Special Issues in Myeloproliferative Neoplasms
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Special issues in myeloproliferative neoplasms (MPN) comprise clinical conditions with high relevance for the duration and quality of the patient’s life, but with limited evidence to support sound diagnostic and therapeutic recommendations and a low probability of being solved by the current standard of clinical research. These issues include MPN in pregnancy and in children, abdominal vein thrombosis, bleeding complications, surgery, pruritus, and leukemic transformation. Practical suggestions to guide clinical decisions in these settings remain largely empirical, but recently developed guidelines based on experts’ consensus may help to tackle these problems. This article reviews the state of the art regarding these issues, with special emphasis on experts’ consensus recommendations.
KeywordsChronic myeloproliferative neoplasms MPN Polycythemia vera Essential thrombocythemia Primary myelofibrosis Pregnancy Pediatric patients Children Splanchnic vein thrombosis Bleeding Surgery Pruritus Leukemic transformation Hydroxyurea
Conflicts of Interest: T. Barbui: Honoraria, speaking fees, and travel expenses from Shire; G. Finazzi: none.
Papers of particular interest, published recently, have been highlighted as: • Of importance
- 1.• Barbui T, Barosi G, Birgegard G, et al.: Philadelphia-negative classical myeloproliferative neoplasms: critical concepts and management recommendations from European LeukemiaNet. J Clin Oncol 2011 (in press). These are the most up-to-date management recommendations for Philadelphia-negative classic myeloproliferative neoplasms, based on the results of a systematic consensus process by a panel of 21 experts appointed by the European LeukemiaNet. Google Scholar
- 4.Barbui T, Barosi G, Grossi A, et al.: Practice guidelines for the therapy of essential thrombocythemia. A statement from the Italian Society of Hematology, the Italian Society of Experimental Hematology and the Italian Group for Bone Marrow Transplantation. Haematologica 2004; 89:215–32.PubMedGoogle Scholar
- 9.• Harrison CN, Bareford D, Butt N, et al.: Guideline for investigation and management of adults and children presenting with a thrombocytosis. Br J Haematol 2010; 149:352-375. This recent and comprehensive guideline produced by the British Committee for Standards in Haematology provides health care professionals with clear guidance on the investigation and management of thrombocytosis in both adult and pediatric patients. CrossRefPubMedGoogle Scholar
- 31.Tefferi A, Mesa RA, Nagorney DM, et al.: Splenectomy in myelofibrosis with myeloid metaplasia: a single-institution experience with 223 patients. Blood 2000; 95:226–33.Google Scholar
- 34.• Verstovsek S, Kantarjian H, Mesa RA, et al.: Safety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosis. N Engl J Med 2010; 363:1117-1127. The first phase I/II clinical trial of a selective JAK2 inhibitor published in full, showing marked and durable clinical benefits in patients with myelofibrosis. CrossRefPubMedGoogle Scholar
- 35.Vannucchi AM, Guglielmelli P, Gattoni E, et al.: RAD001, an inhibitor of mTOR, shows clinical activity in a phase I/II study in patients with primary myelofibrosis (PMF) and post polycythemia vera/essential thrombocythemia myelofibrosis (PPV/PET MF). [abstract 307]. Presented at the 51th American Society of Hematology Congress, New Orleans, Louisiana, December 5–8, 2009.Google Scholar
- 37.Landaw SA. Acute leukemia in polycythemia vera. In Polycythyemia Vera and the Myeloproliferative Disorders. Edited by Wasserman LR, Berk PD, Berlin NI. Philadelhia: WP Saunders Co. 1995:154-165.Google Scholar
- 43.Bjorkholm M, Derolf AR, Ekstrand C, et al.: Clinical risk for AML/MDS transformation in Philadelphia negative chronic myeloproliferative neoplasms. A population-based nested case-control study in Sweden [abstract 1085]. Presented at the 14th European Hematology Association Congress, Berlin, June 4–7, 2009.Google Scholar
- 45.Kiladjian JJ, Chevret S, Dosquet C, et al.: Long-term outcome in polycythemia vera: final analysis of a randomized trial comparing hydroxyurea (HU) to Pipobroman (Pi). [abstract 1746]. Presented at the 50th American Society of Hematology Congress, San Francisco, California, December 6–9, 2008.Google Scholar
- 47.Tefferi A, Thiele J, Orazi A, et al.: Proposals and rationale for revision of the World Health Organization diagnostic criteria for polycythemia vera, essential thrombocythemia, and primary myelofibrosis: recommendations from an ad hoc international expert panel. Blood 2007; 110:1092–1097.CrossRefPubMedGoogle Scholar