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The Use of Novel Therapies to Reconstitute Blood Cell Production and Promote Organ Performance, Using Bone Marrow Failure as a Model

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Technical Report,28 Sep 2010,27 Sep 2017

Corporate Author:

Feinstein Institute for Medical Research Manhasset United States

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This study was a Phase III 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 72014 and the last patient received study drug until 22017. 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.

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  • Medicine and Medical Research

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