Myelofibrosis Treatment Algorithm 2018

Blood Cancer Journal - Tập 8 Số 8
Ayalew Tefferi1, Paola Guglielmelli2, Animesh Pardanani1, Alessandro M. Vannucchi2
1Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN, USA
2Department of Experimental and Clinical Medicine, CRIMM, Center Research and Innovation of Myeloproliferative Neoplasms, Azienda Ospedaliera Universitaria Careggi, University of Florence, Florence, Italy

Tóm tắt

AbstractTwo novel prognostic systems for primary myelofibrosis (PMF) were recently unveiled: GIPSS (genetically inspired prognostic scoring system) and MIPSS70 (mutation-enhanced international prognostic scoring system for transplant-age patients). GIPSS is based exclusively on genetic markers: mutations and karyotype. MIPSS70 includes mutations and clinical risk factors. In its most recent adaptation, the prognostic value of MIPSS70 has been bolstered by the inclusion of a three-tiered cytogenetic risk stratification and use of hemoglobin thresholds that are adjusted for sex and severity (MIPSS70+ version 2.0). GIPSS features four, MIPSS70 three, and MIPSS70+ version 2.0 five risk categories. MIPSS70 is most useful in the absence of cytogenetic information. MIPSS70+ version 2.0 is more comprehensive than MIPSS70 and is the preferred model in the presence of cytogenetic information. Both MIPSS70 and MIPSS70+ version 2.0 require an online score calculator (http://www.mipss70score.it). GIPPS offers a lower complexity prognostic tool that reliably identifies candidates for allogeneic stem cell transplant (GIPSS high-risk disease) or long-term observation with little or no therapeutic intervention (GIPSS low-risk disease). Ultimately, we favor a step-wise prognostication approach that starts with GIPSS but also considers MIPSS70+ version 2.0 for confirming the most appropriate treatment approach for the individual patient.

Từ khóa


Tài liệu tham khảo

Barbui, T. et al. The 2016 WHO classification and diagnostic criteria for myeloproliferative neoplasms: document summary and in-depth discussion. Blood Cancer J. 8, 15 (2018).

Tefferi, A. et al. Driver mutations and prognosis in primary myelofibrosis: Mayo-Careggi MPN alliance study of 1,095 patients. Am. J. Hematol. 93, 348–355 (2018).

Tefferi, A. et al. Targeted deep sequencing in primary myelofibrosis. Blood Adv. 1, 105–111 (2016).

Tefferi, A. Primary myelofibrosis: 2017 update on diagnosis, risk-stratification, and management. Am. J. Hematol. 91, 1262–1271 (2016).

Tefferi, A. et al. Long-term survival and blast transformation in molecularly annotated essential thrombocythemia, polycythemia vera, and myelofibrosis. Blood 124, 2507–2513 (2014).

Tefferi, A. et al. Allogeneic hematopoietic stem cell transplant overcomes the adverse survival effect of very high risk and unfavorable karyotype in myelofibrosis. Am. J. Hematol. 93, 649–654 (2018).

Samuelson Bannow, B. T. et al. Hematopoietic cell transplantation for myelofibrosis: the dynamic International Prognostic Scoring System plus risk predicts post-transplant outcomes. Biol. Blood. Marrow Transplant. 24, 386–392 (2018).

Kroger, N. M. et al. Indication and management of allogeneic stem cell transplantation in primary myelofibrosis: a consensus process by an EBMT/ELN international working group. Leukemia 29, 2126–2133 (2015).

Cervantes, F. et al. New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment. Blood 113, 2895–2901 (2009).

Passamonti, F. et al. A dynamic prognostic model to predict survival in primary myelofibrosis: a study by the IWG-MRT (International Working Group for Myeloproliferative Neoplasms Research and Treatment). Blood 115, 1703–1708 (2010).

Gangat, N. et al. DIPSS plus: a refined Dynamic International Prognostic Scoring System for primary myelofibrosis that incorporates prognostic information from karyotype, platelet count, and transfusion status. J. Clin. Oncol. 29, 392–397 (2011).

Tefferi, A. et al. Application of current prognostic models for primary myelofibrosis in the setting of post-polycythemia vera or post-essential thrombocythemia myelofibrosis. Leukemia 31, 2851–2852 (2017).

Passamonti, F. et al. A clinical-molecular prognostic model to predict survival in patients with post polycythemia vera and post essential thrombocythemia myelofibrosis. Leukemia 31, 2726–2731 (2017).

Rotunno, G. et al. Epidemiology and clinical relevance of mutations in postpolycythemia vera and postessential thrombocythemia myelofibrosis: a study on 359 patients of the AGIMM group. Am. J. Hematol. 91, 681–686 (2016).

Tefferi, A. et al. The prognostic advantage of calreticulin mutations in myelofibrosis might be confined to type 1 or type 1-like CALR variants. Blood 124, 2465–2466 (2014).

Li, B. et al. The different prognostic impact of type-1 or type-1 like and type-2 or type-2 like CALR mutations in patients with primary myelofibrosis. Am. J. Hematol. 91, E320–E321 (2016).

Guglielmelli, P. et al. Validation of the differential prognostic impact of type 1/type 1-like versus type 2/type 2-like CALR mutations in myelofibrosis. Blood Cancer J. 5, e360 (2015).

Vannucchi, A. M. et al. Mutations and prognosis in primary myelofibrosis. Leukemia 27, 1861–1869 (2013).

Tefferi A., et al. U2AF1 mutation types in primary myelofibrosis: phenotypic and prognostic distinctions. Leukemia. https://doi.org/10.1038/s41375-018-0078-0 (2018).

Vaidya, R. et al. Monosomal karyotype in primary myelofibrosis is detrimental to both overall and leukemia-free survival. Blood 117, 5612–5615 (2011).

Tefferi A, et al. Revised cytogenetic risk stratification in primary myelofibrosis: analysis based on 1002 informative patients. Leukemia. https://doi.org/10.1038/s41375-018-0018-z (2018).

Mudireddy M, et al. Prefibrotic versus overtly fibrotic primary myelofibrosis: clinical, cytogenetic, molecular and prognostic comparisons. Br. J. Haematol. https://doi.org/10.1111/bjh.14838 (2017).

Guglielmelli, P. et al. Presentation and outcome of patients with 2016 WHO diagnosis of prefibrotic and overt primary myelofibrosis. Blood 129, 3227–3236 (2017).

Guglielmelli, P., Vannucchi, A. M. & Investigators, A. The prognostic impact of bone marrow fibrosis in primary myelofibrosis. Am. J. Hematol. 91, E454–E455 (2016).

Tefferi A, et al. Monocytosis is a powerful and independent predictor of inferior survival in primary myelofibrosis. Br. J. Haematol. https://doi.org/10.1111/bjh.15061 (2017).

Shah, S. et al. Marked elevation of serum lactate dehydrogenase in primary myelofibrosis: clinical and prognostic correlates. Blood Cancer J. 7, 657 (2017).

Tefferi, A. et al. JAK2 germline genetic variation affects disease susceptibility in primary myelofibrosis regardless of V617F mutational status: nullizygosity for the JAK2 46/1 haplotype is associated with inferior survival. Leukemia 24, 105–109 (2010).

Tefferi, A. et al. Low JAK2V617F allele burden in primary myelofibrosis, compared to either a higher allele burden or unmutated status, is associated with inferior overall and leukemia-free survival. Leukemia 22, 756–761 (2008).

Guglielmelli, P. et al. Identification of patients with poorer survival in primary myelofibrosis based on the burden of JAK2V617F mutated allele. Blood 114, 1477–1483 (2009).

Tefferi, A. et al. Circulating interleukin (IL)-8, IL-2R, IL-12, and IL-15 levels are independently prognostic in primary myelofibrosis: a comprehensive cytokine profiling study. J. Clin. Oncol. 29, 1356–1363 (2011).

Pardanani, A., Begna, K., Finke, C., Lasho, T. & Tefferi, A. Circulating levels of MCP-1, sIL-2R, IL-15, and IL-8 predict anemia response to pomalidomide therapy in myelofibrosis. Am. J. Hematol. 86, 343–345 (2011).

Pardanani, A., Finke, C., Abdelrahman, R. A., Lasho, T. L. & Tefferi, A. Associations and prognostic interactions between circulating levels of hepcidin, ferritin and inflammatory cytokines in primary myelofibrosis. Am. J. Hematol. 88, 312–316 (2013).

Guglielmelli, P. et al. MIPSS70: Mutation-Enhanced International Prognostic Score System for Transplantation-Age Patients With Primary Myelofibrosis. J. Clin. Oncol. 36, 310–318 (2018).

Tefferi A et al. MIPSS70+ version 2.0: mutation and karyotype enhanced international prognostic scoring system for primary myelofibrosis. J. Clin. Oncol. (2018, in press).

Tefferi, A. et al. GIPSS: genetically-inspired prognostic scoring system for primary myelofibrosis. Leukemia 32, 1631–1642 (2018).

Cervantes, F. & Pereira, A. Does ruxolitinib prolong the survival of patients with myelofibrosis? Blood 129, 832–837 (2017).

Verstovsek, S. et al. Safety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosis. N. Engl. J. Med. 363, 1117–1127 (2010).

Tefferi, A. et al. Risk factors and a prognostic model for postsplenectomy survival in myelofibrosis. Am. J. Hematol. 92, 1187–1192 (2017).

Thông tin
Thông tin xuất bản

Blood Cancer Journal

Tập 8 Số 8

Thông tin tác giả