children with aplastic anemia
Conditions
Interventions
Treatment group 1(1-5 years old):Herombopag dose climb: 0.15mg/kg/d, 0.2mg/kg/d, 0.25mg/kg/d, 0.3mg/kg/d, each dose level is continuously administered for 7 days, and PK blood collection is performed
Treatment group 2(6-11 years old):Herombopag dose climb: 7.5mg/d, 10mg/d, 12.5mg/d, 15mg/d, each dose level is continuously administered for 7 days, and PK blood collection is performed on the first d
Treatment group 1(12-17 years old):Herombopag dose climb: 7.5mg/d, 15mg/d, each dose level is continuously administered for 7 days, and PK blood collection is performed on the first day of the startin
Sponsors
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
Eligibility
Sex/Gender
All
Age
1 Years to 17 Years
Inclusion criteria
Inclusion criteria: To be enrolled in this study, subjects must meet all of the following inclusion criteria: 1. Age < 18 years old, gender unlimited; 2. The diagnosis of aplastic anemia should refer to the related guidelines and consensus of Chinese children with aplastic anemia, which are as follows: At least 2 of the following 3 items: ?HGB<100g/L; ?PLT<100×109/L; ?ANC<1.5×109/L(thrombocytopenia must be included if it is two-line reduction); Classification diagnostic criteria: Those meeting the above AA diagnostic criteria were categorized according to bone marrow pathology and peripheral blood cell count: Severe AA(SAA) : (1) The degree of nucleated cell hyperplasia in bone marrow is 25%-50%, the degree of residual hematopoietic cells <30% or the degree of nucleated cell hyperplasia <25%. (2) At least two of the following three parameters in the peripheral blood: ?ANC<0.5×109/L; ?PLT<20×109/L; ? Reticulocyte (RET) <20×109/L, or RET <1% after correction; Extremely severe AA(VSAA) : The absolute value of neutrophils was <0.2×109/L, except for meeting the condition of SAA. (3) Non-severe AA(NSAA) : did not meet the diagnostic criteria of SAA and VSAA; Transfusion-dependent NSAA: All patients with HGB<60g/L and/or PLT<10×109/L requiring regular infusion of red blood cells or platelets within the first 6 months were transfusion-dependent NSAA. Conversely, patients who did not meet the criteria were non-transfusion-dependent; 3. Platelet receptor agonists (TPO-RA) were not treated within 1 week before treatment; 4.ECOG score =2 points; 5. The subject is not suitable for or unwilling to receive hematopoietic stem cell transplantation; 6. Subject and legal guardian agree to participate and sign informed consent.
Exclusion criteria
Exclusion criteria: Subjects will not be enrolled in this study if they have any of the following characteristics or conditions: 1. Failure to understand the nature of the study or to obtain informed consent; 2. Patients with a history of primary myelodysplastic syndrome (MDS), primary paroxysmal sleep hemoglobinuria (PNH), leukemia, and congenital bone marrow failure syndromes (IBMFS), such as Fanconi anemia (FA) and congenital dyskeratosis (DC); 3. Treatment with TPO-RA for =1 month before treatment; 4. During the screening period, alanine aminotransferase (ALT) and aspartate aminotransferase (AST) were more than 2.5 times of the upper limit of normal value, total bilirubin was more than 1.5 times of the upper limit of normal value, and serum creatinine was more than 1.5 times of the upper limit of normal value; 5. History of cirrhosis or portal hypertension; 6. Congestive heart failure, arrhythmia, peripheral arteriovenous thrombosis requiring drug treatment occurred within 1 year before enrollment, or myocardial infarction or cerebral infarction occurred within 3 months before enrollment; 7. Other conditions deemed unsuitable for inclusion in the study by the researcher.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Herombopag PK index (fixed blood concentration at the point of blood collection);Incidence of adverse events during treatment with Herombopag;The recommended dose of Herombopag for the treatment of children with aplastic anemia; | — |
Secondary
| Measure | Time frame |
|---|---|
| Proportion of patients with hematologic responses of either line during treatment with Herombopag; | — |
Countries
China
Contacts
Public Contactxiaofan Zhu
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
Outcome results
None listed