The approach of grading quality of evidence, as well as the strength of recommendations, offers a transparent, more comprehensive technique to develop clinical judgments regarding the use of diagnostic and test strategies. This particular paper utilizes the information captured in the two resources to establish their suitability in impacting the effective treatment and other beneficial forms of therapy by categorizing the evidence on particular levels.
Source 1
Gordon, L. B., Campbell, S. E., Massaro, J. M., D'Agostino Sr, R. B., Kleinman, M. E., Kieran, M. W., & Moses, M. A. (2018). Survey of plasma proteins in children with progeria pre-therapy and on-therapy with lonafarnib. Pediatric research , 83 (5), 982.
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This particular research employs the method of the longitudinal survey to investigate the plasma proteins in children to investigate the Hutchinson-Gilford progeria syndrome. The authors aim at initiating the development of a short-term and clinically meaning biomarkers for the evaluation of the status of the disease as well as improvement of the treatment. The study falls in level II of the evidence hierarchy because it employs the conceptual study approach (Horvath, 2009). The theoretical concepts are used in guiding the selection of the sample basing on the content of the literature. The study is only limited to a single group which comprised of children with the history of Hutchinson-Gilford progeria syndrome with a sample size of 24 individuals.
The study relies on the case report, which ranks the resource to a lower level of the evidence hierarchy. There is no comparison group considered in the research, and therefore, the study only gives outcomes for a single subgroup, which is the children with HGPS condition. This particular research poses the potential for the introduction of bias from the follow-up that is likely to utilize retrospective study designs. Besides the weakness presented in this study design, the findings are vital in studying this rare syndrome. As a result, it has a significant impact on the identification of relevant plasma protein biomarkers for children exhibiting the HGPs. The study can, therefore, be used to effectively design a hypothesis and providing more information about the ultra-rare disease which is associated with the particular procedures.
From the outcomes of the study and the above assessment, I feel the strength of the evidence is moderate. From the GRADE criteria provided by the U.S Preventive Services Task Force, an evidence that is categorized as moderate implies that the evidence provided is not sufficient to establish the effects of a preventive service on outcomes (GRADE Working Group 2004). The confidence, however, is limited by various factors, including limited generalizability of the findings to the routine primary care practice. The authors only provide data that can be used for further research. Also, a small sample of 24 individuals is used, which is not sufficient enough to make evidence-based conclusions.
Source 2
Mateos, J., Fafián-Labora, J., Morente-López, M., Lesende-Rodriguez, I., Monserrat, L., Ódena, M. A., ... & Arufe, M. C. (2018). Next-Generation Sequencing and Quantitative Proteomics of Hutchinson-Gilford progeria syndrome-derived cells point to the role of nucleotide metabolism in premature aging. PloS one , 13 (10),
The study approach for this particular research involved the use of human cell lines from patients with the HGPS and from the healthy parental controls, which were studies together using High-Resolution Quantitative Proteomics and Next-Generation Sequencing technique. This design is similar to the cohort study that is often conducted prospectively and usually follows two groups of the patients. The risk factor of interest characterizes the first group, while the other group does not. The research is, therefore, positioned at higher hierarchy because data collection, as well as the follow-up, is easily monitored and tries to make them as accurate and complete as possible.
The study type is a generalizable form of studies and involves use of the randomized trials which places this research into the higher levels of the evidence of hierarchy. The sampling process is focused on literature and theory, and it is extended due to the analysis aimed at capturing the diversity of experience. It employs analytic procedures, which are clear and comprehensive. Also, the background of the study links the study to relevant literature to establish the relevance of the investigation to other settings. Case studies involve the examination of the groups with a particular issue compared to the controls group using either qualitative or quantitative approach to enable studying the specific cause of an illness. This study involved the use of Human HGPS-derived fibroblast cell lines and healthy parental controls.
The research uses a randomized study design. There is consistency in the findings and techniques of investigation with those captured in the literature. It is established that de-regulation of LMNA promotes the down-regulation of a PRPS1 gene giving rise to a reduction in the levels of PRPS1 in HGPS. This particular finding is much more consistent with the literature. Overall, the randomized trial renders this type of evidence high and falls in level II with regards to the hierarchy of evidence. Doing other repeat trials and further research is less likely to change the level of confidence of the findings in studying the HGPS. The recommendations provided are much more useful than harmful because it is aimed at deepening the understanding of the association between de-regulation of purine and premature aging as well as investigating the therapeutic windows needed for better diagnosis and treatment of the HGPS.
References
Mateos, J., Fafián-Labora, J., Morente-López, M., Lesende-Rodriguez, I., Monserrat, L., Ódena, M. A., ... & Arufe, M. C. (2018). Next-Generation Sequencing and Quantitative Proteomics of Hutchinson-Gilford progeria syndrome-derived cells point to the role of nucleotide metabolism in premature aging. PloS one , 13 (10),
Mateos, J., Fafián-Labora, J., Morente-López, M., Lesende-Rodriguez, I., Monserrat, L., Ódena, M. A., ... & Arufe, M. C. (2018). Next-Generation Sequencing and Quantitative Proteomics of Hutchinson-Gilford progeria syndrome-derived cells point to the role of nucleotide metabolism in premature aging. PloS one , 13 (10),
GRADE Working Group. (2004). Grading quality of evidence and strength of recommendations. BMJ: British Medical Journal , 328 (7454), 1490.
Horvath, A. R. (2009). Grading quality of evidence and strength of recommendations for diagnostic tests and strategies. Clinical chemistry , 55 (5), 853-855.
