MIPSS70: Mutation-Enhanced International Prognostic Score System for Transplantation-Age Patients With Primary Myelofibrosis

被引:404
作者
Guglielmelli, Paola [1 ]
Lasho, Terra L. [7 ]
Rotunno, Giada [1 ]
Mudireddy, Mythri [7 ]
Mannarelli, Carmela [1 ]
Nicolosi, Maura [7 ]
Pacilli, Annalisa [1 ]
Pardanani, Animesh [7 ]
Rumi, Elisa [2 ,3 ]
Rosti, Vittorio [2 ]
Hanson, Curtis A. [7 ]
Mannelli, Francesco [1 ]
Ketterling, Rhett P. [7 ]
Gangat, Naseema [7 ]
Rambaldi, Alessandro [4 ,5 ]
Passamonti, Francesco [6 ]
Barosi, Giovanni [2 ]
Barbui, Tiziano [5 ]
Cazzola, Mario [2 ,3 ]
Vannucchi, Alessandro M. [1 ]
Tefferi, Ayalew [7 ]
机构
[1] Univ Florence, Azienda Osped Univ Careggi, Florence, Italy
[2] Fdn Ist Ricovero & Cura Carattere Sci Policlin Sa, Pavia, Italy
[3] Univ Pavia, Pavia, Italy
[4] Univ Milan, Milan, Italy
[5] Azienda Socio Sanit Terr Papa Giovanni XXIII, Bergamo, Italy
[6] Univ Insubria, Varese, Italy
[7] Mayo Clin, Rochester, MN USA
关键词
CYTOGENETIC-RISK CATEGORIZATION; STEM-CELL TRANSPLANTATION; BONE-MARROW; CALRETICULIN MUTATIONS; WORKING GROUP; SURVIVAL; IMPACT; CALR; NEOPLASMS; DIAGNOSIS;
D O I
10.1200/JCO.2017.76.4886
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
Purpose To develop a prognostic system for transplantation-age patients with primary myelofibrosis (PMF) that integrates clinical, cytogenetic, and mutation data. Patients and Methods The study included 805 patients with PMF age <= 70 years recruited from multiple Italian centers and the Mayo Clinic (Rochester, MN), forming two independent learning and validation cohorts. A Cox multivariable model was used to select from among a list of 22 variables those that were predictive of overall survival (OS). Integrated clinical and genetic prognostic models with (MIPSS70-plus) or without (MIPSS70) cytogenetic information were developed. Results Multivariable analysis identified the following as significant risk factors for OS: hemoglobin, < 100 g/L, leukocytes > 25 * 10(9)/L, platelets, < 100 * 10(9)/L, circulating blasts >= 2%, bone marrow fibrosis grade > 2, constitutional symptoms, absence of CALR type-1 mutation, presence of high-molecular risk mutation (ie, ASXL1, EZH2, SRSF2, IDH1/2), and presence of two or more high-molecular risk mutations. By assigning hazard ratio (HR)-weighted points to these variables, three risk categories were delineated for the MIPSS70 model; 5-year OS was 95% in low-risk, 70% in intermediate-risk, and 29% in high-risk categories, corresponding to median OS of 27.7 years (95% CI, 22 to 34 years), 7.1 years (95% CI, 6.2 to 8.1 years), and 2.3 years (95% CI, 1.9 to 2.7 years), respectively. In the MIPSS70-plus model, which included cytogenetic information, four risk categories were delineated, with 5-year OS of 91% in low-risk, 66% in intermediate-risk (HR, 3.2; 95% CI, 1.9 to 5.2), 42% in high-risk (HR, 6.4; 95% CI, 4.1 to 10.0), and 7% very high-risk categories (HR, 17.0; 95% CI, 9.8 to 29.2). Both models remained effective after inclusion of older patients in the analysis. Conclusion MIPSS70 and MIPSS70-plus provide complementary systems of risk stratification for transplantation-age patients with PMF and integrate prognostically relevant clinical, cytogenetic, and mutation data. (C) 2017 by American Society of Clinical Oncology
引用
收藏
页码:310 / +
页数:15
相关论文
共 31 条
[1]  
AKAIKE H, 1992, No To Hattatsu, V24, P127
[2]  
[Anonymous], BR J HAEMATOL
[3]   The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia [J].
Arber, Daniel A. ;
Orazi, Attilio ;
Hasserjian, Robert ;
Thiele, Jurgen ;
Borowitz, Michael J. ;
Le Beau, Michelle M. ;
Bloomfield, Clara D. ;
Cazzola, Mario ;
Vardiman, James W. .
BLOOD, 2016, 127 (20) :2391-2405
[4]   Philadelphia-Negative Classical Myeloproliferative Neoplasms: Critical Concepts and Management Recommendations From European LeukemiaNet [J].
Barbui, Tiziano ;
Barosi, Giovanni ;
Birgegard, Gunnar ;
Cervantes, Francisco ;
Finazzi, Guido ;
Griesshammer, Martin ;
Harrison, Claire ;
Hasselbalch, Hans Carl ;
Hehlmann, Rudiger ;
Hoffman, Ronald ;
Kiladjian, Jean-Jacques ;
Kroeger, Nicolaus ;
Mesa, Ruben ;
McMullin, Mary F. ;
Pardanani, Animesh ;
Passamonti, Francesco ;
Vannucchi, Alessandro M. ;
Reiter, Andreas ;
Silver, Richard T. ;
Verstovsek, Srdan ;
Tefferi, Ayalew .
JOURNAL OF CLINICAL ONCOLOGY, 2011, 29 (06) :761-770
[5]   Refined cytogenetic-risk categorization for overall and leukemia-free survival in primary myelofibrosis: a single center study of 433 patients [J].
Caramazza, D. ;
Begna, K. H. ;
Gangat, N. ;
Vaidya, R. ;
Siragusa, S. ;
Van Dyke, D. L. ;
Hanson, C. ;
Pardanani, A. ;
Tefferi, A. .
LEUKEMIA, 2011, 25 (01) :82-88
[6]   Does ruxolitinib prolong the survival of patients with myelofibrosis? [J].
Cervantes, Francisco ;
Pereira, Arturo .
BLOOD, 2017, 129 (07) :832-837
[7]   Improving Survival Trends in Primary Myelofibrosis: An International Study [J].
Cervantes, Francisco ;
Dupriez, Brigitte ;
Passamonti, Francesco ;
Vannucchi, Alessandro M. ;
Morra, Enrica ;
Reilly, John T. ;
Demory, Jean-Loup ;
Rumi, Elisa ;
Guglielmelli, Paola ;
Roncoroni, Elisa ;
Tefferi, Ayalew ;
Pereira, Arturo .
JOURNAL OF CLINICAL ONCOLOGY, 2012, 30 (24) :2981-2987
[8]   New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment [J].
Cervantes, Francisco ;
Dupriez, Brigitte ;
Pereira, Arturo ;
Passamonti, Francesco ;
Reilly, John T. ;
Morra, Enrica ;
Vannucchi, Alessandro M. ;
Mesa, Ruben A. ;
Demory, Jean-Loup ;
Barosi, Giovanni ;
Rumi, Elisa ;
Tefferi, Ayalew .
BLOOD, 2009, 113 (13) :2895-2901
[9]   THE EFFICACY OF DIRECT, 24-HOUR CULTURE, AND MITOTIC SYNCHRONIZATION METHODS FOR CYTOGENETIC ANALYSIS OF BONE-MARROW IN NEOPLASTIC HEMATOLOGIC DISORDERS [J].
DEWALD, GW ;
BRODERICK, DJ ;
TOM, WW ;
HAGSTROM, JE ;
PIERRE, RV .
CANCER GENETICS AND CYTOGENETICS, 1985, 18 (01) :1-10
[10]   Allogeneic Hematopoietic Stem-Cell Transplantation for Myelofibrosis: A Practical Review [J].
Farhadfar, Nosha ;
Cerquozzi, Sonia ;
Patnaik, Mrinal ;
Tefferi, Ayalew .
JOURNAL OF ONCOLOGY PRACTICE, 2016, 12 (07) :611-+