Evidence map›Paper›PMID 42151367›Full record

ArticleNPJ precision oncology2026

Lorlatinib monotherapy or combination therapy in anaplastic lymphoma kinase-driven high-risk neuroblastoma.

Weiji Xie, Yu Zhang, Suying Lu, Feifei Sun, Jia Zhu, Yi Que, Junting Huang, Mengjia Song, Zijun Zhen, Juan Wang and 1 more

Abstract read
In one paragraph

Article in NPJ precision oncology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
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

11 authors.

Weiji Xie *State Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Sun Yat-sen University Cancer Center, Guangzhou, PR China.
Yu Zhang *State Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Sun Yat-sen University Cancer Center, Guangzhou, PR China.
Suying LuState Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Sun Yat-sen University Cancer Center, Guangzhou, PR China.
Feifei SunState Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Sun Yat-sen University Cancer Center, Guangzhou, PR China.
Jia ZhuState Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Sun Yat-sen University Cancer Center, Guangzhou, PR China.
Yi QueState Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Sun Yat-sen University Cancer Center, Guangzhou, PR China.
Junting HuangState Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Sun Yat-sen University Cancer Center, Guangzhou, PR China.
Mengjia SongState Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Sun Yat-sen University Cancer Center, Guangzhou, PR China.
Zijun ZhenState Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Sun Yat-sen University Cancer Center, Guangzhou, PR China.
Juan WangState Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Sun Yat-sen University Cancer Center, Guangzhou, PR China. wangjuan@sysucc.org.cn.
Yizhuo ZhangState Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Sun Yat-sen University Cancer Center, Guangzhou, PR China. zhangyzh@sysucc.org.cn.

Funding

National Key Research and Development Program of China 2022YFC2705005National Natural Science Foundation of China 82302940National Natural Science Foundation of China 82573714
6 · The paper itself

Abstract

This study evaluated the clinical efficacy and safety of lorlatinib in 25 patients with anaplastic lymphoma kinase (ALK) -driven neuroblastoma. Among 17 evaluable patients, the objective response rate (ORR) was 64.7% (11/17). All six newly diagnosed high-risk patients who received lorlatinib in combination with induction chemotherapy achieved an objective response. Marked response heterogeneity was observed according to molecular profile: patients with ALK hotspot mutations alone achieved an ORR of 100%, whereas those with concurrent MYCN amplification, rare ALK mutations, or ALK amplification demonstrated significantly reduced efficacy (ORR 25.0%, p = 0.006). Acquired resistance was associated with potential bypass pathway alterations, including BRAF fusions, MET amplification, and NF1 mutations; however, functional validation of these mechanisms is still required. Pulmonary toxicity was observed in patients receiving lorlatinib combined with anti-GD2 immunotherapy or chemotherapy. These preliminary findings suggest potential efficacy of frontline lorlatinib in ALK-driven neuroblastoma, highlight molecular determinants of sensitivity, and reveal challenges of resistance and treatment-related toxicity.

Identifiers

PMID42151367
PMCPMC13447835

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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.