Treatment Patterns Research Articles

Treatment patterns in metastatic early-onset rectal cancer.

Early onset rectal cancer (EORC) is increasing internationally. While EORC cancer presents with some distinct clinical features, there is currently insufficient evidence that age of onset should alter treatment. This study examines treatment patterns for EO versus late-onset (LO) metastatic rectal cancer in Canterbury, New Zealand, to better understand appropriate treatment strategies and there effect on patient outcomes. A retrospective study on all patients diagnosed with stage 4 rectal adenocarcinoma in Canterbury from 2010 to 2021 was undertaken. Patients under 50 were compared to a control group aged 60-74, analysing treatment patterns, hospital stays, and survival outcomes. Between 2010 and 2021, there were 949 rectal cancer diagnoses in Canterbury, of which 23 were EO and 64 were LO with stage 4 cancer. Survival analysis revealed a significant difference in median survival times between EORC (47.9 months) and LORC patients (26.5 months; P = 0.03). There was no significant difference in the surgical or oncological management between age groups (P > 0.05). Mean admissions per 100 days of life was 0.45 in LORC and 0.44 in EORC (P = 0.9119). There was no significant difference in the median proportion of time spent in hospital between EO and LO groups (2.5 vs. 2.2 days for every 100 days of life, P = 0.88). Surgical and oncological treatments were similar for both EORC and LORC groups. The EO group exhibited better survival, with hospitalization burdens comparable for both. These findings underline the importance of maintaining an approach to metastatic RC balancing survival and quality of life.

Real-world perioperative treatment patterns and economic burden of recurrence in early-stage HER2-negative breast cancer: a SEER-Medicare study

ABSTRACT Aim This study aimed to describe treatment patterns and quantify the economic impact of recurrence in early-stage human epidermal growth factor receptor 2 (HER2)-negative breast cancer (BC). Materials & Methods Medicare beneficiaries with stages I–III HER2-negative BC and lumpectomy or partial/total mastectomy were identified from SEER-Medicare data (2010–2019). Perioperative therapies were reported in the neoadjuvant and adjuvant setting. Locoregional recurrence and distant metastasis were identified using a claims-based algorithm developed with clinical input and consisting of diagnosis-based and treatment-based indicators. All-cause and BC-related healthcare resource utilization (HRU) per-patient-month and monthly healthcare costs were estimated from the recurrence date for patients with recurrence and from an imputed index date for patients without recurrence using frequency matching. HRU and costs were compared between groups stratified by hormone receptor-positive (HR+) or triple negative BC (TNBC) using multivariable regression models. Results Of 28,655 patients, 8.5% experienced recurrence, 90.4% had HR + disease, and 5.6% received neoadjuvant therapy. Relative to patients without recurrence, patients with recurrence had more advanced disease (stage II/III: 73.7% vs. 34.0%) and higher-grade tumors (Grade 3/4: 40.6% vs. 18.0%) at diagnosis. Recurrence in HR+/HER2-negative BC and TNBC was associated with higher rates of all-cause hospitalizations (incidence rate ratio [IRR]: 2.84 and 3.65), emergency department (ED) visits (IRR: 1.75 and 2.00), and outpatient visits (IRR: 1.46 and 1.55; all p < 0.001). Similarly, recurrence was associated with higher rates of BC-related HRU, particularly for ED visits in HR+/HER2-negative BC (IRR: 4.24; p < 0.001) and hospitalizations in TNBC (IRR: 11.71; p < 0.001). Patients with HR+/HER2-negative BC and TNBC recurrence incurred higher monthly all-cause (cost difference [CD]: $3,988 and $4,651) and BC-related healthcare costs (CD: $3,743 and $5,819). Conclusions Our findings highlight the considerable economic burden of recurrence in early-stage HER2-negative BC and underscore the unmet need for optimization of therapies that reduce recurrence in this population.

Behçet's disease: incidence, prevalence, and real-word data on the use of biologic agents in Japan.

Behçet's disease (BD) is an autoinflammatory disease that can affect multiple organs, including the gastrointestinal tract. Conventional management comprises anti-inflammatory drugs such as glucocorticoids (GCs) and/or immunomodulators that alleviate symptoms. The introduction of biological agents that target tumor necrosis factorα (TNF-α) has improved disease management. The goal of this work was to analyze the current prevalence and incidence of total BD and gastrointestinal Behçet's disease (GIBD) in Japan, and examine treatment trends, especially regarding the use of TNF-α inhibitors (TNFαi). We performed a retrospective descriptive observational study in which BD and GIBD demographic trends, medical treatment patterns, and reported adverse events (AEs) were assessed among patients with data recorded between 2017 and 2021 in the Japan Medical Data Center Claims Database (now JMDC Inc.). Prevalence of BD and GIBD in Japan during the observation period increased at an annual rate of + 3% and + 4%, respectively, while incidence decreased by - 5% and - 2%, with a more prominent decline in confirmed GIBD cases (- 15%). Although GCs were the most common initial treatment administered, use of TNFαi for BD and GIBD management increased by + 5.6% and + 8.1%, respectively. Severe AEs (mainly pneumonia and GI-associated AEs) were reported in 40% of patients receiving TNFαi; however, a high retention rate (of up to 80%) was observed 3years after treatment initiation. The use of TNFαi for GIBD treatment has increased in Japan in recent years. Additional research is necessary to further evaluate TNFαi effectiveness in GIBD and other BD subtypes.

Trial eligibility, treatment patterns and outcome for venetoclax-based therapy in AML: a prospective cohort study.

Venetoclax (Ven) plus hypomethylating agents are considered standard-of-care for patients with acute myeloid leukemia (AML) judged ineligible for intensive chemotherapy (IC). Real-world studies complement clinical trials, since patterns of patient selection, treatment-exposure and post-remission management may vary. This prospective observational multi-center study included 209 newly diagnosed IC-ineligible patients with a median age 75 years (interquartile range, 71-81 years). A high proportion of patients had secondary AML (53.7%), adverse-risk disease (35.3%), and complex karyotype (15.5%). At a median follow-up of 22.5 months (range 0.1-43), median overall-survival (mOS) was 11.7 months (95% Confidence Interval [CI] 9.9,15.4). Composite complete remission (CRc) was achieved in 65.2% (CR 44.4%; CRi 20.8%). Of responding patients, 21.1% underwent stem-cell transplantation. When stratified based on VIALE-A original eligibility criteria, mOS was 17.8 months for patients meeting eligibility criteria and 10.7 months for patients who did not (p=0.027). AML ontogeny (p=0.024), reduced kidney function (p=0.001), Charlson co-morbidity index (CCI; p=0.0017), ELN-risk (p=0.01) and body-mass index (p=0.0298) were significantly associated with OS. Multivariant Cox-regression analysis confirmed independent association of OS with AML ontogeny (p=0.012), CCI (p=0.033) and ELN-risk (p=0.019). Patients enrolled in the latter half of the study period demonstrated improved OS compared to those enrolled earlier (p=0.026). This prospective observational study highlights outcomes of patient subgroups, including those excluded from registration trials. (clinicaltrials.gov: #NCT03987958).

Buprenorphine prescribing practices for older adults in 2019 and 2020.

Opioid use disorder (OUD) prevalence has increased threefold among Medicare beneficiaries 65 years and older (hereafter "older adults") since 2013, with a prevalence of 15.7 per 1000 Medicare beneficiaries in 2018. Yet, access to treatment that addresses the needs of older adults with OUD is limited, including patterns of buprenorphine prescribing to older adults. Therefore, we sought to describe buprenorphine treatment patterns among older adults and prescribing clinician specialties. We conducted a retrospective observational study using 2019-2020 IQVIA Real World Data-Longitudinal Prescriptions. High-prescribing clinicians are those who prescribed buprenorphine to >15 unique older adults within a calendar year. We used T-test and chi-square tests to compare characteristics associated with differences in prescribing. Among 26,202 clinicians prescribing buprenorphine to 67,921 unique older adults, 1232 (5%) prescribed to more than 15 older adults (mean 28.1, SD = 23.6), corresponding to buprenorphine prescribing for 24,672 (36%) older adults. Among older adults dispensed buprenorphine, the majority (58%) were 66-70 years of age and male (54%). Individuals older than 70 years (44%) and males (51%) were slightly more common among high prescribers than non-high-prescribing clinicians (41% and 49%, respectively). Primary care clinicians (42%) and advance practice providers (APPs) (29%) were the most common specialties prescribing buprenorphine. Most (87%) buprenorphine prescribers were in urban counties; with a slightly higher percentage of high-prescribing clinicians in urban counties (91%). A relatively small subset of clinicians prescribes most buprenorphine to older adults. This work provides evidence of gaps in care delivery. Scalable systems-level interventions should be developed and tested to improve treatment availability mindful of existing clinical infrastructure.

One-Year Insights into the GLOBOSTAD Multinational Prospective Observational Study of Patients Receiving Dupilumab for Atopic Dermatitis.

Currently, limited data are available on long-term use of dupilumab to treat atopic dermatitis (AD) in a multinational real-world setting. The aim of this analysis was to report the interim 1-year data for patients with AD enrolled in the GLOBOSTAD registry, including treatment patterns, dupilumab effectiveness and safety, and healthcare burden. GLOBOSTAD is an ongoing, 5-year, multinational, prospective, observational study of adult/adolescent (aged ≥ 12years at baseline) patients with AD who initiated dupilumab in real-world settings according to their local country-specific prescribing guidelines. Outcomes were evaluated at baseline and at 3, 6 and 12months and included Eczema Area and Severity Index (EASI) total score, SCORing Atopic Dermatitis (SCORAD) total score, percent body surface area (BSA) affected, Patient-Oriented Eczema Measure (POEM), Dermatology Life Quality Index (DLQI) total score for adults or Children's Dermatology Life Quality Index (CDLQI) total score for adolescents and pruritus Numeric Rating Scale (NRS) total score. At the interim 1-year cut-off (March 2023), 955 patients were enrolled in GLOBOSTAD, and follow-up data were obtained from 903 patients. After dupilumab initiation, mean improvements in effectiveness outcome measures from baseline to month 3 were EASI from 25.1 to 6.1, SCORAD 59.3 to 25.3, POEM 19.7 to 8.7, DLQI 13.7 to 5.3, CDLQI 12.2 to 2.7 and pruritus NRS 6.3 to 2.5, with each measure exceeding the minimal clinically important difference. These positive changes in effectiveness outcomes were maintained or further improved through 12months since treatment initiation. AD-related hospitalizations and emergency room or urgent care facility visits decreased from 11.1% to 1.7% from baseline to month 12. In a multinational real-world setting, dupilumab demonstrated rapid, robust and sustained effectiveness in patients with moderate-to-severe AD across multiple disease domains, including AD signs, symptoms, quality of life and emergency/urgent care visits. Safety was consistent with the known dupilumab safety profile. ClinicalTrials.gov Identifier NCT03992417.

A contemporary analysis of the Australian clinical and genetic landscape of spinal muscular atrophy: a registry based study

New paradigms of diagnosis and treatment have changed the neurodegenerative trajectory for individuals with spinal muscular atrophy (SMA). Registries are a critical tool to provide real-world data on treatment patterns, their effects and health care provision within this evolving paradigm of care. This study aimed to evaluate the phenotypic and genotypic landscape, treatment patterns and health impact of SMA in Australia through the national registry. This cross-sectional study investigated demographic, clinical and genetic information, sequelae of weakness, treatment patterns and patient-reported outcomes amongst individuals with SMA enrolled in the Australian Neuromuscular Disease Registry (ANMDR) from 1st January 2020 to 30th April 2023. Descriptive statistics were used for analysis and Chi-Squared or Fisher's exact tests for associations. 195 individuals with SMA enrolled into the ANMDR. 5/195 (2.6%) were deceased by censor date. Of (n=190) individuals living with SMA, 104/190 (54.7%) were children. Minimum Australian prevalence was 0.73/100,000. SMN2 copies were inversely associated with phenotype in those with homozygous SMN1 deletions (p<0.0001)). Treatment was utilised in 154/190 (81%) of the population, with 65/137 (47.6%) of individuals perceiving improvements with therapeutic intervention on Patient/Parent Global Impression of Improvement scale (p<0.0001). Engagement with multidisciplinary care practitioners was significantly higher among children with SMA than adults (93% versus 12%, p<0.0001). Despite diagnostic and therapeutic advances, mortality and the multi-systemic health impact of SMA continue to be experienced within the Australian population. Healthcare provision must align with patient-centred outcomes, adapting to meeting their changing but ongoing care requirements. The study identified the considerable unmet need for multidisciplinary care, not only for adults with SMA but also for the emerging cohort of treated children, emphasising the imperative for comprehensive healthcare provision to address their evolving needs. No funding was received for this study.

Treatment patterns and clinical outcomes of immunocompromised patients with COVID-19 receiving remdesivir in the inpatient setting in Japan.

Remdesivir (RDV) was approved in Japan for the treatment of coronavirus disease 2019 (COVID-19) in May 2020. However, large-scale research describing the characterized use of RDV in the inpatient setting in Japan is limited. This study aimed to describe the treatment patterns and clinical outcomes of immunocompromised patients with COVID-19 treated with RDV. We used a secondary nationwide administrative claims database of acute care hospitals in Japan. The study period was from October 19, 2020, to September 30, 2022. Patients with COVID-19 treated with RDV during hospitalization were included, and the proportion of patients with death and disease progression were calculated. The analysis included a total of 2,171 immunocompromised patients treated with RDV. The mean (standard deviation) age at index date was 75.1 (13.6) years. The median time to RDV initiation from hospitalization was 1.0 days (Q1-Q3: 1.0-2.0), and the median duration of RDV treatment was 5.0 days (Q1-Q3: 3.0-5.0). At RDV initiation, 53.02% (n=1,151) of patients required non-invasive positive pressure ventilation, nasal high-flow or low-flow oxygen, and 2.26% (n=49) required mechanical ventilation, extracorporeal membrane oxygenation, or intensive care unit admission. Inpatient mortality rate by day 28 was 8.98% (95% confidence interval, 7.81-10.26). By day 28, the rate of disease progression was 9.86% (n=214), and 76.83% (n=1,668) were discharged. This study presents practical information on outcomes and treatment patterns of RDV in immunocompromised inpatients diagnosed with COVID-19.

Differential Outcomes and Treatment in Non-traumatic Carotid versus Vertebral Cervical Artery Dissection: A National Inpatient Sample Study.

Cervical artery dissection (CAD) involves the carotid or vertebral artery. However, limited studies have compared their clinical features and outcomes. We examined non-traumatic CAD patients from the National Inpatient Sample (2005-2019). Those diagnosed with both carotid and vertebral artery dissections were excluded. The evaluation included patient demographics, comorbidities, and treatment. Differences between carotid and vertebral dissections were assessed using survey-weighted stepwise backward logistic regression, followed by adjusted multivariable regressions for the primary outcomes of in-hospital mortality and routine discharge (to home or self-care). From 2005 to 2019, we included 123,224 non-traumatic CAD admissions without concurrent vertebral and carotid dissections. There were more carotid dissections than vertebral dissections (67,533 vs. 55,691). Compared to vertebral dissection, carotid dissection was associated with older age (54 [44-66] years vs. 49 [37-62] years, P<0.001), Black race (12.6% vs. 8.8%, P<0.001), White race (73.5% vs. 72.0%, P = 0.024), atrial fibrillation (10.8% vs. 6.3%, P<0.001), aortic dissection (7.7% vs. 0.3%, P<0.001), and fewer concurrent acute ischemic strokes (AIS) (47.8% vs. 56.7%, P<0.001).Within the concurrent AIS group, carotid CAD patients exhibited higher NIHSS (8 [2-17] vs. 2 [0-5], P<0.001), an increased utilization of intravenous thrombolysis (6.7% vs. 2.7% P<0.001), endovascular therapy (13.3% vs. 3.3%, P<0.001), and angioplasties (9.8% vs. 0.3%, P<0.001). Overall, carotid CAD was associated with more inpatient deaths (aOR 1.30, 95% CI 1.04-1.62, P=0.023) and less routine discharges (aOR 0.88, 95% CI 0.79-0.97, P=0.014) than vertebral CAD. Our study underscores distinct comorbidity profiles and treatment patterns between non-traumatic carotid dissection and vertebral dissection groups. These findings advocate for tailored treatment strategies based on dissection type to optimize patient outcomes.

Differences in the treatment patterns of small renal masses: A disaggregated analyses by race/ethnicity

ObjectiveTo characterize differences in the management of small renal masses among disaggregated race/ethnic subgroups. Material and MethodsWe used the National Cancer Database to identify patients diagnosed with clinically localized kidney cancer and tumor size ≤4cm. We studied 16 predefined racial/ethnic subgroups and compared 1) the use of surveillance for tumors <2cm and 2) the use of radical nephrectomy for tumors ≤4cm. We used multivariable logistic regression to evaluate the independent association of race/ethnicity with management, adjusting for baseline characteristics. We also compared our disaggregated analyses to the 6 National Institute of Health aggregate race categories. ResultsWe identified 286,063 patients that met inclusion criteria. For tumors <2cm, Black Non-Hispanic (aOR 1.43) and Mexican patients (aOR 1.29) were significantly more likely to undergo surveillance compared to White patients. For tumors ≤4cm, Black Non-Hispanic (aOR 1.43), Filipino (aOR 1.28), Japanese (aOR 1.28), Mexican (aOR 1.32), and Native Indian patients (aOR 1.15) were significantly more likely to undergo radical nephrectomy compared to White patients. When comparing our disaggregated analyses to the NIH categories, we found that many disaggregated race/ethnic subgroups had associations with management strategies that were not represented by their aggregated group. ConclusionsIn this study, we found that the use of surveillance for tumors <2cm and radical nephrectomy for tumors ≤4cm varied significantly among certain race/ethnic subgroups. Our disaggregated approach provides information on differences in treatment patterns in particular subgroups that warrant further study to optimize kidney cancer care for all patients.

Evaluating transportability of overall survival estimates from US to UK populations receiving first-line treatment for advanced non-small cell lung cancer: a retrospective cohort study

ObjectivesThe objective of this study is to explore how the UK versus the USA compare in patient characteristics, treatment patterns and overall survival (OS) of patients with advanced non-small cell...

Prostate cancer incidence rates, trends, and treatment related to prostate-specific antigen screening recommendations in the United States

BackgroundChanges in US prostate-specific antigen (PSA) screening guidelines have impacted prostate cancer (PCa) incidence rates and trends. This study shows corresponding changes in PCa incidence rates and describes treatment patterns by tumor stage, age, and race/ethnicity. MethodsAnalyses were based on 777,152 cases diagnosed in 17 population-based tumor registries in the SEER Program of the US National Cancer Institute, 2007–2021. Rates were age adjusted and trends assessed using annual percent change and joinpoint regression. ResultsPCa age-adjusted incidence rates (per 100,000) fell from 165.8 in 2007 to 101 in 2014 (APC=-6.51, p<0.05) and then rose to 121.2 in 2021 (APC=1.87, p< 0.05). The significant fall and rise in rates appeared in ages 55–69 and ≥70, all racial/ethnic groups (except in Hispanics during 2014–2021), and in local/regional stage. PCa incidence rates by tumor stage, age, and race/ethnicity were presented and discussed. A few PCa cases were identified through autopsy or death certificate among the racial/ethnic groups (0.68 %-1.37 %). Unstaged cases identified through sources other than autopsy or death certificate significantly varied by race/ethnicity, ranging from 4.13 % for non-Hispanic (NH) Whites to 9.5 % for Hispanic (Chi-square p <.0001). In 2017–2021, surgery occurred in 33 % of cases (primarily in local/regional cases, inversely associated with age), radiation in 30 % of cases (increasing with age in local/regional stage cases and decreasing with age in distant stage cases), and chemotherapy in 2 % of cases (<1 % in local/regional stage cases and 9 %-38 % for age groups in distant stage cases). Several racial/ethnic differences in treatment exist, such as NH Blacks (vs. NH Whites) with local/regional disease were significantly less likely to receive surgery and more likely to receive radiation. ConclusionsUnderstanding trends and patterns of PCa rates and treatment among patients by tumor stage, age, and race/ethnicity can guide public health planning in relation to screening and treatment.

Real-world treatment patterns and clinical outcomes in patients with multiple myeloma previously treated with lenalidomide and an anti-CD38 monoclonal antibody

Real-world treatment patterns and clinical outcomes in patients with multiple myeloma previously treated with lenalidomide and an anti-CD38 monoclonal antibody

Dental Treatment Patterns in Pediatric Patients with Rare Diseases: A Retrospective Analysis at Yonsei University Dental Hospital

This study aimed to investigate dental treatment patterns in pediatric patients with rare diseases at Yonsei University Dental Hospital over 3 years. Data of 786 pediatric patients diagnosed with rare diseases who received dental treatment between January 2020 and December 2022 were analyzed retrospectively. Patients were identified through electronic medical record screening for rare disease codes registered with the Korea Disease Control and Prevention Agency. Demographics, rare disease categories, and dental treatment methods and modalities were analyzed using chi-square tests, Fisher’s exact tests, t-tests, and logistic regression. Significant differences in dental treatment needs and methods were observed among categories. Patients with nervous system diseases were less likely to receive preventive or restorative treatments, such as sealants (&lt;i&gt;p&lt;/i&gt; = 0.0062) and composite restoration (&lt;i&gt;p&lt;/i&gt; = 0.0171), and had a higher prevalence of disabilities (&lt;i&gt;p&lt;/i&gt; &lt; 0.00001), leading to greater dental care challenges. Conversely, patients with congenital malformations required more extensive treatments, including stainless-steel crowns (&lt;i&gt;p&lt;/i&gt; = 0.0118) and surgery (&lt;i&gt;p&lt;/i&gt; = 0.0234), often under general anesthesia (&lt;i&gt;p&lt;/i&gt; = 0.0337). The study underscores the importance of tailored dental care protocols and interdisciplinary collaboration to address the needs of pediatric patients with rare diseases, enhancing their oral health outcomes and quality of life.

Real-world diagnostic, referral, and treatment patterns in early Alzheimer's disease among community-based practices in the United States.

Over 90% of individuals with mild cognitive impairment (MCI) may not receive a timely diagnosis. Understanding community-based practice patterns, where most individuals are seen, is critical to improving patient care. To understand how patients with MCI and mild dementia due to Alzheimer's disease (AD) are diagnosed and managed in community-based settings, including the use of clinical and cognitive assessments, referrals to dementia-related specialties, and receipt of treatment. This observational study recruited community-based primary care physicians (PCPs) (N = 177) and neurologists (N = 147) in August-September 2023, through a verified physician panel with broad geographic representation across the US. Physicians abstracted medical chart data from patients diagnosed with MCI or mild AD within the previous two years. Data collected included use of neurocognitive assessments, biomarker and structural imagine tests, referrals, and treatments. Descriptive statistics were used. Medical records for 817 MCI and 467 mild AD patients were abstracted. The mean age was 70.2 years, 56.4% were female, and 67.2% were White. Symptoms were commonly reported by a family member (67.2%). Nearly 1 in 6 patients did not receive any neurocognitive assessments (16.1%), and nearly 1 in 4 did not receive a structural imaging or AD-specific biomarker test (23.7%). AD-specific biomarker tests were more common among patients aged ≥65 (87.1% versus 75.3%; p < 0.05). Less than 1 in 4 patients were referred for cognitive/behavioral concerns. As the diagnostic and treatment landscape changes, education on symptom recognition, and physician training on new technologies may facilitate timely diagnoses and improve patient outcomes.

Characteristics, treatment patterns and survival of patients with high-risk early hormone receptor-positive breast cancer in French real-world settings: an exploratory study of the CANTO cohort

Patients with hormone receptor-positive, human epidermal growth factor receptor 2 (HER2)-negative breast cancer (HR+ BC) with unfavorable features have an increased risk of relapse and are currently candidate for additional treatment strategies. We evaluated the real-world clinicopathological characteristics, treatment patterns and survival outcomes of these patients within the CANcer TOxicities study (CANTO, NCT01993498). This is a retrospective analysis of the prospective data collected within CANTO between 2012 and 2022. Patients with high-risk HR+ BC were defined either by the identification of at least four positive axillary lymph nodes (LNs) or one to three positive axillary LNs with a tumor size ≥5 cm or histologic grade 3 (cohort 1). The definition 1-3 positive LNs with Ki-67 ≥20% was also considered (cohort 2). The Kaplan-Meier method was used for survival analysis. Patients with high-risk HR+ BC represented 15.0%-19.6% of HR+ BC (cohort 1 and 2, respectively) in the CANTO cohort. Of the 1266 patients in cohort 1, 617 patients (49.0%) had ≥4 LNs, 327 (26.0%) had tumor ≥5 cm and 727 (57.6%) had grade III tumors. 79.9% had a favorable Charlson comorbidity score and 88.1% stage II/IIIA. Patients with ≥10 LNs accounted for 11.8%. (Neo)adjuvant chemotherapy was administered in 94.2%. Endocrine therapy was prescribed in 97.3%, mostly with aromatase inhibitors and discontinued in 34.3%, mainly for adverse events. Patients enrolled at least 6 years before data extraction had a 5-year invasive disease-free survival and 5-year distant relapse-free survival of 79.9% [95% confidence interval (CI) 77.2% to 82.4%] and 83.5% (95% CI 80.9% to 85.7%), respectively. This real-world study confirms that patients with HR+ BC and unfavorable clinicopathological features are at risk of relapse early in their adjuvant treatment trajectory, despite (neo)adjuvant chemotherapy. It is imperative to implement innovative treatment approaches for high-risk patients, ideally adding them as early as possible to the adjuvant treatment.

Immunotherapy Following Anaplastic Lymphoma Kinase Inhibitor Therapy for Patients with Anaplastic Lymphoma Kinase‑Positive Non‑small Cell Lung Cancer in Japan.

Although anaplastic lymphoma kinase inhibitors (ALKis) are the effective initial treatment for patients with anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC), most patients experience resistance to ALKis, leading to the need for alternative therapies. Immune checkpoint inhibitors (ICIs) are a standard NSCLC treatment. On the other hand, their efficacy remains unclear for ALK-positive NSCLC. We aim to describe the treatment patterns and treatment outcomes for patients with ALK-positive NSCLC receiving later-line ICI treatment. This retrospective cohort study used claims data from Japanese acute care hospitals and included patients with lung cancer (International Classification of Diseases, 10th version (ICD-10), code: C34) diagnosed between 1 December 2015 and 31 January 2023. We extracted patients who received ALKis as first-line therapy and subsequent lines of treatment. Patient characteristics and treatment patterns and durations were descriptively summarized. Time to treatment discontinuation (TTD) for ICIs was examined using Kaplan-Meier estimates. Of 478 patients who received ALKi as first-line treatment, 30 received ICIs, 249 ALKis, and 154 non-ICI/ALKi therapy as second-line treatment. Most patient characteristics showed no differences among the groups. ICIs were more likely to be administered to patients who underwent shorter durations of ALKi treatment. The median TTD for ICIs was 66 days, with a 1 year TTD rate of 13%. Given the rarity of ALK-positive NSCLC, this study contributes to add evidence through an expanded database and increased sample size, supporting previous suggestions that ICIs have limited effectiveness in patients positive for ALK.

The IMPACT Survey: the humanistic impact of osteogenesis imperfecta in adults

BackgroundThe IMPACT Survey explored the humanistic, clinical, and economic burden of osteogenesis imperfecta (OI) on individuals with OI, their families, caregivers, and wider society. Two previous publications report research methodology, initial insights of the survey, and cost of illness of OI. Here, we present data on the impact of OI on the quality of life (QoL) of adults with OI and explore potential drivers of this impact.MethodsThe IMPACT Survey was an international mixed methods online survey in eight languages (fielded July–September 2021), aimed at adults (aged ≥ 18 years) or adolescents (aged 12–17 years) with OI, caregivers (with or without OI) of individuals with OI, and other close relatives. Survey domains included demographics, socioeconomic factors, clinical characteristics, treatment patterns, QoL, and health economics. We conducted a descriptive analysis of the QoL data, as well as exploratory regression analyses to identify drivers of impact of OI on QoL (independent associations between patient characteristics and the impact on QoL).Results1,440 adults with OI participated in the survey. The proportion who reported an impact of OI on their QoL across individual areas in the physical, socioeconomic, and mental well-being domains ranged between 49 and 84%. For instance, 84% of adults reported an impact of OI on the types of leisure activities they could do and 74% on the type of job they could do. More severe self-reported OI and higher fracture frequency were consistently identified as drivers of OI’s impact on QoL. The proportion of adults who reported worrying about different aspects of their lives due to their OI, such as mobility loss, future fractures, and ageing, ranged between 31 and 97%.ConclusionIMPACT provides insights into the humanistic burden of OI on adults, revealing that OI has a substantial impact on the QoL of adults. OI severity and fracture frequency were consistently identified as drivers of impact on QoL across all domains. Understanding these drivers may aid in identifying areas for targeted interventions, such as fracture prevention.

Clinical trial emulation in nephrology.

Trial emulation, also known as target trial emulation, has significantly advanced epidemiology and causal inference by providing a robust framework for deriving causal relationships from observational data. This approach aims to reduce biases and confounding factors inherent in observational studies, thereby improving the validity of causal inferences. By designing observational studies to mimic randomized controlled trials (RCTs) as closely as possible, researchers can better control for confounding and bias. Key components of trial emulation include defining a clear time-zero, simulating random assignment using techniques like propensity score matching and inverse probability treatment weighting, assessing group comparability by standardized mean differences and establishing a clear comparison strategy. The increasing availability of large-scale real-world databases, such as research cohorts, patient registries, and hospital records, has driven the popularity of target trial emulation. These data sources offer information on patient outcomes, treatment patterns, and disease progression in real-world settings. By applying the principles of target trial emulation to these rich data sources, researchers can design studies that provide robust causal inferences about the effects of interventions, informing clinical guidelines and regulatory decisions. Despite its advantages, trial emulation faces challenges like data quality, unmeasured confounding, and implementation complexity. Future directions include integrating trial emulation with machine learning techniques and developing methods to address unmeasured confounding. Overall, trial emulation represents a significant advancement in epidemiology, offering a valuable tool for deriving accurate and reliable causal inferences from observational data, ultimately improving public health outcomes.

Oral azacitidine maintenance after intensive chemotherapy versus venetoclax and azacitidine: real world outcomes in newly diagnosed acute myeloid leukemia

Oral azacitidine (Oral-AZA) is recommended as maintenance therapy for patients with newly diagnosed acute myeloid leukemia (ND AML) achieving remission with intensive chemotherapy (IC) but not transplant candidates; venetoclax plus injectable azacitidine (VEN-AZA) is recommended for patients ineligible for IC. Some patients may be considered candidates for either regimen. This retrospective study used Flatiron Health’s database to compare treatment patterns and clinical outcomes with Oral-AZA maintenance after IC (IC🡪Oral-AZA) versus frontline VEN-AZA. Relapse-free survival (RFS) and overall survival (OS) were analyzed at 4 different time points, including from Oral-AZA initiation (IC🡪Oral-AZA cohort) or from remission (VEN-AZA cohort) in the Core Analysis. Median RFS was 14.9 and 8.1 months for IC🡪Oral-AZA and VEN-AZA propensity score-matched cohorts, in the Core Analysis (n = 32 in each; p = 0.027); median OS was 18.7 and 15.2 months (p = 0.034). In patients with AML, IC🡪Oral-AZA significantly improved RFS and OS compared with VEN-AZA.