1. [Transplantation of the bone marrow from a HLA-compatible unrelated donor after immunoablative conditioning in children with acquired aplastic anemia unresponsive to combined immunosuppressive therapy: preliminary results]
- Author
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Novichkova, G. A., Maschan, M. A., Shipitsyna, I. P., Skvortsova, Y. V., Persiantseva, M. I., Lebedeva, L. L., Bobrynina, V. O., Baidildina, D. D., Goronkova, O. V., Solopova, G. G., Khachatryan, L. A., Petrova, U. N., Suntsova, E. V., Kalinina, I. I., Sinitsyna, V. V., Skorobogatova, E. V., Dmitry Balashov, Dyshlevaya, Z. M., Shelikhova, L. N., Kurnikova, E. E., Trakhtman, P. E., and Maschan, A. A.
- Subjects
Transplantation Conditioning ,Adolescent ,Graft Survival ,lcsh:R ,Anemia, Aplastic ,lcsh:Medicine ,thoracoabdominal irradiation ,unrelated donors ,Combined Modality Therapy ,Disease-Free Survival ,Tissue Donors ,Graft vs Host Reaction ,acquired aplastic anemia ,children ,HLA Antigens ,Child, Preschool ,Cyclosporine ,Humans ,Treatment Failure ,Child ,tacrolimus ,Immunosuppressive Agents ,Antilymphocyte Serum ,Bone Marrow Transplantation ,transplantation - Abstract
Aim. To analyze the efficiency of transplantation of the bone marrow from a HLA-compatible unrelated donor and continued immunosuppressive therapy (IST) in children with aplastic anemia (AA) unresponsive to 2 courses of IST. Subjects and methods. The study enrolled 14 children aged 2-16 years (median 9 years). A control group comprised 26 patients in whom IST was continued. The median interval between the diagnosis of AA and transplantation was 26 months (9-156 months). The conditioning regimen consisted of thoracoabdominal irradiation in a dose of 2 Gy, fludarabin (Flu) 100-150 mg/m2, cyclophosphamide (Cy) 100-200 mg/kg, antithymocyte globulin (ATG) in 11 patients and Flu, Cy, and ATG in 3. A graft-versus-host reaction was prevented with mycophenolate mefetil in all the patients, tacrolimus in 11, and cyclosporin A in 3. Donors were compatible for high-resolution typing of 10/10 and 9/10 alleles in 8 and 6 patients, respectively; the source of a transplant was bone marrow in 13 patients and granulocyte colony-stimulating factor-mobilized peripheral blood precursors in one case. Results. Thirteen patients achieved primary engraftment after single transplantation; one patient did after repeat transplantation. Grades I to II graft-versus-host reaction (GVHR) developed in 9 patients; postengraftment life-threatening infections in 3, extensive chronic GVHR in 2, circumscribed GVHR in 7. All fourteen hemopoietic cell transplant recipients followed for a median 17.5 months (range 1-71 months) were survivors. Conclusion. The likelihood of good survival after unrelated transplantations in AA is much higher than that after continued IST: 100% versus 15±11%.