The Grndad Registry: Contemporary Natural History Data And An Analysis Of Real-World Patterns Of Use And Limitations Of Disease Modifying Therapy In Adults With Scd

BLOOD(2020)

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摘要
Incremental improvement in care for children with sickle cell disease (SCD), arising from government-funded research over the last 4 decades, resulted in a dramatically reduced childhood mortality. However, the impact of iterative research and disease modifying therapy (DMT) on adults with SCD has not been as strong. Until now, there has been no coordinated, longitudinal, generalizable, natural history study of SCD that allowed for an assessment of the contemporary adult population.
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