Eteplirsen May Prolong Survival in Patients With Duchenne Muscular Dystrophy

Treatment with eteplirsen is associated with higher survival among patients with Duchenne muscular dystrophy (DMD) compared with those who received no treatment, according to study results presented at the 2023 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting, held from November 1 to 4 in Phoenix, Arizona.

Compared with patients with DMD who do not receive treatment, those treated with eteplirsen experience a delay in ambulatory and pulmonary decline. For the study, researchers assessed the survival of patients with DMD who received eteplirsen vs those who received no treatment, or natural history control individuals.

The researchers compared survival among patients with DMD treated with eteplirsen as routine care vs those with DMD natural history by utilizing real world data from 2 US-based and 2 European studies.

Via the digitization of Kaplan-Meier curves, the researchers replicated patient data. To compare survival age between the 2 patient groups, they used unadjusted Kaplan-Meier curves, log-rank tests, Cox models, and parametric specifications. With the help of a simulation, the researchers randomly matched each patient who received eteplirsen alive at age of treatment initiation with up to 15 natural history control individuals to compare time from treatment initiation to death. They adjusted for baseline age and age-treatment interaction.

The study included a total of 579 patients (mean age at initiation, 11.9 years; mean exposure, 3.7 years) with DMD who received eteplirsen.

Compared with natural history control individuals, those treated with eteplirsen had a higher median age at death (32.8 vs 27.4 years; P <.0001) and a prolonged median survival rate of 5.4 years. Moreover, survival was 66% higher among patients treated with eteplirsen compared with natural history control individuals (HR, 0.64; 95% CI, 0.23-0.50; P <.001). The researchers noted that younger age at initiation and longer treatment exposure to eteplirsen were both independently associated with prolonged survival.

Across 5-year segments, spanning from 5 to 45 years, mortality rates were lower in patients who received eteplirsen compared with those who received no treatment.

“These real-world data suggest that eteplirsen may prolong survival in patients with DMD across a wide age range,” the researchers concluded.

The study was limited by the inability to perform adjusted comparison controlling for prognostic factors due to data limitations.

Disclosures: Some study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.