The Japanese Journal of Pediatric Hematology
Online ISSN : 1884-4723
Print ISSN : 0913-8706
ISSN-L : 0913-8706
An Evaluation of Current Aplastic Anemia Therapies and in Vitro Culture Findings for Therapy Selection and Prognostic Assessment
Tadaatsu ITOShuko OKANOTakuya HATTORIYorikazu ISHIKAWAKozue NAKAMURA
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JOURNAL FREE ACCESS

1999 Volume 13 Issue 2 Pages 79-85

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Abstract
Nine of severe aplastic anemia (SAA) patients who lacked an identical HLA donor for a marrow transplant underwent testing of various therapeutic modalities. Actuarial survivals of these patients at 3 and 5 years were 100% and 87.5%, respectively. Response rates were greater in patients treated with antithymocyte globulin (ATG) /antilymphocyte globulin (ALG) plus methylprednisolone (mPSL). The long-term administration of the granulocyte colony-stimulating factor (G-CSF) induced a multilineage hematological recovery in 3 of 4 patients, and a dose-dependent increase/decrease in the neutrophilic and platelet counts was observed in 2. A patient who underwent lymphocytapheresis 14 years ago remains in hematological remission without need of further therapy. The prognoses have been good for patients with subnormal to normal bone marrow CFU-C and for patients with an early increase of CFU-Cs after immunosuppressive therapy. Three patients with CFU-C-suppressing T lymphocytes have exhibited a good response to ATG/ALG therapy. Long-term bone marrow cultures (LTBMCs) revealed an impaired CFU-C generation; however, the prognoses of 2 patients in whom CFU-C production was maintained for 8 weeks remain good. Although ATG/ALG were found to be effective therapies for patients with SAA, the HD γgl and G-CSF therapies are also beneficial. Lymphocytapheresis might be one of the useful therapeutic modalities for SAA patients. A bone marrow CFU-C assay and an LTBMC may be useful for selecting the proper treatment regimen for SAA patients.
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