BESPONSA Injection 1 mg Special Investigation

An adverse drug reaction (ADR) was a treatment-related adverse event, and any untoward medical occurrence attributed to BESPONSA Injection 1mg in a participant who received BESPONSA Injection 1mg. A serious adverse drug reaction (SADR) was a treatment-related adverse event resulting in any of the following outcomes or deemed significant for any other reason: results in death; is life-threatening; requires inpatient hospitalization or prolongation of hospitalization; results in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); results in congenital anomaly/birth defect.

Risk ratios for the proportion of participants with ADRs in different subgroups were determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category.

ADRs meeting definition of liver disorder including safety specification events of VOD/SOS were analyzed by each of the following factors: Age; Hepatic impairment; Medical history (past) - VOD/SOS, Hepatitis or hepatic disease; Medical history (present) - VOD/SOS, Hepatitis or hepatic disease; Eastern Cooperative Oncology Group performance status (ECOG PS); aspartate aminotransferase (AST) and alanine aminotransferase (ALT) levels; gamma glutamyl transpeptidase (γ-GTP) level; Total bilirubin level; Platelet count; Peripheral blast count; Salvage line of the induction treatment with this drug; HSCT before the start of this drug.

Risk ratios for the proportion of participants with ADRs in different subgroups was determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category. In category(A) The risk ratio was calculable; however, the confidence interval could not be estimated due to the absence of ADRs in the numerator.

ADRs meeting definition of liver disorder including safety specification events of VOD/SOS were analyzed by each of the following factors: Age; Hepatic impairment; Medical history (past) - VOD/SOS, Hepatitis or hepatic disease; Medical history (present) - VOD/SOS, Hepatitis or hepatic disease; ECOG PS; AST and ALT levels; γ-GTP level; Total bilirubin level; Platelet count; Peripheral blast count; Salvage line of the induction treatment with this drug; HSCT before the start of this drug.

Risk ratios for the proportion of participants with ADRs in different subgroups was determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category.

ADRs meeting definition of safety specification events of myelosuppression were analyzed by each of the following factors: Age; Hepatic impairment; Renal impairment; Total dose per cycle > 1.8 mg/m2; ECOG PS; White blood cell count; Neutrophil count; Platelet count; Salvage line of the induction treatment with this drug; HSCT before the start of this drug.

Risk ratios for the proportion of participants with ADRs in different subgroups was determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category.

ADRs meeting definition of safety specification events of myelosuppression were analyzed by each of the following factors: Age; Hepatic impairment; Renal impairment; Total dose per cycle > 1.8 mg/m2; ECOG PS; White blood cell count; Neutrophil count; Platelet count; Salvage line of the induction treatment with this drug; HSCT before the start of this drug.

Risk ratios for the proportion of participants with ADRs in different subgroups was determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category. In category(A) The risk ratio was calculable; however, the confidence interval could not be estimated due to the absence of ADRs in the numerator. In category (B) The risk ratio and confidence interval could not be estimated because no ADRs were observed in either the numerator, the denominator, or both.

ADRs meeting definition of safety specification events of infection were analyzed by each of the following factors: Age; Hepatic impairment; Renal impairment; Total dose per cycle > 1.8 mg/m2; ECOG PS; Salvage line of the induction treatment with this drug; HSCT before the start of this drug.

Risk ratios for the proportion of participants with ADRs in different subgroups was determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category. In category(A) The risk ratio was calculable; however, the confidence interval could not be estimated due to the absence of ADRs in the numerator. In category (B) The risk ratio and confidence interval could not be estimated because no ADRs were observed in either the numerator, the denominator, or both.

ADRs meeting definition of safety specification events of infection were analyzed by each of the following factors: Age; Hepatic impairment; Renal impairment; Total dose per cycle > 1.8 mg/m2; ECOG PS; Salvage line of the induction treatment with this drug; HSCT before the start of this drug.

Risk ratios for the proportion of participants with ADRs in different subgroups was determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category. In category(A) The risk ratio was calculable; however, the confidence interval could not be estimated due to the absence of ADRs in the numerator. In category (B) The risk ratio and confidence interval could not be estimated because no ADRs were observed in either the numerator, the denominator, or both.

ADRs meeting definition of safety specification events of hemorrhage were analyzed by each of the following factors: Age; Hepatic impairment; Renal impairment; Total dose per cycle > 1.8 mg/m2; ECOG PS; Salvage line of the induction treatment with this drug; HSCT before the start of this drug.

Risk ratios for the proportion of participants with ADRs in different subgroups was determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category.

ADRs meeting definition of safety specification events of hemorrhage were analyzed by each of the following factors: Age; Hepatic impairment; Renal impairment; Total dose per cycle > 1.8 mg/m2; ECOG PS; Salvage line of the induction treatment with this drug; HSCT before the start of this drug.

Risk ratios for the proportion of participants with early death in different subgroups was determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category.

Participants in the safety analysis set who underwent HSCT after the start of BESPONSA Injection 1mg and have completed the study were included in the analysis. Early death after HSCT was defined as death occurring within 100 days after the first HSCT following the start of BESPONSA Injection 1mg. Early death was considered to be absent if the participant died on Day 101 or later, or was confirmed alive at the time of study completion/discontinuation.

The best overall response was evaluated for its clinical effect of hematologic remission during the observation period.

The proportions of CR or CRi and their 95% confidence intervals were determined.

However, if subsequent treatment for the target disease was started or HSCT was performed, the best overall response before the start of the subsequent treatment or HSCT, whichever occurred earlier, was recorded.

OS was defined and evaluated as the number of months from the start of BESPONSA Injection 1mg to all-cause death. The participant's survival status was confirmed at the time of completion or discontinuation of the study. The endpoint was censored on the day when participant's survival was last confirmed.

For time-to-event data, the median was determined using the Kaplan-Meier method.