Evidence map›Paper›PMID 41823156›Full record

ArticlePediatric blood & cancer2026

Efficacy and Tolerability of Topotecan/Cyclophosphamide/Dinutuximab in Relapsed and Refractory High-Risk Neuroblastoma: A Multi-Institutional Retrospective Study.

Benjamin J Lerman, Madeleine King, Rajen Mody, Sara-Jane Onyeama, Araz Marachelian, Thomas Cash, Sara M Federico, Meaghan Granger, Ami V Desai, Suzanne Shusterman and 8 more

Abstract readMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Article in Pediatric blood & cancer, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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.

Benjamin J LermanDepartment of Pediatrics, University of California San Francisco and Benioff Children's Hospitals, San Francisco, California, USA.
Madeleine KingPediatric Hematology Oncology, C.S. Mott Children's Hospital, Ann Arbor, Michigan, USA.
Rajen ModyPediatric Hematology Oncology, C.S. Mott Children's Hospital, Ann Arbor, Michigan, USA.
Sara-Jane OnyeamaDepartment of Pediatrics, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, California, USA.
Araz MarachelianDepartment of Pediatrics, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, California, USA.
Thomas CashDepartment of Pediatrics, Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA.
Sara M FedericoDepartment of Pediatrics, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
Meaghan GrangerDepartment of Pediatrics, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
Ami V DesaiDepartment of Pediatrics, University of Chicago, Chicago, Illinois, USA.
Suzanne ShustermanDepartment of Pediatrics, Dana-Farber/Boston Children's Cancer and Blood Disorders Centers, Harvard Medical School, Boston, Massachusetts, USA.
Jennifer H FosterPediatric Hematology/Oncology, Texas Children's Hospital, Houston, Texas, USA.
Elizabeth SokolDivision of Pediatric Hematology, Oncology, Neuro-oncology, and Stem Cell Transplant, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern Feinberg School of Medicine, Chicago, Illinois, USA.
Chelsee GreerDepartment of Pediatrics, Cook Children's Medical Center, Fort Worth, Texas, USA.
Keri StrebyDepartment of Pediatrics, Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus, Ohio, USA.
Holly J MeanyCenter for Cancer and Blood Disorders, Children's National Hospital, Washington, Columbia, USA.
Kieuhoa T VoDepartment of Pediatrics, University of California San Francisco and Benioff Children's Hospitals, San Francisco, California, USA.
Margaret E MacyDepartment of Pediatrics, University of Colorado Anschutz Medical Campus and Children's Hospital Colorado, Aurora, Colorado, USA.
Rochelle BagatellDivision of Oncology, Department of Pediatrics, Perelman School of Medicine, Children's Hospital of Philadelphia, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Funding

Training Program in Translational Brain Tumor ResearchT32CA151022 · NCI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI Joseph F Costello · 2010 to 2026
$6.6M
Research Training in Childhood CancerT32CA128583 · NCI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI Eric Alejandro Sweet-Cordero · 2007 to 2026
$6.0M
UCSF Paul Calabresi K12 Career Development Program in Clinical OncologyK12CA260225 · NCI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI BERGSLAND, EMILY K, BIVONA, TREVER G · 2021 to 2025
$2.6M
Alex's Lemonade Stand Centers of ExcellenceNCI NIH HHS K12 CA260225NCI NIH HHS T32 CA128583NCI NIH HHS T32 CA151022NIH/NCI 5T32CA128583-17
6 · The paper itself

Abstract

purposeChemoimmunotherapy with irinotecan, temozolomide, and dinutuximab (I/T/DIN) has emerged as first-line therapy for relapsed/refractory (r/r) high-risk neuroblastoma (HRNB) in North America. Topotecan and cyclophosphamide (T/C) are often used in combination with dinutuximab in the setting of lack of response, progression, or incomplete response following I/T/DIN, but efficacy and tolerability are unknown.

methodsEligible patients received one or more cycles of T/C/DIN for r/r HRNB. Response was assessed using the 2017 International Neuroblastoma Response Criteria.

resultsFifty-four patients with r/r HRNB were included. Patients received a median of four cycles of T/C/DIN (range = 1-16); 48 (89%) had previously received I/T/DIN. Twenty-three patients (42.6%) had an objective response (complete/partial/minor; OR) to T/C/DIN, 21 (38.9%) had stable disease, and 10 (18.5%) had progressive disease as best response. Among patients who previously received I/T/DIN, objective response rate (ORR) to T/C/DIN was 54% in those with prior OR to I/T/DIN and 23% in those without prior OR (adjusted odds ratio 4.40, 95% confidence interval [CI]: 1.09-17.75). Patients who had experienced on-therapy progression with I/T/DIN had a similar ORR to T/C/DIN compared to those who never progressed (38% vs. 41%, p = 1.0). Twenty-one patients (39%) required T/C/DIN dose modification-11 (52%) due to hematologic toxicity. Seven patients (13%) discontinued T/C/DIN due to toxicity.

conclusionT/C/DIN is an effective regimen for patients with r/r HRNB, producing ORRs similar to prior reports in chemoimmunotherapy-naïve patients treated with I/T/DIN. Prior chemoimmunotherapy response may predict response to T/C/DIN, but even patients who progress while receiving I/T/DIN may benefit from the T/C/DIN combination.

Indexed as

Antineoplastic Combined Chemotherapy ProtocolsNeoplasm Recurrence, LocalNeuroblastomaAdolescentAntibodies, MonoclonalChildChild, PreschoolCyclophosphamideDrug Resistance, NeoplasmFemaleFollow-Up StudiesHumansInfantMalePrognosisRetrospective StudiesAntibodies, MonoclonalCyclophosphamidedinutuximabTopotecanchemoimmunotherapydinutuximabimmunotherapyneuroblastoma

Identifiers

PMID41823156
PMCPMC13271835

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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.