Dataset Information

The EUTOS long-term survival (ELTS) score is superior to the Sokal score for predicting survival in chronic myeloid leukemia.

ABSTRACT: Prognostic scores support clinicians in selecting risk-adjusted treatments and in comparatively assessing different results. For patients with chronic-phase chronic myeloid leukemia (CML), four baseline prognostic scores are commonly used. Our aim was to compare the prognostic performance of the scores and to arrive at an evidence-based score recommendation. In 2949 patients not involved in any score development, higher hazard ratios and concordance indices in any comparison demonstrated the best discrimination of long-term survival with the ELTS score. In a second step, of 5154 patients analyzed to investigate risk group classification differences, 23% (n?=?1197) were allocated to high-risk by the Sokal score. Of the 1197 Sokal high-risk patients, 56% were non-high-risk according to the ELTS score and had a significantly more favorable long-term survival prognosis than the 526 high-risk patients according to both scores. The Sokal score identified too many patients as high-risk and relatively few (40%) as low-risk (versus 60% with the ELTS score). Inappropriate risk classification jeopardizes optimal treatment selection. The ELTS score outperformed the Sokal score, the Euro, and the EUTOS score regarding risk group discrimination. The recent recommendation of the European LeukemiaNet for preferred use of the ELTS score was supported with significant statistical evidence.

SUBMITTER: Pfirrmann M

PROVIDER: S-EPMC7387299 | biostudies-literature | 2020 Aug

REPOSITORIES: biostudies-literature

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The EUTOS long-term survival (ELTS) score is superior to the Sokal score for predicting survival in chronic myeloid leukemia.

Pfirrmann Markus M Clark Richard E RE Prejzner Witold W Lauseker Michael M Baccarani Michele M Saussele Susanne S Guilhot François F Heibl Sonja S Hehlmann Rüdiger R Faber Edgar E Turkina Anna A Ossenkoppele Gert G Höglund Martin M Zaritskey Andrey A Griskevicius Laimonas L Olsson-Strömberg Ulla U Everaus Hele H Koskenvesa Perttu P Labar Boris B Sacha Tomasz T Zackova Daniela D Cervantes Francisco F Colita Adriana A Zupan Irena I Bogdanovic Andrija A Castagnetti Fausto F Guilhot Joëlle J Hasford Joerg J Hochhaus Andreas A Hoffmann Verena S VS

Leukemia 20200629 8

Prognostic scores support clinicians in selecting risk-adjusted treatments and in comparatively assessing different results. For patients with chronic-phase chronic myeloid leukemia (CML), four baseline prognostic scores are commonly used. Our aim was to compare the prognostic performance of the scores and to arrive at an evidence-based score recommendation. In 2949 patients not involved in any score development, higher hazard ratios and concordance indices in any comparison demonstrated the bes ...[more]

PMID: 32601376

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Project description:BackgroundPediatric CML is very rare. Before the introduction of tyrosine kinase inhibitors (TKIs), allogeneic hematopoietic stem cell transplantation (HSCT) from a donor -if available- was the standard cure attempt. Data on the long-term outcome and health-related quality of life (HRQOL) in former pediatric CML patients undergoing HSCT are lacking.Study questionWe investigated long-term survivors' self-reporting to a questionnaire sent out to patients formerly enrolled in pediatric CML-HSCT trials.MethodsIndividuals with CML transplanted at age <18 years were identified from the German Childhood Cancer Registry database. Long-term survivors received a questionnaire based on the SF-36 and FACT-BMT asking them to self-report HRQOL issues. (Ethical vote #541_20 B, Medical Faculty, University of Erlangen-Nürnberg).Results111/171 (64.9%) individuals survived HSCT long-term and 86/111 (77.5%) fulfilled all inclusion criteria and received the questionnaire. 37/86 (43%) participants (24 female, 13 male, median age at HSCT 12 years [range 2-18], median age at the time of the survey 29 years [range 18-43]) responded after a median follow-up period of 19 years (range 4-27) after HSCT. 10/37 (27%) participants underwent no regular medical follow-up examinations. Self-reported symptoms like chronic graft-versus-host disease (cGvHD)-associated organ impairments and conditioning regimen consequences could causatively not sharply be separated in each case. Complains comprised hypothyroidism (N=11, 30%), infertility (N=9, 24%), lung problems, dry eyes (each N=7, 19%), skin alterations (N=6, 17%), hair problems (N=4, 11%), and sexual dysfunction (N=3, 9%). 10 (27%) participants experienced 13 CML relapses after a median interval from HSCT of 31 months (range 2-93). Only one patient underwent 2nd SCT after failure of relapse treatment with TKIs. Six secondary malignancies (dysplastic melanocytic nevus and ALL, basal cell carcinoma (N=2), rhabdomyosarcoma, and thyroid carcinoma developed in 5 (13%) participants. As assessed by the SF-36 questionnaire, impaired physical health was mainly associated with cGvHD. The mental component summary score showed that also participants without cGvHD scored significantly lower than the general population. When assessed by the FACT-BMT, participants with cGvHD scored significantly lower while participants without cGvHD scored even 5 points higher than the data from controls. 18 (49%) participants considered the sequelae of HSCT an obstacle to education. Out of the total cohort, N=20 (54%), N=7 (19%), N=5 (14%), and N=4 (11%) participants worked full time, part-time, were unemployed, or had not yet finalized their education, respectively. 20 (54%) participants lived as singles, 8 (22%) lived in a partnership, 6 (16%) were married, and 3 (8%) had been divorced. Four (11%) participants reported a total number of 7 children.ConclusionThis first assessment of HRQOL in former pediatric patients with CML surviving HSCT for more than two decades demonstrates self-reported satisfactory well-being only in the absence of cGvHD. Research-based on self-reported outcomes sheds light on former patients' perspectives and provides an additional layer of valuable knowledge for pediatric and adult hematologists. Regular follow-up examinations are mandatory helping to avoid that late secondary neoplasias, CML-relapse, and disorders forming the broad range of possible long-term consequences of HSCT are not detected too late.

Project description:BackgroundAcute myeloid leukemia (AML) is a group of highly heterogeneous diseases, for which approximately 35-40% of patients younger than 60 years old can be cured. However, the multi-omics characteristics and immune cell infiltration (ICI) status of adult long-term survival patients with AML patients compared with healthy controls are still relatively under-explored.MethodsA total of 10 healthy transplant donors (control group) and 11 long-term survival patients with AML with de novo sampling from 2019 to 2020 at the Institute of Hematology in the Hospital of Blood Diseases were enrolled. We simultaneously performed 850 K methylation and bulk RNA-seq on these 21 patients for comparing the differential gene methylation and expression levels between the two groups. The analysis of immune cell gene expression was based on 4 algorithms single sample gene set enrichment analysis (ssGSEA), EPIC, ESTIMATE and immunophenotype score (IPS) on the bulk RNA-seq data.ResultsThe differential methylation positions (DMPs) of the control group was significantly higher than that of the long-term survival group (P<0.01). The hypomethylated probes for Kyoto Encyclopedia of Genes and Genomes (KEGG) enrichment is summarized as follows: the significant pathway was related to NK-cell-mediated cytotoxicity and amino acid metabolism. We also found the Differential expression genes (DEGs) of long-term survival AML were roughly similar, and the DEGs were highly relevant to the cellular amino acid metabolic process pathway by gene set enrichment analysis (GSEA). Based on the further univariate and multivariate Cox survival analyses in GSE37642, genes crosslinked of DEGs and DMPs: LOXL1 and PDZRN4, which characterized as hypomethylated and upregulated, may become an AML prognostic marker (P<0.05). Besides, compared with the long-term-survival AML patients who discontinued chemotherapy after >3 years and the healthy donors, T cell-, natural killer cell-, MHC- and effector cell (EC)-related genes were downregulated; suppressor cells (SC) and checkpoint (CP) cells were significantly upregulated in the long-term-survival AML patients who discontinued chemotherapy after <3 years.ConclusionsIn terms of DNA methylation, RNA expression and ICI, AML patients with long-term survival were slightly different than that of healthy people. The profile of long-term AML survivors, especially those who discontinued chemotherapy less than 3 years, still differed from that of healthy people.

Dataset Information

The EUTOS long-term survival (ELTS) score is superior to the Sokal score for predicting survival in chronic myeloid leukemia.

Publications

The EUTOS long-term survival (ELTS) score is superior to the Sokal score for predicting survival in chronic myeloid leukemia.

Similar Datasets

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