This prospective, multicenter, randomized study aim to evaluate the efficacy and safety of romiplostim N01 combined with glucocorticoids as the first-line treatment for newly diagnosed adult primary immune thrombocytopenia (ITP).
The primary endpoint of this study is to assess the efficacy of romiplostim N01 combined with glucocorticoids in untreated newly diagnosed adult ITP patients after 6 months of administration. 129 eligible subjects were randomized to either romiplostim N01 combining glucocorticoids or glucocorticoids treatment in 2:1 ratio. Administration protocol: Experimental group: Dexamethasone (HD-DXM) 40mg/d × 4d, one cycle. If there is no response on the 10th day, repeat once, administered orally or intravenously. Simultaneously, romiplostim N01 is administered, with an initial dose of 3µg/kg, by subcutaneous injection once a week, for up to 6 months. Control group: Dexamethasone (HD-DXM) 40mg/d × 4d, one cycle. If there is no response on the 10th day, repeat once, administered orally or intravenously. The initial dose of romiplostim N01 administration was 3µg/kg and can be initiated within 4 days of dexamethasone treatment. The dose of romiplostim N01 was adjusted according to the subject platelet count during the treatment period. When the platelet count is \< 50 × 10\^9/L, the patient will receive an increment in the dose of romiplostim N01 by 2µg/kg weekly, with a maximum dose of 10µg/kg. When 200 × 10\^9/L \> platelet count ≥ 50 × 10\^9/L, the administration dosage remains unchanged. When 400 × 10\^9/L \> platelet count ≥ 200 × 10\^9/L for two consecutive weeks, the dose is reduced by 1µg/kg. When the platelet count is ≥ 400 × 10\^9/L, discontinue the drug. When the platelet count \< 200 × 10\^9/L, resume administration, and the administration dose is 1µg/kg less than before drug cessation. All subjects were followed up until the 24th week after the end of treatment through clinical follow-up or telephone follow-up. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events, concomitant medications and concomitant treatments are also recorded throughout the study. The researcher can increase the number of visits as necessary for AE follow-up to monitor the alleviation of AEs.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
Dexamethasone (HD-DXM) at a dose of 40mg/d for 4 days constitutes one cycle. If there is no response on the 10th day, repeat it once. The administration can be either oral or intravenous. Meanwhile, romiplostim N01 is administered with an initial dose of 3µg/kg by subcutaneous injection within 4 days of dexamethasone treatment once a week for up to 6 months.
Dexamethasone (HD-DXM) 40mg/d × 4 days, one cycle. If there is no response on the 10th day, repeat once, administered either orally or intravenously.
Chinese Academy of Medical Science and Blood Disease Hospital
Tianjin, Tianjin Municipality, China
RECRUITINGThe proportion of patients with continuous remission
Continuous remission is defined as the maintenance of the therapeutic effect of patients for at least 6 months since achieving remission, without the need for additional ITP-specific treatment.
Time frame: 6 months
The total effective rate OR
The total effective rate OR = CR + R (Complete response CR, platelet count ≥ 100×10\^9/L, and no bleeding symptoms; Response R, platelet count is between 30×10\^9/L and 100×10\^9/L, and at least doubles compared to the baseline value, and no bleeding symptoms).
Time frame: 6 months
The proportion of patients with the initial response (reaching the effective standard within one month of the start of treatment)
The total effective rate OR = CR + R (Complete response CR, platelet count ≥ 100×10\^9/L, and no bleeding symptoms; Response R, platelet count is between 30×10\^9/L and 100×10\^9/L, and at least doubles compared to the baseline value, and no bleeding symptoms within one month).
Time frame: one month of the start of treatment
The proportion of patients reaching the effective standard 3 months after the start of treatment
The total effective rate OR = CR + R (Complete response CR, platelet count ≥ 100×10\^9/L, and no bleeding symptoms; Response R, platelet count is between 30×10\^9/L and 100×10\^9/L, and at least doubles compared to the baseline value, and no bleeding symptoms within 3 month).
Time frame: 3 months after the start of treatment]
The proportion of patients reaching the effective standard 6 months after the start
The total effective rate OR = CR + R (Complete response CR, platelet count ≥ 100×10\^9/L, and no bleeding symptoms; Response R, platelet count is between 30×10\^9/L and 100×10\^9/L, and at least doubles compared to the baseline value, and no bleeding symptoms within 6 month).
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TREATMENT
Masking
NONE
Enrollment
129
Time frame: 6 months after the start of treatment
The proportion of patients reaching the effective standard 9 and 12 months after the start of treatment
The total effective rate OR = CR + R (Complete response CR, platelet count ≥ 100×10\^9/L, and no bleeding symptoms; Response R, platelet count is between 30×10\^9/L and 100×10\^9/L, and at least doubles compared to the baseline value, and no bleeding symptoms within 9 and 12 month).
Time frame: 9 and 12 months after the start of treatment
The maximum consecutive weeks of platelet response
The consecutive weeks with platelet count \> 30×10\^9/L in the absence of any rescue treatment.
Time frame: 6 months
The proportion of subjects receiving rescue treatment.
The proportion of subjects receiving rescue treatment.
Time frame: 6 months
According to the WHO bleeding score standard, the incidence and severity of bleeding symptoms.
According to the WHO bleeding score standard, the incidence and severity of bleeding symptoms
Time frame: 6 months
The incidence of adverse events
Evaluated using Version 5.0 of the "Common Terminology Criteria for Adverse Events (NCI CTC AE)".
Time frame: 6 months