Evidence map›Paper›PMID 40549985›Full record

ArticleJournal of clinical oncology : official journal of the American Society of Clinical Oncology2025

Phase I Study of

Thomas Cash, Araz Marachelian, Steven G DuBois, Yueh-Yun Chi, Anahit Baregamyan, Susan G Groshen, Hunter C Jonus, Anasheh Shamirian, Mary Crowley, Fariba Goodarzian and 19 more

Abstract readClinical Trial, Phase I
In one paragraph

Article in Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing 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

9 citing papers in PubMed.

  1. Article
  2. Review
  3. Review
  4. Article
  5. Article
  6. Article
  7. Review
  8. Review
  9. Article
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

29 authors.

Thomas CashDepartment of Pediatrics, Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA.ORCID 0000-0002-1128-784X
Araz MarachelianDepartment of Pediatrics, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA.
Steven G DuBoisDepartment of Pediatrics, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA.ORCID 0000-0003-0882-738X
Yueh-Yun ChiDepartment of Pediatrics, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA.
Anahit BaregamyanDepartment of Pediatrics, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA.
Susan G GroshenDepartment of Preventive Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA.ORCID 0000-0001-9705-1012
Hunter C JonusDepartment of Pediatrics, Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA.
Anasheh ShamirianDepartment of Pediatrics, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA.
Mary CrowleyDepartment of Pediatrics, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA.
Fariba GoodarzianDepartment of Radiology, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA.
Patricia T AcharyaDepartment of Radiology, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA.
Bruce PawelDepartment of Pathology and Laboratory Medicine, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA.ORCID 0000-0001-7678-5324
Amy K ErbeDepartments of Pediatrics, Human Oncology, or Biostatistics, University of Wisconsin-Madison School of Medicine and Public Health, Madison, WI.ORCID 0000-0003-1175-4062
Ankita ShahiDepartments of Pediatrics, Human Oncology, or Biostatistics, University of Wisconsin-Madison School of Medicine and Public Health, Madison, WI.
Jen ZaborekDepartments of Pediatrics, Human Oncology, or Biostatistics, University of Wisconsin-Madison School of Medicine and Public Health, Madison, WI.ORCID 0000-0002-5247-0632
Eleanor KennedyDepartments of Pediatrics, Human Oncology, or Biostatistics, University of Wisconsin-Madison School of Medicine and Public Health, Madison, WI.
Shahab AsgharzadehDepartment of Pediatrics, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA.ORCID 0000-0002-0510-3848
Judith G VillablancaDepartment of Pediatrics, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA.
Navin PintoDepartment of Pediatrics, Seattle Children's Hospital, University of Washington School of Medicine, Seattle, WA.ORCID 0000-0002-8775-6710
Brian D WeissDepartment of Pediatrics, Division of Oncology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.ORCID 0000-0001-5154-3134
Yael P MosseDepartment of Pediatrics, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.ORCID 0000-0002-3463-0399
Ami V DesaiDepartment of Pediatrics, University of Chicago, Chicago, IL.ORCID 0000-0001-9658-3156
Margaret E MacyDepartment of Pediatrics, Children's Hospital Colorado, University of Colorado Anschutz Medical Campus, Aurora, CO.ORCID 0000-0001-6446-9180
Meaghan GrangerCook Children's Medical Center, Fort Worth, TX.
Kieuhoa T VoDepartment of Pediatrics, UCSF Benioff Children's Hospital, University of California San Francisco School of Medicine, San Francisco, CA.ORCID 0000-0002-9281-7485
Paul M SondelDepartments of Pediatrics, Human Oncology, or Biostatistics, University of Wisconsin-Madison School of Medicine and Public Health, Madison, WI.ORCID 0000-0002-0981-8875
Katherine K MatthayDepartment of Pediatrics, UCSF Benioff Children's Hospital, University of California San Francisco School of Medicine, San Francisco, CA.ORCID 0000-0002-0146-2499
Julie R ParkDepartment of Pediatrics, St Jude Children's Research Hospital, Memphis, TN.ORCID 0000-0002-6777-9165
Kelly C GoldsmithDepartment of Pediatrics, Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA.ORCID 0000-0001-8672-2298

Funding

Winship Cancer Institute Cancer Center Support GrantP30CA138292 · NCI · EMORY UNIVERSITY · PI Suresh S Ramalingam · 2009 to 2026
$47.5M
Tumor microenvironment-dependent therapy resistanceP01CA217959 · NCI · CHILDREN'S HOSP OF PHILADELPHIA · PI JOHN M MARIS · 2017 to 2026
$20.7M
NCI NIH HHS P01 CA217959NCI NIH HHS P30 CA138292
6 · The paper itself

Abstract

purposeWe conducted a phase I trial to determine the safety, tolerability, and preliminary antitumor activity of

methodsIn part A, patients with MIBG-avid rNBL received MIBG intravenously (IV) on day 1 at 12, 15, or 18 mCi/kg per the rolling six design and dinutuximab (17.5 mg/m

resultsForty-five eligible patients enrolled, of whom 31 were evaluable. The median age was 7.5 (range, 2.9-24.1) years. For part A (n = 19), no dose-limiting toxicities (DLTs) occurred across all dose levels and courses, establishing the RP2D of MIBG to be 18 mCi/kg. In part B (n = 12), 1 DLT (grade 3 hypokalemia) occurred during course 1, and 3 of 11 patients who received a second course experienced DLT: grade 3 ALT increase, grade 4 hypoxia and grade 5 pneumonitis, and grade 3 fatigue. The best overall response rate (BORR; complete response [CR] + partial response [PR]) on part A was 42% with a CR/PR/minor response (MR) rate of 46%, and 19% progressive disease (PD) rate. For part B, the BORR was 42% with a CR/PR/MR rate of 75% and 0% PD rate.

conclusionMIBG combined with dinutuximab was well tolerated with encouraging antitumor activity. Vorinostat added to this combination may augment responses in this heavily pretreated patient population.

Indexed as

3-IodobenzylguanidineAntineoplastic Combined Chemotherapy ProtocolsNeuroblastomaAdolescentAdultAntibodies, MonoclonalChildChild, PreschoolFemaleHumansInfantMaleNeoplasm Recurrence, LocalVorinostat3-IodobenzylguanidineAntibodies, MonoclonaldinutuximabVorinostat

Identifiers

PMID40549985
PMCPMC12226806

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.