[CIS PIDD] BMT for newborn with CGD

[Jane Peake]

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