Evidence map›Paper›PMID 38200233›Full record

ArticleBritish journal of cancer2024

Phase 1 study of high-dose DFMO, celecoxib, cyclophosphamide and topotecan for patients with relapsed neuroblastoma: a New Approaches to Neuroblastoma Therapy trial.

Michael D Hogarty, David S Ziegler, Andrea Franson, Yueh-Yun Chi, Denice Tsao-Wei, Kangning Liu, Rohan Vemu, Eugene W Gerner, Elizabeth Bruckheimer, Anasheh Shamirian and 8 more

2 registry-linked trialsOpen access · hybridAbstract readClinical Trial, Phase I
PubMed Full text at PMC Publisher
In one paragraph

Article in British journal of cancer, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
5.9field-weighted citation impact, top 3% 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.

NCT02030964 phase1completednot on this map

N2012-01: Phase 1 Study of Difluoromethylornithine (DFMO) and Celecoxib With Cyclophosphamide/Topotecan for Patients With Relapsed or Refractory Neuroblastoma

TypeinterventionalSponsorNew Approaches to Neuroblastoma Therapy ConsortiumRan2014 to 2023Enrolled30ConditionsNeuroblastomaArmsDFMO, Celecoxib, Cyclophosphamide, Topotecan
NCT03794349 phase2active not recruitingnot on this map

A Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab With or Without Eflornithine (DFMO) in Children With Relapsed, Refractory or Progressive Neuroblastoma

TypeinterventionalSponsorChildren's Oncology GroupRan2019 to 2029Enrolled94ConditionsHigh Risk Neuroblastoma, Recurrent Neuroblastoma, Refractory NeuroblastomaArmsDinutuximab, Eflornithine Hydrochloride, Irinotecan Hydrochloride, Sargramostim, Temozolomide
3 · Its place in the literature

Who cites it

8 citing papers in PubMed, 16 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

18 authors at 8 institutions in 2 countries.

Michael D HogartyDivision of Oncology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA. hogartym@chop.edu.ORCID 0000-0002-9221-4852
David S ZieglerChildren's Cancer Institute, Lowy Cancer Research Centre, Randwick, NSW, Australia.
Andrea FransonDepartment of Pediatrics, University of Michigan Medical School, Ann Arbor, MI, USA.ORCID 0000-0002-5361-7683
Yueh-Yun ChiChildren's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA.
Denice Tsao-WeiDepartment of Preventive Medicine, University of Southern California, Los Angeles, CA, USA.
Kangning LiuDivision of Oncology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Rohan VemuDivision of Oncology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Eugene W GernerCancer Prevention Pharmaceuticals, Tucson, AZ, USA.
Elizabeth BruckheimerPanbela Pharmaceuticals, Inc., Waconia, MN, USA.
Anasheh ShamirianChildren's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA.
Beth HasenauerChildren's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA.
Frank M BalisDivision of Oncology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Susan GroshenChildren's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA.
Murray D NorrisChildren's Cancer Institute, Lowy Cancer Research Centre, Randwick, NSW, Australia.ORCID 0000-0002-0632-4589
Michelle HaberChildren's Cancer Institute, Lowy Cancer Research Centre, Randwick, NSW, Australia.
Julie R ParkSt. Jude Children's Research Hospital, University of Tennessee, Memphis, TN, USA.
Katherine K MatthayUCSF Benioff Children's Hospital, UCSF School of Medicine, University of California San Francisco, San Francisco, CA, USA.
Araz MarachelianChildren's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA.
University of Southern California · USChildren's Hospital of Philadelphia · USChildren's Cancer Institute Australia · AUCancer Prevention Pharmaceuticals (United States) · USSt. Jude Children's Research Hospital · USUniversity of California, San Francisco · USUniversity of Michigan · USUNSW Sydney · AU

Funding

Vascular endothelial cell integrins in angiogenesisP01CA081403 · NCI · CHILDREN'S HOSPITAL OF LOS ANGELES · PI SEEGER, ROBERT CHARLES · 2000 to 2014
$28.5M
Tumor microenvironment-dependent therapy resistanceP01CA217959 · NCI · CHILDREN'S HOSP OF PHILADELPHIA · PI JOHN M MARIS · 2017 to 2026
$20.7M
NCI NIH HHS P01 CA081403NCI NIH HHS P01 CA217959
6 · The paper itself

Abstract

backgroundMYC genes regulate ornithine decarboxylase (Odc) to increase intratumoral polyamines. We conducted a Phase I trial [NCT02030964] to determine the maximum tolerated dose (MTD) of DFMO, an Odc inhibitor, with celecoxib, cyclophosphamide and topotecan.

methodsPatients 2-30 years of age with relapsed/refractory high-risk neuroblastoma received oral DFMO at doses up to 9000 mg/m

resultsTwenty-four patients (median age, 6.8 years) received 136 courses. Slow platelet recovery with 21-day courses (dose-levels 1 and 2) led to subsequent dose-levels using 28-day courses (dose-levels 2a-4a). There were three course-1 dose-limiting toxicities (DLTs; hematologic; anorexia; transaminases), and 23 serious adverse events (78% fever-related). Five patients (21%) completed 1-year of therapy. Nine stopped for PD, 2 for DLT, 8 by choice. Best overall response included two PR and four MR. Median time-to-progression was 19.8 months, and 3 patients remained progression-free at >4 years without receiving additional therapy. The MTD of DFMO with this regimen was 6750 mg/m

conclusionHigh-dose DFMO is tolerable when added to chemotherapy in heavily pre-treated patients. A randomized Phase 2 trial of DFMO added to chemoimmunotherapy is ongoing [NCT03794349].

Indexed as

Neoplasm Recurrence, LocalNeuroblastomaAdolescentAdultAntineoplastic Combined Chemotherapy ProtocolsCelecoxibChildChild, PreschoolCyclophosphamideHumansTopotecanYoung AdultCelecoxibCyclophosphamideTopotecan

Identifiers

PMID38200233
PMCPMC10912730
OpenAlexW4390691382

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

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.