Abstract:Introduction:In Europe, despite recent advances in clinical development, most of the drugs currently used to treat childhood cancers are adult medicines, prescribed outside of the authorized indication. In this context, a monocentric retrospective cohort analysis was conducted, evaluating pediatric, adolescent, and young adult patients affected by onco-hematologic disease, treated with targeted therapies used off-label or as compassionate use.
Methods:The analysis was conducted on 45 patients aged less than or… Show more
“…Креативная хирургия и онкология / Creative Surgery and Oncology 2024;14(1):69-77 нения «вне инструкции» у пациентов со злокачественными новообразованиями (ЗНО) центральной нервной системы (ЦНС) [4]. R. Cenna и соавторы опубликовали результаты моноцентрового ретроспективного когортного анализа, в котором проводилось изучение клинических ситуаций c использованием терапии таргетными препаратами «вне инструкции» и по программам расширенного доступа у 45 пациентов со злокачественными новообразованиями в возрасте до 30 лет в крупном педиатрическом центре Италии [12]. Двадцать пять пациентов из 45 имели онкогематологические заболевания.…”
Section: обзорные статьи / Reviewsunclassified
“…Как минимум одна побочная реакция отмечена при использовании 76 % препаратов (n = 22) у пациентов с онкогематологическими заболеваниями, и 43 % препаратов (n = 10), применявшихся у больных с солидными опухолями. Авторы исследования подтвердили частое использование таргетной терапии «вне инструкции» детям с онкологическими заболеваниями, особенно при рецидивах, отмечая при этом то, что данное лечение в большинстве случаев эффективно и переносимо [12]. A. Spini и соавторы, опубликовав результаты систематического обзора и метаанализа ряда клинических испытаний и обсервационных исследований, сообщили о том, что у каждого второго педиатрического пациента (≤18 лет) с солидной опухолью, для лечения которых применялись антиангиогенные препараты в качестве монотерапии, наблюдались серьезные нежелательные явления (НЯ) [7].…”
Safety is recognized as a crucial issue of off-label use of anticancer drugs. The potential benefits of such prescriptions in oncology are associated with prevailing the expected clinical benefits over the risks of complications. However, in certain clinical situations with uncertain benefit/risk ratio, an off-label use of drugs may threaten the life and health of the patient. The present paper explores the safety of off-label anticancer drug therapy in real clinical practice. Health care and routine clinical practice are given particular emphasis on systematic recording and careful monitoring of adverse events associated with the off-label use of medicinal products. The creation of a unified registration system for off-label use of drug therapy in oncology along with the creation of large databases (on the sites of institutions with an option to combine the data obtained at the level of districts, regions and the country) enables a significant amount of information on the safety and effectiveness of this approach to be gathered. As a result, a predictable nature of treatment and manageable toxic effects are potentially provided. The study into reasons behind off-label use of drugs in oncology, as well as the study into spectrum and severity of adverse events resulting from the implementation of these prescriptions, will provide detailed information on the safety of off-label use of anticancer agents in patients with malignant neoplasms at different stages of oncological treatment.
“…Креативная хирургия и онкология / Creative Surgery and Oncology 2024;14(1):69-77 нения «вне инструкции» у пациентов со злокачественными новообразованиями (ЗНО) центральной нервной системы (ЦНС) [4]. R. Cenna и соавторы опубликовали результаты моноцентрового ретроспективного когортного анализа, в котором проводилось изучение клинических ситуаций c использованием терапии таргетными препаратами «вне инструкции» и по программам расширенного доступа у 45 пациентов со злокачественными новообразованиями в возрасте до 30 лет в крупном педиатрическом центре Италии [12]. Двадцать пять пациентов из 45 имели онкогематологические заболевания.…”
Section: обзорные статьи / Reviewsunclassified
“…Как минимум одна побочная реакция отмечена при использовании 76 % препаратов (n = 22) у пациентов с онкогематологическими заболеваниями, и 43 % препаратов (n = 10), применявшихся у больных с солидными опухолями. Авторы исследования подтвердили частое использование таргетной терапии «вне инструкции» детям с онкологическими заболеваниями, особенно при рецидивах, отмечая при этом то, что данное лечение в большинстве случаев эффективно и переносимо [12]. A. Spini и соавторы, опубликовав результаты систематического обзора и метаанализа ряда клинических испытаний и обсервационных исследований, сообщили о том, что у каждого второго педиатрического пациента (≤18 лет) с солидной опухолью, для лечения которых применялись антиангиогенные препараты в качестве монотерапии, наблюдались серьезные нежелательные явления (НЯ) [7].…”
Safety is recognized as a crucial issue of off-label use of anticancer drugs. The potential benefits of such prescriptions in oncology are associated with prevailing the expected clinical benefits over the risks of complications. However, in certain clinical situations with uncertain benefit/risk ratio, an off-label use of drugs may threaten the life and health of the patient. The present paper explores the safety of off-label anticancer drug therapy in real clinical practice. Health care and routine clinical practice are given particular emphasis on systematic recording and careful monitoring of adverse events associated with the off-label use of medicinal products. The creation of a unified registration system for off-label use of drug therapy in oncology along with the creation of large databases (on the sites of institutions with an option to combine the data obtained at the level of districts, regions and the country) enables a significant amount of information on the safety and effectiveness of this approach to be gathered. As a result, a predictable nature of treatment and manageable toxic effects are potentially provided. The study into reasons behind off-label use of drugs in oncology, as well as the study into spectrum and severity of adverse events resulting from the implementation of these prescriptions, will provide detailed information on the safety of off-label use of anticancer agents in patients with malignant neoplasms at different stages of oncological treatment.
“…8 In an Italian single-center study describing 45 patients treated with off-label or compassionate use of targeted anticancer therapies, the majority of use was in relapsed or refractory disease, 71% experienced at least one adverse drug reaction, and 52% achieved a complete response. 9 Over the last decade, immune checkpoint inhibition (ICI) has revolutionized the field of medical oncology, and the number of patients with adult malignancies treated with ICI has dramatically increased. 10,11 Retrospective studies and early phase clinical trials have demonstrated ICI to be safe and tolerable in pediatric patients with a variety of malignancies, [12][13][14][15][16][17][18][19][20][21][22][23] including central nervous system tumors.…”
Section: Introductionmentioning
confidence: 99%
“…One study evaluating the off‐label use of targeted therapy in pediatric cancers from 2007 to 2017 observed an overall prevalence of 9.2% 8 . In an Italian single‐center study describing 45 patients treated with off‐label or compassionate use of targeted anticancer therapies, the majority of use was in relapsed or refractory disease, 71% experienced at least one adverse drug reaction, and 52% achieved a complete response 9 …”
Section: Introductionmentioning
confidence: 99%
“… 8 In an Italian single‐center study describing 45 patients treated with off‐label or compassionate use of targeted anticancer therapies, the majority of use was in relapsed or refractory disease, 71% experienced at least one adverse drug reaction, and 52% achieved a complete response. 9 …”
BackgroundImmune checkpoint inhibitors (ICI) have improved outcomes in a variety of adult cancers and are prescribed with increasing frequency across oncology. However, patterns of off‐label use of ICI in pediatrics remain unclear.MethodsThis is a single‐institution, retrospective cohort study evaluating off‐label ICI use in pediatric and young adult patients with cancer treated at our institution from 2014 to 2022. Response was based on clinician assessment derived from clinical records. Immune‐related adverse events (iRAEs) were classified according to CTCAE v5.0.ResultsWe identified 50 unique patients treated with off‐label ICI (28 with solid tumors, 20 with central nervous system (CNS) tumors, 2 with hematologic malignancies). At time of ICI initiation, only five patients (10%) had localized disease, and all but one patient was treated in the relapsed/refractory setting. All patients were treated with the FDA‐approved weight‐based dosing recommendations. Overall, there was disease control in 21 patients (42%), with best response including one complete response (melanoma), two partial responses (high‐grade glioma, CNS nongerminomatous germ cell tumor), and 18 patients with stable disease. Forty‐four patients (88%) eventually experienced disease progression. Among 22 patients (44%) experiencing iRAEs, 10 (20%) had a grade ≥3 irAE, 12 (24%) required corticosteroids, and 14 (28%) required ICI discontinuation. irAE occurrence was associated with significantly improved progression‐free survival (HR 0.35; 95% CI: 0.18 to 0.68; p = 0.002) and overall survival (HR 0.33; 95% CI: 0.17 to 0.66; p = 0.002).ConclusionsAt our institution, ICI was most commonly prescribed in the relapsed/refractory setting to patients with metastatic disease. The treatment was generally well‐tolerated in the pediatric population. The overall response rate was low, and the majority of patients eventually experienced disease progression. A few patients, however, had durable treatment responses. Further studies are needed to identify which pediatric patients are most likely to benefit from ICI.
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