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

Fri Jul 11 12:26:18 EDT 2014

Jack and Cary- I double checked, on stem cell processing lab sheet and TNC/kg=1.69x10^8 is listed, so previous email was a typo taken from EMR chart. Today chimerism is back: Unseparated 6.7%; T lymph 5.47%; B lymph 8.82%; grans 2.72%, 40d post tx. Also match was 6/6 (there was a mismatch at C locus so BMT coordinator called it a "7/8").
Summary of chimerism:
	unsep	T	B	gran
20d	2.75	0	2.6	1.6
30d	5.77	insuff	insuff	3.56
40d	6.7	5.47	8.82	2.72

Baby is stable, room air, feeding.
Looking into PEG-ADA; discussing with transplanters here the next steps...
Appreciate all the comments/suggestions.

Thanks again!
Elena

Elena E. Perez, M.D.,Ph.D.
Associate Professor 
Chief, Pediatric Allergy and Immunology
Jeffrey Modell Diagnostic and Research Center for Primary Immunodeficiencies
Division of Immunology and Infectious Diseases
Batchelor Children's Research Institute, Suite 316
University of Miami Miller School of Medicine
1580 NW 10th Avenue
Miami, FL 33136

Office: 305-243-4863
Nurse (Maria Rodriguez) 305-243-9514
FAX: 305-243-7409
Email: e.perez13 at med.miami.edu

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-----Original Message-----
From: Bleesing, Jacob [mailto:Jack.Bleesing at cchmc.org] 
Sent: Thursday, July 10, 2014 9:28 AM
To: CIS-PIDD
Subject: RE: [cis-pidd] ADA SCID tx

Elena:

Are you sure about the cell dose that you shared with us?

Jack

________________________________________
From: Perez, Elena [e.perez13 at med.miami.edu]
Sent: Thursday, July 10, 2014 9:10 AM
To: CIS-PIDD
Subject: RE: [cis-pidd] ADA SCID tx

Thanks for all the comments so far. I will pass them along. He has been off of cyclosporine for a couple of weeks and results of next chimerism (whole blood and lineage specific) are due today. I believe that match was 8/8 but will double check. Will keep you posted & thanks again.
Elena

-----Original Message-----
From: Szabolcs, Paul [mailto:paul.szabolcs at chp.edu]
Sent: Thursday, July 10, 2014 8:32 AM
To: CIS-PIDD
Subject: Re: [cis-pidd] ADA SCID tx

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 http://www.chp.edu/CHP/bmt

One Children's Hospital Drive
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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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