Spontaneous remission in high risk MDS/AML
Posted: Mon Jul 13, 2020 7:52 pm
High risk MDS/AML has a rare spontaneous remission rate. Here is what I found so far:
https://ashpublications.org/bloodadvanc ... -high-risk
They report a 64 year period with 46 cases each a little different. Its pretty rare. Not too sure about lower risk and MF but it makes me wonder if RWD might be applied to further analyze the PII MDS study since there are so many more patients that might be available for a purer "control" matching group. Geron has a highly skilled small group of statistical analysts. Clearly the FDA doesn't care and won't budge failing some catastrophic blood supply shortage, but the more thoughtful and reasonable Europeans might. We know the MDS study has not enrolled to the half way point yet (a promised PR when they do), and I wonder if some of the new sites (where are they by the way) might also be used to provide a RWD look that could be presented to the EU. Continuing to hope for conditional MA in EU but odds are unknown. One hopes reason prevails. It is a terrible thought to think patients will be waiting until 2024 for this drug. Is anyone listening to the KOL's? There is very little downside to a conditional MA while more data is collected. bp
https://ashpublications.org/bloodadvanc ... -high-risk
They report a 64 year period with 46 cases each a little different. Its pretty rare. Not too sure about lower risk and MF but it makes me wonder if RWD might be applied to further analyze the PII MDS study since there are so many more patients that might be available for a purer "control" matching group. Geron has a highly skilled small group of statistical analysts. Clearly the FDA doesn't care and won't budge failing some catastrophic blood supply shortage, but the more thoughtful and reasonable Europeans might. We know the MDS study has not enrolled to the half way point yet (a promised PR when they do), and I wonder if some of the new sites (where are they by the way) might also be used to provide a RWD look that could be presented to the EU. Continuing to hope for conditional MA in EU but odds are unknown. One hopes reason prevails. It is a terrible thought to think patients will be waiting until 2024 for this drug. Is anyone listening to the KOL's? There is very little downside to a conditional MA while more data is collected. bp