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.