[CIS PIDD] [cis-pidd] Wiskott Aldrich and Very low platelets

[CIS-PIDD]

i saw the case on line. We and others have treated at least 12 kids with WAS-XLT with eltrombopag with reasonably good but not great results. First 8 were published in blood in 2015 late in the year.
The problem for this baby is that you need to not have milk for ideally 4 hours before and 4 hours after the eltrombopag and it is hard to believe that this can happen.  Therefore i would use romiplostim (plate). I have not seen published data on it but anecdotally a number of patients including 3 of ours have used it and at least the same number at Necker in Paris. We have a baby who has been on it for the reason above taken care of by Susan Prockop and he is doing reasonably well with it.   I think as a bridge to transplant i would give 10 micrograms/kg weekly and see how it goes. The platelets may still fall with infections but will probably bounce back quicker and more reliably.
Love to get f/u.
Jim Bussel


On Mar 11, 2017, at 11:15 AM, CIS-PIDD <cis-pidd at lists.clinimmsoc.org<mailto:cis-pidd at lists.clinimmsoc.org>> wrote:


All,

I have a 4 month old male patient with Wiskott-Aldrich. His baseline platelet count is about 30-40. He was admitted to our hospital when he was 3 months old with RSV and moraxella pneumonia. At that time, his platelet count dropped to 2-3. That lasted about a week. As he recovered from his infections, his platelets improved.

He was then re-admitted on March 2nd when he was 4 months old- this time with Coronavirus and Flu A. Again his platelets have dropped and do not really respond to platelet transfusions. He is no longer symptomatic from his viral infections, but his platelets have yet to recover. We have given him IVIG without any response. I have looked at his blood smear, and his platelet count does appear to really be that low.

We are getting him prepared for a bone marrow transplant. In the meantime, does anyone have any good recommendations of how to increase his platelet count. I am hesitant to take out his spleen. Has anyone tried eltrombopag?

Thanks.

Evan

Evan Shereck, MD, MEd
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