[CIS-PAGID] IPEX question

[Cowan, Mort]

I agree with Kate. We had a child with IPEX in a very similar situation who didn't respond all that well to tacrolimus but stablilized a bit and we went ahead with a cord blood using Campath Flu Mel. Our patient did very well although the diabetes has remained.

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 Sullivan, Kathleen
Sent: Tuesday, October 04, 2011 7:43 AM
To: pagid at list.clinimmsoc.org
Subject: Re: [CIS-PAGID] IPEX question

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

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