Contributions
Abstract: PB1727
Type: Publication Only
Session title: Myeloproliferative neoplasms - Clinical
Background
Ruxolitinib (RXL) is an inhibitor of JAK 1 and 2, used in the management of patients with MPD, leading to improvements in splenomegaly and constitutional symptoms.
Aims
to characterize the efficacy and toxicity of RXL in patients with MPD treated in our center.
Methods
retrospective analysis of the patients treated with RXL during the previous 5 years.
Results
13 patients were included, 10 (71%) males, median age 63.5 yo (min 52; max 78). 7 (54%) patients had primary myelofibrosis (PMF); 4 (30%) patients had secondary myelofibrosis (SMF) - 1 post Polycythemia Vera (PV) and 3 post Essential Thrombocytosis (ET); 1 (8%) patient with PV and 1 (8%) patient with ET. Results are presented as follows:
PMF – at diagnosis 3 (43%) patients presented with intermediate-2 DIPSS score, 3 patients with intermediate-1 DIPSS score and 1 (14%) patient had low risk DIPSS. 4 patients were JAK2V617F+. As first line treatment, 3 patients were treated with ESA, 2 with HU and 1 patient received first line treatment with RXL. RXL was offered as 2nd line treatment in 3 patients, and as 3rd line treatment in 3 patients. Median duration of treatment with RXL was 15 months (min-9; max-54). No patient had PMF-related symptoms. Splenomegaly was present in 4 (57%) patients: 1 received RXL as first line treatment, 2 received RXL after 2nd line HU therapy and 1 received RXL as 3rd line of treatment (after HU and subsequent therapy with thalidomide + PDN). Response to RXL, including spleen reduction, was observed in the patient who received treatment as 1st line treatment, in 1 patient after 2º line treatment and in the patient treated as 3rd line of treatment. Overall, 4 (57%) patients had hematological toxicities and 1 (14%) patient had gastrointestinal adverse effects.
SMF – 3 patients had post ET myelofibrosis, all had received cytoreductive therapy as first line treatment; only 1 patient were JAK2V617F+ and had splenomegaly, and RXL was effective in reducing splenomegaly in this patient; the 2 other patients had no splenomegaly or JAK2V617F+ mutation and evolved with splenomegaly despite RXL therapy. Mean duration of treatment was 18 months (min 3; max 33). Hematological toxicity was registered in 2 patients. 1 patient had post PV myelofibrosis, was JAK2V617F+ and had splenomegaly responding to RXL treatment. There was no toxicity registered after a treatment duration of 10 months.
PV – 1 patient with PV received RXL due to intolerance to cytoreductive therapy; he was JAK2V617F+ and had splenomegaly at diagnosis. RXL was no able to reduce splenomegaly after 20 months of treatment. Hematological toxicity as thrombocytopenia has occurred.
ET – 1 patient with ET received RXL due to resistance to hydroxyurea; he was JAK2V617F+; had symptoms related to thrombocytosis, but no splenomegaly. RXL was effective in reducing symptoms but had no effect on the platelet count. No hematological toxicity was registered after 12 months of treatment with RXL.
Conclusion
Overall, response to RXL measured as spleen reduction or symptomatic control, was verified in 6 patients (46%) and 8 (61,5%) patients suffered toxicity, mainly hematological.
Keyword(s):
Abstract: PB1727
Type: Publication Only
Session title: Myeloproliferative neoplasms - Clinical
Background
Ruxolitinib (RXL) is an inhibitor of JAK 1 and 2, used in the management of patients with MPD, leading to improvements in splenomegaly and constitutional symptoms.
Aims
to characterize the efficacy and toxicity of RXL in patients with MPD treated in our center.
Methods
retrospective analysis of the patients treated with RXL during the previous 5 years.
Results
13 patients were included, 10 (71%) males, median age 63.5 yo (min 52; max 78). 7 (54%) patients had primary myelofibrosis (PMF); 4 (30%) patients had secondary myelofibrosis (SMF) - 1 post Polycythemia Vera (PV) and 3 post Essential Thrombocytosis (ET); 1 (8%) patient with PV and 1 (8%) patient with ET. Results are presented as follows:
PMF – at diagnosis 3 (43%) patients presented with intermediate-2 DIPSS score, 3 patients with intermediate-1 DIPSS score and 1 (14%) patient had low risk DIPSS. 4 patients were JAK2V617F+. As first line treatment, 3 patients were treated with ESA, 2 with HU and 1 patient received first line treatment with RXL. RXL was offered as 2nd line treatment in 3 patients, and as 3rd line treatment in 3 patients. Median duration of treatment with RXL was 15 months (min-9; max-54). No patient had PMF-related symptoms. Splenomegaly was present in 4 (57%) patients: 1 received RXL as first line treatment, 2 received RXL after 2nd line HU therapy and 1 received RXL as 3rd line of treatment (after HU and subsequent therapy with thalidomide + PDN). Response to RXL, including spleen reduction, was observed in the patient who received treatment as 1st line treatment, in 1 patient after 2º line treatment and in the patient treated as 3rd line of treatment. Overall, 4 (57%) patients had hematological toxicities and 1 (14%) patient had gastrointestinal adverse effects.
SMF – 3 patients had post ET myelofibrosis, all had received cytoreductive therapy as first line treatment; only 1 patient were JAK2V617F+ and had splenomegaly, and RXL was effective in reducing splenomegaly in this patient; the 2 other patients had no splenomegaly or JAK2V617F+ mutation and evolved with splenomegaly despite RXL therapy. Mean duration of treatment was 18 months (min 3; max 33). Hematological toxicity was registered in 2 patients. 1 patient had post PV myelofibrosis, was JAK2V617F+ and had splenomegaly responding to RXL treatment. There was no toxicity registered after a treatment duration of 10 months.
PV – 1 patient with PV received RXL due to intolerance to cytoreductive therapy; he was JAK2V617F+ and had splenomegaly at diagnosis. RXL was no able to reduce splenomegaly after 20 months of treatment. Hematological toxicity as thrombocytopenia has occurred.
ET – 1 patient with ET received RXL due to resistance to hydroxyurea; he was JAK2V617F+; had symptoms related to thrombocytosis, but no splenomegaly. RXL was effective in reducing symptoms but had no effect on the platelet count. No hematological toxicity was registered after 12 months of treatment with RXL.
Conclusion
Overall, response to RXL measured as spleen reduction or symptomatic control, was verified in 6 patients (46%) and 8 (61,5%) patients suffered toxicity, mainly hematological.
Keyword(s):