[CIS-PAGID] IPEX question

[Sullivan, Kathleen]

Personally- I would hit him hard with FK506.  It seems to work a little faster than Rapamycin for me and you are only using it as a bridge to transplant so I would worry less about long term side effects.

can the lungs be due to IPEX- yes.  In our two who dies we found lymphocytic infiltrates everywhere.

I would start the FK, try to find a great match, include campath as the conditioning and transplant as soon as possible.

kate
On Oct 4, 2011, at 10:23 AM, Perez, Elena wrote:


> 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

>  

>  


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


-------------- next part --------------
An HTML attachment was scrubbed...
URL: <http://seven.pairlist.net/mailman/private/pagid/attachments/20111004/ddc40f7a/attachment.htm>