Evidence-Based Focused Review

Case 1. Kierra Malcom,† a 17 year old with homozygous sickle cell disease (HbSS), was on chronic erythrocytapheresis after she suffered a stroke at 8 years of age. With the intervention, her hemoglobin S level was consistently maintained between 25% and 35%. Iron chelation was commenced at the age of 10 years. She is currently on oral deferasirox and has a ferritin level of 1400 ng/mL. T2* liver magnetic resonance imaging is normal and indicates no ironoverload.Her functional score is appropriate forher age, except for poor school performance mostly attributed to frequent absences from school for sickle cell complication–related hospital admissions. Specifically, after a period of well-being for 2 to 3 years following commencement of erythrocytapheresis, she reports increasing pain symptoms over the last 5 or 6 years, chronic opioid use, and hospital admissions lasting 4 to 5 days each time, 4 to 6 times per year, interrupting her education and resulting in poor quality of life (QOL). She is keen to discuss hematopoietic stem cell transplantation (HSCT) because she has heard that it promises a cure. Herhematologist concurswith her desire tomeet the transplant team to discuss HSCT. Should Kierra undergo an HSCT? Case 2. Sonal Scher† is a 13-year-old adopted child with b thalassemia major who commenced chronic red cell transfusion therapy 8 months after birth. Iron chelation with subcutaneous desferrioxamine was started at 7 years of age following adoption. Oral chelation therapy was substituted for desferrioxamine 3 years ago, but her ferritin has remained consistently >3000 ng/mL, suggesting poor compliance. By liver magnetic resonance imaging, her hepatic iron concentrationwas calculated as 20mg/gdryweight. She is of small stature and has hepatosplenomegaly. The liver is palpable 3 cm below the right costal margin and the spleen is palpable 5 cm below the left. Otherwise, she appears to be in good health. Should Sonal undergo an HSCT? The purpose of this evidence-based review is to discuss the indications and outcomes of HSCT for sickle cell disease (SCD) and transfusion-dependent thalassemia.