Emerging drugs in randomized controlled trials for sickle cell disease: are we on the brink of a new era in research and treatment?

Alessandro Matte, Maria Domenica Cappellini, Achille Iolascon, Federti Enrica, Lucia De Franceschi

Research output: Contribution to journalReview articlepeer-review

Abstract

Introduction: Sickle cell disease (SCD) is caused by a mutation in the HBB gene which is key for making a component of hemoglobin. The mutation leads to the formation of an abnormal hemoglobin molecule called sickle hemoglobin (HbS). SCD is a chronic, complex disease with a multiplicity of pathophysiological targets; it has high morbidity and mortality. Hydroxyurea has for many years been the only approved drug for SCD; hence, the development of new therapeutics is critical. Areas covered: This article offers an overview of the key studies of new therapeutic options for SCD. We searched the PubMed database and Cochrane Database of Systemic Reviews for agents in early phase clinic trials and preclinical development. Expert opinion: Although knowledge of SCD has progressed, patient survival and quality of life must be improved. Phase II and phase III clinical trials investigating pathophysiology-based novel agents show promising results in the clinical management of SCD acute events. The design of long-term clinical studies is necessary to fully understand the clinical impact of these new therapeutics on the natural history of the disease. Furthermore, the building of global collaborations will enhance the clinical management of SCD and the design of primary outcomes of future clinical trials.

Original languageEnglish
Pages (from-to)23-31
Number of pages9
JournalExpert Opinion on Investigational Drugs
Volume29
Issue number1
DOIs
Publication statusPublished - Jan 2 2020

Keywords

  • hemoglobinopathies
  • inflammatory vasculopathy
  • new treatment
  • Sickle cell disease

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)

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