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EBMT 2025 | The benefits of GCSF-primed bone marrow as a graft source in transplantation for severe thalassemia
Lawrence Faulkner, MD, Cure2Children Foundation, Florence, Italy, discusses the potential of granulocyte-colony stimulating factor (GCSF)-primed bone marrow as a source of transplantable stem cells that can decrease the risk of graft-versus-host disease (GvHD), a major transplant-related complication in patients receiving a transplant for severe thalassemia. Dr Faulkner highlights that this graft source can benefit both the donor and recipient by reducing bone marrow collection volumes and speeding up hematological recovery. This interview took place at the 51st Annual Meeting of the EBMT in Florence, Italy.
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Transcript
The reason why we focused on granulocyte-colony stimulating factor primed bone marrow is because it is a source of transplantable stem cells, which is known to be associated with decreased graft-versus-host disease. Especially chronic graft-versus-host disease, which is something that for benign diseases like hemoglobinopathies, it’s a problem. And even more relevant in developing countries where having graft-versus-host disease is a major problem, both in terms of finances and in terms of health-related quality of life...
The reason why we focused on granulocyte-colony stimulating factor primed bone marrow is because it is a source of transplantable stem cells, which is known to be associated with decreased graft-versus-host disease. Especially chronic graft-versus-host disease, which is something that for benign diseases like hemoglobinopathies, it’s a problem. And even more relevant in developing countries where having graft-versus-host disease is a major problem, both in terms of finances and in terms of health-related quality of life. So that, in some instances, having a moderate to severe chronic graft-versus-host disease can make your quality of life worse than the hemoglobinopathy itself. And that’s why we try to minimize the chance of that happening.
For the donor, because essentially it enriches the donor’s bone marrow, so you can decrease collection volumes. And especially in children, I am a pediatrician, this is very important, particularly for thalassemia, and because most of the sibling donors are thalassemia carriers, so are mildly anemic, and of course we want to avoid giving a third-party blood transfusion to donors. And actually, we essentially never transfuse a donor. So decreasing bone marrow collection volume in donor is the first point for the donor. For the recipient, of course, it decreases the chance of getting graft-versus-host disease, and it also speeds up engraftment because stem cells are sort of exposed to GCSF, so they are primed and will give a faster hematological recovery.
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