ArticlePediatric blood & cancer2011
Phase 1 trial and pharmacokinetic study of the farnesyl transferase inhibitor tipifarnib in children and adolescents with refractory leukemias: a report from the Children's Oncology Group.
Article in Pediatric blood & cancer, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 12 papers, 1 of them a synthesis that pooled it.
What it found
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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.
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.
A Phase I Trial and Pharmacokinetic Study of R115777 in Pediatric Patients With Refractory Leukemia
A Phase II Study of Binimetinib in Children and Adults With NF1 Associated Plexiform Neurofibromas (PNOC010)
Who cites it
12 citing papers in PubMed, 1 synthesis or guideline pooled it, 26 citations in OpenAlex.
- The under reporting of recruitment strategies in research with children with life-threatening illnesses: A systematic review.Palliative medicine · 2017Pooled it
- Phase II/III trial of a pre-transplant farnesyl transferase inhibitor in juvenile myelomonocytic leukemia: a report from the Children's Oncology Group.Pediatric blood & cancer · 2015Trial
- Lipids: Driving Forces in the Underlying Biology of Carcinogenesis.ACS pharmacology & translational science · 2025Review
- Design of a Highly Active Peptide Inhibitor of Farnesyltransferase and Its Protective Effect Against Acute Liver Failure.Drug design, development and therapy · 2025Article
- Targeting farnesylation as a novel therapeutic approach in HRAS-mutant rhabdomyosarcoma.Oncogene · 2022Article
- The Use of Inhibitors of Tyrosine Kinase in Paediatric Haemato-Oncology-When and Why?International journal of molecular sciences · 2021Review
- Molecular profiling of childhood cancer: Biomarkers and novel therapies.BBA clinical · 2014Review
- Advances in paediatric cancer treatment.Translational pediatrics · 2014Review
- Pediatric relapsed or refractory leukemia: new pharmacotherapeutic developments and future directions.Drugs · 2013Review
- Gene mutations and molecularly targeted therapies in acute myeloid leukemia.American journal of blood research · 2013Article
- 2-Hydroxyoleate, a nontoxic membrane binding anticancer drug, induces glioma cell differentiation and autophagy.Proceedings of the National Academy of Sciences of the United States of America · 2012Article
- Neurotoxicity of biologically targeted agents in pediatric cancer trials.Pediatric neurology · 2012Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 6 institutions in 1 country.
Funding
Abstract
backgroundThe objectives of this trial were to define the toxicity profile, dose, pharmacokinetics, and pharmacodynamics of the farnesyl transferase (FTase) inhibitor, tipifarnib, in children and adolescents with hematological malignancies. PROCEDURE: Tipifarnib was administered twice daily for 21 days, repeated every 28 days starting at a dose of 300 mg/m(2) /dose. Pharmacokinetic sampling was performed for 36 hr after the first dose and leukemic blasts were collected pre-treatment and at steady state for determination of FTase activity.
resultsOf 29 patients enrolled, 18 were fully evaluable for toxicity, and 23 for response; 26 had pharmacokinetic and pharmacodynamic sampling. The recommended dose is 300 mg/m(2) /dose and toxicities included skin rash, mucositis, nausea, vomiting, and diarrhea. Neurotoxicity, which was dose-limiting in adults at doses exceeding 600 mg/dose, was infrequent and mild. The plasma pharmacokinetics of tipifarnib were highly variable but comparable to adults with acute leukemia and children with solid tumors. The median apparent clearance of tipifarnib was 630 ml/min/m(2) and the median half-life was 4.7 hr. At steady state on 300 mg/m(2) /dose, FTase activity was inhibited by 82% in leukemic blasts. No objective responses were observed.
conclusionsOral tipifarnib is well tolerated in children with leukemia on a twice daily for 2 days schedule at 300 mg/m(2) /dose.
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Registered trials
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.