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2023 ACMG Annual Clinical Genetics Meeting Digital ...
Survival in Eteplirsen-treated vs Duchenne Muscula ...
Survival in Eteplirsen-treated vs Duchenne Muscular Dystrophy Natural History Patients: An Indirect Treatment Comparison Using Real-world Data
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A study presented at the 2023 American College of Medical Genetics and Genomics Annual Meeting compared the survival of patients with Duchenne muscular dystrophy (DMD) receiving the drug eteplirsen to those receiving standard care. The study used real-world data from 579 eteplirsen-treated patients and compared them to patient-level data from published DMD natural history (NH) studies. <br /><br />The main findings from the study were as follows:<br /><br />1. Eteplirsen-treated patients had significantly longer survival compared to DMD NH controls, with a median difference of at least 5.4 years.<br /><br />2. Patients treated with eteplirsen for longer exposure periods may experience increasing survival benefits.<br /><br />3. Patients who initiate eteplirsen at younger ages may have higher survival benefits compared to those initiating at older ages. The survival differences were most evident for those initiating eteplirsen between ages 10-28 years and were attenuated past 28 years of age.<br /><br />The study also found that mortality rates were lower for eteplirsen-treated patients compared to DMD NH controls across all age intervals ranging from 5-45 years. The results were generally robust to sensitivity analyses with different cohorts included in the DMD NH control group.<br /><br />The study had some limitations, such as data limitations that did not allow for adjusted comparisons controlling for prognostic factors, and the analysis of age at event not matching patients with similar characteristics at treatment initiation, potentially leading to selection bias. However, the findings suggest that eteplirsen may prolong survival in patients with DMD across a wide age range.<br /><br />Further research is needed to confirm these findings and to investigate the impact of eteplirsen on other outcomes, such as ambulatory and pulmonary function.
Asset Subtitle
Presenting Author - Erin O’Rourke, MS, CGC; Co-Author - Joel Iff, PhD, PharmD; Co-Author - Nicolae Done, PhD; Co-Author - Ed Tuttle, MBA; Co-Author - Yi Zhong, PhD; Co-Author - Fangzhou Wei, MS; Co-Author - Basil T. Darras, MD; Co-Author - Craig McDonald, MD; Co-Author - Eugenio Mercuri, MD; Co-Author - Francesco Muntoni, MD;
Meta Tag
Databases
Musculoskeletal system
Therapy
X-Inactivation/X-Linked Disease
Co-Author
Joel Iff, PhD, PharmD
Co-Author
Nicolae Done, PhD
Co-Author
Ed Tuttle, MBA
Co-Author
Yi Zhong, PhD
Co-Author
Fangzhou Wei, MS
Co-Author
Basil T. Darras, MD
Co-Author
Craig McDonald, MD
Co-Author
Eugenio Mercuri, MD
Co-Author
Francesco Muntoni, MD
Presenting Author
Erin O’Rourke, MS, CGC
Keywords
Duchenne muscular dystrophy
eteplirsen
survival
patient-level data
real-world data
mortality rates
age at treatment initiation
prognostic factors
selection bias
ambulatory and pulmonary function
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