Ruxolitinib for patients with polycythemia vera who have had an inadequate response or are intolerant to hydroxyurea: a critical appraisal

The goal of this review is to critically analyze data supporting the use of ruxolitinib in polycythemia vera patients resistant or intolerant to hydroxyurea. We analyzed the randomized Phase III study (RESPONSE) and we applied the Grading of Evidence, Assessment, Development and Evaluation approach by evaluating five dimensions of evidence: overall risk of bias, imprecision, inconsistency, indirectness and publication bias. We upgraded the quality of evidence because of large effect size on splenomegaly, hematocrit and symptoms but we downgraded it for performance bias and for indirectness of the comparator. In conclusion, by identifying factors affecting the quality of evidence, we rated the outcomes of ruxolitinib in polycythemia vera patients resistant or intolerant to hydroxyurea as having moderate level of evidence.