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Blood, Vol. 95 No. 4 (February 15), 2000:
pp. 1188-1194
Allogeneic and syngeneic marrow transplantation for
myelodysplastic syndrome in patients 55 to 66 years of age
H. J. Deeg,
H. M. Shulman,
J. E. Anderson,
E. M. Bryant,
T. A. Gooley,
J. T. Slattery,
C. Anasetti,
A. Fefer,
R. Storb, and
F.
R. Appelbaum
From the Fred Hutchinson Cancer Research Center and the University
of Washington, Seattle, WA.
We carried out bone marrow transplantation (BMT) in 50 patients with
myelodysplastic syndrome (MDS) who were 55.3 to 66.2 years of
age (median, 58.8 years). According to the criteria of the
French-American-British (FAB) classification, 13 patients had
refractory anemia (RA), 19 had RA with excess blasts (RAEB), 16 had
RAEB in transformation or acute myelogenous leukemia (RAEB-T/AML), and
2 had chronic myelomonocytic leukemia (CMML). According to the
recently established International Prognostic Scoring System (IPSS),
available for 45 patients, 2 patients were considered low risk; 14, intermediate 1 risk; 19, intermediate 2 risk; and 10, high risk.
Conditioning regimens were cyclophosphamide (CY) (120 mg/kg of body
weight) plus 12-Gy fractionated total-body irradiation (FTBI)
(n = 15), CY plus FTBI with lung and liver shielding
(n = 4), busulfan (7 mg/kg) plus FTBI (n = 4), or busulfan (16 mg/kg) plus CY (n = 27). The busulfan-plus-CY group included 16 patients in whom busulfan was targeted to plasma levels of 600 to 900 ng/mL. In these 16 patients, steady-state levels of busulfan actually
achieved were 714 to 961 ng/mL (mean ± SD, 845 ± 64 ng/mL; median,
838 ng/mL). The donors were HLA-identical siblings for 34 patients,
HLA-nonidentical family members for 4, identical twins for 4, and
unrelated volunteers for 6. All 46 patients surviving > 21 days had
engraftment, and 22 patients (44%) are surviving 9 to 80 months after
BMT. Specifically, among 13 patients with RA, 1 had relapse (cumulative
incidence [CI] at 3 years, 8%) and 8 are surviving, for a
Kaplan-Meier (KM) estimate of survival at 3 years of 59% (disease-free
survival [DSF], 53%). Among 19 patients with RAEB, 3 had relapse (CI at 3 years, 16%), and 8 are surviving disease free (KM
estimate at 3 years, 46%). Among 18 patients with RAEB-T/AML or CMML,
6 had relapse (CI at 3 years, 28%), and the KM estimate of DSF at 3 years is 33%. Relapse-free survival had an inverse correlation with
cytogenetic risk classification and with the risk score according to
the IPSS. Survival in all FAB categories was highest among patients
enrolled in a protocol in which busulfan plasma levels were targeted to
600 to 900 ng/mL. These data indicate that BMT can be carried out
successfully in patients with MDS who are older than 55 years of age.
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