Background: Blinatumomab used to treat acute B lymphoblastic leukemia, while the actual safety data for it in different age groups is limited. Method: FAERS data were interrogated with ROR, PRR, BCPNN, and MGPS to identify significant safety signals. Subgroup analysis by age was performed to explore differences. The age groups were divided into minors (<18 years old), young adults (18-44 years old), middle-aged adults (45-64 years old), and old people (≥65 years old). In addition, minors were further stratified based on prognosis, into those under 1 year old, 1-9 years old, and 10-17 years old. Results: The common adverse reactions across different age groups were Acute lymphoblastic leukemia recurrence, Cytokine release syndrome and Central nervous system leukemia. However, there were distinct patterns of adverse events in different age groups. The results indicated that the Venoocclusive liver disease was regarded as a positive signal of adverse events in the under 1 years old subgroup, hepatotoxicity was prevalent in the 18-44 age group and psychiatric/neurologic events were more preponderant at greater than age greater than 65. Conclusion: This study provides evidence on the epidemiological characteristics of blinatumomab, age-related drug risk heterogeneity, and the authenticity of the early-onset model.

Background Thalidomide has been reported as a promising treatment for reducing transfusion volume in adults with β-thalassemia. However, the evidence about the safety and efficacy of thalidomide on children with transfusion dependent β-thalassemia (TDT) is scarce. Methods Seventy-seven children with TDT treated with thalidomide at least for 6 months were included and retrospectively analyzed. Oral dose was started at 2.5 mg•kg-1•d-1 Blood volume for maintenance of hemoglobin above 90 g•L-1 compared with pre-treatment volume is the evaluation index for response. Results After the sixth month treatment, 51/77 (66.2%) maintained Hb over 90 g•L-1 without transfusion. Adverse events were reported in 48 (63.2%) patients.Age, sex, genotype category, dosage and transfusion interval before thalidomide treatment were not correlated to treatment response. The AUC was 0.806 for the HbFat the third month of treatment in predicting probability of major responders at the sixth month treatment. Based on Youden’s index algorithm in the ROC curve, 47.298 g•L-1 was the optimal cut-off value of the HbFat the third month of treatment in predicting major responders at the sixth month treatment, with sensitivity of 67.5%, specificity of 93.3%. Conclusions The dose of thalidomide between 2.5 mg•kg-1•d-1to 3.6 mg•kg-1•d-1 is effective in TDT children. Severe side effects are uncommon. HbF concentration of 47.298 g•L-1 at the third month is recommended as the predictor for further major responders.