title: Acquired amegakaryocytic thrombocytopenia treated with allogeneic BMT: a case report and review of the literature.
abstract: Despite recent advances in understanding the biology of thrombopoiesis, autoimmune thrombocytopenia caused by inhibition of megakaryocytic precursors, remains a treatment dilemma. We report a case of a 43-year-old female who developed amegakaryocytic thrombocyto- penia refractory to intravenous immunoglobulin (IVIG), prednisone, cytoxan and vincristine. She was subsequently treated with myeloablative chemotherapy (busulfan and cyclophosphamide) followed by allogeneic bone marrow transplant from a 6/6 HLA-matched sibling. The patient is currently more than 1 year after transplant with complete donor chimerism and restoration of normal thrombopoiesis. A review of the literature shows that the clinical syndrome known as amegakaryocytic thrombocytopenia represents a heterogeneous group of disorders, and clinical experience with immunosuppression varies. Appropriate initial treatment for these patients requires immunosuppressive agents, including antithymocyte globulin (ATG) for steroid refractory disease. However, in the case of symptomatic patients who have an appropriate sibling donor, early hematopoietic progenitor cell transplant, even before administration of ATG, may be necessary. Further studies are needed to better define the pathogenesis and mechanism of this heterogeneous disorder before more definitive treatment algorithms can be established.
authors.affiliation: Department of Medicine, Emory University, Atlanta, GA 30322, USA.
authors.forename: S
authors.initials: S
authors.lastname: Lonial
authors.forename: P A
authors.initials: PA
authors.lastname: Bilodeau
authors.forename: A A
authors.initials: AA
authors.lastname: Langston
authors.forename: C
authors.initials: C
authors.lastname: Lewis
authors.forename: S
authors.initials: S
authors.lastname: Mossavi-Sai
authors.forename: J T
authors.initials: JT
authors.lastname: Holden
authors.forename: E K
authors.initials: EK
authors.lastname: Waller
mesh_terms: D000328:Adult; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use; D016026:Bone Marrow Transplantation; D005260:Female; D006801:Humans; D007136:Immunoglobulins / Q000008:administration & dosage / Q000009:adverse effects; D008533:Megakaryocytes / Q000473:pathology*; D013921:Thrombocytopenia / Q000139:chemically induced* / Q000628:therapy*; D018183:Transplantation Chimera; D014184:Transplantation, Homologous
chemical_list: D007136:Immunoglobulins