[CIS PIDD] [cis-pidd] ADA SCID tx

[Notarangelo, Luigi]

Dear Elena,

I assume chimerism is on total blood, correct? What is T cell count now? Can you do lineage specific chimerism? What immunosuppression? (You may consider reducing it to favor donor cells, although risk of GvHD would still be there). I see no advantage in rushing toward haplo. Lastly, did you adjust Bu exposure?

Gigi

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Luigi D. Notarangelo, MD
Jeffrey Modell Chair of Pediatric Immunology Research
Division of Immunology, Boston Children's Hospital
Professor of Pediatrics and Pathology, Harvard Medical School
Karp Research Building, Room 10217
1, Blackfan Circle
Boston, MA 02115
USA

Tel: 617-919-2277
FAX: 617-730-0709

> On Jul 9, 2014, at 9:55 PM, "Perez, Elena" <e.perez13 at med.miami.edu> wrote:
> 
> Dear "transplant-for-Primary Immunodeficiency" community:
> 
> We have a 2mo boy with ADA deficiency picked up on NBS who received an HLA matched umbilical cord transplant on DOL45 after being conditioned with 50% dose reduced Busulphan, Fludarabine and ATG and rituximab, which was well tolerated. Stem Cell Dose: 16.7 x 10e5 CD34 pos cells/kg; 1.69 x 10e7 TNC/kg. 
> 
> Early evaluation of peripheral cell genotype unfortunately revealed mixed chimerism of 3% donor suggestive of primary graft failure. His most recent chimerism revealed 5% donor, and the transplanters are planning to send another one this week before deciding about retransplant. Today is +41d post transplant.
> 
> In anticipation of need for retransplant, I offered to post his case on the list serve for feedback to our transplanters. They are considering retransplant with umbilical cord blood but hesitant to redose chemo vs maternal haplo but clinimacs cell purification IND is not available here.  
> 
> He is infection free but has developed seizures that are controlled on Keppra. He is maintained on IVIG and all the usual prophylaxis and so far has remained free of detectable infections, except on presentation required oxygen which has improved to room air. 
> 
> He is on medicaid with very limited family resources, which makes it difficult to transfer care out of state.
> 
> Feedback from transplanters in the group appreciated, and will share with transplanters here.
> 
> thank you,
> Elena Perez
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