ETV6::RUNX1 Acute Lymphoblastic Leukemia: how much therapy is needed for cure?

Publikation: Bidrag til tidsskriftTidsskriftartikelForskningfagfællebedømt

  • Anna Østergaard
  • Marta Fiocco
  • Hester de Groot-Kruseman
  • Anthony V. Moorman
  • Ajay Vora
  • Martin Zimmermann
  • Martin Schrappe
  • Andrea Biondi
  • Gabriele Escherich
  • Jan Stary
  • Chihaya Imai
  • Toshihiko Imamura
  • Mats Heyman
  • Schmiegelow, K.
  • Rob Pieters

Recent trials show 5-year survival rates >95% for ETV6::RUNX1 Acute Lymphoblastic Leukemia (ALL). Since treatment has many side effects, an overview of cumulative drug doses and intensities between eight international trials is presented to characterize therapy needed for cure. A meta-analysis was performed as a comprehensive summary of survival outcomes at 5 and 10 years. For drug dose comparison in non-high risk trial arms, risk group distribution was applied to split the trials into two groups: trial group A with ~70% (range: 63.5–75%) of patients in low risk (LR) (CCLSG ALL2004, CoALL 07-03, NOPHO ALL2008, UKALL2003) and trial group B with ~45% (range: 38.7–52.7%) in LR (AIEOP-BFM ALL 2000, ALL-IC BFM ALL 2002, DCOG ALL10, JACLS ALL-02). Meta-analysis did not show evidence of heterogeneity between studies in trial group A LR and medium risk (MR) despite differences in treatment intensity. Statistical heterogeneity was present in trial group B LR and MR. Trials using higher cumulative dose and intensity of asparaginase and pulses of glucocorticoids and vincristine showed better 5-year event-free survival but similar overall survival. Based on similar outcomes between trials despite differences in therapy intensity, future trials should investigate, to what extent de-escalation is feasible for ETV6::RUNX1 ALL.

OriginalsprogEngelsk
TidsskriftLeukemia
Antal sider11
ISSN0887-6924
DOI
StatusE-pub ahead of print - 2024

Bibliografisk note

Funding Information:
A\u00D8 received funding from the Princess M\u00E1xima Center Foundation Talent Programme.

Funding Information:
This study was a collaboration within the I-BFM study network. The Children\u2019s Cancer and Leukemia Study Group Japan, Childhood Acute Lymphoblastic Leukemia Group Germany, Nordic Society of Pediatric Hematology and Oncology, United Kingdom Acute Lymphoblastic Leukemia, Associazione Italiana di Ematologia e Oncologia Pediatrica and Berlin Frankfurt M\u00FCnster, Acute Lymphoblastic Leukemia Intercontinental-Berlin Frankfurt M\u00FCnster, Dutch Childhood Oncology Group and the Japan Childhood Leukemia Study Group contributed to this study. This work is part of the evaluation of ALL-BFM trials in Germany funded by the Deutsche Krebshilfe, Bonn, Germany (No. 50-2698-Schr 1). This work is part of the Danish nation-wide research program Childhood Oncology Network Targeting Research, Organization & Life expectancy (CONTROL) and supported by the Danish Cancer Society (R-257-A14720) and the Danish Childhood Cancer Foundation (2019-5934 and 2020-5769).

Publisher Copyright:
© The Author(s) 2024.

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