Optimal Design of Clinical Trials Involving Persons with Disorders of Consciousness.
Neurocrit Care
; 40(1): 74-80, 2024 Feb.
Article
en En
| MEDLINE
| ID: mdl-37535178
ABSTRACT
BACKGROUND:
Limited data exist regarding the optimal clinical trial design for studies involving persons with disorders of consciousness (DoC), and only a few therapies have been tested in high-quality clinical trials. To address this, the Curing Coma Campaign Clinical Trial Working Group performed a gap analysis on the current state of clinical trials in DoC to identify the optimal clinical design for studies involving persons with DoC.METHODS:
The Curing Coma Campaign Clinical Trial Working Group was divided into three subgroups to (1) review clinical trials involving persons with DoC, (2) identify unique challenges in the design of clinical trials involving persons with DoC, and (3) recommend optimal clinical trial designs for DoC.RESULTS:
There were 3055 studies screened, and 66 were included in this review. Several knowledge gaps and unique challenges were identified. There is a lack of high-quality clinical trials, and most data regarding patients with DoC are based on observational studies focusing on patients with traumatic brain injury and cardiac arrest. There is a lack of a structured long-term outcome assessment with significant heterogeneity in the methodology, definitions of outcomes, and conduct of studies, especially for long-term follow-up. Another major barrier to conducting clinical trials is the lack of resources, especially in low-income countries. Based on the available data, we recommend incorporating trial designs that use master protocols, sequential multiple assessment randomized trials, and comparative effectiveness research. Adaptive platform trials using a multiarm, multistage approach offer substantial advantages and should make use of biomarkers to assess treatment responses to increase trial efficiency. Finally, sound infrastructure and international collaboration are essential to facilitate the conduct of trials in patients with DoC.CONCLUSIONS:
Conduct of trials in patients with DoC should make use of master protocols and adaptive design and establish international registries incorporating standardized assessment tools. This will allow the establishment of evidence-based practice recommendations and decrease variations in care.Palabras clave
Texto completo:
1
Bases de datos:
MEDLINE
Asunto principal:
Trastornos de la Conciencia
/
Lesiones Traumáticas del Encéfalo
Tipo de estudio:
Clinical_trials
/
Guideline
/
Observational_studies
Límite:
Humans
Idioma:
En
Revista:
Neurocrit Care
Asunto de la revista:
NEUROLOGIA
/
TERAPIA INTENSIVA
Año:
2024
Tipo del documento:
Article
País de afiliación:
Estados Unidos