Evidence map›Paper›PMID 32171069›Full record

ArticleThe Lancet. Oncology2020

Venetoclax in combination with cytarabine with or without idarubicin in children with relapsed or refractory acute myeloid leukaemia: a phase 1, dose-escalation study.

Seth E Karol, Thomas B Alexander, Amit Budhraja, Stanley B Pounds, Kristin Canavera, Lei Wang, Joshua Wolf, Jeffery M Klco, Paul E Mead, Soumyasri Das Gupta and 7 more

Abstract readClinical Trial, Phase I
In one paragraph

Article in The Lancet. Oncology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 91 papers.

0numbers the graph read from it
0cells of the map it votes in
91citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

91 citing papers in PubMed.

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  11. [Allogeneic hematopoietic stem cell transplantation for pediatric acute leukemia harboring theZhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2025
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  14. Haematologica · 2025
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31 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

17 authors.

Seth E KarolDepartment of Oncology, St Jude Children's Research Hospital, Memphis, TN, USA.
Thomas B AlexanderDepartment of Pediatrics, University of North Carolina, Chapel Hill, NC, USA.
Amit BudhrajaDepartment of Cell and Molecular Biology, St Jude Children's Research Hospital, Memphis, TN, USA.
Stanley B PoundsDepartment of Biostatistics, St Jude Children's Research Hospital, Memphis, TN, USA.
Kristin CanaveraDepartment of Psychology, St Jude Children's Research Hospital, Memphis, TN, USA.
Lei WangDepartment of Biostatistics, St Jude Children's Research Hospital, Memphis, TN, USA.
Joshua WolfDepartment of Infectious Diseases, St Jude Children's Research Hospital, Memphis, TN, USA.
Jeffery M KlcoDepartment of Pathology, St Jude Children's Research Hospital, Memphis, TN, USA.
Paul E MeadDepartment of Pathology, St Jude Children's Research Hospital, Memphis, TN, USA.
Soumyasri Das GuptaDepartment of Cell and Molecular Biology, St Jude Children's Research Hospital, Memphis, TN, USA.
Su Y KimAbbVie, North Chicago, IL, USA.
Ahmed Hamed SalemAbbVie, North Chicago, IL, USA; Department of Clinical Pharmacy, Faculty of Pharmacy, Ain Shams University, Cairo, Egypt.
Tammy PalenskiAbbVie, North Chicago, IL, USA.
Norman J LacayoDepartment of Pediatrics, Stanford University, Palo Alto, CA, USA.
Ching-Hon PuiDepartment of Oncology, St Jude Children's Research Hospital, Memphis, TN, USA.
Joseph T OpfermanDepartment of Cell and Molecular Biology, St Jude Children's Research Hospital, Memphis, TN, USA.
Jeffrey E RubnitzDepartment of Oncology, St Jude Children's Research Hospital, Memphis, TN, USA. Electronic address: jeffrey.rubnitz@stjude.org.

Funding

Viral Vector Technology (VVTSR)P30CA021765 · NCI · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI CHARLES ROBERTS · 1985 to 2026
$166.9M
Pathway Specific Combinational Therapy in Poor Prognosis B-Lineage Acute Lymphoblastic LeukemiaR01CA201069 · NCI · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI OPFERMAN, JOSEPH T. · 2016 to 2020
$2.0M
NCI NIH HHS P30 CA021765NCI NIH HHS R01 CA201069
6 · The paper itself

Abstract

backgroundOutcomes for children with relapsed or refractory acute myeloid leukaemia remain poor. The BCL-2 inhibitor, venetoclax, has shown promising activity in combination with hypomethylating agents and low-dose cytarabine in older adults for whom chemotherapy is not suitable with newly diagnosed acute myeloid leukaemia. We aimed to determine the safety and explore the activity of venetoclax in combination with standard and high-dose chemotherapy in paediatric patients with relapsed or refractory acute myeloid leukaemia.

methodsWe did a phase 1, dose-escalation study at three research hospitals in the USA. Eligible patients were aged 2-22 years with relapsed or refractory acute myeloid leukaemia or acute leukaemia of ambiguous lineage with adequate organ function and performance status. During dose escalation, participants received venetoclax orally once per day in continuous 28-day cycles at either 240 mg/m

findingsBetween July 1, 2017, and July 2, 2019, 38 patients were enrolled (aged 3-22 years; median 10 [IQR 7-13]), 36 of whom received combination therapy with dose escalation, with a median follow-up of 7·1 months (IQR 5·1-11·2). The recommended phase 2 dose of venetoclax was found to be 360 mg/m

interpretationThe safety and activity of venetoclax plus chemotherapy in paediatric patients with heavily relapsed and refractory acute myeloid leukaemia suggests that this combination should be tested in newly diagnosed paediatric patients with high-risk acute myeloid leukaemia.

fundingUS National Institutes of Health, American Lebanese Syrian Associated Charities, AbbVie, and Gateway for Cancer Research.

Indexed as

AdolescentAntineoplastic Combined Chemotherapy ProtocolsBridged Bicyclo Compounds, HeterocyclicChildChild, PreschoolCytarabineFemaleHumansIdarubicinLeukemia, Myeloid, AcuteMaleNeoplasm Recurrence, LocalSulfonamidesYoung AdultBridged Bicyclo Compounds, HeterocyclicCytarabineIdarubicinSulfonamidesvenetoclax

Identifiers

PMID32171069
PMCPMC7153631

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.