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Long-term survivors in myelodysplastic syndromes: a report on 63 cases and comparison with short and intermediate
Abstract:
Sixty-three of 373 patients (17%) with de novo myelodysplastic syndrome (MDS) survived more than 5 yr (long survivors). At diagnosis, they usually had no or only moderate cytopenias; 63% of them had marrow blasts < 5%; only 1 patient had circulating blasts; 60% had refractory anaemia (RA) or refractory anaemia with ringed sideroblasts (RARS); 19% of the 43 patients who were karyotyped had an abnormal clone, and only 2 patients had complex cytogenetic findings. Only 4 of the 63 patients progressed to another FAB type within 5 yr of diagnosis, from RA to refractory anaemia with excess of blasts (RAEB) or chronic myelomonocytic leukaemia (CMML). After 5 yr, 9 patients showed progression (including progression to AML in 5 patients). The other patients remained stable (43 cases) or died from cytopenias or unrelated disorders (11 cases). Except for 3 patients who achieved prolonged complete remission with intensive chemotherapy, and possibly 3 patients who responded to low dose Ara C, prolonged survival seemed to result mainly from the low natural course of the disease. Although, as expected, significant differences for age, cytopenias, circulating and bone marrow blasts, and karyotype were seen between short (< 2 yr) and long (> 5 yr) very few significant differences were seen between intermediate and long survivors. These 2 subgroups only differed by significantly lower age and higher haemoglobin level in long survivors. Multivariate scoring systems (Bournemouth, Sanz's and Lille scores) proved of relatively limited value in differentiating them.(ABSTRACT TRUNCATED AT 250 WORDS)
Insights
Seventeen percent of myelodysplastic syndrome (MDS) patients survive over five years, often with mild symptoms and favorable genetics. Their prolonged survival is mainly due to the disease
Area of Science:
- Hematology
- Oncology
- Internal Medicine
Background:
- Myelodysplastic syndromes (MDS) are a group of clonal hematopoietic stem cell disorders.
- Long-term survival in MDS is variable and influenced by disease characteristics and patient factors.
Purpose of the Study:
- To identify characteristics of de novo myelodysplastic syndrome (MDS) patients who achieve long-term survival (>5 years).
- To analyze progression patterns and factors differentiating long-term survivors from others.
Main Methods:
- Retrospective analysis of 373 de novo MDS patients.
- Evaluation of clinical, laboratory, and cytogenetic data at diagnosis.
- Assessment of survival, progression, and response to treatment.
Main Results:
- 17% (63/373) of patients survived >5 years.
- Long survivors typically had no/moderate cytopenias, <5% marrow blasts, and often refractory anemia (RA) or refractory anemia with ringed sideroblasts (RARS).
- Few long survivors progressed to other FAB types or acute myeloid leukemia (AML); most remained stable.
Conclusions:
- De novo MDS patients with favorable initial characteristics (mild cytopenias, low blast counts, specific cytogenetics) have a higher likelihood of long-term survival.
- Disease progression is infrequent in long-term survivors, with survival often attributed to the indolent nature of the disease.
- Multivariate scoring systems showed limited value in differentiating intermediate and long-term survivors.