thalidomide and Agnogenic Myeloid Metaplasia studies

Thalidomide: A piperidinyl isoindole originally introduced as a non-barbiturate hypnotic, but withdrawn from the market due to teratogenic effects. It has been reintroduced and used for a number of immunological and inflammatory disorders. Thalidomide displays immunosuppressive and anti-angiogenic activity. It inhibits release of TUMOR NECROSIS FACTOR-ALPHA from monocytes, and modulates other cytokine action.
thalidomide : A racemate comprising equimolar amounts of R- and S-thalidomide.
2-(2,6-dioxopiperidin-3-yl)-1H-isoindole-1,3(2H)-dione : A dicarboximide that is isoindole-1,3(2H)-dione in which the hydrogen attached to the nitrogen is substituted by a 2,6-dioxopiperidin-3-yl group.

Research Excerpts

Excerpt	Relevance	Reference
"Low-dose thalidomide and prednisone alone or combined are effective therapies in some persons with primary myelofibrosis (PMF) and anemia with or with RBC transfusion dependence."	9.27	Thalidomide plus prednisone with or without danazol therapy in myelofibrosis: a retrospective analysis of incidence and durability of anemia response. ( Fang, L; Hu, N; Huang, G; Li, B; Luo, X; Pan, L; Peter Gale, R; Qin, T; Qu, S; Xiao, Z; Xu, Z; Zhang, H; Zhang, P; Zhang, Y, 2018)
"We evaluated pomalidomide with prednisone for myelofibrosis (MF) with significant anemia (hemoglobin < 10 g/dL)."	9.19	Phase II study of pomalidomide in combination with prednisone in patients with myelofibrosis and significant anemia. ( Cortes, J; Daver, N; Jabbour, E; Kadia, T; Kantarjian, H; Newberry, K; Pemmaraju, N; Pierce, S; Shastri, A; Verstovsek, S; Zhou, L, 2014)
"We evaluated single agent pomalidomide for myelofibrosis-associated anemia."	9.17	Modest activity of pomalidomide in patients with myelofibrosis and significant anemia. ( Cortes, J; Daver, N; Jabbour, E; Kadia, T; Kantarjian, H; Konopleva, M; O'Brien, S; Pierce, S; Quintas-Cardama, A; Shastri, A; Verstovsek, S; Zhou, L, 2013)
" Pomalidomide therapy is effective for alleviating anemia in myelofibrosis; we examined the relationship between plasma cytokine/chemokine levels and response to treatment with pomalidomide."	9.15	Circulating levels of MCP-1, sIL-2R, IL-15, and IL-8 predict anemia response to pomalidomide therapy in myelofibrosis. ( Begna, K; Finke, C; Lasho, T; Pardanani, A; Tefferi, A, 2011)
"5 or 2 mg/d with or without an abbreviated course of prednisone is well tolerated in patients with myelofibrosis and active in the treatment of anemia."	9.14	Pomalidomide is active in the treatment of anemia associated with myelofibrosis. ( Barosi, G; Bekele, BN; Cervantes, F; Deeg, HJ; Gale, RP; Gisslinger, H; Kantarjian, HM; Kvasnicka, HM; Mesa, RA; Paquette, RL; Passamonti, F; Rivera, CE; Roboz, GJ; Tefferi, A; Thiele, J; Vardiman, JW; Verstovsek, S; Zhang, Y, 2009)
"To observe the clinical effects of low-dose thalidomide (THAL) and prednisone (PRED) with or without danazol (DANA) in patients with primary myelofibrosis (PMF) associated anemia."	7.80	[Comparison of low-dose thalidomide and prednisone combined with or without danazol for the treatment of primary myelofibrosis-associated anemia]. ( Fang, L; Hu, N; Li, B; Pan, L; Qin, T; Qu, S; Xiao, Z; Xu, J; Xu, Z; Zhang, H; Zhang, Y, 2014)
"Venous thrombosis is a well-recognized complication of lenalidomide therapy in patients with multiple myeloma, but its occurrence during the treatment of other hematologic malignancies is less well described."	7.74	Thrombosis of the corpora cavernosa complicating lenalidomide therapy of myelofibrosis. ( Carwile, J; El Rassi, FA; Tam, CS; Verstovsek, S, 2008)
"Primary myelofibrosis (PMF) and overlap myelodysplastic/myeloproliferative neoplasms (MDS/MPN) are clonal hematopoietic disorders that share similar clinical features and molecular abnormalities, such as the Janus kinase 2 (JAK2) valine to phenylalanine mutation at codon 617 (V617F) and the tet methylcytosine dioxygenase 2 (TET2) mutation."	6.77	A phase 2 trial of combination therapy with thalidomide, arsenic trioxide, dexamethasone, and ascorbic acid (TADA) in patients with overlap myelodysplastic/myeloproliferative neoplasms (MDS/MPN) or primary myelofibrosis (PMF). ( Advani, A; Bejanyan, N; Chan, J; Jankowska, A; Kalaycio, M; Maciejewski, JP; Mooney, L; Raza, A; Saunthararajah, Y; Sekeres, MA; Tiu, RV, 2012)
"Primary myelofibrosis is a clonal haematopoietic stem cell disease, characterised by marrow stromal fibrosis, extramedullary haematopoiesis, splenomegaly, hepatomegaly and progressive cytopenia."	6.46	Thalidomide and lenalidomide in primary myelofibrosis. ( de Witte, T; Holle, N; Mandigers, C; Raymakers, R; Schaap, N, 2010)
"Erythema nodosum (EN) is a panniculitis frequently encountered secondary to medical therapy."	5.43	Erythema Nodosum-like Septal Panniculitis Secondary to Lenalidomide Therapy in a Patient With Janus Kinase 2-Positive Myelofibrosis. ( Fung, MA; Jagdeo, J; Mayo, K; Petukhova, TA; Tartar, DM; Tuscano, J, 2016)
"Primary myelofibrosis (PMF) is a chronic myeloproliferative neoplasm characterized by progressive anemia, massive splenomegaly, leukoerythroblastosis, extramedullary hematopoiesis and in about 50% of cases the presence of JAK2V617F mutation."	5.35	Toxic epidermal necrolysis in a patient with primary myelofibrosis receiving thalidomide therapy. ( Colagrande, M; Coletti, G; Di Ianni, M; Fargnoli, MC; Lapecorella, M; Moretti, L; Peris, K; Tabilio, A, 2009)
"We report a patient with very advanced myelofibrosis and huge splenomegaly who showed a complete hematological response to low dose thalidomide with reversal of splenomegaly and bone narrow fibrosis after 30 months of the treatment."	5.34	Mini-dose of thalidomide for treatment of primary myelofibrosis. Report of a case with complete reversal of bone marrow fibrosis and splenomegaly. ( Berrebi, A; Feldberg, E; Shvidel, L; Spivak, I, 2007)
"Thalidomide was started at a daily dose of 100 mg/d and increased to 400 mg/d."	5.33	Hairy cell leukemia (HCL) with extensive myelofibrosis responds to thalidomide. ( Fenk, R; Gattermann, N; Germing, U; Haas, R; Kündgen, A; Strupp, C, 2005)
"Low-dose thalidomide and prednisone alone or combined are effective therapies in some persons with primary myelofibrosis (PMF) and anemia with or with RBC transfusion dependence."	5.27	Thalidomide plus prednisone with or without danazol therapy in myelofibrosis: a retrospective analysis of incidence and durability of anemia response. ( Fang, L; Hu, N; Huang, G; Li, B; Luo, X; Pan, L; Peter Gale, R; Qin, T; Qu, S; Xiao, Z; Xu, Z; Zhang, H; Zhang, P; Zhang, Y, 2018)
"Our aim was to evaluate the relationship between anemia response to therapy with pomalidomide with or without corticosteroids and patient-reported outcomes using the FACT-An instrument."	5.19	Use of the Functional Assessment of Cancer Therapy--anemia in persons with myeloproliferative neoplasm-associated myelofibrosis and anemia. ( Cervantes, F; Gale, RP; Hudgens, S; Khan, ZM; Mesa, R; Passamonti, F; Rivera, C; Tefferi, A; Tencer, T; Verstovsek, S, 2014)
"We evaluated pomalidomide with prednisone for myelofibrosis (MF) with significant anemia (hemoglobin < 10 g/dL)."	5.19	Phase II study of pomalidomide in combination with prednisone in patients with myelofibrosis and significant anemia. ( Cortes, J; Daver, N; Jabbour, E; Kadia, T; Kantarjian, H; Newberry, K; Pemmaraju, N; Pierce, S; Shastri, A; Verstovsek, S; Zhou, L, 2014)
"We evaluated single agent pomalidomide for myelofibrosis-associated anemia."	5.17	Modest activity of pomalidomide in patients with myelofibrosis and significant anemia. ( Cortes, J; Daver, N; Jabbour, E; Kadia, T; Kantarjian, H; Konopleva, M; O'Brien, S; Pierce, S; Quintas-Cardama, A; Shastri, A; Verstovsek, S; Zhou, L, 2013)
"5 mg) pomalidomide and prednisone and pomalidomide alone (2 mg/day), for the treatment of anemia associated with myelofibrosis (MF)."	5.15	A phase-2 trial of low-dose pomalidomide in myelofibrosis. ( Begna, KH; Hogan, WJ; Litzow, MR; McClure, RF; Mesa, RA; Pardanani, A; Tefferi, A, 2011)
" Pomalidomide therapy is effective for alleviating anemia in myelofibrosis; we examined the relationship between plasma cytokine/chemokine levels and response to treatment with pomalidomide."	5.15	Circulating levels of MCP-1, sIL-2R, IL-15, and IL-8 predict anemia response to pomalidomide therapy in myelofibrosis. ( Begna, K; Finke, C; Lasho, T; Pardanani, A; Tefferi, A, 2011)
"5 or 2 mg/d with or without an abbreviated course of prednisone is well tolerated in patients with myelofibrosis and active in the treatment of anemia."	5.14	Pomalidomide is active in the treatment of anemia associated with myelofibrosis. ( Barosi, G; Bekele, BN; Cervantes, F; Deeg, HJ; Gale, RP; Gisslinger, H; Kantarjian, HM; Kvasnicka, HM; Mesa, RA; Paquette, RL; Passamonti, F; Rivera, CE; Roboz, GJ; Tefferi, A; Thiele, J; Vardiman, JW; Verstovsek, S; Zhang, Y, 2009)
"We conducted a nonrandomized prospective phase II study of thalidomide in anemic patients with myelofibrosis with myeloid metaplasia (MMM), with or without preceding polycythemia vera or essential thrombocythemia, with a primary aim to improve anemia."	5.10	Negligible clinical effects of thalidomide in patients with myelofibrosis with myeloid metaplasia. ( Birgergård, G; Björkholm, M; Kutti, J; Maim, C; Markevärn, B; Mauritzson, N; Merup, M; Palmblad, J; Westin, J, 2002)
" Tipifarnib may be added to conventional therapeutic instruments for symptomatic primary myelofibrosis."	4.85	Novel strategies for patients with chronic myeloproliferative disorders. ( Barosi, G; Rosti, V, 2009)
"The purpose of the present study was to determine whether genes involved in the organization of the hematopoietic niche were dysregulated in patients with primary myelofibrosis (MF) treated with lenalidomide."	3.81	Genes Involved in Maintaining the Bone Marrow Stroma Are Dysregulated in Patients with Myelofibrosis: Lenalidomide Treatment Up-regulates SOCS3. ( Kusec, R; Livun, A; Manshouri, T; Newberry, KJ; Verstovsek, S, 2015)
"To observe the clinical effects of low-dose thalidomide (THAL) and prednisone (PRED) with or without danazol (DANA) in patients with primary myelofibrosis (PMF) associated anemia."	3.80	[Comparison of low-dose thalidomide and prednisone combined with or without danazol for the treatment of primary myelofibrosis-associated anemia]. ( Fang, L; Hu, N; Li, B; Pan, L; Qin, T; Qu, S; Xiao, Z; Xu, J; Xu, Z; Zhang, H; Zhang, Y, 2014)
"Venous thrombosis is a well-recognized complication of lenalidomide therapy in patients with multiple myeloma, but its occurrence during the treatment of other hematologic malignancies is less well described."	3.74	Thrombosis of the corpora cavernosa complicating lenalidomide therapy of myelofibrosis. ( Carwile, J; El Rassi, FA; Tam, CS; Verstovsek, S, 2008)
"Prednisolone was added if there was no response after 3 months in cohort 1 and based on up-front randomization in cohort 2 if there was no response at 3 or 6 months."	2.84	Pomalidomide in myeloproliferative neoplasm-associated myelofibrosis. ( Benner, A; Blau, IW; Döhner, H; Döhner, K; Gattermann, N; Grießhammer, M; Hebart, H; Heidel, FH; Hochhaus, A; Jost, E; Kreipe, H; Platzbecker, U; Reiter, A; Schafhausen, P; Schlenk, RF; Stegelmann, F; Teleanu, V; Verbeek, W; Waller, C; Werner, M, 2017)
"Improvements in bone marrow fibrosis and serial reductions in lactate dehydrogenase >50% were noted in 17% and 50% of evaluable responders, respectively."	2.80	Ruxolitinib in combination with lenalidomide as therapy for patients with myelofibrosis. ( Borthakur, G; Cortes, J; Daver, N; Jabbour, E; Kadia, T; Kantarjian, H; Newberry, K; Pemmaraju, N; Pierce, S; Ravandi, F; Sasaki, K; Verstovsek, S; Wang, X; Zhou, L, 2015)
"Ninety-four Mayo Clinic patients with myelofibrosis (MF) participated in two consecutive clinical trials of pomalidomide (0."	2.77	Long-term outcome of pomalidomide therapy in myelofibrosis. ( Begna, KH; Hanson, CA; Hogan, WJ; Litzow, MR; Mesa, R; Pardanani, A; Tefferi, A, 2012)
"Primary myelofibrosis (PMF) and overlap myelodysplastic/myeloproliferative neoplasms (MDS/MPN) are clonal hematopoietic disorders that share similar clinical features and molecular abnormalities, such as the Janus kinase 2 (JAK2) valine to phenylalanine mutation at codon 617 (V617F) and the tet methylcytosine dioxygenase 2 (TET2) mutation."	2.77	A phase 2 trial of combination therapy with thalidomide, arsenic trioxide, dexamethasone, and ascorbic acid (TADA) in patients with overlap myelodysplastic/myeloproliferative neoplasms (MDS/MPN) or primary myelofibrosis (PMF). ( Advani, A; Bejanyan, N; Chan, J; Jankowska, A; Kalaycio, M; Maciejewski, JP; Mooney, L; Raza, A; Saunthararajah, Y; Sekeres, MA; Tiu, RV, 2012)
"Lenalidomide therapy was continued indefinitely in patients exhibiting clinical benefit."	2.74	Lenalidomide plus prednisone results in durable clinical, histopathologic, and molecular responses in patients with myelofibrosis. ( Bueso-Ramos, C; Cortes, J; Ferrajoli, A; Garcia-Manero, G; Kantarjian, HM; Manshouri, T; Quintás-Cardama, A; Ravandi, F; Thomas, D; Verstovsek, S, 2009)
"Thalidomide is a putative antiangiogenesis agent with activity in several hematologic malignancies."	2.72	Thalidomide therapy for myelofibrosis with myeloid metaplasia. ( Albitar, M; Cortes, JE; Faderl, S; Garcia-Manero, G; Giles, FJ; Kantarjian, HM; Keating, MJ; O'Brien, SM; Pierce, S; Thomas, DA; Verstovsek, S; Zeldis, J, 2006)
"Thalidomide was administered together with current therapy to 63 patients, starting at 50 mg daily and increasing to 400 mg as tolerated."	2.71	Low-dose thalidomide ameliorates cytopenias and splenomegaly in myelofibrosis with myeloid metaplasia: a phase II trial. ( Balestri, F; Barosi, G; Barulli, S; Bauduer, F; Bendotti, C; Bordessoule, D; Broccia, G; Buccisano, F; Caenazzo, A; Demory, JL; Dupriez, B; Falcone, A; Gentili, S; Grossi, A; Ilariucci, F; Le Bousse-Kerdiles, MC; Marchetti, M; Pecci, A; Viarengo, G; Volpe, A, 2004)
"Thalidomide is an anti-angiogenic and immunomodulatory drug with a wide spectrum of activities, which are not clearly understood."	2.71	Thalidomide for the treatment of idiopathic myelofibrosis. ( Gattermann, N; Germing, U; Haas, R; Kündgen, A; Mödder, U; Scherer, A; Strupp, C, 2004)
"Myelofibrosis with myeloid metaplasia (MMM) is uniquely characterized by macroscopic bone marrow stromal changes that are believed to be both reactive and cytokine mediated."	2.70	Thalidomide treatment in myelofibrosis with myeloid metaplasia. ( Ansell, SM; Elliott, MA; Geyer, SM; Hook, CC; Levitt, RM; Li, CY; Mesa, RA; Tefferi, A, 2002)
"Myelofibrosis with myeloid metaplasia (MMM) is a clonal disorder involving disregulation of angiogenesis and immunomodulatory mechanisms."	2.70	Thalidomide in agnogenic and secondary myelofibrosis. ( Ballerini, F; Canepa, L; Clavio, M; Gobbi, M; Miglino, M; Pierri, I; Quintino, S; Reni, L; Varaldo, R, 2001)
"However, as myelofibrosis is not a disease with spontaneous remissions, even non-randomized trials carry weight."	2.50	Does anything work for anaemia in myelofibrosis? ( Birgegård, G, 2014)
"Primary myelofibrosis is a clonal haematopoietic stem cell disease, characterised by marrow stromal fibrosis, extramedullary haematopoiesis, splenomegaly, hepatomegaly and progressive cytopenia."	2.46	Thalidomide and lenalidomide in primary myelofibrosis. ( de Witte, T; Holle, N; Mandigers, C; Raymakers, R; Schaap, N, 2010)
"Idiopathic myelofibrosis a Philadelphia-negative chronic myeloproliferative disorder."	2.45	[Advances in thalidomide therapy for idiopathic myelofibrosis]. ( Chen, JL; Song, L, 2009)
"The conventional treatment of myelofibrosis involves a wait-and-see approach for asymptomatic patients, oral chemotherapy for the hyperproliferative forms of the disease, androgens or erythropoietin for the anaemia, and splenectomy in selected patients."	2.43	Modern management of myelofibrosis. ( Cervantes, F, 2005)
"Myelofibrosis with myeloid metaplasia (MMM) is a clonal stem cell disorder that is characterized by florid bone marrow stromal reaction including collagen fibrosis, osteosclerosis, and angiogenesis."	2.42	Angiogenesis and anti-angiogenic therapy in myelofibrosis with myeloid metaplasia. ( Arora, B; Mesa, R; Tefferi, A, 2004)
"Myelofibrosis with myeloid metaplasia (MMM) is currently classified as a chronic myeloproliferative disorder (CMPD) and is characterized by prominent bone marrow stromal reaction including collagen fibrosis, osteosclerosis, and angiogenesis."	2.42	Treatment approaches in myelofibrosis with myeloid metaplasia: the old and the new. ( Tefferi, A, 2003)
"Median survival of patients with myelofibrosis with myeloid metaplasia (MMM) ranges from 3."	2.41	Prognostic factors and current practice in treatment of myelofibrosis with myeloid metaplasia: an update anno 2000. ( Cervantes, F, 2001)
"Anemia in myelofibrosis (MF) occurs frequently, is poorly addressed by US Food and Drug Administration-approved JAK inhibitors, and negatively impacts quality of life."	1.56	Retrospective Analysis of the Clinical Use and Benefit of Lenalidomide and Thalidomide in Myelofibrosis. ( Al Ali, N; Castillo-Tokumori, F; Komrokji, R; Kuykendall, AT; Lancet, J; Padron, E; Sallman, D; Sweet, K; Talati, C; Yun, S, 2020)
"Erythema nodosum (EN) is a panniculitis frequently encountered secondary to medical therapy."	1.43	Erythema Nodosum-like Septal Panniculitis Secondary to Lenalidomide Therapy in a Patient With Janus Kinase 2-Positive Myelofibrosis. ( Fung, MA; Jagdeo, J; Mayo, K; Petukhova, TA; Tartar, DM; Tuscano, J, 2016)
"Primary myelofibrosis is a clonal disease of chronic myeloproliferative neoplasm, and is a progressive clinical course with short median survival of less than 5 years after diagnosis."	1.40	Thalidomide treatment in a myelofibrosis patient with leukemia transformation. ( Chu, SC; Huang, WH; Kao, RH; Li, MS; Wang, TF; Wu, YF, 2014)
"A diagnosis of ALL with myelofibrosis is rare in the adult population, acute myelofibrosis (AMF) is more commonly associated with myeloproliferative disorders, and the development of acute leukemia in myeloma is rare."	1.39	Acute myelofibrosis and acute lymphoblastic leukemia in an elderly patient with previously treated multiple myeloma. ( Chen, L; Gonzalez, MM; Kidd, L; Nguyen, N; Quesada, J, 2013)
"Lenalidomide is an immunomodulator used to treat 5q-myelodysplastic syndrome, myelofibrosis, and multiple myeloma."	1.38	Lenalidomide-induced elevated bilirubin. ( Kolesar, JM; Simondsen, KA, 2012)
"Primary myelofibrosis (PMF) is a chronic myeloproliferative neoplasm characterized by progressive anemia, massive splenomegaly, leukoerythroblastosis, extramedullary hematopoiesis and in about 50% of cases the presence of JAK2V617F mutation."	1.35	Toxic epidermal necrolysis in a patient with primary myelofibrosis receiving thalidomide therapy. ( Colagrande, M; Coletti, G; Di Ianni, M; Fargnoli, MC; Lapecorella, M; Moretti, L; Peris, K; Tabilio, A, 2009)
"We report a patient with very advanced myelofibrosis and huge splenomegaly who showed a complete hematological response to low dose thalidomide with reversal of splenomegaly and bone narrow fibrosis after 30 months of the treatment."	1.34	Mini-dose of thalidomide for treatment of primary myelofibrosis. Report of a case with complete reversal of bone marrow fibrosis and splenomegaly. ( Berrebi, A; Feldberg, E; Shvidel, L; Spivak, I, 2007)
"Thalidomide was started at a daily dose of 100 mg/d and increased to 400 mg/d."	1.33	Hairy cell leukemia (HCL) with extensive myelofibrosis responds to thalidomide. ( Fenk, R; Gattermann, N; Germing, U; Haas, R; Kündgen, A; Strupp, C, 2005)
"She was also diagnosed as having a myelofibrosis with myeloid metaplasia (MMM)."	1.33	Endolymphatic hydrops as a cause of audio-vestibular manifestations in relapsing polychondritis. ( Horii, A; Kubo, T; Mitani, K; Mizuki, M; Murata, J; Tamura, M, 2006)
"Thalidomide-based therapy has the potential to produce durable responses in MMM-associated cytopenias, even after discontinuation of the drug."	1.32	Durable responses to thalidomide-based drug therapy for myelofibrosis with myeloid metaplasia. ( Elliott, MA; Mesa, RA; Schroeder, G; Tefferi, A, 2004)
"Thalidomide was administered at daily doses increasing from 100 to 600 mg."	1.31	Clinical efficacy and antiangiogenic activity of thalidomide in myelofibrosis with myeloid metaplasia. A pilot study. ( Ascani, S; Baccarani, M; Finelli, C; Grafone, T; Isidori, A; Malagola, M; Martinelli, G; Piccaluga, PP; Pileri, SA; Ricci, P; Tura, S; Visani, G, 2002)

Research

Studies (91)

Authors

Clinical Trials (8)

Trial Overview

Trial Outcomes

Number of Participants With Best Overall Response

Timeframe	Studies, this research(%)	All Research%
pre-1990	0 (0.00)	18.7374
1990's	0 (0.00)	18.2507
2000's	45 (49.45)	29.6817
2010's	43 (47.25)	24.3611
2020's	3 (3.30)	2.80

Authors	Studies
Qu, S	3
Xu, Z	3
Qin, T	3
Li, B	3
Pan, L	3
Chen, J	1
Yan, X	1
Wu, J	1
Zhang, Y	4
Zhang, P	2
Gale, RP	5
Xiao, Z	3
Chowdhury, O	1
O'Sullivan, J	1
Barkas, N	1
Wang, G	1
Buck, G	1
Hamblin, A	1
Tefferi, A	21
Al-Ali, HK	2
Barosi, G	7
Devos, T	2
Gisslinger, H	4
Jiang, Q	2
Kiladjian, JJ	2
Mesa, R	5
Passamonti, F	4
Ribrag, V	2
Schiller, G	2
Vannucchi, AM	3
Zhou, D	2
McMullin, MF	3
Zhong, J	2
Mead, AJ	1
Castillo-Tokumori, F	1
Talati, C	2
Al Ali, N	2
Sallman, D	1
Yun, S	1
Sweet, K	2
Padron, E	2
Lancet, J	1
Komrokji, R	1
Kuykendall, AT	2
Sallman, DA	1
Lancet, JE	2
List, AF	2
Zuckerman, KS	1
Komrokji, RS	1
Luo, X	1
Zhang, H	2
Fang, L	2
Hu, N	2
Huang, G	1
Peter Gale, R	1
Fan, WJ	1
Wu, T	1
Chen, JW	1
Bai, H	1
Cervantes, F	6
Martinez-Trillos, A	1
Burgstaller, S	1
Fridrik, M	1
Hojas, S	1
Kühr, T	1
Ludwig, H	1
Mayrbäurl, B	1
Pöhnl, R	1
Pötscher, M	1
Schlögl, E	1
Zauner, D	1
Thaler, J	1
Machaczka, M	1
Laurizohn, C	1
Gonzalez, MM	1
Kidd, L	1
Quesada, J	1
Nguyen, N	1
Chen, L	1
Daver, N	3
Shastri, A	2
Kadia, T	3
Quintas-Cardama, A	2
Jabbour, E	4
Konopleva, M	1
O'Brien, S	1
Pierce, S	6
Zhou, L	4
Cortes, J	8
Kantarjian, H	6
Verstovsek, S	13
Guglielmelli, P	1
Huang, WH	1
Li, MS	1
Chu, SC	1
Wang, TF	1
Kao, RH	1
Wu, YF	1
Hudgens, S	1
Rivera, C	1
Tencer, T	1
Khan, ZM	1
Andrei, M	1
Sindhu, H	1
Wang, JC	1
Newberry, K	2
Pemmaraju, N	2
Xu, J	1
Birgegård, G	1
Yoshimoto, G	1
Miyamoto, T	1
Wang, X	1
Sasaki, K	1
Borthakur, G	1
Ravandi, F	3
Aylı, M	1
Özcan, M	1
Cengiz Seval, G	1
Livun, A	1
Newberry, KJ	2
Manshouri, T	2
Kusec, R	1
Sharma, S	1
Chandra, J	1
Gupta, R	1
Chauhan, R	1
Chihara, D	1
Masarova, L	1
Maeng, H	1
Garcia-Manero, G	3
Ferrajoli, A	2
Petukhova, TA	1
Tartar, DM	1
Mayo, K	1
Fung, MA	1
Tuscano, J	1
Jagdeo, J	1
Reiser, D	1
Schlenk, RF	1
Stegelmann, F	1
Reiter, A	1
Jost, E	1
Gattermann, N	4
Hebart, H	1
Waller, C	1
Hochhaus, A	1
Platzbecker, U	1
Schafhausen, P	1
Blau, IW	1
Verbeek, W	1
Heidel, FH	1
Werner, M	1
Kreipe, H	1
Teleanu, V	1
Benner, A	1
Döhner, H	1
Grießhammer, M	1
Döhner, K	1
Ross, DM	1
Weinkove, R	1
Reilly, JT	1
Curtin, NJ	1
Radia, D	1
Harrison, CN	1
Colagrande, M	1
Di Ianni, M	1
Coletti, G	1
Peris, K	1
Fargnoli, MC	1
Moretti, L	1
Lapecorella, M	1
Tabilio, A	1
Mesa, RA	11
Hanson, CA	3
Ketterling, RP	3
Schwager, S	2
Knudson, RA	1
Rosti, V	1
Crystal, SC	1
Leonidas, J	1
Jakubowski, A	1
Di Rocco, A	1
Roboz, GJ	1
Paquette, RL	1
Rivera, CE	1
Deeg, HJ	1
Thiele, J	1
Kvasnicka, HM	1
Vardiman, JW	1
Bekele, BN	1
Kantarjian, HM	4
Thomas, D	3
Bueso-Ramos, C	1
Song, L	1
Chen, JL	1
Pardanani, AD	1
Hussein, K	1
Wu, W	2
Litzow, MR	4
Hogan, WJ	4
Lacy, MQ	2
Rajkumar, SV	1
Yao, X	1
Cripe, LD	1
Li, CY	3
Litzow, M	1
Paietta, E	1
Rowe, JM	1
Tallman, MS	1
Biemond, BJ	1
Holle, N	1
de Witte, T	1
Mandigers, C	1
Schaap, N	1
Raymakers, R	1
Begna, KH	2
Pardanani, A	6
McClure, RF	2
Thapaliya, P	1
Steensma, DP	2
Camoriano, J	1
Geyer, S	1
Hattori, Y	1
Miyakawa, Y	1
Yokoyama, K	1
Yamada, T	1
Du, W	1
Jinzaki, M	1
Shinmoto, H	1
Okamoto, S	1
Begna, K	1
Finke, C	1
Lasho, T	1
Wémeau, M	1
Gauthier, J	1
Leleu, X	2
Yakoub-Agha, I	2
Bejanyan, N	1
Tiu, RV	1
Raza, A	1
Jankowska, A	1
Kalaycio, M	1
Advani, A	1
Chan, J	1
Saunthararajah, Y	1
Mooney, L	1
Maciejewski, JP	1
Sekeres, MA	2
Kreuziger, LB	1
Carlson, M	1
Mesa, H	1
Gupta, P	1
Simondsen, KA	1
Kolesar, JM	1
Hebballi, S	1
Akiki, S	1
Bareford, D	1
Takahashi, K	1
Abruzzo, L	1
Merup, M	1
Kutti, J	1
Birgergård, G	1
Mauritzson, N	1
Björkholm, M	1
Markevärn, B	1
Maim, C	1
Westin, J	1
Palmblad, J	1
Piccaluga, PP	3
Visani, G	3
Pileri, SA	1
Ascani, S	1
Grafone, T	1
Isidori, A	2
Malagola, M	2
Finelli, C	2
Martinelli, G	1
Ricci, P	1
Baccarani, M	1
Tura, S	1
Elliott, M	2
Kaufmann, SH	1
Wiseman, G	1
Gray, LA	1
Schroeder, G	2
Reeder, T	1
Zeldis, JB	2
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Giovanni, B	1
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Letizia, C	1
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Giuseppe, V	1
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Monia, M	1
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Strupp, C	3
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Trial	Phase	Enrollment	Study Type	Start Date	Status
A Phase II, Prospective, Open Label Study (PO-MMM-PI-0011) to Determine the Safety and Efficacy of Pomalidomide (CC-4047) in Subjects With Primary, Post Polycythemia Vera, or Post Essential Thrombocythemia Myelofibrosis (PMF; Post-PV MF, or Post-ET MF)[NCT00946270]	Phase 2	70 participants (Actual)	Interventional	2009-07-22	Completed
The Benefit/Risk Profile of Pegylated Proline-Interferon Alpha-2b (AOP2014) Added to the Best Available Strategy Based on Phlebotomies in Low-risk Patients With Polycythemia Vera (PV). The Low-PV Randomized Trial[NCT03003325]	Phase 2	127 participants (Actual)	Interventional	2017-02-02	Completed
Evaluation of Ruxolitinib and Lenalidomide Combination as a Therapy for Patients With Myelofibrosis[NCT01375140]	Phase 2	31 participants (Actual)	Interventional	2011-09-22	Completed
A Phase-3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Compare Efficacy and Safety of Pomalidomide in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis and Red Blood Cell-Transfusion-Dependence[NCT01178281]	Phase 3	267 participants (Actual)	Interventional	2010-09-08	Completed
Multi-Center Phase II Study With Pomalidomide in Patients With Myeloproliferative Neoplasms in Fibrotic Stage[NCT00949364]	Phase 2	103 participants (Actual)	Interventional	2009-12-31	Completed
Evaluation of Lenalidomide (CC-5013) and Prednisone as a Therapy for Patients With Myelofibrosis (MF)[NCT00352794]	Phase 2	40 participants (Actual)	Interventional	2006-07-07	Completed
A Phase II Study of Lenalidomide (CC-5013) in Combination With Prednisone for the Treatment of Myelofibrosis With Myeloid Metaplasia[NCT00227591]	Phase 2	48 participants (Actual)	Interventional	2005-12-31	Completed
Evaluation of TNF-Alpha Modulator for Clinical and Molecular Indicators of Analgesic Effect[NCT00121563]	Phase 2	90 participants	Interventional	2005-07-31	Completed
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Primary endpoint is best overall response. An evaluable subject classified as a treatment success for the primary endpoint if the subject's best overall response is clinical improvement (CI) as determined by International Working Group Criteria over the first 6 cycles of study treatment. International Working Group (IWG) consensus criteria for treatment response in myelofibrosis - Clinical improvement (CI) in anemia 1/ A minimum 20g/L increase in hemoglobin level or 2. becoming transfusion independent for at least 8 week duration. (NCT00946270)
Timeframe: 6 months

Participants With Objective Response

Intervention	Participants (Count of Participants)
Group 1 CC-4047	0
Group 2	3
Group 3 CC-4047 + Prednisone	6

To determine the efficacy of the combination of Ruxolitinib + Lenalidomide in patients with Myelofibrosis (MF). Objective response rate equals Complete and Partial Response, and Clinical Improvement as defined by International Working Group for Myelofibrosis Research and Treatment (IWG-MRT). Objective response rate (ORR), defined as a clinical improvement (CI), partial remission (PR), and complete remission (CR) according to the International Working Group (IWG) Criteria. Complete remission (CR): bone marrow blasts <5%, hemoglobin >/= 10, absolute neutrophil count (ANC) >/= 1000, platelets >/= 100, <2% immature myeloid cell, spleen and liver not palpable. Partial Response (PR): CR plus one or more of the following: ANC >/= 1000, decreased platelets by 50%, hemoglobin >/= 8.5 but < 10, <2% immature myeloid cells. Clinical improvement (CI): hemoglobin increase of 2g/dl, transfusion independence or reduction splenomegaly and/or hepatomegaly >/= 50%, >/=50% reduction in MPN-SAF TSS (NCT01375140)
Timeframe: 3 cycles (28 days each) up to 3 months

China Extension: Number of Participants Achieving a Hemoglobin Increase of ≥ 15 g/L Compared to Baseline for ≥ 84 Consecutive Days

Intervention	Participants (Count of Participants)
Ruxolitinib + Lenalidomide	7

A response in the China extension study was defined as an increase in hemoglobin ≥ 15 g/L above baseline value (in the absence of RBC transfusion) for ≥ 84 consecutive days. (NCT01178281)
Timeframe: From the first dose of study drug until treatment discontinuation; median treatment duration was 24.0 weeks.

Duration of RBC-Transfusion Independence

Intervention	Participants (Count of Participants)
China Extension: Pomalidomide 0.5 mg	1

The duration of RBC-transfusion independence is the time from the date at which the first RBC-transfusion independence started to the date of another RBC-transfusion given at least 84 days after the time the transfusion independence started. The duration of the RBC-transfusion independence was analyzed using the Kaplan-Meier method. Data were censored at the end of the treatment phase for participants who had not received another RBC-transfusion after the start of transfusion independence by the end of treatment phase. (NCT01178281)
Timeframe: From first dose of study drug up to 28 days after last dose, as of the data cut-off date of 16 Jan 2013; median treatment duration was 23.6 weeks in the pomalidomide arm and 23.9 weeks in the placebo arm.

Overall Survival

Intervention	months (Median)
Pomalidomide 0.5 mg	NA
Placebo	5.8

The time from randomization to the death or to the latest date when participants are known to be alive. Overall survival was analyzed using Kaplan-Meier method; participants who were alive or lost to follow-up were censored at the latest date they were known to be alive. (NCT01178281)
Timeframe: From first dose of study drug up to end of study; median follow-up time was 19.1 months in the pomalidomide 0.5 mg arm and 17.6 months in the placebo arm.

Percentage of Participants Who Achieved RBC-Transfusion Independence

Intervention	months (Median)
Pomalidomide 0.5 mg	24.2
Placebo	26.2

RBC-transfusion independence was defined as the absence of RBC transfusions for any consecutive 84-day interval. (NCT01178281)
Timeframe: 168 days

Time to RBC-Transfusion Independence

Intervention	percentage of participants (Number)
Pomalidomide 0.5 mg	17.3
Placebo	16.7

Time to response was measured from first dose of study drug to the start of the first response. The start date of the response was defined as one day after the last date of an RBC-transfusion for participants who received a RBC-transfusion after the first dose, and as the date of the first dose of study drug for participants who received no RBC-transfusions during the 84 days after the first dose of study drug. (NCT01178281)
Timeframe: 168 days

Change From Baseline in EuroQoL-5D (EQ-5D) Health Index Score

Intervention	weeks (Median)
Pomalidomide 0.5 mg	6.9
Placebo	2.4

"EQ-5D is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D includes 2 components: the EQ-5D health state profile (descriptive system) and the EQ-5D visual analog scale (VAS). For the health state profile participants rate their perceived health state today on 5 dimensions: mobility, selfcare, usual activities, pain/discomfort, and anxiety/depression on a Likert-type scale from 1 to 3, where 1 = no problems, 2 = some problems, and 3 = extreme problems. The EQ-5D Health Utility Index (HUI) was generated from the five health state domain scores, and ranges from -0.594 (worst) and 1 (best) imaginable health state, with -0.594 representing an unconscious health state." (NCT01178281)
Timeframe: Baseline and Days 85 and 169

Change From Baseline in EuroQoL-5D (EQ-5D) Visual Analog Scale

Intervention	score on a scale (Mean)
	Day 85	Day 169
Placebo	-0.0298	0.0766
Pomalidomide 0.5 mg	-0.0385	-0.0202

EQ-5D is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D includes 2 components: the EQ-5D health state profile (descriptive system) and the EQ-5D visual analog scale (VAS). On the VAS the participant rates his/her health state on a line from 0 (worst imaginable health) to 100 (best imaginable health). (NCT01178281)
Timeframe: Baseline and Days 85 and 169

Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Total Score

Intervention	units on a scale (Mean)
	Day 85	Day 169
Placebo	-1.4	0.3
Pomalidomide 0.5 mg	2.0	2.9

The FACT-An is a 47-item, cancer-specific questionnaire consisting of a core 27-item general questionnaire measuring the four general domains of QoL (physical, social/family, emotional and functional well-being), and an additional 20-item anemia questionnaire (FACT-An Anemia subscale) that measures 13 fatigue-associated items (FACT-F Fatigue subscale) and seven non-fatigue-related items. Each item is scored using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a bit; and 4 = Very much). FACT-An total score is calculated by adding all the FACT-An subscales together. The total score ranges from 0-188 with higher scores representing better QOL. (NCT01178281)
Timeframe: Baseline and Days 85 and 169

Number of Participants With Treatment-emergent Adverse Events (TEAE)

Intervention	units on a scale (Mean)
	Day 85	Day 169
Placebo	4.3	11.9
Pomalidomide 0.5 mg	-2.1	6.2

A TEAE is an adverse event (AE) that starts on or after the first dose of study drug. The severity of each AE was graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE),Version 4.0 and according to the following scale: Grade 1 = Mild (transient or mild discomfort; no limitation in activity; no medical intervention/therapy required); Grade 2 = Moderate (mild to moderate limitation in activity, some assistance may be needed; minimal medical intervention/therapy required); Grade 3 = Severe (marked limitation in activity, assistance usually required; medical intervention/therapy required, hospitalization possible); Grade 4 = Life-threatening (extreme limitation in activity, significant assistance or medical intervention/therapy required, hospitalization or hospice care probable); Grade 5 = Death Drug-related (related) AEs are those suspected by the Investigator as being related to administration of study drug (NCT01178281)
Timeframe: From the first dose of study drug until 28 days after last dose; median treatment duration was 23.7 weeks in the pomalidomide arm, 23.9 weeks in the placebo arm, and 24.0 weeks in the China extension pomalidomide arm.

Number of Patients With Objective Response (Complete and Partial Response + Hematological Improvement)

Intervention	Participants (Count of Participants)
	Any adverse event (AE)	Adverse event suspected as related to study drug	Adverse event leading to dose interruption	Drug-related AE leading to dose interruption	AE leading to discontinuation of study drug	Related AE leading to study drug discontinuation	Grade 3/4 adverse event	Grade 3/4 AE related to study drug	Grade 3/4 AE leading to study drug discontinuation	Grade 3/4 AE leading to dose interruption	Grade 5 adverse event	Grade 5 AE related to study drug	Serious adverse event (SAE)	SAE related to study drug	SAE leading to discontinuation of study drug	SAE leading to dose interruption
China Extension: Pomalidomide 0.5 mg	12	3	2	1	0	0	4	0	0	1	0	0	0	0	0	0
Placebo	81	32	17	6	14	8	44	13	9	14	10	3	29	7	8	7
Pomalidomide 0.5 mg	164	90	48	26	53	21	100	45	33	36	17	1	76	24	31	22

Time to response defined as the time from start of therapy until the response criteria are fulfilled. Response duration defined as the time from response until relapse (progressive disease) or death. (NCT00352794)
Timeframe: 6 months

Overall Response Rate

Intervention	Participants (Count of Participants)
Lenalidomide + Prednisone	14

"Response was evaluated for Anemia and Spleen:~Major anemia response: hemoglobin increase to within normal limits in the absence of transfusion. Minor anemia response: hemoglobin improvement of at least 2 grams per deciliter independent of transfusion support, or achievement of transfusion independence in transfusion-dependent patients. Major spleen response: normalization of spleen size to the range of 12-14 centimeters by ultrasound. Minor spleen response: a 50% or more decrease in excess spleen size by ultrasound. Complete remission (CR): complete resolution of disease-related symptoms, splenomegaly, normalization of peripheral blood count, white cell differential and smear, and normalization of bone marrow histology. Partial remission (PR): a major or minor response in anemia or splenomegaly. Overall Response (OR)=CR + PR, assessed among eligible, treated patients." (NCT00227591)
Timeframe: Assessed at the end of cycle 3

Article	Year
[Research and Applications Progress of Lenalidomide for Myelofibrosis--Review]. Zhongguo shi yan xue ye xue za zhi, 2018, Volume: 26, Issue:6 Topics: Humans; Lenalidomide; Prednisone; Primary Myelofibrosis; Quality of Life; Thalidomide	2018
Myelofibrosis: an update on current pharmacotherapy and future directions. Expert opinion on pharmacotherapy, 2013, Volume: 14, Issue:7 Topics: Age Factors; Androgens; Anemia; Humans; Immunologic Factors; Janus Kinase 2; Mutation; Nitriles; Pri	2013
Does anything work for anaemia in myelofibrosis? Best practice & research. Clinical haematology, 2014, Volume: 27, Issue:2 Topics: Androgens; Anemia; Blood Transfusion; Erythropoietin; Hematinics; Humans; Immunosuppressive Agents;	2014
[Treatment strategy for myeloproliferative neoplasms]. [Rinsho ketsueki] The Japanese journal of clinical hematology, 2014, Volume: 55, Issue:10 Topics: Adrenal Cortex Hormones; Aspirin; Calreticulin; Carbamates; Clinical Trials as Topic; Drug Therapy,	2014
Novel strategies for patients with chronic myeloproliferative disorders. Current opinion in hematology, 2009, Volume: 16, Issue:2 Topics: Chronic Disease; Humans; Interferon alpha-2; Interferon-alpha; Janus Kinase 2; Mutation; Myeloprolif	2009
[Advances in thalidomide therapy for idiopathic myelofibrosis]. Zhongguo yi xue ke xue yuan xue bao. Acta Academiae Medicinae Sinicae, 2009, Volume: 31, Issue:5 Topics: Angiogenesis Inhibitors; Humans; Immunosuppressive Agents; Primary Myelofibrosis; Thalidomide; Treat	2009
Pomalidomide: a new IMiD with remarkable activity in both multiple myeloma and myelofibrosis. American journal of hematology, 2010, Volume: 85, Issue:2 Topics: Cytokines; Humans; Immunologic Factors; Multiple Myeloma; Neovascularization, Pathologic; Primary My	2010
Thalidomide and lenalidomide in primary myelofibrosis. The Netherlands journal of medicine, 2010, Volume: 68, Issue:1 Topics: Angiogenesis Inhibitors; Antineoplastic Agents; Antineoplastic Agents, Hormonal; Hematopoiesis; Huma	2010
Pomalidomide therapy for multiple myeloma and myelofibrosis: an update. Leukemia & lymphoma, 2011, Volume: 52, Issue:4 Topics: Antineoplastic Agents; Boronic Acids; Bortezomib; Clinical Trials as Topic; Humans; Lenalidomide; Mu	2011
[IMiDs in hematology]. Bulletin du cancer, 2011, Volume: 98, Issue:8 Topics: Chronic Disease; Hematologic Neoplasms; Humans; Immunologic Factors; Lenalidomide; Leukemia, Lymphoi	2011
Treatment approaches in myelofibrosis with myeloid metaplasia: the old and the new. Seminars in hematology, 2003, Volume: 40, Issue:1 Suppl 1 Topics: Angiogenesis Inhibitors; Animals; Hematopoietic Stem Cell Transplantation; Humans; Palliative Care;	2003
Angiogenesis and anti-angiogenic therapy in myelofibrosis with myeloid metaplasia. Leukemia & lymphoma, 2004, Volume: 45, Issue:12 Topics: Angiogenesis Inhibitors; Animals; Cytokines; Humans; Neovascularization, Pathologic; Primary Myelofi	2004
[Thalidomide: mechanisms of action and new insights in hematology]. La Revue de medecine interne, 2005, Volume: 26, Issue:2 Topics: Amyloidosis; Angiogenesis Inhibitors; Clinical Trials as Topic; Cytokines; Follow-Up Studies; Foreca	2005
Modern management of myelofibrosis. British journal of haematology, 2005, Volume: 128, Issue:5 Topics: Adult; Angiogenesis Inhibitors; Anti-Inflammatory Agents; Humans; Middle Aged; Palliative Care; Pred	2005
Myelofibrosis: biology and treatment options. European journal of haematology. Supplementum, 2007, Issue:68 Topics: Benzamides; Chronic Disease; Darbepoetin alfa; Erythropoietin; Hematopoietic Stem Cell Transplantati	2007
[Pathophysiology of and therapy for primary myelofibrosis]. Nihon Naika Gakkai zasshi. The Journal of the Japanese Society of Internal Medicine, 2007, Aug-10, Volume: 96, Issue:8 Topics: Animals; Cytokines; Hematopoietic Stem Cell Transplantation; Humans; Janus Kinase 2; Mutation; Prima	2007
Prognostic factors and current practice in treatment of myelofibrosis with myeloid metaplasia: an update anno 2000. Pathologie-biologie, 2001, Volume: 49, Issue:2 Topics: Aged; Anemia; Aneuploidy; Angiogenesis Inhibitors; Bone Marrow Transplantation; Case Management; Com	2001

Article	Year
Thalidomide plus prednisone with or without danazol therapy in myelofibrosis: a retrospective analysis of incidence and durability of anemia response. Blood cancer journal, 2018, 01-15, Volume: 8, Issue:1 Topics: Adult; Aged; Anemia; Danazol; Female; Humans; Male; Middle Aged; Prednisolone; Primary Myelofibrosis	2018
Modest activity of pomalidomide in patients with myelofibrosis and significant anemia. Leukemia research, 2013, Volume: 37, Issue:11 Topics: Aged; Anemia; Angiogenesis Inhibitors; Antineoplastic Combined Chemotherapy Protocols; Female; Follo	2013
Use of the Functional Assessment of Cancer Therapy--anemia in persons with myeloproliferative neoplasm-associated myelofibrosis and anemia. Clinical therapeutics, 2014, Apr-01, Volume: 36, Issue:4 Topics: Anemia; Bayes Theorem; Double-Blind Method; Female; Glucocorticoids; Humans; Male; Myeloproliferativ	2014
Phase II study of pomalidomide in combination with prednisone in patients with myelofibrosis and significant anemia. Leukemia research, 2014, Volume: 38, Issue:9 Topics: Aged; Aged, 80 and over; Anemia; Drug Administration Schedule; Drug Therapy, Combination; Female; Hu	2014
Ruxolitinib in combination with lenalidomide as therapy for patients with myelofibrosis. Haematologica, 2015, Volume: 100, Issue:8 Topics: Adult; Aged; Aged, 80 and over; Antineoplastic Combined Chemotherapy Protocols; Bone Marrow; Chromos	2015
Long-term results of a phase II trial of lenalidomide plus prednisone therapy for patients with myelofibrosis. Leukemia research, 2016, Volume: 48 Topics: Adult; Aged; Aged, 80 and over; Antineoplastic Combined Chemotherapy Protocols; Female; Follow-Up St	2016
A randomized study of pomalidomide vs placebo in persons with myeloproliferative neoplasm-associated myelofibrosis and RBC-transfusion dependence. Leukemia, 2017, Volume: 31, Issue:4 Topics: Adult; Aged; Aged, 80 and over; Biomarkers; Erythrocyte Transfusion; Female; Humans; Immunologic Fac	2017
Pomalidomide in myeloproliferative neoplasm-associated myelofibrosis. Leukemia, 2017, Volume: 31, Issue:4 Topics: Aged; Aged, 80 and over; Alleles; Biomarkers; Chromosome Banding; Female; Humans; Immunologic Factor	2017
Low-dose thalidomide in myelofibrosis. Haematologica, 2008, Volume: 93, Issue:7 Topics: Aged; Aged, 80 and over; Blood Transfusion; Female; Humans; Immunosuppressive Agents; Male; Middle A	2008
Pomalidomide is active in the treatment of anemia associated with myelofibrosis. Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2009, Sep-20, Volume: 27, Issue:27 Topics: Adult; Aged; Aged, 80 and over; Anemia; Double-Blind Method; Female; Glucocorticoids; Hematinics; Hu	2009
Lenalidomide plus prednisone results in durable clinical, histopathologic, and molecular responses in patients with myelofibrosis. Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2009, Oct-01, Volume: 27, Issue:28 Topics: Adult; Aged; Aged, 80 and over; Anemia; Antineoplastic Agents; Drug Therapy, Combination; Enzyme-Lin	2009
Phase1/-2 study of Pomalidomide in myelofibrosis. American journal of hematology, 2010, Volume: 85, Issue:2 Topics: Adult; Aged; Aged, 80 and over; Anemia; Anti-Inflammatory Agents; Female; Humans; Immunosuppressive	2010
Lenalidomide and prednisone for myelofibrosis: Eastern Cooperative Oncology Group (ECOG) phase 2 trial E4903. Blood, 2010, Nov-25, Volume: 116, Issue:22 Topics: Antineoplastic Agents; Antineoplastic Combined Chemotherapy Protocols; Follow-Up Studies; Humans; Le	2010
A phase-2 trial of low-dose pomalidomide in myelofibrosis. Leukemia, 2011, Volume: 25, Issue:2 Topics: Aged; Aged, 80 and over; Anemia; Basophils; Erythrocyte Transfusion; Female; Humans; Janus Kinase 2;	2011
Prospective study of combination therapy with low-dose thalidomide plus prednisolone ameliorating cytopenia in primary myelofibrosis. International journal of hematology, 2011, Volume: 93, Issue:1 Topics: Adult; Aged; Angiogenesis Inhibitors; Antineoplastic Agents, Hormonal; Drug Therapy, Combination; Fe	2011
Circulating levels of MCP-1, sIL-2R, IL-15, and IL-8 predict anemia response to pomalidomide therapy in myelofibrosis. American journal of hematology, 2011, Volume: 86, Issue:4 Topics: Adult; Aged; Aged, 80 and over; Anemia; Chemokine CCL2; Cytokines; Female; Humans; Interleukin-15; I	2011
Comparison of thalidomide and lenalidomide as therapy for myelofibrosis. Blood, 2011, Jul-28, Volume: 118, Issue:4 Topics: Adult; Aged; Aged, 80 and over; Drug Therapy, Combination; Female; Humans; Immunosuppressive Agents;	2011
Long-term outcome of pomalidomide therapy in myelofibrosis. American journal of hematology, 2012, Volume: 87, Issue:1 Topics: Adult; Aged; Aged, 80 and over; Female; Humans; Male; Middle Aged; Primary Myelofibrosis; Prognosis;	2012
A phase 2 trial of combination therapy with thalidomide, arsenic trioxide, dexamethasone, and ascorbic acid (TADA) in patients with overlap myelodysplastic/myeloproliferative neoplasms (MDS/MPN) or primary myelofibrosis (PMF). Cancer, 2012, Aug-15, Volume: 118, Issue:16 Topics: Aged; Antineoplastic Combined Chemotherapy Protocols; Arsenic Trioxide; Arsenicals; Ascorbic Acid; D	2012
Chromosome 5q deletion is extremely rare in patients with myelofibrosis. Leukemia research, 2013, Volume: 37, Issue:5 Topics: Adult; Aged; Antineoplastic Agents; Chromosome Deletion; Chromosomes, Human, Pair 5; Female; Humans;	2013
Negligible clinical effects of thalidomide in patients with myelofibrosis with myeloid metaplasia. Medical oncology (Northwood, London, England), 2002, Volume: 19, Issue:2 Topics: Adult; Aged; Anemia; Blood Transfusion; Bone Marrow; Female; Humans; Leprostatic Agents; Male; Middl	2002
A phase 2 trial of combination low-dose thalidomide and prednisone for the treatment of myelofibrosis with myeloid metaplasia. Blood, 2003, Apr-01, Volume: 101, Issue:7 Topics: Adrenal Cortex Hormones; Aged; Anemia; Angiogenesis Inhibitors; Antineoplastic Agents, Hormonal; Ant	2003
Low-dose thalidomide ameliorates cytopenias and splenomegaly in myelofibrosis with myeloid metaplasia: a phase II trial. Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2004, Feb-01, Volume: 22, Issue:3 Topics: Adolescent; Adult; Aged; Aged, 80 and over; Anemia; Female; Humans; Immunosuppressive Agents; Leukop	2004
Thalidomide for the treatment of idiopathic myelofibrosis. European journal of haematology, 2004, Volume: 72, Issue:1 Topics: Aged; Blood Transfusion; Female; Humans; Magnetic Resonance Imaging; Male; Middle Aged; Palliative C	2004
Combined treatment with thalidomide, corticosteroids, and erythropoietin in patients with idiopathic myelofibrosis. European journal of haematology, 2005, Volume: 74, Issue:3 Topics: Adrenal Cortex Hormones; Child, Preschool; Drug Therapy, Combination; Erythropoietin; Female; Humans	2005
Thalidomide therapy for myelofibrosis with myeloid metaplasia. Cancer, 2006, May-01, Volume: 106, Issue:9 Topics: Adult; Aged; Aged, 80 and over; Angiogenesis Inhibitors; Female; Humans; Male; Middle Aged; Neovascu	2006
Lenalidomide therapy in myelofibrosis with myeloid metaplasia. Blood, 2006, Aug-15, Volume: 108, Issue:4 Topics: Administration, Oral; Adult; Aged; Anemia; Anemia, Myelophthisic; Female; Hemoglobins; Humans; Janus	2006
Thalidomide versus placebo in myeloid metaplasia with myelofibrosis: a prospective, randomized, double-blind, multicenter study. Haematologica, 2006, Volume: 91, Issue:8 Topics: Angiogenesis Inhibitors; Double-Blind Method; France; Humans; Placebos; Primary Myelofibrosis; Repro	2006
Thalidomide in agnogenic and secondary myelofibrosis. British journal of haematology, 2001, Volume: 115, Issue:2 Topics: Adult; Aged; Angiogenesis Inhibitors; Female; Follow-Up Studies; Hemoglobins; Humans; Immunosuppress	2001
Thalidomide treatment in myelofibrosis with myeloid metaplasia. British journal of haematology, 2002, Volume: 117, Issue:2 Topics: Adult; Aged; Drug Administration Schedule; Female; Hematopoiesis, Extramedullary; Humans; Immunosupp	2002

Article	Year
Ruxolitinib combined with prednisone, thalidomide and danazol in patients with myelofibrosis: Results of a pilot study. Hematological oncology, 2022, Volume: 40, Issue:4 Topics: Anemia; Danazol; Hemoglobins; Humans; Myeloproliferative Disorders; Nitriles; Pilot Projects; Predni	2022
Spliceosome mutations are common in persons with myeloproliferative neoplasm-associated myelofibrosis with RBC-transfusion-dependence and correlate with response to pomalidomide. Leukemia, 2021, Volume: 35, Issue:4 Topics: Disease Management; Disease Susceptibility; Erythrocyte Transfusion; Humans; Mutation; Myeloprolifer	2021
Retrospective Analysis of the Clinical Use and Benefit of Lenalidomide and Thalidomide in Myelofibrosis. Clinical lymphoma, myeloma & leukemia, 2020, Volume: 20, Issue:12 Topics: Aged; Anemia; Drug Therapy, Combination; Female; Humans; Lenalidomide; Male; Primary Myelofibrosis;	2020
The Treatment Landscape of Myelofibrosis Before and After Ruxolitinib Approval. Clinical lymphoma, myeloma & leukemia, 2017, Volume: 17, Issue:12 Topics: Aged; Drug Approval; Drug Therapy; Female; Hematinics; Humans; Immunosuppressive Agents; Male; Nitri	2017
Experience with lenalidomide in an Austrian non-study population with advanced myelofibrosis. Wiener klinische Wochenschrift, 2013, Volume: 125, Issue:7-8 Topics: Aged; Aged, 80 and over; Anemia; Austria; Dose-Response Relationship, Drug; Female; Humans; Immunolo	2013
Oral lesions in the course of myelofibrosis successfully treated using combination therapy with thalidomide, betamethasone, and cytarabine. Polskie Archiwum Medycyny Wewnetrznej, 2013, Volume: 123, Issue:4 Topics: Aged; Betamethasone; Candida albicans; Cytarabine; Drug Therapy, Combination; Humans; Male; Oral Ulc	2013
Acute myelofibrosis and acute lymphoblastic leukemia in an elderly patient with previously treated multiple myeloma. Annals of clinical and laboratory science, 2013,Spring, Volume: 43, Issue:2 Topics: Aged; Blood Cell Count; Bone Marrow; Humans; Immunohistochemistry; Lenalidomide; Maintenance Chemoth	2013
Struggling with myelofibrosis-associated anemia. Leukemia research, 2013, Volume: 37, Issue:11 Topics: Anemia; Angiogenesis Inhibitors; Antineoplastic Combined Chemotherapy Protocols; Female; Humans; Mal	2013
Thalidomide treatment in a myelofibrosis patient with leukemia transformation. International journal of hematology, 2014, Volume: 99, Issue:2 Topics: Adult; Angiogenesis Inhibitors; Bone Marrow; Cell Transformation, Neoplastic; Drug Resistance, Neopl	2014
Two cases of myelofibrosis with severe thrombocytopenia and symptomatology successfully treated with combination of pomalidomide and ruxolitinib. Leukemia & lymphoma, 2015, Volume: 56, Issue:2 Topics: Aged; Aged, 80 and over; Antineoplastic Combined Chemotherapy Protocols; Fatigue; Humans; Leukocyte	2015
[Comparison of low-dose thalidomide and prednisone combined with or without danazol for the treatment of primary myelofibrosis-associated anemia]. Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi, 2014, Volume: 35, Issue:8 Topics: Adult; Aged; Anemia; Danazol; Drug Therapy, Combination; Female; Follow-Up Studies; Humans; Male; Mi	2014
Ruxolitinib Treatment in a Patient with Primary Myelofibrosis Resistant to Conventional Therapies and Splenectomy: A Case Report. Turkish journal of haematology : official journal of Turkish Society of Haematology, 2015, Volume: 32, Issue:2 Topics: Aged; Aspergillosis; Blood Transfusion; Danazol; Disease Progression; Drug Resistance; Fatal Outcome	2015
Genes Involved in Maintaining the Bone Marrow Stroma Are Dysregulated in Patients with Myelofibrosis: Lenalidomide Treatment Up-regulates SOCS3. Anticancer research, 2015, Volume: 35, Issue:10 Topics: Aged; Aged, 80 and over; Antineoplastic Agents; Case-Control Studies; Clinical Trials, Phase II as T	2015
Human Immunodeficiency Virus Related Myelofibrosis Responsive to Low Dose Thalidomide. Indian journal of pediatrics, 2016, Volume: 83, Issue:9 Topics: Angiogenesis Inhibitors; HIV Infections; Humans; Primary Myelofibrosis; Thalidomide	2016
Erythema Nodosum-like Septal Panniculitis Secondary to Lenalidomide Therapy in a Patient With Janus Kinase 2-Positive Myelofibrosis. Journal of drugs in dermatology : JDD, 2016, Aug-01, Volume: 15, Issue:8 Topics: Aged; Erythema Nodosum; Humans; Immunologic Factors; Janus Kinase 2; Lenalidomide; Male; Panniculiti	2016
Responses to pomalidomide and placebo in myelofibrosis-related anaemia. Leukemia, 2017, Volume: 31, Issue:3 Topics: Anemia; Humans; Immunologic Factors; Primary Myelofibrosis; Thalidomide	2017
Toxic epidermal necrolysis in a patient with primary myelofibrosis receiving thalidomide therapy. International journal of hematology, 2009, Volume: 89, Issue:1 Topics: Aged; Humans; Male; Neovascularization, Pathologic; Primary Myelofibrosis; Stevens-Johnson Syndrome;	2009
Trisomy 13: prevalence and clinicopathologic correlates of another potentially lenalidomide-sensitive cytogenetic abnormality. Blood, 2009, Jan-29, Volume: 113, Issue:5 Topics: Antineoplastic Agents; Chromosomes, Human, Pair 13; Female; Humans; Lenalidomide; Leukemia, Myeloid,	2009
Thalidomide induced acute worsening of Parkinson's disease. Movement disorders : official journal of the Movement Disorder Society, 2009, Sep-15, Volume: 24, Issue:12 Topics: Aged; Humans; Immunosuppressive Agents; Male; Parkinson Disease; Primary Myelofibrosis; Thalidomide	2009
Treatment considerations for primary myelofibrosis. The Netherlands journal of medicine, 2010, Volume: 68, Issue:1 Topics: Anemia; Angiogenesis Inhibitors; Antineoplastic Agents; Humans; Lenalidomide; Leukemia, Myeloid, Chr	2010
International working group for myelofibrosis research and treatment response assessment and long-term follow-up of 50 myelofibrosis patients treated with thalidomide-prednisone based regimens. American journal of hematology, 2011, Volume: 86, Issue:1 Topics: Aged; Aged, 80 and over; Cohort Studies; Female; Humans; Male; Middle Aged; Prednisone; Primary Myel	2011
Perinephric extramedullary haematopoiesis in primary myelofibrosis. British journal of haematology, 2012, Volume: 157, Issue:2 Topics: Aged; Angiogenesis Inhibitors; Heart; Hematopoiesis, Extramedullary; Humans; Kidney; Liver; Male; Me	2012
Lenalidomide-induced elevated bilirubin. Journal of oncology pharmacy practice : official publication of the International Society of Oncology Pharmacy Practitioners, 2012, Volume: 18, Issue:4 Topics: Bilirubin; Humans; Lenalidomide; Male; Middle Aged; Polycythemia Vera; Primary Myelofibrosis; Thalid	2012
Resolution of post-polycythaemic myelofibrosis with a combination of thalidomide and interferon. Journal of clinical pathology, 2012, Volume: 65, Issue:8 Topics: Bone Marrow Examination; DNA Mutational Analysis; Drug Therapy, Combination; Female; Humans; Immunol	2012
Clinical efficacy and antiangiogenic activity of thalidomide in myelofibrosis with myeloid metaplasia. A pilot study. Leukemia, 2002, Volume: 16, Issue:9 Topics: Adult; Aged; Angiogenesis Inhibitors; Bone Marrow; Endothelial Growth Factors; Female; Fibroblast Gr	2002
Thalidomide: no benefit in myelofibrosis with myeloid metaplasia. The Lancet. Oncology, 2003, Volume: 4, Issue:2 Topics: Bone Marrow; Humans; Metaplasia; Primary Myelofibrosis; Thalidomide; Treatment Failure	2003
Thalidomide in myelofibrosis with myeloid metaplasia: a pooled-analysis of individual patient data from five studies. Leukemia & lymphoma, 2002, Volume: 43, Issue:12 Topics: Adult; Aged; Aged, 80 and over; Anemia; Clinical Trials, Phase II as Topic; Female; Hemoglobins; Hum	2002
Signet ring-like light chain myeloma with systemic spread. European journal of haematology, 2003, Volume: 70, Issue:4 Topics: Anemia; Antineoplastic Agents; Bone Marrow Examination; Combined Modality Therapy; Diagnostic Errors	2003
Sequential combination of thalidomide and erythropoietin determines transfusion independence and disease control in idiopathic myelofibrosis previously insensitive to both drugs used as single agents. Leukemia, 2003, Volume: 17, Issue:8 Topics: Blood Transfusion; Disease Management; Drug Resistance; Drug Synergism; Drug Therapy, Combination; E	2003
Myelofibrosis: thalidomide finds a new disease. Mayo Clinic proceedings, 2004, Volume: 79, Issue:7 Topics: Humans; Primary Myelofibrosis; Thalidomide	2004
Durable responses to thalidomide-based drug therapy for myelofibrosis with myeloid metaplasia. Mayo Clinic proceedings, 2004, Volume: 79, Issue:7 Topics: Adult; Aged; Drug Combinations; Female; Follow-Up Studies; Humans; Male; Middle Aged; Prednisone; Pr	2004
Hairy cell leukemia (HCL) with extensive myelofibrosis responds to thalidomide. Leukemia research, 2005, Volume: 29, Issue:8 Topics: Aged; Cladribine; Humans; Leukemia, Hairy Cell; Male; Primary Myelofibrosis; Remission Induction; Th	2005
Current treatment of myelofibrosis. Clinical advances in hematology & oncology : H&O, 2005, Volume: 3, Issue:4 Topics: Blood Transfusion; Bone Marrow Transplantation; Clinical Trials as Topic; Dexamethasone; Disease Man	2005
Endolymphatic hydrops as a cause of audio-vestibular manifestations in relapsing polychondritis. Acta oto-laryngologica, 2006, Volume: 126, Issue:5 Topics: Acute Disease; Audiometry, Evoked Response; Autoimmune Diseases; C-Reactive Protein; Diagnosis, Diff	2006
Neutrophilic dermatosis (Sweet syndrome) of the hands associated with lenalidomide. Archives of dermatology, 2006, Volume: 142, Issue:8 Topics: Aged; Diagnosis, Differential; Hand Dermatoses; Humans; Immunologic Factors; Lenalidomide; Male; Pri	2006
Thalidomide use and digital gangrene. Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2006, Nov-20, Volume: 24, Issue:33 Topics: Angiogenesis Inhibitors; Antineoplastic Agents; Chronic Disease; Debridement; Female; Fingers; Gangr	2006
Treatment of deletion 5q acute myeloid leukemia with lenalidomide. Leukemia, 2007, Volume: 21, Issue:3 Topics: Acute Disease; Antineoplastic Agents; Bone Marrow; Chromosome Deletion; Chromosomes, Human, Pair 5;	2007
Mini-dose of thalidomide for treatment of primary myelofibrosis. Report of a case with complete reversal of bone marrow fibrosis and splenomegaly. Haematologica, 2007, Volume: 92, Issue:2 Topics: Aged, 80 and over; Angiogenesis Inhibitors; Blood Transfusion; Bone Marrow; Combined Modality Therap	2007
Lenalidomide therapy in del(5)(q31)-associated myelofibrosis: cytogenetic and JAK2V617F molecular remissions. Leukemia, 2007, Volume: 21, Issue:8 Topics: Aged; Antineoplastic Agents; Chromosome Deletion; Chromosomes, Human, Pair 5; Cytogenetic Analysis;	2007
Thrombosis of the corpora cavernosa complicating lenalidomide therapy of myelofibrosis. Leukemia research, 2008, Volume: 32, Issue:5 Topics: Aged; Humans; Lenalidomide; Male; Penis; Primary Myelofibrosis; Thalidomide; Thrombosis	2008
Serious myeloproliferative reactions associated with the use of thalidomide in myelofibrosis with myeloid metaplasia. Blood, 2000, Dec-01, Volume: 96, Issue:12 Topics: Adult; Aged; Female; Humans; Leukocyte Count; Male; Middle Aged; Myeloproliferative Disorders; Pilot	2000
Thalidomide therapy in compensated and decompensated myelofibrosis with myeloid metaplasia. Haematologica, 2001, Volume: 86, Issue:7 Topics: Angiogenesis Inhibitors; Bone Marrow; Drug Evaluation; Humans; Neovascularization, Pathologic; Prima	2001
Safety and efficacy of thalidomide in patients with myelofibrosis with myeloid metaplasia. British journal of haematology, 2001, Volume: 114, Issue:1 Topics: Adult; Aged; Aged, 80 and over; Angiogenesis Inhibitors; Fatigue; Feasibility Studies; Female; Follo	2001
[Dynamic contrast-enhanced MRI for evaluating bone marrow microcirculation in malignant hematological diseases before and after thalidomide therapy]. Der Radiologe, 2002, Volume: 42, Issue:3 Topics: Adult; Aged; Angiogenesis Inhibitors; Bone Marrow; Contrast Media; Female; Gadolinium DTPA; Humans;	2002

thalidomide and Agnogenic Myeloid Metaplasia