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Long‐term outcome of acquired aplastic anaemia in children: comparison between immunosuppressive therapy and bone marrow transplantation
Author(s) -
Kojima Seiji,
Horibe Keizo,
Inaba Jun,
Yoshimi Ayami,
Takahashi Yoshiyuki,
Kudo Kazuko,
Kato Koji,
Matsuyama Takaharu
Publication year - 2000
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/j.1365-2141.2000.02289.x
Subject(s) - medicine , aplastic anemia , cumulative incidence , bone marrow , incidence (geometry) , transplantation , gastroenterology , salvage therapy , surgery , bone marrow failure , pediatrics , chemotherapy , haematopoiesis , stem cell , physics , optics , biology , genetics
A total of 100 children under the age of 17 years with acquired aplastic anaemia (AA) were initially treated with immunosuppressive therapy (IST) ( n = 63) or bone marrow transplantation (BMT) ( n = 37) from an HLA‐matched family donor. The projected 10‐year survival rates were 55 ± 8% and 97 ± 3% respectively ( P = 0·004). Because the IST group included 11 non‐responders who were salvaged by BMT from an HLA‐matched unrelated donor, we compared failure‐free survival (FFS) between the groups. The probability of FFS at 10 years was 97 ± 3% for the BMT group, compared with 40 ± 8% for the IST group ( P = 0·0001). Seven patients evolved to myelodysplastic syndrome (MDS) with monosomy 7 and the estimated cumulative incidence of MDS 10 years after diagnosis was 20 ± 7% in the IST group. We compared the outcome of children treated with IST during the two consecutive periods of 1983–91 (group A, n = 40) and 1991–8 (group B, n = 23) to assess the impact of combined therapy with anti‐thymocyte globulin and cyclosporin. The probability of FFS at 7 years follow‐up was the same in the two groups (50 ± 8% vs. 40 ± 15%, P = 0·40). We recommend BMT as first‐line therapy in paediatric severe AA patients with an HLA‐matched family donor. Alternative donor BMT is recommended as salvage therapy in patients who relapse or do not respond to initial IST.