Evidence map›Paper›PMID 38807260›Full record

ArticlePediatric blood & cancer2024

Anaplastic sarcoma of the kidney (DICER1-sarcoma of the kidney): A report from the International Pleuropulmonary Blastoma/DICER1 Registry.

Peter J Schoettler, Caroline C Smith, Miki Nishitani, Anne K Harris, Alexander T Nelson, Dave A Watson, Junne Kamihara, Elizabeth A Mullen, D Ashley Hill, Yoav H Messinger and 6 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Pediatric blood & cancer, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing 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

10 citing papers in PubMed.

  1. Review
  2. Article
  3. Article
  4. Review
  5. Article
  6. Prognostic Significance of GermlineJCO precision oncology · 2025
    Article
  7. Review
  8. Update on Pediatric Surveillance Recommendations for PTEN Hamartoma Tumor Syndrome, DICER1-Related Tumor Predisposition, and Tuberous Sclerosis Complex.Clinical cancer research : an official journal of the American Association for Cancer Research · 2025
    Review
  9. Article
  10. DICER1-Related Tumor Predisposition: Identification of At-risk Individuals and Recommended Surveillance Strategies.Clinical cancer research : an official journal of the American Association for Cancer Research · 2024
    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

16 authors.

Peter J SchoettlerDepartment of Pediatrics, Division of Hematology/Oncology, University of Texas Southwestern Medical Center, Dallas, Texas, USA.
Caroline C SmithDepartment of Pediatrics, Division of Hematology/Oncology, University of Texas Southwestern Medical Center, Dallas, Texas, USA.
Miki NishitaniDana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.ORCID https://orcid.org/0000-0001-9778-1197
Anne K HarrisInternational Pleuropulmonary Blastoma/DICER1 Registry, Children's Minnesota, Minneapolis, Minnesota, USA.
Alexander T NelsonInternational Pleuropulmonary Blastoma/DICER1 Registry, Children's Minnesota, Minneapolis, Minnesota, USA.ORCID https://orcid.org/0000-0002-1611-4577
Dave A WatsonResearch Institute, Children's Minnesota, Minneapolis, Minnesota, USA.
Junne KamiharaDana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.
Elizabeth A MullenDana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.
D Ashley HillInternational Pleuropulmonary Blastoma/DICER1 Registry, Children's Minnesota, Minneapolis, Minnesota, USA.
Yoav H MessingerInternational Pleuropulmonary Blastoma/DICER1 Registry, Children's Minnesota, Minneapolis, Minnesota, USA.ORCID https://orcid.org/0000-0001-7990-1802
Douglas B FairDepartment of Pediatrics, Division of Hematology/Oncology, Primary Children's Hospital, University of Utah, Salt Lake City, Utah, USA.ORCID https://orcid.org/0000-0001-9742-4640
Kiran A KumarDepartment of Pediatrics, Division of Hematology/Oncology, University of Texas Southwestern Medical Center, Dallas, Texas, USA.
Louis P DehnerInternational Pleuropulmonary Blastoma/DICER1 Registry, Children's Minnesota, Minneapolis, Minnesota, USA.
Shifra AshPediatric Hematology-Oncology and Bone Marrow Transplantation, Ruth Rappaport Children's Hospital, Rambam Health Care Campus, Technion-Israel Institute of Technology, Haifa, Israel.ORCID https://orcid.org/0000-0002-0461-1867
Kenneth S ChenDepartment of Pediatrics, Division of Hematology/Oncology, University of Texas Southwestern Medical Center, Dallas, Texas, USA.ORCID https://orcid.org/0000-0003-2304-4631
Kris Ann P SchultzInternational Pleuropulmonary Blastoma/DICER1 Registry, Children's Minnesota, Minneapolis, Minnesota, USA.ORCID https://orcid.org/0000-0002-1788-5832

Funding

DICER1 and the Pleuropulmonary Blastoma Family Cancer SyndromeR01CA143167 · NCI · CHILDREN'S RESEARCH INSTITUTE · PI HILL, DANA ASHLEY · 2010 to 2020
$3.7M
Detecting DICER1: A global partnership to cure pediatric lung cancerR37CA244940 · NCI · CHILDREN'S HOSPITALS AND CLINICS · PI Kris Ann Pinekenstein Schultz · 2020 to 2026
$2.4M
Cancer Prevention and Research Institute of Texas RR180071Children's Minnesota Internal Research Grant Program 20435-47609National Cancer Institute of the National Institutes of Health R01CA143167National Cancer Institute of the National Institutes of Health R37CA244940NCI NIH HHS R01 CA143167NCI NIH HHS R37 CA244940Pine Tree Apple Tennis Classic 21433-47809Rein in Sarcoma Foundation 20435-97066Rein in Sarcoma Foundation 20435-97804
6 · The paper itself

Abstract

backgroundAnaplastic sarcoma of the kidney (ASK) is a DICER1-related neoplasm first identified as a distinctive tumor type through the evaluation of unusual cases of putative anaplastic Wilms tumors. Subsequent case reports identified the presence of biallelic DICER1 variants as well as progression from cystic nephroma, a benign DICER1-related neoplasm. Despite increasing recognition of ASK as a distinct entity, the optimal treatment remains unclear.

methodsIndividuals with known or suspected DICER1-related tumors including ASK were enrolled in the International Pleuropulmonary Blastoma/DICER1 Registry. Additionally, a comprehensive review of reported cases of ASK was undertaken, and data were aggregated for analysis with the aim to identify prognostic factors and clinical characteristics to guide decisions regarding genetic testing, treatment, and surveillance.

resultsTen cases of ASK were identified in the Registry along with 37 previously published cases. Staging data, per Children's Oncology Group guidelines, was available for 40 patients: 13 were stage I, 12 were stage II, 10 were stage III, and five were stage IV. Outcome data were available for 37 patients. Most (38 of 46) patients received upfront chemotherapy and 14 patients received upfront radiation. Two-year event-free survival (EFS) for stage I-II ASK was 81.8% (95% confidence interval [CI]: 67.2%-99.6%), compared with 46.6% EFS (95% CI: 24.7%-87.8%) for stage III-IV (p = .07). Two-year overall survival (OS) for stage I-II ASK was 88.9% (95% CI: 75.5%-100.0%), compared with 70.0% (95% CI: 46.7%-100.0%) for stage III-IV (p = .20). Chemotherapy was associated with improved EFS and OS with hazard ratios of 0.09 (95% CI: 0.02-0.31) and 0.08 (95% CI: 0.02-0.42), respectively.

conclusionASK is a rare DICER1-related renal neoplasm. In the current report, we identify clinical and treatment-related factors associated with outcome including the importance of chemotherapy in treating ASK. Ongoing data collection and genomic analysis are indicated to optimize outcomes for children and adults with these rare tumors.

Indexed as

DEAD-box RNA HelicasesKidney NeoplasmsPulmonary BlastomaRegistriesRibonuclease IIISarcomaAdolescentChildChild, PreschoolFemaleFollow-Up StudiesHumansInfantMalePrognosisSurvival RateDEAD-box RNA HelicasesDICER1 protein, humanRibonuclease IIIanaplastic sarcoma of the kidneychildhood cancercystic nephromaDICER1pleuropulmonary blastomarenal sarcoma

Identifiers

PMID38807260
PMCPMC11590164

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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.