USAResearch and clinical trials
Oral bitopertin did not correct anemia in DBA in a first study
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
In the USA, bitopertin was tested in 15 adults with DBA who did not respond to cortisone. All patients were receiving regular blood transfusions. Twelve patients completed eight months of treatment. The drug was well tolerated, but anemia did not improve in any patient. The researchers are planning a new study with slower dose increases.
What does it mean for patients and families?
This is a negative result, and the drug is not currently recommended for DBA. It was tested in a small group of people who had had the disease for a long time. The results of new studies should be awaited.
Source
Scientific paper: Results from the first clinical trial of bitopertin for steroid-relapsed/refractory Diamond-Blackfan anemia. Blood Red Cells & Iron, 2026. DOI: 10.1016/j.brci.2026.100090 · PubMed 42583025
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Diamond-Blackfan anemia (DBA)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.