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
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