Prognosis of Patients With Heart Failure Receiving Autologous Myoblast Patchesã- Comparison of Single-Arm Trial Data to Registry Data.
Circ J
; 87(4): 481-486, 2023 03 24.
Article
em En
| MEDLINE
| ID: mdl-36384895
ABSTRACT
BACKGROUND:
Clinical studies in regenerative medicine remain insufficient in Japan due to ethical concerns regarding the control group and a lack of statistical methodology to evaluate efficacy in a small treatment group. This study evaluated the efficacy of autologous myoblast patch (AMP) treatment for heart failure using restricted mean survival time (RMST) analysis by comparing data from a small single-arm trial to epidemiological data from a registry.MethodsâandâResults:
The clinical trial arm included 55 patients with advanced ischemic cardiomyopathy who received an AMP between 2010 and 2020. The registry-based control group comprised 937 participants with severely impaired left ventricular function who were hospitalized for heart failure during the study period. Due to the limited number of patients, RMST analysis was used to compare survival between the 2 groups. Cox regression analyses revealed non-significant differences in survival between the groups at 3, 3.5, and 4 years. In contrast, RMST analyses revealed significant differences in survival at 3 years (P=0.008) and 3.5 (P=0.024) years, but not at 4 years.CONCLUSIONS:
This small single-arm trial using RMST analyses was able to detect the efficacy of AMP transplantation for advanced heart failure (compared with a registry-based control group), with better survival until 3.5 years. This approach may be useful for efficacy analyses in regenerative medicine, where traditional clinical trials are difficult.Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Isquemia Miocárdica
/
Insuficiência Cardíaca
Tipo de estudo:
Clinical_trials
/
Prognostic_studies
Limite:
Humans
Idioma:
En
Ano de publicação:
2023
Tipo de documento:
Article