A retrospective analysis of overall response and survival to 1st+ salvage therapy in T-cell and 2nd+ salvage therapy in B-cell pediatric acute lymphoblastic leukemia (20180065) (Retrospective pediatric ALL retrospective response)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medical condition to be studied:
  T-cell type acute leukaemia

  B-cell type acute leukaemia
• Additional medical condition(s):
  T-cell and B-cell pediatric acute lymphoblastic leukemia

Population studied

• Age groups:
  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)
• Estimated number of subjects:
  1000

Study design details

• Main study objective:
  Assess the complete remission response rates among pediatric patients treated for relapsed or refractory acute lymphoblastic leukemia.
• Outcomes:
  Complete remission rate, event free survival, overall survival, transplant rate.
• Data analysis plan:
  The primary objective of describing the proportion of patients achieving a CR and BOR after salvage therapy will be estimated as a proportion with a 95% CI. Patients will be categorized according to standard hematologic definitions of response. The secondary endpoint of CR+CRp will estimate the proportion of patients achieving a CR with a 95% CI. BOR after salvage therapy will be estimated as a proportion for each response with a 95% CI. MRD will be assessed among patients achieving CR+CRp. Secondary endpoints EFS and OS will be described as a median in months with a 95% CI by the Kaplan-Meier method. KM curves of EFS and OS will be plotted. EFS and OS 3-, 6- 12-, 24-, 60- month probability survival point estimates will be calculated. Additionally, the proportion of patients that undergo HSCT will also be calculated with a 95% CI, particularly among patients who achieve a CR.

Documents

Study results

20180065 Abstract ORSR 10Jan25.pdf