Hj. Deeg et al., Allogeneic and syngeneic marrow transplantation for myelodysplastic syndrome in patients 55 to 66 years of age, BLOOD, 95(4), 2000, pp. 1188-1194
We carried out bone marrow transplantation (BMT) in 50 patients with myelod
ysplastic syndrome (MDS) who were 55.3 to 66.2 years of age (median, 58.8 y
ears). According to the criteria of the French-American-British (FAB) class
ification, 13 patients had refractory anemia (RA), 19 had RA with excess bl
asts (RAEB), 16 had RAEB in transformation or acute myelogenous leukemia (R
AEB-T/AML), and 2 had chronic myelomonocytic leukemia (CMML). According to
the recently established International Prognostic Scoring System (IPSS), av
ailable for 45 patients, 2 patients were considered low risk; 14, intermedi
ate 1 risk; 19, intermediate 2 risk; and 10, high risk. Conditioning regime
ns were cyclophosphamide (CY) (120 mg/kg of body weight) plus 12-Gy fractio
nated total-body irradiation (FTBI) (n = 15), CY plus FTBI with lung and li
ver shielding (n = 4), busulfan (7 mg/kg) plus FTBI (n = 4), or busulfan (1
6 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 thes
e 16 patients, steady-state levels of busulfan actually achieved were 714 t
o 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 member
s for 4, identical twins for 4, and unrelated volunteers for 6. AII 46 pati
ents surviving > 21 days had engraftment, and 22 patients (44%) are survivi
ng 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, fo
r 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 y
ears, 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, 2
8%), and the KM estimate of DSF at 3 years is 33%. Relapse-free survival ha
d an inverse correlation with cytogenetic risk classification and with the
risk score according to the IPSS. Survival in ail FAB categories was highes
t among patients enrolled in a protocol in which busulfan plasma levels wer
e targeted to 600 to 900 ng/mL. These data indicate that BMT can be carried
out successfully in patients with MDS who are order than 55 years of age.