Evidence map›Paper›PMID 41405208›Full record

Trial reportCancer2025

Real-world efficacy and adverse events of frontline nilotinib in pediatric chronic myeloid leukemia in chronic phase: A prospective multicenter study from SCCCG[PLUS]-CML 2023.

Zhong Fan, Qi-Wen Chen, Xiu-Ya Huang, Qiang Fu, Hui-Rong Mai, Xing-Jiang Long, Qiao-Ru Li, Li-Li Liu, Ri-Yang Liu, Fei He and 21 more

Abstract readClinical TrialMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Cancer, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Trial
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

31 authors.

Zhong FanDepartment of Pediatrics, First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China.ORCID https://orcid.org/0000-0003-2632-011X
Qi-Wen ChenDepartment of Pediatrics, The First Affiliated Hospital of Nanchang University, Nanchang, China.
Xiu-Ya HuangDepartment of Pediatrics, First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China.
Qiang FuDepartment of Hematology, Wuhan Children's Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Hui-Rong MaiHematology and Oncology Department, Shenzhen Children's Hospital, Shenzhen, China.
Xing-Jiang LongDepartment of Pediatrics, Liuzhou People's Hospital, Liuzhou, China.
Qiao-Ru LiDepartment of Pediatrics, Zhongshan City People's Hospital, Zhongshan, Guangdong, China.
Li-Li LiuDepartment of Pediatrics, Affiliated Hospital of Guangdong Medical University, Zhanjiang, China.
Ri-Yang LiuDepartment of Pediatrics, Huizhou Central People's Hospital, Huizhou, Guangdong, China.
Fei HeDepartment of Hematology and Oncology, Jiangxi Provincial Children's Hospital, Nanchang, China.
Bi-Yun GuoDepartment of Pediatrics, The First Affiliated Hospital of Xiamen University, Xiamen, China.
Min-Hui ZengDepartment of Hematology and Oncology, Children's Medical Center, Hunan Provincial People's Hospital/First Hospital of Hunan Normal University, Changsha, China.ORCID https://orcid.org/0000-0001-7394-6188
Wu-Qing WanDepartment of Pediatrics, The Second Xiangya Hospital of Central South University, Changsha, China.
Wei-Na ZhangDepartment of Hematology, Guangzhou Women and Children's Medical Center, Guangzhou Medical University, Guangzhou, China.
Hai-Xia GuoPediatric Hematology/Oncology, Children's Center, Nanfang Hospital, Southern Medical University, Guangzhou, China.
Dun-Hua ZhouChildren's Medical Center, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, China.
Ya-Wei ZouDepartment of Pediatrics, The First Affiliated Hospital of Guangzhou Medical University, Guangzhou, China.
Wen-Jie XiongDepartment of Hematology, Foresea Life Insurance Guangzhou General Hospital, Guangzhou, China.
Xiao-Bo ZhouInstitute of Pediatric Hemato-Oncology, Shunde Women and Children's Hospital of Guangdong Medical University, Foshan, China.
Hui-Qin ChenDepartment of Pediatrics, The Third Affiliated Hospital, Sun Yat-sen University, Guangzhou, China.
Li-Min LinDepartment of Pediatrics, The Second Affiliated Hospital of Shantou University Medical College, Shantou, China.
Hao-Yan RenDepartment of Pediatric Hematology, Zhuhai Center for Maternal and Child Health Care, Zhuhai, China.
Xin TianDepartment of Hematology, Kunming Children's Hospital, Kunming, Yunnan, China.
Hong-Ying WeiDepartment of Pediatrics, the Second Affiliated Hospital of Guangxi Medical University, Nanning, China.
He-Kui LanDepartment of Pediatrics, Zhujiang Hospital, Southern Medical University, Guangzhou, China.
Jiao JinChildren's Medical Center, Affiliated Hospital of Guizhou Medical University, Guizhou, China.
Cong LiangDepartment of Pediatrics, First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China.
Xue-Qun LuoDepartment of Pediatrics, First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China.
Li-Bin HuangDepartment of Pediatrics, First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China.
Li-Hua YangDepartment of Pediatric Hematology, Zhujiang Hospital, Southern Medical University, Guangzhou, China.
Xiao-Li ZhangDepartment of Pediatrics, First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China.

Funding

Guangdong Basic and Applied Basic Research Foundation 2023A1515220176Medical Science and Technology Research Project of Guangdong Province A2024559Shenzhen Medical Research A2402007
6 · The paper itself

Abstract

backgroundChronic myeloid leukemia (CML) is rare in children and often shows more aggressive features than in adults. Real-world data on the efficacy and safety of frontline nilotinib in pediatric CML in chronic phase (CML-CP) remain limited.

methodsThis prospective multicenter real-world study evaluated the efficacy, safety, and pharmacokinetics of frontline nilotinib in newly diagnosed pediatric CML-CP across 26 hospitals in China between January 2023 and April 2025. Patients received nilotinib 230 mg/m

resultsAmong 59 enrolled patients (median age, 10.6 years), 71.8% (28/39) achieved MMR at 12 cycles and all achieved CCyR at six cycles. Median time to MMR was 6.2 (3.3, 12.2) months, and 2-year EFS was 93.3% (84.4%-100%). Nilotinib trough concentrations (Ctrough) correlated negatively with BCR::ABL1 transcript levels at cycles 6 and 12. Patients with Ctrough >950 ng/mL were three to five times more likely to reach MMR, whereas those with Ctrough ≥1500 ng/mL had a 6.5-fold higher risk of hyperbilirubinemia.

conclusionsFrontline nilotinib thus shows strong efficacy and manageable safety in pediatric CML-CP. Higher drug exposure predicts deeper molecular responses but increases toxicity, supporting the potential role of therapeutic drug monitoring.

Indexed as

Leukemia, Myelogenous, Chronic, BCR-ABL PositiveProtein Kinase InhibitorsPyrimidinesAdolescentChildChild, PreschoolChinaFemaleHumansInfantMaleProspective StudiesTreatment OutcomenilotinibProtein Kinase InhibitorsPyrimidinesmolecular responsenilotinibpediatric chronic myeloid leukemiaplasma trough concentrationreal‐world study

Identifiers

PMID41405208
PMCPMC12710452

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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.