[CIS PIDD] [cis-pidd] Transplant Question

[David Buchbinder]

Dear Colleagues,
 
I wanted to see if anyone has any comments / suggestions on the
approach to transplant for a interesting CVID patient....
 
She is an adolescent with a documented history of recurrent
sinopulmonary infections (Penicillium, Corynebacterium propinquum, and
Pseudomonas aeruginosa), viral infections (shingles), and recurrent
autoimmune disease (autoimmune cytopenias, vitiligo).  Whole exome
sequencing identified compound heterozygous mutations in RAG1.  She has
granulomatous-lymphocytic interstitial lung disease.  She has been on
immunoglobulin replacement due to her history of progressive
hypogammagloublinemia, poor antibody responses, and recurrent
sinopulmonary infections. She is lymphopenic with abnormal mitogen and
antigen T cell proliferation responses She was treated with
corticosteroids and infliximab (for her lung disease), which resulted in
radiographic / clinical improvement.  She is now on IVIG and Humira with
abnomral PFTs (FVC was 83% predicted, FEV1 was 75% predicted, FEV1/FVC
ratio was 81%, FEF 25-75% was 67% predicted w/ improvement after
bronchodilators).  Her most recent autoimmune issue has been enteritis.

 
We have identified a 10/10 matched unrelated donor bone marrow donor. 
He is a young male (ABO mismatched, CMV positive).  Given her risk of
infection and ongoing autoimmunity we have decided to move to
transplant.  My plan was to proceed with a conditioning regimen
consisting of distal Campath, Fludarabine 150 mg/m2, Thiotepa 8 mg/kg,
Melphalan 140 mg/m2.   She is quite a bit older and a patient with more
comorbidities including lung disease, etc.  I wanted to avoid a standard
myeloablative approach given risks of toxicity.  Her autoimmunity is
also quite pronounced so I would like to make sure we ablate enough of
her immune system so we limit her risk of additional autoimmunity.  Any
comments or suggestions would be more than welcomed on the transplant
front.
 
Thank you as always….
 
Dave Buchbinder
CHOC Children's - UC Irvine

CHOCLX-MGSM made the following annotations
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