Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Objectives: To compare the efficacy and safety in childhood and adolescent patients (<20 years) diagnosed as essential thrombocythemia treated with the Pegylated Interferon Alfa-2b vs. Interferon Alfa.
Study Design: A prospective, open-label, nonrandomized, single-center clinical trial
This is a prospective, open-label, nonrandomized, single-center clinical trial between Interferon Alfa and Pegylated Interferon Alfa-2b in childhood and adolescent essential thrombocythemia (<20 years).
Patients will be divided into the following two treatment groups: 1. Recombinant Interferon Alpha, with an initial dose of 300 wu twice a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available, and the specific dose will be determined by the researchers; 2. Pegylated Interferon Alfa-2b, with an initial dose of 135 ug once a week (body surface area < 1.73 m2) or 180 ug once a week ( body surface area≥1.73 m2).
The current drug therapies and possible risks of Pegylated Interferon Alfa-2b and Interferon Alfa in the treatment of childhood and adolescent essential thrombocythemia will be fully introduced to the guardians (childhood patients) or patients (adolescent patients) by the researchers. Then the patients will be divided into one of the two groups according to the guardians' (childhood patients) or patients' (adolescent patients) will.
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.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Recombinant Interferon Alpha | Active Comparator | Recombinant Interferon Alpha, with an initial dose of 300 wu twice a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available, and the specific dose will be determined by the researchers. |
|
| Pegylated Interferon Alfa-2b | Experimental | Pegylated Interferon Alfa-2b, with an initial dose of 135 ug once a week (body surface area < 1.73 m2) or 180 ug once a week ( body surface area≥1.73 m2). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Recombinant Interferon Alpha | Drug | Recombinant Interferon Alpha, with an initial dose of 300 wu twice a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available, and the specific dose will be determined by the researchers; |
| Measure | Description | Time Frame |
|---|---|---|
| Change in platelet count | Proportion of subjects with a continuous platelet count ≤600×109/L or decrease ≥50% (<1000×109/L ) (at least 12 weeks) from baseline during treatment will be evaluated. | From the start of study treatment (Day 1) up to the end of month 12 |
| Measure | Description | Time Frame |
|---|---|---|
| The complete hematologic response rates | To compare the complete hematologic response rates between different treatment groups | From the start of study treatment (Day 1) up to the end of month 12 |
| Time to response in platelet count |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Rongfeng Fu, MD | Contact | +862223909009 | furongfeng@ihcams.ac.cn | |
| Lei Zhang, MD | Contact | +862223909240 | zhanglei1@ihcams.ac.cn |
| Name | Affiliation | Role |
|---|---|---|
| Lei Zhang, MD | Institute of Hematology & Blood Diseases Hospital, China | Principal Investigator |
| Rongfeng Fu | Institute of Hematology & Blood Diseases Hospital, China | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Institute of Hematology & Blood Diseases Hospital | Recruiting | Tianjin | Tianjin Municipality | 300020 | China |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
| Pegylated interferon alfa-2b | Drug | Pegylated Interferon Alfa-2b, with an initial dose of 135 ug once a week (body surface area < 1.73 m2) or 180 ug once a week ( body surface area≥1.73 m2). |
|
Time to response in platelet count (<600×109/L) between different treatment groups
| From the start of study treatment (Day 1) up to the end of month 12 |
| Impact of therapy on key biomarkers | To compare the proportion of subjects that display change on key biomarkers of the disease- JAK2V617F, CALR, MPL mutations. | From the start of study treatment (Day 1) up to the end of month 12 |
| Incidence of major cardiovascular and thrombotic events | To estimate incidence of major cardiovascular and thrombotic events (defined as cardiovascular death, myocardial infarction, stroke, transient ischemic attack, pulmonary embolism, Budd Chiari syndrome, deep vein thrombosis, and any other clinically relevant thrombotic event) while on active treatment or observation following end of treatment between different treatment groups | From the start of study treatment (Day 1) up to the end of month 12 |
| Incidence of development of myelodysplastic disorders, myelofibrosis, or leukemic transformation. | To estimate incidence of development of myelodysplastic disorders, myelofibrosis, or leukemic transformation between different treatment groups | From the start of study treatment (Day 1) up to the end of month 12 |
| 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. | 12 months |
| 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. | From the start of study treatment (Day 1) up to the end of month 12 |
| Impact of therapy on bone marrow histopathology (selectable) | To compare the proportion of subjects that display change on bone marrow histopathology | From the start of study treatment (Day 1) up to the end of month 12 |
| Impact of therapy on cytogenetic abnormalities (selectable) | To compare the proportion of subjects that display change on cytogenetic abnormalities. | From the start of study treatment (Day 1) up to the end of month 12 |
| Death while on active treatment or observation following end of treatment | To compare the incidence of death while on active treatment or observation following end of treatment | From the start of study treatment (Day 1) up to the end of month 12 |
| Change in platelet count | Proportion of subjects with a continuous platelet count <1000×109/L in those with platelet count ≥1000×109/L before treatment (at least 12 weeks) will be evaluated. | From the start of study treatment (Day 1) up to the end of month 12 |
| ID | Term |
|---|---|
| D013920 | Thrombocythemia, Essential |
| ID | Term |
|---|---|
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D013922 | Thrombocytosis |
| D001791 | Blood Platelet Disorders |
| D009196 | Myeloproliferative Disorders |
| D001855 | Bone Marrow Diseases |
| D006474 | Hemorrhagic Disorders |
Not provided
Not provided
| ID | Term |
|---|---|
| C417083 | peginterferon alfa-2b |
Not provided
Not provided
Not provided