Abstract
Over 15 years, 42 children aged 2-14 years were diagnosed as having acquired aplastic anaemia. Adequate clinical details were available for 38 children who were categorised as very severe (n = 13), severe (n = 16), or nonsevere (n = 9) by the modified Camitta criteria. Treatment varied over the study period. Seven children received a bone marrow allograft from a full match family donor and three a matched unrelated donor transplant after failed treatment with antilymphocyte globulin. The remainder were treated with antilymphocyte globulin (n = 11), antilymphocyte globulin and oxymetholone (n = 4), oxymetholone with or without prednisolone (n = 12), or supportive treatment alone (n = 1). With a minimum follow up of one year since treatment, the five year survival was 70% for bone marrow transplantation with a family donor, 30% for antilymphocyte globulin, and 25% for oxymetholone. All three children with a matched unrelated donor transplant died. The prognosis of acquired aplastic anaemia remains poor for most children and new approaches to treatment are urgently required.
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- Alter B. P., Potter N. U., Li F. P. Classification and aetiology of the aplastic anaemias. Clin Haematol. 1978 Oct;7(3):431–465. [PubMed] [Google Scholar]
- Bacigalupo A., Hows J., Gordon-Smith E. C., Gluckman E., Van Lint M. T., Congiu M., James D. C., Barrett A. J., Gmur J., De Planque M. M. Bone marrow transplantation for severe aplastic anemia from donors other than HLA identical siblings: a report of the BMT Working Party. Bone Marrow Transplant. 1988 Nov;3(6):531–535. [PubMed] [Google Scholar]
- Bayever E., Champlin R., Ho W., Lenarsky C., Storch S., Ladisch S., Gale R. P., Feig S. A. Comparison between bone marrow transplantation and antithymocyte globulin in treatment of young patients with severe aplastic anemia. J Pediatr. 1984 Dec;105(6):920–925. doi: 10.1016/s0022-3476(84)80078-5. [DOI] [PubMed] [Google Scholar]
- Camitta B., O'Reilly R. J., Sensenbrenner L., Rappeport J., Champlin R., Doney K., August C., Hoffmann R. G., Kirkpatrick D., Stuart R. Antithoracic duct lymphocyte globulin therapy of severe aplastic anemia. Blood. 1983 Oct;62(4):883–888. [PubMed] [Google Scholar]
- Clausen N. A population study of severe aplastic anemia in children. Incidence, etiology and course. Acta Paediatr Scand. 1986 Jan;75(1):58–63. doi: 10.1111/j.1651-2227.1986.tb10157.x. [DOI] [PubMed] [Google Scholar]
- Halpérin D. S., Grisaru D., Freedman M. H., Saunders E. F. Severe acquired aplastic anemia in children: 11-year experience with bone marrow transplantation and immunosuppressive therapy. Am J Pediatr Hematol Oncol. 1989 Fall;11(3):304–309. [PubMed] [Google Scholar]
- Hoffman R., Zanjani E. D., Lutton J. D., Zalusky R., Wasserman L. R. Suppression of erythroid-colony formation by lymphocytes from patients with aplastic anemia. N Engl J Med. 1977 Jan 6;296(1):10–13. doi: 10.1056/NEJM197701062960103. [DOI] [PubMed] [Google Scholar]
- Hows J. M., Yin J. L., Marsh J., Swirsky D., Jones L., Apperley J. F., James D. C., Smithers S., Batchelor J. R., Goldman J. M. Histocompatible unrelated volunteer donors compared with HLA nonidentical family donors in marrow transplantation for aplastic anemia and leukemia. Blood. 1986 Dec;68(6):1322–1328. [PubMed] [Google Scholar]
- Jones T. C. Theoretical and practical issues concerning the use of rhGM-CSF and rhIL-3: present status. Bone Marrow Transplant. 1990 Jan;5 (Suppl 1):36–37. [PubMed] [Google Scholar]
- Leonard E. M., Raefsky E., Griffith P., Kimball J., Nienhuis A. W., Young N. S. Cyclosporine therapy of aplastic anaemia, congenital and acquired red cell aplasia. Br J Haematol. 1989 Jun;72(2):278–284. doi: 10.1111/j.1365-2141.1989.tb07695.x. [DOI] [PubMed] [Google Scholar]
- Liang D. C., Lin K. H., Lin D. T., Yang C. P., Hung K. L., Lin K. S. Post-hepatitic aplastic anaemia in children in Taiwan, a hepatitis prevalent area. Br J Haematol. 1990 Apr;74(4):487–491. doi: 10.1111/j.1365-2141.1990.tb06339.x. [DOI] [PubMed] [Google Scholar]
- Locasciulli A., van't Veer L., Bacigalupo A., Hows J., Van Lint M. T., Gluckman E., Nissen C., McCann S., Vossen J., Schrezenmeier A. Treatment with marrow transplantation or immunosuppression of childhood acquired severe aplastic anemia: a report from the EBMT SAA Working Party. Bone Marrow Transplant. 1990 Sep;6(3):211–217. [PubMed] [Google Scholar]
- Marsh J. C., Hows J. M., Bryett K. A., Al-Hashimi S., Fairhead S. M., Gordon-Smith E. C. Survival after antilymphocyte globulin therapy for aplastic anemia depends on disease severity. Blood. 1987 Oct;70(4):1046–1052. [PubMed] [Google Scholar]
- McGlave P. B., Haake R., Miller W., Kim T., Kersey J., Ramsay N. K. Therapy of severe aplastic anemia in young adults and children with allogeneic bone marrow transplantation. Blood. 1987 Nov;70(5):1325–1330. [PubMed] [Google Scholar]
- Najean Y., Girot R., Baumelou E. Prognostic factors and evolution of acquired aplastic anemia in childhood. A prospective analysis of 48 androgen-treated cases. Am J Pediatr Hematol Oncol. 1982 Fall;4(3):273–283. [PubMed] [Google Scholar]
- Ozsoylu S., Coşkun T., Minassazi S. High dose intravenous glucocorticoid in the treatment of childhood acquired aplastic anaemia. Scand J Haematol. 1984 Sep;33(3):309–316. doi: 10.1111/j.1600-0609.1984.tb02233.x. [DOI] [PubMed] [Google Scholar]
- Sullivan R., Quesenberry P. J., Parkman R., Zuckerman K. S., Levey R. H., Rappeport J., Ryan M. Aplastic anemia: lack of inhibitory effect of bone marrow lymphocytes on in vitro granulopoiesis. Blood. 1980 Oct;56(4):625–632. [PubMed] [Google Scholar]
- Tichelli A., Gratwohl A., Würsch A., Nissen C., Speck B. Late haematological complications in severe aplastic anaemia. Br J Haematol. 1988 Jul;69(3):413–418. doi: 10.1111/j.1365-2141.1988.tb02382.x. [DOI] [PubMed] [Google Scholar]
- Werner E. J., Stout R. D., Valdez L. P., Harris R. E. Immunosuppressive therapy versus bone marrow transplantation for children with aplastic anemia. Pediatrics. 1989 Jan;83(1):61–65. [PubMed] [Google Scholar]
- Young N. S., Issaragrasil S., Chieh C. W., Takaku F. Aplastic anaemia in the Orient. Br J Haematol. 1986 Jan;62(1):1–6. doi: 10.1111/j.1365-2141.1986.tb02893.x. [DOI] [PubMed] [Google Scholar]
- de Planque M. M., Kluin-Nelemans H. C., van Krieken H. J., Kluin P. M., Brand A., Beverstock G. C., Willemze R., van Rood J. J. Evolution of acquired severe aplastic anaemia to myelodysplasia and subsequent leukaemia in adults. Br J Haematol. 1988 Sep;70(1):55–62. doi: 10.1111/j.1365-2141.1988.tb02434.x. [DOI] [PubMed] [Google Scholar]