Current understanding in the management of sickle cell disease

Area of Science:

• Hematology
• Genetics
• Public Health

Background:

• Sickle cell disease (SCD) is the most common monogenetic disorder globally, posing a substantial public health challenge due to high morbidity and mortality.
• Advances in molecular and cellular biology have deepened the understanding of SCD pathophysiology and its complex, heterogeneous manifestations.

Purpose of the Study:

• To highlight modern management strategies for sickle cell disease (SCD).
• To discuss the impact of contemporary SCD treatments on patients' lives.

Main Methods:

• Review of natural history studies and clinical trials in SCD.
• Analysis of data on clinical features, complications, and severity predictors.
• Evaluation of recent disease-modifying therapies.

Main Results:

• Significant improvements in survival for SCD patients due to new therapies.
• Continued mortality, particularly in older patients, despite treatment advances.
• Ongoing challenges for healthcare providers in managing SCD.

Conclusions:

• Modern management has improved SCD patient survival.
• Addressing age-related complications and persistent challenges in SCD care is crucial.
• Further advancements are needed to overcome the remaining hurdles in SCD treatment and patient well-being.