Objectives: To compare the efficacy and safety in Adult patients (≥18 years) diagnosed as essential thrombocythemia treated with the Pegylated Interferon Alfa-2b vs. Interferon Alfa. Study Design: A prospective, open-label, multicenter, randomized controlled clinical trial.
This is a prospective, open-label, multicenter, randomized controlled clinical trial between Interferon Alfa and Pegylated Interferon Alfa-2b in adult essential thrombocythemia (≥18 years). Patients will be randomly divided into the following two treatment groups: 1. Recombinant Interferon Alpha, with an initial dose of 300 wu three times a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available. 2. Pegylated Interferon Alfa-2b, with an initial dose of 135 ug at week 0 , and then 180 ug once a week from week 1 to week 52. The dosage will be adjusted according to the results of laboratory examinations and patient tolerance. The patient will be transferred to the other group if intolerance or resistance occurs.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
194
Recombinant Interferon Alpha, with an initial dose of 300 wu three times a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available.
Pegylated Interferon Alfa-2b, with an initial dose of 135 ug at week 0 , and then 180 ug once a week from week 1 to week 52.
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, China
RECRUITINGComplete hematological remission (CHR) rates
The CHR rates defined as European Leukemia Net will be compared between the two groups.
Time frame: From the start of study treatment (Week 0) up to the end of Week 52
CHR rates at week 24 and 36
The CHR rates at week 24 and 36 will be compared between the two groups
Time frame: From the start of study treatment (Week 0) up to the end of Week 24 and Week 36, respectively
Time to CHR
The time of reaching CHR will be compared between the two groups
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
The proportion of patients crossed to the contralateral group
The proportion of patients crossed to the contralateral group will be compared between the two groups
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
The CHR rates after crossover
The CHR rates within 52 weeks after crossover
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
Impact of therapy on driver mutations
To compare the proportion of subjects that display change on key biomarkers of the disease- JAK2V617F, CALR, MPL mutations.
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
Impact of therapy on non-driver mutations
To compare the proportion of subjects that display change on key non-driver biomarkers of the disease-DNMT3A, ASXL1, TET2 or other mutations.
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Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
Change of splenomegaly
To compare the proportion of subject with improvement and no progress rate of splenomegaly between the two groups.
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
Change of bone marrow pathology
To compare the rate of patients with improvement and no progress rate of bone marrow pathology between the two groups
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
The incidence of major thrombotic events
To compare the incidence of major thrombotic events between the two groups.
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
The incidence of major bleeding events
To compare the incidence of major bleeding events between the two groups.
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
The incidence of progressing to bone marrow fibrosis
The incidence of progressing to bone marrow fibrosis will be compared between the two groups
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
The incidence of progressing to acute leukemia
The incidence of progressing to acute leukemia will be compared between the two groups
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
Change in Myeloproliferative Neoplasm Symptom Assessment Form total symptom score
To compare the proportion of subjects that display change in Myeloproliferative Neoplasm Symptom Assessment Form total symptom score (0-100 scores, higher scores mean a worse outcome) between different treatment groups.
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
Change of quality of life
Compare the rate of patients with improvement in quality of life between the two groups (assessed by EORTC quality of life scale QLQ-C30 V3.0 questionnaire).
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
Change of microcirculation disturbance
The rate of patients with improvement in microcirculation disturbance (such as pruritus, headache, dizziness, chest tightness, erythematous limb pain and limb paresthesia) will be compared between the two groups
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
Specific pre-defined toxicity
To compare incidence of specific pre-defined toxicity including fatigue, flu-like symptoms, dizziness, injection site necrosis, dyspnea, pain, depression, blurred Vision, insomnia, anorexia, weight Loss, weakness, pruritis, sweating, fever, decreased Libido, hot Flashes, flushing.
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.