June 2022. Volume 18. Number 2

Cell therapy appears to stop deterioration of upper limb and heart functions in Duchenne muscular dystrophy

 
 
 
 
 
 
 
 
 
 
Rating: 0 (0 Votes)
Newsletter Free Subscription
Regularly recieve most recent articles by e-mail
Subscribe
Print
Add to library
Discuss this article

AVC | Critically appraised articles

McDonald CM, Marbán E, Hendrix S, Hogan N, Ruckdeschel Smith R, Eagle M, et al. HOPE-2 Study Group. Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial. Lancet. 2022; 399:1049-58.

Reviewers: Cuestas Montañés EJ1, Pérez-Moneo Agapito B2.
1Servicio de Pediatría y Neonatología. Hospital Privado Universitario de Córdoba. Córdoba. Argentina.
2Servicio de Pediatría. Hospital Universitario Infanta Leonor. Universidad Complutense de Madrid. Madrid. España.
Correspondence: Eduardo José Cuestas Montañés. Email: eduardo.cuestas@gmail.com
Reception date: 18/05/2022
Acceptance date: 25/05/2022
Publication date: 01/06/2022

Abstract

Authors´conclusions: CAP-1002 cell therapy appears to be safe and effective in reducing deterioration of upper limb function in patients with late-stage Duchenne muscular dystrophy. Various measures of cardiac function and structure were also improved in the CAP-1002 group compared with the placebo group. Longer-term extension studies are needed to confirm the therapeutic durability and safety of CAP-1002 beyond 12 months for the treatment of skeletal myopathy and cardiomyopathy in Duchenne muscular dystrophy.

Reviewers´commentary: this clinical trial has shown that cellular therapy is safe and effective in stopping the deterioration of upper limb and heart functions in patients with late-stage Duchenne muscular dystrophy.

How to cite this article

Cuestas Montañés EJ, Pérez-Moneo Agapito B. La terapia celular parece detener el deterioro de las funciones de las extremidades superiores y del corazón en la distrofia muscular de Duchenne. Evid Pediatr. 2022;18:16.

AVC | Critically appraised articles

McDonald CM, Marbán E, Hendrix S, Hogan N, Ruckdeschel Smith R, Eagle M, et al. HOPE-2 Study Group. Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial. Lancet. 2022; 399:1049-58.

Reviewers: Cuestas Montañés EJ1, Pérez-Moneo Agapito B2.
1Servicio de Pediatría y Neonatología. Hospital Privado Universitario de Córdoba. Córdoba. Argentina.
2Servicio de Pediatría. Hospital Universitario Infanta Leonor. Universidad Complutense de Madrid. Madrid. España.
Correspondence: Eduardo José Cuestas Montañés. Email: eduardo.cuestas@gmail.com
Reception date: 18/05/2022
Acceptance date: 25/05/2022
Publication date: 01/06/2022

How to cite this article

Cuestas Montañés EJ, Pérez-Moneo Agapito B. La terapia celular parece detener el deterioro de las funciones de las extremidades superiores y del corazón en la distrofia muscular de Duchenne. Evid Pediatr. 2022;18:16.

01/06/2022

Linked Comment