NCCN Guidelines Insights: Myeloproliferative Neoplasms, Version 2.2018

Mesa, Ruben A.; Jamieson, Catriona; Bhatia, Ravi; Deininger, Michael W.; Fletcher, Christopher D.�M.; Gerds, Aaron T.; Gojo, Ivana; Gotlib, Jason; Gundabolu, Krishna; Hobbs, Gabriela; McMahon, Brandon; Mohan, Sanjay; Oh, Stephen T.; Padron, Eric; Papadantonakis, Nikolaos; Pancari, Philip; Podoltsev, Nikolai A.; Rampal, Raajit K.; Ranheim, Erik A.; Reddy, V.; Rein, Lindsay; Scott, Bart L.; Snyder, David S.; Stein, Brady L.; Talpaz, Moshe; Verstovšek, Srđan; Wadleigh, Martha; Wang, Eunice S.; Bergman, Mary Anne; Gregory, Kristina M.; Sundar, Hema

doi:10.6004/jnccn.2017.0157

Cited by 127 publications

(115 citation statements)

References 60 publications

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“…1 Treatment goals comprise prevention of thrombohemorrhagic events (THEs), progression to myelofibrosis (MF) or acute leukemia (AL), and the onset of secondary malignancies. Many guidelines recommend antiplatelet and cytoreduction therapy for patients who are at high risk of thrombosis [2][3][4] ; in particular, the recently revised European LeukemiaNet (ELN) recommendations that recommend hydroxyurea and interferon-α as first-line therapies for cytoreduction therapies. If hydroxyurea is ineffective or cannot be tolerated, anagrelide and interferon-α are recommended as secondline therapies.…”

Section: Introductionmentioning

confidence: 99%

Efficacy and safety of anagrelide as a first‐line drug in cytoreductive treatment‐naïve essential thrombocythemia patients in a real‐world setting

Ito

Hashimoto

Tanaka

et al. 2019

European J of Haematology

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Objective: This study aimed to retrospectively assess the efficacy and safety of anagrelide in cytoreduction therapy-naïve essential thrombocythemia (ET) patients in a real-world setting.Method: Data from 53 ET patients who received anagrelide as a first-line therapy were reviewed for patient characteristics, antiplatelet status, cytoreduction status, therapeutic effects, adverse events, thrombohemorrhagic event development, progression to myelofibrosis or acute leukemia, and cause of death. Results:The rate of achieving a platelet count of <600 × 10 9 /L during anagrelide monotherapy was 83.0%. Adverse events occurred in 32 of 53 patients, and tended to be slightly more severe in patients with cardiac failure; however, they were mostly tolerable. The therapeutic effect of anagrelide was consistent, regardless of genetic mutation profiles. The incidence of anemia as an adverse event was significantly higher in the CALR mutation-positive group. Favorable platelet counts were also achieved in patients for whom hydroxyurea was introduced as a replacement for anagrelide or in addition to anagrelide because of unresponsiveness or intolerance to treatment. Conclusion:In Japanese cytoreduction therapy-naïve ET patients, anagrelide administration as a first-line therapy demonstrated favorable effects in reducing platelet counts, and its safety profile that was generally consistent with those in previous reports. K E Y W O R D S anagrelide, essential thrombocythemia, first-line, treatment | 117 ITO eT al. Complete response, n (%) 27 (50.9) Partial response, n (%) 18 (34.0) No response, n (%) 8 (15.1) AEs (≥5% of patients) Palpitations, n (%) 14 (26.4) Headache, n (%) 11 (20.8) Anemia, n (%) 10 (18.9) Diarrhea, n (%) 4 (7.5) Cardiac failure, n (%) 3 (5.7) AEs (grade 3) Anemia, n (%) 2 (3.8) Cardiac failure, n (%) 2 (3.8) Abbreviations: AE: adverse event; SD: standard deviation.

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Section: Introductionmentioning

confidence: 99%

Efficacy and safety of anagrelide as a first‐line drug in cytoreductive treatment‐naïve essential thrombocythemia patients in a real‐world setting

Ito

Hashimoto

Tanaka

et al. 2019

European J of Haematology

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“…5 Both NCCN and the European LeukemiaNet (ELN) guidelines recommend cytoreductive therapy with hydroxyurea (HU) as frontline therapy for patients with high-risk ET. 6,7 HU is an oral chemotherapeutic agent that inhibits ribonucleotide reductase and interferes with the process of DNA synthesis and repair. 8 Three randomized trials have evaluated the effect of HU among patients with high-risk ET.…”

Section: Introductionmentioning

confidence: 99%

“…11 Current guidelines recommend anagrelide (if not previously used as initial therapy) as a second-line treatment for patients refractory to or intolerant of HU. 6,7 To date, the impact of HU on thrombosis and survival among patients with high-risk ET has rarely been evaluated outside of clinical trials, and some experts have expressed concern about the safety and efficacy of HU in the real-world setting. 12 To address these knowledge gaps, a large, population-based, nationally representative cohort study was conducted to assess the impact of HU on survival and thrombosis risk among older patients with ET.…”

Section: Introductionmentioning

confidence: 99%

Impact of Hydroxyurea on Survival and Risk of Thrombosis Among Older Patients With Essential Thrombocythemia

Podoltsev

Zhu²,

Zeidan

et al. 2019

Journal of the National Comprehensive Cancer Network

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Background: Current guidelines recommend hydroxyurea (HU) as frontline therapy for patients with high-risk essential thrombocythemia (ET) to prevent thrombosis. However, little is known about the impact of HU on thrombosis or survival among these patients in the real-world setting. Patients and Methods: A retrospective cohort study was conducted of older adults (aged $66 years) diagnosed with ET from 2007 through 2013 using the linked SEER-Medicare database. Multivariable Cox proportional hazards regression models were used to assess the effect of HU on overall survival, and multivariable competing risk models were used to assess the effect of HU on the occurrence of thrombotic events. Results: Of 1,010 patients, 745 (73.8%) received HU. Treatment with HU was associated with a significantly lower risk of death (hazard ratio [HR], 0.52; 95% CI, 0.43-0.64; P,.01). Every 10% increase in HU proportion of days covered was associated with a 12% decreased risk of death (HR, 0.88; 95% CI, 0.86-0.91; P,.01). Compared with nonusers, HU users also had a significantly lower risk of thrombotic events (HR, 0.51; 95% CI, 0.41-0.64; P,.01). Conclusions: Although underused in our study population, HU was associated with a reduced incidence of thrombotic events and improved overall survival in older patients with ET.

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“…Recent translational and clinical experience with immuno‐oncology therapies have generated excitement, and there is a real risk that for the present, overshadow other potential investigational drug development efforts. In this regard, the development of drug resistance and clonal evolution, well documented for TKIs and ruxolitinib, should underscore the potential value of combining different molecular therapy approaches . The evolving story of SM and updated views on its biology and KIT ‐targeted therapy suggest the importance of the rationally designed midostaurin and the emergence of other candidate agents.…”

Section: Future Prospectsmentioning

confidence: 99%

“…In this regard, the development of drug resistance and clonal evolution, well documented for TKIs and ruxolitinib, should underscore the potential value of combining different molecular therapy approaches. 104 The evolving story of SM and updated views on its biology and KIT-targeted therapy suggest the importance of the rationally designed midostaurin and the emergence of other candidate agents.…”

Section: Future Prospectsmentioning

confidence: 99%

Emerging translational science discoveries, clonal approaches, and treatment trends in chronic myeloproliferative neoplasms

Mughal

Pemmaraju

Radich

et al. 2019

Hematological Oncology

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The 60th American Society of Hematology (ASH) held in San Diego in December 2018 was followed by the 13th Post‐ASH chronic myeloproliferative neoplasms (MPNs) workshop on December 4 and 5, 2018. This closed annual workshop, first introduced in 2006 by Goldman and Mughal, was organized in collaboration with Alpine Oncology Foundation and allowed experts in preclinical and clinical research in the chronic MPNs to discuss the current scenario, including relevant presentations at ASH, and address pivotal open questions that impact translational research and clinical management. This review is based on the presentations and deliberations at this workshop, and rather than provide a resume of the proceedings, we have selected some of the important translational science and treatment issues that require clarity. We discuss the experimental and observational evidence to support the intimate interaction between aging, inflammation, and clonal evolution of MPNs, the clinical impact of the unfolding mutational landscape on the emerging targets and treatment of MPNs, new methods to detect clonal heterogeneity, the challenges in managing childhood and adolescent MPN, and reflect on the treatment of systemic mastocytosis (SM) following the licensing of midostaurin.

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NCCN Guidelines Insights: Myeloproliferative Neoplasms, Version 2.2018

Cited by 127 publications

References 60 publications

Efficacy and safety of anagrelide as a first‐line drug in cytoreductive treatment‐naïve essential thrombocythemia patients in a real‐world setting

Efficacy and safety of anagrelide as a first‐line drug in cytoreductive treatment‐naïve essential thrombocythemia patients in a real‐world setting

Impact of Hydroxyurea on Survival and Risk of Thrombosis Among Older Patients With Essential Thrombocythemia

Emerging translational science discoveries, clonal approaches, and treatment trends in chronic myeloproliferative neoplasms

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