To evaluate the safety and efficacy of avatrombopag in the treatment of pediatric primary immune thrombocytopenia in patients who have been treated with eltrombopag before and switched to avatrobopag because of poor efficacy, excessive platelet fluctuation or intolerance, or patient preference, economic reasons, and other reasons.
Immune thrombocytopenia (ITP) is an organ-specific autoimmune disease, which is characterized by decreased platelet count and skin and mucosal bleeding. ITP is a kind of disease with increased platelet destruction and impaired platelet production caused by autoimmunity. Conventional treatment of adult ITP includes first-line glucocorticoid and immunoglobulin therapy, second line TPO and TPO receptor agonist, splenectomy and other immunosuppressive treatments (such as rituximab, vincristine, azathioprine, etc.). Eltrombopag is currently the only TPO receptor agonist with indications for pediatric immune thrombocytopenia. However, at present, the treatment response of pediatric ITP is not good, and a considerable number of patients need to switched to other TPO receptor agonist, such as avatrombopag, because of poor efficacy, excessive platelet fluctuation or intolerance, or patient preference, economic reasons, and other reasons. Therefore, the investigators designed this clinical trial to evaluate the efficacy and safety of avatrombopag in the treatment of pediatric immune thrombocytopenia in patients who who have been treated with eltrombopag before and switched to avatrobopag because of different reasons.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
60
After enrollment, all subjects receive Avatrombopag treatment. The initial dose of Avatrombopag administration was an oral 10 mg (\<30kg) or 20mg (≥30kg) once daily in all participants. ps. For subjects weighing ≥30kg, the dose can be started from 40mg once daily if the platelet count is \< 10×10\^9/L or if there is severe bleeding or risk of bleeding. Complete blood count including platelet count was done once a week. The dose of Avatrombopag was adjusted according to the subject platelet count during the period from week 1 to week 24. If the platelet count was less than 30×10\^9/L for 2 weeks, avatrobopag was gradually increased. If the platelet count was greater than 150 x 10\^9/L, avatrobopag was gradually reduced. If the platelet count \>250×10\^9/L, avatrobopag will be stoped until the platelet count \<100×10\^9/L.
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
Tianjin, China
RECRUITINGOverall efficacy response after AVA treatment within 12 weeks
Overall response rate defined as proportion of subjects with a platelet count ≥ 30 × 10\^9/L and at least 2-fold from baseline without bleeding at the meanwhile within 12 weeks after initial administration in absence of rescue therapy.
Time frame: 12 weeks
Treatment response-1
Number of participants achieving a platelet count ≥ 50×10\^9/L at week 1,2,4,6,8,12 in absence of rescue therapy.
Time frame: 12 weeks
Treatment response-2
Number of participants achieving a platelet count ≥ 100×10\^9/L at week 1,2,4,6,8,12 in absence of rescue therapy.
Time frame: 12 weeks
Time to Response
The time required from the start of treatment to the first time a subject's platelet count was greater than or equal to 30×10\^9/L and at least a two-fold increase from the baseline platelet count without bleeding in absence of rescue therapy.
Time frame: 12 weeks
Persistent response
Persistent response defined as proportion of subjects with a platelet count ≥ 30 × 10\^9/L and at least 2-fold from baseline without bleeding for 4 consecutive weeks or more within 12 weeks after initial administration in absence of rescue therapy.
Time frame: 12 weeks
Emergency treatment
The proportion of subjects receiving emergency treatment
Time frame: 12 weeks
Reduction of concomitant drug
Percentage of patients with reduced doses of concomitant drugs at baseline
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Time frame: 12 weeks
Number of participants with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale.
The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss.
Time frame: 12 weeks
Number of participants with clinically significant bleeding as assessed using the bleeding scale for pediatric patients with ITP.
The bleeding scale for pediatric patients with ITP is a measure of bleeding severity with the following grades: Grade 1 (minor) Minor bleeding, few petechiae (≤100 total) and/or ≤5 small bruises (≤3 cm in diameter), no mucosal bleeding；Grade 2 (mild) Mild bleeding, many petechiae (\>100 total) and/or \>5 large bruises (\>3 cm in diameter), no mucosal bleeding；Grade 3 (moderate) Moderate bleeding, overt mucosal bleeding, troublesome lifestyle；Grade 4 (severe) Severe bleeding, mucosal bleeding leading to decrease in Hb\>2 g/dL or suspected internal hemorrhage；
Time frame: 12 weeks
Health-related quality of life survey of subjects(HRQoL)-1
In all participants ,use ITP-PAQ (ITP Patient Assessment Questionnaire) to assess the HRQoL before and after treatment.
Time frame: 12 weeks
Health-related quality of life survey of subjects(HRQoL)-2
In all participants ,use FACIT-F（functional assessment of chronic illness therapy- fatigue）to assess the HRQoL before and after treatment.
Time frame: 12 weeks
Health-related quality of life survey of subjects(HRQoL)-3
In all participants ,use Kids' ITP tool KIT、to assess the HRQoL before and after treatment.
Time frame: 12 weeks
Health-related quality of life survey of subjects(HRQoL)-4
In all participants ,use Pediatric Quality of Life Inventory PedsQL to assess the HRQoL before and after treatment.
Time frame: 12 weeks