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Publications

Journal Article Research Support, Non-U.S. Gov't Epublish 1 citation

PubMed: 36746535 DOI: 10.1136/bmjopen-2022-064872

Efficacy and safety of pharmacological interventions for managing sickle cell disease in children and adolescents: protocol for a systematic review with network meta-analysis.

BMJ Open 13(2):e064872

Authors: Fernanda S Tonin, Catarina Ginete, Fernando Fernandez-Llimos, Joana Ferreira, Mariana Delgadinho, Miguel Brito

Affiliations

  • Health & Technology Research Center (H&TRC), Escola Superior de Tecnologia da Saúde de Lisboa, Instituto Politécnico de Lisboa, Lisbon, Portugal.
  • Pharmaceutical Sciences Postgraduate Programme, Federal University of Paraná, Curitiba, Brazil.
  • Laboratory of Pharmacology, Department of Drug Sciences, Faculty of Pharmacy, University of Porto, Porto, Portugal.
  • Health & Technology Research Center (H&TRC), Escola Superior de Tecnologia da Saúde de Lisboa, Instituto Politécnico de Lisboa, Lisbon, Portugal.

Abstract

INTRODUCTION

Sickle cell disease (SCD), an inherited haemoglobinopathy, has important impact on morbidity and mortality, especially in paediatrics. Previous systematic reviews are limited to adult patients or focused only on few therapies. We aim to synthesise the evidence on efficacy and safety of pharmacological interventions for managing SCD in children and adolescents.

METHODS AND ANALYSIS

This systematic review protocol is available at Open Science Framework (doi:10.17605/OSF.IO/CWAE9). We will follow international recommendations on conduction and report of systematic reviews and meta-analyses. Searches will be conducted in PubMed, Scopus and Web of Science (no language nor time restrictions) (first pilot searches performed in May 2022). We will include randomised controlled trials comparing the effects of disease-modifying agents in patients with SCD under 18 years old. Outcomes of interest will include: vaso-occlusive crisis, haemoglobin levels, chest syndrome, stroke, overall survival and adverse events. We will provide a narrative synthesis of the findings, and whenever possible, results will be pooled by means of pairwise or Bayesian network meta-analyses with surface under the cumulative ranking curve analyses. Different statistical methods and models will be tested. Dichotomous outcomes will be reported as OR, risk ratio or HR, while continuous data will be reported as standard mean differences, both with 95% CI/credibility interval. The methodological quality of the trials will be evaluated using the Risk of Bias 2.0 tool, and the certainty of the evidence will be assessed with the Grading of Recommendations Assessment, Development and Evaluation approach.

ETHICS AND DISSEMINATION

This study refers to a systematic review, so no ethics approval is necessary. We intent to publish our findings in international, peer-reviewed journal. Data will also be presented to peers in scientific events. Additionally, the results obtained in this study may contribute towards the update of therapeutic guidelines and for the development of health policies for SCD.

PROSPERO REGISTRATION NUMBER

CRD42022328471.

MeSH

Cells, Safety, Anemia, Sickle Cell, Adolescent, Network Meta-Analysis, Therapeutics, Child