[CIS-PAGID] looking for transplant advice

[Cowan, Mort]

We've had success with clofazimine for patients not responding to the more standard anti-MAI drugs. Steve Holland at NIH might be able to offer more info about it. We're treating one child with it now and you need a IRB-approved protocol and have to get an IND for each patient you treat but it has worked for us (so far).  Mort

Morton J. Cowan, M.D.
Professor of Pediatrics
Chief, Blood and Marrow Transplant Division
UCSF Children's Hospital, Room M659
505 Parnassus Ave
San Francisco, CA 94143-1278
 
Phone: 415-476-2188
FAX: 415-502-4867
 
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-----Original Message-----
From: pagid-bounces at list.clinimmsoc.org [mailto:pagid-bounces at list.clinimmsoc.org] On Behalf Of Sullivan, Kathleen
Sent: Thursday, June 23, 2011 12:20 PM
To: pagid at list.clinimmsoc.org
Subject: [CIS-PAGID] looking for transplant advice

I am posting this question on behalf of others but the big question is:

Does it make more sense to transplant now or to try to achieve some clearance of mycobacteria prior to BMT?

The patient is a two year old with very low T cell numbers (CD3 about 100-200) and no TRECS.  Her mitogens are not completely flat but about 2-4% of the control.  She has immunoglobulin and B cells and has had some responses to vaccines.  She had PCP and now presents with MAI and huge nodes.  She was treated with triple therapy for about three weeks for her MAI and the nodes enlarged.  We have increased her MAI coverage to 5 drugs and are thinking about adding gamma-interferon.  We do not have a genetic type of SCID identified although she has a mutation of uncertain significance in the IL-7Ra gene and she has uniparental isodisomy of that chromosome.

Given this picture, what do other people think about hurrying to do a transplant on the theory that this is the only curative maneuver that can clear her MAI vs waiting to achieve some level of control and then transplanting?


Kate

Kate Sullivan, MD PhD
Professor of Pediatrics
ARC 1216 Immunology CHOP
3615 Civic Center Blvd.
Philadelphia, PA 19104
(p) 215-590-1697
(f) 267-426-0363