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← Disease database

sickle cell disease due to hemoglobin S and a non-S/non-C hemoglobin variant

Sickle cell disease (SCD) is a group of inherited hemoglobinopathies caused by a mutation in HBB that produces hemoglobin S (HbS). HbS polymerizes under deoxygenation, deforming erythrocytes into rigid sickle shapes that cause hemolytic anemia, vaso-occlusion, and end-organ injury. Severity spans silent carrier status to life-threatening acute complications and progressive chronic organ damage.

Therapeutic area
Hematology · Rare Disease
Rarity
Rare
Prevalence
~100k US / ~20M globally (est.)
MONDO
MONDO:0979354

Also known as: SCD · sickle cell anemia · HbSS · HbSC · HbS/beta-thalassemia

Overview

Sickle cell disease encompasses hemoglobin genotypes that include at least one HbS allele paired with another beta-globin variant (classically HbSS, HbSC, HbS/beta-thalassemia, and rarer compound heterozygotes). The prototypical HbSS form is the most common and generally most severe.

The central pathophysiologic events are intravascular hemolysis, microvascular vaso-occlusion, and a pro-inflammatory, pro-thrombotic vascular phenotype. Acute vaso-occlusive episodes (VOEs) cause severe pain; chronic complications include stroke, acute chest syndrome, avascular necrosis, nephropathy, retinopathy, and pulmonary hypertension.

Mechanism classesgene therapycrispr

Content last reviewed: 2026-05