Burden For Patients Research Articles

The impact of experiential avoidance on anxiety and depressive disorders in hematological cancer patients

Abstract Anxiety disorders and/or depressive disorders co-occurring with hematological cancer are an additional burden for patients. Experiential avoidance (EA; efforts to avoid negative emotions, thoughts, or memories) is an empirically evident transdiagnostic factor for the onset and maintenance of anxiety and depressive disorders in non-cancer populations. There is lack of evidence on the impact of EA in predicting anxiety and depression in cancer patients. A total of 291 patients with hematological cancer (60% male, mean age 55 years) were included in this cross-sectional observational study. Participants were assessed using the Structured Clinical Interview for DSM-5 mental disorders (SCID-5). EA was assessed via self-report using the Brief Experiential Avoidance Questionnaire (BEAQ). Hierarchical binomial logistic regression was conducted in order to estimate the impact of EA on anxiety and depressive disorders. A total of 38 patients (13.3%) met the diagnostic criteria for a current anxiety disorder, while 49 patients (17.2%) met the criteria for a current depressive disorder. In bivariate analyses, EA was significantly elevated in patients with an anxiety disorder in comparison to those without (54.4 vs. 48.9; p = 0.01). The same was true for depressive disorder (54.9 vs. 48.6; p < 0.01). After controlling for relevant sociodemographic and medical factors, EA did not predict anxiety or depressive disorder in separate regression models. The presence of an anxiety disorder was significantly predicted by female sex, younger age and elevated comorbidity burden. In contrast, the presence of a depressive disorder was predicted by comorbidity burden. Sociodemographic and medical predictors have greater predictive potential than EA regarding current anxiety and depressive disorder in hematological cancer patients.

Mavorixafor; CXCR4 antagonist a novel treatment for whim syndrome, first FDA approval 2024 (Editorial)

WHIM syndrome is a rare autosomal dominant disorder characterized by gain-of-function mutations in the Chemokine receptor type-4 (CXCR4 receptor). The acronym WHIM stands for Warts, Hypogammaglobulinemia, recurrent bacterial Infections, and Myelokathexis. Traditionally, WHIM syndrome has been managed with intravenous immunoglobulin (IVIG) and filgrastim (Granulocyte Colony-Stimulating Factor, G-CSF). IVIG is used to provide passive immunity to counteract hypogammaglobulinemia, while filgrastim stimulates the production and release of neutrophils from the bone marrow to address neutropenia. However, these treatments have limited efficacy, require frequent administration, and involve significant costs and patient burden. The recent Food and Drug Administration (FDA) approval of Mavorixafor, a small molecule CXCR4 antagonist, marks a significant advancement in the management of WHIM syndrome. Mavorixafor enhances white blood cell mobilization from the bone marrow, directly targeting the underlying cause of the disease. In a pivotal Phase-3 trial conducted by Badalato R et al. (2024), involving 30 participants randomized to receive Mavorixafor 400 mg daily or placebo for 52 weeks, Mavorixafor demonstrated a statistically significant increase in absolute neutrophil count (ANC) (p<0.01) and a notable reduction in annual infection frequency compared to placebo. Additionally, a Phase-2 study reported a 75% reduction in cutaneous warts and an annual infection rate of 2.27 occurrences with adequate dosing. Mavorixafor maintained a favorable safety profile and required fewer administration complexities than IVIG and filgrastim, with 78% of patients achieving an overall response compared to 10% in the placebo group. These findings underscore the potential of Mavorixafor to significantly improve clinical outcomes for patients with WHIM syndrome, offering a more effective and targeted treatment option. The approval of Mavorixafor not only enhances current therapeutic strategies but also paves the way for future research into CXCR4 antagonists, potentially revolutionizing the management of WHIM syndrome and similar immunodeficiencies.

Design considerations for future renoprotection trials in the era of multiple therapies for chronic kidney disease.

Nephrology has benefited from conducting increasingly large high-quality trials in the last 5-10 years. In addition to the long-standing known benefits of renin-angiotensin system inhibitors, we now have multiple pharmacotherapies that provide kidney and/or cardiovascular protection for certain types of patient with chronic kidney disease (CKD). These include sodium-glucose co-transporter 2 inhibitors (SGLT2i), a non-steroidal mineralocorticoid receptor antagonist and a glucagon-like peptide-1 receptor agonist. Trials of SGLT2i have had particularly important impact, as wide eligibility criteria in pivotal trials have enabled safety and efficacy across a wide range of causes of CKD to be demonstrated. These findings support the concept of final common pathways of CKD progression and should encourage similar trial designs recruiting broad ranges of patients at risk of CKD progression. This is important as these new drugs do not completely arrest CKD progression nor do they mitigate the full excess of cardiovascular disease. In the current era of multiple therapies to manage risk of CKD progression, trial design and conduct also need to consider new challenges. These include falling event rates, establishing standard of care for participants pre-randomization and improving the inclusion of trial participants understudied in previous trials. Streamlining trial design and conduct and reducing participation burden for patients and clinicians is increasingly important to facilitate larger sample sizes and to optimize adherence to study interventions and follow-up. Potential other solutions include maintaining a focus on wide generalizability (to include understudied patient groups) and empowering patients to volunteer for trials (through public and patient involvement and large-scale invitation methods), as well as innovations in trial design (including use of pre-randomization run-in periods to implement standard of care and factorial or platform trials to assess multiple treatments simultaneously).

Distress and Symptom Burden in Patients with Monoclonal Gammopathy of Undetermined Significance and Smoldering Myeloma.

Distress and Symptom Burden in Patients with Monoclonal Gammopathy of Undetermined Significance and Smoldering Myeloma.

Treatment landscape and disease burden of patients with multiple myeloma in Japan: a real-world survey.

Multiple myeloma (MM) is a hematological malignancy associated with poor health-related quality of life (HRQoL). Safe and effective treatments for MM are limited. There is a need for real-world data to improve understanding of treatment patterns and sequencing in routine clinical practice in Japan. This study evaluated disease burden, treatment patterns, treatment sequencing, and reasons for treatment selection in patients with MM in Japan. This analysis used survey data of hematologists or hemato-oncologists and their adult patients with MM who received active treatment in a real-world setting in Japan between September 2022 and May 2023. Treatment and retreatment patterns and data from several validated patient reported outcome tools were analyzed. Formal sample size calculations were not applicable. Fifty-one physicians provided data for 309 patients, of whom 52 completed a quality-of-life survey (median [interquartile range] overall health status by EQ-5D-3L questionnaire: 0.7 [0.6-1.0]). Of 309 patients, most (77%) of the first-line cohort received a lenalidomide-based therapy. Lenalidomide retreatment was common in patients with relapsed/refractory MM (80%). Poor HRQoL and high retreatment rates indicate a need for new therapy options in patients with MM in Japan. These findings may guide healthcare policies and clinical practice in Japan.

The hidden interplay between sex and adverse outcomes in incident dialysis patients: the role of aortic calcification

Abstract Background Research on the sex disparity in the prognosis of chronic kidney disease (CKD), particularly among those who are newly initiating dialysis, is limited and inconclusive. This study aimed to investigate the associations between sex and all-cause mortality and major cardiovascular adverse events (MACE), with a particular focus on the presence of aortic calcification (AC). Methods We conducted a post hoc analysis of 1459 incident dialysis patients included in this prospective cohort study. The primary outcome of interest was all-cause mortality, and the secondary endpoint was a composite of MACE. Results During a median follow-up period of 3.55 years, 362 (269 male and 93 female) patients died and 477 (342 male and 135 female) patients developed MACE. The risks for all-cause mortality (HR 0.61, 95% CI 0.47-0.79) and MACE (HR 0.74, 95% CI 0.60-0.93) were lower in females than in males. This finding was robust across multiple sensitivity analyses and most subgroups. Moreover, the associations between sex and adverse outcomes were significantly modified by AC status at dialysis initiation (p for interaction < 0.05). Specifically, among patients without AC, females exhibited lower risks for all-cause mortality (HR 0.45, 95% CI 0.29-0.69; p < 0.001) and MACE (HR 0.67, 95% CI 0.49-0.93; p=0.015),whereas no differences were observed for all-cause mortality (HR 0.82; 95% CI 0.59-1.15; p=0.256) or MACE (HR 0.80, 95% CI 0.59-1.10; p=0.174) among patients with AC. Conclusions In patients with renal failure receiving dialysis, AC abolished the survival and cardiovascular protection observed in female versus male patients. This finding supports the need for greater awareness of the AC burden in female dialysis patients.

Association between psychological flexibility and self-perceived burden in patients with cervical cancer: A computer-simulated network analysis.

The study aimed to identify the profiles of psychological flexibility in patients with cervical cancer and how self-perceived burden networked to different psychological flexibility profiles. The Personalized Psychological Flexibility Index and the Self-Perceived Burden for Cancer Patients were used to measure psychological flexibility and self-perceived burden in patients from the "Be Resilient to Cancer" project. Latent profile analysis was used to identify profiles and computer-simulated network analysis was conducted to determine if self-perceived burden networked to any of the psychological flexibility profiles. A total of 325 patients with cervical cancer were recruited in this study. Mean age was 42.26 years, the majority were married (71%) and 75.4% had early stage disease (I or II). Three profiles of psychological flexibility were identified: moderate avoidance-low acceptance/harnessing (34.8%), high avoidance-moderate acceptance/harnessing (36.9%), low avoidance and high acceptance/harnessing (28.3%). Furthermore, "worrying about damaging family relationships" and "feeling guilty about affecting family's life plans" were the most bridge symptoms as well as the targeted intervention symptoms for alleviating and aggravating the self-perceived burden respectively. Three patterns of psychological flexibility were recognized with two symptoms might be targeted to improve the self-perceived burden in women with cervical cancer.

Risk factors for mortality and financial burdens in patients with colon cancer.

300 Background: Colon cancer remains one of the most common cancers of the digestive system and continues to rank high among the leading causes of cancer-related deaths, despite the increased prevalence of screening. In this study, we aimed to investigate risk factors for mortality and health care costs in hospitalized patients diagnosed with colon cancer. Methods: Patients admitted to the hospital in 2019 and 2020 were identified using the Nationwide Inpatient Sample database and categorized into two groups based on the presence of colon cancer (with relevant ICD-10 codes). To account for confounding factors, multivariate regression analysis was applied to calculate mortality. Statistical analyses were conducted to assess mortality rates, length of stay, and hospital charges. Results: A total of 169,805 patients were admitted with a primary diagnosis of colon cancer, with a mean age of 67.6 years. The majority were White (69.9%), and 50.2% were male. Age over 60 (OR: 1.67, p < 0.001) and sepsis (OR: 16.01, p < 0.001) were associated with increased in-hospital mortality among colon cancer patients, while higher household income (76th to 100th percentile, OR: 0.71, p = 0.011) and treatment at an urban teaching facility (OR: 0.54, p < 0.001) were linked to a lower risk of in-hospital mortality. Female gender (OR: 0.88, p = 0.080) and having inflammatory bowel disease (OR: 0.67, p = 0.055) did not significantly affect in-hospital mortality, nor did Black race (OR: 1.1, p = 0.348) or Hispanic race (OR: 1.15, p = 0.287) compared to White patients. Sepsis (+11.8 days, p<0.001; +$192,699, p< 0.001), age over 60 (+0.99 days, p<0.001; +$6,134, p<0.001), and care at urban teaching hospitals (+0.54 days, p< 0.001; +$32,230, p< 0.001) were associated with longer hospital stays and higher total charges. Conversely, female gender (-0.21 days, p=0.001; -$5,529, p<0.001) and higher income (-0.69 days, p<0.001; -$1,565, p=0.466) were linked to shorter hospital stays and lower total charges. Conclusions: Early recognition of sepsis in colon cancer patients could potentially reduce in-hospital mortality rates, as well as significantly decrease costs and length of stay. The higher mortality rates observed in patients with sepsis may be attributed to the immunocompromised state associated with cancer. Implementing targeted interventions to address the higher risks in older patients and those with sepsis could improve outcomes and reduce the overall burden of colon cancer-related hospitalizations. Impact of patient demographics, socioeconomic factors, and clinical conditions on mortality, length of hospital stay, and total hospital charges. Data Mortality Length of Hospital stay Total Charge Age>60 1.67 (p< 0.001) 0.99 days, p<0.001 $6428, p< 0.001 Race (Hispanic compared to White race) 1.15 (p=0.287) 0.38 days, p= 0.004 $14,127, p<0.001 Race (Black compared to White race) 1.1 (p=0.348) 1.4 days, p< 0.001 $5370, p=0.002 Female gender 0.88 (p=0.08) -0.21 days, p= 0.001 -$5529, p< 0.001 High house hold income 0.71 (p= 0.011) -0.69 days, p<0.001 -$1565, p= 0.466 Treatment at urban teaching facility 0.54 (p<0.001 0.54 days, p<0.001 $32230, p< 0.001 Sepsis 16.01 (p<0.001) 11.8 days, p<0.001 $192699, p< 0.001 IBD 1.05 (p=0.888) 0.67 days, p=0.055 $5348, p=0.293

HER2DX in HER2-positive inflammatory breast cancer: correlative insights and comparative analysis with noninflammatory breast cancers.

The HER2DX assay predicts long-term prognosis and pathologic complete response (pCR) in patients with early-stage human epidermal growth factor receptor 2 (HER2)-positive breast cancer receiving neoadjuvant systemic therapy but has not been evaluated in inflammatory breast cancer (IBC). HER2DX was analyzed in baseline biopsy tissues from 23 patients with stage III HER2-positive IBC on a phase II trial (NCT01796197) treated with neoadjuvant trastuzumab, pertuzumab, and paclitaxel (THP). To assess the assay's predictive accuracy for pCR in IBC, clinical-pathological features and outcomes from this IBC cohort were compared with 156 patients with stage III HER2-positive non-IBC from four different cohorts. Comparative analyses included HER2DX scores, gene signatures, and expression of individual genes between patients with IBC and non-IBC. Notable differences in clinicopathological characteristics included higher pertuzumab and chemotherapy usage and lower axillary burden in patients with IBC compared with non-IBC. In the combined cohort (n= 179), HER2DX pCR score and pertuzumab use were significant predictors of pCR, but not IBC status. The pCR rates in patients treated with trastuzumab-based chemotherapy (including IBC and non-IBC) were 68.9%, 58.5%, and 16.3% in the HER2DX pCR-high, -medium, and -low groups, respectively. Comparative gene expression analysis indicated minor differences between IBC and non-IBC affecting individual HER2, immune, and proliferation genes. The HER2DX pCR score could predict pCR in stage III HER2-positive IBC following treatment with de-escalated neoadjuvant systemic therapy and in stage III HER2-positive non-IBC. Elevated pCR rates in HER2-positive IBC with high HER2DX pCR scores suggest there may be a role for treatment de-escalation in these patients and confirmatory studies are justified.

Analysis of disease burden in patients with hereditary angioedema from Japan by patient-reported outcomes.

Hereditary angioedema (HAE) symptoms can vary greatly. Disease burden evaluation is essential for providing adequate treatments for patients. Patient-reported outcome measures (PROMs), including the 12-Item Short Form Health Survey (SF-12), the Angioedema Quality of Life (AE-QoL), the Hospital Anxiety and Depression Scale (HADS), and the Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) questionnaires, were collected in 2021, before modern medications for long-term prophylaxis (LTP) of HAE were licensed in Japan. Patients also reported their HAE attack frequency as "annual" (several attacks annually), "monthly" (several attacks monthly) or "weekly" (several attacks weekly). Multiple linear regression analyses were conducted on the relationship between independent parameters (sex, age, attack frequency, HAE type, and HADS scores) and dependent parameters (AE-QoL and SF-12 scores). Fifty-four patients reported PROMs. All PROMs showed substantial health-related quality of life (HRQoL) impairment. Overall, the higher the attack frequencies, the greater the reported impairment in the PROMs tended to be. In multiple linear regression analyses, higher AE-QoL Fatigue/Mood and Fears/Shame domain scores (greater impairment) were associated with higher HADS anxiety subscale scores; higher AE-QoL total scores (greater HRQoL impairment) and lower SF-12 Physical and Mental Health Composite scores (greater general health impairment) were associated with higher HADS depression subscale scores. Patients with monthly or weekly HAE attacks reported numerically low absenteeism and numerically high presenteeism and work productivity loss as measured by the WPAI:SHP questionnaire. In this study, conducted before modern LTP options were available in Japan, patients with HAE reported notable impairment in HRQoL and work productivity. Weekly or monthly HAE attack frequencies were associated with a high disease burden. Furthermore, a substantial number of patients reported notable fatigue/mood impairment as measured by the AE-QoL and depression as measured by the HADS regardless of attack frequency. These results provide a basis for future studies evaluating the effect of LTP on the clinical manifestations and HRQoL in patients with HAE.

A novel 2-2 5-5 model for stratifying liver metastasis burden in patients with pancreatic cancer.

711 Background: In metastatic pancreatic cancer (MPC), hepatic metastasis is uniformly counted as just ‘one organ metastasis’ regardless of its metastatic tumor burden. This study aims to develop a novel stratification system for liver metastasis based on tumor burden, and to evaluate its prognostic significance in MPC patients. Methods: Patients with MPC treated at Seoul National University Bundang Hospital from 2011 to 2021 were included as the training cohort. Patients with liver-only metastasis were selected for analysis. Liver metastasis burden was categorized by the number and maximum diameter of metastases as measured from diagnostic CT images. Optimal cutoffs for metastasis number and size were determined using Cox proportional hazards regression, adjusted for chemotherapy regimen. An independent cohort from Seoul St. Mary’s Hospital and Hallym University Sacred Heart Hospital served as the validation group. The ‘H category classification’ suggested by the Japanese Society for Cancer of the Colon and Rectum (JSCCR) was also referenced. Results: A total of 1,257 patients were included, with 1,084 in the training cohort and 173 in the validation cohort. In the training cohort, data from 311 patients with liver-only metastasis were analyzed to derive the Two-two five-five (‘2-2 5-5’) criteria, classifying metastasis into Oligo-metastasis (≤2 metastases and ≤2 cm), Meso-metastasis (non-oligo and non-mega), and Mega-metastasis (≥5 metastases or ≥5 cm). Median overall survival (OS) significantly differed between the groups: Oligo (11.4 months), Meso (9.2 months, HR 1.66, 95% CI 1.14–2.43), Mega (5.6 months, HR 2.63, 95% CI 1.94–3.58) in the training cohort; Oligo (5.8 months) vs Mega (1.81 months, HR 1.94, 95% CI 1.16–3.22) in the validation cohort. Conclusions: This 2-2 5-5 model effectively stratifies MPC patients with hepatic metastasis into three prognostic groups, demonstrating significant survival differences in both training and validation cohorts. This could rationalize the independent therapeutic strategies by extent of hepatic metastasis. Demographic characteristics of patients classified according to “two-two five-five” criteria. Oligo-metastasis(n = 88) Meso-metastasis(n = 59) Mega-metastasis(n = 164) Total(n = 311) P-value* Age, median (range) 66.0 (33.2-91.0) 67.1(37.2-86.9) 68.7 (40.1-94.6) 67.9 (33.2-94.6) 0.587 Male, n (%) 43 (48) 36 (61) 91 (55) 170 (54) 0.330 Tumor location(Head), n(%) 47 (53) 27 (45) 60 (36) 134 (43) <0.05 Number of liver metastasis, median (range) 1 (1-2) 3 (1-4) 12 (2-14) 5 (1-14) <0.05 Maximal size of liver metastasis, cm (IQR)Average size of liver metastasis, cm (IQR) 10.6 (8.1-13.1)10.3 (8.0-12.1) 15.9 (10.1-24.2)13.5 (7.8-19.1) 21.6 (16.8-33.7)13.2 (10.5-19.2) 17.2 (10.9-25.2)11.9 (9.2-17.1) <0.05<0.05 CA19-9 (U/mL) 462 (103-1,334) 560 (89-1,480) 1,170 (100-9,500) 745 (100-3,700) <0.05

Prognostic Implications of Magee Equation 3 and Residual Cancer Burden in patients receiving Neoadjuvant Chemotherapy for HR-Positive HER2-Negative Breast Cancer

Prognostic Implications of Magee Equation 3 and Residual Cancer Burden in patients receiving Neoadjuvant Chemotherapy for HR-Positive HER2-Negative Breast Cancer

Oxygen consumption, effort, and weaning in the mechanically ventilated patient in the intensive care unit: The extubate study: A protocol for an observational study.

The spontaneous breathing trial (SBT) is often performed to determine whether the patient can be extubated with a minimal risk of failure. The rapid shallow breathing index (RSBI) is an important parameter used in an SBT. However, weaning failure rates are between 15 and 20%. Because of the high incidence of extubation failure a search for a better parameter is warranted. The aim of this study is to investigate whether oxygen consumption, pressure-time product, work of breathing and pressure swings predict weaning failure. This is a single-center, prospective observational study conducted at the Leiden University Medical Center. Adult patients undergoing an SBT as per the ruling protocol in the LUMC will be included. Measurements of oxygen consumption, pressure-time product, work of breathing and pressure swings will be continuously recorded during 10min prior, during and 10min after the SBT. Data collection will not interfere with clinical decision making. The primary outcome is the ability of these parameters to predict extubation success or failure. Patient enrollment started in January 2024 and inclusions are expected to be complete in January 2026. Patient's risk and burden are minimal. We hypothesize that patients who fail extubation will have higher absolute baseline efforts but will not have the potential to increase those efforts when subjected to an SBT. The study is retrospectively registered at ClinicalTrials.gov under identifier NCT06391424, submitted on 2024-04-30.

WCN25-1664 Epidemiology, disease burden and treatment patterns for primary IgA nephropathy patients: A real-world data study in The Netherlands

WCN25-1664 Epidemiology, disease burden and treatment patterns for primary IgA nephropathy patients: A real-world data study in The Netherlands

Impact of Multimorbidity on Symptom Burden and Symptom Clusters in Patients Receiving Chemotherapy.

Detailed information on patient characteristics and symptom burden associated with multimorbidity in oncology patients is extremely limited. Purposes were to determine the prevalence of low (≤ 2) and high (≥ 3) multimorbidity in a sample of oncology outpatients (n = 1343) undergoing chemotherapy and evaluate for differences between the two multimorbidity groups in demographic and clinical characteristics; the occurrence, severity, and distress of 38 symptoms; and the stability and consistency of symptom clusters. Using the Self-Administered Comorbidity Questionnaire, patients were classified into low and high multimorbidity groups. Memorial Symptom Assessment Scale was used to assess the occurrence, severity, and distress of 38 symptoms prior to the patients' second or third cycle of chemotherapy. For each multimorbidity group, symptom clusters based on occurrence rates were identified using exploratory factor analysis. Compared to the low group (61.4%), patients in the high group (38.6%) were older, had fewer years of education, were less likely to be married or partnered, less likely to be employed, and had a lower annual income. In addition, they had a higher body mass index, poorer functional status, were a longer time since their cancer diagnosis, and were more likely to have received previous cancer treatments and have metastatic disease. Patients in the low and high groups reported 12.7 (±6.7) and 15.9 (±7.5) concurrent symptoms, respectively. Eight and seven symptom clusters were identified for the low and high groups, respectively. Psychological, gastrointestinal, weight gain, hormonal, and respiratory clusters were stable across multimorbidity groups. Weight gain and respiratory clusters were consistent. Three unstable clusters were identified in the low group and two in the high group. Findings suggest that higher multimorbidity is associated with various social determinants of health and a higher symptom burden. Differences between multimorbidity groups may be related to aging, treatments, and/or comorbid conditions.

Exploration of symptom cluster patterns and their trajectory in esophageal cancer surgery patients.

To investigate symptom burden and symptom cluster trajectories in patients undergoing surgery for esophageal cancer. A convenience sample of 210 patients who underwent thoracoscopic surgery for esophageal cancer was included from July to December 2023. The symptoms of the patients were evaluated at the following time points: preoperatively (T0), 1-3 days postoperatively (T1), 7 days postoperatively (T2), 1 month postoperatively (T3), and 3 months postoperatively (T4). This was done via the Chinese version of the MD Anderson Symptom Scale and the Supplementary Questionnaire for Perioperative Esophageal Cancer Symptoms. Exploratory factor analysis was used to identify symptom clusters, and latent class growth modeling was used to analyze the trajectories of each different symptom cluster. Exploratory factor analysis revealed the presence of four symptom clusters: mood-related symptom clusters (worst at T1, T2, and T4), gastrointestinal symptom clusters (worst at T0), fatigue-related symptom clusters (worst at T3), and surgery-related symptom clusters, with cumulative variance contribution rates of 64.34%, 62.29%, 68.23%, 70.29%, and 63.82%, respectively. The latent category growth model identified the existence of 2-3 distinct trajectories in each symptom cluster. This study identified 4 distinct symptom clusters in patients undergoing esophageal cancer surgery. The mood-related and gastrointestinal symptom clusters exhibited worsening and recurrence within 7 days post-surgery, necessitating particular attention. A subset of patients in the fatigue-related symptom cluster showed a tendency for continued exacerbation, emphasizing the need for long-term monitoring. Furthermore, symptom management strategies can be prioritized based on the severity of the symptom clusters.

Evaluation of the correlation coefficient to determine the severity of bone marrow infiltration between bone marrow burden, fat fraction and Lyso GL1 in adult patients with Gaucher disease. A Study from the Argentine group for the diagnosis and treatment of GD

Evaluation of the correlation coefficient to determine the severity of bone marrow infiltration between bone marrow burden, fat fraction and Lyso GL1 in adult patients with Gaucher disease. A Study from the Argentine group for the diagnosis and treatment of GD

A single arm phase II trial of trastuzumab deruxtecan in patients with gastrooesophageal adenocarcinoma cancer who are ctDNA and HER2 positive: DECIPHER.

TPS512 Background: Oesophagogastric adenocarcinoma (OGA) is globally prevalent and frequently advanced at presentation. Less than 50% of patients with operable OGA are cured when treated with multimodality therapy. Patients in the UK with operable OGA, are treated with FLOT prior to and following surgery. However, patients who have circulating tumour DNA (ctDNA) in their blood after surgery have worse survival than ctDNA negative patients. Therefore, use of novel treatment approaches aiming to cure this micrometastatic disease are considered a rational and appropriate treatment approach. Trastuzumab deruxtecan (T-DXd), a novel HER2-targeting antibody drug conjugate, is licensed to treat advanced, previously treated HER2 positive OGA. Therefore, there is a need to evaluate T-DXd in resectable HER2 and ctDNA positive OGA, where options are limited. Methods: Co-ordinated by the Cancer Research UK Southampton Clinical Trials Unit, DECIPHER is a single arm, multicentre, phase II trial testing the effect of T-DXd on reducing micrometastatic disease burden in HER2 positive OGA patients who are ctDNA positive after chemotherapy and surgery. To be eligible, participants must have been treated with chemotherapy before surgery for at least 6 weeks and must have recovered from surgery with no evidence of metastatic disease on post-surgical imaging. HER2 positive patients are recruited to the tissue testing stage of the trial prior to surgery. Patients who are ctDNA positive after surgery will be treated with T-DXd at a dose of 6.4 mg/kg intravenously every 21 days for a maximum of 8 cycles or until disease recurrence. If required, patients may dose reduce, but no dose re-escalation will be permitted. A clinically validated, personalised, tumour-informed ctDNA assay (signatera, Natera, Inc.) will be utilised to detect ctDNA in patient plasma samples. The primary endpoint is the percentage of patients who are ctDNA negative after 4 cycles of treatment. Secondary endpoints include ctDNA clearance after each cycle, disease free survival, overall survival, quality of life and frequency of adverse events. DECIPHER (NCT05965479) opened in April 2024 and will run in approximately 15 UK secondary care hospitals with the aim of recruiting 25 evaluable patients (recruitment to the tissue testing stage is currently 27 on 23-Sep-2024). Clinical trial information: NCT05965479 .

Retrospective cohort study evaluating the economic burden of patients with Pompe disease treated with enzyme replacement therapy in the United States

Retrospective cohort study evaluating the economic burden of patients with Pompe disease treated with enzyme replacement therapy in the United States

Mortality and other outcomes of hospitalizations due to pancreatic cancer.

780 Background: Pancreatic cancer continues to be a highly fatal malignancy of the digestive system and imposes a significant financial strain on healthcare systems. Although it is often diagnosed incidentally, several risk factors have been identified, including smoking, family history, age, race, gender, and alcohol use. In this study, we aimed to examine inpatient outcomes and the healthcare burden of patients hospitalized with pancreatic cancer. Methods: Patients admitted to the hospital in 2019 and 2020 were identified using the Nationwide Inpatient Sample database and categorized into two groups based on the presence of a pancreatic cancer diagnosis (with relevant ICD-10 codes). To account for confounding factors, multivariate regression analysis was applied when calculating mortality. Statistical analyses were conducted to assess mortality rates, length of stay and hospital charges. Results: : A total of 74,584 patients met the inclusion criteria, with a mean age of 68.5 years. The majority were white (69%), while black patients made up 13% of the study population, and 49% were female (36,570 patients). Thrombosis (OR: 2.08, p < 0.001), age over 60 (OR: 1.22, p = 0.042), and being of black race (compared to white race, OR: 1.45, p = 0.001) were linked to higher mortality rates. Thrombosis was also associated with longer hospital stays (+1.9 days, p<0.001) and increased costs (+$16,003, p= 0.001). However, age over 60 (+0.26 days, p = 0.088; +$589, p = 0.833) and black race (+1.01 days, p< 0.001; +$1,493.7, p = 0.649) showed trends toward longer stays and higher costs, these were not statistically significant. On the other hand, pancreatic conditions such as chronic pancreatitis, pancreatic cysts, pseudocysts, or exocrine pancreatic insufficiency (OR: 0.589, p < 0.001), female gender (OR: 0.78, p = 0.002), and care at urban teaching facilities (OR: 0.37, p < 0.001) were associated with lower mortality. While female gender (+0.06 days, p=0.603; -$1699, p=0.448) did not significantly impact hospital length of stay or costs, pancreatic disease (+0.33 days, p = 0.031; +$10,826, p < 0.001) and being treated at urban teaching facilities (+1.8 days, p < 0.001; +$57,106, p < 0.001) were linked to longer stays and higher costs. Conclusions: Although some unmodifiable risk factors significantly increase mortality, thrombosis contributes not only to higher mortality but also to increased healthcare costs and longer hospital stays. However, it may be preventable with anticoagulation therapy. The inverse relationship between pancreatic disease and mortality could potentially be explained by more frequent imaging and earlier detection of malignancies in these patients. Analysis of factors influencing in-hospital mortality, length of hospital stay, and total charges in hospitalized patients. Data Mortality Length of hospital stay Total Charge Age>60 1.22(p=0.042) +0.26 days, p=0.088 $589, p=0.833 Race (black compared to white race) 1.45 (p=0.001) +1.01 days, p< 0.001 $1493.7, p=0.649 Female gender 0.78 (p=0.002) +0.06 days, p=0.603 -$1699, p=0.448 Treatment at urban teaching facility 0.37(p<0.001) +1.8 days, p<0.001 $57,106, p<0.001 Thrombosis 2.08(p<0.001) +1.9 days, p< 0.001 $16,003, p= 0.001 Pancreatic conditions 0.589(p<0.001) +0.33 days, p= 0.031 $10,826, p<0.001