International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study

Sperr, Wolfgang R.; Kundi, Michael; Álvarez‐Twose, Iván; Anrooij, Björn van; Elberink, J. N. G. Oude; Górska, Aleksandra; Niedoszytko, Marek; Gleixner, Karoline V.; Hadzijusufovic, Emir; Zanotti, Roberta; Bonadonna, Patrizia; Bonifacio, Massimiliano; Perkins, Cecelia; Illerhaus, Anja; Elena, Chiara; Merante, Serena; Shoumariyeh, Khalid; Bubnoff, Nikolas von; Parente, Roberta; Jawhar, Mohamad; Fortina, Anna Belloni; Caroppo, Francesca; Brockow, Knut; Zink, Alexander; Fuchs, David; Kilbertus, Alex; Yavuz, Akif Selim; Doubek, Michael; Hägglund, Hans; Panse, Jens; Sabato, Vito; Bretterklieber, Agnes; Niederwieser, Dietger; Breynaert, Christine; Hartmann, Karin; Triggiani, Massimo; Nedoszytko, Bogusław; Reiter, Andreas; Órfão, Alberto; Hermine, Olivier; Gotlib, Jason; Arock, Michel; Kluin‐Nelemans, Hanneke C.; Valent, Peter

doi:10.1016/s2352-3026(19)30166-8

Cited by 107 publications

(164 citation statements)

References 31 publications

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“…We utilized R (version 3.6.1) to generate a matrix that included the clinical information and DEG expression. We used Cox regression analysis to build the prognostic model using the R package “survival” ( 25 , 26 ) and online tool “SangerBox”. Then, we utilized the R package “randomForest” to validate the prognostic model through risk score and calculate the accuracy, rrror rate, sensitivity and precision from a confusion matrix.…”

Section: Methodsmentioning

confidence: 99%

Differential Expression Analysis Revealing CLCA1 to Be a Prognostic and Diagnostic Biomarker for Colorectal Cancer

et al. 2020

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Colorectal cancer (CRC) is a common malignant tumor of the digestive tract and lacks specific diagnostic markers. In this study, we utilized 10 public datasets from the NCBI Gene Expression Omnibus (NCBI-GEO) database to identify a set of significantly differentially expressed genes (DEGs) between tumor and control samples and WGCNA (Weighted Gene Co-Expression Network Analysis) to construct gene co-expression networks incorporating the DEGs from The Cancer Genome Atlas (TCGA) and then identify genes shared between the GEO datasets and key modules. Then, these genes were screened via MCC to identify 20 hub genes. We utilized regression analyses to develop a prognostic model and utilized the random forest method to validate. All hub genes had good diagnostic value for CRC, but only CLCA1 was related to prognosis. Thus, we explored the potential biological value of CLCA1. The results of gene set enrichment analysis (GSEA) and immune infiltration analysis showed that CLCA1 was closely related to tumor metabolism and immune invasion of CRC. These analysis results revealed that CLCA1 may be a candidate diagnostic and prognostic biomarker for CRC.

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Section: Methodsmentioning

confidence: 99%

Differential Expression Analysis Revealing CLCA1 to Be a Prognostic and Diagnostic Biomarker for Colorectal Cancer

et al. 2020

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“…The validity of these scores is generally demonstrated on large patient cohorts and also confirmed in external series [4,26], and they have the undeniable merit to provide a reference framework for SM patient management. However, some limitations have to be acknowledged: data are obtained by retrospective studies, and wide recruitment periods are needed to collect sufficient patients, with all consequent variabilities in terms of diagnostic methods, treatment opportunities, and geographical-related differences in approaches.…”

Section: Integrated Prognostic Scoresmentioning

confidence: 82%

“…The basis for treatment of systemic mastocytosis is the distinction between indolent/smoldering from advanced variants according to WHO classification. In routine practice, the clinician often deals with some challenging subsets, where the choice of the most appropriate therapeutic modality should be evaluated carefully in individual cases Although prognostically meaningful [2][3][4], WHO classification is limited by some relevant flaws. In clinical practice, the distinction between some categories is not always clearcut and the appraisal of some key parameters may be affected by variable interpretation.…”

Section: Who Classificationmentioning

confidence: 99%

“…To this end, several models incorporating clinical and molecular variables have been devised: the Mayo alliance prognostic system (MAPS) [24], the International prognostic scoring system (IPSM) [4], the Mutation-Adjusted Risk Score (MARS) [25] among the most relevant ones. Some models include clinical information only and are particularly useful in routine practice [4,24]. Other scores are hybrid, in that add biological, mutational data to clinical ones [24,25].…”

Section: Integrated Prognostic Scoresmentioning

confidence: 99%

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Catching the clinical and biological diversity for an appropriate therapeutic approach in systemic mastocytosis

Mannelli

2020

Ann Hematol

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Systemic mastocytosis (SM) is a rare disease calling for integrated approaches involving onco-hematologic competences for appropriate clinical management and treatment. The wide variability of manifestations and disease course claims for an accurate risk stratification, currently relying on the appraisal of the benefit/risk ratio of treatment modalities within indolent and advanced variants according to WHO classification. More objective parameters are progressively incorporated and integrated into comprehensive models, on which to support the adoption of therapeutic strategies, since the mere clinical distinction between mediator-related signs/symptoms and "true" organ damage can sometimes be complicated. The development of novel targeted drugs is progressively extending the therapeutic alternatives available, which ranges from conventional agents such as interferon and cladribine, to the more modern approach based on KIT inhibition. Ultimately, the choice of the most appropriate therapy should be rationalized on the basis of the clinical picture and molecular data. The focus of the present review is on the areas still open in the current evaluation of SM patients, particularly when considering the need of a treatment.

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“…patients usually have a high disease burden, multiple-organ damage and poor prognosis with median overall survival of approximately 3-4 years (Jawhar et al 2016(Jawhar et al , 2017a(Jawhar et al , b, 2019Valent et al 2017;Sperr et al 2019).…”

mentioning

confidence: 99%

An increased bone mineral density is an adverse prognostic factor in patients with systemic mastocytosis

Riffel

Schwaab

Lutz

et al. 2020

J Cancer Res Clin Oncol

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Purpose Systemic mastocytosis (SM) is characterized by the expansion of clonal mast cells that infiltrate various organ systems. The extent of organ infiltration and subsequent organ damage distinguishes between indolent SM (ISM) defined by a nearly normal life expectancy and advanced SM (AdvSM) defined by poor prognosis. In ISM, measurement of the bone mineral density (BMD) frequently reveals osteoporosis. In contrast, the clinical implication of an increased BMD and osteosclerosis remains unclear. Methods BMD was evaluated in 61 patients with mastocytosis (ISM, n = 29, 48%; AdvSM, n = 32, 52%). We correlated the prevalence of osteoporosis, increased BMD and osteosclerosis with clinical parameters, disease variant and prognosis. Results Osteoporosis was detected in 11/29 (38%) patients with ISM but only in 2/32 (6%) patients with AdvSM (p = 0.004). An increased BMD was detected in 1/29 (3%) patients with ISM and 24/32 (75%) patients with AdvSM (p < 0.001) while osteosclerosis was only detected in AdvSM patients (16/32, 50%). AdvSM patients with increased BMD had higher levels of bone marrow mast cell infiltration, higher serum tryptase and alkaline phosphatase levels compared to ISM as well as higher number of high-molecular risk mutations (p < 0.05). In addition, we found that the prognosis of AdvSM patients with increased BMD is inferior compared to those without increased BMD (median overall survival 3.6 years versus not reached, p = 0.031). Conclusions Osteoporosis is a common feature in ISM but not in AdvSM. An increased BMD is frequently present in AdvSM but not in ISM and is associated with more advanced disease and inferior outcome.

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International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study

Cited by 107 publications

References 31 publications

Differential Expression Analysis Revealing CLCA1 to Be a Prognostic and Diagnostic Biomarker for Colorectal Cancer

Differential Expression Analysis Revealing CLCA1 to Be a Prognostic and Diagnostic Biomarker for Colorectal Cancer

Catching the clinical and biological diversity for an appropriate therapeutic approach in systemic mastocytosis

An increased bone mineral density is an adverse prognostic factor in patients with systemic mastocytosis

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