Safety of Anticancer Agents Used in Children: A Focus on Their Off-Label Use Through Data From the Spontaneous Reporting System

Mascolo, Annamaria; Scavone, Cristina; Bertini, Michele; Brusco, Simona; Punzo, Francesca; Pota, Elvira; Martino, Martina Di; Pinto, Daniela Di

doi:10.3389/fphar.2020.00621

Cited by 14 publications

(10 citation statements)

References 59 publications

(58 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“…For both reasons, the comparison of the incidence of ADR between the use of off-label and on-label drugs was considered inappropriate. A similar trend was documented in the study of Mascolo et al, which aimed to assess off-label use of Individual Case Safety Reports concerning antineoplastic drugs in children and found that only 18 (7.6%) out of 236 were classified as off-label cases ( Mascolo et al, 2020 ). These authors considered the low number of cases were due to underreporting.…”

Section: Discussionsupporting

confidence: 73%

Adverse Reactions to Drugs of Special Interest in a Pediatric Oncohematology Service

et al. 2021

View full text Add to dashboard Cite

Introduction: Drugs used in oncological diseases are frequently related to adverse drug reactions (ADR). Few studies have analyzed the toxicity of cancer treatments in children in real practice.Methods: An observational, longitudinal and prospective study has been carried out in an Oncohematology Service of a tertiary hospital. During 2017, patients exposed to one or more drugs of a previously agreed list were identified and followed-up for at least 6 months each. Characteristics of ADR, incidence, causality and possible preventability, have been evaluated.Results: 72 patients have been treated with at least one study drug, and 159 ADR episodes involving at least one of these drugs have been identified, with a total of 293 ADR. Most episodes required hospital admission (35.2%) or happened during the hospital stay (33%), and 91.2% were severe. Blood disorders were the most frequent ADR (96; 32.8%), related to thioguanine (42) and pegaspargase (39) mainly, followed by infections (86; 29.4%) related to thioguanine (32), pegaspargase (27), Erwinia asparaginase (14) and rituximab (13). Two ADR were unknown. Most ADR were dose-dependent or expectable (>90%). The global incidence of ADR was 3.1/100 days at risk (95% CI 2.7–3.5), with 3.5 ADR/100 days at risk with pegaspargase (95% CI 2.9–4.2), 1.2/100 days at risk with rituximab (95% CI 0.8–1.8) and 11.6/100 days at risk with thioguanine (95% CI 9.4–14.2). Controversial additional measures of prevention, other than those already used, were identified.Conclusion: ADR are frequent in pediatric oncohematological patients, mainly blood disorders and infectious diseases. Findings regarding incidence and preventability may be useful to compare data between different centers and to evaluate new possibilities for action or prevention.

show abstract

Section: Discussionsupporting

confidence: 73%

Adverse Reactions to Drugs of Special Interest in a Pediatric Oncohematology Service

et al. 2021

View full text Add to dashboard Cite

show abstract

“…To the best of the author’s knowledge, this study addressed the adverse events of off-label use for the first time through AE reporting system databases in a comprehensive manner. Relatively few studies have recently described AEs associated with off-label uses in limited populations 60 or specific cases. 61 The results here can be useful when making predictions of AEs associated with off-label use.…”

Section: Discussionmentioning

confidence: 99%

Adverse Events Related to Off-Label Drugs Using Spontaneous Adverse Event Reporting Systems

Han¹,

Oh²,

Kim³

2021

TCRM

View full text Add to dashboard Cite

“…However, there are concerns that there is an underreporting phenomenon occurring through national spontaneous reporting systems. [ 94 ]…”

Section: Safety Initiativesmentioning

confidence: 99%

Off-Label Medication: From a Simple Concept to Complex Practical Aspects

Rusz

Ősz

Jîtcă

et al. 2021

IJERPH

View full text Add to dashboard Cite

Off-label use of drugs is widely known as unapproved use of approved drugs, and it can be perceived as a relatively simple concept. Even though it has been in existence for many years, prescribing and dispensing of drugs in an off-label regimen is still a current issue, triggered especially by unmet clinical needs. Several therapeutic areas require off-label approaches; therefore, this practice is challenging for prescribing physicians. Meanwhile, the regulatory agencies are making efforts in order to ensure a safe practice. The present paper defines the off-label concept, and it describes its regulation, together with several complex aspects associated with clinical practices regarding rare diseases, oncology, pediatrics, psychiatry therapeutic areas, and the safety issues that arise. A systematic research of the literature was performed, using terms, such as “off-label”, ”prevalence”, ”rare diseases”, ”oncology”, ”psychiatry”, ”pediatrics”, and ”drug repurposing”. There are several reasons for which off-label practice remains indispensable in the present; therefore, efforts are made worldwide, by the regulatory agencies and governmental bodies, to raise awareness and to ensure safe practice, while also encouraging further research.

show abstract

Safety of Anticancer Agents Used in Children: A Focus on Their Off-Label Use Through Data From the Spontaneous Reporting System

Cited by 14 publications

References 59 publications

Adverse Reactions to Drugs of Special Interest in a Pediatric Oncohematology Service

Adverse Reactions to Drugs of Special Interest in a Pediatric Oncohematology Service

Adverse Events Related to Off-Label Drugs Using Spontaneous Adverse Event Reporting Systems

Off-Label Medication: From a Simple Concept to Complex Practical Aspects

Contact Info

Product

Resources

About