ASH 2024 | Key challenges that remain in the treatment and management of Fanconi anemia
Agnieszka Czechowicz, MD, PhD, Stanford University, Stanford, CA, comments on the challenges in treating and managing Fanconi anemia, highlighting the need for earlier diagnosis and improved access to care. Dr Czechowicz emphasizes the importance of innovative treatments, such as cell and gene therapies, to address hematopoietic disease and reduce the incidence of solid tumors. This interview took place at the 66th ASH Annual Meeting and Exposition, held in San Diego, CA.
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Transcript
So I think diagnosis is still an issue. Patients get diagnosed much later than they should in many cases. And then access to care continues to be an issue. Patients that are in large treatment centers or close to large treatment centers get improved care, get access to more innovative treatments, have an easier time. We’ve really been trying to do a lot to expand those types of efforts to be able to bring patients to our institution and to other institutions to be able to provide top care through both telehealth opportunities as well as through various different resources to be able to bring patients to us...
So I think diagnosis is still an issue. Patients get diagnosed much later than they should in many cases. And then access to care continues to be an issue. Patients that are in large treatment centers or close to large treatment centers get improved care, get access to more innovative treatments, have an easier time. We’ve really been trying to do a lot to expand those types of efforts to be able to bring patients to our institution and to other institutions to be able to provide top care through both telehealth opportunities as well as through various different resources to be able to bring patients to us. So I think those continue to be large unmet needs and opportunities and then as I mentioned the improvement in the transplant treatments or cell and gene therapy treatments to really resolve the hematopoietic disease NFA is not present in the original text, it is likely that it should be NF or FA, likely referring to Fanconi anemia, is critically important and then unfortunately many patients still do develop solid tumors and so our hope is that through the improved use of new innovative cell and gene therapies that are much safer hopefully we can decrease or eliminate the use of these toxic agents and thereby decrease the malignancies that we see in these patients long term. Although there are also other programs that are looking at specifically improved treatment of those malignancies or even systemic gene therapies I think are part of the future of the treatment of this disease.
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