[CIS PIDD] [cis-pidd] ADA-deficient SCID

[CIS-PIDD]

Dear Howard,
I would go with gene therapy. The results of the last protocol are excellent.
At this age, I would send the patient to Don Kohn at UCLA. In the meantime, I would begin PEG-ADA as soon as possible.
All the best
Elie Haddad



Elie Haddad, MD, PhD,
Professor of Pediatrics, University of Montreal,
Head, Pediatric Immunology and Rheumatology Division,
CHU Sainte-Justine, 3175 Cote Sainte-Catherine
Montreal, QC, H3T 1C5, Canada
Ph: 1 514 345 4713
fax: 1 514 345 4897
e-mail: elie.haddad at umontreal.ca





> Le 2017-03-07 à 14:45, CIS-PIDD <cis-pidd at lists.clinimmsoc.org> a écrit :
> 
> I have a new ADA deficient SCID.  Three weeks old.  Perfectly healthy. No sibs.
> We have one 6/6 cord blood donor, and ten 10/10 bone marrow donor options (<40 years old) from the NMDP.
>  
> Would people transplant now without a prep regimen, transplant now with RIC, give PEG-ADA for 2 years and then transplant when conditioning toxicity might be less, or try gene therapy?  (If the latter, who has an active program?
>  
>  
> Howard
>  
>  
> Howard M. Lederman, M.D., Ph.D. 
> Professor of Pediatrics, Medicine and Pathology 
> Division of Pediatric Allergy and Immunology 
> Johns Hopkins Hospital - CMSC 1102 
> 600 N. Wolfe Street 
> Baltimore, MD 21287-3923 
> Phone: 410-955-5883 
> Fax: 410-955-0229 
> Email: Hlederm1 at jhmi.edu <https://mobile.johnshopkins.edu/OWA/redir.aspx?C=02aee18ab96e42ab8cb61c09ecb79487&URL=mailto%3aHlederm1%40jhem.jhmi.edu> 
> 
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