Case 3:
A 1-year-old female with Radioulnar Synostosis and Amegakaryocytic
Thrombocytopenia presented with SD aGVHD of the skin around day +177.
The patient was started on prednisone and sirolimus. Attempts to wean
from prednisone led to multiple skin GVHD flares. Rituximab was given
without success. She was deemed underweight for ECP catheter placement,
so bortezomib was initiated and increased as tolerated for total of 24
weekly doses (Table 1). There was initial PR to bortezomib, allowing
wean of steroids; however, she was unable to completely discontinue
prednisone. All bortezomib doses were well tolerated, with the only
complications including delayed dose due to rhinovirus infection, and a
temporary dose reduction due to pancytopenia. The patient ultimately
continued to have skin flare-ups after the initial response, and
bortezomib was discontinued.