Evidence map›Paper›PMID 39011126›Full record

ArticleHemaSphere2024

Oncogenic and immunological targets for matched therapy of pediatric blood cancer patients: Dutch iTHER study experience.

Judith M Boer, Uri Ilan, Aurélie Boeree, Karin P S Langenberg, Jan Koster, Marco J Koudijs, Jayne Y Hehir-Kwa, Stefan Nierkens, Corinne Rossi, Jan J Molenaar and 3 more

Abstract read
In one paragraph

Article in HemaSphere, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed, 1 pooled it
–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

2 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. 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

13 authors.

Judith M BoerPrincess Máxima Center for Pediatric Oncology Utrecht The Netherlands.ORCID https://orcid.org/0000-0003-4848-7789
Uri IlanPrincess Máxima Center for Pediatric Oncology Utrecht The Netherlands.
Aurélie BoereePrincess Máxima Center for Pediatric Oncology Utrecht The Netherlands.
Karin P S LangenbergPrincess Máxima Center for Pediatric Oncology Utrecht The Netherlands.
Jan KosterAmsterdam UMC University of Amsterdam, Center for Experimental and Molecular Medicine, Laboratory of Experimental Oncology and Radiobiology Amsterdam The Netherlands.
Marco J KoudijsPrincess Máxima Center for Pediatric Oncology Utrecht The Netherlands.
Jayne Y Hehir-KwaPrincess Máxima Center for Pediatric Oncology Utrecht The Netherlands.
Stefan NierkensPrincess Máxima Center for Pediatric Oncology Utrecht The Netherlands.
Corinne RossiDepartment of Pediatric Oncology, Hematology, and Immunology Heidelberg University Hospital Heidelberg Germany.
Jan J MolenaarPrincess Máxima Center for Pediatric Oncology Utrecht The Netherlands.
Bianca F GoemansPrincess Máxima Center for Pediatric Oncology Utrecht The Netherlands.
Monique L den BoerPrincess Máxima Center for Pediatric Oncology Utrecht The Netherlands.
C Michel ZwaanPrincess Máxima Center for Pediatric Oncology Utrecht The Netherlands.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Over the past 10 years, institutional and national molecular tumor boards have been implemented for relapsed or refractory pediatric cancer to prioritize targeted drugs for individualized treatment based on actionable oncogenic lesions, including the Dutch iTHER platform. Hematological malignancies form a minority in precision medicine studies. Here, we report on 56 iTHER leukemia/lymphoma patients for which we considered cell surface markers and oncogenic aberrations as actionable events, supplemented with ex vivo drug sensitivity for six patients. Prior to iTHER registration, 34% of the patients had received allogeneic hematopoietic cell transplantation (HCT) and 18% CAR-T therapy. For 51 patients (91%), a sample with sufficient tumor percentage (≥20%) required for comprehensive diagnostic testing was obtained. Up to 10 oncogenic actionable events were prioritized in 49/51 patients, and immunotherapy targets were identified in all profiled patients. Targeted treatment(s) based on the iTHER advice was given to 24 of 51 patients (47%), including immunotherapy in 17 patients, a targeted drug matching an oncogenic aberration in 12 patients, and a drug based on ex vivo drug sensitivity in one patient, resulting in objective responses and a bridge to HCT in the majority of the patients. In conclusion, comprehensive profiling of relapsed/refractory hematological malignancies showed multiple oncogenic and immunotherapy targets for a precision medicine approach, which requires multidisciplinary expertise to prioritize the best treatment options for this rare, heavily pretreated pediatric population.

Identifiers

PMID39011126
PMCPMC11247331

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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 OpenQuestion graph.