The Use of Novel Therapies to Reconstitute Blood Cell Production and Promote Organ Performance, Using Bone Marrow Failure as a Model
Abstract
This study was a Phase I/II study to study the use of L-leucine in subjects with transfusion-dependent Diamond Blackfan anemia, a rare, inherited bone marrow failure syndrome. The study accrued subjects appropriately and closed to accrual in May 2016. In total the study opened to patient accrual in 7/2014 and the last patient received study drug until 2/2017. There were 55 patients consented; 12 screen failures; 43 patients evaluable. There were 21 males; the median age was 10 years 4 months. No untoward side effects were attributable to L-leucine. Two patients had a complete remission of their anemia and 5 patients had a partial remission with elevated reticulocyte counts. Ten of the 22 patients with growth potential and complete data had an average increase of 8 percentile in growth velocity, independent of the hematologic response at the end of treatment.
Document Details
- Document Type
- Technical Report
- Publication Date
- Dec 01, 2017
- Accession Number
- AD1104068
Entities
People
- Adrianna Vlachos
Organizations
- The Feinstein Institute for Medical Research