International Working Group (IWG) consensus criteria for treatment response in myelofibrosis with myeloid metaplasia, for the IWG for Myelofibrosis Research and Treatment (IWG-MRT)

Tefferi, Ayalew; Barosi, Giovanni; Mesa, Ruben A.; Cervantes, Francisco; Deeg, H. Joachim; Reilly, John T.; Verstovšek, Srđan; Dupriez, Brigitte; Silver, Richard T.; Odenike, Olatoyosi; Cortes, Jorge; Wadleigh, Martha; Solberg, Lawrence A.; Camoriano, John; Gisslinger, Heinz; Noël, Pierre; Thiele, Jüergen; Vardiman, James W.; Hoffman, Ronald; Cross, Nicholas C. P.; Gilliland, D. Gary; Kantarjian, Hagop M.

doi:10.1182/blood-2006-03-009746

Cited by 291 publications

(240 citation statements)

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“…In a substantial proportion of patients, HU was effective in controlling the manifestations of MF, especially the hyperproliferative signs or symptoms, and, overall, 40% of the patients fulfilled the stringent IWG-MRT category of clinical improvement [16]. Median duration of the response exceeded one year, although there was a wide variability.…”

Section: Discussionmentioning

confidence: 94%

“…The present study included 40 MF patients who were uniformly treated at the same institution and in whom the response to treatment was assessed using modern criteria of response [15,16]. In a substantial proportion of patients, HU was effective in controlling the manifestations of MF, especially the hyperproliferative signs or symptoms, and, overall, 40% of the patients fulfilled the stringent IWG-MRT category of clinical improvement [16].…”

Section: Discussionmentioning

confidence: 99%

“…The response was evaluated using the criteria of the European Myelofibrosis Network (EUMNET) [15] and those of the International Working Group for Myelofibrosis research and Treatment (IWG-MRT) [16]. The minimum time to assess the response was established as 3 months.…”

Section: Amongmentioning

confidence: 99%

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Efficacy and tolerability of hydroxyurea in the treatment of the hyperproliferative manifestations of myelofibrosis: results in 40 patients

et al. 2010

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Hydroxyurea (HU) is frequently given as treatment for myelofibrosis (MF), but data on its efficacy and tolerability are scarce. The results of HU therapy were evaluated in 40 patients with hyperproliferative manifestations of primary (n= 32), post-polycythemia vera (n= 6) or post-essential thrombocythemia (n= 2) myelofibrosis. Median interval between diagnosis and HU start was 6.2 months (range: 0-141.7). Reasons for treatment were: constitutional symptoms (55%), symptomatic splenomegaly (45%), thrombocytosis (40%), leukocytosis (28%), pruritus (10%), and bone pain (8%). The starting dose was 500 mg/day, subsequently adjusted to the individual efficacy and tolerability.Response was: bone pain 100%, constitutional symptoms 82%, pruritus 50%, splenomegaly 40%, and anemia 12.5%. According to the International Working Group for Myelofibrosis Research and Treatment criteria, clinical improvement was achieved in 16 patients (40%). Median duration of response was 13.2 months (range: 3-126.2). Worsening of the anemia or appearance of pancytopenia were observed in 18 patients, requiring administration of erythropoietin-stimulating agents (n=17) and/or danazol (n=9). Oral or leg ulcers appeared in 5 patients and one had gastrointestinal symptoms. HU is an effective and generally well-tolerated therapy for the hyperproliferative manifestations of MF. The accentuation of the anemia often induced by HU is usually manageable with concomitant treatment.Response to Reviewers: Answers to the comments by reviewer 1:1. Retrospective design of the study. As already stated in the Introduction and in the Discussion, although there is limited bibliographic support for the use of hydroxyurea (HU) in MF, in clinical practice, this drug has been considered for many years (and still is) as the standard therapy for the hyperproliferative manifestations of this disease. Actually, until the recent development of the JAK2 inhibitors, no real alternative therapy to HU was available for comparison. As a result, for the time being, a retrospective study is the only possibility to assess the efficacy and tolerability of HU in MF.Having said this, it must be remarked that the data from the present study have been generated in a single institution, in which the drug was used following uniform rules for the starting dose, the dose adjustments and the use of adjuvant drugs for the treatment of the associated anemia. Moreover, more than 80% of the patients in the study were treated by the same physician, with long-lasting and specific dedication to MF.2. Selection of the patients. As stated in the Patients and Methods, the 40 patients included in the study were the only ones receiving HU among the 157 with MF followed in our institution during the study period. As also stated in the same section, HU was given to these 40 patients exclusively as a treatment for the hyperproliferative manifestations of MF (i.e., constitutional symptoms, symptomatic splenomegaly, pruritus, bone pain, leukocytosis, and thrombocytosis). This means that a patient ...

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Section: Discussionmentioning

confidence: 94%

Section: Discussionmentioning

confidence: 99%

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Efficacy and tolerability of hydroxyurea in the treatment of the hyperproliferative manifestations of myelofibrosis: results in 40 patients

et al. 2010

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“…Oral pomalidomide (0.5-3.5 mg/day) was administered daily in either 21-day or 28-day cycles. Response was assessed by the IWG-MRT criteria [11]. Statistical analyses were performed using Stat-View (SAS Institute, Cary, NC).…”

Section: Methodsmentioning

confidence: 99%

“…Lenalidomide (5-10 mg/day), with or without prednisone, also alleviated anemia in a similar proportion of patients with MF [3,8,9], and was most useful in the presence of del(5q) [10]. Two recent studies reassessed treatment response using the International Working Group for Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria [11]; one study reported up to 38% response rate for lenalidomide plus prednisone therapy [12] whereas the other study reported 28% response rate for thalidomide-based therapy [13]. However, in a prospective multi-center study [8], the combination of lenalidomide and prednisone resulted in only 19% anemia response and 23% overall response.…”

Section: Introductionmentioning

confidence: 99%

Long‐term outcome of pomalidomide therapy in myelofibrosis

et al. 2011

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Ninety-four Mayo Clinic patients with myelofibrosis (MF) participated in two consecutive clinical trials of pomalidomide (0.5-3.5 mg/day), with or without prednisone. Overall anemia response was 27% and increased to 53% in JAK2V617F-positive patients with <10 cm palpable splenomegaly and <5% circulating blasts; response rate was 0% in mutation-negative patients with either 10 cm splenomegaly or 5% circulating blasts (P 5 0.0001). Median duration of anemia response was 16 months. Treatment effect on splenomegaly was negligible. To date, pomalidomide therapy has been discontinued in 86 (91%) patients at a rate of 68% at 1 year and 89% at 2 years. Grade 1 sensory neuropathy developed in 4 (13%) of 30 patients treated for 1 year. Risk-adjusted survival in pomalidomide-treated primary MF patients (n 5 72) was similar to their counterparts not exposed to the drug (n 5 471; P 5 0.19). Long-term follow-up of pomalidomide treatment in MF reveals palliative value for a select group of patients and treatment-emergent sensory neuropathy. Am. J. Hematol. 87:66-68, 2012. V V C 2011 Wiley Periodicals, Inc. IntroductionPomalidomide is an immunomodulatory drug that is structurally related to both lenalidomide and thalidomide [1]. All three drugs display anti-angiogenic, anti-tumor necrosis factor-a, and T cell modulatory effect, but the precise mechanism of action in vivo is poorly understood. Thalidomide [2], lenalidomide [3], and pomalidomide [4] have all been shown to have therapeutic activity in myelofibrosis (MF), including primary (PMF), post-polycythemia vera (post-PV MF), and post-essential thrombocythemia (post-ET MF). Higher doses (100-400 mg/day) of thalidomide were associated with an increased adverse drop-out rate whereas low-dose (50 mg/day) thalidomide, alone or in combination with prednisone, was better tolerated and alleviated anemia in approximately 20% of treated patients with MF [5][6][7]. Lenalidomide (5-10 mg/day), with or without prednisone, also alleviated anemia in a similar proportion of patients with MF [3,8,9], and was most useful in the presence of del(5q) [10]. Two recent studies reassessed treatment response using the International Working Group for Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria [11]; one study reported up to 38% response rate for lenalidomide plus prednisone therapy [12] whereas the other study reported 28% response rate for thalidomide-based therapy [13]. However, in a prospective multi-center study [8], the combination of lenalidomide and prednisone resulted in only 19% anemia response and 23% overall response. Regardless, the use of thalidomide or lenalidomide in MF is limited by the respective occurrence of peripheral neuropathy [12] and moderate to severe myelosuppression [8], in the majority of treated patients.We have recently communicated the short-term results of two consecutive clinical trials using pomalidomide therapy for MF [4,14,15]. The maximum tolerated dose of pomalidomide in MF was 3 mg/day (dose-limiting toxicity was myelosuppression) but...

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Philadelphia Chromosome‐Negative Myeloproliferative Neoplasms

Mesa

Verstovšek

2010

Leukemias: Principles and Practice of Therapy

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International Working Group (IWG) consensus criteria for treatment response in myelofibrosis with myeloid metaplasia, for the IWG for Myelofibrosis Research and Treatment (IWG-MRT)

Cited by 291 publications

References 55 publications

Efficacy and tolerability of hydroxyurea in the treatment of the hyperproliferative manifestations of myelofibrosis: results in 40 patients

Efficacy and tolerability of hydroxyurea in the treatment of the hyperproliferative manifestations of myelofibrosis: results in 40 patients

Long‐term outcome of pomalidomide therapy in myelofibrosis

Philadelphia Chromosome‐Negative Myeloproliferative Neoplasms

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