Evidence map›Paper›PMID 41154380›Full record

ReviewCancers2025

Molecularly Targeted Small Molecule Inhibitor Therapy for Pediatric Acute Lymphoblastic Leukemia: A Comprehensive Review of Clinical Trials.

Nicolò Peccatori, Erica Brivio, Andrej Lissat, Francisco Bautista Sirvent, Elisabeth Salzer, Andrea Biondi, Grazia Fazio, Carmelo Rizzari, Sarah K Tasian, Christian Michel Zwaan

Abstract readReview
In one paragraph

Review in Cancers, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Review
  2. Review
  3. 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

10 authors.

Nicolò PeccatoriTettamanti Center, Fondazione IRCCS San Gerardo dei Tintori, 20900 Monza, Italy.ORCID 0009-0004-9779-4165
Erica BrivioPrincess Máxima Center for Pediatric Oncology, 3584 CS Utrecht, The Netherlands.
Andrej LissatCharité Universitätsmedizin, 10117 Berlin, Germany.
Francisco Bautista SirventPrincess Máxima Center for Pediatric Oncology, 3584 CS Utrecht, The Netherlands.ORCID 0000-0002-0421-8862
Elisabeth SalzerWillem Alexander Children's Hospital, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.
Andrea BiondiTettamanti Center, Fondazione IRCCS San Gerardo dei Tintori, 20900 Monza, Italy.ORCID 0000-0002-6757-6173
Grazia FazioTettamanti Center, Fondazione IRCCS San Gerardo dei Tintori, 20900 Monza, Italy.ORCID 0000-0001-7077-8422
Carmelo RizzariSchool of Medicine and Surgery, University of Milano-Bicocca, 20126 Milan, Italy.
Sarah K TasianPrincess Máxima Center for Pediatric Oncology, 3584 CS Utrecht, The Netherlands.
Christian Michel ZwaanPrincess Máxima Center for Pediatric Oncology, 3584 CS Utrecht, The Netherlands.

Funding

U24-Uncovering the Shared Genetic Origins of Childhood Cancer and Structural Birth Defects Through Enhanced Data Integration and Analysis with the CFDE Data Distillery Knowledge Graph.U24OD038422 · OD · CHILDREN'S HOSP OF PHILADELPHIA · PI DISKIN, SHARON, GOLDMUNTZ, ELIZABETH · 2024 to 2025
$4.4M
Identifying Relapse Predictors and Therapeutic Vulnerabilities in Ph+ and Ph-like Acute Lymphoblastic LeukemiaR01CA293587 · NCI · SEATTLE CHILDREN'S HOSPITAL · PI LOH, MIGNON LEE-CHEUN, TASIAN, SARAH KATHLEEN · 2025 to 2025
$3.5M
NCI NIH HHS R01 CA293587NIH HHS U24 OD038422
6 · The paper itself

Abstract

In the past decades, significant advancements in the biological and genetic characterization of acute leukemias and optimization of risk-adapted multi-agent treatment protocols have dramatically improved cure rates and quality of life for children with acute lymphoblastic leukemia (ALL). Despite these optimal results, patients with relapsed or chemotherapy-refractory (R/R) disease or with high-risk genetic features still face unsatisfactory outcomes. Further intensification of conventional chemotherapy has reached its limits in achieving the desired efficacy without undue side effects, necessitating innovative approaches to improve cure rates while continuing to minimize the toxicities associated with chemotherapy and hematopoietic stem cell transplantation. In the era of precision medicine, two key therapeutic strategies have emerged in hemato-oncology: molecularly targeted therapies and immunotherapies. Antibody-based and cellular immunotherapies have undoubtedly reshaped the landscape of childhood ALL treatment and have significant potential to play leading roles in current and future frontline regimens; these important therapies are well delineated in recent reviews. Molecularly targeted small molecule inhibitor therapies remain a cornerstone of precision medicine, supported by recent advancements in next-generation sequencing, which have enabled the application of transcriptomic and genomic profiling data to risk stratification and therapy optimization. Clinical trials for children with ALL have been instrumental in refining therapies and improving outcomes, a paradigm that remains critical as treatment strategies become increasingly complex. This comprehensive review focuses upon molecularly targeted therapy approaches for childhood ALL and aims to summarize findings from completed clinical trials to highlight the current landscape of ongoing and upcoming trials and to provide insights into future directions for the precision-driven optimization of pediatric B-ALL and T-ALL treatment.

Indexed as

acute lymphoblastic leukemiaclinical trialsinhibitorpediatricprecision medicinerelapsed/refractorytargeted therapy

Identifiers

PMID41154380
PMCPMC12562350

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.