BESPONSA Injection 1 mg Special Investigation

BESPONSA® INJECTION 1 MG SPECIAL INVESTIGATION

The purpose of this study is to learn about the safety and effectiveness of BESPONSA. BESPONSA is approved for treatment of relapsed or refractory CD22-positive acute lymphocytic leukemia.

Registration criteria for this study is all patients who starting BESPONSA in Japan from its launch to the market to April 30, 2020.

All patients in this study will receive BESPONSA according to the prescriptions.

Patients will be followed up as follow.

• 52 weeks for patients who did not have a HSCT (Hematopoietic Stem Cell Transplant) within 52 weeks after starting BESPONSA.
• Up to 52 weeks after a HSCT for patients who had a HSCT within 52 weeks after starting BESPONSA.

Study Overview

• Status: Completed
• Conditions: Acute Lymphocytic Leukemia

• Study Type: Observational
• Enrollment (Actual): 421

Contacts and Locations

Study Locations

• Japan
  • Tokyo, Japan
    • Pfizer

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

All patients who received BESPONSA

Description

Inclusion Criteria:

• All patients prescribed BESPONSA

Exclusion Criteria:

• None

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The Incidence of Adverse Drug Reactions	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.	From first dose up to 28 days after last dose. VOD/SOS: No HSCT: up to 52 weeks, HSCT: up to 104 weeks
The Incidence of Liver Disorder Including VOD/SOS (ADRs)/ (All CTCAE Grades)	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.	No HSCT: up to 52 weeks, HSCT: up to 104 weeks
The Incidence of Liver Disorder Including VOD/SOS (ADRs)/ (CTCAE Grade 3 or Higher)	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.	No HSCT: up to 52 weeks, HSCT: up to 104 weeks
The Incidence of Myelosuppression (ADRs)/ (All CTCAE Grades)	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.	From the start date of administration to Day 28 post-final dose, approximately 27 weeks for HSCT and no HSCT participants
The Incidence of Myelosuppression (ADRs)/ (CTCAE Grade 3 or Higher)	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.	From the start date of administration to Day 28 post-final dose, approximately 27 weeks for HSCT and no HSCT participants
The Incidence of Infections (ADRs)/ (All CTCAE Grades)	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.	From the start date of administration to Day 28 post-final dose, approximately 27 weeks for HSCT and no HSCT participants
The Incidence of Infections (ADRs)/ (CTCAE Grade 3 or Higher)	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.	From the start date of administration to Day 28 post-final dose, approximately 27 weeks for HSCT and no HSCT participants
The Incidence of Hemorrhage (ADRs)/ (All CTCAE Grades)	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.	From the start date of administration to Day 28 post-final dose, approximately 27 weeks for HSCT and no HSCT participants
The Incidence of Hemorrhage (ADRs)/ (CTCAE Grade 3 or Higher)	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.	From the start date of administration to Day 28 post-final dose, approximately 27 weeks for HSCT and no HSCT participants
Early Death After HSCT	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.	Within 100 days or greater than 100 days after the first HSCT after the start of BESPONSA 1 mg injection up to 52 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Hematologic Remission Rate	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.	52 Weeks. 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 was earlier, was entered.
Overall Survival (OS)	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.	No HSCT: up to 52 weeks, HSCT: up to 104 weeks

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): July 2, 2018
• Primary Completion (Actual): September 6, 2024
• Study Completion (Actual): September 6, 2024

Study Registration Dates

• First Submitted: June 20, 2023
• First Submitted That Met QC Criteria: June 20, 2023
• First Posted (Actual): June 28, 2023

Study Record Updates

• Last Update Posted (Actual): December 5, 2025
• Last Update Submitted That Met QC Criteria: November 14, 2025
• Last Verified: October 1, 2025

More Information

Terms related to this study

• Keywords: Drug use investigation in Japan
• Additional Relevant MeSH Terms: Neoplasms; Immune System Diseases; Neoplasms by Histologic Type; Hematologic Diseases; Lymphatic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Leukemia, Lymphoid; Leukemia; Hemic and Lymphatic Diseases; Precursor Cell Lymphoblastic Leukemia-Lymphoma
• Other Study ID Numbers: B1931024; NCT05923112 (Registry Identifier: ClinicalTrials.gov)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.