[CIS PIDD] [cis-pidd] Transplanted boy with WAS

[Sokolic, Robert (NIH/NHGRI) [E]]

If What was the preparative regimen for the original transplant? If it was
non-myeloablative, I would consider myeloablative preparation.

Did you look at NK function in the patient, or in the donors?

Would consider sending blood to Jordan Orange to see if immune synapse
formation by pt NK cells is abnormal. If so, this would argue for either
myeloablative transplant or an alternative donor.
Rob Sokolic

On 8/7/14 4:31 AM, "Anders Fasth" <anders at fasth.com> wrote:

>Dear All,
>This case below I have asked for advice earlier in July, but nobody
>replied. Too difficult case ???
>
>I am still anxious for your advice. The update is even more troublesome:
>He was started on the HLH04 protocol again and after dexamethasone and
>two etopside his ferritin has gone from 2000 to 18 000. The pediatric
>oncologist are afraid of retransplantation, but does he has a chance
>without a new transplantation?.
>
>The case as from my mail in late July.
>
>I need your advice re a six year old boy with WAS transplanted with a
>double cord 14 months ago. Donors were his sibling twins (sister -
>non-carrier and brother) born after HLA-PGD. More or less eventful course
>for the first 3 months. Trombocytes returned to normal, full donors
>chimera, today about ⅔ female donor and ⅓ male donor. In late September
>last year he developed a rash that nobody could put a dx to: biopsy - no
>GvhD, possible eczema. In December, 8 months post SCT, he develops HLH
>with all typical findings and ferritin >100 000. New skin biopsy at time
>of HLH showed massive infiltrates of histeocytes. Nu mutation in FHL
>genes. Treated according to HLH-04 protocol and quickly clinically
>better, but only slowly normalizing his ferritin over many months.
>Now for about a month normal ferritin, but as steroids was tapered down
>his skin rash is back since a couple of weeks. And last week ferritin was
>slowly raising and is today 1900 + fever = relapse of his HLH.
>
>My concern is of course  - how to get a sustained response. Should we
>re-transplant him? He has his matched twin siblings that now are 1 ½
>years of age so we can use bone marrow this time.
>
>Anders
>
>
>Anders Fasth, MD, PhD
>Professor of Pediatric Immunology,
>Dept of Pediatrics, University of Gothenburg
>Address: The Queen Silvia Children’s Hospital,
>SE-416 85 Göteborg, Sweden
>Tel +46-31-343 5220 (343 4000 switchboard)
>Mobile +46-76-050 6117 (work) +46-70-687 5970 (private)
>Fax +46-31-707 0694
>
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