Outcomes of patients with hereditary transthyretin-mediated amyloidosis with early onset V30M versus all other mutations in APOLLO, a Phase 3 study of patisiran - 27/03/19
Résumé |
Introduction |
hATTR is multi-systemic, life-threatening, caused by transthyretin gene mutations. Investigational RNAi therapeutic resulted in statistically significant improvement in neuropathy and Quality of Life measures compared to placebo in Phase 3.
Objective |
We evaluated patisiran efficacy and safety in patients with early onset V30M versus all other mutations.
Patients and methods |
APOLLO was a multi-center, international, randomized (2:1), double-blind study of patisiran 0.3mg/kg or placebo IV q3W in hATTR amyloidosis patients with polyneuropathy (NCT01960348). Primary endpoint was changed from baseline at 18-months in mNIS+7 with multiple secondary endpoints including Norfolk QOL-DN. Pre-specified subgroup analyses were conducted to evaluate patients with early onset V30M (≤50 years of age at onset) and those with all other mutations including late onset V30M (>50 years of age at onset).
Results |
APOLLO enrolled 225 patients with 39 different TTR mutations including 42.7% with V30M mutations with 10.2% considered to have early onset V30M disease. Similar to the overall patient population, patisiran demonstrated improvement in mNIS+7 and Norfolk QOL-DN compared to placebo in early onset V30M and as well as in all other mutations at 18-months.
Discussion |
Efficacy and safety data to be presented.
Conclusion |
Patisiran, investigated in patients with early and late onset V30M as well as a wide range of non-V30M genotypes, demonstrated consistent benefit over placebo in mNIS+7 and Norfolk QOL-DN.
Le texte complet de cet article est disponible en PDF.Mots clés : Mutations, ARNi therapeutic, Amylose héréditaire à transthyrétine
Plan
Vol 175 - N° S1
P. S129 - avril 2019 Retour au numéro
Article précédent
- Phase 1 study of ALN-TTRsc02, a subcutaneously administered investigational RNAi therapeutic for the treatment of transthyretin-mediated amyloidosis
- David Adams, Karsten Verena
- Fasciculations induites par les fluoroquinolones : une présentation clinique piégeuse
- Sacha Posener, Marie-Christine Durand, Benjamin Davido, Aurélien Dinh, Julie Paquereau
Bienvenue sur EM-consulte, la référence des professionnels de santé.
L’accès au texte intégral de cet article nécessite un abonnement.
Déjà abonné à cette revue ?