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Peripheral blood versus bone marrow transplant in patients with aplastic anemia, an unresolved issue

Bone Marrow Transplantation volume 51, pages 1628–1630 (2016)Cite this article

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Aplastic anemia is a rare disorder with an incidence of ~2–4 cases per million people per year.1 If left untreated, only 30 percent of aplastic anemia patients will survive after 1 year, and the rest will die from complications of pancytopenia.2 Options for definitive therapy for aplastic anemia include hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy.3 Bone marrow transplantation (BMT) is considered the treatment of choice in patients younger than 20 years due to better overall survival (OS) and the preferred treatment in healthy patients older than 20 years of age with a fully HLA-matched sibling donor.4, 5 As GvHD is the principal cause of morbidity and mortality after HSCT in patients with aplastic anemia,6 the choice of suitable source of stem cell for transplantation is very important.

This study included 185 patients with aplastic anemia who were referred to our center between 1991 and 2013 to compare the results of BMT and peripheral blood stem cell transplantation (PBSCT). Forty patients (median age 17.5 years) received BMT and 145 patients (median age 24) received PBSCT. All participants received HSCT from a fully HLA-matched sibling donor. Patients conditioned by cyclophosphamide 50 mg/kg once daily i.v. for 4 days for BMT and PBSCT plus Anti-thymocyte globulin (ATG: Thymoglobulin, Genzyme) 2.5 mg/kg/ i.v. on days −4 till −2 for BMT and PBSCT and 17 other patients received cyclophosphamide alone. Prophylaxis for GvHD consisted of cyclosporine (1.5 mg/kg intravenously from −3, then 3 mg/kg/day intravenously from +7 in PBSCT and +11 in BMT then 12.5 mg/kg/day per oral) alone or plus a short course of methotrexate (10 mg/m2 on Day +1 and 6 mg/m2 on Days +3, +6, and +11). Cyclosporine was started 3 days prior to transplantation and continued orally for 6 to 7 months post HSCT; it was then discontinued in the absence of GvHD.

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Authors and Affiliations

  1. Hematology-Oncology and Stem Cell Transplantation Research Center/Tehran University of Medical Sciences, Tehran, Iran

    A Ghavamzadeh, K Alimoghaddam, M Jalili, S A Mousavi, B Bahar, A Kasaeian, A A Hamidieh, M Behfar, M Vaezi & M Jahani

  2. Non-communicable Diseases Research Center, Endocrinology & Metabolism Population Sciences Institute, Tehran University of Medical Sciences, Tehran, Iran

    A Kasaeian

  3. Tehran University of Medical Sciences, Tehran, Iran

    A Jalali

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  1. A Ghavamzadeh
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  2. K Alimoghaddam
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  3. M Jalili
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  4. S A Mousavi
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  5. B Bahar
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  6. A Kasaeian
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  7. A A Hamidieh
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  8. M Behfar
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  9. M Vaezi
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  11. M Jahani
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Correspondence to K Alimoghaddam.

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Ghavamzadeh, A., Alimoghaddam, K., Jalili, M. et al. Peripheral blood versus bone marrow transplant in patients with aplastic anemia, an unresolved issue. Bone Marrow Transplant 51, 1628–1630 (2016). https://doi.org/10.1038/bmt.2016.235

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