Evidence map›Paper›PMID 40749151›Full record

ArticleJCO precision oncology2025

Mucin 16-Directed Therapy in Pediatric Sarcomas: Case Evidence of Ubamatamab Efficacy in Epithelioid Sarcoma and Its Implications for Other Sarcoma Subtypes.

Denise M Connolly, Gabriel Revon-Rivière, Rose Chami, Denise Mills, Ailish C Coblentz, Thomas S Uldrick, David A Knorr, Priscila Goncalves, Michael Dobosz, Sumreen Jalal and 2 more

Registry-linked trialAbstract readCase Reports
In one paragraph

Article in JCO precision oncology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06444880 (Phase II Trial of Ubamatamab Alone or in Combination With Cemiplimab in MUC16-Expressing SMARCB1-Deficient Malignancies), which is not on this map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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.

NCT06444880 phase2active not recruitingnot on this map

Phase II Trial of Ubamatamab Alone or in Combination With Cemiplimab in MUC16-Expressing SMARCB1-Deficient Malignancies

TypeinterventionalSponsorM.D. Anderson Cancer CenterRan2024 to 2028Enrolled15ConditionsSMARCB1-Deficient Malignancies, Epithelioid Sarcoma, Renal Medullary CarcinomaArmsUbamatamab, Cemiplimab
3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Review
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

12 authors.

Denise M ConnollyDivision of Hematology/Oncology, Department of Pediatrics, The Hospital for Sick Children and University of Toronto, Toronto, ON, Canada.ORCID 0009-0009-7379-3439
Gabriel Revon-RivièreDivision of Hematology/Oncology, Department of Pediatrics, The Hospital for Sick Children and University of Toronto, Toronto, ON, Canada.ORCID 0000-0001-6512-1004
Rose ChamiDepartment of Pediatric Pathology and Laboratory Medicine, The Hospital for Sick Children and the University of Toronto, Toronto, ON, Canada.
Denise MillsDivision of Hematology/Oncology, Department of Pediatrics, The Hospital for Sick Children and University of Toronto, Toronto, ON, Canada.
Ailish C CoblentzDepartment of Diagnostic Imaging, The Hospital for Sick Children, Toronto, ON, Canada.ORCID 0000-0002-4073-5939
Thomas S UldrickRegeneron Pharmaceuticals, Inc, Tarrytown, NY.ORCID 0000-0001-6959-0924
David A KnorrRegeneron Pharmaceuticals, Inc, Tarrytown, NY.
Priscila GoncalvesRegeneron Pharmaceuticals, Inc, Tarrytown, NY.
Michael DoboszRegeneron Pharmaceuticals, Inc, Tarrytown, NY.
Sumreen JalalRegeneron Pharmaceuticals, Inc, Tarrytown, NY.
Sarah Cohen-GogoDivision of Hematology/Oncology, Department of Pediatrics, The Hospital for Sick Children and University of Toronto, Toronto, ON, Canada.ORCID 0000-0002-8852-1104
Daniel A MorgensternDivision of Hematology/Oncology, Department of Pediatrics, The Hospital for Sick Children and University of Toronto, Toronto, ON, Canada.ORCID 0000-0002-4859-1108

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purposeEpithelioid sarcoma (ES) and malignant rhabdoid tumor (MRT) are rare soft tissue sarcomas with poor prognoses. Although mucin 16 (MUC16) and its soluble form, cancer antigen 125 (CA125), are established as biomarkers and therapeutic targets in ovarian cancer, emerging data suggest MUC16 may also be expressed in ES and MRT. In this study, we present a patient with ES, who demonstrated a response to ubamatamab, a novel bispecific T-cell engager (MUC16xCD3), and analyze treatment resistance after disease progression. Additionally, we examine MUC16 expression across pediatric and adolescent and young adult (AYA) sarcomas, to evaluate the frequency of this target and explore the broader application of ubamatamab in this population. MATERIALS AND

methodsWe performed a retrospective clinical case review and immunohistochemical analysis of pediatric and AYA (0-25 years) sarcoma samples from 2015 to 2021, evaluating MUC16 expression using anti-CA125 immunohistochemistry (IHC) on the DAKO Omnis platform.

resultsA 23-year-old female patient with multiply relapsed metastatic ES, harboring MUC16 expression by IHC and elevated serum CA125, received intravenous ubamatamab (250 mg) once per week as part of a single patient study. After 11 weeks of ubamatamab, a RECIST v1.1 partial response was demonstrated, along with serum CA125 normalization, lasting 43 weeks. During the initial step-up dosing, the patient experienced grade 2 cytokine release syndrome. Treatment-emergent adverse events included grade 2 pleural effusion, pericardial effusion, and palmar-plantar erythrodysesthesia, all resolving without intervention. IHC analysis of retrospective samples showed positive MUC16 staining in six of eight (75%) ES and two of four (50%) MRT samples, with no immunoreactivity observed in other pediatric/AYA sarcoma subtypes.

conclusionMUC16 is frequently detected in ES and MRTs. Ubamatamab is an encouraging anti-MUC16 therapy, demonstrating clinical efficacy. Ongoing trials (ClinicalTrials.gov identifier: NCT06444880) are evaluating ubamatamab in other rare MUC16-positive tumors.

Indexed as

CA-125 AntigenMembrane ProteinsSarcomaAdolescentAdultChildChild, PreschoolFemaleHumansRetrospective StudiesYoung AdultCA-125 AntigenMembrane ProteinsMUC16 protein, human

Identifiers

PMID40749151
PMCPMC12316166

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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.