[CIS PIDD] BMT for newborn with CGD

[Fleisher, Thomas (NIH/CC/DLM) [E]]

I deferred to Steve Holland (and also Harry Malech who I assume will respond later). Steve's response is:

I would do it. I don't know that that is the right preparative regimen (I just don't know anything about the regimens for infants) but the Swiss regimen from Tayfun Gungor and Reinhard Seger has the most exposure and has been extremely successful with high success rates and very low toxicity.

Steve
--
Chief, Laboratory of Clinical Infectious Diseases CRC B3-4141 MSC 1684 Bethesda, MD 20892-1684
301-402-7684 (v)
301-480-4508 (fax)
smh at nih.gov
Assistant: Adrienne Snyder
301-451-9019 (v)
301-480-4506 (fax)
asnyder at niaid.nih.gov

Thomas A. Fleisher, M.D. 
Chief, Department of Laboratory Medcine
NIH Clinical Center
Bethesda, MD 20892
301 496-5668 (tel)
301 402-1612 (fax)


-----Original Message-----
From: Jane Peake [mailto:j.peake at uq.edu.au] 
Sent: Monday, August 27, 2012 10:39 PM
To: pagid at list.clinimmsoc.org
Subject: [CIS PIDD] BMT for newborn with CGD



Hi 

I was wondering if I could canvas opinions. We have a  baby boy who is now 7 weeks old with confirmed  AR (p67) CGD that has been born to a family that has had 2 previous infants die with CGD. They come from a remote part of Australia and on presentation the other children had a large number of fungal organisms in their lungs and a lot of lung damage. One died  very suddenly with a febrile illness when they presented to their local hospital and  he could not be resuscitated. The family refused a postmortem. The other young boy underwent a matched sibling donot BMT buit died of respiratory failure.
I belive that this infant whilst he is still well should undergo BMT as we have a matched sibling donor sooner rather than later but I have some concerns regading the conditioning regieme in a baby so young both from the increased toxicity from full dose busulfan in neonatal population and long term neurocognitive outcomes.
 
I would be very interested in others thoughts.
Kind regards
Jane
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