Abstract
Sickle cell disease (SCD) is an inherited red blood cell disorder that leads to substantial morbidity and early mortality. Acute and chronic SCD-related complications increase with older age, and therapies are urgently needed to treat adults. Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative therapy, but has been used less frequently in adults compared to children. This is, in part, due to (1) greater chronic organ damage, limiting tolerability to myeloablative conditioning regimens, (2) a higher rate of HSCT-related complications in adults versus children with SCD, and (3) limited coverage by public and private health insurance. Newer approaches using nonmyeloablative and reduced-intensity conditioning HSCT regimens have demonstrated better safety and tolerability, with high rates of stable engraftment in SCD adults. This review will focus on the impacts of HSCT, using more contemporary approaches to SCD-related complications in adults.
Highlights
Sickle cell disease (SCD) is among the most common monogenetic disorders, affecting approximately 25 million people worldwide [1]
The Comprehensive Study of Sickle Cell Disease (CSSCD) cohort study demonstrated that the median survival for men and women was 42 and 48 years in Hb SS SCD and 60 and 68 years in Hb SC SCD, respectively [4]
The tricuspid regurgitant jet velocity (TRJV) is a noninvasive marker of systolic pulmonary artery blood pressure obtained by transthoracic echocardiography that is elevated in SCD patients with pulmonary hypertension or diastolic dysfunction
Summary
Sickle cell disease (SCD) is among the most common monogenetic disorders, affecting approximately 25 million people worldwide [1]. Sickle cell disease is due to homozygous inheritance of the hemoglobin S mutation (Hb SS) or compound heterozygous inheritance of the Hb S mutation with another β-globin chain abnormality, such as Hb C (Hb SC) or β-thalassemia (Hb Sβ+-thalassemia, Hb Sβ0-thalassemia). Hb S polymerizes, resulting in vaso-occlusion, chronic red blood cell hemolysis, acute and chronic organ complications, and reduced overall survival [2]. Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative therapy that has been applied more frequently in children than adults with SCD [3]
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Free) 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call
