Early Use of Disease-Modifying Therapy May Prevent Diffuse Myocardial Fibrosis in Sickle Cell Anemia
Diffuse myocardial fibrosis is pervasive in sickle cell anemia (SCA) and strongly associated with diastolic dysfunction, but a small, single-institution study suggests early initiation and uninterrupted use of disease-modifying therapy for SCA can prevent this complication. The study investigators had previously observed diffuse myocardial fibrosis in every patient in an SCA cohort of 25 patients who ranged in age from 6 to 61 years. They used cardiac magnetic resonance imaging to quantify diffuse myocardial fibrosis in a cohort of SCA patients (N=7; mean age, 13.4 years) who have received uninterrupted treatment with hydroxyurea or chronic transfusion therapy since ≤4 years of age. They compared these findings with those observed in a control group without SCA (N=16).
Key findings were as follows:
- Only 1 of the 7 patients had evidence of mild diffuse fibrosis (extracellular volume fraction [ECV], 0.339), with the other 6 patients having no detectable disease (ECV, <0.304);
- Mean ECV was 0.283 ± 0.012, which was similar to that of normal controls and significantly lower than the ECV observed in individuals with SCA who have not received early uninterrupted therapy.
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Quinn CT, Niss O, Wood J, et al. Early initiation of disease-modifying therapy prevents diffuse myocardial fibrosis in sickle cell anemia. Presented at: 2018 American Society of Pediatric Hematology/Oncology; May 2-5, 2018; Pittsburgh, Pennsylvania. Abstract 319.