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.
- 2020-10-06
- Clinical Trial · n = 55
- Pediatric blood & cancer 67(12)
- Adrianna Vlachos
- Evangelia Atsidaftos
- Mohammad Lutfi Lababidi
- Ellen Muir
- Zora R Rogers
- Waseem Alhushki
- Jonathan Bernstein
- Bertil Glader
- Barbara Gruner
- Helge Hartung
- Christine Knoll
- Thomas Loew
- Grzegorz Nalepa
- Anupama Narla
- Arun R Panigrahi
- Colin A Sieff
- Kelly Walkovich
- Jason E Farrar
- Jeffrey M Lipton
- PubMed: 33025707
- DOI: 10.1002/pbc.28748
- High evidence
- Clinical
Two subjects had a complete erythroid response and five had a partial response.
- Effect
- Beneficial
- Effect size
- Small
- Significant
- Yes
- Dose
- 700 mg/m^2 orally three times daily