Background and Objective: Immune thrombocytopenia (ITP) is an autoimmune-mediated hemorrhagic disease. Anti-glycoprotein autoantibodies play a key role in the pathophysiology of ITP, but the relationship between platelet-specific antibodies and bleeding severity is unclear. This study aimed to analyze the relationship between anti-glycoprotein autoantibodies and bleeding severity in children with newly diagnosed ITP and platelet count <10×109/L. Method: This was a single-center prospective observational study that analyzed children with newly diagnosed ITP and platelet count <10×109/L between June 2018 and September 2021 at our hospital. The children were classified into the mild and severe groups based on the bleeding scores. The type and titer of anti-glycoprotein autoantibodies were detected using an ELISA kit (PAKAUTO). We analyzed the relationship between bleeding severity and anti-glycoprotein autoantibodies. Results: A total of 86 cases were enrolled, including 42 in the mild group and 44 in the severe group. Patients with anti-GPIIb/IIIa or anti-GPIb/IX antibodies suffered more severe bleeding than patients without them (c2=7.303, p=0.007; c2=3.875, p=0.049), but there was no significant difference between patients with or without anti-GPIa/IIa antibody (c2=0.745, p=0.388). When antibodies were analyzed together, patients with three antibodies suffered more severe bleeding than those without three antibodies (c2=5.053, p=0.025). Patients with higher antibody titer in the eluent, but not in the plasma, suffered more severe bleeding in all three antibodies (Z=-2.389, p=0.017; Z=-2.108, p=0.035; Z=-2.557, p=0.011). Conclusion: Anti-glycoprotein autoantibodies led to more severe bleeding in children under 18 years of age without drug treatment with newly diagnosed ITP and platelet count <10×109/L.

Severe aplastic anemia (SAA) is caused by immune-mediated destruction. Standard immunosuppressive therapy (IST) is effective, but needs to be improved. A total of 115 patients (60 males; median age of 5.77 years and median follow-up time of 45 months) were enrolled in this historical control study. The complete response (CR) rates in the eltrombopag group were 30.3% at 3 months, 50.0% at 6 months, conpared to 8.2% at 3 months, 10.2% at 6 months in the control group. There were significant differences between the two groups at 3 months and 6 months after IST. The overall response rates in the two groups showed no significant differences during the study. There were significant differences in the times separated from granulocyte colony stimulating factor (G-CSF) G-CSF, red blood cell transfusion and Platelet transfusion between the two groups. Overall survival rates were 97.0% in the eltrombopag group and 96.0% in the control group (P=0.998). In the eltrombopag group 10.2% cases relapsed compared to 4.1% in the control group (P=0.703). No case progressed to myelodysplastic syndrome or myeloid leukemia in the eltrombopag group; one patient (2.0%) progressed to myelodysplastic syndrome in the control group. Totally 11 patients (16.7%) showed myeloid gene mutations in the eltrombopag group. Event-free survival (EFS) was 66.6% in the eltrombopag group and 57.1% in the control group. There were no significant differences in EFS between the two groups (P=0.967). In the eltrombopag group the common adverse reactions were transient and reversible hyperbilirubinemia, elevated liver enzymesand hyperuricemia.

Objective: To analyze the effect of a novel second-line escalating treatment strategy (high-dose dexamethasone (HDD), low-dose rituximab to eltrombopag) for children with severe chronic immune thrombocytopenia (SCITP). Materials and Methods: This study was a single-center, retrospective cohort study. The second-line escalating strategy included 3 steps: Step I (6 courses high-dose dexamethasone: HDD), Step II (HDD combined with low-dose rituximab), and Step III (eltrombopag). Results: A total of 30 cases (18 males and 12 females) were included; the median age was 8.83 (1.42-13.9) year-old, the duration time of ITP was 20.5 (12.0-96.0) months, and the platelet counts were 15 (3-29) ×109/L. After the median 14 (12-37) months’ treatment, the remission rate was 36.7% (11/30), and the sustained response (SR) rate was 68.2% (15/22). In eltrombopag (step III) cases, 47.5% (8/17) maintained platelet ≥50×109/L, 37.5% (3/8) dose tapering, and 25% (2/8) were successfully discontinued from medication. The number of patients at 12th, 24th, and 36th months was 30, 7, and 2, with the total response (TR) and remission rates of 80% (36.7%), 57.1% (28.6%), and 50% (50%), respectively. The total relapse rate was 26.7% (8/30)，three cases(75%, 3/4)from Step II and 5 cases (41.7% ,5/12)from Step III, none in Step I. Conclusion: The new second-line escalating strategy for children SCITP has an effective improving rate with 36.7% remission and 68.2% SR; 30% could benefit and retain stable response from HDD treatment. Combined treatment with eltrombopag can reduce the relapse rate of low-dose rituximab.

Background: Eltrombopag (E-PAG) is being investigated for the treatment of aplastic anemia (AA) by stimulating hematopoietic stem cell (HSC) proliferation. Objective: To evaluate the effectiveness and safety of E-PAG in first-line therapy for pediatric AA. Methods: The present retrospective study reviewed the pediatric patients with newly diagnosed AA in immunosuppressive therapy (IST) therapy with E-PAG at the single center from March to September in 2017. All patients were followed up for >2 years. Results: A total of 14 patients (8 males), aged 86 months, were enrolled in this study. E-PAG was administered with a median time to initiation of 19.5 days after IST, and the median course of treatment was 253 days. The rate of complete response and overall response at 6 months were 64.3% (9/14 case) and 78.6% (11/14 cases) respectively. The survival rate was 100%, and no relapses occurred in responders. E-PAG was well-tolerated; however, the most common adverse events included indirect bilirubin elevation, jaundice, and transient liver-enzyme elevation. By the end of follow-up, bone marrow chromosomes were normal, and no abnormal myelodysplastic syndromes (MDS) -related clones appeared. Conclusions: The addition of E-PAG to IST was associated with the markedly increased complete response with respect to hematology in pediatric patients with SAA than in a historical cohort without the unacceptable side effects.