Current Medical Research and Opinion最新文献

Objective: In the phase 3 RATIONALE-312 trial (ClinicalTrials.gov Identifier: NCT04005716), the addition of tislelizumab to chemotherapy as first-line treatment significantly improved overall survival and progression-free survival compared to placebo plus chemotherapy in patients with extensive-stage small-cell lung cancer (ES-SCLC), with an acceptable safety profile. This analysis reports the patient-reported outcomes (PROs) from RATIONALE-312.

Methods: Overall, 457 adults with ES-SCLC were randomized (1:1) to tislelizumab plus chemotherapy (n = 227) or placebo plus chemotherapy (n = 230). PROs were protocol-prespecified secondary end points that assessed health-related quality of life (HRQoL) using the EORTC QLQ-C30 and EORTC QLQ-LC13 questionnaires. PRO end points at Cycles 4 and 6 were analyzed using a mixed model for repeated measures. Time to deterioration (TTD) was evaluated.

Results: At Cycle 4, the tislelizumab arm demonstrated clinically meaningful improvement in coughing, hemoptysis, and chest pain. Clinically meaningful improvement was observed in the tislelizumab arm, but not in the placebo arm, for global health status (GHS)/QoL, dyspnea, and arm or shoulder pain. By Cycle 6, the tislelizumab arm achieved clinically meaningful improvement in GHS/QoL, with a statistically significant between-group difference favoring the tislelizumab arm. Clinically meaningful improvements in dyspnea, coughing, hemoptysis, and chest pain were maintained in the tislelizumab arm through Cycle 6. No between-group differences were observed in the TTD analysis.

Conclusions: Tislelizumab plus chemotherapy maintained or improved HRQoL and patient-reported symptoms compared to placebo plus chemotherapy. Along with prior efficacy and safety data, these data support tislelizumab plus chemotherapy as first-line treatment in patients with advanced ES-SCLC.

Background: This study presents patient-reported outcomes (PROs) from the phase 1/2 BRUIN (NCT03740529) trial of pirtobrutinib monotherapy for the treatment of B-cell malignancies, including chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) and mantle cell lymphoma (MCL).

Methods: PROs were collected at each cycle using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire and item library (IL) sets for CLL/SLL- and MCL-related symptoms and an Expanded Fatigue measure. Prespecified analyses included descriptive change from baseline, time to worsening (TTW) using Kaplan-Meier method, and longitudinal analyses using a mixed model for repeated measures.

Results: A total of 263 patients with CLL/SLL and 124 with non-blastoid MCL who received pirtobrutinib monotherapy after prior BTKi were included in the final PRO analysis. The proportion of patients with CLL/SLL who improved or remained stable from baseline through Cycle 31 remained above 80% for physical function (PF), CLL/SLL-related symptoms, fatigue, and global health status/quality of life (GHS/QoL). The proportion of patients with MCL who improved or remained stable through Cycle 20 remained above 70% for PF, MCL-related symptoms, fatigue, and GHS/QoL. Median TTW was not reached in either CLL or MCL. Longitudinal analyses for PF, CLL/SLL-related symptoms, fatigue, and GHS/QoL consistently met statistically significant and clinically-meaningful improvement from baseline for CLL. PRO assessments remained stable over time for MCL.

Conclusions: The final analysis from the BRUIN trial demonstrates stability in PROs throughout the duration of treatment with pirtobrutinib. Most patients with CLL/SLL and MCL reported stable or improved outcomes throughout the study.

Over the past few decades, patient centricity has evolved from an idea to a clearly defined component of cancer care, integrated into clinical practice: patients with cancer are now more involved in their care. The aim of this opinion article is to highlight the urgent need to involve them in preclinical and clinical research stages, across cancer types, ensuring their voices are heard, and their care needs are addressed as new and evolving cancer therapies are developed. The current oncology landscape makes patient integration particularly pertinent, as it is becoming more complex and technical, therefore creating a need for patient-level translation. This is combined with the rise of the patient voice and patients' increasing willingness to be involved in shaping research. Additionally, this article presents preliminary ideas, approaches, and current real-world initiatives to encourage greater patient integration in drug development. This article is based on the authors' experiences and is supported by a review of current literature, but it is not aiming to replicate existing comprehensive literature reviews on the subject of patient integration.

Background: This retrospective observational study was designed to evaluate healthcare resource utilization and hospital costs among patients who underwent total hip arthroplasty (THA) with a cementless, medial collared, triple tapered femoral stem (TTS) compared to the morphometric wedge (MMW) design based femoral stems.

Methods: Patients >21 years of age, undergoing cementless THA from January 2016-September 2023, and implanted with TTS vs. MMW, in Premier Healthcare Database were analyzed. Primary outcomes included length of hospital stay and 90- and 365-day all-cause readmission. Cohorts were balanced using covariate balance propensity score method and generalized linear model was used to compute outcomes.

Results: 14,238 TTS and 17,578 MMW patients were included in the analysis. Balanced cohorts had an average age of 66 years. Length of stay was significantly shorter for TTS (1.65 days vs. 1.93 days for control, p-value < 0.01). The 90-day all-cause readmission rate was 2.62% for TTS and 3.15% for MMW and the mean difference of 0.52% [0.13% to 0.92%] was significantly lower (p-value < 0.01) for TTS. The mean difference for 365-day all-cause readmission was also significantly lower for TTS (1.19% [0.53% to 1.84%], p-value < 0.01). Fewer patients were discharged to a skilled nursing facility and total cost of care at 365-day follow-up was lower for TTS compared to the MMW cohort.

Conclusions: The comparative analyses showed favorable outcomes for the TTS cohort in terms of shorter length of stay, fewer all-cause readmissions, fewer discharges to skilled nursing facility and lower cost at 365-day follow-up compared to MMW.

Objectives: Several indices are available to predict poor outcomes in older patients admitted to the emergency department (ED); however, there remains a need for more practical, sensitive, and easily applicable tools. This study aimed to develop a novel prognostic index to predict the risk of disability, hospitalization, and mortality in older patients presenting to the ED.

Methods: This study included 356 patients aged 65 and older presenting to the ED. Exclusion criteria were: poor general condition preventing response to questions, low level of consciousness (GCS <15), inability to provide informed consent, and lack of prospective follow-up. The G8 scale, PRISMA-7 questionnaire, Katz Activities of Daily Living Questionnaire (ADL) scale, and Charlson Comorbidity Index were administered. Follow-up assessments were conducted via telephone on the 30th and 180th days post-application, using the Katz ADL scale. Patients or their relatives were also queried about repeated outpatient clinic visits, emergency admissions, hospitalizations, nursing home admissions, and survival status.

Results: Parameters significantly associated with adverse outcomes in univariate analysis were further analyzed using multivariate regression, resulting in the development of the GAZI Index. This new index includes six simple parameters independently associated with adverse outcomes. The predictive performance of the GAZI Index was compared to that of the PRISMA-7, a validated tool in the ED. The GAZI Index demonstrated significantly superior predictive ability for poor outcomes compared to the PRISMA-7 (p = 0.008).

Conclusion: The GAZI Index is a novel, valid, and practical tool for predicting adverse outcomes in older patients presenting to the ED.

Background: Ustekinumab (UST) reduced symptom prevalence, work productivity loss (WPL) and indirect costs among Crohn's disease (CD) patients in clinical trial. However, little is known on real-world effectiveness of UST in relation to symptom prevalence, WPL, and indirect costs, which we aimed to evaluate in this study.

Methods: Data were drawn from the Adelphi Real World CD Disease Specific Programme, a cross-sectional survey of gastroenterologists and their CD patients in the United States of America. Gastroenterologists provided data on consulting patients capturing symptoms and treatment use; a proportion of those patients completed a voluntary questionnaire capturing the Work Productivity and Activity Impairment (WPAI) questionnaire. Outcomes were descriptively analyzed between patients receiving and not receiving UST, while symptom prevalence was compared using McNemars test.

Results: Gastroenterologists reported data on 197 UST patients and 72 non-UST patients with moderate-to-severe (M-S) disease activity. At last consultation, the proportion of patients with symptoms decreased from UST initiation to most recent consultation (p < 0.0001). Of those with WPAI data available, for UST patients (n = 39) (vs non-UST M-S, n = 39), mean percentage impairment in overall work was 21.1% (vs 51.0%), presenteeism was 19.8% (vs 44.9%) and absenteeism was 3.2% (vs 16.7%). Mean annual total work productivity loss cost/patient was $12,211 for UST vs $30,096 non-UST MS patients.

Conclusions: Patients with CD receiving UST had lower mean annual WPL costs and work impairment compared to moderate-to-severe patients with CD not receiving UST along with reduced symptomology over their UST treatment period, suggesting UST offers economic and health related quality of life benefits to this population.

Background: Natural killer/T-cell lymphoma (NKTCL) is a highly invasive non-Hodgkin lymphoma (NHL) characterized by extranodal onset, commonly observed in the nose and nasopharynx. NHL in the female reproductive system is rare, mostly secondary, occurring in the ovaries, cervix, uterine body, and endometrium. The prognosis of this disease is extremely poor, with most patients dying within a few months of diagnosis. Owing to its rarity, the prognostic factors are difficult to ascertain. Here, we report a case of extranodal NK/T-cell lymphoma in the uterus related to pregnancy.

Case report: A 31-year-old woman sought treatment for continuous vaginal discharge after cesarean section, however, anti-inflammatory treatment had no effect. A lump was found on the anterior wall of the uterus on transvaginal ultrasonography, so uterine lesion resection was performed. Owing to pathological misdiagnosis as uterine fibroids, treatment was delayed for 10 months. After readmission with a clear diagnosis, radiotherapy, chemotherapy, and autologous stem cell transplantation were done, and the disease was put into completely remission.

Conclusion: Primary uterine NKTCL is rare, and special attention should be given to women infected with Epstein-Barr Virus (EBV) during their pregnancy and post-partum period, especially patients with abnormal uterine bleeding, vaginal discharge, fever, or high LDH.

Background: As the cornerstone of ablation, cryoballoon Ablation (CBA) still has certain limitations, whereas Pulse Field Ablation (PFA) offers a non-thermal and tissue specific alternative. This review aims to compare the safety and efficacy of PFA and CBA.

Methods: A systematic search for eligible studies was conducted in trial registries and databases until August 31, 2024. A total of 1090 patients from eight studies were included.

Results: Most studies reported higher 1-year free recurrence rate in PFA, but did not yield statistical significance (RR 1.03, 95%CI: 0.94, 1.12, p = 0.55, I2 = 0%). PFA was associated with a significantly lower contrast volume (MD -41.63, 95% CI: -49.19 to -34.06, p < 0.00001, I² = 0%), but showed non-significant differences in procedural time (MD -5.84, 95% CI: -12.35 to 0.66, p = 0.08, I² = 83%) and fluoroscopy time (MD 1.43, 95% CI: -0.09 to 2.95, p = 0.07, I² = 75%). Complications were comparable, except for phrenic nerve palsy (PNP) that occurred exclusively in CBA. PFA and CBA are comparable in the terms of acute success and 1-year free recurrence rates. Repeat procedures were more frequent after PFA. However, organized atrial tachycardia (AT) was more commonly observed in the CBA group, while recurrences in both groups were predominantly Atrial Fibrillation (AF).

Conclusion: PFA offers advantages in procedural time and contrast medium use. Complication rate for PFA and CBA was similar except for PNP which exclusively occurred in CBA.