For those with lifelong bleeding, bruising, and/or low platelets but no diagnosis, the NHGRI RUNX1 Program can offer focused testing for those without the resources to get more comprehensive testing elsewhere at no cost. Enrollment in our study is also free of cost.

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Enrollment in our study is absolutely free. For those who can join us in Bethesda at the NIH we are able to provide transportation and lodging. And we cover the costs of all testing (which is only to your level of comfort and desired involvement) #RUNX1

We also encourage all of our study participants and their families to connect with RUNX1 Research Program where they will find an amazing community of caring providers, researchers, and most importantly other people also on their RUNX1 Journeys. #RUNX1 #HemeSky #Platelets

www.runx1-fpd.org

This not only deprives these patients of the best care and prevention, but blinds us to the full extent of RUNX1 biology.

Sadly, Dr. Bisi Lawal shows that minorities continue to struggle to receive attention at both the local and national level, and when they do, they are more likely to be the ones with more severe and/or advanced disease, or already progressed to malignancy. #ASH24 #HemeEquity

At an #ASH24 that saw the wonderful blinatumomab results, it is frightening that pediatric AIHA can have mortality rates approaching 10%. We need better diagnostics, understanding and treatments.

Importantly, side effects and reactions were as good as, if not better than, iron sucrose

Although there is confounding, such as lead time, HCT for HRF has a better prognosis than after the development of malignancy. Importantly, younger patients do better. Work like this will help us to come to better-honed consensus guidelines for treating SDS and others. #ASH24

In #SDS, the accumulation of TP53 clones over 3% VAF and other features (HRF) defines a high risk subgroup that may be more amenable to early intervention #ASH24