L-leucine improves anemia and growth in patients with transfusion-dependent Diamond-Blackfan anemia: Results from a multicenter pilot phase I/II study from the Diamond-Blackfan Anemia Registry.
Adrianna VlachosEvangelia AtsidaftosMohammad Lutfi LababidiEllen MuirZora R RogersWaseem AlhushkiJonathan BernsteinBertil GladerBarbara GrunerHelge HartungChristine KnollThomas LoewGrzegorz NalepaAnupama NarlaArun R PanigrahiColin A SieffKelly WalkovichJason E FarrarJeffrey M LiptonPublished in: Pediatric blood & cancer (2020)
L-leucine is safe, resulted in an erythroid response in 16% of subjects with DBA, and led to an increase in weight and linear growth velocity in 36% and 44% of evaluable subjects, respectively. Further studies will be critical to understand the role of L-leucine in the management of patients with DBA.