[CIS-PAGID] looking for transplant advice

[Sullivan, Kathleen]

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 100-200) and no TRECS.  Her mitogens are not completely flat 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