[CIS PIDD] BMT for newborn with CGD

[Bleesing, Jacob]

In my last CGD patient, diagnosed at birth (family history), I got him on the appropriate anti-microbials and waited until he was 6 months of age (for the reasons outlined below) and then used our Bu/Cy/ATG regimen (without any problems and using a UCB donor).

I would be careful with using fludarabine in a very young patient as well - and why we didn't consider the RIC approach in our young boy.

Since, I would not classify this as a "high-risk"  patient, not sure why he should be transplanted right now (or why a RIC would be the first option). The fact that a MSD is available should be taken into consideration as well, as far as preparative regimen is concenerd.

Jack Bleesing

-----Original Message-----
From: pagid-bounces at list.clinimmsoc.org [mailto:pagid-bounces at list.clinimmsoc.org] On Behalf Of Fleisher, Thomas (NIH/CC/DLM) [E]
Sent: Tuesday, August 28, 2012 2:12 PM
To: pagid at list.clinimmsoc.org
Subject: Re: [CIS PIDD] BMT for newborn with CGD


Advice from Harry Malech:

Most pediatric transplanters avoid busulfan in very early infancy, making the Gungor/Seger regimen likely not appropriate in the age group of this infant (7 weeks).  However, I am not familiar with what should be used.  I would refer them to Joann Kurtzberg at Duke for advice in this regard, and if she does not feel she has the best answer, she will know who has been transplanting infants of this age.  I do agree that this is a situation where going forward now is likely the right thing to do, even if it is not the best approach for CGD transplant in general. H.

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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