Is taxomifene useful for preserving cardiac function in DMD?

The investigators of the TAMDMD trial, designed to study the possible beneficial effects of tamoxifen in Duchenne muscular dystrophy (DMD), have carried out a post-hoc analysis despite disappointing results concerning motor function:

• the trial involved 79 double-blind patients treated with oral tamoxifen,
• analysis of the echocardiographic data available for 14 of these patients (7 treated and 7 on placeboLe placebo est un produit dont la présentation est identique à celle d’un médicament, mais qui ne contient pas de principe actif. Dans un essai clinique, un placebo est utilisé pour mesurer l’action réelle du médicament testé, en comparant les effets du médicament testé contenant le principe actif et ceux du placebo.) showed stabilisation of systolic function and shortening fraction in the treated group at 48 weeks.

These encouraging results do not, however, allow us to draw any definitive conclusions, but they do open the door to future trials targeting cardiac function.

Tamoxifen may contribute to preserve cardiac function in Duchenne muscular dystrophy. Henzi BC, Lava SAG, Spagnuolo C et al. Eur J Pediatr. 2024 Jul 3.