Evidence map›Paper›PMID 37036306›Full record

ArticlePediatric blood & cancer2023

Venetoclax in combination with chemotherapy as treatment for pediatric advanced hematologic malignancies.

Amanda E Marinoff, Kathryn Aaronson, Anurag K Agrawal, Benjamin S Braun, Carla Golden, Benjamin J Huang, Jennifer Michlitsch, Erica Southworth, Allyson Thrall, Kieuhoa T Vo and 1 more

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PubMed Full text at PMC Publisher
In one paragraph

Article in Pediatric blood & cancer, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
3.2field-weighted citation impact, top 8% of its field
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

7 citing papers in PubMed, 11 citations in OpenAlex.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

11 authors at 1 institution in 1 country.

Amanda E MarinoffDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, San Francisco, CA, USA.ORCID 0000-0001-8600-6400
Kathryn AaronsonDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, San Francisco, CA, USA.
Anurag K AgrawalDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, San Francisco, CA, USA.ORCID 0000-0003-4898-9615
Benjamin S BraunDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, San Francisco, CA, USA.ORCID 0000-0001-6996-0833
Carla GoldenDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, San Francisco, CA, USA.
Benjamin J HuangDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, San Francisco, CA, USA.
Jennifer MichlitschDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, San Francisco, CA, USA.
Erica SouthworthDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, San Francisco, CA, USA.
Allyson ThrallDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, San Francisco, CA, USA.
Kieuhoa T VoDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, San Francisco, CA, USA.ORCID 0000-0002-9281-7485
Elliot StieglitzDepartment of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, San Francisco, CA, USA.ORCID 0000-0001-7032-4623
University of California, San Francisco · US

Funding

Research Training in Childhood CancerT32CA128583 · NCI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI Eric Alejandro Sweet-Cordero · 2007 to 2026
$6.0M
Co-targeting BET Bromodomain Proteins and Aberrant Signaling in AML.K08CA256489 · NCI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI HUANG, BENJAMIN · 2021 to 2025
$1.2M
NCI NIH HHS K08 CA256489NCI NIH HHS T32 CA128583
6 · The paper itself

Abstract

backgroundVenetoclax is frequently used as salvage treatment in pediatric, adolescent, and young adult (AYA) patients with advanced hematologic malignancies. However, more data are needed from real-world studies to guide the safe and appropriate use of venetoclax in this population. PROCEDURE: We retrospectively reviewed the medical records of all patients diagnosed with hematologic malignancies less than 30 years of age treated with venetoclax outside of clinical trials at the University of California San Francisco Benioff Children's Hospitals from 2016 to 2022.

resultsWe identified 13 patients (acute myeloid leukemia, n = 8; B-acute lymphoblastic leukemia, n = 3; myelodysplastic syndrome, n = 2) aged 4 months to 27 years. A median of 3 prior lines of therapy weregiven (range 0-5). All patients received venetoclax in combination with either a hypomethylating agent or conventional chemotherapy. Three (23%) patients achieved complete remission (CR); two (15%) achieved partial remission (PR); 3 (23%) had stable disease (SD), and five (42%) had progressive disease. Median survival and time to progression from venetoclax initiation was 9 months (range 2.5-52 months) and 3 months (range 2 weeks to 7.5 months), respectively. Six patients (46%) developed grade 3 or higher infections while receiving venetoclax, including bacteremia due to atypical organisms, invasive pulmonary infections with Aspergillus, cytomegalovirus (CMV) viremia, skin infections, and encephalitis with bacterial brain abscesses.

conclusionsVenetoclax in combination with hypomethylating agents or cytotoxic chemotherapy was effective in a subset of pediatric/AYA patients with advanced hematologic malignancies, but multiple severe infections were observed, particularly among patients who received venetoclax in combination with chemotherapy. Prospective studies will be required to determine the optimal dose and duration of venetoclax in this population.

Indexed as

Hematologic NeoplasmsLeukemia, Myeloid, AcuteAdolescentAdultAntineoplastic Combined Chemotherapy ProtocolsBridged Bicyclo Compounds, HeterocyclicChildHumansProspective StudiesRetrospective StudiesSulfonamidesYoung AdultBridged Bicyclo Compounds, HeterocyclicSulfonamidesvenetoclaxadolescent and young adultsinfectionleukemiapediatricvenetoclax

Identifiers

PMID37036306
PMCPMC10133180
OpenAlexW4363645402

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

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.