Myelodysplastic syndrome in the new millennium. How to classify and cure patients?

Czech version

Authors: J. Čermák ¹; A. Vítek ¹; K. Michalová ¹; J. Březinová ¹; Z. Zemanová ²
Authors‘ workplace: Ústav hematologie a krevní transfuze, Praha, ředitel prof. MUDr. Pavel Klener, DrSc. ¹; Centrum nádorové cytogenetiky Ústavu klinické biochemie a laboratorní diagnostiky 1. LF UK a VFN, Praha, ředitel prof. MUDr. Tomáš Zima, DrSc. ²
Published in: Vnitř Lék 2005; 51(1): 20-30
Category: Original Contributions

Overview

Aim of the study and methods:
A retrospective analysis of the relationship between the initial classification according to either FAB or WHO classification and survival was performed in a group of 197 patients with primary myelodysplastic syndrome (MDS), who were also subdivided into risk groups according to the International Prognostic Scoring System (IPSS). The influence of stem cell transplantation (SCT) or supportive therapy on survival of different subgroups of patients was also studied.

Results:
A classification of 107 patients on the base of WHO criteria revealed a significant difference in median survival between patients with „pure“ RA and RCMD (78.2 months vs. 42.3 months, P = 0.001). Molecular cytogenetics methods (fluorescent in situ hybridization – FISH) were crucial for classification and prognosis of patients with 5q deletion and enabled us to discriminate patients with additional karyotype abnormalities and adverse prognosis from those with isolated deletion of 5q. In patients with advanced MDS (RAEB, RAEB-T), SCT substantially prolonged median survival in both patients with RAEB and RAEB-T (38.4 months vs. 11.5 months, p < 0.001) and patients with intermediate II. and high risk according to IPSS (36.8 months vs. 12.0 months, p < 0.001) in comparison to supportive treatment or/and chemotherapy only. On the other hand, SCT did not significantly prolong median survival either in RA patients (62.3 months vs. 62.4 months, P > 0.8) or in those with low or intermediate I. IPSS risk (57.2 months vs. 60.8 months, P = 0.8), mainly because of a high rate of SCT related mortality. Classification of RA patients according to the WHO criteria showed no benefit from SCT for survival in both RA and RCMD subgroups. Only more detailed subclassification on the base of combined WHO morphologic and IPSS cytogenetic criteria revealed a subgroup of non-transplanted RCMD patients and „poor“ karyotype, who survived only 9.2 months in contrast to transplanted with median survival of 81.8 months. Presence of adverse karyotype abnormalities was the most important independent variable determining survival and leukemic transformation in multivariate regression analysis.

Conclusion:
Combination of classical morphology with molecular cytogenetics methods may improve identification of risk patients within RA subgroup, who may benefit from early SCT despite a high risk of SCT related complications.

Key words:
myelodysplastic syndrome – classification – prognosis – treatment – transplantation – karyotype

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