Summary:
Congenital sideroblastic anemia (CSA) is a dyserythropoietic disorder that leads to transfusion dependency and subsequent iron overload. Nonmyeloablative allogeneic hematopoietic stem cell transplantation (NST) was performed for a patient with CSA, who had contraindications to conventional allografting. Conditioning was fludarabine, low-dose total body irradiation and antithymocyte globulin, followed by peripheral blood stem cell transplant. Cyclosporine and mycophenolate mofetil were used for graft-versus-host disease prophylaxis. Complete donor chimerism was observed day +131. Early after transplant, the patient became transfusion independent, allowing a regular phlebotomy program. On day +190, refractory lactic acidosis followed by fatal cardiovascular collapse developed, without evidence of infection. Data from this case demonstrates that NST may correct the erythropoietic defect of CSA.
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References
Bottomley SS, Muller-Eberhard U . Pathophysiology of heme synthesis. Semin Hematol 1988; 25: 282–302.
Urban C, Binber B, Hauer C, Lanzer G . Congenital sideroblastic anemia successfully treated by allogeneic bone marrow transplantation. Bone Marrow Transplant 1992; 10: 373–375.
Lucarelli G, Giardini C, Baronciani D . Bone marrow transplantation in thalassemia. Semin Hematol 1995; 32: 297–303.
Gonzalez MI, Caballero D, Vazquez L et al. Allogeneic peripheral stem cell transplantation in a case of hereditary sideroblastic anaemia. Br J Haematol 2000; 109: 658–660.
Slavin S, Nagler A, Naparstek E . Nonmyeloablative stem cell transplantation and cell therapy as an alternative to conventional bone marrow transplantation with lethal cytoreduction for the treatment of malignant and nonmalignant hematologic diseases. Blood 1998; 91: 756–763.
McSweeney P, Storb R . Mixed chimerism: preclinical studies and clinical application. Biol Blood Marrow Transplant 1999; 5: 192–203.
Richards J, Childs R . Non-myeloablative stem cells transplants. Br J Haematol 2000; 111: 6–17.
McSweeney PA, Niederwieser D, Shizuru JA et al. Hematopoietic cell transplantation in older patients with hematologic malignancies: replacing high-dose cytotoxic therapy with graft-versus-host disease. Blood 2001; 97: 3390–3400.
Angelucci E, Muretto P, Lucarelli G et al. Treatment of iron overload in the ‘Ex-Thalassemic’. Ann NY Acad Sci 1998; 850:288–293.
Muretto P et al. Reversibility of cirrhosis in patients cured of thalassemia by bone marrow transplantation. Ann Intern Med 2002; 136: 667–672.
Marioti E, Angelucci E, Agostini A . Evaluation of cardiac status in iron-loaded thalassemia patients following bone marrow transplantation: improvement in cardiac function during reduction in body iron burden. Br J Haematol 1998; 103: 916–921.
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Medeiros, B., Kolhouse, J., Cagnoni, P. et al. Nonmyeloablative allogeneic hematopoietic stem cell transplantation for congenital sideroblastic anemia. Bone Marrow Transplant 31, 1053–1055 (2003). https://doi.org/10.1038/sj.bmt.1704038
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DOI: https://doi.org/10.1038/sj.bmt.1704038