Risk Factors and Prognostic Scoring Systems in Argentinean Patients with MDS: A Multicentric Study

BELLI, CAROLINA; DE DIOS SOLER, MARCELA; BARCALÁ, VIRNA; FLORES, GABRIELA; GONZALEZ, JACQUELINE; IASTREBNER, MARCELO

Myelodysplastic Syndromes (MDS): Risk Factors, Treatment and Prognosis

Nova Science Publishers, Inc

Lugar: New York; Año: 2016; p. 115 - 140

One of the most challenging problems in hematology is the heterogeneous group of clonal disorders that were formally defined as Myelodysplastic Syndromes (MDS) by the French¬American¬British Cooperative Group (FAB) in 1982, and subsequently by the World Health Organization (WHO) in 2001 and in 2008. The bone marrow (BM) is usually normo/hiper-cellular, displays various morphologic abnormalities and the stem cells show defective capacity for self-renewal and differentiation leading into an ineffective hematopoiesis and cytopenias.The clinical course is highly variable, ranging from stable disease over 10 years to death within a few months due to complications associated with their cytopenias or leukemic transformation. This great variability in the outcome of MDS complicates decision-making regarding therapies and prognostic characterization of individual patients is vital prior initiating treatment. In order to overcome the limitations of the FAB classification, various scoring systems have been designed during this last 30 years. The International Prognostic Scoring System (IPSS) has been widely adopted becoming in the gold standard for risk assessment. In 2012, it has been revised (IPSS-R) defining five groups of risk based on five cytogenetic groups, new clinical cut-points for relevant cytopenias and for the percentage of BM blasts. However, the IPSS and IPSS-R exclude secondary MDS and chronic myelomonocytic leukemia with leukocytosis, which represent a large proportion of patients. Other disease related factors have been proposed such us the presence of myelofibrosis, LDH and ferritin level or the transfusion dependency instead of the hemoglobin level. Some others, related to patient?s characteristic, which include age, performance status and the co-existence of comorbidities, have also been included in different scoring systems.Argentinean MDS Registry was created in 2008 and is sponsored by the Argentinean Society of Hematology. The main objective is to collect MDS related data in order to describe epidemiological characteristics, prognostic factors and scoring risk stratifications in our population. After 7 years, 14 institutions from Argentine have been reporting data from 532 patients (89% with de novo MDS) diagnosed mostly since 2007. The median age was 72 (17-95) years with a gender ratio (M/F) of 1.3. During the follow-up (median: 18 months, range: 1-129 m), 104 (20%) patients evolved to AML and 211 (40%) died. Gender, percentage of BM blast, hemoglobin level, platelet and neutrophil counts, cytogenetic groups of risk, presence of myelofibrosis, and red blood cell transfusion requirement were significant predictive variables for prognosis The IPSS, IPSS-R, WPSS revised according to hemoglobin levels, MDA score, and Charlson?s Comorbidities Index allowed us to differentiate groups with different outcomes (Kaplan-Meier and Long-Rank test, p < 0.05).This chapter thus reviews different prognostic factors and stratifications of risk that have been published during this past years evaluating them in our population based registry. Descriptive studies are necessary not only to establish epidemiological features useful for public health strategies but also to confirm prognosis factors and to validate scoring systems to properly adapt therapeutic schemes.