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 Lipton

Published 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.

Keyphrases

iron deficiency
chronic kidney disease
body mass index
physical activity
cardiac surgery
weight loss
cross sectional
weight gain
study protocol
blood flow
body weight