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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