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Blood, 1 August 2002, Vol. 100, No. 3, pp. 791-798
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Myelodysplastic syndrome is not merely "preleukemia"
Maher Albitar,
Taghi Manshouri,
Yu Shen,
Diane Liu,
Miloslav Beran,
Hagop M. Kantarjian,
Anna Rogers,
Iman Jilani,
Chung Wu Lin,
Sherry Pierce,
Emil J. Freireich, and
Elihu H. Estey
From the Departments of Hematopathology, Biostatistics,
and Leukemia, The University of Texas MD Anderson Cancer Center,
Houston.
Myelodysplastic syndrome (MDS) is a disease characterized by
ineffective hematopoiesis. There are significant biologic and clinical
differences between MDS and acute myeloid leukemia (AML). We studied a
cohort of 802 patients, 279 (35%) with newly diagnosed MDS and 523 (65%) with newly diagnosed AML, and compared clinical and biologic
characteristics of the 2 groups. Complete clinical and cytogenetic data
were available on all patients, and a subgroup of patients was studied
for apoptosis, angiogenesis, proliferation, and growth factors. Our
results demonstrate that MDS is a discrete entity that is different
from AML and is characterized primarily by increased apoptosis in early
and mature hematopoietic cells. Using cell sorting and loss of
heterozygosity, we demonstrate that the leukemic cells from MDS
patients are capable of differentiation into mature myeloid cells and
monocytes. We also demonstrate that there is a significant overlap
between AML and MDS when MDS is defined on the basis of an arbitrary
percentage of blasts of 20% or 30%. These data suggest that despite
similarities between AML and MDS in their responses to treatment and
outcomes, MDS is biologically and clinically different from AML and
should not be considered an early phase of AML. The data indicate that
MDS must be better defined on the basis of its biology rather than the
percentage of blasts; further, the data suggest that there is a need to
develop therapeutic approaches that specifically address the biologic abnormalities of MDS.
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