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Unmet clinical need...
Unmet clinical needs in the management of CALR-mutated essential thrombocythaemia : a consensus-based proposal from the European LeukemiaNet
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- Alvarez-Larran, Alberto (author)
- Inst Invest Biomed August Pi & Sunyer IDIBAP, Hosp Clin, Hematol Dept, Barcelona, Spain.
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- Sant'Antonio, Emanuela (author)
- Univ Hosp Paolo Giaccone, Hematol Div, Palermo, Italy.
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- Harrison, Claire (author)
- Guys & StThomasNHS Fdn Trust, Dept Haematol, London, England.
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- Kiladjian, Jean-Jacques (author)
- Univ Paris, Ctr Invest Clin, Hop St Louis, AP HP,INSERM,CIC1427, Paris, France.
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- Griesshammer, Martin (author)
- Univ Bochum, Johannes Westing Med Ctr Minden UKRUB, Oncol Hemostaseol & Palliat Care, Bochum, Germany.
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- Mesa, Ruben (author)
- UT Hlth San Antonio MD Anderson, Mays Canc Ctr, San Antonio, TX USA.
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- Ianotto, Jean Christophe (author)
- CHRU Brest, Serv Hematol Clin, Inst Cancerol Hematol, Brest, France.
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- Palandri, Francesca (author)
- Univ Bologna, Ist Ematol Seragnor, IRCCS Azienda Osped, Bologna, Italy.
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- Hernandez-Boluda, Juan Carlos (author)
- Hosp Clin Valencia, Hematol Dept, Valencia, Spain.
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- Birgegård, Gunnar, 1944- (author)
- Uppsala universitet,Hematologi
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- Nangalia, Jyoti (author)
- Wellcome Sanger Inst, Cambridge, England.
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- Koschmieder, Steffen (author)
- Rhein Westfal TH Aachen, Fac Med, Dept Hematol Oncol Hemostaseol & Stem Cell Transp, Aachen, Germany.
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- Rumi, Elisa (author)
- Univ Pavia, Dept Mol Med, Pavia, Italy.
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- Barbui, Tiziano (author)
- Res Fdn Papa Giovanni XXIII Hosp, I-24127 Bergamo, Italy.
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Inst Invest Biomed August Pi & Sunyer IDIBAP, Hosp Clin, Hematol Dept, Barcelona, Spain Univ Hosp Paolo Giaccone, Hematol Div, Palermo, Italy. (creator_code:org_t)
- Elsevier, 2021
- 2021
- English.
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In: The Lancet Haematology. - : Elsevier. - 2352-3026. ; 8:9, s. E658-E665
- Related links:
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https://urn.kb.se/re...
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https://doi.org/10.1...
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Abstract
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- Recommendations regarding management of essential thrombocythaemia rely on studies done before the discovery of the CALR mutation. On May 20, 2020, the European LeukemiaNet annual meeting was held with the goal to identify unmet clinical needs in myeloproliferative neoplasms. Because patients with a CALR mutation have specific clinical characteristics, treatment of CALR-mutated essential thrombocythaemia was considered an unmet clinical need by the European LeukemiaNet. The elaboration of a consensus document with recommendations according to current evidence was proposed as a solution for resolving uncertainties in the treatment of CALR-mutated essential thrombocythaemia. A steering committee comprising four European LeukemiaNet members was then formed and a panel of ten experts in the field was recruited. The experts proposed 51 potential unmet clinical needs in the management of CALR-mutated essential thrombocythaemia and were asked to score the relevance of each topic. Those topics that obtained the highest scores as relevant unmet clinical needs were identified, including antiplatelet therapy in patients at low risk, definition of extreme thrombocytosis and its management in patients at low risk, indications of cytoreduction and targets of therapy, first-line treatment of choice in young patients (<60 years), and management of pregnancy. After the steering committee revised the available evidence for each topic, a consensus on management and proposal for improving knowledge was achieved by use of an email-based, two round, Delphi approach. Consensus was achieved when 90% of the panellists agreed with a statement and included 14 recommendations and six solution proposals. Key recommendations included careful observation for asymptomatic patients with classical, low-risk, CALR-mutated essential thrombocythaemia without cardiovascular risk factors; caution in the use of antiplatelet therapy for symptomatic patients at low risk with platelet counts of 1000-1500 × 109 platelets per L, in such cases cytoreduction is an adequate option, especially if adquired Von Willebrand disease is present; cytoreduction is recommended for extreme thrombocytosis (platelet count >1500 × 109 platelets per L) with pegylated interferon alfa being the preferred option for younger patients; both hydroxycarbamide and anagrelide might be given to patients ineligible for pegylated interferon alfa; and treatment algorithms for patients with high-risk pregnancies should not be changed according to genotype. The European LeukemiaNet proposes to use these recommendations in the routine management of patients with CALR-mutated essential thrombocythaemia, and designing new clinical studies in this field might be useful.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Hematologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Hematology (hsv//eng)
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Alvarez-Larran, ...
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Sant'Antonio, Em ...
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Harrison, Claire
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Kiladjian, Jean- ...
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Griesshammer, Ma ...
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Mesa, Ruben
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Ianotto, Jean Ch ...
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Palandri, France ...
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Hernandez-Boluda ...
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Birgegård, Gunna ...
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Nangalia, Jyoti
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Koschmieder, Ste ...
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Rumi, Elisa
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Barbui, Tiziano
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Hematology
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The Lancet Haema ...
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Uppsala University