ETV6::RUNX1 Acute Lymphoblastic Leukemia: how much therapy is needed for cure?
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ETV6::RUNX1 Acute Lymphoblastic Leukemia : how much therapy is needed for cure? / Østergaard, Anna; Fiocco, Marta; de Groot-Kruseman, Hester; Moorman, Anthony V.; Vora, Ajay; Zimmermann, Martin; Schrappe, Martin; Biondi, Andrea; Escherich, Gabriele; Stary, Jan; Imai, Chihaya; Imamura, Toshihiko; Heyman, Mats; Schmiegelow, Kjeld; Pieters, Rob.
I: Leukemia, 2024.Publikation: Bidrag til tidsskrift › Tidsskriftartikel › Forskning › fagfællebedømt
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TY - JOUR
T1 - ETV6::RUNX1 Acute Lymphoblastic Leukemia
T2 - how much therapy is needed for cure?
AU - Østergaard, Anna
AU - Fiocco, Marta
AU - de Groot-Kruseman, Hester
AU - Moorman, Anthony V.
AU - Vora, Ajay
AU - Zimmermann, Martin
AU - Schrappe, Martin
AU - Biondi, Andrea
AU - Escherich, Gabriele
AU - Stary, Jan
AU - Imai, Chihaya
AU - Imamura, Toshihiko
AU - Heyman, Mats
AU - Schmiegelow, Kjeld
AU - Pieters, Rob
N1 - Publisher Copyright: © The Author(s) 2024.
PY - 2024
Y1 - 2024
N2 - 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.
AB - 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.
U2 - 10.1038/s41375-024-02287-7
DO - 10.1038/s41375-024-02287-7
M3 - Journal article
C2 - 38844578
AN - SCOPUS:85195259396
JO - Leukemia
JF - Leukemia
SN - 0887-6924
ER -
ID: 395138150