Long-Term Remission and Bone Marrow Findings in Children with Severe Aplastic Anemia Immunosuppressed with High Doses of CyclophosphamideJosé Carlos Jaime-Pérez*Oscar González-Llano Olga G. Cantú-Rodríguez Luis Javier Marfil-Rivera David Gómez-Almaguer
Hematology Department, Dr. José E González University Hospital of the School of Medicine of the Autonomous University of Nuevo León, Monterrey, México, USA
- Corresponding Author:
- José Carlos Jaime-Pérez
Departament de Hematología
Edificio "Dr. Rodrigo Barragán"
Hospital Universitario "Dr. José E González"
Avenida Madero y Gonzalitos
Colonia Mitras Centro, Monterrey
N.L, México, C.P. 64460, USA
Tel: 52 (81) 1257-2905
Fax: 52 (81) 1257-2906
E-mail: [email protected]
Received Date: January 30, 2013; Accepted Date: February 27, 2013; Published Date: March 01, 2013
Citation: Jaime-Pérez JC, González-Llano O, Cantú-Rodríguez OG, Marfil-Rivera LJ, Gómez-Almaguer D (2013) Long-Term Remission and Bone Marrow Findings in Children with Severe Aplastic Anemia Immunosuppressed with High Doses of Cyclophosphamide. J Bone Marrow Res 1:112. doi: 10.4172/2329-8820.1000112
Copyright: © 2013 Jaime-Pérez JC, et al. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
Background: Most cases of aplastic anemia are acquired and of autoimmune etiology. Treatment of patients lacking a stem cell donor habitually consists of two curses of immunosuppression with anti-thymocyte globuline plus cyclosporine (ATG/CSA), whereas intensive immunosuppression consisting exclusively of high doses of cyclophosphamide (HDCY) has been successfully employed mostly in adults, detailed long-term information on children receiving HDCY is lacking.
Patients and methods: Five children suffering from severe acquired aplastic anemia and without an HLAcompatible stem cell donor were immunosuppressed with high-dose cyclophosphamide (HDCY) at a total dose of 200 mg/kg over four days. Granulocyte colony-stimulating factor (G-CSF) was administered at 5 μg/kg/day until sustained neutrophil and platelet count recovery. The surviving patients were followed-up for twelve years and their bone marrows examined at this time.
Results:Three of the five children obtained a complete sustained hematological remission. Current ages are 17, 19 and 27. After twelve years of follow-up two patients have normal hematological values, with no relapse nor clonal or displasic hematological disorders, their bone marrow aspirate was morphologically normal, whereas bone marrow trephine biopsy histopathology demonstrated a reduced cell content to about 60% of normal. One patient developed myelodisplasia 12 years after HDCY and currently he is being considered for a bone marrow transplant.
Conclusions: Sustained long-term trilineage hematopoiesis can be rescued in children suffering from SAA employing HDCY as the only immunosuppressor; the bone marrow did not fully recuperate its normal cellularity after twelve years post-treatment, mielodysplasia can develop more than ten years after HDCY.