s):  
Systematic Review ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Meta Analysis

scholarly journals Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models

Gene Therapy ◽  
2021 ◽  
Author(s):  
Ellie M. Chilcott ◽  
Evalyne W. Muiruri ◽  
Theodore C. Hirst ◽  
Rafael J. Yáñez-Muñoz
Keyword(s):  
Systematic Review ◽  
Experimental Design ◽  
Spinal Muscular Atrophy ◽  
Publication Bias ◽  
Median Survival ◽  
Muscular Atrophy ◽  
Meta Analysis ◽  
Significant Heterogeneity ◽  
Rodent Models ◽  
Genetic Therapy

AbstractSpinal muscular atrophy (SMA) is a severe childhood neuromuscular disease for which two genetic therapies, Nusinersen (Spinraza, an antisense oligonucleotide), and AVXS-101 (Zolgensma, an adeno-associated viral vector of serotype 9 AAV9), have recently been approved. We investigated the pre-clinical development of SMA genetic therapies in rodent models and whether this can predict clinical efficacy. We have performed a systematic review of relevant publications and extracted median survival and details of experimental design. A random effects meta-analysis was used to estimate and compare efficacy. We stratified by experimental design (type of genetic therapy, mouse model, route and time of administration) and sought any evidence of publication bias. 51 publications were identified containing 155 individual comparisons, comprising 2573 animals in total. Genetic therapies prolonged survival in SMA mouse models by 3.23-fold (95% CI 2.75–3.79) compared to controls. Study design characteristics accounted for significant heterogeneity between studies and greatly affected observed median survival ratios. Some evidence of publication bias was found. These data are consistent with the extended average lifespan of Spinraza- and Zolgensma-treated children in the clinic. Together, these results support that SMA has been particularly amenable to genetic therapy approaches and highlight SMA as a trailblazer for therapeutic development.

Early vs Late Use of Anti-TNFa Therapy in Adult Patients With Crohn Disease: A Systematic Review and Meta-Analysis

2020 ◽  
Vol 26 (12) ◽  
pp. 1808-1818
Author(s):  
Shadi Hamdeh ◽  
Muhammad Aziz ◽  
Osama Altayar ◽  
Mojtaba Olyaee ◽  
Mohammad Hassan Murad ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Response ◽  
Disease Progression ◽  
Crohn Disease ◽  
Meta Analysis ◽  
Adult Patients ◽  
Cochrane Central Register ◽  
Significant Difference ◽  
Clinical Series ◽  
Early Use

Abstract Objectives While anti-tumor necrosis factor alpha (anti-TNFa) therapies for Crohn disease (CD) were initially introduced in 1998 for biologic therapies are often introduced after a minimum of 6 years after diagnosis. The benefit of anti-TNFa early in the course of CD is still controversial, with some studies showing better outcomes but others not. To determine whether earlier introduction of anti-TNFa therapy improves efficacy in clinical trials or clinical series, we aimed to perform a meta-analysis comparing early vs late anti-TNFa use in the management of CD. Methods A comprehensive search of MEDLINE, EMBASE, the Cochrane Central Register of Controlled Trials, the Cochrane Database of Systematic Reviews, and Scopus was conducted from each database’s inception to November 3, 2019. We included comparative studies of early vs late use of anti-TNFa therapy in adult patients with CD. Results Eleven studies were included in the analysis, with a total of 2501 patients. Meta-analysis demonstrated that the early use of anti-TNFa was associated with a statistically significant decrease in the need for surgery (relative risk [RR] = 0.43; 95% confidence interval [CI], 0.26–0.69; I2 = 68%) and disease progression (RR = 0.51; 95% CI, 0.35–0.75; I2 = 61%). Early use also showed an increase in early remission (RR = 1.94; 95% CI, 1.54–2.46; I2 = 0%) and clinical response. There was no statistically significant difference in achieving late remission (RR = 1.39; 95% CI, 0.94–2.05; I2 = 65%) or mucosal healing (RR = 1.10; 95% CI, 0.63–1.91; I2 = 0%). Conclusion This systematic review suggests that using anti-TNFa earlier in the treatment of CD (within 3 years) may improve clinical outcomes compared to late administration in terms of achieving early clinical remission, clinical response, disease progression, and the need for surgery.

scholarly journals How to intervene in the caries process: proximal caries in adolescents and adults—a systematic review and meta-analysis

2020 ◽  
Vol 24 (5) ◽  
pp. 1623-1636 ◽  
Author(s):  
C.H. Splieth ◽  
P. Kanzow ◽  
A. Wiegand ◽  
J. Schmoeckel ◽  
A. Jablonski-Momeni
Keyword(s):  
Systematic Review ◽  
Meta Analysis ◽  
Proximal Caries ◽  
Adolescents And Adults

scholarly journals Antioxidant agents for delaying diabetic kidney disease progression: A systematic review and meta-analysis

PLoS ONE ◽  
2017 ◽  
Vol 12 (6) ◽  
pp. e0178699 ◽  
Author(s):  
Davide Bolignano ◽  
Valeria Cernaro ◽  
Guido Gembillo ◽  
Rossella Baggetta ◽  
Michele Buemi ◽  
...  
Keyword(s):  
Systematic Review ◽  
Kidney Disease ◽  
Disease Progression ◽  
Diabetic Kidney Disease ◽  
Meta Analysis ◽  
Diabetic Kidney ◽  
Antioxidant Agents ◽  
Kidney Disease Progression
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