[CIS PIDD] [cis-pidd] Bone marrow transplant for X-linked CGD
Prof. Dr. Antonio Condino Neto
condino at icb.usp.br
Fri Oct 19 11:35:41 EDT 2012
I would try gene therapy for CGD
Contact Thrasher in London
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Antonio Condino-Neto
Professor of Experimental Medicine
Institute of Biomedical Sciences, University of São Paulo
1730 Lineu Prestes Avenue, São Paulo - SP. ZIP 05508-000. Brazil
Tel (55) (11) 3091-7387 / Fax (55) (11) 3091-7224
Citando "Newburger, Peter" <Peter.Newburger at umassmed.edu>:
> I can't comment on the donor/HSCT issues, but would suggest that you
> evaluate and possibly treat the patient for secondary HLH prior to
> transplant.
> Regards,
> Peter Newburger
> -------------------------------------------------------------
> Peter E. Newburger, MD
> Ali and John Pierce Professor of
> Pediatric Hematology/Oncology
> Vice Chair for Research
> Department of Pediatrics
> University of Massachusetts Medical School
> 55 Lake Avenue North
> Worcester, MA 01655
> tel. 508 856-4225
> fax 508 856-4282
> peter.newburger at umassmed.edu<mailto:peter.newburger at umassmed.edu>
> -------------------------------------------------------------
>
> ________________________________
> From: Howard Lederman [hlederm1 at jhmi.edu]
> Sent: Thursday, October 18, 2012 2:00 PM
> To: CIS-PIDD
> Subject: [cis-pidd] Bone marrow transplant for X-linked CGD
>
> We have a 4 y/o boy with X-linked CGD who has had a stormy course
> with a history of multiple infections and mild inflammatory bowel
> disease. He currently has generalized adenopathy,
> hepatosplenomegaly and bone marrow granulomas without an
> identifiable microbial cause. He has not responded to a variety of
> antibiotics, but is improving with corticosteroids. We would like
> to do a bone marrow transplant.
>
> The available donor options are:
>
>
> 1. Unrelated 4/6 matched cord blood (match at DRB1)
>
> 2. Haploidentical parent (probably the father, as the mother
> has a mixed connective tissue disease)
>
> 3. One possible NMDP unrelated donor who is mismatched at A
>
> I am interested in the experience of others with donor choice. I am
> also interested in recommendations for full myeloablation vs.
> reduced intensity conditioning, particularly in view of the recent
> publication from Duke (Tewari et al 2012. Biol Blood Marrow
> Transplant 18:1368-77).
>
>
> 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<mailto:Hlederm1 at jhmi.edu>
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