Evidence map›Paper›PMID 37012551›Full record

ArticleNature medicine2023

Lorlatinib with or without chemotherapy in ALK-driven refractory/relapsed neuroblastoma: phase 1 trial results.

Kelly C Goldsmith, Julie R Park, Kimberly Kayser, Jemily Malvar, Yueh-Yun Chi, Susan G Groshen, Judith G Villablanca, Kateryna Krytska, Lillian M Lai, Patricia T Acharya and 25 more

Open access · hybridAbstract readClinical Trial, Phase I
PubMed Full text at PMC Publisher
In one paragraph

Article in Nature medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 77 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
77citing papers in PubMed, 1 pooled it
23.9field-weighted citation impact, top 1% of its field
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

77 citing papers in PubMed, 1 synthesis or guideline pooled it, 116 citations in OpenAlex.

  1. Pooled it
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  12. Targeted therapies in pediatric oncology: A start.Molecular therapy. Oncology · 2026
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17 more citing papers are in PubMed but not listed here.

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

35 authors at 19 institutions in 5 countries.

Kelly C GoldsmithAflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, GA, USA.
Julie R ParkSeattle Children's Hospital, Seattle, WA, USA.
Kimberly KayserDepartment of Psychiatry and Behavioral Sciences, Vanderbilt University Medical Center, Nashville, TN, USA.
Jemily MalvarCancer and Blood Disease Institute, Children's Hospital Los Angeles, Los Angeles, CA, USA.
Yueh-Yun ChiCancer and Blood Disease Institute, Children's Hospital Los Angeles, Los Angeles, CA, USA.
Susan G GroshenKeck School of Medicine, University of Southern California, Los Angeles, CA, USA.
Judith G VillablancaCancer and Blood Disease Institute, Children's Hospital Los Angeles, Los Angeles, CA, USA.
Kateryna KrytskaDivision of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Lillian M LaiDepartment of Radiology, University of Iowa Hospital and Clinics, Iowa City, IA, USA.
Patricia T AcharyaKeck School of Medicine, University of Southern California, Los Angeles, CA, USA.
Fariba GoodarzianKeck School of Medicine, University of Southern California, Los Angeles, CA, USA.
Bruce PawelKeck School of Medicine, University of Southern California, Los Angeles, CA, USA.
Hiroyuki ShimadaDepartment of Pathology and Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
Susan GhazarianCancer and Blood Disease Institute, Children's Hospital Los Angeles, Los Angeles, CA, USA.
Lisa StatesPerelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
Lynley MarshallThe Royal Marsden Hospital, London, UK.
Louis CheslerThe Royal Marsden Hospital, London, UK.ORCID http://orcid.org/0000-0001-7842-2068
Meaghan GrangerCook Children's Medical Center, Fort Worth, TX, USA.
Ami V DesaiDepartment of Pediatrics, Section of Hematology/Oncology/Stem Cell Transplantation, University of Chicago, Chicago, IL, USA.
Rajen ModyDepartment of Pediatrics, University of Michigan, Ann Arbor, MI, USA.
Daniel A MorgensternDivision of Haematology and Oncology, Hospital for Sick Children, Toronto, ON, Canada.ORCID http://orcid.org/0000-0002-4859-1108
Suzanne ShustermanDana-Farber Cancer Institute, Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA, USA.
Margaret E MacyUniversity of Colorado Anschutz Medical Campus, Aurora, CO, USA.ORCID http://orcid.org/0000-0001-6446-9180
Navin PintoSeattle Children's Hospital, Seattle, WA, USA.ORCID http://orcid.org/0000-0002-8775-6710
Gudrun SchleiermacherRTOP (Recherche Translationelle en Oncologie Pédiatrique), INSERM U830, Research Center, PSL Research University, Institut Curie, Paris, France.ORCID http://orcid.org/0000-0002-0133-5879
Kieuhoa VoDepartment of Pediatrics, UCSF Benioff Children's Hospital, University of California, San Francisco School of Medicine, San Francisco, CA, USA.
Holger C ThurmGlobal Product Development, Clinical Pharmacology, Pfizer Oncology, Pfizer, Inc., New York, NY, USA.
Joseph ChenGlobal Product Development, Clinical Pharmacology, Pfizer Oncology, Pfizer, Inc., New York, NY, USA.ORCID http://orcid.org/0000-0002-5998-7765
Marlon LiyanageGlobal Product Development, Clinical Pharmacology, Pfizer Oncology, Pfizer, Inc., New York, NY, USA.
Gerson PeltzGlobal Product Development, Clinical Pharmacology, Pfizer Oncology, Pfizer, Inc., New York, NY, USA.
Katherine K MatthayDepartment of Pediatrics, UCSF Benioff Children's Hospital, University of California, San Francisco School of Medicine, San Francisco, CA, USA.
Esther R BerkoDivision of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
John M MarisDivision of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.ORCID http://orcid.org/0000-0002-8088-7929
Araz MarachelianCancer and Blood Disease Institute, Children's Hospital Los Angeles, Los Angeles, CA, USA.
Yael P MosséDivision of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA. mosse@chop.edu.ORCID http://orcid.org/0000-0002-3463-0399
University of Southern California · USChildren's Hospital of Philadelphia · USPfizer (United States) · USChildren's Hospital of Los Angeles · USInstitute of Cancer Research · GBSeattle Children's Hospital · USUniversity of California, San Francisco · USChildren's Hospital Colorado · USCook Children's Medical Center · USDana-Farber Cancer Institute · USEmory University · USInserm · FRStanford University · USUniversity of Chicago · USUniversity of Iowa Hospitals and Clinics · USUniversity of Michigan · USUniversity of Pennsylvania · USUniversity of Toronto · CAVanderbilt University Medical Center · US

Funding

Tumor microenvironment-dependent therapy resistanceP01CA217959 · NCI · CHILDREN'S HOSP OF PHILADELPHIA · PI JOHN M MARIS · 2017 to 2026
$20.7M
Discovering mechanisms of neuroblastoma tumorigenesis to improve patient outcomesR35CA220500 · NCI · CHILDREN'S HOSP OF PHILADELPHIA · PI JOHN M MARIS · 2017 to 2026
$8.7M
Targeting Oncogenic ALK Signaling in NeuroblastomaR01CA140198 · NCI · CHILDREN'S HOSP OF PHILADELPHIA · PI MOSSE, YAEL P · 2009 to 2023
$5.6M
NCI NIH HHS P01 CA217959NCI NIH HHS R01 CA140198NCI NIH HHS R35 CA220500
6 · The paper itself

Abstract

Neuroblastomas harbor ALK aberrations clinically resistant to crizotinib yet sensitive pre-clinically to the third-generation ALK inhibitor lorlatinib. We conducted a first-in-child study evaluating lorlatinib with and without chemotherapy in children and adults with relapsed or refractory ALK-driven neuroblastoma. The trial is ongoing, and we report here on three cohorts that have met pre-specified primary endpoints: lorlatinib as a single agent in children (12 months to <18 years); lorlatinib as a single agent in adults (≥18 years); and lorlatinib in combination with topotecan/cyclophosphamide in children (<18 years). Primary endpoints were safety, pharmacokinetics and recommended phase 2 dose (RP2D). Secondary endpoints were response rate and

Indexed as

Carcinoma, Non-Small-Cell LungLung NeoplasmsNeuroblastoma3-IodobenzylguanidineAdolescentAdultAminopyridinesAnaplastic Lymphoma KinaseChildChild, PreschoolHumansInfantLactamsLactams, MacrocyclicNeoplasm Recurrence, LocalProtein Kinase Inhibitors3-IodobenzylguanidineAminopyridinesAnaplastic Lymphoma KinaseLactamsLactams, MacrocycliclorlatinibProtein Kinase InhibitorsPyrazoles

Identifiers

PMID37012551
PMCPMC10202811
OpenAlexW4362522571

What OpenQuestion holds

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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.