免疫调节剂治疗骨髓纤维化研究进展
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摘要
<正>骨髓纤维化(MF)是指骨髓造血组织被纤维组织所代替,以致影响造血功能所致的病理状态,患者主要的临床特征表现为髓外造血有关的肝脾肿大、外周血血细胞计数异常和体质性症状(疲劳、盗汗、发热等),并有一定的几率转化为急性白血病~([1])。近年来研究表明MF是一种慢性炎症相关性疾病~([2]),
引文
[1]Mesa R A,Green A,Barosi G,et al.MPN-associated myelofibrosis(MPN-MF)[J].Leuk Res,2011,35(1):12-13.
[2]Lussana F,R ambaldi A.Inflammation and myeloproliferative neoplasms[J].Autoimmun,2017,85:58-63.
[3]Hasselbalch HC.Perspectives on the impact of JAK-inhibitor therapy upon inflammation-mediated comorbidities in myelofibrosis and related neoplasms[J].Expert Rev Hematol,2014,7(2):203-216.
[4]Pemmaraju N,Kantarjian H,Kadia,et al.A phase I/II study of the Janus kinase(JAK)1 and 2 inhibitor ruxolitinib in patients with relapsed or refractory acute myeloid leukemia[J].Clin Lymphoma Myeloma Leuk,2015,15(3):171-176.
[5]Kleppe M,Kwak M,Koppikar P,et al.JAK-STAT pathway activation in malignant and nonmalignant cells contributes to MPN pathogenesis and therapeutic response[J].Cancer Discov,2015,5(3):316-331.
[6]Fisher DAC,Malkova O,Engle EK,et al.Mass cytometry analysis reveals hyperactive NF Kappa B signaling in myelofibrosis and secondary acute myeloid leukemia[J].Leukemia,2017,31(9):1962-1974.
[7]Tefferi A,Vaidya R,Caramazza D,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].JClin Oncol,2011,29(10):1356-1363
[8]Kastritis E,Dimopoulos MA.The evolving role of lenalidomide in the treatment of hematologic malignancies[J].Expert Opin Pharmacother,2007,8(4):497-509.
[9]Thomas DA,Giles FJ,Albitar M,et al.Thalidomide therapy for myelofibrosis with myeloid metaplasia[J].Cancer,2006,106(9):1974-1984.
[10]Marchetti M,Barosi G,Balestri F,et al.Low-Dose Thalidomide Ameliorates Cytopenias and Splenomegaly in Myelofibrosis With Myeloid Metaplasia:A Phase II Trial[J].J Clin Oncol,2004,22(3):424-431.
[11]Barosi G,Grossi A,Comotti B,et al.Safety and efficacy of thalidomide in patients with myelofibrosis with myeloid metaplasia[J].Br JHaematol,2001,114(1):78-83.
[12]Tefferi A,Elliot MA:Serious myeloproliferative reactions associated w ith the use of thalidomide in myelof ibrosis w ith myeloid metaplasia[J].Blood,2000,96(12):4007.
[13]Barosi G,Rosti V,Vannucchi AM.Therapeutic approaches in myelofibrosis[J].Expert Opin pharmacother,2011,12(10):1597-1611.
[14]Tefferi A,Cortes J,Verstovsek S,et al.Lenalidomide therapy in myelofibrosis with myeloid metaplasia[J].Blood,2006,108(4):1158-1164.
[15]Mesa RA,Pardanani AD,Hussein K,et al.[J]Phase1/-2 study of Pomalidomide in myelofibrosis.Am J hematol,2010,85(2):129-130.
[16]Begna KH,Mesa RA,Pardanani A,et al.A phase-2 trial of low-dose pomalidomide in myelofibrosis[J].Leukemia,2011,25(2):301-304.
[17]Tefferi A,Al-Ali HK,Barosi G,et al.A randomized study of pomalidomide vs placebo in persons with myeloproliferative neoplasmassociated myelofibrosis and RBC-transfusion dependence[J].Leukemia,2017,31(4):896-902.
[18]Begna KH,Pardanani A,Mesa R,et al.Long-term outcome of pomalidomide therapy in myelofibrosis[J].Am J Hematol,2012,87(1):66-68.
[19]Hideshima T,Richardson PG,Anderson KC.Current therapeutic uses of lenalidomide in multiple myeloma[J].Expert Opin Investig Drugs,2006,15(2):171-179.
[20]Mesa RA,Steensma DP,Pardanani A,et al.A phase 2 trial of combination low-dose thalidomide and prednisone for the treatment of myelofibrosis with myeloid metaplasia[J].Blood,2003,101(7):2534-2541.
[21]Mesa RA,Elliott MA,Schroeder G,et al.Durable responses to thalidomide-based drug therapy for myelofibrosis with myeloid metaplasia[J].Mayo Clin Proc,2004,79(7):883-889.
[22]Quintas-Cardama A,Kantarjian HM.Lenalidomide plus prednisone results in durable clinical,histopathologic,and molecular responses in patients with myelofibrosis[J].J Clin Oncol,2009,27(28):4760-4766.
[23]Chihara D,Masarova L,Newberry KJ,et al.Long-term results of a phase II trial of lenalidomide plus prednisone therapy for patients with myelofibrosis.Leukemia Res,2016,48:1-5.
[24]Schlenk R,Stegelmann F,Reiter A,et al.Pomalidomide in myeloproliferative neoplasm-associated myelofibrosis[J].Leukemia,2017,31(4):889-895.
[25]Thapaliya P,Tefferi A,Pardanani A,et al.International working group for myelofibrosis research and treatment response assessment and longterm follow-up of 50 myelofibrosis patients treated with thalidomideprednisone based regimens[J].Am J Hematol,2011,86(1):96-98.
[26]Mesa RA,Yao X,Cripe LD,et al.Lenalidomide and prednisone for myelofibrosis:Eastern Cooperative Oncology Group(ECOG)phase 2trial E4903[J].Blood,2010,116(22):4436-4438.
[27]Jabbour E,Thomas D,Kantarjian H,et al.Comparison of thalidomide and lenalidomide as therapy for myelofibrosis[J].Blood,2011,118(4):899-902.
[28]Tefferi A,Verstovsek S,Barosi G,et al.Pomalidomide is active in the treatment of anemia associated with myelofibrosis[J].J Clin Oncol,2009,27(27):4563-4569.
[29]Cervantes F,Isola IM,Alvarez-Larran A,et al.Danazol therapy for the anemia of myelofibrosis:assessment of efficacy with current criteria of response and long-term results[J].Ann Hematol,2015,94(11):1791-1796.
[30]Luo X,Xu Z,Li B,et al.Thalidomide plus prednisone with or without danazol therapy in myelofibrosis:a retrospective analysis of incidence and durability of anemia response[J].Blood Cancer J,2018,8(1):9.
[31]Ward AC,Touw I,Yoshimura A:The Jak-Stat pathway in normal and perturbed hematopoiesis[J].Blood,2000,95(1):19-29.
[32]Verstovsek S,Mesa RA,Gotlib J,et al.A double-blind,placebocontrolled trial of ruxolitinib for myelofibrosis[J].N Engl J Med,2012,366(9):799-807.
[33]Verstovsek S,Gotlib J,Mesa RA,et al.Long-term survival in patients treated with ruxolitinib for myelofibrosis:COMFORT-I and-II pooled analyses[J].J Hematol Oncol,2017,10(1):156.
[34]Harrison C,Kiladjian J,Al-Ali HK,et al.JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis[J].N Engl J Med,2012,366(9):787-798.
[35]Daver N,Cortes J,Newberry K,et al.Ruxolitinib in combination with lenalidomide as therapy for patients with myelofibrosis.[J]Haematologica,2015,100(8):1058-1063.
[36]Yacoub A,Odenike O,Verstovsek S:Ruxolitinib:long-term management of patients with myelofibrosis and future directions in the treatment of myeloproliferative neoplasms[J].Curr Hematol Malig Rep,2014,9(4):350-359.
[2]Lussana F,R ambaldi A.Inflammation and myeloproliferative neoplasms[J].Autoimmun,2017,85:58-63.
[3]Hasselbalch HC.Perspectives on the impact of JAK-inhibitor therapy upon inflammation-mediated comorbidities in myelofibrosis and related neoplasms[J].Expert Rev Hematol,2014,7(2):203-216.
[4]Pemmaraju N,Kantarjian H,Kadia,et al.A phase I/II study of the Janus kinase(JAK)1 and 2 inhibitor ruxolitinib in patients with relapsed or refractory acute myeloid leukemia[J].Clin Lymphoma Myeloma Leuk,2015,15(3):171-176.
[5]Kleppe M,Kwak M,Koppikar P,et al.JAK-STAT pathway activation in malignant and nonmalignant cells contributes to MPN pathogenesis and therapeutic response[J].Cancer Discov,2015,5(3):316-331.
[6]Fisher DAC,Malkova O,Engle EK,et al.Mass cytometry analysis reveals hyperactive NF Kappa B signaling in myelofibrosis and secondary acute myeloid leukemia[J].Leukemia,2017,31(9):1962-1974.
[7]Tefferi A,Vaidya R,Caramazza D,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].JClin Oncol,2011,29(10):1356-1363
[8]Kastritis E,Dimopoulos MA.The evolving role of lenalidomide in the treatment of hematologic malignancies[J].Expert Opin Pharmacother,2007,8(4):497-509.
[9]Thomas DA,Giles FJ,Albitar M,et al.Thalidomide therapy for myelofibrosis with myeloid metaplasia[J].Cancer,2006,106(9):1974-1984.
[10]Marchetti M,Barosi G,Balestri F,et al.Low-Dose Thalidomide Ameliorates Cytopenias and Splenomegaly in Myelofibrosis With Myeloid Metaplasia:A Phase II Trial[J].J Clin Oncol,2004,22(3):424-431.
[11]Barosi G,Grossi A,Comotti B,et al.Safety and efficacy of thalidomide in patients with myelofibrosis with myeloid metaplasia[J].Br JHaematol,2001,114(1):78-83.
[12]Tefferi A,Elliot MA:Serious myeloproliferative reactions associated w ith the use of thalidomide in myelof ibrosis w ith myeloid metaplasia[J].Blood,2000,96(12):4007.
[13]Barosi G,Rosti V,Vannucchi AM.Therapeutic approaches in myelofibrosis[J].Expert Opin pharmacother,2011,12(10):1597-1611.
[14]Tefferi A,Cortes J,Verstovsek S,et al.Lenalidomide therapy in myelofibrosis with myeloid metaplasia[J].Blood,2006,108(4):1158-1164.
[15]Mesa RA,Pardanani AD,Hussein K,et al.[J]Phase1/-2 study of Pomalidomide in myelofibrosis.Am J hematol,2010,85(2):129-130.
[16]Begna KH,Mesa RA,Pardanani A,et al.A phase-2 trial of low-dose pomalidomide in myelofibrosis[J].Leukemia,2011,25(2):301-304.
[17]Tefferi A,Al-Ali HK,Barosi G,et al.A randomized study of pomalidomide vs placebo in persons with myeloproliferative neoplasmassociated myelofibrosis and RBC-transfusion dependence[J].Leukemia,2017,31(4):896-902.
[18]Begna KH,Pardanani A,Mesa R,et al.Long-term outcome of pomalidomide therapy in myelofibrosis[J].Am J Hematol,2012,87(1):66-68.
[19]Hideshima T,Richardson PG,Anderson KC.Current therapeutic uses of lenalidomide in multiple myeloma[J].Expert Opin Investig Drugs,2006,15(2):171-179.
[20]Mesa RA,Steensma DP,Pardanani A,et al.A phase 2 trial of combination low-dose thalidomide and prednisone for the treatment of myelofibrosis with myeloid metaplasia[J].Blood,2003,101(7):2534-2541.
[21]Mesa RA,Elliott MA,Schroeder G,et al.Durable responses to thalidomide-based drug therapy for myelofibrosis with myeloid metaplasia[J].Mayo Clin Proc,2004,79(7):883-889.
[22]Quintas-Cardama A,Kantarjian HM.Lenalidomide plus prednisone results in durable clinical,histopathologic,and molecular responses in patients with myelofibrosis[J].J Clin Oncol,2009,27(28):4760-4766.
[23]Chihara D,Masarova L,Newberry KJ,et al.Long-term results of a phase II trial of lenalidomide plus prednisone therapy for patients with myelofibrosis.Leukemia Res,2016,48:1-5.
[24]Schlenk R,Stegelmann F,Reiter A,et al.Pomalidomide in myeloproliferative neoplasm-associated myelofibrosis[J].Leukemia,2017,31(4):889-895.
[25]Thapaliya P,Tefferi A,Pardanani A,et al.International working group for myelofibrosis research and treatment response assessment and longterm follow-up of 50 myelofibrosis patients treated with thalidomideprednisone based regimens[J].Am J Hematol,2011,86(1):96-98.
[26]Mesa RA,Yao X,Cripe LD,et al.Lenalidomide and prednisone for myelofibrosis:Eastern Cooperative Oncology Group(ECOG)phase 2trial E4903[J].Blood,2010,116(22):4436-4438.
[27]Jabbour E,Thomas D,Kantarjian H,et al.Comparison of thalidomide and lenalidomide as therapy for myelofibrosis[J].Blood,2011,118(4):899-902.
[28]Tefferi A,Verstovsek S,Barosi G,et al.Pomalidomide is active in the treatment of anemia associated with myelofibrosis[J].J Clin Oncol,2009,27(27):4563-4569.
[29]Cervantes F,Isola IM,Alvarez-Larran A,et al.Danazol therapy for the anemia of myelofibrosis:assessment of efficacy with current criteria of response and long-term results[J].Ann Hematol,2015,94(11):1791-1796.
[30]Luo X,Xu Z,Li B,et al.Thalidomide plus prednisone with or without danazol therapy in myelofibrosis:a retrospective analysis of incidence and durability of anemia response[J].Blood Cancer J,2018,8(1):9.
[31]Ward AC,Touw I,Yoshimura A:The Jak-Stat pathway in normal and perturbed hematopoiesis[J].Blood,2000,95(1):19-29.
[32]Verstovsek S,Mesa RA,Gotlib J,et al.A double-blind,placebocontrolled trial of ruxolitinib for myelofibrosis[J].N Engl J Med,2012,366(9):799-807.
[33]Verstovsek S,Gotlib J,Mesa RA,et al.Long-term survival in patients treated with ruxolitinib for myelofibrosis:COMFORT-I and-II pooled analyses[J].J Hematol Oncol,2017,10(1):156.
[34]Harrison C,Kiladjian J,Al-Ali HK,et al.JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis[J].N Engl J Med,2012,366(9):787-798.
[35]Daver N,Cortes J,Newberry K,et al.Ruxolitinib in combination with lenalidomide as therapy for patients with myelofibrosis.[J]Haematologica,2015,100(8):1058-1063.
[36]Yacoub A,Odenike O,Verstovsek S:Ruxolitinib:long-term management of patients with myelofibrosis and future directions in the treatment of myeloproliferative neoplasms[J].Curr Hematol Malig Rep,2014,9(4):350-359.
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