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EBMT 2026 | Optimizing mobilization and apheresis in preparation for sickle cell gene therapy

In this video, Mohammed Essa, MD, Ministry of National Guard Health Affairs, Riyadh, Saudi Arabia, shares insights on optimizing mobilization and apheresis in preparation for gene therapy for sickle cell disease (SCD). Dr Essa highlights an approach that has been trialed at his institution, combining granulocyte colony-stimulating factor (G-CSF) with plerixafor for mobilization, resulting in improved collection of the target cell dose compared with plerixafor alone. This interview took place at the 52nd Annual Meeting of the EBMT in Madrid, Spain.

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Transcript

Thank you very much for this question. And actually, this is a very challenging and hot topic in the field because the different centers have been trying to modify and approach those patients by different manners. In our center, in collaboration with other centers as well, we have initiated, led by my colleague, Dr. Al-Sahrani, a protocol which included, in addition to Plerixafor as an agent, a very low dose G-CSF with a strict program with frequent exchange transfusion and minimizing the level of hemoglobin S to be less than 20 at all given times...

Thank you very much for this question. And actually, this is a very challenging and hot topic in the field because the different centers have been trying to modify and approach those patients by different manners. In our center, in collaboration with other centers as well, we have initiated, led by my colleague, Dr. Al-Sahrani, a protocol which included, in addition to Plerixafor as an agent, a very low dose G-CSF with a strict program with frequent exchange transfusion and minimizing the level of hemoglobin S to be less than 20 at all given times. And based on that, we have shared in our poster our data, starting from the initial challenges with first patients who we used the classic approach where patients were having challenges in mobilization. And finally, we find that we’ve been able to minimize the time to one cycle for those patients who are being mobilized for gene therapy. 

Now, another point to remember as well, there has been also changes and modification in the apheresis technique by our apheresis team, such as incorporation of unfractionated heparin in the circuit with the citrate, in addition to making sure that the patient is on antiplatelet or aspirin, preventing clotting, if that would help. These are the modifications that we’ve done so far. We know it’s a moving science and we also heard about our colleagues in the US using other agents with promising results as well. In addition to one small point that especially with our older adolescent patients who undergo collection is to use the fixed dose of plerixafor for those patients.

 

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Disclosures

Advisory board: Vertex.