A Case of Successful Non-myeloablative Allogeneic Peripheral Blood Stem Cell Transplant (APBSCT) for Refractory POEMS Syndrome

초록

This is the first report in the world of a successful APBSCT for POEMS syndrome, which affected a 46-year-old man. He underwent APBSCT in January 2002 from his HLA identical sister, and his disease is getting better as of this writing, 10 months post APBSCT. Although his disease began in 1993 with weakness and ascites, the diagnosis of POEMS syndrome was made in 2001, based on polyneuropathy, hepatosplenomegaly, endocrinopathy (hypothyroidism), monoclonal protein (IgA lambda), marrow plasmocytosis (10%), and skin discoloration. Osteosclerotic bone lesions were absent. Cytogenetic study of marrow cells disclosed a karyotype of 46, XY, inv(9)(p12q13). He used to be placed elsewhere on various treatments; prednisone, melphalan, cyclophosphamide, and transjugular intrahepatic portosystemic shunt, under the impression of lymphoma or Castleman's disease with little benefit from the therapies. On referral to us, he was bedridden in marked emaciation with massive right pleural effusion and ascites, which had us opt non-myeloablative APBSCT rather than autologous transplant for the treatment. He was prepared with fludarabine (50mg on days -8 to -4) and melphalan (120mg on days -3 to -2). His sister was given G-CSF (300ug) on days -3 to 1, and a total of 6.98X10(8) NMC/kg or 9.79X10(6) CD34+ cell/kg were collected and given to him on days 0-1 using Cobe Spectra. GVHD prophylaxis consisted of cyclosporine. Engraftment of marrow was documented on day 14, in mixed chimerism with host DNA of 1.8%. Marrow studies at 2, 4, and 6 months repeatedly showed hypocellularity (20%) and good activity of all hematopoietic elements in persistent mixed chimerism (host DNA of 2%). His performance is getting better with gain of muscle power.