ASH 2022 | Prospective study evaluating complement activation in patients with sickle cell disease
Eleni Gavriilaki, MD, PhD, G Papanikolaou Hospital, Thessaloniki, Greece, discusses a study which evaluated the impact of complement activation in patients with sickle cell disease (SCD). Increased complement activation was observed in patients that developed SCD complications, and these findings may allow clinicians to detect which patients are likely to benefit from complement inhibitors at an earlier stage. This interview took place at the 64th ASH Annual Meeting and Exposition congress in New Orleans, LA.
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Transcript (edited for clarity)
We’re very proud to present this abstract on sickle cell disease. It was the primary outcome of our ASH Global Research Award that we received two years ago. And so we looked at patients with sickle cell disease at steady state, and then during the follow-up. Some of them had serious complications so we took a sample during the complications, some of them did not...
We’re very proud to present this abstract on sickle cell disease. It was the primary outcome of our ASH Global Research Award that we received two years ago. And so we looked at patients with sickle cell disease at steady state, and then during the follow-up. Some of them had serious complications so we took a sample during the complications, some of them did not. So we took a sample just on the regular follow-up and we compared these patients. So we found increased complement activation at steady state in patients that then developed a complication. And we also found a marker of complement activation, which is soluble C5b-9 which was significantly increased during the complication and was also significantly increased from the beginning in some of the patients that then developed the complication.
So the main point from that is that maybe we can use this marker to characterize patients that will benefit from complement inhibitors. And we know that complement inhibitors are safe and effective, and we have published in the past that we just need a small dose of complement inhibitor to overcome such a complication of sickle cell disease.
And to support our results we also did genetic analysis showing that there is complement dysregulation at the genetic level of these patients and it is associated with our functional assays as expected. So sickle cell disease, and especially its complications, is an area that might need to have further studies on the role of complement inhibitors in the future.
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