A prognostic score for patients with lower risk myelodysplastic syndrome

Garcia‐Manero, Guillermo; Shan, Jianqin; Faderl, S.; Cortes, Jorge; Ravandi, Farhad; Borthakur, Gautam; Wierda, William G.; Pierce, Sherry; Estey, Elihu H.; Liu, J.; Huang, Xuelin; Kantarjian, Hagop M.

doi:10.1038/sj.leu.2405070

Cited by 297 publications

(244 citation statements)

References 15 publications

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“…Improvement in the IPSS scoring method has been suggested by Germing et al, who proposed adding LDH as a prognostic variable in addition to the IPSS group [38], and Garcia-Manero et al [39], who proposed refinement of criteria for classifying lower-risk groups. Here, we propose that patients with MDS stratification may be improved using additional recommended risk factors, such as those described earlier.…”

Section: Discussionmentioning

confidence: 99%

Analyzing transformation of myelodysplastic syndrome to secondary acute myeloid leukemia using a large patient database

et al. 2012

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One‐third of patients with myelodysplastic syndrome (MDS) progress to secondary acute myeloid leukemia (sAML), with its concomitant poor prognosis. Recently, multiple mutations have been identified in association with MDS‐to‐sAMLtransition, but it is still unclear whether all these mutations are necessary for transformation. If multiple independent mutations are required for the transformation, sAML risk should increase with time from MDS diagnosis. In contrast, if a single critical biological event determines sAML transformation; its risk should be constant in time elapsing from MDS diagnosis. To elucidate this question, we studied a database of 1079 patients with MDS. We classified patients according to the International Prognostic Scoring System (IPSS), using either the French‐American‐British (FAB) or the World Health Organization (WHO) criteria, and statistically analyzed the resulting transformation risk curves of each group. The risk of transformation after MDS diagnosis remained constant in time within three out of four risk groups, and in all four risk groups, when patients were classified according to FAB or to the WHO‐determined criteria, respectively. Further subdivision by blast percentage or cytogenetics had no influence on this result. Our analysis suggests that a single random biological event leads to transformation to sAML, thus calling for the exclusion of time since MDS diagnosis from the clinical decision‐making process. Am. J. Hematol. © 2012 Wiley Periodicals, Inc.

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Section: Discussionmentioning

confidence: 99%

Analyzing transformation of myelodysplastic syndrome to secondary acute myeloid leukemia using a large patient database

et al. 2012

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“…ORR and OS were also assessed according to age (aged <70 years vs !70 years), transfusion dependence at baseline (no vs yes), azacitidine dose schedule (75 mg/m 2 daily vs 100 mg as a daily fixed dose), total median azacitidine dose per cycle ( 700 mg vs >700 mg), and prior therapy (no vs yes). Patients also were analyzed according to IPSS subgroups (low risk or Int-1 risk) and according to the more recent WHO-based prognostic scoring system (WPSS) 38 and The University of Texas M. D. Anderson Cancer Center (MDACC) model, 39 which is specific to lower risk MDS. Both the WPSS and the MDACC models include parameters that are not present in the IPSS, such as transfusion requirement and WHO classification 38 or age and degree of thrombocytopenia, 39 respectively.…”

Section: Discussionmentioning

confidence: 99%

Azacitidine for the treatment of lower risk myelodysplastic syndromes

Musto

Maurillo

Spagnoli

et al. 2010

Cancer

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BACKGROUND:Azacitidine induces responses and prolongs overall survival compared with conventional care regimens in patients who have high‐risk myelodysplastic syndromes (MDS). However, limited data are available concerning the efficacy and safety of azacitidine in patients who have lower risk MDS.METHODS:The authors retrospectively evaluated 74 patients with International Prognostic Scoring System low‐risk or intermediate 1‐risk MDS, who received azacitidine on a national named patient program. At baseline, 84% of patients were transfusion‐dependent, 57% had received erythropoietin, and 51% were aged >70 years. Azacitidine was administered subcutaneously for 5 days (n = 29 patients), 7 days (n = 43 patients), or 10 days (n = 2 patients) every month at a dose of 75 mg/m2 daily (n = 45 patients) or at a fixed dose of 100 mg daily (n = 29 patients) and for a median of 7 cycles (range, 1‐30 cycles).RESULTS:According to the 2006 International Working Group criteria, overall response rate (ORR) was 45.9%, including complete responses (10.8%), partial responses (9.5%), hematologic improvements (20.3%), and bone marrow complete responses (5.4%). The ORR was 51.6% in 64 patients who completed ≥4 cycles of treatment. The median duration of response was 6 months (range, 1‐30 months). After a median follow‐up of 15 months, 71% of patients remained alive. A survival benefit was observed in responders versus nonresponders (94% vs 54% of patients projected to be alive at 2.5 years, respectively; P < .0014). The most common grade 3 or 4 adverse events were myelosuppression (21.6%) and infection (6.8%).CONCLUSIONS:The current results indicated that azacitidine may be a feasible and effective treatment for patients with lower risk MDS. Cancer 2010. © 2010 American Cancer Society.

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“…Malcovati et al [10], Gattermann et al [19] and Garcia-Manero et al [23] also underlined a negative influence of transfusion-related iron overload on survival in MDS patients. Park et al [16] found no correlation of ferritin concentration with survival, but only with dependency of RBC-transfusions.…”

Section: Discussionmentioning

confidence: 99%

Red Blood Cell Transfusion Dependency and Hyperferritinemia Are Associated with Impaired Survival in Patients Diagnosed with Myelodysplastic Syndromes: Results from the First Polish MDS-PALG Registry

Waszczuk‐Gajda¹,

Mądry²,

Machowicz³

et al. 2016

Adv Clin Exp Med

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Background. Myelodysplastic syndromes (MDS) are a heterogeneous group of clonal stem cell disorders characterized by ineffective hematopoiesis, cytopenias and a risk of progression to acute myeloid leukemia (AML). Anemia is the most frequent cytopenia diagnosed in patients with MDS. Regular RBC transfusions are the only treatment option for about 40% of patients. Transfusion-dependent patients develop secondary iron overload. The influence of serum ferritin (SF) concentration on survival and acute myeloid leukemia transformation in MDS patients remains controversial. The data for the Central European population is scarce and so far there is no description for Poland. Objectives. The aim of this study was to perform a retrospective analysis of the relationship of SF concentration with red blood cell transfusion dependency, survival and transformation to acute myeloid leukemia. Material and Methods. We retrospectively evaluated the data of the 819 MDS patients (58% male; median age 70 years) included in the MDS Registry of the MDS Section of the Polish Adult Leukemia Group (PALG). Results. Analyses were performed on 190 patients diagnosed with MDS, maximal 6 months before inclusion to the registry in order to avoid selection bias (a shorter survival of higher risk MDS patients). Patients with hyperferritinemia higher than 1000 ng/L vs. patients with SF concentration lower than 1000 ng/L had a median survival of 320 days vs. 568 days, respectively (p log-rank = 0.014). The following factors were found to significantly worsen survival: RBC-transfusion dependence (p = 0.0033; HR 2.67L), platelet transfusion dependence (p = 0.0071; HR 3.321), hemoglobin concentration lower than 10 g/dL (p = 0.0036; HR 2.97), SF concentration higher than 1000 ng/L (p = 0.0023; HR = 2.94), platelet count lower than 10 G/L (p = 0.0081 HR = 5.04), acute leukemia transformation (p = 0.0081; HR 1.968).

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A prognostic score for patients with lower risk myelodysplastic syndrome

Cited by 297 publications

References 15 publications

Analyzing transformation of myelodysplastic syndrome to secondary acute myeloid leukemia using a large patient database

Analyzing transformation of myelodysplastic syndrome to secondary acute myeloid leukemia using a large patient database

Azacitidine for the treatment of lower risk myelodysplastic syndromes

Red Blood Cell Transfusion Dependency and Hyperferritinemia Are Associated with Impaired Survival in Patients Diagnosed with Myelodysplastic Syndromes: Results from the First Polish MDS-PALG Registry

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