Tag: statistics

BMJ Open. 2023 Aug 31;13(8):e066948. doi: 10.1136/bmjopen-2022-066948.

ABSTRACT

INTRODUCTION: Although allogeneic stem cell transplantation (allo-SCT) is a curative treatment for many haematological malignancies, it is often associated with a high morbidity and mortality. Yet, little is known about the needs for supportive and palliative care among allo-SCT recipients. Moreover, targeted interventions that reduce symptom burden and suffering are still lacking. The present study aims to inform a supportive-palliative care intervention for patients with allo-SCT and their informal carers by exploring their experience and assessing their needs, especially their existential concerns, regarding four research topics: symptom burden and quality of life; coexistence of a chance for cure and a relevant risk of dying; change in goals of care; dying phase.

METHODS AND ANALYSIS: This is a descriptive mixed-methods study in progress with a convergent parallel design. Data on the four research topics will be collected and analysed separately in three steps: (1) qualitative semi-structured interviews among 20 patients, 20 informal carers and 12 healthcare providers (HCPs) and focus groups among 12-24 HCPs; (2) a quantitative cross-sectional survey with validated questionnaires and self-developed questions among 100 patients, 100 informal carers and 50 HCPs; (3) a retrospective case analysis of all deceased patients who underwent an allo-SCT between 2010 and 2019, with collection of quantitative and qualitative data. The qualitative and quantitative data sets will be finally merged for comparison and interpretation. Results will serve to develop a supportive-palliative care intervention.

ETHICS AND DISSEMINATION: The Ethics Commission of the Faculty of Medicine of the University of Cologne approved this study (20-1370_2). The study results will be published in peer-review journals, be presented at congresses and will be translated into clinical practice through the development of the palliative-supportive care intervention.

TRIAL REGISTRATION NUMBER: DRKS00027290 (German Clinical Trials Register).

PMID:37652589 | DOI:10.1136/bmjopen-2022-066948

BMJ Open. 2023 Aug 31;13(8):e071785. doi: 10.1136/bmjopen-2023-071785.

ABSTRACT

OBJECTIVE: The COVID-19 pandemic led to an increase in mortality in most countries in 2020, deviating from prior decreasing trends. In Japan, however, mortality was suggested to decrease in 2020. This study investigated long-term mortality trends and cause-specific contributions, focusing on the period of the COVID-19 pandemic in Japan.

DESIGN: We analysed Japanese age-standardised mortality rates (ASMRs) from 1995 to 2021 using vital statistics.

MAIN OUTCOME MEASURES: The cause-specific annual ASMR changes were calculated in comparison with the previous year over the abovementioned period.

RESULTS: There was a general downward trend in overall ASMR for both sexes until 2020 followed by a small increase in 2021. In men, the all-cause ASMR (per 100 000 persons) decreased from 1352.3 to 1328.8 in 2020 (-1.74% from 2019), and increased to 1356.3 in 2021 in men (+2.07% from 2020). In women, the all-cause ASMR decreased from 746.0 to 722.1 in 2020 (-3.20% from 2019), and increased to 737.9 (+2.19% from 2020) in 2021. ASMRs from malignant neoplasms, pneumonia, accidents and suicide (men only) continued to decrease during the COVID-19 pandemic while the trend of cardiovascular mortality increased in 2021. Analysis of ASMR changes revealed that COVID-19, senility, cardiovascular disease and ‘other causes not classified as major causes’ contributed to the all-cause mortality increase in 2021.

CONCLUSIONS: In Japan, the decreasing trend in overall mortality continued in 2020 despite the COVID-19 pandemic. However, approximately 2% mortality increase was observed in 2021, which was attributable to COVID-19, senility, cardiovascular disease and ‘other causes’. The year 2021 was a turning point of mortality trends in Japan, although continued monitoring is warranted.

PMID:37652585 | DOI:10.1136/bmjopen-2023-071785

Reg Anesth Pain Med. 2023 Aug 31:rapm-2023-104630. doi: 10.1136/rapm-2023-104630. Online ahead of print.

ABSTRACT

INTRODUCTION: Cancer-related pain is ubiquitous. Targeted procedural interventions may be an important and underutilized method for improving cancer-related pain and quality of life. The goal of this study was to determine the baseline educational experience and perceived post-graduation comfort of Pain Medicine fellows in performing procedures that can be used for cancer-related pain.

METHODS: Using Qualtrics, a 16-question survey was distributed to graduating fellows at accredited Pain Medicine Fellowship programs in the USA in June 2022. The fellows’ experience and comfort level performing eight procedures was analyzed using summary descriptive statistics and contingency table analysis with statistical significance determined by Pearson’s χ² test.

RESULTS: Survey respondents represented 30% of fellows among 44% of Pain Medicine programs during the 2021-2022 academic year. The majority of respondents reported no experience performing 7 of the 8 procedures for cancer-related pain (56.1%-90.6%). Graduating fellows reported overall comfort performing sympathetic neurolysis (65.7%), spinal cord stimulator trials (85.7%) and implantations (66.0%), but not intrathecal pump trials (36.9%) and implantations (31.3%), peripheral nerve stimulator implantations (41.7%), vertebral augmentations (31.3%), and vertebral body radiofrequency ablations (16.7%).

CONCLUSIONS: Experience performing 10 or more of the surveyed procedures, personal interest in treating cancer-related pain, and attendance of cancer-related pain lectures were found to significantly increase comfortability in performing cancer-related pain procedures post graduation, whereas cadaver-based learning did not. This study highlights the need for more robust procedural education for cancer-related pain and identifies procedural experience in non-cancer patients and lectures on cancer-related pain as ways to bridge this educational gap.

PMID:37652574 | DOI:10.1136/rapm-2023-104630

RMD Open. 2023 Aug;9(3):e003190. doi: 10.1136/rmdopen-2023-003190.

ABSTRACT

OBJECTIVES: In psoriatic arthritis (PsA), self-management is important for patient function and quality of life. Behaviour change can be difficult, patients could benefit from high-quality support to initiate change. Our aim was to codesign the project as theory-informed, evidence-based, patient-focused, materials supporting healthy lifestyle changes for patients diagnosed with PsA.

METHODS: Development of the materials was overseen by a steering group of patients with PsA, psychologists, rheumatologists, a design team and researchers. First, a literature review was performed to establish the evidence base for behaviours and potential interventions in PsA, including diet, weight, alcohol, smoking, exercise, anxiety, depression and stress. An initial roundtable of patients with PsA prioritised areas and content ideas. Draft materials including a website and downloadable materials were produced. A second roundtable of patients with PsA collected feedback on the draft content and design. A third roundtable was held with patients with PsA and a fourth with clinicians to refine the materials and ensuring that they were evidence based, accessible, interesting, and helpful to initiate and maintain change. A final evaluation survey was performed to review the draft website before launching the final materials.

RESULTS: 15 candidate topics were prioritised. A website and set of postcards summarising the topics were developed by the design team and refined following feedback from the roundtable groups.

CONCLUSION: This project created patient-focused resources to support behaviour change. It addresses common concerns of patients with PsA about how they may optimise their health by providing practical and brief interventions to challenge and support them to make changes.

PMID:37652555 | DOI:10.1136/rmdopen-2023-003190

RMD Open. 2023 Aug;9(3):e003268. doi: 10.1136/rmdopen-2023-003268.

ABSTRACT

OBJECTIVES: To evaluate the efficacy and safety of ustekinumab (UST) in a multicentre, randomised, double-blind, placebo-controlled trial in adult Japanese patients with active polymyositis (PM) and dermatomyositis (DM).

METHODS: Fifty-one Japanese adults diagnosed with active PM/DM who did not respond adequately to one or more standard-of-care treatments were randomised 1:1 to receive UST (n=25) or placebo (n=26). Participants received body weight-range based intravenous administration of UST (6 mg/kg) or placebo at week 0 followed by 90 mg subcutaneous (SC) administration of UST or placebo every 8 weeks from week 8 to week 24. At week 24, placebo group crossed over to receive body weight-range based intravenous administration of UST, and thereafter, all participants received/were to receive SC administration of UST 90 mg every 8 weeks (week 32 through to week 72). The primary efficacy endpoint was the proportion of participants who achieved minimal improvement (≥20) in the International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS TIS) at week 24.

RESULTS: No statistically significant difference was seen in the proportion of participants who achieved minimal improvement (≥20) in IMACS TIS at week 24 between the treatment groups (UST 64.0% vs placebo 61.5%, p=0.94) based on the primary estimand of the primary endpoint analysis.

CONCLUSIONS: UST was safe and well tolerated but did not meet the primary efficacy endpoint in adult Japanese participants with active PM/DM based on the primary analysis at week 24 in the study.

TRIAL REGISTRATION NUMBER: NCT03981744.

PMID:37652554 | DOI:10.1136/rmdopen-2023-003268

J Nucl Med. 2023 Aug 31:jnumed.123.265642. doi: 10.2967/jnumed.123.265642. Online ahead of print.

ABSTRACT

Molecular markers are of increasing importance for classifying, treating, and determining the prognosis for central nervous system tumors. Isocitrate dehydrogenase (IDH) is a critical regulator of glucose and amino acid metabolism. Our objective was to investigate metabolic reprogramming of glioma using compartmental uptake (CU) characteristics in O-(2-¹⁸F-fluoroethyl)-l-tyrosine (FET) PET and to evaluate its diagnostic potential for IDH genotyping. Methods: Between 2017 and 2022, patients with confirmed glioma were preoperatively investigated using static ¹⁸F-FET PET. Metabolic tumor volume (MTV), MTV for 60%-100% uptake (MTV₆₀), and T2-weighted and contrast-enhancing lesion volumes were automatically segmented using U-Net neural architecture and isocontouring. Volume intersections were determined using the Dice coefficient. Uptake characteristics were determined for metabolically defined compartments (central [80%-100%] and peripheral [60%-75%] areas of ¹⁸F-FET uptake). CU ratio was defined as the fraction between the peripheral and central compartments. Mean target-to-background ratio was calculated. Comparisons were performed using parametric and nonparametric tests. Receiver-operating-characteristic curves, regression, and correlation were used for statistical analysis. Results: In total, 52 participants (male, 27, female, 25; mean age ± SD, 51 ± 16 y) were evaluated. MTV₆₀ was greater and distinct from contrast-enhancing lesion volume (P = 0.046). IDH-mutated tumors presented a greater volumetric CU ratio and SUV CU ratio than IDH wild-type tumors (P < 0.05). Volumetric CU ratio determined IDH genotype with excellent diagnostic performance (area under the curve [AUC], 0.88; P < 0.001) at more than 5.49 (sensitivity, 86%, specificity, 90%), because IDH-mutated tumors presented a greater peripheral metabolic compartment than IDH wild-type tumors (P = 0.045). MTV₆₀ and MTV were not suitable for IDH classification (P > 0.05). SUV CU ratio (AUC, 0.72; P = 0.005) and target-to-background ratio (AUC, 0.68; P = 0.016) achieved modest diagnostic performance-inferior to the volumetric CU ratio. Furthermore, the classification of loss of heterozygosity of chromosomes 1p and 19q (AUC, 0.75; P = 0.019), MGMT promoter methylation (AUC, 0.70; P = 0.011), and ATRX loss (AUC, 0.73; P = 0.004) by amino acid PET was evaluated. Conclusion: We proposed parametric ¹⁸F-FET PET as a noninvasive metabolic biomarker for the evaluation of CU characteristics, which differentiated IDH genotype with excellent diagnostic performance, establishing a critical association between spatial metabolic heterogeneity, mitochondrial tricarboxylic acid cycle, and genomic features with critical implications for clinical management and the diagnostic workup of patients with central nervous system cancer.

PMID:37652542 | DOI:10.2967/jnumed.123.265642

J Nucl Med. 2023 Aug 31:jnumed.123.265853. doi: 10.2967/jnumed.123.265853. Online ahead of print.

ABSTRACT

Predictive biomarkers of response to human epidermal growth factor receptor 2 (HER2)-directed therapy are essential to inform treatment decisions. The TBCRC026 trial reported that early declines in tumor SUVs corrected for lean body mass (SUL_max) on ¹⁸F-FDG PET/CT predicted a pathologic complete response (pCR) to HER2 therapy with neoadjuvant trastuzumab and pertuzumab (HP) without chemotherapy in estrogen receptor (ER)-negative, HER2-positive breast cancer. We hypothesized that ¹⁸F-FDG PET/CT SUL_max parameters would predict recurrence-free survival (RFS) and overall survival (OS). Methods: Patients with stage II/III ER-negative, HER2-positive breast cancer received neoadjuvant HP (n = 88). pCR after HP alone was 22% (18/83), additional nonstudy neoadjuvant therapy was administered in 28% (25/88), and the majority received adjuvant therapy per physician discretion. ¹⁸F-FDG PET/CT was performed at baseline and at cycle 1, day 15 (C1D15). RFS and OS were summarized using the Kaplan-Meier method and compared between subgroups using logrank tests. Associations between ¹⁸F-FDG PET/CT (≥40% decline in SUL_max between baseline and C1D15, or C1D15 SUL_max ≤ 3) and pCR were evaluated using Cox regressions, where likelihood ratio CIs were reported because of the small numbers of events. Results: Median follow-up was 53.7 mo (83/88 evaluable), with 6 deaths and 14 RFS events. Estimated RFS and OS at 3 y was 84% (95% CI, 76%-92%) and 92% (95% CI, 87%-98%), respectively. A C1D15 SUL_max of 3 or less was associated with improved RFS (hazard ratio [HR], 0.36; 95% CI, 0.11-1.05; P = 0.06) and OS (HR, 0.14; 95% CI, 0.01-0.85; P = 0.03), the latter statistically significant. The association of an SUL_max decline of at least 40% (achieved in 59%) with RFS and OS did not reach statistical significance. pCR was associated with improved RFS (HR, 0.25; 95% CI, 0.01-1.24; P = 0.10) but did not reach statistical significance. Conclusion: For the first time, we report a potential association between a C1D15 SUL_max of 3 or less on ¹⁸F-FDG PET/CT and RFS and OS outcomes in patients with ER-negative, HER2-positive breast cancer receiving neoadjuvant HP alone. If confirmed in future studies, this imaging-based biomarker may facilitate early individualization of therapy.

PMID:37652539 | DOI:10.2967/jnumed.123.265853

Int J Gynecol Cancer. 2023 Aug 31:ijgc-2023-004621. doi: 10.1136/ijgc-2023-004621. Online ahead of print.

ABSTRACT

OBJECTIVE: To evaluate the safety and the effectiveness of thoracic epidural analgesia as part of the enhanced recovery after surgery (ERAS) multimodal analgesic protocol in patients with gynecologic oncology who have undergone laparotomy for suspected or confirmed malignancy.

METHODS: We conducted a prospective cohort study, following an enhanced recovery after surgery pathway, among patients who had undergone laparotomy for confirmed or suspected gynecological malignancy between January 2020 and September 2021. All patients who underwent laparotomy at the gynecologic oncology department for the aforementioned reason during that time were considered eligible. Patients (n=217) were divided into two groups: epidural (n=118) and non-epidural (n=99) group. Both groups were treated with the standard ERAS departmental analgesic protocol. The primary outcomes were length of hospital stay, complications, and readmission rates.

RESULTS: Data from 217 patients (epidural group, n=118 vs non-epidural group, n=99) with median age of 61 years (IQR 53-68) were analyzed. The most common type of cancer was of ovarian origin (85/217, 39.2%, p=0.055) and median (Aletti) surgical complexity score was 3 (p=0.42). No differences were observed in the patients’ demographics, clinical, and surgical characteristics. Primarily, median length of stay was 4 days in both groups with statistically significant lower IQR in the epidural group (3-5 vs 4-5, p=0.021). Complication rates were more common in the non-epidural group (38/99, 38.3% vs 36/118, 30.5%, p<0.001) with similar rates of grade III (p=0.51) and IV (0%) complications and readmission rates (p=0.51) between the two groups. Secondarily, the epidural group showed lower pain scores (p<0.001) on the day of surgery and in the first post-operative day (p<0.001), higher mobilization rates on the day of surgery (94.1% vs 57.6%, p<0.001), faster removal of urinary catheter (p<0.001), shorter time to flatus (p<0.001), and less nausea on the day of surgery (p<0.001).

CONCLUSION: In this study we showed that thoracic epidural analgesia, when used as part of an ERAS protocol, is safe and offers more favorable pain relief along with a number of additional benefits, improving the peri-operative experience of patients with gynecologic cancer.

PMID:37652530 | DOI:10.1136/ijgc-2023-004621

In Vivo. 2023 Sep-Oct;37(5):2244-2252. doi: 10.21873/invivo.13326.

ABSTRACT

BACKGROUND/AIM: The aim of this study was to evaluate patient preferences regarding cervical dysplasia clinics. Specifically, preferences in terms of diagnostic and therapeutic pathways as well as logistical and structural aspects were addressed to recognize unmet needs and improve existing structures of cervical dysplasia care.

PATIENTS AND METHODS: This questionnaire-based study was conducted between June and December 2022 at an academic medical center in Southwestern Germany. A total of 226 patients who had an appointment at the certified dysplasia clinic were included.

RESULTS: The vast majority of patients (74.8%) preferred counseling at the certified dysplasia clinic in the case of an abnormal finding of the cervix or labia. A prompt appointment (within a maximum of 4 weeks), a timely notification about test results (within a maximum of 2 weeks), a travel time <60 minutes and seeing the same doctor during follow-up appointments were recognized as important aspects. While about half of the patients (53.5%) were indifferent to the sex of the gynecologist, almost all of the remaining patients stated they would prefer to be seen by a female doctor (44.3% female doctor vs. 2.2% male doctor).

CONCLUSION: Most women expect very timely appointments and result notifications. Moreover, they favor short travel times and continuity of care. The identified patient preferences should be considered to increase patient satisfaction and quality of care when developing and optimizing management at specialized dysplasia clinics.

PMID:37652527 | DOI:10.21873/invivo.13326

In Vivo. 2023 Sep-Oct;37(5):2219-2223. doi: 10.21873/invivo.13322.

ABSTRACT

BACKGROUND/AIM: To investigate the possible association of kisspeptin levels with the ovarian reserves of women of reproductive age.

PATIENTS AND METHODS: Eighty women aged 19-40 participated after signing an informed consent. Of these, 74 were finally included as in 6 women the blood samples were considered inappropriate due to hemolysis. They were divided into three main groups according to their ovarian reserve patterns: women with adequate ovarian reserves (Group A – AOR) (n=30), women with increased ovarian reserves (Group B – PCOS) (n=31), and women with diminished ovarian reserves (Group C – DOR) (n=13).

RESULTS: Women with diminished ovarian reserves had statistically significantly increased age and FSH compared to the other two groups. No statistically significant difference was found between the three groups for estradiol and thyroid stimulating hormone. Moreover, body mass index, luteinizing hormone, total testosterone, 17-hydroxyprogesterone, dehydroepiandrosterone, anti-Mullerian hormone (AMH), and antral follicle count (AFC) were increased in group B compared to the other two groups. AMH and AFC were decreased in women with diminished ovarian reserves compared to the other two groups, as expected. The comparison of kisspeptin levels between the three groups showed that kisspeptin levels were increased in women with diminished ovarian reserves, compared to the other two groups, but without a statistically significant difference. However, kisspeptin levels in group C were statistically significantly higher than those in group A.

CONCLUSION: There are no strong indications that kisspeptin levels are associated with the ovarian reserve in women of reproductive age.

PMID:37652519 | DOI:10.21873/invivo.13322