[PAGID] LAD3

[Amos Etzioni]

Dear Javier
I believe also people outside the USA can give advice regarding LAD 3. What is the origin of your patient? Does she has the two gene mutations or just Kindlin3? As you know up to now there were less than 20 patients reported world wide. I believe that if you have a related matched donor you should go for BMT. From our expierence even if infecions wise no severe problems, blood transfusion may be frequent and the question for BMT in MUD cases will depend on the clinical conditions.
Regards from the other continent
Amos etzioni
________________________________
From: pagid-bounces at list.clinimmsoc.org [pagid-bounces at list.clinimmsoc.org] On Behalf Of Chinen, Javier [jxchinen at texaschildrens.org]
Sent: Wednesday, September 22, 2010 4:39 PM
To: 'pagid at list.clinimmsoc.org'
Subject: [PAGID] LAD3


Dear all,

I am seeing a 7 yo girl with LAD3 (confirmed) and would appreciate any experience in the US with these patients, besides those already reported in the literature.

My question is indication (or NOT) for BMT, and what have been the outcomes (of BMT vs conservative management)

Best regards,



Javier

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