[CIS-PAGID] IPEX question

[Perez, Elena]

Thanks very much to all for the prompt responses. We are instituting FK506 and consulting with BMT for Campath or other rx.  Will keep you posted.
E

From: pagid-bounces at list.clinimmsoc.org [mailto:pagid-bounces at list.clinimmsoc.org] On Behalf Of Cowan, Mort
Sent: Tuesday, October 04, 2011 1:11 PM
To: pagid at list.clinimmsoc.org
Subject: Re: [CIS-PAGID] IPEX question

Shalini Shenoy at Washington University in St Louis reported 4 children with IPEX who got unrelated cord bloods using her Campath Flu Mel. The campath is given on day -21 (or something like that) so that might help quiet the disease down before giving flu and mel.  Mort

Morton J. Cowan, M.D.
Professor of Pediatrics
Chief, Allergy Immunology and 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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From: pagid-bounces at list.clinimmsoc.org [mailto:pagid-bounces at list.clinimmsoc.org] On Behalf Of Elie Haddad
Sent: Tuesday, October 04, 2011 8:13 AM
To: pagid at list.clinimmsoc.org
Subject: Re: [CIS-PAGID] IPEX question

You could also try ATG (or Campath) in order to be even more immunosuppressive. The question of myasthenia is important because if it is indeed a myasthenia, you could then consider plasmapheresis/rituximab.
In all cases, these therapeutical strategies come with an as fast as possible BMT with the best available donor. Cord blood may be there a good option (if no related matched donors) to get quickly a donor.
Elie


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<mailto:elie.haddad at umontreal.ca>





Le 2011-10-04 à 10:23, Perez, Elena a écrit :



Dear All,

We have a 3mo baby with IPEX confirmed (1156 C>T; R386C) in the hospital, with DM on insulin drip, diarrhea, dermatitis who is vent/trach dependent (able to breathe over the vent but requiring a little extra PEEP to help), unable to wean off (transferred from outside hosp w/ trach and on vent). He only weighs 3.1 kg. We have him on sirolimus (levels ~15-25) and steroids, although not yet noting improvement in stools. On TPN and minimal NG feeds.

My question(s) to group is:
1.       Any thoughts on possible reversible/treatable reasons why he could be trach/vent dependent related to IPEX? (neuro consult --considered Myasthenia testing and muscle bx pending)...
2.       Prognosis doesn't seem too good, but family meeting w/ PICU, Transplant, Immunology on Friday to discuss BMT. Any insights? hope?

Appreciate thoughts.

Thanks,

Elena Perez, MD, PhD
Division of Allergy, Immunology, Rheumatology
University of South Florida
All Children's Hospital
St. Petersburg, Florida



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