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Blood, 1 August 2002, Vol. 100, No. 3, pp. 799-803
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Outcome of 154 patients with severe aplastic anemia who received
transplants from unrelated donors: the Japan Marrow Donor
Program
Seiji Kojima,
Takaharu Matsuyama,
Shunichi Kato,
Hisato Kigasawa,
Ryoji Kobayashi,
Atsushi Kikuta,
Hisashi Sakamaki,
Koichiro Ikuta,
Masahiro Tsuchida,
Yasutaka Hoshi,
Yasuo Morishima, and
Yoshihisa Kodera
From the Japan Marrow Donor Program. Participating
centers are listed in an appendix at the end of this article.
We retrospectively analyzed results for 154 patients with acquired
severe aplastic anemia who received bone marrow transplants between
1993 and 2000 from unrelated donors identified through the Japan Marrow
Donor Program. Patients were aged between 1 and 46 years (median, 17 years). Seventy-nine donor-patient pairs matched at HLA-A, -B, and
-DRB1 loci, as shown by DNA typing. Among the 75 mismatched pairs, DNA
typing of 63 pairs showed that 51 were mismatched at 1 HLA locus (18 HLA-A, 11 HLA-B, 22 HLA-DRB1) and 12 were mismatched at 2 or more loci.
Seventeen patients (11%) experienced either early or late graft
rejection. The incidence of grade III/IV acute graft versus host
disease and chronic graft versus host disease was 20% (range,
7%-33%) and 30% (range, 12%-48%), respectively. Currently, 99 patients are alive, having survived for 3 to 82 months (median, 29 months) after their transplantations. The probability of overall
survival at 5 years was 56% (95% confidence interval, 34%-78%).
Multivariate analysis revealed the following unfavorable factors:
transplantation more than 3 years after diagnosis (relative risk
[RR], 1.86; P = .02), patients older than 20 years (RR,
2.27; P = .03), preconditioning regimen without
antithymocyte globulin (RR 2.28; P = .04), and HLA-A or
-B locus mismatching as determined by DNA typing. Matching of HLA class
I alleles and improvement of preparative regimens should result in
improved outcomes in patients with severe aplastic anemia who receive
transplants from unrelated donors.
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