ISAL 2025 | The biggest challenges in managing APL: differentiation syndrome and early deaths

Yeah, there are some still open challenges and I would say one is differentiation syndrome and this is due to the differentiating agents ATRA and ATO that we are now using more and more. We have learned to deal with this complication which is strongly associated with leukocytosis and also to coagulopathy, the typical APL coagulopathy. We have learned how to prevent these by using low-dose steroids and to treat it by increasing the dose. And indeed, also in our Apollo trial, we didn’t have any deaths due to differentiation syndrome by adopting these measures, but it is still a challenge for clinicians, having a patient with high white blood cell counts and sometimes needing ventilation because of lung complications. The second big issue, and this is still a big issue, are early deaths in APL. These are known to occur during the first month after diagnosis, but there are also cases occurring in the peri-diagnostic period and even before the diagnosis is made. They’re usually due to bleeding complications and they may still be about 15 to 20 percent of the patients with APL who may die of early death. So in this case, the challenge is to diagnose it as quickly as possible and start supportive treatment with plasma derivatives and ATRA, and also cytoreductive treatment to avoid bleeding complications and platelet support, of course. The second cause of early death in these patients is infectious complications, but these are, I would say, easier to deal with. The thing we are most worried about are those patients who cannot profit from this so efficient treatment because of a lack of diagnosis or a delayed diagnosis.

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