Risk prediction in MDS: independent validation of the IPSS-M-ready for routine?
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ABSTRACT:
SUBMITTER: Baer C
PROVIDER: S-EPMC10079546 | biostudies-literature | 2023 Apr
REPOSITORIES: biostudies-literature
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Risk prediction in MDS: independent validation of the IPSS-M-ready for routine?
Baer Constance C Huber Sandra S Hutter Stephan S Meggendorfer Manja M Nadarajah Niroshan N Walter Wencke W Platzbecker Uwe U Götze Katharina S KS Kern Wolfgang W Haferlach Torsten T Hoermann Gregor G Haferlach Claudia C
Leukemia 20230201 4
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Project description:Higher-risk Myelodysplastic Syndromes/Neoplasms (MDS) represent an ongoing therapeutic challenge, with few effective therapies, many of which may have limited use in this older patient population often with considerations around comorbidities. Outside of transplant, azacitidine and decitabine remain the only disease-modifying therapies, and are palliative in nature. Recent interest has grown in extending combination chemotherapies used to treat acute myeloid leukemia (AML) to patients with MDS, including novel combination chemotherapy "doublets" and "triplets." In this review, we discuss considerations around combination chemotherapy in MDS, specifically as relates to study design, appropriate endpoints, supportive considerations, and how to integrate these into the current treatment paradigm. New therapies in MDS are desperately needed but also require considerations particular to this unique patient population.
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| S-EPMC10539168 | biostudies-literature
Project description:BackgroundThe treatment strategies for Myelodysplastic Syndromes (MDS) are usually based on the risk stratification system. However, few risk signatures which integrate the revised international prognostic scoring system (IPSS-R) with gene mutations can be easily applied in the real world.MethodsThe training cohort of 63 MDS patients was conducted at Zhongda Hospital of Southeast University from January 2013 to April 2020. The validation cohort of 141 MDS patients was obtained from GSE129828. The mutation scoring system was based on the number of mutations and a unique favorable prognostic factor, which is SF3B1 mutation. Univariate Cox, multivariate Cox, and LASSO regression analyses were used to determine the significant factors that influenced the overall survival. The receiver operating characteristic curve (ROC) was used to evaluate the efficiency of the prognostic model.ResultsA novel risk scoring system we named "mutation combined with revised international prognostic scoring system (MIPSS-R)" was developed based on the results derived from multivariate analysis which assigned points to the IPSS-R and the mutation scores according to their relative statistical weight. Based on the quintile of the new scores, patients were divided into five risk levels. The Kaplan-Meier curves showed the superiority of MIPSS-R in separating patients from different groups, comparing with IPSS-R both in the training cohort (p = 1.71e-08 vs. p = 1.363e-04) and validation cohort (p = 1.788e-04 vs. p = 2.757e-03). The area under the ROC of MIPSS-R was 0.79 in the training cohort and 0.62 in the validation cohort. The retrospective analysis of our house patients showed that the risk levels of 57.41% of patients would adjust according to MIPSS-R. After changing risk levels, 38.71% of patients would benefit from treatment strategies that MIPSS-R recommends.ConclusionA mutation scoring system was conducted based on the number of mutations and a unique favorable prognostic factor. MIPSS-R, the novel integral risk stratification system was developed by integrating IPSS-R and the mutation scores, which is more effective on prognosis and treatment guidance for MDS patients.
| S-EPMC7866647 | biostudies-literature
Project description:BackgroundThe Movement Disorder Society-sponsored Non-motor Rating Scale (MDS-NMS) assess the severity and disability caused by non-motor symptoms (NMS) in Parkinson's disease (PD).ObjectiveThis article encapsulates the formal process for completing this program and the data on the first officially approved non-English version of the MDS-NMS (Spanish).MethodsThe MDS-NMS translation program involves four steps: translation and back-translation; cognitive pre-testing to ensure that raters and patients understand the scale and are comfortable with its content; field testing of the finalized version; analysis of the factor structure of the tested version against the original English language version for the nine domains that could be analyzed in a confirmatory factor analysis. To be designated an "Official MDS translation," the confirmatory factor analysis Comparative Fit Index had to be ≥0.90.ResultsThe Spanish MDS-NMS was tested in 364 native-Spanish-speaking patients with PD from seven countries. For all subjects with fully computable data with all domains of the MDS-NMS (n = 349), the Comparative Fit Index was ≥0.90 for the nine eligible domains. Missing data were negligible and moderate floor effect (42.90%) was found for the Non-Motor Fluctuations subscale. Item homogeneity coefficient was adequate, and the correlation of the MDS-NMS domains with other measures for related constructs was acceptable (r s ≥ 0.50).ConclusionsThe Spanish version of the MDS-NMS followed the IPMDS Translation Program protocol, reached the criterion to be designated as an Official Translation, and is now available on the MDS website.
| S-EPMC10105117 | biostudies-literature
Project description:ObjectivesTo evaluate the predictive validity of an adapted version of the Minimum Data Set (MDS) Mortality Risk Index-Revised (MMRI-R) based on MDS version 3.0 assessment items (MMRI-v3) and to compare the predictive validity of the MMRI-v3 with that of a single MDS item indicating limited life expectancy (LLE).DesignRetrospective, cross-sectional study of MDS assessments. Other data sources included the Veterans Affairs (VA) Residential History File and Vital Status File.SettingVA nursing homes (NHs).ParticipantsVeterans aged 65 and older newly admitted to VA NHs between July 1, 2012, and September 30, 2015.MeasurementsThe dependent variable was death within 6 months of admission date. Independent variables included MDS items used to calculate MMRI-v3 scores (renal failure, chronic heart failure, sex, age, dehydration, cancer, unintentional weight loss, shortness of breath, activity of daily living scale, poor appetite, acute change in mental status) and the MDS item indicating LLE.ResultsThe predictive ability of the MMRI-v3 for 6-month mortality (c-statistic 0.81) is as good as that of the original MMRI-R (c-statistic 0.76). Scores generated using the MMRI-v3 had greater predictive ability than that of the single MDS indicator for LLE (c-statistic 0.76); using the 2 together resulted in greater predictive ability (c-statistic 0.86).ConclusionThe MMRI-v3 is a useful tool in research and clinical practice that accurately predicts 6-month mortality in veterans residing in Veterans Affairs NHs. Identification of residents with LLE has great utility for studying palliative care interventions and may be helpful in guiding allocation of these services in clinical practice. J Am Geriatr Soc 66:2353-2359, 2018.
| S-EPMC7477706 | biostudies-literature
Project description:Utilizing assays that assess specific T-cell-mediated immunity against cytomegalovirus (CMV) holds the potential to enhance personalized strategies aimed at preventing and treating CMV in organ transplantation. This includes improved risk stratification during transplantation compared to relying solely on CMV serostatus, as well as determining the optimal duration of antiviral prophylaxis, deciding on antiviral therapy when asymptomatic replication occurs, and estimating the risk of recurrence. In this review, we initially provide an overlook of the current concepts into the immune control of CMV after transplantation. We then summarize the existent literature on the clinical experience of the use of immune monitoring in organ transplantation, with a particular interest on the outcomes of interventional trials. Current evidence indicates that cell-mediated immune assays are helpful in identifying patients at low risk for replication for whom preventive measures against CMV can be safely withheld. As more data accumulates from these and other clinical scenarios, it is foreseeable that these assays will likely become part of the routine clinical practice in organ transplantation.
| S-EPMC10661902 | biostudies-literature
Project description:BackgroundClinical prediction rules (CPRs) should be externally validated by independent researchers. Although there are many cardiovascular CPRs, most have not been externally validated. It is not known why some CPRs are externally validated by independent researchers and others are not.MethodsWe analyzed cardiovascular risk CPRs included in a systematic review. Independent external validations were identified by forward citation searches of derivation studies. Time between the publication of a cardiovascular CPR and the first independent external validation was calculated. We assessed Kaplan-Meier estimates of the probability to have an independent external validation. Using univariable Cox regression, we explored whether characteristics of derivation (design, location, sample size, number of predictors, presentation format, validation in derivation), reporting (participants, predictors, outcomes, performance measure, information for risk calculation), and publication (journal impact factor) are associated with time to the first independent external validation.ResultsOf 125 cardiovascular risk CPRs, 29 had an independent external validation. The median follow-up was 118 months (95% CI, 99-130). The 25th percentile of event time was 122 months (95% CI, 91-299). Cardiovascular risk CPRs from the USA were 4.15 times (95% CI, 1.89-9.13) more likely to have an independent external validation. Increasing the sample size of derivation by ten times was associated with a 2.32-fold (95% CI, 1.37-3.91) increase in the probability of having an independent external validation. CPRs presented with an internal validation tend to get an independent external validation sooner (HR = 1.73, 95% CI, 0.77-3.93). CPRs reporting all the information necessary for calculating individual risk were 2.65 (95% CI, 1.01-6.96) times more likely to have an independent external validation. Publishing a cardiovascular risk CPR in a journal that has one unit higher impact factor was associated with a 6% (95% CI, 3-9) higher likelihood of an independent external validation.ConclusionsThe probability for cardiovascular risk CPRs to get an independent external validation was low even many years after their derivations. Authors of new cardiovascular risk CPRs should consider using adequate sample size, conducting an internal validation, and reporting all the information needed for risk calculation as these features were associated with an independent external validation.
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Project description:Serum ferritin (SF) has been identified as a potential prognostic factor for patients undergoing stem cell transplantation, but the prognostic value of SF in acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) patients and the impact of iron chelation therapy (ICT) on MDS patients are controversial. The present meta-analysis aimed to better elucidate these relationships.Three electronic databases were searched systematically to identify reports on the prognostic role of SF in MDS and AML patients, and those investigating the impact of ICT on prognosis of MDS patients. The hazard ratios (HRs) and its 95% confidence interval (95%CI) were extracted from the identified studies using Cox proportional hazard regression model for overall survival (OS) and progression of MDS to AML.Twenty reports including 1066 AML patients and 4054 MDS patients were included in present study. The overall pooled HRs for OS of AML and MDS patients with elevated SF prior to transplantation was 1.73 (1.40-2.14), subgroup analyses stratified by the cut-off value of SF ≥1400/1000 ng/mL showed that the pooled HRs were 1.45 (0.98-2.15) and 1.65 (1.30-2.10), respectively. The pooled HRs for ICT in MDS patients was 0.30 (0.23-0.40). For ICT, the pooled HRs for the progression of MDS to AML was 0.84 (0.61-1.61).SF has a negative impact on the OS of AML and MDS patients when it is higher than 1000 ng/mL. ICT can improve the OS of MDS patients with iron overload but it is not associated with the progression of MDS to AML.
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Project description:The International Prognostic Scoring System-Revised (IPSS-R) is one standard for myelodysplastic syndrome (MDS) risk stratification. It divides patients into five categories including an intermediate subset (IPSS-R int-risk). Outcomes and clinical interventions for patients with IPSS-R int-risk are not well defined. We performed an analysis of outcomes of this group of patients. Out of 3167 patients, a total of 298 were identified with IPSS-R int-risk MDS and retrospectively analyzed to assess characteristics affecting outcomes. Cox proportional hazard models for overall survival (OS) were performed to identify statistically significant clinical factors that influence survival. Age of 66 years or greater, peripheral blood blasts of 2% or more, and history of red blood cell (RBC) transfusion were significantly associated with inferior survival. Based on these features, MDS patients with IPSS-R int-risk were classified into two prognostic risk groups for analysis, an int-favorable group and an int-adverse group, and had significantly divergent outcomes. Sequential prognostication was validated using two independent datasets comprising over 700 IPSS-R int-risk patients. The difference in median survival between int-favorable and int-adverse patients was 3.7 years in the test cohort, and 1.8 and 2.0 years in the two validation cohorts. These results confirm significantly variable outcomes of patients with IPSS-R int-risk and need for different prognostic systems.
| S-EPMC6750209 | biostudies-literature