Evidence map›Paper›PMID 33723397›Full record

ReviewBritish journal of cancer2021

Rationale for the use of tyrosine kinase inhibitors in the treatment of paediatric desmoid-type fibromatosis.

Monika Sparber-Sauer, Daniel Orbach, Fariba Navid, Simone Hettmer, Stephen Skapek, Nadège Corradini, Michela Casanova, Aaron Weiss, Matthias Schwab, Andrea Ferrari

Open access · bronzeAbstract readReview
In one paragraph

Review in British journal of cancer, 2021. 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
3.7field-weighted citation impact, top 6% of its field
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, 30 citations in OpenAlex.

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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

10 authors at 9 institutions in 4 countries.

Monika Sparber-Sauer *Klinikum Stuttgart - Olgahospital, Stuttgart Cancer Center, Zentrum für Kinder-, Jugend- und Frauenmedizin, Pediatrics 5 (Oncology, Hematology, Immunology), Stuttgart, Germany.
Daniel Orbach *SIREDO Oncology Center (Care, Innovation and Research for Children, Adolescents and Young Adults with Cancer), PSL University, Institut Curie, Paris, France. daniel.orbach@curie.fr.ORCID http://orcid.org/0000-0002-2520-139X
Fariba NavidDepartment of Pediatrics, Children's Hospital Los Angeles, University of Southern California, Keck School of Medicine, Los Angeles, CA, USA.
Simone HettmerDepartment of Pediatrics and Adolescent Medicine, Division of Pediatric Hematology and Oncology, Faculty of Medicine, University of Freiburg, Freiburg, Germany.ORCID http://orcid.org/0000-0003-1709-4448
Stephen SkapekDepartment of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA.
Nadège CorradiniInstitut d'Hematologie et d'Oncologie Pediatrique, Centre Leon Berard, Lyon, France.
Michela CasanovaPediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale Tumori, Milano, Italy.
Aaron WeissDepartment of Pediatrics, Maine Medical Center, Portland, ME, USA.
Matthias SchwabDr. Margarete Fischer-Bosch Institute of Clinical Pharmacology, Stuttgart, Germany.
Andrea FerrariPediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale Tumori, Milano, Italy.
Fondazione IRCCS Istituto Nazionale dei Tumori · ITDr. Margarete Fischer-Bosch-Institute of Clinical Pharmacology · DEInstitut d’Hématologie et d’Oncologie Pédiatrique · FRMaine Medical Center · USOlgahospital · DEThe University of Texas Southwestern Medical Center · USUniversité Paris Sciences et Lettres · FRUniversity of Freiburg · DEUniversity of Southern California · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

In children with desmoid-type fibromatosis (DTF) in whom disease progression occurs after an initial watch-and-wait strategy, prolonged low-dose chemotherapy using vinblastine and methotrexate (VBL-MTX) is currently the standard of care. These conventional drugs have been prospectively evaluated but their efficacy and safety profiles are limited, and alternative therapeutic options are therefore essential. Based on the results of clinical trials, the use of tyrosine kinase inhibitors (TKIs) in the treatment of DTF is currently considered only in adult patients. TKIs such as imatinib show superior therapeutic efficacy to VBL-MTX and tolerable short-term side effects for the treatment of adult DFT, supporting the concept of the use of TKIs for the treatment of paediatric DFT. Moreover, new-generation TKIs, such as pazopanib and sorafenib, have shown improved therapeutic efficacy compared to imatinib in adult non-comparative studies. A tolerable safety profile of TKI therapy in children with disease entities other than DTF, such as leukaemia, has been reported. However, the efficacy and, in particular, the long-term safety of TKIs, including childhood-specific aspects such as growth and fertility, for the treatment of children with DTF should be investigated prospectively, as DFT therapy requires long-term drug exposure.

Indexed as

AdultAge of OnsetAntineoplastic Combined Chemotherapy ProtocolsChildDesmoid TumorsDrug-Related Side Effects and Adverse ReactionsHumansProtein Kinase InhibitorsProtein Kinase Inhibitors

Identifiers

PMID33723397
PMCPMC8110972
OpenAlexW3138103076

What OpenQuestion holds

Textmetadata
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.