[CIS PIDD] (no subject)

[Infante, Anthony J]

There are BMT protocols out there for "severe autoimmune disease." The allo vs auto question is a good one. I tend to favor allo but others may know better.

Tony Infante
Sent from my iPhone

On Sep 1, 2012, at 11:51 AM, "Sorensen, Ricardo" <RSoren at lsuhsc.edu<mailto:RSoren at lsuhsc.edu>> wrote:


Hi everybody,
I would like to ask you for advise on a patient with  a severe systemic-onset Juvenile rheumatoid arthritis (JRA).  JRA is not defined as an imunodeficiency jet, but it certainly is a severe immunological disorder. The questions is: does anybody have experience and/or advise on performing a stem cell transplant for this condition.
The patient is a 14 female with early onset JRA at age 2.  She has received initially treatment with steroids and methotrexate with partial response.  Enbrel (etanercept) treatment did not decrease her active disease. IVIG infusions, another TNF blocker, remicade (infliximab), IL-1 blocker, kineret (anakinra), IL-6 blocker (actemra), and even orencia (abatacept) were  tried by our rheumatologist without success.  Finally, in the last 2 months she received a total of 3 infusions of rituxan (rituximab) at 3 week intervals, again without significant improvement. Currently, the patient is in a wheelchair, consuming a significant dose of steroids but  still having a very active disease.  In summary this patient has an early onset,  severe refractory active-destructive systemic-JRA.
We wonder if she might benefit from stem cell transplantation.  If we consider that this is really an immunodeficiency with an as jet undetected molecular defect as suggested by the early once and refractory course of the disease,  an autologous BMT probably would not work?
Any advise about additional diagnosis and treatment of this patients would be much appreciated.

Thanks,

Ricardo U Sorensen, M.D.
For  Abraham Gedalia,M.D.
Professor of Pediatrics
Head, Division of Pediatric Rheumatology
LSU Health Sciences Center and Children's Hospital
New Orleans,Louisiana
Office: (504) 896-9385
E-Mail: agedal at lsuhsc.edu<mailto:agedal at lsuhsc.edu>

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