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

[Szabolcs, Paul]

Elena

The data is not that hopeless with these details. A second donor search
can be initiated but this graft is not lost yet. I would follow now Q2
weeks chimerism and holding ADA at least till next dataset is worthwhile
indeed.
Best, Paul 
 
On 7/12/14, 9:09 AM, "Sokolic, Robert (NIH/NHGRI) [E]"
<sokolicr at mail.nih.gov> wrote:

>Elena-
>  I agree with what the others have said in terms of withdrawal of
>immunosuppression. Would then start PEG-ADA. Rather than going to haplo
>or another cord, I would advise looking for a MUD, b/c the child should
>be stable after a 3-4 months on PEG. This is assuming that a MUD is less
>toxic than a haplo as done by your transplanters  You could try
>withholding the PEG while immunosuppression is withdrawn to give an added
>advantage to the donor cells.
>Rob
>
>
>
>From: Perez, Elena [e.perez13 at med.miami.edu]
>Sent: Friday, July 11, 2014 12:26 PM
>To: CIS-PIDD
>Subject: RE: [cis-pidd] ADA SCID tx
>
>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
>
>
>The information contained in this transmission may contain privileged and
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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
>4401 Penn Avenue, Rangos, Room 5125
>Pittsburgh, PA 15224
>Phone:  412-692-6225
>Fax:  412 692-7816
>LR page: 412 958-6985
>E-mail:  paul.szabolcs at chp.edu
>
>
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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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