FOR YOUR INFORMATION

The DBA Registry is planning another clinical trial. 

We are pursuing a second clinical trial using a higher dose of L-leucine for both transfusion dependent patients AND steroid dependent patients!

The results of our pilot trial of leucine were quite promising (https://onlinelibrary.wiley.com/doi/abs/10.1002/pbc.28748). We had 43 patients who completed the trial. There were no significant adverse events in any of our patients. Two patients had a complete response and no longer require transfusions and 5 patients showed an increase in their reticulocyte count. Of the patients with growth potential 44% showed an increase in growth velocity. Given the safety of L-leucine along with these results we plan to pursue our next clinical trial using an even higher dose of leucine.

 

Who May Be Eligible?

• Patients that have been transfusion dependent for at least 6 months,

• Patients who have baseline hemoglobin less than or equal to 9.5 g/dL and are taking more than 0.3 mg/kg/day of steroid medication OR greater than 20 mg of Prednisone a day for more than 6 months’ duration

• Patients must be at least 2 years of age

• Patients must not be on L-leucine for at least 3 months to be allowed to enroll on this study

As always, we thank each of you for your help in advancing the treatment of DBA. We look forward to the start of this clinical trial.

If you are interested in learning more about this exciting trial and be on the mailing list when it opens, please email at DBARegistry@northwell.edu. You will be contacted as soon as the trial becomes available for enrollment. 

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Thank you again and stay safe,

Adrianna Vlachos, MD

Director, Diamond Blackfan Anemia Registry