A Prospective, Single-center Clinical Trial of Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in the Treatment of Childhood and Adolescent Essential Thrombocythemia
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Change in platelet count
研究概览
简要总结
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
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 19 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •<20 years old
- •Male or Female
- •Diagnosis of essential thrombocythemia according to the 2016 WHO criteria.
- •Platelet count ≥ 450 × 109 / L for more than 6 months（If the patient has JAK2 V617F, CALR or MPL gene mutation, the history may be less than 6 months）
- •Platelet count ≥ 1000 × 109 / L or other therapeutic indications at screening.
- •The guardians has provided written informed consent prior to enrollment
排除标准
- •Known to meet the criteria for primary myelofibrosis or polycythemia vera by 2016 WHO criteria
- •Presence of any life-threatening co-morbidity
- •Secondary thrombocytosis
- •Familial thrombocytosis
- •Resistance, or intolerance, or any contraindications to interferon
- •Interferon is used in the past 1 month before enrollment
- •Patients with previous or present thrombosis or active bleeding
- •WBC<4× 109 / L
- •HGB<110g/L
- •Poor control of thyroid dysfunction
- •Patients with a prior malignancy within the last 3 years
- •Patients with severe cardiac or pulmonary dysfunction
- •Severe renal damage (creatinine clearance < 30 ml / min)
- •Severe liver dysfunction (ALT or AST > 2.5×ULN)
- •Patients diagnosed as diabetes with poor control
- •Patients with hepatitis B virus, hepatitis C virus replication or HIV infection
- •Patients with a history of drug / alcohol abuse (within 2 years before the study)
- •Patients that have participated in other experimental researches within one month before enrollment
- •History of psychiatric disorder
- •Any other circumstances that the investigator considers that the patient is not suitable to participate in the trial
研究组 & 干预措施
Recombinant Interferon Alpha
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.
干预措施: Recombinant Interferon Alpha (Drug)
Pegylated Interferon Alfa-2b
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).
干预措施: Pegylated interferon alfa-2b (Drug)
结局指标
主要结局
Change in platelet count
时间窗: From the start of study treatment (Day 1) up to the end of month 12
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.
次要结局
- The complete hematologic response rates(From the start of study treatment (Day 1) up to the end of month 12)
- Time to response in platelet count(From the start of study treatment (Day 1) up to the end of month 12)
- Impact of therapy on key biomarkers(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.(From the start of study treatment (Day 1) up to the end of month 12)
- Incidence of major cardiovascular and thrombotic events(From the start of study treatment (Day 1) up to the end of month 12)
- Change in platelet count(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(12 months)
- Specific pre-defined toxicity(From the start of study treatment (Day 1) up to the end of month 12)
- Impact of therapy on bone marrow histopathology (selectable)(From the start of study treatment (Day 1) up to the end of month 12)
- Impact of therapy on cytogenetic abnormalities (selectable)(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(From the start of study treatment (Day 1) up to the end of month 12)