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Off-label prescribing of targeted anticancer therapy at a large pediatric cancer center.

ABSTRACT: BACKGROUND:Off-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. METHODS:We 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. RESULTS:The 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. CONCLUSIONS:In 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.

SUBMITTER: Lim M

PROVIDER: S-EPMC7520353 | biostudies-literature | 2020 Aug

REPOSITORIES: biostudies-literature

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Off-label prescribing of targeted anticancer therapy at a large pediatric cancer center.

Lim Mir M Shulman David S DS Roberts Holly H Li Anran A Clymer Jessica J Bona Kira K Al-Sayegh Hasan H Ma Clement C DuBois Steven G SG

Cancer medicine 20200804 18

<h4>Background</h4>Off-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.<h4>Methods</h4>We 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 bo ...[more]

PMID: 32750219

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Dataset Information

Off-label prescribing of targeted anticancer therapy at a large pediatric cancer center.

Publications

Off-label prescribing of targeted anticancer therapy at a large pediatric cancer center.

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