ASH Clinical News September 2017 New | Page 37

CLINICAL NEWS
Over a median follow-up of 2.61 years ( range = 0.23-4.12 years ), ruxolitinibtreated patients received a median dose of 19 mg twice-daily ( range not provided ), and the most commonly used treatments in the BAT arm were hydroxycarbamide ( n = 37 ), anagrelide ( n = 25 ), and interferon ( n = 21 ).
Thirty-five patients in the ruxolitinib group discontinued treatment , compared with nine patients in the BAT group . “ However , 30 BAT patients switched their
initially assigned BAT treatment for various reasons , which indicates a similar rate of treatment ineffectiveness or intolerance ,” the authors wrote .
Rates of complete response ( CR ; primary outcome ) at two-year follow-up were similar in the ruxolitinib and BAT groups ( 27 patients [ 46.5 %] vs . 23 patients [ 44.2 %], respectively ; p = 0.4 ). Rates of partial response ( PR ) were also similar between the two treatment groups : 27 ( 46.5 %) versus 27
( 51.9 %), respectively . No baseline factors ( including hydroxycarbamide resistance , platelet counts , or JAK2 status ) influenced response rates in multivariate analyses .
Thirteen patients in the study transformed to post-ET myelofibrosis : eight in the ruxolitinib group and five in the BAT group . One ruxolitinib-treated patient transformed to acute myeloid leukemia ( AML ), and , again , transformation-free probability was not significantly different
between the two arms ( p = 0.29 ).
Ruxolitinib treatment appeared to reduce patients ’ symptom burden , according to an analysis of 85 patients ( 47 in the ruxolitinib group and 38 in the BAT group ) who completed a total symptom score ( TSS ) assessment at baseline and at least once after baseline .
The overall symptom response rate ( defined as a 50 % reduction in TSS during the first 12 months of treatment ) did not differ significantly between the ruxolitinib and BAT groups ( 12 [ 29 %] vs . 6 [ 19 %], respectively ; p = 0.37 ), but the magnitude of symptom reduction appeared to be greater in the ruxolitinib group ( median reduction in TSS = 32 % vs . 0 %; p = 0.03 ). Symptom response was “ rapid ” in the ruxolitinib arm , occurring within two months in eight patients ( 19 %), compared with in one patient ( 3 %) in the BAT group ( p = 0.04 ).
In the safety analysis , which included all 115 patients in the MAJIC-ET trial who received ruxolitinib or BAT , 128 grade 3 / 4 adverse events ( AEs ) occurred in 89 patients , including :
• grade 3 / 4 anemia : 12 patients ( 21 %) in the ruxolitinib arm versus 0 in the BAT arm ( p < 0.005 )
• grade 3 / 4 thrombocytopenia : 2 patients ( 3.4 %) versus 0 ( p = 0.32 )
• grade 3 infections : 9 patients ( 15.5 %) versus 2 ( 3.5 %; p = 0.03 )
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There were five patient deaths in the ruxolitinib arm and two in the BAT arm , but none was considered treatment related . Thrombotic events were more common in the ruxolitinib group than in the BAT group : 10 ruxolitinib-treated patients ( 17.2 %) experienced 11 events , and three BAT patients ( 5.8 %) experienced five events ( p = 0.09 ). Hemorrhagic events appeared to be more common in the BAT group ( 1 [ 1.7 %] vs . 5 [ 8.9 %]; p = 0.14 ).
“ Overall thrombosis , hemorrhage , or transformation considered separately or together as a composite endpoint were not statistically different between the ruxolitinib and BAT [ cohorts ],” the authors concluded .
The study ’ s findings are limited by the variation in treatment choice in the BAT group , and , the authors noted , “ the use of hydroxycarbamide as a BAT and frequent switching of BAT therapies also reflect real-life constraints and limited treatment options ” for this group of patients with high-risk ET . ●
Dr . Harrison reports financial relationships with Novartis , the manufacturer of ruxolitinib .
REFERENCE
Harrison CN , Mead AJ , Panchai A , et al . Ruxolitinib versus best available therapy for ET intolerant or resistant to hydroxycarbamide in a randomized trial . Blood . 2017 August 9 . [ Epub ahead of print ]
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