Use of newer prognostic indices for patients with myelodysplastic syndromes in the low and intermediate-1 risk categories: a population-based study

Valcárcel, David; Sanz, Guillermo; Ortega, Margarita; Nomdedeu, Benet; Luño, Elisa; Díez-Campelo, María; Ardanaz, María Teresa; Pedro, Carmen; Montoro, María Julia; Collado, Rosa; Andreu, Rafael; Marco, Víctor; Cedena, María Teresa; Paz, Raquel de; Tormo, Mar; Xicoy, Blanca; Ramos, Fernando; Bargay, Joan; González, Bernardo; Brunet, Salut; Muñoz, Juan Antonio; Gómez, Valle; Bailén, Alicia; Sánchez, Joaquín; Merchán, Brayan; Cañizo, Consuelo del; Vallespı́, Teresa

doi:10.1016/s2352-3026(15)00067-8

Cited by 25 publications

(28 citation statements)

References 18 publications

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“…Of note, the median survival of the highest risk LR-PSS category 3 in our analysis was significantly longer than originally reported by Garcia-Manero et al (39.8 vs. 14.2 months) [12]. In a recent Spanish analysis of 2,373 IPSS low and INT-1 patients, the median OS of patients reclassified as LR-PSS category 3 (15%) was lower than that of our cohort at 26.2 months (95% CI, 21.2–31.1) [15] but was also significantly higher than originally reported by Garcia-Manero et al [12]. Similarly, 8.5% of our IPSS low and INT-1 patients were reclassified by the IPSS-R to high and very-high risk categories, groups which are currently recommended to be treated with disease-modifying interventions, compared to 4% of the Spanish cohort [15].…”

Section: Discussioncontrasting

confidence: 60%

Comparing the Prognostic Value of Risk stratifying Models for Patients with Lower-Risk Myelodysplastic Syndromes: Is one model better?

et al. 2015

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Some patients classified as having lower-risk (LR)-disease by the International Prognostic Scoring System (IPSS) fare more poorly than predicted. We examined the prognostic utility of IPSS, the MD Anderson LR-Prognostic System (LR-PSS), and the revised IPSS (IPSS-R) in a large cohort of patients classified as having IPSS LR-MDS in the MDS Clinical Research Consortium database. Actual overall survival (OS) was assessed in patients with IPSS LR-MDS (i.e. low and intermediate-1) using Kaplan–Meier methods. Harrell’s c index (HCI) and Akaike information criteria (AIC) were used to compare the models. Median OS of 1,140 eligible patients was 47 months (95% CI, 44–52). Median follow-up was 62 months. HCI values indicating the discriminatory power of the models (higher is better) were better for LR-PSS (0.74, 95% CI, 0.70–0.78) than IPSS-R (0.64, 95% CI, 0.60–0.67) and IPSS (0.64, 95% CI, 0.60–0.68). Similarly, AIC values indicating the goodness of the fit were better for LR-PSS than IPSS-R and IPSS (8,110, 8,147, and 8,150, respectively, lower is better). LR-PSS assigned 25.1% and 37.4% of patients with IPSS LR-MDS into LR-PSS Category 3 and IPSS-R Categories ≥Intermediate, respectively. Of 291 patients (25.5%) who survived ≤24 months from diagnosis, only 37.1% and 45% were classified as LR-PSS category 3 and IPSS-R categories ≥Intermediate, respectively (P = 0.06). While both LR-PSS and IPSS-R distinguish groups with varied survival outcome among patients with IPSS LR-MDS, both tools fail to identify a significant subset with poor OS. Future studies should assess whether patients identified as at increased risk will benefit from earlier interventions with disease-modifying therapies.

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Section: Discussioncontrasting

confidence: 60%

Comparing the Prognostic Value of Risk stratifying Models for Patients with Lower-Risk Myelodysplastic Syndromes: Is one model better?

et al. 2015

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“…WPSS is suggested to mainly improve risk assessment of patients with early‐stage disease (Della Porta et al , ). Another study concluded that the IPSS‐R was the best scoring system to identify high‐risk patients within the lower risk groups in IPSS (Valcarcel et al , ). In our study, 15% of patients with IPSS intermediate‐1 were redistributed to IPSS‐R high risk or very high‐risk group.…”

Section: Discussionmentioning

confidence: 99%

Prognostic scoring systems for myelodysplastic syndromes (MDS) in a population‐based setting: a report from the Swedish MDS register

et al. 2018

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The myelodysplastic syndromes (MDS) have highly variable outcomes and prognostic scoring systems are important tools for risk assessment and to guide therapeutic decisions. However, few population-based studies have compared the value of the different scoring systems. With data from the nationwide Swedish population-based MDS register we validated the International Prognostic Scoring System (IPSS), revised IPSS (IPSS-R) and the World Health Organization (WHO) Classification-based Prognostic Scoring System (WPSS). We also present population-based data on incidence, clinical characteristics including detailed cytogenetics and outcome from the register. The study encompassed 1329 patients reported to the register between 2009 and 2013, 14% of these had therapy-related MDS (t-MDS). Based on the MDS register, the yearly crude incidence of MDS in Sweden was 2·9 per 100 000 inhabitants. IPSS-R had a significantly better prognostic power than IPSS (P < 0·001). There was a trend for better prognostic power of IPSS-R compared to WPSS (P = 0·05) and for WPSS compared to IPSS (P = 0·07). IPSS-R was superior to both IPSS and WPSS for patients aged ≤70 years. Patients with t-MDS had a worse outcome compared to de novo MDS (d-MDS), however, the validity of the prognostic scoring systems was comparable for d-MDS and t-MDS. In conclusion, population-based studies are important to validate prognostic scores in a 'real-world' setting. In our nationwide cohort, the IPSS-R showed the best predictive power.

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“…Median time from diagnosis to treatment in our study was 45 days (range, 21-52 days) for lower-risk patients, while in other studies of lower-risk newly diagnosed MDS patients, it was 130 days (range, 60-450 days; ARCADE study) and 92 days (range, 1-5683 days; SPRESAS study [Spanish registry of ESAs in anemic lowrisk MDS patients]) 22,23 The estimated 3-year OS in the MDS patients we diagnosed was 71% and was significantly higher among lower-risk (IPSS-R ≤ 3.5) than higher-risk patients (IPSS-R > 3.5; 100% vs 0%; P < .001), consistent with previous reports. 16,24,25 Recently, somatic mutations have been described in >80%-90% of patients with MDS. 26 The presence of myeloid mutations appears to be related to MDS pathogenesis; for example, the SF3B1 mutation induces the formation of ring sideroblasts and other MDS-specific phenotypes.…”

Section: Recent Data From the Us Surveillance Epidemiology And Endmentioning

confidence: 99%

Hidden myelodysplastic syndrome (MDS): A prospective study to confirm or exclude MDS in patients with anemia of uncertain etiology

Bastida

López‐Godino

Vicente-Sánchez

et al. 2018

Int J Lab Hematology

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Introduction Diagnosis of myelodysplastic syndromes (MDSs) when anemia is the only abnormality can be complicated. The aim of our study was to investigate the primary causes of anemia and/or macrocytosis of uncertain etiology. Methods We conducted a multicenter, prospective study over 4 months in three hematology laboratories. In step 1, we used an automated informatics system to screen 137 453 hemograms for cases of anemia and/or macrocytosis (n = 2702). In step 2, we excluded all patients whose anemia appeared to be due to a known cause. This left 290 patients had anemia of uncertain etiology. In step 3, we conducted further investigations, including a peripheral blood smear, and analysis of iron, vitamin B12, folate, and thyroid hormone levels. Results A differential diagnosis was obtained in 139 patients (48%). The primary causes of anemia were iron deficiency (n = 59) and megaloblastic anemia (n = 39). In total, 25 hematologic disorders were diagnosed, including 14 patients with MDS (56%). The median age of MDS patients was 80 years, 12 had anemia as an isolated cytopenia, and most (n = 10) had lower‐risk disease (IPSS‐R ≤ 3.5). SF3B1 mutations were most frequent (n = 6) and correlated with the presence of ring sideroblasts (100%) and associated with better prognosis (P = 0.001). Conclusions Our prospective, four‐step approach is an efficient and logical strategy to facilitate the diagnosis of MDS on the basis of unexplained anemia and/or macrocytosis, and may allow the early diagnosis of the most serious causes of anemia. Molecular analysis of genes related to MDS could be a promising diagnostic and prognostic approach.

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Use of newer prognostic indices for patients with myelodysplastic syndromes in the low and intermediate-1 risk categories: a population-based study

Cited by 25 publications

References 18 publications

Comparing the Prognostic Value of Risk stratifying Models for Patients with Lower-Risk Myelodysplastic Syndromes: Is one model better?

Comparing the Prognostic Value of Risk stratifying Models for Patients with Lower-Risk Myelodysplastic Syndromes: Is one model better?

Prognostic scoring systems for myelodysplastic syndromes (MDS) in a population‐based setting: a report from the Swedish MDS register

Hidden myelodysplastic syndrome (MDS): A prospective study to confirm or exclude MDS in patients with anemia of uncertain etiology

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