Bone marrow transplantation for patients with myelodysplasia. Pretreatment variables and outcome.
OBJECTIVE
To determine the efficacy of allogeneic bone marrow transplantation for severe myelodysplasia, and to identify variables predictive of outcome.


METHODS
Case series study.


METHODS
A referral-based bone marrow transplant center.


METHODS
Consecutive series of 59 patients with myelodysplasia or closely related disorders and either life-threatening cytopenia or a progressive increase in marrow blast percentage.


METHODS
Patients were treated with high-dose cyclophosphamide and total body irradiation followed by allogeneic bone marrow transplantation from either an HLA-identical (n = 45) or HLA-partially matched (n = 14) donor.


RESULTS
The product-limit estimate for disease-free survival 3 years after transplant is 45% (95% CI, 32% to 59%). The commonest causes of death after transplant were disease recurrence, interstitial pneumonia, and graft-versus-host disease, accounting for eight deaths each. In a univariate analysis, younger patients, those with shorter disease duration, and those whose disease was characterized by an abnormal cytogenetic karyotype had better survival and disease-free survival than the group as a whole. In a multivariate analysis, younger age and abnormal karyotype were independent predictors of improved disease-free survival and overall survival. Patients who received transplants when they had fewer blasts in their bone marrow had a decreased chance for disease recurrence when compared with patients with excess blasts.


CONCLUSIONS
Bone marrow transplantation offers a potential cure for many patients with myelodysplasia. Best results can be expected in younger patients who receive transplants relatively early in their disease course.
Appelbaum
F R
FR
Fred Hutchinson Cancer Research Center, Seattle, Washington.
Barrall
J
J
Storb
R
R
Fisher
L D
LD
Schoch
G
G
Ramberg
R E
RE
Shulman
H
H
Anasetti
C
C
Bearman
S I
SI
Beatty
P
P
D000293:Adolescent; D000328:Adult; D000367:Age Factors; D016026:Bone Marrow Transplantation; D002066:Busulfan / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000627:therapeutic use; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007621:Karyotyping; D008297:Male; D008875:Middle Aged; D015999:Multivariate Analysis; D009190:Myelodysplastic Syndromes / Q000235:genetics / Q000401:mortality / Q000601:surgery*; D011379:Prognosis; D012008:Recurrence; D014916:Whole-Body Irradiation
D003520:Cyclophosphamide; D002066:Busulfan