Evidence map›Paper›PMID 42049404›Full record

ArticleIn vivo (Athens, Greece)

Comparison of Early Response to Selumetinib Therapy in Adolescents and Children With NF1-related Plexiform Neurofibromas.

Jagoda Styczyńska, Agata Marjańska, Agnieszka Jatczak-Gaca, Marek Karwacki, Justyna Walenciak, Katarzyna Garus, Katarzyna Bilska, Agnieszka Mizia-Malarz, Wanda Badowska, Dorota Sławińska and 7 more

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

Article in In vivo (Athens, Greece). The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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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

17 authors.

Jagoda StyczyńskaDepartment of Pediatric Hematology, Oncology, Immunology and Transplantology, Collegium Medicum, Nicolaus Copernicus University Torun, Jurasz University Hospital, Bydgoszcz, Poland; jagasadlok@gmail.com.
Agata MarjańskaDepartment of Pediatric Hematology, Oncology, Immunology and Transplantology, Collegium Medicum, Nicolaus Copernicus University Torun, Jurasz University Hospital, Bydgoszcz, Poland.
Agnieszka Jatczak-GacaDepartment of Pediatric Hematology, Oncology, Immunology and Transplantology, Collegium Medicum, Nicolaus Copernicus University Torun, Jurasz University Hospital, Bydgoszcz, Poland.
Marek KarwackiDepartment of Pediatric Hematology and Oncology, Clinical Transplantology and Pediatrics, Medical University of Warsaw, Warsaw, Poland.
Justyna WalenciakDepartment of Pediatrics, Oncology and Hematology, Medical University, Łódź, Poland.
Katarzyna GarusDepartment of Pediatric Oncology and Hematology, Institute of Pediatrics, Jagiellonian University Medical College, Kraków, Poland.
Katarzyna BilskaDepartment of Oncology and Surgical Oncology for Children and Youth, Institute of Mother and Child, Warsaw, Poland.
Agnieszka Mizia-MalarzDepartment of Oncology, Hematology and Chemotherapy, Division of Pediatrics, Medical University of Silesia, Upper Silesian Child Health Centre, Katowice, Poland.
Wanda BadowskaDepartment of Pediatrics and Hematology and Oncology, Province Children's Hospital, Olsztyn, Poland.
Dorota SławińskaDepartment of Pediatric Haematology and Oncology and Transplantology, Medical University, Lublin, Poland.
Jadwiga Węcławek-TompolDepartment of Pediatric Hematology/Oncology and BMT, Medical University, Wrocław, Poland.
Radosław ChaberDepartment of Pediatrics, Institute of Medical Sciences, Collegium Medicum, University of Rzeszów, Rzeszów, Poland.
Ewa BieńDepartment of Pediatrics, Hematology and Oncology, Medical University, Gdańsk, Poland.
Michalina JezierskaDepartment of Pediatrics, Hematology and Oncology, Medical University, Gdańsk, Poland.
Grażyna KarolczykDepartment of Pediatric Oncology and Hematology, Children's Hospital in Kielce, Kielce, Poland.
Jolanta Skalska-SadowskaDepartment of Pediatric Oncology, Hematology and Transplantology, Poznań University of Medical Sciences, Poznań, Poland.
Jan StyczyńskiDepartment of Pediatric Hematology, Oncology, Immunology and Transplantology, Collegium Medicum, Nicolaus Copernicus University Torun, Jurasz University Hospital, Bydgoszcz, Poland.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

BACKGROUND/

aimFollowing approval from the European Medicines Agency, selumetinib has emerged as a targeted therapy for pediatric patients aged ≥3 years with inoperable, symptomatic NF1-related plexiform neurofibromas (PNs). In Poland, this treatment has been reimbursed since January 2024 under a national therapeutic program funded by the National Health Service. Nevertheless, the efficacy and safety of selumetinib in subgroups within the pediatric population remain unclear. This study aimed to evaluate the efficacy and safety of selumetinib treatment in adolescents (≥16 years old) compared with younger children with NF1-related PNs. PATIENTS AND

methodsA total of 111 patients were included in the program over a two-year period. Patients were divided into two groups according to age: adolescents (≥16 years of age) and children (<16 years of age). Demographic characteristics, PN location, treatment efficacy and adverse effects were analyzed and compared between the two groups.

resultsResponse rates at the first and second evaluation time point were similar between the groups. However, at the third time point, the response rate was significantly higher in children

conclusionEarly response to selumetinib therapy is achieved faster in children than in adolescents. Selumetinib is generally well tolerated in both age groups.

Indexed as

BenzimidazolesNeurofibroma, PlexiformNeurofibromatosis 1AdolescentChildChild, PreschoolFemaleHumansMaleTreatment OutcomeAZD 6244BenzimidazolesadolescentschildrenNeurofibromatosis type 1plexiform neurofibromaselumetinib

Identifiers

PMID42049404
PMCPMC13133820

What Socratic holds

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LicenceCC BY-NC-ND
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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 Socratic graph.