International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study.,The Lancet Haematology

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International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study.
The Lancet Haematology ( IF 15.4 ) Pub Date : 2019-10-31 , DOI: 10.1016/s2352-3026(19)30166-8
Wolfgang R Sperr ₁ , Michael Kundi ₂ , Ivan Alvarez-Twose ₃ , Bjorn van Anrooij ₄ , Joanna N G Oude Elberink ₅ , Aleksandra Gorska ₆ , Marek Niedoszytko ₆ , Karoline V Gleixner ₁ , Emir Hadzijusufovic ₇ , Roberta Zanotti ₈ , Patrizia Bonadonna ₉ , Massimiliano Bonifacio ₈ , Cecelia Perkins ₁₀ , Anja Illerhaus ₁₁ , Chiara Elena ₁₂ , Serena Merante ₁₂ , Khalid Shoumariyeh ₁₃ , Nikolas von Bubnoff ₁₄ , Roberta Parente ₁₅ , Mohamad Jawhar ₁₆ , Anna Belloni Fortina ₁₇ , Francesca Caroppo ₁₇ , Knut Brockow ₁₈ , Alexander Zink ₁₈ , David Fuchs ₁₉ , Alex J Kilbertus ₂₀ , Akif Selim Yavuz ₂₁ , Michael Doubek ₂₂ , Hans Hägglund ₂₃ , Jens Panse ₂₄ , Vito Sabato ₂₅ , Agnes Bretterklieber ₂₆ , Dietger Niederwieser ₂₇ , Christine Breynaert ₂₈ , Karin Hartmann ₂₉ , Massimo Triggiani ₁₅ , Boguslaw Nedoszytko ₃₀ , Andreas Reiter ₁₆ , Alberto Orfao ₃₁ , Olivier Hermine ₃₂ , Jason Gotlib ₁₀ , Michel Arock ₃₃ , Hanneke C Kluin-Nelemans ₃₄ , Peter Valent ₁

Affiliation

Department of Internal Medicine I, Division of Hematology and Hemostaseology, and Ludwig Boltzmann Institute for Hematology and Oncology, Medical University of Vienna, Vienna, Austria.
Institute of Environmental Health, Medical University of Vienna, Vienna, Austria.
Instituto de Estudios de Mastocitosis de Castilla La Mancha (CLMast), Hospital Virgen del Valle, Toledo, Spain.
Department of Hematology, University Medical Center Groningen, University of Groningen, Groningen, Netherlands; Department of Allergology, University Medical Center Groningen, University of Groningen, Groningen, Netherlands.
Department of Allergology, University Medical Center Groningen, University of Groningen, Groningen, Netherlands.
Department of Allergology, Medical University of Gdańsk, Gdańsk, Poland.
Department of Internal Medicine I, Division of Hematology and Hemostaseology, and Ludwig Boltzmann Institute for Hematology and Oncology, Medical University of Vienna, Vienna, Austria; Internal Medicine Small Animals, University Clinic for Small Animals, Department/University Clinic for Companion Animals and Horses, University of Veterinary Medicine, Vienna, Austria.
Section of Hematology, Department of Medicine, Verona University Hospital, Verona, Italy.
Allergy Unit, Verona University Hospital, Verona, Italy.
Division of Hematology, Department of Medicine, Stanford University School of Medicine, Stanford, CA, USA.
Department of Dermatology, University of Cologne, Cologne, Germany.
Department of Molecular Medicine and Department of Hematology Oncology, University of Pavia and Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
Department of Hematology, Oncology and Stem Cell Transplantation, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
Department of Hematology, Oncology and Stem Cell Transplantation, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany; Department of Hematology and Oncology, Medical Center, University of Schleswig-Holstein, Campus Lübeck, Lübeck, Germany.
Division of Allergy and Clinical Immunology, University of Salerno, Salerno, Italy.
III Medizinische Klinik, Universitätsmedizin Mannheim, Universität Heidelberg, Mannheim, Germany.
Pediatric Dermatology Unit, Department of Medicine, University of Padua, Padua, Italy.
Department of Dermatology and Allergy, School of Medicine, Technical University of Munich, Munich, Germany.
University Clinic for Hematology and Internal Oncology, Kepler University Hospital, Johannes Kepler University, Linz, Austria.
Department of Dermatology and Venerology, Kepler University Hospital, Johannes Kepler University, Linz, Austria.
Division of Hematology, Istanbul Medical School, University of Istanbul, Istanbul, Turkey.
University Hospital and CEITEC Masaryk University, Brno, Czech Republic.
Division of Hematology, Department of Medical Sciences Uppsala University, Uppsala, Sweden.
Department of Oncology, Haematology, Haemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen, Germany.
Faculty of Medicine and Health Sciences, Department of Immunology-Allergology-Rheumatology, University of Antwerp and Antwerp University Hospital, Antwerp, Belgium.
Department of Dermatology and Venereology, University Hospital Graz, Graz, Austria.
University Hospital of Leipzig, Leipzig, Germany.
KU Leuven Department of Microbiology, Immunology and Transplantation, Allergy and Clinical Immunology Research Group and MASTeL, University Hospitals Leuven, Leuven, Belgium.
Department of Dermatology, University of Cologne, Cologne, Germany; Division of Allergy, Department of Dermatology, and Department of Biomedicine, University of Basel, Basel, Switzerland.
Department of Dermatology, Medical University of Gdańsk, Gdańsk, Poland.
Centro de Investigación del Cáncer/IBMCC (USAL/CSIC), CIBERONC and IBSAL, Departamento de Medicina and Servicio General de Citometría, University of Salamanca, Salamanca, Spain.
Imagine Institute Université Paris Descartes, Sorbonne, Paris Cité, Centre national de référence des mastocytoses, Paris, France.
Department of Hematological Biologie, Pitié-Salpêtrière Hospital, Paris Sorbonne University, Paris UMR8113, Ecole, France.
Department of Hematology, University Medical Center Groningen, University of Groningen, Groningen, Netherlands.

Background

The WHO classification separates mastocytosis into distinct variants, but prognostication remains a clinical challenge. The aim of this study was to improve prognostication for patients with mastocytosis.

Methods

We analysed data of the registry of the European Competence Network on Mastocytosis including 1639 patients (age 17–90 years) diagnosed with mastocytosis according to WHO criteria between Jan 12, 1978, and March 16, 2017. Univariate and multivariate analyses with Cox regression were applied to identify prognostic variables predicting survival outcomes and to establish a prognostic score. We validated this International Prognostic Scoring System in Mastocytosis (IPSM) with data of 462 patients (age 17–79 years) from the Spanish network Red Española de Mastocitosis diagnosed between Jan 22, 1998, and Nov 2, 2017.

Findings

The prognostic value of the WHO classification was confirmed in our study (p<0·0001). For patients with non-advanced mastocytosis (n=1380), we identified age 60 years or older (HR 10·75, 95% CI 5·68–20·32) and a concentration of alkaline phosphatase 100 U/L or higher (2·91, 1·60–5·30) as additional independent prognostic variables for overall survival. The resulting scoring system divided patients with non-advanced mastocytosis into three groups: low (no risk factors), intermediate 1 (one risk factor), and intermediate 2 (two risk factors). Overall survival and progression-free survival differed significantly among these groups (p<0·0001). In patients with advanced mastocytosis (n=259), age 60 years or older (HR 2·14, 95% CI 1·42–3·22), a concentration of tryptase 125 ng/mL or higher (1·81, 1·20–2·75), a leukocyte count of 16 × 10⁹ per L or higher (1·88, 1·27–2·79), haemoglobin of 11 g/dL or lower (1·71, 1·13–2·57), a platelet count of 100 × 10⁹ per L or lower (1·63, 1·13–2·34), and skin involvement (0·46, 0·30–0·69) were prognostic variables. Based on these variables, a separate score for advanced mastocytosis with four risk categories was established, with significantly different outcomes for overall survival and progression-free survival (p<0·0001). The prognostic value of both scores was confirmed in 413 patients with non-advanced disease and 49 with advanced mastocytosis from the validation cohort.

Interpretation

The IPSM scores for patients with non-advanced and advanced mastocytosis can be used to predict survival outcomes and guide treatment decisions. However, the predictive value of the IPSM needs to be confirmed in forthcoming trials.

Funding

Austrian Science Fund, Deutsche Forschungsgemeinschaft, Koeln Fortune Program, Charles and Ann Johnson Foundation, Instituto de Salud Carlos III, Fondos FEDER, Research-Foundation Flanders/Fonds Wetenschappelijk Onderzoek, Clinical Research-Fund of the University Hospitals Leuven, and Research-Foundation Flanders/Fonds Wetenschappelijk Onderzoek.

中文翻译：

肥大细胞增多症的国际预后评分系统（IPSM）：一项回顾性队列研究。

背景

WHO分类将肥大细胞增多症分为不同的变体，但预后仍然是临床挑战。这项研究的目的是改善肥大细胞增多症患者的预后。

方法

我们根据1978年1月12日至2017年3月16日之间的WHO标准，分析了欧洲抗脂肪细胞病能力网络注册中心的数据，包括1639名（17-90岁）诊断为脂肪细胞增多症的患者。采用Cox回归的单因素和多因素分析为用于识别预测生存结果的预后变量并建立预后评分。我们通过西班牙网络RedEspañolade Mastocitosis在1998年1月22日至2017年11月2日之间诊断出的462例患者（年龄17-79岁）的数据，验证了该国际上的肥大细胞增多症预后评分系统（IPSM）。

发现

我们的研究证实了WHO分类的预后价值（p <0·0001）。对于非晚期肥大细胞增多症（n = 1380）的患者，我们确定年龄在60岁以上（HR 10·75，95％CI 5·68-20·32），且碱性磷酸酶的浓度为100 U / L或更高（ 2·91、1·60-5·30）作为整体生存率的附加独立预后变量。最终的评分系统将非晚期肥大细胞增多症患者分为三组：低（无危险因素），中级1（一个危险因素）和中级2（两个危险因素）。这些组的总生存期和无进展生存期差异显着（p <0·0001）。在年龄大于或等于60岁（HR 2·14，95％CI 1·42-3·22）的晚期肥大细胞增多症患者（n = 259）中，胰蛋白酶浓度为125 ng / mL或更高（1·81、1 ·20–2·75），白细胞计数为16×10⁹每升或更高（1·88，1·27-2·79）11克，血红蛋白/ dL或更低（1·71，1·13-2·57），一个100×10血小板计数⁹每L或更低（1·63、1·13-2·34）和皮肤受累（0·46、0·30-0·69）是预后变量。基于这些变量，建立了具有四个风险类别的晚期肥大细胞增多症的单独评分，其总生存期和无进展生存期的结果显着不同（p <0·0001）。在验证队列中，这两项评分的预后价值在413例非晚期疾病患者和49例晚期肥大细胞增多症患者中得到了证实。

解释

非晚期和晚期肥大细胞增多症患者的IPSM评分可用于预测生存结果并指导治疗决策。但是，IPSM的预测价值需要在即将进行的试验中得到证实。

资金

奥地利科学基金，Deutsche Forschungsgemeinschaft，Koeln财富计划，Charles and Ann Johnson基金会，Salud Carlos III研究所，Fondos FEDER，研究基金会Flanders / Fonds Wetenschappelijk Onderzoek，鲁汶大学医院临床研究基金和研究基金会Flanders / Fonds Wetenschappelijk Onderzoek。

更新日期：2019-11-01

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