Abstract
The survival of pediatric patients with multiply relapsed and/or refractory (R/R) B-cell acute lymphoblastic leukemia has historically been very poor; however, data are limited in the current era. We conducted a retrospective study to determine the outcome of multiply R/R childhood B-ALL treated at 24 TACL institutions between 2005 and 2013. Patient information, treatment, and response were collected. Prognostic factors influencing the complete remission (CR) rate and event-free survival (EFS) were analyzed. The analytic set included 578 salvage treatment attempts among 325 patients. CR rates (mean ± SE) were 51 ± 4% for patients with bone marrow R/R B-ALL who underwent a second salvage attempt, 37 ± 6% for a third attempt, and 31 ± 6% for the fourth through eighth attempts combined. For patients achieving a CR after their second, third, and fourth through eighth attempts, the 2 year EFS was 41 ± 6%, 13 ± 7%, and 27 ± 13% respectively. Our results showed slight improvement when compared to previous studies. This is the largest and most recent study to date that evaluates the outcome of this patient population. Our data will provide detailed reference for the evaluation of new agents being developed for childhood B-ALL.
Highlights
Acute lymphoblastic leukemia (ALL) is the most common malignancy in children and adolescents, with ~85% of cases being B-cell precursor ALL (B-ALL)
The majority of salvage attempts were due to bone marrow (BM) relapse (458/578, 79.2%), while 13.5% were due to isolated extramedullary disease (Supplementary Table S1)
This is the second retrospective pooled data analysis from Therapeutic Advances in Childhood Leukemia consortium (TACL) that evaluates the outcome of pediatric patients with multiply R/R B-ALL treated during a contemporary period
Summary
Acute lymphoblastic leukemia (ALL) is the most common malignancy in children and adolescents, with ~85% of cases being B-cell precursor ALL (B-ALL). Over the past few decades, the overall survival rate in children with newly diagnosed ALL has improved dramatically from ~10% in the 1960s to almost 90% today [1, 2] Despite this remarkable improvement, ~2% patients are refractory to induction chemotherapy [3], and an additional 10–15% of ALL patients still experience a relapse [4]. Extended author information available on the last page of the article subsequent second complete remission (CR) can be achieved in most patients [5,6,7,8], ~55% of those patients will relapse again [6, 9] Those children are generally managed with intensive chemotherapy, with or without novel agents to induce a third remission, followed by hematopoietic stem cell transplant (HSCT) if indicated [10, 11]. New strategies are needed to improve the outcome of these patients
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