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Real-world data on spinal muscular atrophy in Spain: Insights from over 500 individuals in the CuidAME project

Fecha de publicación: Fecha Ahead of Print:

Autores de IIS La Fe

Participantes ajenos a IIS La Fe

  • Puig-Ram, C
  • Segovia, S
  • Garcia-Uzquiano, R
  • Romero, MG
  • Expósito-Escudero, JM
  • Carrera-García, L
  • López-Lobato, M
  • Paradas, C
  • Mera, LG
  • Molinero, MA
  • Andrés, DG
  • Toro, E
  • Ramos, JAF
  • Grimalt, MA
  • de Laguna, LTB
  • Barrios, DG
  • Tizzano, EF
  • Cattinari, MG
  • Medina, J
  • Medina, RC
  • Munell, F
  • Sotoca, J
  • Martínez-Salcedo, E
  • Escribano, AM
  • Panadés, MP
  • Fernández-García, MA
  • Benito, DND
  • Nascimento, A

Grupos

Abstract

Background The new treatment paradigm in Spinal Muscular Atrophy (SMA) has introduced novel phenotypes, changes in trajectories and clinical questions not fully addressed in clinical trials. To explore these challenges, several international initiatives have emerged. CuidAME was created as a nationwide clinical network in Spain designed to standardise SMA care, facilitate knowledge sharing, and capture data in a longitudinal comprehensive registry. Objective Evaluating the usefulness of the CuidAME project to capture data in a real-world setting. Methods This multicentric cohort study includes individuals with SMA followed at participating hospitals. Clinical examinations and multidisciplinary assessments were performed during routine clinical visits. We present a cross-sectional analysis of the registry population. Results As of February 2025, 543 participants from 25 hospitals were recruited: 12 were presymptomatic, 125 (23%) had SMA type 1, 208 (35%) type 2, 194 (38%) type 3, and 4 (<1%) type 4. Among the cohort, 91% (n = 495) had received at least one disease-modifier treatment, with 17 discontinuations. The registry included 5092 motor assessments, 1565 performed before treatment initiation. Conclusions CuidAME is an academic, longitudinal, real-world data collection project that demonstrated a fast and effective model for implementation facilitating the standardization of clinical practices and outcome measures across Spain. By aligning with core dataset used in other registries and establishing multidisciplinary working groups, the initiative will contribute to sharing knowledge to advance SMA care and improve patient outcomes.

Datos de la publicación

ISSN/ISSNe:
2214-3599, 2214-3602

Journal of Neuromuscular Diseases  SAGE PUBLICATIONS INC

Tipo:
Article
Páginas:
837-848
PubMed:
40726133
Factor de Impacto:
1,054 SCImago ℠
Cuartil:
Q1 SCImago ℠

Citas Recibidas en Web of Science: 4

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Keywords

  • spinal muscular atrophy; multicentre; prospective; registry; real-world evidence; real-life outcome data; neuromuscular disease; motor function scale; long-term follow-up; orphan drugs

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