Therapeutic Advances in Medical Oncology最新文献

Background: The prognosis for patients with ultra high-risk gestational trophoblastic neoplasia (GTN) is significantly worse, and there is currently no consensus regarding the optimal treatment strategies for this specific patient population.

Objectives: This study aims to investigate the clinical characteristics, treatment responses, outcomes, and prognostic risk factors in ultra high-risk GTN patients with an International Federation of Gynecology and Obstetrics (FIGO) score of 13 or higher.

Design: Retrospective study.

Methods: Medical records of 36 GTN patients with FIGO score ⩾ 13, treated at the first affiliated hospital of Zhengzhou University, China, from January 2015 to June 2024, were retrospectively reviewed. Chi-square tests, univariate analysis, and Kaplan-Meier survival analysis were employed for data analysis.

Results: Etoposide, methotrexate, actinomycin D, cyclophosphamide, and vincristine (EMA-CO) was the most commonly used chemotherapy regimen (17/36, 47.2%). Twenty-one patients responded well to the initial chemotherapy regimen and achieved complete remission (CR). One patient was switched to salvage chemotherapy due to resistance to the initial regimen and subsequently achieved CR. Four patients experienced relapse; of these, three attained CR. The median follow-up time was 46 months (range 12-85 months). The CR rate was 69.4% (25/36). A total of 20 patients (55.6%) received adjuvant treatments, including surgery and radiotherapy. Stage IV disease, liver metastases, uncommon distant metastatic sites, and ⩾3 metastatic sites were significant predictors of mortality.

Conclusion: In our study, the overall CR rate for ultra high-risk GTN patients with FIGO score ⩾ 13 was 69.4%. Five patients (45.5%) experienced early mortality. All patients with brain metastases who received chemotherapy in conjunction with whole-brain or stereotactic radiotherapy achieved CR. Immune checkpoint inhibitors demonstrated potential efficacy in treating chemotherapy-resistant GTN.

What is this summary about? This summary describes the results of a clinical study that compared two different doses of a treatment, called inotuzumab ozogamicin (inotuzumab for short), for acute lymphoblastic leukemia (ALL for short). This summary describes the results for people aged 18 years and older who took part in the study. Why was this study done? People who took part in the study had a higher risk of developing a side effect called sinusoidal obstruction syndrome (SOS for short), also known as veno-occlusive disease (VOD for short), which is a rare condition where some of the small blood vessels in the liver become blocked. Researchers wanted to find out if receiving a lower dose than the recommended dose of inotuzumab reduced the likelihood of people developing SOS after a stem cell transplant. Researchers wanted to find out if a lower dose of inotuzumab would also impact the efficacy (how well inotuzumab works to treat ALL) in people with ALL and what other side effects occurred.

What is the purpose of this PLS-P? The purpose of this plain language summary of publication is to help you understand recent findings from Part 1 of the RUBY study (full name ENGOT-EN6/GOG3031/RUBY trial). • Researchers sometimes study new combinations of already approved individual treatments to see if the combinations work well together and if they are safe to prescribe to patients. • In Part 1 of the RUBY study, the combination of dostarlimab with carboplatin-paclitaxel was investigated to see how well it worked for patients with primary advanced or recurrent endometrial cancer when compared with patients who were given placebo plus carboplatin-paclitaxel. • Patients in the RUBY study were divided into subgroups according to their biomarker status. This allowed researchers to see if biomarker status could help determine how well patients with specific biomarkers would respond to the combination of dostarlimab with carboplatin-paclitaxel. • This summary reports the results of the second data cut (the second time investigators looked at all of the data since the start of the study) of the RUBY study. At this time, researchers were looking specifically at how long patients lived and how safe the treatment was in patients who received dostarlimab with carboplatin-paclitaxel compared with those patients who received placebo plus carboplatin-paclitaxel.

Background: Adapted physical activity (APA) is key supportive care in cancer patients. Very few studies have evaluated APA in digestive cancers, and the eligibility and feasibility of an APA program in this population have not been clearly defined.

Objectives: Primary objective: to analyze the reasons for failure of an APA program in digestive cancer patients. Secondary objectives: to assess patient eligibility, feasibility of an APA program, and the potential changes in patient quality of life, physical performance, and nutritional status.

Design: APACADIG is a pilot, single-center, prospective non-randomized study.

Methods: All consecutive patients with digestive cancer seen from January 18, 2021 to February 15, 2021, were proposed a supervised 2-month APA program.

Results: Sixty-five consecutive patients (men 69%, median age 69.8 years old, PS 0/1: 78.5%) with mainly colorectal (38%) and pancreaticobiliary (35%) cancers were included. Forty-seven of these patients (72%) were eligible, and 22 (46.8%) accepted the APA program. The latter were more often active women, living in urban areas (59.1%) with higher socioeconomic status (40.9%). The barriers to APA were clinical, the cost of transport, lack of awareness of the benefits of APA, and a sedentary lifestyle before the cancer diagnosis. Withdrawal from APA was mainly due to changes in physical status. APA improved patients' physical performance (6-min walk test) (distance 516.1 m ± 102.7 m vs 414.5 m ± 111.9 m; p = 0.0430).

Conclusion: Only one-sixth of the population completed the program, and we identified several barriers to APA.

Background: Biliary tract cancers (BTC) are often diagnosed after the age of 70, when comorbidities and compromised performance status (PS) are more prevalent.

Objectives: This study compared clinical and disease characteristics and outcomes in BTC patients aged ⩽70 and >70 years.

Design and methods: PRONOBIL-ACABI is a cohort study including 1256 BTC patients treated across 16 French centers from January 2003 to June 2021. We analyzed demographics, clinical characteristics, treatment modalities, molecular profiles, overall survival (OS) as the primary endpoint, and progression-free survival (PFS).

Results: Among the 1256 BTC patients (53% male; median age: 64.5), 31% were aged >70. Patients >70 exhibited poorer PS (PS ⩾2, 17% vs 8%; p < 0.0001), a higher rate of comorbidities (⩾1, 89% vs 78%; p < 0.0001), and were less often proposed a molecular profile (43% vs 65%; p < 0.0001) than those ⩽70. Patients with unresectable BTC aged >70 had significantly shorter OS compared to younger patients (median OS: 14.6 vs 17.4 months, p < 0.0001), despite similar PFS (median PFS: 6.6 vs 5.8 months, p = 0.61). They were also less likely to receive first-line chemotherapy (87% vs 97%, p < 0.0001). In resected BTC, survival outcomes were comparable across age groups, with a median OS of 47.0 months in patients >70 vs 48.8 months in those ⩽70.

Conclusion: Patients aged >70 years with unresectable BTC had a significantly shorter OS compared to those aged ⩽70, despite similar first-line PFS. In resected BTC, elderly patients achieved OS and PFS outcomes comparable to those aged ⩽70.

The treatment of metastatic castration-resistant prostate cancer (mCRPC) remains a huge challenge. Newer radionuclides, such as ²²³Ra, ¹⁷⁷Lu, ²²⁵Ac, etc., have shown efficacy in alleviating cancer-related pain and also in enhancing the prognosis of mCRPC patients. These novel radionuclides have emerged as important weapons in the treatment of mCRPC. This article reviews recent advances and key clinical trials in various radionuclides and their combinations in the application for treating mCRPC. Further directions of this new treatment strategy are also discussed in this review.

Background: The LITESPARK-005 trial demonstrated the efficacy and safety of belzutifan in patients with previously treated clear cell renal cell carcinoma (ccRCC). This study aims to evaluate the cost-effectiveness of belzutifan compared to everolimus in treating patients with advanced ccRCC who have received at least one systemic therapy from the perspective of the Chinese healthcare system and the US payers.

Objectives: To provide previously treated ccRCC patients with the option of belzutifan and to offer recommendations regarding in China.

Design: The cost-effectiveness analysis.

Methods: A partitioned survival model was constructed based on data from the LITESPARK-005 trial. Patients transitioned through three mutually exclusive health states: progression-free survival (PFS), progressive disease, and death. The model cycle length was set at 28 days, with a lifetime horizon. Direct medical costs and utility values were obtained from published literature and real-world healthcare data. The model estimated total costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs). Price simulations, sensitivity analyses, and scenario analyses were conducted to assess model robustness.

Results: The base-case analysis showed that belzutifan in China generated 2.038 QALYs at a total cost of $102,561.26 and an ICER of $54,430.16/QALY, which exceeded the willingness to pay (WTP) threshold ($39,076.44/QALY). Belzutifan in the United States generated 2.280 QALYs at a total cost of $796,227.28 with an ICER of $270,864.46/QALY, which significantly exceeded the WTP threshold ($150,000/QALY). The PFS utility value and the drug cost of belzutifan were the main factors affecting the change in ICER, whether in China or the United States. At current pricing, belzutifan was unlikely to be cost-effective. Price simulations indicated the belzutifan would be cost-effective when the price of belzutifan remained below $5.524/mg in the United States and $0.779/mg in China.

Conclusion: Compared to everolimus, belzutifan is not cost-effective at its current price for treating previously treated RCC in the United States. In China, the belzutifan group was cost-effective when the price of belzutifan was less than $0.779/mg. This study suggests that reducing the price could substantially improve the economic viability of belzutifan.

Background: Fertility loss due to chemotherapy is a major concern for young patients with breast cancer (BC), influencing treatment decisions and quality of life. Despite established guidelines recommending fertility counseling, access to fertility preservation remains limited in Latin America.

Objectives: This study evaluated attitudes and preferences regarding fertility-related concerns and chemotherapy decision-making among young Latin American women with early-stage BC.

Design: A prospective cohort study was conducted at seven institutions in Brazil, Mexico, and Peru.

Methods: Premenopausal women aged 18-40 years with stage I-III BC requiring (neo)adjuvant chemotherapy completed a fertility questionnaire before treatment, along with validated quality-of-life assessments (EORTC QLQ-C30 and EORTC QLQ-BR23). One year after chemotherapy initiation, the patients were reassessed for ovarian function status and quality of life. Factors associated with chemotherapy acceptance despite potential infertility risks were analyzed using univariate and multivariate Poisson regression models.

Results: A total of 270 patients were included (mean age, 33.9 years). Prior to diagnosis, 41.5% of the women had children, and 31.1% expressed a desire for future childbearing. Among the participants, 8.5% were unaware of chemotherapy-induced infertility risks, 21.5% would decline chemotherapy if the infertility risk exceeded 25%, and 20.0% would accept treatment despite a 76%-100% infertility risk. In addition, 44.1% of patients required at least a 20% increase in survival probability to accept chemotherapy. In the multivariate analysis, married patients were significantly less likely to refuse chemotherapy (risk ratio: 0.88, 95% confidence interval: 0.82-0.94; p < 0.01). One year post-treatment, 73.1% of the patients experienced chemotherapy-induced amenorrhea.

Conclusion: Fertility concerns significantly impact chemotherapy decision-making in young Latin American patients with BC. Limited fertility awareness, socioeconomic disparities, and restricted access to fertility preservation contribute to these challenges. Strengthening fertility counseling and improving access to preservation options are essential for supporting informed treatment decisions in this population.

Trial registration: (ClinicalTrials.gov): NCT02862990.