Abstract
Patients (pts) with polycythemia vera (PV) suffer from pruritus, night sweats, and other symptoms, as well as from thromboembolic complications and progression to post-PV myelofibrosis. Ruxolitinib (RUX) is approved for second-line therapy in high-risk PV pts with hydroxyurea intolerance or resistance. The RuxoBEAT trial (NCT02577926, registered on October 1, 2015, at clinicaltrials.gov) is a multicenter, open-label, two-arm phase-IIb trial with a target population of 380 pts with PV or ET, randomized to receive RUX or best available therapy. This pre-specified futility analysis assesses the early clinical benefit and tolerability of RUX in previously untreated PV pts (6-week cytoreduction was allowed). Twenty-eight patients were randomly assigned to receive RUX. Compared to baseline, after 6 months of treatment, there was a significant reduction of median hematocrit (46 to 41%), the median number of phlebotomies per year (4.0 to 0), and median patient-reported pruritus scores (2 to 1), and a trend for reduced night sweat scores (1.5 to 0). JAK2V617F allele burden, as part of the scientific research program, also significantly decreased. One hundred nine adverse events (AEs) occurred in 24/28 patients (all grade 1 to 3), and no pt permanently discontinued treatment because of AEs. Thus, treatment with ruxolitinib in untreated PV pts is feasible, well-tolerated, and efficient regarding the above-mentioned endpoints.
| Original language | English |
|---|---|
| Journal | Annals of Hematology |
| Volume | 102 |
| Issue number | 2 |
| Pages (from-to) | 349-358 |
| Number of pages | 10 |
| ISSN | 0939-5555 |
| DOIs | |
| Publication status | Published - 02.2023 |
Funding
We thank Kim Kricheldorf for support with the GSG-MPN bioregistry coordination, Carmen Fera, Michelle Haaße, Yvonne Schumacher, Amrei Pelzer, Christina Grohe, Irina Prell, Verena Deserno, and Rainer Schuckelt for support at the Center for Translational and Clinical Research Aachen (CTC-A), Kristina Pannen (née Feldberg) for logistic support with samples, and Britta Wagner for excellent secretarial assistance. We thank our colleagues and study staff at all RuxoBEAT study centers in Germany and our patients for participation in this trial. Additional centers participating in this study include Aschaffenburg (Martine Klausmann), Berlin (Philip LeCoutre), Chemnitz (Mathias Hänel), Düsseldorf University Hospital (Norbert Gattermann), Essen (Joachim R. Göthert), Mainz (Thomas Kindler), Mannheim (Andreas Reiter), Münster (Eva Eßeling), Nürnberg (Marinela Augustin), Duisburg (Sebastian Balleisen), Halle (Haifa Kathrin Al-Ali), Düsseldorf Marien-Hospital (Aristoteles Giagounidis), Siegburg (Stefan Fronhoffs), and Cologne (Christof Scheid). We thank Novartis for the financial support of the clinical trial and for providing the study medication. We thank the Deutsche Forschungsgemeinschaft (DFG) for supporting concomitant basic research associated with this entire trial within the Clinical Research Unit CRU344 (KO 2155/7-1, BR 1782/5-1). We thank Kim Kricheldorf for support with the GSG-MPN bioregistry coordination, Carmen Fera, Michelle Haaße, Yvonne Schumacher, Amrei Pelzer, Christina Grohe, Irina Prell, Verena Deserno, and Rainer Schuckelt for support at the Center for Translational and Clinical Research Aachen (CTC-A), Kristina Pannen (née Feldberg) for logistic support with samples, and Britta Wagner for excellent secretarial assistance. We thank our colleagues and study staff at all RuxoBEAT study centers in Germany and our patients for participation in this trial. Additional centers participating in this study include Aschaffenburg (Martine Klausmann), Berlin (Philip LeCoutre), Chemnitz (Mathias Hänel), Düsseldorf University Hospital (Norbert Gattermann), Essen (Joachim R. Göthert), Mainz (Thomas Kindler), Mannheim (Andreas Reiter), Münster (Eva Eßeling), Nürnberg (Marinela Augustin), Duisburg (Sebastian Balleisen), Halle (Haifa Kathrin Al-Ali), Düsseldorf Marien-Hospital (Aristoteles Giagounidis), Siegburg (Stefan Fronhoffs), and Cologne (Christof Scheid). We thank Novartis for the financial support of the clinical trial and for providing the study medication. We thank the Deutsche Forschungsgemeinschaft (DFG) for supporting concomitant basic research associated with this entire trial within the Clinical Research Unit CRU344 (KO 2155/7-1, BR 1782/5-1).
Research Areas and Centers
- Research Area: Luebeck Integrated Oncology Network (LION)
- Centers: University Cancer Center Schleswig-Holstein (UCCSH)
