불응성 진성적혈구 형성부전 환자에서 시행한 자가 CD34+ 말초 조혈모세포 이식 1례 = Autologous CD34+ Stem Cell Transplantation for Patient with Refractory Pure Red Cell Aplasia

다국어 초록 (Multilingual Abstract)

Acquired pure red cell aplasia(PRCA) is a rare hematological condition, characterized by severe anemia and reticulocytopenia, with normal white cell and platelet counts, and selective aplasia of the erythroid cell line in the marrow. It is considered an autoimmune disorder. Antibodies directed against heme synthesis or erythroid progenitors have been described. Based upon the autoimmune nature of PRCA, a variety of immunomodulatory agents and method including corticosteroids, antithymocyte or antilymphocyte globulins, azathioprine, cyclophosphamide, cyclosporine, and plasmapheresis is used for patients with PRCA but the optimal therapeutic strategy for refractory patients to the conventional treatment has not been defined.
For PRCA patients with refractory to conventional treatments, disease morbidity is increased and life expectancy is significantly shortened because of increased infection risk or multiorgan failure due to chronic immune suppression status and prolonged transfusions. Results of recent studies and observations of severe refractory autoimmune disease control in autologous peripheral stem cell transplantation setting have recently prompted interest in a potential role for high dose immunosuppressive therapy as a treatment modality.
We report our experience the high dose immune suppression with cyclophosphamide (50mg/ Kg/day, for 4 days) followed by autologous CD34＋ enriched peripheral stem cell transplantation for refractory PRCA patient, and she is hematologically remission for 6 months after transplantation. To our knowledge, this is the first report of autografting for patient with PRCA.