Novel Insights into the Pathophysiology and Treatment of Sickle Cell Disease

Abstract

The polymerization of hemoglobin under deoxygenation is the main pathophysiological event in sickle cell diseases, described more than 70 years ago. The last two decades have seen a major increase in knowledge about the cascade of events that follow the polymerization of hemoglobin and the ensuing sickling of red blood cells. Several distinctive therapeutic targets have been discovered as a result, and a few drugs with innovative mechanisms of action are already on the market, while several others are the focus of ongoing trials. The aim of this narrative review is to describe some of the more recent data in the SCD literature regarding pathophysiology and novel treatments.

Keywords:

• Sickle cell disease
• pathophysiology
• treatment
• perspective

Acknowledgements

The authors would like to thank Dr Mariangela Correa, MD, PhD, for providing writing assistance on behalf of Springer Healthcare. This manuscript was prepared according to the International Society for Medical Publication Professionals–Good Publication Practice for Communicating Company-Sponsored Medical Research: the GPP3 Guidelines.

Disclosure statement

The authors report no conflicts of interest. The authors alone are responsible for the content and writing of this article.

Additional information

Funding

Funding to support the preparation of this manuscript was provided by Novartis. ASA declares receiving research grants and participating in Advisory Boards at Novartis. ACSP declares receiving honoraria from Novartis and EMS for consulting services. MSF declares receiving honoraria from Pfizer for consulting services. STOS has participated in clinical studies sponsored by Novartis. SFMG has participated in clinical studies sponsored by Novartis and Alexion as the principal investigator. The authors take full responsibility for the content and conclusions stated in this manuscript.