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

Thu Jul 10 08:32:00 EDT 2014

Agree with Gigi that withdrawal of Imm Supp drugs is your last hope but if
the repeat chimerism is falling below 5% it is going to be hopeless.
I have never seen clinically significant GVHD in this setting ( n: 5-7)
but the chances are very poor that it will work being so close to UCBT. If
You were at 100days or beyond with 5% donor cells  you may have a bit more
likely graft survival but even that would be against the odds

I doubt that ADA SCID could engraft with Bu doses <12

Best wishes, Paul

Paul Szabolcs, M.D.

Professor of Pediatrics and Immunology
University of Pittsburgh School of Medicine
Chief, Division of Blood and Marrow Transplantation and Cellular Therapies,
Children's Hospital of Pittsburgh of UPMC
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On 7/9/14, 10:04 PM, "Notarangelo, Luigi"
<Luigi.Notarangelo at childrens.harvard.edu> wrote:

>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
>
>Sent from my iPad
>
>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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