ArticleCancer medicine2020
Off-label prescribing of targeted anticancer therapy at a large pediatric cancer center.
Article in Cancer medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers, 1 of them a synthesis that pooled it.
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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.
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Who cites it
17 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Risk and Benefit for Targeted Therapy Agents in Pediatric Phase II Trials in Oncology: A Systematic Review with a Meta-Analysis.Targeted oncology · 2021Pooled it
- Gilteritinib and chemotherapy in children with relapsed/refractory FLT3-ITD AML: results from the phase 1/2 SKIPPER trial.Blood advances · 2026Trial
- The global landscape of pediatric monoclonal antibody (mAb) clinical trials: an analysis of ClinicalTrials.gov.Frontiers in pediatrics · 2026Article
- Ag(e)nostic precision oncology therapy approvals across the years.Trends in cancer · 2025Review
- Hoping Against Hope: Ethical Considerations when Trying Unproven Treatments for Seriously Ill Children.Asian bioethics review · 2025Article
- Integrating MAPK pathway inhibition into standard-of-care therapy for pediatric low-grade glioma.Frontiers in oncology · 2025Review
- Statistical Fragility of Findings From Randomized Phase 3 Trials in Pediatric Oncology.Cancer medicine · 2024Article
- Off-label prescribing of immune checkpoint inhibitor therapy at a single pediatric cancer center.Cancer medicine · 2024Article
- Access to innovative therapies in pediatric oncology: Report of the nationwide experience in Canada.Cancer medicine · 2024Article
- Editorial: Systems and network approaches to precision medicine and healthcare.Frontiers in molecular biosciences · 2024Article
- BRAF/MEK inhibitors use for pediatric gliomas; real world experience from a resource-limited country.Frontiers in oncology · 2024Article
- Article
- Clinical development of new drugs for adults and children with cancer, 2010-2020.Journal of the National Cancer Institute · 2023Article
- Real-world treatment outcomes of medicines used in special situations (off-label and compassionate use) in oncology and hematology: A retrospective study from a comprehensive cancer institution.Cancer medicine · 2023Article
- Measuring Safety and Outcomes for the Use of Compassionate and Off-Label Therapies for Children, Adolescents, and Young Adults With Cancer in the SACHA-France Study.JAMA network open · 2023Article
- Off-Label Medication: From a Simple Concept to Complex Practical Aspects.International journal of environmental research and public health · 2021Review
- Off-label prescribing of targeted anticancer therapy at a large pediatric cancer center.Cancer medicine · 2020Article
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Authors and funding
9 authors.
Funding
Abstract
backgroundOff-label drug prescribing is common in pediatric clinical medicine, though the extent and impact of this practice in pediatric oncology has not yet been characterized.
methodsWe completed a retrospective single-institution cohort study evaluating prevalence, characteristics, and clinical outcomes of off-label prescribing of 108 FDA-approved targeted anticancer drugs in patients < 30 years old treated for cancer from 2007 to 2017. Dosing strategies were adjusted for body size and compared to FDA-approved adult dosing regimen. A composite toxicity endpoint was defined as a patient having unplanned clinic visits, emergency department visits, or unplanned hospital admissions that were at least possibly related to the off-label treatment.
resultsThe overall prevalence of off-label use of targeted therapies was 9.2% (n = 374 patients). The prevalence increased significantly over the study period (P < .0001). Patients treated off-label were more likely to have neuro-oncology diagnoses compared to patients not treated off-label (46% vs 29%; P < .0001). Of the 108 potential agents, 38 (35%) were used by at least one patient. The median starting dose was below the FDA-approved normalized dose for 44.4% of agents. Fifteen percent of patients had a complete response while receiving off-label therapy, 38% experienced toxicity as defined, and 13% discontinued off-label therapy due to toxicity.
conclusionsIn this real-world evaluation of prescribing at a large pediatric cancer center, off-label prescribing of FDA-approved targeted therapies was common, increasing in prevalence, encompassed a broad sample of targeted agents, and was tolerable. Clinicians commonly start dosing below the equivalent FDA-approved dose.
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