[CIS-PAGID] looking for transplant advice

[Sergio Rosenzweig]

I'm not sure if Kate's patient was BCG vaccinated or not, but during the
last 2y we have been collecting data from BCG vaccinated SCID patients
form all around the world (25 centers from 15 countries, more than 200
patient so far) to learn about their outcome and the best therapeutic
approach for them. Bottom line, more than 50% of this patients develop
BCG complications, 1/3 localized, 2/3 disseminated. Preliminary analysis
shows that preemptive treatment helps, not so clear at this point which
scheme and for how long works better (my approach used to be 4 drugs
-including aminoglucoside for 1-2 mos-, parenteral route, treat until
immunerecontitution; however others have had good success with 2 oral
drugs). We are still accepting patients until the end of July, when
final analysis will start.
Thanks,
Sergio

Sergio D. Rosenzweig, MD, PhD
Chief, Infectious Diseases Susceptibility Unit
Laboratory of Host Defenses, NIAID, NIH
10 Center Dr., Bldg. 10, CRC 5W-3888
Bethesda, MD 20892-1456
Phone (301) 451 8971
Fax (301) 451 7901
Cell (240) 361 7617
Pager 102 10678
srosenzweig at niaid.nih.gov
 
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>>> "Notarangelo, Luigi" <Luigi.Notarangelo at childrens.harvard.edu>

06/23/11 5:08 PM >>>
I agree with Antonio that this patient needs multi-drug treatment of BCG
and that this needs to be continued across transplant, including
engraftment (because this is the time when inflammatory complications
would otherwise develop). I would start treatment now but I would try to
perform the transplant within the next month or so. This is the only way
to save the patient. Who will be the donor? I would personally favor an
unmanipulated MUD (as long as full match)vs. a T-cell depleted haplo.

Gigi

Luigi D. Notarangelo
Division of Immunology
Children's Hospital boston
Sent from my Verizon Wireless BlackBerry

-----Original Message-----
From: "Prof. Dr. Antonio Condino Neto" <condino at icb.usp.br>
Sender: "pagid-bounces at list.clinimmsoc.org"
	<pagid-bounces at list.clinimmsoc.org>
Date: Thu, 23 Jun 2011 15:52:44 
To: pagid at list.clinimmsoc.org<pagid at list.clinimmsoc.org>;
Sullivan,Kathleen<sullivak at mail.med.upenn.edu>
Reply-To: "pagid at list.clinimmsoc.org" <pagid at list.clinimmsoc.org>
Subject: Re: [CIS-PAGID] looking for transplant advice

Dear Kate

All of my CGD cases that underwent BMT started receiving drugs for BCG  
at least 6 weeks before the BMT or even earlier if they developed  
complications with BCG (25% of them complicate with BCG). The drugs  
against BCG were given continuously, before and after the BMT  
procedure and ALL of them worsened during BMT procedure, bringing it  
to real risk that has to explained to the family. As the BMT engrafted  
the BCG was brought to control.

In your case I would try to have it as more stable as possible before  
BMT and do it as quick as possible, the patient́s only chance to cure  
it.

Hope this helps your decision

All best

Condino
-- 
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 "Sullivan, Kathleen" <sullivak at mail.med.upenn.edu>:


> 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 10> 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

>

>

>




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