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Real-world data regarding patients with non-small cell lung cancer (NSCLC) with EGFR exon 20 insertion (ex20ins) mutations receiving mobocertinib are limited. This study describes these patients' characteristics and outcomes. A chart review was conducted across three countries (Canada, France, and Hong Kong), abstracting data from eligible patients (NCT05207423). The inclusion criteria were: ≥ 18 years old; diagnosis of stage IIIB-IV NSCLC with EGFR ex20ins between January 1, 2017 and November 30, 2021; received mobocertinib. Data on demographics, clinical parameters, treatment patterns, mobocertinib exposure, real-world outcomes, and adverse events (AEs) were collected. Results are also reported by Asian/Non-Asian races. Overall, 105 patients were enrolled (median [IQR] age at initial diagnosis: 64.0 years [56, 71]; women: 62.9%). The most common first-line of therapy (LoT) was chemotherapy; the most common second LoT was EGFR tyrosine kinase inhibitors. Most patients received mobocertinib during LoT two and three (74.3%); the maximum dose was 160 mg/day for 67.6% of the cohort (mean [SD] daily dose: 130.6 mg [36.68]). The median real-world progression-free survival (PFS) on mobocertinib was 4.76 months (95% CI: 3.98, 6.21). The overall response rate and disease control rate were 20.0% and 48.6%, respectively (median duration of response: 8.34 months [95% CI: 3.61, 9.49]). The median overall survival (OS) was 26.28 months (95% CI: 20.21, 36.44). Asian patients had numerically superior PFS and OS compared with non-Asian patients. Regarding safety analysis, 73 patients (69.5%) experienced any AE. The most common AE was diarrhea (any grade) (52 patients; 49.5%). These data illustrate the real-world effectiveness of mobocertinib.

Background Early (often continuous) treatment of multiple myeloma (MM) with lenalidomide has become common practice, leading to an increase in lenalidomide‐refractory disease. Methods We report real‐world treatment patterns, health care resource utilization (HCRU), and outcomes for patients with lenalidomide‐refractory MM using data from Optum US Claims and Optum electronic health record (EHR) databases with index date from January 2016 to March 2022 (Claims) or December 2021 (EHR). Eligible patients had received 1–3 prior lines of therapy (LOT), including a proteasome inhibitor. Results A total of 1383 and 1597 patients with lenalidomide‐refractory disease were included from the Claims and EHR databases, respectively, with median ages of 72 and 68 years and mean Charlson Comorbidity Index scores of 4.0 and 3.1. The most common treatment combinations were daratumumab–pomalidomide–dexamethasone, daratumumab–bortezomib–dexamethasone, and pomalidomide–dexamethasone (~5% each). From LOT 2 to LOT 6, treatment attrition (patients who died or received no further treatment) was 95.2% to 95.9%. Median time to next treatment was 5.4 (Claims) and 5.9 months (EHR). Median OS was 35.2 (Claims) and 41.2 months (EHR). HCRU was consistent across LOT. Conclusions Patients with lenalidomide‐refractory MM who received 1–3 prior LOT had poor outcomes and moved quickly through available therapies, demonstrating an unmet need to improve outcomes in this difficult‐to‐treat patient population.

Background Head and neck squamous cell carcinoma (HNSCC) is a globally significant disease with poor survival outcomes. Immune checkpoint inhibitors (ICIs) such as pembrolizumab and nivolumab have improved treatment paradigms, yet their real‐world efficacy and the factors influencing treatment outcomes remain underexplored. Aims This study aimed to evaluate real‐world outcomes of pembrolizumab and nivolumab therapy in patients with HNSCC and to identify clinical and laboratory factors associated with overall survival (OS), progression‐free survival (PFS), and objective response rate (ORR). Methods and Results We conducted a retrospective analysis of 45 HNSCC patients treated with pembrolizumab or nivolumab at the University Medical Center Mannheim. Patient‐specific factors, including tumor characteristics, PD‐L1 expression, and laboratory parameters, were assessed using Kaplan–Meier estimation, log‐rank tests, and multivariate regression models. The median OS and PFS were 10.4 months and 7.4 months, respectively, with an ORR of 22%. A tumor proportion score (TPS) ≥ 50% and absence of smoking or alcohol abuse significantly improved ORR, while female sex, high neutrophil‐to‐lymphocyte ratio (NLR), and elevated leukocyte counts were associated with inferior OS and PFS. Real‐world outcomes largely aligned with the pivotal trials Keynote‐048 and CheckMate 141. Conclusion This study underscores the predictive value of TPS and patient lifestyle factors in ICI treatment for HNSCC. The findings also highlight sex‐specific differences, as well as NLR and leukocyte count as potential prognostic factors. Larger, more diverse cohorts are needed to confirm these results and refine patient selection strategies.

Background The efficacy and safety of second catheter ablation (CA) sessions for idiopathic premature ventricular contractions (PVCs) from the same origin as the initial session remain unclear. Methods and Results We analyzed 138 patients (median age 55 [43–68] years; 74 males [53.6%]) who underwent second CA sessions for idiopathic PVCs from the same origin category, using the Japanese Catheter Ablation Registry data collected between August 2017 and December 2020. PVC origins included 77 from the right ventricular outflow tract (RVOT) (55.8%), 8 from other right ventricular (RV) origins (5.8%), 23 from the left ventricular outflow tract (LVOT) (16.7%), and 30 from other left ventricular (LV) origins (21.7%). Acute success was achieved in 114 patients (82.6%), with significant variations by origins (RVOT: 83.1%, other RV: 75.0%, LVOT: 76.9%, other LV: 90.0%; p < 0.01). In‐hospital recurrence despite acute success occurred in 6 patients (5.3%), most frequently in other RV and LVOT sites. Success at discharge cases included more females (57.4% vs. 40.0%; p = 0.04) and were treated at higher‐volume centers (median 304 vs. 234 cases/year; p < 0.01). No significant predictors of success at discharge were identified in univariable or multivariable analyses. One patient (0.7%) experienced a cardiac tamponade. Conclusion Second CA sessions for idiopathic PVCs are generally safe and effective. However, additional efforts are needed to improve the LVOT and other RV origin efficacy. Trial Registration: The J‐AB registry has been registered in both the UMIN Clinical Trial Registry (UMIN000028288) and ClinicalTrials.gov (NCT03729232). The second session of idiopathic premature ventricular contraction ablation, targeting the same origin, demonstrated comparable efficacy, particularly for RVOT and other LV sites. However, outcomes for LVOT and other RV sites remained suboptimal. Overall, repeat procedures were safely performed in Japan.

Aims The TOPCAT trial showed no benefit for spironolactone in heart failure patients with preserved ejection fraction (HFpEF). Post‐hoc, spironolactone helped participants from the Americas, but not Eastern Europe. Determining which patients with HFpEF could respond like TOPCAT's responders should help guide their care. We aimed to develop a TOPCAT Trial Score (TS) as a composite metric to identify such patients. Methods and results From the TOPCAT individual‐level data, we calculated a TS of age, body mass index, systolic blood pressure, heart rate, creatinine, potassium, glucose, left ventricular ejection fraction, and left atrial volume for each participant as a weighted distance in multidimensional space from the theoretical perfectly average Americas participant. Logistic regression was used to measure TS and spironolactone as predictors of TOPCAT's primary outcome. The relationship between TS and the H2FPEF score was also determined in TOPCAT and a registry cohort of real‐world patients in the U.S. with HFpEF. A bimodal distribution of TS separated American (n = 1766) and Eastern European (n = 1,677) participants. Those with lower TS showed no significant response to spironolactone. Spironolactone's benefit rose with rising TS [βinteraction = ‐0.28 (P < 0.01)]. Significantly more American participants had benefit from spironolactone based on higher TS (> 1.14), in addition to higher likelihood of HFpEF based on higher H2FPEF scores (≥3). The cohort of real‐world patients with HFpEF had even higher TS than American TOPCAT participants. Conclusions Patients with HFpEF can be quantified by the TS to capture the likelihood of benefit from spironolactone.

Abstract Context Glucagon-like peptide-1 receptor agonists (GLP-1RAs) and sodium-glucose cotransporter-2 inhibitors (SGLT2is) are used off-label in the management of type 1 diabetes mellitus (T1DM) in real-world practice as adjuvant therapies to insulin. There are few real-world data regarding efficacy and safety of this practice. Objective This work aimed to determine the efficacy and safety of GLP-1RAs and sodium-glucose SGLT2is in the management of T1DM in real-world practice. Methods A retrospective chart review was performed of all instances of GLP-1RA and/or SGLT2i use greater than 90 days in adult patients with T1DM at a single academic center. We report the clinical and safety outcomes over the duration of use. Results We identified 104 patients with T1DM who ever used a GLP-1RA (76 patients) or SGLT2i (39 patients) for more than 90 days. After 1 year of therapy, GLP-1RA users had statistically significant reductions in weight (90.5 kg to 85.4 kg; P < .001), glycated hemoglobin A1c (HbA1c) (7.7% to 7.3%; P = .007), and total daily dose of insulin (61.8 units to 41.9 units; P < .001). SGLT2i users had statistically significant reductions in HbA1c (7.9% to 7.3%; P < .001) and basal insulin (31.3 units to 25.6 units; P = .003). GLP-1RA users compared to SGLT2i users had greater reduction in weight (P = .027) while HbA1c reduction was comparable between the groups. Over a mean total duration of use of 29.5 months/patient for both groups, more SGLT2i users experienced diabetic ketoacidosis (DKA) (12.8% vs 3.9%). Therapy was discontinued because of adverse events 26.9% of the time for GLP-1RA users vs 27.7% for SGLT2i users. Conclusion GLP-1RA and SGLT2i use in T1DM is associated with clinically relevant benefits. DKA remains a clinical concern with SGLT2i use, requiring careful patient selection and monitoring, with the risk to benefit ratio of treatment evaluated at an individual level.

Abstract Background Treatment of metastatic renal cell carcinoma (mRCC) is rapidly evolving with new combination therapies demonstrating improved response rates and survival. There are no head-to-head prospective trials comparing an immunotherapy doublet with an immunotherapy/tyrosine-kinase inhibitor-based combination. We compare real-world outcomes in patients treated with axitinib/pembrolizumab (axi/pembro) or ipilimumab/nivolumab (ipi/nivo). The primary endpoints were overall-survival (OS) and real-world progression-free survival (rwPFS). Patients and Methods We used a de-identified database to select patients diagnosed with clear cell mRCC and treated with front-line axi/pembro or ipi/nivo from 2018 to 2022. Analyses are adjusted using propensity score-based inverse probability of treatment weighting, balancing age, gender, insurance, race, IMDC risk, and nephrectomy status. We compared survival by treatment groups using weighted and unweighted Kaplan–Meier curves with log-rank tests and weighted Cox proportional hazards regressions. Results We included a total of 1506 patients with mRCC who received frontline axi/pembro (n = 547) or ipi/nivo (n = 959). Median follow-up time was 20.0 months (range: 0.2-47.6). Baseline demographics were similar between the 2 cohorts. Adjusted median OS for the full population was 28.9 months for axi/pembro and was 24.3 months for ipi/nivo (P = .09). Twenty-four-month survival was 53.8% for axi/pembro treated patients and 50.2% for ipi/nivo treated patients. rwPFS was 10.6 months for axi/pembro treated patients and 6.9 months for ipi/nivo treated patients. Treatment with axi/pembro conferred improved survival in the IMDC favorable risk strata, with no significant difference in survival observed within the full cohort. Conclusions In this retrospective, real-world study of patients treated with front-line combination therapy, patients with IMDC favorable risk disease had better survival when treated with axi/pembro compared to ipi/nivo. However, survival for the entire population and the 24-month median overall survival were not statistically different between treatment groups. Longer follow-up is necessary to discern any emerging significant differences. This article reports real-world outcomes of patients with metastatic renal cell carcinoma treated with combination therapies (either axitinib/pembrolizumab or ipilimumab/nivolumab).]

Abstract Background Lutetium-177 (177Lu)–PSMA-617 is a beta-emitting radioligand approved for treatment of metastatic castration-resistant prostate cancer (mCRPC), despite the underrepresentation of Black patients in pivotal trials. We analyzed outcomes of 177Lu-PSMA-617 in a racially diverse cohort. Methods Retrospective analysis of patients with mCRPC treated with 177Lu-PSMA-617 was conducted at the Emory Winship Cancer Institute. Primary outcomes assessed were progression-free survival (PFS), overall survival (OS), and prostate-specific antigen (PSA) reduction ≥50% (PSA50). Cox proportional hazard models were used for univariate and multivariate OS and PFS, and logistic regression was used for PSA50 analysis. Results Among 163 patients treated with 177Lu–PSMA-617, 97 (59.5%) self-identified as White or other racial groups and 66 (40.5%) self-identified as Black. On univariate analysis, Black patients had comparable OS, PFS, and PSA50 responses to non-Black patients, with a trend toward improved outcomes (OS HR: 0.82, P = .446; PFS HR 0.92, P = .655; PSA50 OR = 1.79, P = .088). Multivariate analysis demonstrated a non-significant prolonged PFS and reduction in mortality risk for Black patients (PFS HR: 0.65, P = .106; OS: HR 0.59, HR P-value .081). The odds of a PSA50 response were 2.45 times higher for Black patients (OR = 2.45, P = .027). Conclusions In our racially diverse cohort of patients with mCRPC, Black patients had PFS and OS comparable to non-Black patients, although wide confidence intervals limit definitive conclusions. Black patients had a significantly greater odds of achieving a PSA50 response. Our findings suggest efficacy of 177Lu-PSMA-617 among Black patients in real-world settings and underscore the importance of improved representation in prospective studies.

Background The treatment landscape for advanced non-small cell lung cancer (aNSCLC) has evolved rapidly since immuno-oncology (IO) therapies were introduced. This study used recent data to assess real-world treatment patterns and clinical outcomes in aNSCLC in the United Kingdom. Methods Electronic prescribing records of treatment-naive patients starting first-line (1 L) treatment for aNSCLC between June 2016 and March 2018 (follow-up until December 2018) in the United Kingdom were assessed retrospectively. Patient characteristics and treatment patterns were analyzed descriptively. Outcomes assessed included overall survival (OS), time to treatment discontinuation, time to next treatment, and real-world tumor response. Results In all, 1003 patients were evaluated (median age, 68 years [range, 28–93 years]; 53.9% male). Use of 1 L IO monotherapy (0–25.9%) and targeted therapy (11.8–15.9%) increased during the study period, but chemotherapy remained the most common 1 L treatment at all time points (88.2–58.2%). Median OS was 9.5 months (95% CI, 8.8–10.7 months) for all patients, 8.1 months (95% CI, 7.4–8.9 months) with chemotherapy, 14.0 months (95% CI, 10.7–20.6 months) with IO monotherapy, and 20.2 months (95% CI, 16.0–30.5 months) with targeted therapy. In the 28.6% of patients who received second-line treatment, IO monotherapy was the most common drug class (used in 51.6%). Conclusions Although use of 1 L IO monotherapy for aNSCLC increased in the United Kingdom during the study period, most patients received 1 L chemotherapy. An OS benefit for first-line IO monotherapy vs chemotherapy was observed but was numerically smaller than that reported in clinical trials. Targeted therapy was associated with the longest OS, highlighting the need for improved treatment options for tumors lacking targetable mutations.

Study comparing decitabine and cedazuridine taken by mouth with other hypomethylating agents given through the veins or under the skin Why was the study done? A new type of chemotherapy drug called a hypomethylating agent (HMA) that is taken by mouth (oral) has been approved for patients with myelodysplastic syndromes (MDS). This drug is decitabine and cedazuridine together (DEC-C). Previous HMA treatments had to be given through a patient’s veins (IV) or under the skin (SC). Data collected from patients being treated in the real-world setting, rather than a clinical trial setting, are needed to see whether oral DEC-C can be considered in place of the IV/SC HMAs. What did the researchers do? The study team looked at electronic medical records in ConcertAI’s RWD360 ® dataset of adult patients with MDS. The researchers grouped patients by their first HMA treatment received after MDS diagnosis: oral DEC-C or IV/SC HMA. Then the researchers matched the patients based on characteristics such as age, race, and when they received the initial HMA treatment to make sure the groups were similar. What did the researchers find? Patients who received oral DEC-C had a similar time to death (median 22.7 months versus 19.5 months) and time to leukemia development (median 16.1 months versus 14.3 months) compared with patients who received IV/SC HMAs. However, patients who received oral DEC-C had a longer time until another treatment was needed compared to patients who received IV/SC HMAs (9.3 months versus 7.8 months). What do the findings mean? The results of this study support the consideration of oral DEC-C as a treatment option in place of IV/SC HMAs for patients with MDS.