Efficacy of Immune Checkpoint Inhibitors in Rare Tumours: A Systematic Review

Petrelli, Fausto; Consoli, Francesca; Ghidini, Antonio; Perego, Gianluca; Luciani, Andrea; Mercurio, Paola; Berruti, Alfredo; Grisanti, Salvatore

doi:10.3389/fimmu.2021.720748

Cited by 5 publications

(4 citation statements)

References 86 publications

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“…If for many rare cancer types a genomic understanding remains incomplete or absent, a number of them may benefit from molecular target therapy [ 21 ]. A very recent systematic review has also evidenced a clinical benefit from treatment with immune checkpoint inhibitors for some conditions [ 22 ].…”

Section: Discussionmentioning

confidence: 99%

Descriptive Epidemiology of Hospitalization of Patients with a Rare Tumor in an Italian Region

et al. 2022

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Objectives: Rare tumors (RT) collectively account for one quarter of all malignancies in Italy. The low frequency and the large heterogeneity in natural history and outcome of individual diseases, together with a scarcity of epidemiological information make them a challenge for clinical practice, as well as for public healthcare organizations. We conducted a retrospective study to quantify the burden of hospitalization in a real-word setting in patients diagnosed with these diseases in an Italian region. Methods: RT patients were tracked along all hospital stays from 2000 to 2019 using hospital discharge records. Frequency of hospitalizations, average time spent in hospital and median timespan between consecutive admissions were considered. Re-hospitalization rates were analyzed through a multivariable negative binomial regression analysis to adjust for confounding and allowing for over-dispersion in count data. Results: As a whole, 57,329 patients were identified at first stay for all studied tumors. A total of 183,959 admissions were retrieved, along a median of 3 hospitalizations per patient. Median timespan between hospitalizations shortened in the course of the study years (12.5 months in 2000–2004 to 5.4 months in 2015–2019). The overall re-hospitalization rate increased from 0.92 per patient/year (95% CI = 0.81–1.04) in 2000–2004 to 2.17 (95% CI = 1.90–2.47) in 2015–2019. Conclusions: Overall, the hospitalization rate of patients with a RT increased in the twenty years since the 2000 and particularly doubled starting from 2015. A higher burden of hospitalizations was found for tumors of the central nervous system, thoracic cavity, digestive tract and sarcomas. To the best of our knowledge this is the first paper related to access to Italian healthcare facilities of patients with these tumors.

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Section: Discussionmentioning

confidence: 99%

Descriptive Epidemiology of Hospitalization of Patients with a Rare Tumor in an Italian Region

et al. 2022

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“…These inhibitors induce sustained antitumour immune responses by blocking the inhibitory effects of tumour cells on immune cells. The immune checkpoint inhibitors approved by the FDA include ipilimumab [cytotoxic T-lymphocyte-associated protein 4 (CTLA-4) inhibitor], nivolumab (PD-1 inhibitor) and avelumab (PD-L1 inhibitor) ( 87 ). The successful clinical application of these agents has brought new life to the immunotherapeutic treatment of malignant gliomas.…”

Section: Mirnas and Glioma Drug Resistancementioning

confidence: 99%

NcRNAs: Multi‑angle participation in the regulation of glioma chemotherapy resistance (Review)

et al. 2022

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As the most common primary tumour of the central nervous system, gliomas have a high recurrence rate after surgical resection and are resistant to chemotherapy, particularly high-grade gliomas dominated by glioblastoma multiforme (GBM). The prognosis of GBM remains poor despite improvements in treatment modalities, posing a serious threat to human health. At present, although drugs such as temozolomide, cisplatin and bevacizumab, are effective in improving the overall survival of patients with GBM, most patients eventually develop drug resistance, leading to poor clinical prognosis. The development of multidrug resistance has therefore become a major obstacle to improving the effectiveness of chemotherapy for GBM. The ability to fully understand the underlying mechanisms of chemotherapy resistance and to develop novel therapeutic targets to overcome resistance is critical to improving the prognosis of patients with GBM. Of note, growing evidence indicates that a large number of abnormally expressed noncoding RNAs (ncRNAs) have a central role in glioma chemoresistance and may target various mechanisms to modulate chemosensitivity. In the present review, the roles and molecular mechanisms of ncRNAs in glioma drug resistance were systematically summarized, the potential of ncRNAs as drug resistance markers and novel therapeutic targets of glioma were discussed and prospects for glioma treatment were outlined. ncRNAs are a research direction for tumor drug resistance mechanisms and targeted therapies, which not only provides novel perspectives for reversing glioma drug resistance but may also promote the development of precision medicine for clinical diagnosis and treatment.

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“…Despite the efficacy of ICI and ICI-based combination therapies, their role in rare tumors like ACC is heterogeneous and less well established [ 14 ]. For example, ICIs have been effective in Merkel cell carcinoma and cutaneous squamous cell carcinoma, while they have not been as effective in low-grade neuroendocrine tumors [ 14 , 15 ]. In ACC, Raj.…”

Section: Introductionmentioning

confidence: 99%

The Efficacy and Safety of Immune Checkpoint Inhibitors in Adrenocortical Carcinoma: A Systematic Review and Meta-Analysis

Ababneh,

Ghazou,

Alawajneh

et al. 2024

Cancers

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Immune checkpoint inhibitors (ICIs) have revolutionized the treatment of different malignancies. However, their efficacy in advanced adrenocortical carcinoma (ACC) remains uncertain. Thus, we conducted a systematic review and meta-analysis to summarize the efficacy and tolerability of ICIs in patients with advanced ACC. We searched PubMed, Scopus, and CENTRAL for studies that used ICIs in ACC. Studies with more than five patients were included in the meta-analysis of the objective response rate (ORR), disease control rate (DCR), overall survival (OS), progression-free survival (PFS), and grade 3/4 adverse events. Twenty studies with 23 treatment arms and 250 patients were included. Single-agent anti-PD1 or anti-PD-L1 treatment was utilized in 13 treatment arms, whereas an anti-PD1 or anti-PD-L1 and anti-CTLA4 combination was used in 4 treatment arms. Other anti-PD1- or anti-PD-L1-based combinations were used in five treatment arms. The ORR was 14% (95% CI = 10–19%, I2 = 0%), and the DCR was 43% (95% CI = 37–50%, I2 = 13%). The combination anti-PD1- or anti-PD-L1-based treatment strategies did not correlate with higher responses compared with monotherapy. The median OS was 13.9 months (95% CI = 7.85–23.05), and the median PFS was 2.8 months (95% CI = 1.8–5.4). ICIs have a modest efficacy in advanced ACC but a good OS. Further studies are needed to investigate predictive biomarkers for ICI response and to compare ICI-based strategies with the current standard of care.

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Efficacy of Immune Checkpoint Inhibitors in Rare Tumours: A Systematic Review

Cited by 5 publications

References 86 publications

Descriptive Epidemiology of Hospitalization of Patients with a Rare Tumor in an Italian Region

Descriptive Epidemiology of Hospitalization of Patients with a Rare Tumor in an Italian Region

NcRNAs: Multi‑angle participation in the regulation of glioma chemotherapy resistance (Review)

The Efficacy and Safety of Immune Checkpoint Inhibitors in Adrenocortical Carcinoma: A Systematic Review and Meta-Analysis

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