Outcomes of treatment for relapsed acute lymphoblastic leukaemia in children with Down syndrome

Franziska Meyr1, Gabriele Escherich, Georg Mann

  • 1Paediatric Haematology/Oncology, Charité-Universitätsmedizin Berlin, Augustenburger Platz 1, Berlin, Germany. Franziska.Meyr@charite.de

Insights

Children with Down syndrome (DS) treated for relapsed acute lymphoblastic leukemia (ALL) had lower survival rates due to treatment complications. However, recent outcomes show no significant difference, suggesting improved care for pediatric leukemia patients with DS.

Area of Science:

  • Pediatric Oncology
  • Hematology
  • Clinical Genetics

Background:

  • Children with Down syndrome (DS) face increased risks for acute lymphoblastic leukemia (ALL) and chemotherapy side effects.
  • Relapsed ALL in children necessitates optimized treatment protocols, particularly for vulnerable populations like those with DS.

Purpose of the Study:

  • To compare outcomes and treatment tolerance for children with DS and relapsed ALL versus a matched cohort without DS.
  • To identify prognostic factors influencing survival in pediatric patients with relapsed ALL and DS.

Main Methods:

  • Retrospective analysis of 49 children with DS and 98 matched controls treated for relapsed ALL (1983-2012).
  • Comparison of event-free survival (EFS) and overall survival (OS) rates.
  • Analysis of causes of treatment failure, including induction deaths and treatment-related mortality.

Main Results:

  • Children with DS had significantly lower EFS (17%) and OS (17%) compared to controls (41% and 51%, respectively).
  • Fatal treatment complications were more frequent in children with DS (34% vs. 10%).
  • In the last decade, EFS and OS differences between groups diminished, with EFS at 31% for DS and 36% for controls, and OS at 31% for DS and 53% for controls.

Conclusions:

  • Down syndrome is an independent negative prognostic factor for relapsed ALL outcomes.
  • Induction deaths and treatment-related mortality are primary barriers to successful relapse therapy in children with DS.
  • Recent therapeutic advancements have improved outcomes for children with DS and relapsed ALL, narrowing the survival gap.

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