EBMT 2025 | Bridging therapy options for patients with aplastic anemia and selecting between them

So, bridging therapy is essentially very important for patients with severe aplastic anemia while awaiting allogeneic transplant or when a suitable donor is not immediately available. The goal is to maintain blood counts, to prevent infections and bleeding, and stabilize the patient until definitive therapy can be pursued. 

So, first one is immunosuppressive therapy. This is best for patients without an immediate HLA-matched sibling donor or other patients where allogeneic may not be the first choice, or patients who decline or are not fit for transplantation. The most common immunosuppressive therapy regimens are with ATG, cyclosporine, and eltrombopag. This is the standard first-line therapy. We can obtain around 60-70% of response rates at six months, but it’s a slow set of action, it can take around three months. And up to 30% of responders may relapse. So 10-15 may develop clonal evolution. And this is the preferred option in patients with above 40 years or without a matched donor. 

Another option is the supportive care. So this is the best therapy for patients who are waiting for a transplant or response to immunosuppressive therapy or patients who are unfit for immunosuppressive therapy or transplantation. This includes blood transfusions, antimicrobial prophylaxis, and also growth factors. 

Also, another option, it can be the androgen therapy with danazol. This is an option for patients who cannot tolerate immunosuppressive therapy or require long-term non-transplant options. So, older patients with non-severe aplastic anemia who are non-transplant candidates, and also for those patients that immunosuppressive therapy has failed, and another option is needed. We have to consider that this is a very limited effectiveness, so it can provide a response in around 50% of the cases. It has also important side effects such as virilization, fluid retention, or hepatotoxicity. It’s not a first-line option but can be used in some selected cases.

So I would say that in conclusion, bridging therapy in aplastic anemia should be tailored and based on donor availability, the patient’s age, the severity of the cytopenias, and the overall fitness for transplantation. So immunosuppressive therapy remains the standard for patients lacking an immediate matched sibling donor. Supportive care is essential in all cases, but should be minimized to avoid transfusion-related complications, and lastly, stem cell transplantation should not be delayed unnecessarily, especially in younger patients.

This transcript is AI-generated. While we strive for accuracy, please verify this copy with the video.