The clinical data of patients with PV treated with
ruxolitinib in Peking Union Medical College Hospital from January 1, 2013 to December 31, 2019 were retrospectively analyzed. The starting dose of oral
ruxolitinib was 10 mg twice daily and could be increased after 3 months of treatment if hematocrit (HCT) control was not achieved. HCT control was defined as HCT<45% in the absence of phlebotomy.
RESULTS: Thirty-three patients (17 males and 16 females) were treated with
ruxolitinib at a median age of 50 (21-72) years. JAK2V617F and JAK2exon12 alleles were detected in 31 and 2 patients, respectively. Before treatment, median
hemoglobin level was 187 (166-
208) g/L, median white blood cell and platelet level was 10.4 (5.0-15.8)×109/L and 457(237-677)×109/L, respectively. Totally 17 patients (51.5%) who were resistant to or intolerant of
hydroxyurea were treated with
ruxolitinib as second-line
therapy, and 16 patients (48.5%) were treated with
ruxolitinib as first-line
therapy voluntarily. The median time since PV diagnosis to treatment of
ruxolitinib was 47 (3-188) months. By December 31, 2019, all the patients continued to receive
ruxolitinib. The median duration of
ruxolitinib exposure was 19 (2-91) months. Both in the first-line therapy group and second-line therapy group, 15 cases (accounting for 93.8% and 88.2%, respecitvely) achieved HCT control. The median time from start of
therapy to HCT control was 2.2 (0.8-11.6) months. One patient (3.0%) had
disease progression after HCT control. The most common hematologic adverse events included
anemia and
thrombocytopenia, according to CTCAE classification, including 1 case of grade 1
anemia (3.0%) and 1 case of grade 2
thrombocytopenia (3.0%). There was no thromboembolic event occurred during the
therapy of
ruxolitinib.
CONCLUSION: The remission rate of HCT in PV patients treated with
ruxolitinib is high, and adverse reactions are rare.
Ruxolitinib is effective in HCT control and generally well tolerated in patients with PV.