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1.
Resuscitation ; 187: 109799, 2023 06.
Artigo em Inglês | MEDLINE | ID: mdl-37080335

RESUMO

Meta-analyses and systematic reviews (MSR) have been conceived as tools to summarize evidence on a specific health question. However, in the last years, an exaggerated number of MSRs published by scientific journals has been observed, i.e. 286 MSRs in the field of Resuscitation Science over the last 3 years, i.e. approximately 95 per year. Thus, doubts on the real scientific need of such a high number of MSRs may arise, potentially being only a way to rapidly improve authors' citation index and fame and sometimes the journals' impact factor.


Assuntos
Reanimação Cardiopulmonar , Fator de Impacto de Revistas , Humanos
2.
Orphanet J Rare Dis ; 17(1): 48, 2022 02 14.
Artigo em Inglês | MEDLINE | ID: mdl-35164810

RESUMO

BACKGROUND: Metachromatic Leukodystrophy (MLD) is a rare lysosomal disorder. Patients suffer from relentless neurological deterioration leading to premature death. Recently, new treatment modalities, including gene therapy and enzyme replacement therapy, have been developed. Those advances increase the need for high-quality research infrastructure to adequately compare treatments, execute post-marketing surveillance, and perform health technology assessments (HTA). To facilitate this, a group of MLD experts started the MLD initiative (MLDi) and initiated an academia-led European MLD registry: the MLDi. An expert-based consensus procedure, namely a modified Delphi procedure, was used to determine the data elements required to answer academic, regulatory, and HTA research questions. RESULTS: Three distinct sets of data elements were defined by the 13-member expert panel. The minimal set (n = 13) contained demographics and basic disease characteristics. The core set (n = 55) included functional status scores in terms of motor, manual, speech and eating abilities, and causal and supportive treatment characteristics. Health-related quality of life scores were included that were also deemed necessary for HTA. The optional set (n = 31) contained additional clinical aspects, such as findings at neurological examination, detailed motor function, presence of peripheral neuropathy, gall bladder involvement and micturition. CONCLUSION: Using a modified Delphi procedure with physicians from the main expert centers, consensus was reached on a core set of data that can be collected retrospectively and prospectively. With this consensus-based approach, an important step towards harmonization was made. This unique dataset will support knowledge about the disease and facilitate regulatory requirements related to the launch of new treatments.


Assuntos
Leucodistrofia Metacromática , Consenso , Humanos , Leucodistrofia Metacromática/genética , Qualidade de Vida , Sistema de Registros , Estudos Retrospectivos
3.
Sci Rep ; 9(1): 3580, 2019 03 05.
Artigo em Inglês | MEDLINE | ID: mdl-30837662

RESUMO

Cardiac Magnetic Resonance (CMR) is the gold standard for left ventricular (LV) function assessment in small rodents and, though echocardiography (ECHO) has been proposed as an alternative method, LV volumes may be underestimated when marked eccentric remodeling is present. In the present study we described a novel echocardiographic method and we tested the agreement with CMR for LV volumes and ejection fraction calculation in mice with experimental myocardial infarction. Sham-operated and infarcted mice, subjected to Coronary Artery Ligation, underwent ECHO and CMR. Volumes and ejection fraction were calculated by ECHO using a standard Simpson's modified method (ECHO pLAX) or a method from sequential parasternal short axis (ECHO pSAX) acquired mechanically by translating the probe every 1 mm along the left ventricle. The mean differences ±1.96 standard deviation near to zero suggested close agreement between ECHO pSAX and CMR; contrarily ECHO pLAX agreement with CMR was lower. In addition, ECHO was three times shorter and cheaper (Relative cost difference: pLAX: -66% and pSAX -57%) than CMR. In conclusion, ECHO pSAX is a new, fast, cheap and accurate method for LV function assessment in mice.


Assuntos
Ecocardiografia , Coração/diagnóstico por imagem , Imageamento por Ressonância Magnética , Infarto do Miocárdio/diagnóstico por imagem , Infarto do Miocárdio/fisiopatologia , Função Ventricular Esquerda , Animais , Camundongos
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