Diagnosis Of Polycythemia Vera, Ruxolitinib versus Standard Therapy for the Treatment of Polycythemia Vera
Polycythemia vera is an acquired myeloproliferative disorder that causes overproduction of all three hematopoietic cell lines, most prominently red blood cells. A mutation of JAK2, a signaling molecule, has been demonstrated in 95% of cases
Symptoms and Signs:
Signs and symptoms are related to expanded blood volume and increased blood viscosity which include:
1.Headache 2.Dizziness 3.Tinnitus 4.Blurred vision 5.Night Sweats 6.Fatigue 7.Generalized pruritus especially following a warm shower 8.Splenomegaly 9.Plethora
Hct > 52% in males or 48% in females
In the absence of JAK2 Mutation:
Four Major criteria
Three Major & Two Minor criteria
Hct > 60% in males, Hct > 56% in females
No Secondary erythrocytosis
Presence of genetic mutations (excludes JAK2)
Platelets > 450000/cumm
Neutrophils > 10000/cumm
Low serum erythropoietin
Goal Of Therapy:
The main goal of therapy is to prevent thrombotic events while avoiding iatrogenic harm and minimizing the risk of transformation to
Most patients receive low-dose aspirin and undergo phlebotomy, with a goal of maintaining hematocrit values of less than 45%.
Cytoreductive therapy is recommended in patients having high risk for thrombosis, persistent or progressive hematologic abnormalities or symptoms, increased requirement for phlebotomies or who cannot undergo phlebotomy.
First-line cytoreductive agent is hydroxyurea
Patients having an inadequate response to the drug or have unacceptable side effects at the doses required to consistently control the hematocrit, these patients would be classified as having resistance or intolerance to hydroxyurea
Ruxolitinib versus Standard Therapy for the Treatment of Polycythemia Vera:
Ruxolitinib is a Janus kinase (JAK) inhibitor. It is FDA approved drug for
It was a Randomized Controlled Phase 3 study, to evaluate the safety and efficacy of a JAK inhibitor in patients with PV who have an inadequate response to or have unacceptable side effects from hydroxyurea
Patients randomly assigned phlebotomy-dependent with splenomegaly, in a 1:1 ratio, to receive ruxolitinib (110 patients) or standard therapy (112 patients).
Primary End Point:
Primary end point was both hematocrit control through week 32 and at least a 35% reduction in spleen volume at week 32, as assessed by means of imaging.
The primary end point was achieved in 21% of the patients in the ruxolitinib group versus 1% of those in the standard-therapy group (P<0.001)
Hematocrit control was achieved in 60% of patients receiving ruxolitinib and 20% of those receiving standard therapy.
38% of patients receiving ruxolitinib and 1% of those receiving standard therapy had at least a 35% reduction in spleen volume.
A complete hematologic remission was achieved in 24% of patients in the ruxolitinib group and 9% of those in the standard-therapy group .
Reduction in the total symptom score at week 32 was achieved in 49% of patients in the ruxolitinib group and 5% of those in the standard-therapy group.
In the ruxolitinib group, grade 3 or 4 anemia occurred in 2% of patients, and grade 3 or 4 thrombocytopenia occurred in 5%
In the standard-therapy group anemia occurred in 0% and thrombocytopenia in 4%
Herpes zoster infection was reported in 6% of patients in the ruxolitinib group and 0% of those in the standard-therapy group.
Thromboembolic events occurred in one patient receiving ruxolitinib and in six patients receiving standard therapy.
In patients who had an inadequate response to or had unacceptable side effects from hydroxyurea, ruxolitinib was superior to standard therapy in controlling the hematocrit, reducing the spleen volume, and improving symptoms associated with polycythemia vera.
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