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- Pellat, A., et al.
(författare)
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Comprehensive mapping review of real-world evidence publications focusing on targeted therapies in solid tumors : A collaborative work from ESMO real-world data and Digital Health Working Group
- 2023
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Ingår i: Annals of Oncology. - : Elsevier. - 0923-7534 .- 1569-8041. ; 34:Suppl. 2, s. S925-S925
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Tidskriftsartikel (övrigt vetenskapligt/konstnärligt)abstract
- Background: A growing body of real-world evidence (RWE) aims to better reflect outcomes of cancer patients treated in real-world settings. We aimed to conduct a first comprehensive mapping review of the RWE produced over the past 3 years in terms of tumor type, treatment strategies, setting, and data sources, focusing on targeted therapies (TT) in solid tumors.Methods: We conducted a systematic review in PubMed of RWE studies published between 01/2020 and 12/2022. We identified non-interventional studies using observational data, focusing on solid tumors exposure to targeted therapies, excluding immunotherapies. Abstract and full-text screening were performed by 11 independent reviewers.Results: A total of 7,774 publications were retrieved with 1,251 considered eligible and extracted. The number of publications per year progressively increased during this period (328 in 2020; 421 in 2021; 502 in 2022). Most studies (50%) were performed in Asia, followed by Europe (25%) and North America (17%). Only 8% of studies had patients treated in more than one country. Treatment effectiveness and safety were assessed in 71% and 42% of studies respectively. Main data sources were medical records.Conclusions: RWE publications on TT for solid tumors are heterogeneous and mostly rely on retrospective data such as medical records. Population-based and international studies are rare. Collaborative efforts towards international representativeness and the use of routinely collected and/or standardized data sources must be encouraged to increase the relevance and future quality of publications and their potential impact on oncology practice.
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- Derksen, J. W. G., et al.
(författare)
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Real-world evidence contributions to European medicines agency's safety and efficacy evaluations of oncology targeted therapies between 2018-2022
- 2023
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Ingår i: Annals of Oncology. - : Elsevier. - 0923-7534 .- 1569-8041. ; 34:Suppl. 2, s. S930-S930
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Tidskriftsartikel (övrigt vetenskapligt/konstnärligt)abstract
- Background: While Real-world Evidence (RWE) has documented value for safety monitoring and disease epidemiology, its objective contribution to safety and efficacy evaluations for regulatory purposes is still unclear. Here, we aim to describe the prevalence and type of RWE considered by European Medicines Agency (EMA) as contribution to efficacy and safety-related evidence generation among approved oncology targeted therapies...Methods: On March 10, 2023, we screened the medicines listing of EMA to identify all anti-cancer targeted therapies for solid malignancies with a decision date (initial marketing authorizations and extension of indications) between 2018-2022. We screened the European public assessment reports (EPARs) using a standardized approach to collect data on RWE. When generated pre-authorization, the RWE contribution to the final regulatory decision was classified as definitive, supportive, or non-supportive. For...Results: Out of a total of 1976 medicines, we identified 55 oncology targeted therapies, corresponding to 75 EPARs (indications), which are described in the table. The use of RWE in regulatory deliberations occurred in 24/75 (32%) EPARs, increasing from 30% in 2018-2020, to 34% in 2021-2022. Pre-authorization RWE was described in 20/24 (83%) EPARs, among which none were definitive, 8 RWE studies (in 7 EPARs) non-supportive, and 20 RWE studies (in 15 EPARs) were supportive of the decision. Published RWE...Conclusions: Over the past 5 years, RWE involvement in the approval of oncology targeted therapies in Europe tends to increase, with the majority being supportive for EMA regulatory decision making complementary to traditional clinical trials...
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- Karihtala, P., et al.
(författare)
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Current treatment landscape of HR+/HER2-advanced breast cancer in the Nordics : A modified Delphi study
- 2022
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Ingår i: Annals of Oncology. - : Elsevier. - 0923-7534 .- 1569-8041. ; 33:Suppl. 3, s. S218-S218
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Tidskriftsartikel (övrigt vetenskapligt/konstnärligt)abstract
- Background: The aim of this Delphi study was to assess current perspectives on HR+/HER2- advanced breast cancer (aBC) treatment strategies among Nordic BC oncologists, and to gain broader understanding of the uptake and implementation of novel treatments.Methods: A modified, three round Delphi method was followed. A steering committee was appointed for study coordination, panellist selection and questionnaires development. The questionnaires covered clinically relevant topics related to HR+/HER2- aBC treatment: treatment patterns in different lines of therapy (first- (1L), second- (2L) and third-line (3L)), oligometastatic disease, de novo aBC, brain metastases, age as influential factor, visceral crisis, radiotherapy, diagnostics and clinical guidelines. Both open and closed-ended questions were included. Consensus was defined as at least 70% agreement.Results: In total 28 panelists participated in the study. In rounds one and two, 14 and 21 questions reached consensuses, respectively. Thirteen non-consensus reaching questions were reposted in round three, where 10 reached consensus. Overall, topics that reached a high consensus level included: treatment approaches in 1L and 2L treatment setting for HR+/HER2- aBC, treatment of oligometastatic disease, visceral crisis and brain metastases, and age-related treatment considerations. No consensus was reached for aspects regarding 3L therapy and de novo aBC. Endocrine therapy (ET) combined with CDK4/6i was the treatment of choice for both 1L and 2L therapy. Regarding implementation of clinical guidelines, a discrepancy was observed between treatments recommended in guidelines and those used in clinical practice, especially in cases where novel treatments were proposed.Conclusions: Endocrine therapy combined with a CDK4/6i is the frontline treatment choice for HR+/HER2- aBC in the Nordics. Observed discrepancies between international clinical guidelines and practice are partly due to difference in the availability of novel treatment strategies that might lead to differences in clinical experience in the Nordics. The lack of consensus might reflect limited evidence on these topics and the need for collaborative research efforts. Written on behalf of Nordic Delphi Panellist group.
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- Nicolaescu, T-M, et al.
(författare)
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Prognostic relevance of pre-treatment c-reactive protein to albumin ratio in patients with diffuse large b cell lymphoma
- 2022
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Ingår i: Annals of Oncology. - : Elsevier. - 0923-7534 .- 1569-8041. ; 33:7, s. S832-S832
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Tidskriftsartikel (övrigt vetenskapligt/konstnärligt)abstract
- Background: Previous studies have shown that a high level of pre-treatment C-reactive protein to albumin ratio (CAR) is associated with poor outcomes in patients with diffuse large B cell lymphoma (DLBCL). However, these were single-centre studies with a relatively small number of patients. The aim of our study was to further investigate the prognostic value of CAR in a larger cohort and whether the addition of CAR to the International Prognostic Index (IPI) would result in a better discriminatory ability.Methods: All adult patients treated 2000–2013 with R-CHOP/CHOP-like treatment for DLBCL in four counties of Sweden were included (n=414). The study population was divided into high respectively low CAR group using the Budczies et al.’s cut-off finder. The groups were compared in terms of differences in clinical characteristics, response to treatment and survival. The prognostic ability of IPI vs IPI plus CAR was compared by receiver-operating-characteristic curve (ROC), net reclassification improvement (NRI) and the integrated discrimination improvement index (IDI).Results: The high CAR group was associated with higher IPI score, lower performance status, high LDH, bulky disease and more advanced Ann Arbour stage. The high CAR group had a higher proportion of patients with progressive disease (24.2% vs 6.4%, p<0.001) and a lower proportion of patients with complete remission (61.5% vs 85.7%, p<0.01). The high CAR group had poorer 5-year OS (49% vs 70%; p<0.001) and EFS (45% vs 68%; p<0.001). After adjustment for BMI, bulky disease and IPI, high CAR values independently predicted poor OS (HR: 1.58, 95% CI 1.18–2.11; p=0.002) and EFS (HR: 1.57, 95% CI 1.18–2.10; p=0.002). When assessed by NRI, the addition of CAR to IPI seems to better identify patients with better prognosis compared with IPI alone. However, the area under the ROC curve and IDI did not show any significant improvement in model performance.Conclusions: CAR seems to be a useful prognostic biomarker in patients with DLBCL. Although the addition of CAR to IPI could identify some additional patients with better prognosis, the discriminatory ability of IPI was not improved. IPI remains the standard model for risk stratification in patients with DLBCL.
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