Evidence map›Paper›PMID 38917371›Full record

ArticleJournal of clinical oncology : official journal of the American Society of Clinical Oncology2024

US Food and Drug Administration Approval Summary: Eflornithine for High-Risk Neuroblastoma After Prior Multiagent, Multimodality Therapy.

Elizabeth S Duke, Diana Bradford, Arup K Sinha, Pallavi S Mishra-Kalyani, Catherine C Lerro, Donna Rivera, Emily Wearne, Claudia P Miller, John Leighton, Hairat Sabit and 8 more

Registry-linked trialAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT07278674 (Clinical Study of Eflornithine After Immunotherapy for High-risk Neuroblastoma（CSHEIN）), which is not on this map. Cited by 12 papers.

0numbers the graph read from it
0cells of the map it votes in
12citing 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.

NCT07278674 active not recruitingnot on this mapstarted 2025, after this paper: background citation

Clinical Study of Eflornithine After Immunotherapy for High-risk Neuroblastoma（CSHEIN）

Typeobservational_patient_registrySponsorShanghai Children's HospitalRan2025 to 2029Enrolled20ConditionsHigh Risk NeuroblastomaArmsOral administration of eflornithine for the prevention of high-risk neuroblastoma
3 · Its place in the literature

Who cites it

12 citing papers in PubMed.

  1. Review
  2. Article
  3. Article
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  5. Article
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  8. Article
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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.

Elizabeth S DukeCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.ORCID 0000-0002-3361-1436
Diana BradfordCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Arup K SinhaCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Pallavi S Mishra-KalyaniCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Catherine C LerroOncology Center of Excellence, US Food and Drug Administration, Silver Spring, MD.
Donna RiveraOncology Center of Excellence, US Food and Drug Administration, Silver Spring, MD.ORCID 0000-0002-4565-4556
Emily WearneCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Claudia P MillerCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
John LeightonCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Hairat SabitCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Hong ZhaoCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Ashley LaneCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Barbara ScepuraCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Richard PazdurCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.ORCID 0000-0002-4771-9923
Harpreet SinghCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.ORCID 0000-0003-1526-0916
Paul G KluetzCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Martha DonoghueOncology Center of Excellence, US Food and Drug Administration, Silver Spring, MD.
Nicole DreznerCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.

Funding

Intramural FDA HHS FD999999
6 · The paper itself

Abstract

On December 13, 2023, the US Food and Drug Administration (FDA) approved eflornithine (IWILFIN, US WorldMeds) to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma who have demonstrated at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy. The approval was based on an externally controlled trial (ECT) consisting of a single-arm trial, study 3(b), compared with an external control (EC) derived from a National Cancer Institute/Children's Oncology Group-sponsored clinical trial (Study ANBL0032) and supported by confirmatory evidence. In the protocol-specified primary analysis, the event-free survival hazard ratio (HR) was 0.48 (95% CI, 0.27 to 0.85) and overall survival HR was 0.32 (95% CI, 0.15 to 0.70). The most common adverse reactions (≥5%) were hearing loss, otitis media, pyrexia, pneumonia, and diarrhea. Notably, this is the first oncology drug approval which relies on an ECT as the primary clinical data to support substantial evidence of effectiveness. This was made possible by a distinctly high-quality, comparable EC data set with consistent treatment effect estimations demonstrated in multiple sensitivity and supportive analyses. Eflornithine's manageable safety profile and strong nonclinical and mechanistic data provided further support for the approval, and the evidentiary package was evaluated in the context of high unmet need in a rare, life-threatening cancer.

Indexed as

Drug ApprovalEflornithineNeuroblastomaUnited States Food and Drug AdministrationAdolescentAdultChildChild, PreschoolCombined Modality TherapyFemaleHumansInfantMaleUnited StatesEflornithine

Identifiers

PMID38917371
PMCPMC11365752

What OpenQuestion holds

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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.