C007 Validation of the WHO proposals for the classification of unclassifiable myelodysplastic syndromes

Germing, Ulrich; Nachtkamp, Kathrin; Strupp, C.; Hildebrandt, Barbara; Haas, Richard de Goeij-de; Giagounidis, Aristoteles; Kuendgen, Andrea; Gattermann, Norbert

doi:10.1016/s0145-2126(09)70045-1

Cited by 54 publications

(76 citation statements)

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“…Although RCMD without RS and RCMD-RS entities were merged in the 2008 WHO classification [3], we differentiated them in Table I to point out that more evident differences were observed in the pure RARS subtype. RARS accounts for 1.5-12% of MDS patients and occurs primarily in older cases with median age of 60-73 years [3,22,[33][34][35] and more frequent in male than females [3,33] or with a similar sex ratio [34,35]. The median age for pure RARS subtype, which accounted for 5.0% of the whole series, was 70 years old (mean age: 67 years) with a M/F ratio of 0.8 and was detected in 2.5, 10.4, and 1.2% of Ar, Br and Ch cases, respectively.…”

Section: Discussionmentioning

confidence: 99%

Myelodysplastic syndromes in South America: A multinational study of 1080 patients

et al. 2015

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There are previously reported data describing differences between Asian and European patients with Myelodysplastic Syndromes (MDS), few direct comparisons based on cancer registration characteristics or using cohorts to validate scoring systems. This is the first study from South-America, which attempts to describe demographic, clinical features, and outcome of MDS patients. We retrospectively analyzed 1,080 patients with de novo MDS from Argentina (635), Brazil (345), and Chile (100). Chilean patients were younger (P 5 0.001) with female preponderance (P 5 0.071). Brazilian series showed a higher predominance of RARS subtype regarding FAB and WHO classifications (P < 0.001). Hemoglobin levels were significantly lower in Brazilian and Chilean series (P < 0.001), and Chilean series also showed a lower platelet count (P 5 0.028), with no differences concerning the neutrophil count, % BM blast, and the distribution of cytogenetic risk groups (P > 0.05). Chilean series depicted a lower overall survival (OS; 35 months vs. 56 months-Argentine; 55 monthsBrazil, P 5 0.030), which was consistent with a higher predominance of the high-risk group according both to the IPSS and IPSS-R (P 5 0.046 and P < 0.001). The IPSS-R system and its variables showed a good reproducibility to predict clinical outcome for the whole South-American population. Epidemiological and clinical characteristics, distribution among prognostic subgroups, the OS, and the access to disease modifying therapies were more similar between Argentinean and Brazilian compared with Chilean MDS series. This will need further analysis in a larger group of patients. Descriptive and comparative studies are necessary to establish epidemiological features useful for public health attitudes to generate suitable therapeutic schemes.

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Section: Discussionmentioning

confidence: 99%

Myelodysplastic syndromes in South America: A multinational study of 1080 patients

et al. 2015

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“…Several classifications of MDS exist, two of which are frequently used. The World Health Organization (WHO) classification is being used as a diagnostic tool, based on morphologic factors and cytogenetic findings [3][4][5], and the international prognostic scoring system (IPSS) serves as a prognostic tool.…”

Section: Discussionmentioning

confidence: 99%

Cost of transfusion-dependent myelodysplastic syndrome (MDS) from a German payer’s perspective

et al. 2010

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Background: No curative treatment exists for patients with myelodysplastic syndrome (MDS) besides allogeneic stem cell transplantation. Hence, palliative treatment is provided for a life time accruing high health care cost. As no cost study of MDS exists in Germany, the objective of this study was to assess and analyse costs of transfusion-dependent low/intermediate-1 risk MDS in Germany from a payers' perspective. Patients and methods: 116 low/intermediate-1 risk transfusion-dependent MDS patients with and without isolated 5q-deletion from seven centers were identified. Claims data and patient records of the previous five years were used to collect health care utilization data retrospectively. Publicly available tariff books and remuneration schemes were applied to evaluate mean costs per year in Euro with 2007 as base year. Results: The annual cost of MDS patients was estimated at €14,883. Subgroup analyses showed differences in patient's characteristics and outcomes among patients treated at a hospital based versus an office based setting. Patients treated at the hospital based registry show higher cost where as the reasons for that still need to be detected. Overall per annum direct costs range from €12,543 (SD 12,967) to €24,957 (SD 36,399) in different subgroups of patients. In both groups, patients with 5q-deletion use more medication than those without deletion. Mean costs for medication in the office based setting are €5,902 for patients with isolated 5q-deletion vs. €3,932 with no deletion, respectively. Conclusion: MDS leads to a high health care utilization and resulting costs for the health care system which requires a detailed analysis of underlying services.Response to Reviewers: For reasons of a better outline, we decided to respond to your comments in a word document which is attached. Response to reviewer's comments Reviewer's comments ResponsesReviewer #1:In this retrospective study, the authors have evaluated the annual cost of MDS transfused patients in Germany. They stated that patients with 5q-deletion used significantly more resources and especially more medication than those without deletion. This paper raise two majors Criticisms : -1st. The way the data were retrospectively collected raise some issues.In fact the authors compared MDS and del5q but the collection of data were rather different in the two groups and this might also (in part) explain the difference observed in the two groups.Thank you for raising this point. We are aware of this limitation and tried to point it out in the discussion. As this was addressed as a major point by the first reviewer we decided that the respective section needed a revision, which we did in the results and discussion section.To further address this point that was also raised by the editor we conducted additional analyses of the data and present results for the center 7 (MDS registry at a university) separately in a new table 3. By this the differences in data collection and composition of the respective cohorts can be reflected. The fi...

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“…4 Especially disappointing is Table 1 in the letter, citing current COG indications for HSCT. 1 It includes ALL with t(9;22) genotype or hypodiploidy despite COG's recent publications suggesting no advantage of HSCT for these subtypes of ALL.…”

Section: Conflict Of Interestmentioning

confidence: 99%

“…This risk is increased from refractory cytopenia with unilineage dysplasia, for example, refractory anemia (RA), with about 2% of cases progressing at 5 years to about 40% in cases with RA with excess blasts (RAEB-2). 1 Cytogenetic abnormalities have prognostic relevance and these have been incorporated into the IPSS score. 2 In contrast, only little is known about molecular aberrations in MDS, especially those associated with progression from MDS to AML.…”

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confidence: 99%

Mutation analysis for RUNX1, MLL-PTD, FLT3-ITD, NPM1 and NRAS in 269 patients with MDS or secondary AML

et al. 2010

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C007 Validation of the WHO proposals for the classification of unclassifiable myelodysplastic syndromes

Cited by 54 publications

References 0 publications

Myelodysplastic syndromes in South America: A multinational study of 1080 patients

Myelodysplastic syndromes in South America: A multinational study of 1080 patients

Cost of transfusion-dependent myelodysplastic syndrome (MDS) from a German payer’s perspective

Mutation analysis for RUNX1, MLL-PTD, FLT3-ITD, NPM1 and NRAS in 269 patients with MDS or secondary AML

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