Treatment Patterns Research Articles

Disparities in Real-World Treatment Patterns Among Patients with Von Willebrand Disease in a Large US Population-Based Dataset

Disparities in Real-World Treatment Patterns Among Patients with Von Willebrand Disease in a Large US Population-Based Dataset

Real-World Patient-Reported Treatment Patterns and Treatment Satisfaction for Sickle Cell Disease Patients in the United States

Real-World Patient-Reported Treatment Patterns and Treatment Satisfaction for Sickle Cell Disease Patients in the United States

Real-World Bleeding Risk, Outcomes and Treatment Patterns of Patients with CLL/SLL Receiving BTKi with or without Prescription Anticoagulants or Antiplatelets

Real-World Bleeding Risk, Outcomes and Treatment Patterns of Patients with CLL/SLL Receiving BTKi with or without Prescription Anticoagulants or Antiplatelets

Real-World Treatment Patterns, Resource Utilization, and Cost of Care in Medicare Beneficiaries Newly Diagnosed with Acute Myeloid Leukemia and Treated with Venetoclax-Based Regimens

Real-World Treatment Patterns, Resource Utilization, and Cost of Care in Medicare Beneficiaries Newly Diagnosed with Acute Myeloid Leukemia and Treated with Venetoclax-Based Regimens

The journey of patients affected by metastatic hormone receptor-positive/HER2-negative breast cancer from CDK 4/6 inhibitors to second-line treatment: A real-world analysis of 701 patients enrolled in the GIM14/BIOMETA study

PurposeThe aim of this study was to evaluate the effectiveness of CDK 4/6 inhibitors (CDK 4–6i) according to HER2 status (low/zero), and endocrine resistance/sensitivity, as well as the efficacy of second-line treatments, in a large real-world cohort. MethodsThe GIM14/BIOMETA study (NCT02284581) is a retrospective/prospective study of the Gruppo Italiano Mammella evaluating treatment patterns and survival outcomes in patients with metastatic breast cancer (MBC). We retrieved data on patients with hormone receptor-positive/HER2-negative MBC receiving first-line CDK 4/6i. ResultsAmong 3832 patients enrolled in the GIM14-BIOMETA study, 701 were eligible. At a median follow-up of 24.80 months, no significant differences were found between HER2-zero and HER2-low subgroups in terms of first-line time to treatment discontinuation (TTD) (26.16 months [IQR 12.84-NR] vs. 27.60 months [IQR 12.12–64.44], p = 0.972) or overall survival (OS) (mOS>60 months for both groups, p = 0.398). Median TTD was 33.24 months (IQR 16.32-NR) for the endocrine sensitive subgroup, 19.92 months (IQR 8.88–51.24) for the secondary endocrine resistant subgroup and 17.40 months (IQR 7.44–24.72) for the primary endocrine resistant subset, respectively (p < 0.001). Among 239 patients receiving second-line treatment, no significant difference (p = 0.188) was found in terms of second-line TTD between those treated with capecitabine (6.11 months, IQR 2.96–11.47), taxane-based chemotherapy (5.06 months, IQR 2.99–9.99), everolimus plus exemestane (5.39 months, IQR 2.53–9.03) or fulvestrant (6.44 months, IQR 3.38-NR). ConclusionsEndocrine therapy plus CDK 4/6i represents an effective treatment, regardless of HER2 status (low/zero). Second-line agents did not differ significantly in terms of TTD. Endocrine resistant cancers exhibit poor response to CDK 4/6i.

Treatment Patterns and Glycaemic Control Between 2015 and 2019 in Tianjin, China: A Real-World Study of Adults with Type 2 Diabetes.

Diabetes is associated with a high economic burden in China; therefore, strategies to prevent diabetes, improve glycaemic control, delay disease-related complications and maintain quality of life are essential. This study was conducted to evaluate trends in treatment patterns and glycaemic control in people with type 2 diabetes (T2D) in real-world clinical practice in Tianjin, China. This retrospective, cross-sectional, multicentre study analysed data from adults with T2D living in Tianjin, China between 2015 and 2019, based on information obtained from a regional electronic medical record database. Temporal trends in treatment patterns and glycaemic control were assessed using linear regression (continuous variables), and Cochran-Armitage (two categories) or Cochran-Mantel-Haenszel (≥ 3 categories) tests. Between 2015 and 2019, data from 312,203 individuals treated at 75 hospitals were included. Over this period, there was an upward trend in the prevalence of hypertension, hyperlipidaemia, obesity, cardiovascular disease, stroke and retinopathy each year (all P < 0.001). The use of metformin or dipeptidyl peptidase-4 inhibitors increased, while thiazolidinedione, alpha-glucosidase inhibitor and glinide use decreased; the use of basal insulin (BI), glucagon-like peptide-1 receptor agonists (GLP-1 RAs), GLP-1 RAs + BI, bolus insulin and BI + bolus insulin increased, whereas the use of premixed insulin showed a downward trend (all P < 0.001). From 2015 to 2019, an increased proportion of individuals achieved glycated haemoglobin (HbA1c) < 7% (< 53 mmol/mol; 28.1-33.7%), fasting plasma glucose (FPG) < 7 mmol/l (21.7-26.9%) and postprandial glucose (PPG) < 10 mmol/l (22.0-48.2%; all P < 0.001). There was no change in the proportion of individuals with an FPG ≥ 7 mmol/l and a PPG ≥ 10 mmol/l, while the prevalence of residual hyperglycaemia increased (P < 0.001). Glycaemic control improved between 2015 and 2019 in people with T2D in Tianjin, China; however, there is an unmet need for more effective glycaemic control.

Health care utilization among Medicare beneficiaries with newly diagnosed back pain

BackgroundLow back pain (LBP) is the most common medical cause of disability among adults 65 or older. No previous study has characterized health care costs and treatment patterns of LBP among Medicare beneficiaries. MethodsThis retrospective cohort study quantifies health care utilization costs among Medicare beneficiaries with newly diagnosed LBP, compares costs between patients managed operatively and nonoperatively, identifies costs associated with treatment guideline nonadherence, and characterizes opioid prescribing patterns. Patients were queried via ICD codes from a 20% random sample of Medicare claims records. Patients with concomitant or previous “red flag” diagnoses, neurological deficits, or diagnoses that could cause nondegenerative LBP were excluded. Total costs of care in the year of diagnosis were calculated and stratified by operative versus nonoperative management. To assess for guideline adherence, utilization and costs of different services were tabulated. Opioid prescription patterns were characterized by quantity, cost, duration, and medication type. ResultsAbout 1,269,896 patients were identified; 23,919 (1.8%) underwent surgery. These accounted for 7% of the cohort's total cost ($514 million total, $21,496 per person). Patients treated nonoperatively accounted for over $7 billion in costs ($5,880 per person; p<.001). Within the nonoperative cohort, 626,896 (50.3%) patients were nonadherent to current guidelines for conservative management of LBP. Guideline nonadherence increased total annual costs by $4,012 per person ($7,873 for nonadherent vs. $3,861 for adherent patients, p<.001). About 460,867 opioid prescriptions were filled for 303,796 unique patients (23.9%) within 30 days of LBP diagnosis. Within the nonsurgical cohort, patients nonadherent to imaging guidelines were more likely to have an opioid prescription within this window than adherent patients (26.5% vs. 21.2%; p<.001). ConclusionsNonoperative management of LBP is associated with significantly lower costs per patient. Early imaging and opioid prescription are significant drivers of excess cost. Adherence to proposed treatment guidelines can save over $2.8 billion in total health care costs.

Treatment Patterns and Attrition in Metastatic Renal Cell Carcinoma: Real-Life Experience from the Turkish Oncology Group Kidney Cancer Consortium (TKCC) Database

IntroductionDespite the rapid evolution in management of metastatic renal cell carcinoma (mRCC) over the past decade, challenges remain in accessing new therapies in some parts of the world. Despite therapeutic advancements, attrition rates remain persistently high. This study aims to assess the treatment patterns and attrition rates of patients with mRCC in oncology clinics across Turkey. Patients and MethodsPatients diagnosed with mRCC between January 1, 2008, and December 31, 2022, with first-line systemic treatment data, were retrospectively evaluated using the Turkish Oncology Group Kidney Cancer Consortium (TKCC) Database. ResultsThe final analysis included a total of 1126 patients. The percentages of patients treated in the 2nd, 3rd, 4th, and 5th lines of therapy were 62.8%, 27.4%, 8.9%, and 2.1%, respectively. The drugs that were most commonly used in the groups were tyrosine kinase inhibitors (TKIs) (52.2%) and interferon (IFN)-alpha (43.3%) for the first line, TKIs (66.3%) and immunotherapy (IO) monotherapy (25.9%) for the second line, TKI (41.4%) and mTOR inhibitors (28.8%) for the third line, TKI (44.4%) and mTOR inhibitors (29%) for the fourth line, and IO monotherapy (37.5%) and TKI (25%) for the fifth line. For the first-line treatment, the primary cause of attrition was disease progression (66.4%), followed by toxicity (16.5%), death (11.2%), and patient preference (5.9%). The primary reason for attrition across all treatment lines was disease progression. Over time, the use of TKIs in first-line treatment increased, while IFN-alpha usage declined. IOs began to be utilized in earlier lines, predominantly in second-line treatment, though use of IO-based combination therapies remains limited. ConclusionThis study underscores that despite significant progress in therapeutic options, the adoption of novel agents remains slow, and attrition rates are still high. These findings indicate a disparity in systemic therapy compared to developed countries.Micro-abstract: The inclusion of patients with more favorable prognoses and performance status in clinical trials can complicate the generalizability of treatment patterns to a broader and more diverse patient group. The management of metastatic renal cell carcinoma (mRCC) in real-world settings involves complexities that usually do not occur in controlled clinical studies. This study evaluates the treatment patterns and rates of patient attrition in oncology clinics in Turkey for patients with metastatic renal cell carcinoma. The percentages of patients receiving treatment in the second, third, and fourth lines of therapy were 62.8%, 27.4%, and 8.9%, respectively. The disease's progression was the primary reason for attrition, with toxicity being the secondary cause. Over time, the use of the new agents has increased, but the progress has been slow, and there is a significant discrepancy in systemic therapy compared to developed countries.

P250 Treatment patterns in treatment naïve high-risk non-muscle invasive bladder cancer across EU4 + UK: A real-world study

P250 Treatment patterns in treatment naïve high-risk non-muscle invasive bladder cancer across EU4 + UK: A real-world study

Real-world patient characteristics and survival outcomes in patients with advanced or recurrent endometrial cancer in England: a retrospective, population-based study

ObjectiveThis study defined a retrospective cohort of patients in England with primary advanced or recurrent (A/R) endometrial cancer (EC) who may have been eligible for clinical trials evaluating immune checkpoint...

Characteristics and Treatment Patterns of Long-surviving Patients With Multiple Myeloma: Over 13 Years of Follow-up in the ConnectⓇ MM Registry

BackgroundOver the last 15 years, improvements in patient management and treatments have been associated with longer survival in patients with multiple myeloma (MM). The Connect MM Registry is a long-running, US, multicenter, prospective observational cohort study of patients with newly diagnosed MM (NDMM). We assessed the demographics, clinical characteristics, and treatment patterns of long-term survivors (LTS) enrolled in this registry. MethodsAdults with NDMM (n = 3,011) were enrolled from 250 community, academic, and government sites across the US from 2009-2016. Baseline characteristics, treatment patterns, quality of life (QoL), and overall survival (OS) were examined among LTS, defined as patients with follow-up of ≥ 8 years after enrollment. ResultsAs of February 7, 2023, 518 patients were LTS and 2,493 were non-LTS. LTS were generally younger and had better performance status at enrollment compared with non-LTS. Most (65%) LTS received stem cell transplants and few (2%) experienced disease progression within 6 months of starting first line of therapy. At data cutoff, 63% of LTS were still on treatment at their most recent visit. QoL scores and QoL questionnaire completion rates were consistently higher among LTS than non-LTS. The estimated 8-year OS rate of all patients enrolled in the registry was 40%, comparable to an observed 8-year survival of 39% from the Surveillance, Epidemiology, and End Results (SEER) database. ConclusionThis analysis provides insights on long-surviving patients with MM using real-world data and therefore presents generalizability beyond data obtained in long-term follow-up of clinical trials, underscoring the need for longitudinal follow-up through registries.

P275 A real world study (LADDER) of characteristics, treatment patterns and clinical outcomes among high-risk non-muscle invasive bladder cancer patients - preliminary results

P275 A real world study (LADDER) of characteristics, treatment patterns and clinical outcomes among high-risk non-muscle invasive bladder cancer patients - preliminary results

Outcomes Following Treatment for Progression in Patients Treated With Durvalumab Consolidation in LA-NSCLC

IntroductionPACIFIC established consolidative durvalumab for LA-NSCLC, but only about half of patients completed a year of therapy. Data on treatment patterns and outcomes after durvalumab are limited. MethodsOur analysis included patients from a US nationwide database with LA-NSCLC who received consolidative durvalumab between 2017 and 2023 and had subsequent systemic therapy, classified as PD-L1 monotherapy, PD-L1+chemotherapy, chemotherapy alone, PD-L1+CTLA4, or targeted therapy (TT). Time to next treatment (TTNT) was analyzed from durvalumab start and finish to next line of therapy initiation. Overall survival (OS) from start of postdurvalumab therapy was analyzed using Kaplan Meier methodology. ResultsOur cohort included 751 patients, median age 68 (IQR, 61-74), 53% female, 80% White, 91% ECOG 0-1, 90% smoking history, and 53% nonsquamous histology. The most common postdurvalumab treatment was chemotherapy alone in 349 (46%), followed by PD-L1+chemotherapy in 147 (20%), PD-L1 monotherapy in 114 (15%), and TT in 104 (14%).Median duration of durvalumab treatment was 5.5 months (IQR 2.3-10.6); only 9% of patients received a full year of durvalumab, and 64% started next treatment within a year of initiation. Patients treated with chemotherapy-containing regimens had shorter TTNT from durvalumab start/end, as well as shorter median OS [10.8 (5.6-18.8) months for chemotherapy and 12.9 (6.0-24.2) months for chemoimmunotherapy, versus 23.8 (8.7-34.5) months for PD-L1 monotherapy and 30.1 (9.5-NR) months for TT (P < .001)]. ConclusionPatients treated with systemic therapy after consolidative durvalumab, particularly those requiring chemotherapy-based treatment, have poor outcomes and are in need of improved treatment strategies.

Impact of social determinants of health on hepatocellular carcinoma surveillance, treatment, and health care costs.

The impact of clinical factors and social determinants of health on treatment patterns and health care costs among patients with HCC is unknown. Using 100% Medicare Fee-For-Service claims and a commercial multipayor claims database, we identified patients diagnosed with HCC from January 1, 2017, to December 31, 2020. Surveillance receipt was defined 12 months prior to HCC diagnosis, whereas treatment and health care costs were assessed post-HCC diagnosis. Multinomial logistic regression was used to assess the association between demographics, social determinants of health, and surveillance or HCC treatment. Multivariable generalized linear regression was used to identify factors associated with total health care costs. Of the 32,239 patients with HCC (mean age 68 y, 67% male, 73% White), 70% received surveillance and only half (51%) received any treatment. Curative treatment receipt was higher among those with prior surveillance (24% with CT/MRI and 18% with ultrasound vs. 9% with no surveillance). Curative treatment was independently associated with HCC surveillance and inversely associated with Black race, lower education level, and diagnosis in the year 2020 (COVID-19 year). Higher health care costs were independently associated with Black race, low English proficiency, living alone, and diagnosis in 2018-2020, and inversely associated with CT/MRI-based surveillance. Race and social determinants of health were independently associated with curative treatment receipt and health care costs. Increasing access to high-quality HCC surveillance may improve treatment receipt and reduce health disparities among patients with HCC.

P260 Real-world data on treatment patterns and guideline adherence in patients with locally advanced and metastatic urothelial bladder cancer (la/mUC) in Germany

P260 Real-world data on treatment patterns and guideline adherence in patients with locally advanced and metastatic urothelial bladder cancer (la/mUC) in Germany

Guidelines versus real-world data in metastatic bladder cancer: A population-based study on first-line chemotherapy treatment patterns

BackgroundFor patients with metastatic bladder cancer (mBC) palliative chemotherapy is one of the main treatment options. Real-world insights into outcomes are available, but a comprehensive overview of specific treatment details like number of chemotherapy cycles received and (reasons for) adjustments is lacking. MethodsA population-based study was conducted, including all patients diagnosed with mBC in the Netherlands between 2016 and 2021 who started chemotherapy as initial treatment. Data on patient, tumor, and treatment characteristics, including number of cycles, adjustments and reasons for adjustments, and survival were collected from the Netherlands Cancer Registry. Treatment patterns and outcomes were analyzed descriptively. Logistic regression analysis was used to identify factors associated with receiving the full guideline-recommended treatment (4–6 cycles). ResultsA total of 684 patients started first-line chemotherapy, mostly carboplatin-based (54%). Of these patients, 35% did not receive the full course of treatment. Among these patients who received <4 cycles, 24% died within one month of stopping treatment. Male sex and good performance status were independently associated with receiving the full course of treatment. Among patients who did receive a full course of treatment, half still had adjustments to their treatment schedule, which mainly included dose reductions due to side effects. ConclusionsAmong patients with mBC starting first-line chemotherapy, only a small majority received the recommended number of cycles, and treatment adjustments were common. This suggests that adhering to recommended treatment is challenging, emphasizing the importance of integrating insights on treatment discontinuation and modifications into the shared decision-making process and guideline development.

Patient and family burden in pediatric atopic dermatitis and its treatment pattern in Japan.

This study evaluated the level of burden in pediatric and adolescent atopic dermatitis (AD) patients in Japan, the associated burden on caregivers/families, and whether this burden varied with age. Data were drawn from the Adelphi Pediatric AD Disease Specific Programme (DSP)™, a cross-sectional survey of physicians and their patients conducted in Japan between July and December 2022. Physicians reported patient demographics, clinical characteristics, disease burden, and current/previous therapies. Patients and/or caregivers reported perceived disease severity and impact of AD, including the Children's Dermatology Life Quality Index (CDLQI) and Dermatitis Family Impact questionnaire (DFI). Overall, 55 physicians provided data for 537 AD patients aged ≤17. Mean (SD) overall scores for CDLQI, POEM, and DFI were 9.3 (6.3), 8.3 (6.8), and 11.7 (7.2), respectively. Age was associated with higher patient and/or caregiver-reported CDLQI scores, which increased by 0.543 points per year of age (P = 0.01). Patients with severe disease reported a more significant impact on quality of life factors compared with mild patients (P < 0.001). Age was associated with higher caregiver-reported burden, with DFI scores increasing by 0.325 per year (P = 0.01). Physician-reported impact on caregivers showed that age was significantly associated with increased burden on sleep, daily activities, work, and mood (P < 0.05), with disease severity associated with impact across all factors (P < 0.01). Both increasing age and disease severity were associated with the increased impact of AD on patients and their caregivers. Disease control/modification through appropriate therapeutic intervention at a younger age may relieve the burden of pediatric AD on patients and their families.

Changing Treatment and Metastatic Disease Patterns in Patients with EGFR Mutated NSCLC: An Academic Thoracic Medical Investigator’s Consortium Registry Analysis

IntroductionOsimertinib is now a standard first-line (1L) therapy for EGFR-mutated (EGFRm) advanced NSCLC. We aimed to characterize patterns of therapy and longitudinal risk of brain and liver metastasis in a cohort of EGFRm NSCLC. MethodsPatients with metastatic EGFRm NSCLC who received 1L systemic therapy at sites within the Academic Thoracic Medical Investigator’s Consortium were included; demographic and clinical data including treatment patterns were described. Analyses of overall survival, time to next treatment, and incident brain and liver metastasis were performed using the Kaplan-Meier method, Cox regression, and cumulative incidence functions on patients who started 1L therapy in 2015 or later. ResultsThe full cohort included 1132 patients and the mean age of the participants was 63.4 years; among the participants, 53% were White individuals, 68% were female individuals, and 67% were nonsmokers. Among the participants, 830 patients received 1L systemic therapy in 2015 or later. The predominant first EGFR–tyrosine kinase inhibitor was erlotinib (65%) before 2018 and osimertinib (81%) after 2018. The median time to the next treatment after the start of 1L therapy was 13.9 months overall and the longest in patients receiving 1L osimertinib (28 months). In the post-2015 cohort, the baseline prevalence of brain metastasis (BM) was 54% and among patients without baseline brain metastasis, the probability of incident BM at 12, 24, and 48 months was 8%, 22%, and 44%, respectively. Development of an on-treatment brain metastasis among patients without baseline brain metastasis was associated with a 3.2 times higher risk of death. ConclusionEven in a contemporary era with prevalent osimertinib use, the baseline and longitudinal risk of BM development was high. The ongoing risk of developing BM, together with the associated survival detriment, argues for routine surveillance of the brain through magnetic resonance imaging for patients with EGFRm NSCLC, which is not currently included in the guidelines.

Treatment patterns and outcomes of patients with stage I, high-risk endometrial cancer histologies

Treatment patterns and outcomes of patients with stage I, high-risk endometrial cancer histologies

Treatment patterns and outcomes of endometrial cancer patients with uterine-confined high-risk histology

Treatment patterns and outcomes of endometrial cancer patients with uterine-confined high-risk histology