The Ruxo-BEAT Trial in Patients With High-risk Polycythemia Vera or High-risk Essential Thrombocythemia

Summary

The Philadelphia chromosome negative myeloproliferative neoplasms (MPN) comprise a group of clonal hematological malignancies that are characterized by chronic myeloproliferation, splenomegaly, different degrees of bone marrow fibrosis, and disease-related symptoms including pruritus, night sweats, fever, weight loss, cachexia, and diarrhea. In addition, due to elevated numbers of leucocytes, erythrocytes and/or platelets, the disease course can be complicated by thromboembolic disease, hemorrhage, and leukemic transformation as well as myelofibrosis. Patients with polycythemia vera (PV) typically harbor an increased number of blood cells from all three hematopoietic cell lineages due to clonal amplification of hematopoetic stem cells, while patients with essential thrombocythemia (ET) typically show a predominant expansion of the megakaryocytic lineage. Most patients with PV below the age of 60 years are currently being treated with acetylsalicylic acid +/- phlebotomy only, and patients with low-risk ET have an almost normal life expectancy and often do not require specific treatment. However, PV- as well as ET-patients with a higher risk for complications require cytoreductive treatment. In addition, constitutional symptoms can be unbearable to patients even in the absence of bona fide high risk factors, and these patients may similarly benefit from antineoplastic therapy.

Timeline

Start

2015-10

Primary completion

2027-12

Completion

2028-12

Publications

• Isfort S, Manz K, Teichmann LL, Crysandt M, Burchert A, Hochhaus A, Saussele S, Kiani A, Gothert JR, Illmer T, Schafhausen P, Al-Ali HK, Stegelmann F, Hanel M, Pfeiffer T, Giagounidis A, Franke GN, Koschmieder S, Fabarius A, Ernst T, Warnken-Uhlich M, Wolber U, Kohn D, Pfirrmann M, Wolf D, Brummendorf TH; German CML study group. Step-in dosing of bosutinib in pts with chronic phase chronic myeloid leukemia (CML) after second-generation tyrosine kinase inhibitor (TKI) therapy: results of the Bosutinib Dose Optimization (BODO) Study. Ann Hematol. 2023 Oct;102(10):2741-2752. doi: 10.1007/s00277-023-05394-0. Epub 2023 Aug 18.
• Koschmieder S, Isfort S, Wolf D, Heidel FH, Hochhaus A, Schafhausen P, Griesshammer M, Wolleschak D, Platzbecker U, Dohner K, Jost PJ, Parmentier S, Schaich M, von Bubnoff N, Stegelmann F, Maurer A, Crysandt M, Gezer D, Kortmann M, Franklin J, Frank J, Hellmich M, Brummendorf TH; German Study Group for Myeloproliferative Neoplasms (GSG-MPN). Efficacy and safety of ruxolitinib in patients with newly-diagnosed polycythemia vera: futility analysis of the RuxoBEAT clinical trial of the GSG-MPN study group. Ann Hematol. 2023 Feb;102(2):349-358. doi: 10.1007/s00277-022-05080-7. Epub 2022 Dec 23.

Drugs

Indications