Time To Next Treatment Research Articles

Real-world outcomes following ibrutinib dose reduction in patients with chronic lymphocytic leukemia/small lymphocytic lymphoma

This study used real-world data from three separate United States (US) databases to evaluate dosing patterns and time to next treatment (TTNT) following the first-incident adverse event (AE) in patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) treated with first-line ibrutinib with and without dose reduction (DR). Median TTNT or death in patients with and without a DR following an AE in each database was as follows: Optum Clinformatics Data Mart (CDM): 59.5 and 30.6 months; ConcertAI: 27.1 and 18.0 months; and Medicare Fee-for-Service (FFS): 49.8 and 22.0 months, respectively. Median TTNT or death in patients with cardiac AEs, with and without a DR, was: Optum CDM: 44.4 and 22.9 months; ConcertAI: 29.9 and 18.3 months; and Medicare FFS: 49.6 and 14.0 months, respectively. Ibrutinib DR was associated with fewer outpatient visits and lower CLL/SLL-related medical costs. These findings suggest that utilizing ibrutinib DR may effectively manage tolerability without compromising clinical efficacy.

Thrombocytopenia With High C-reactive Protein in Myeloma Patients Treated With Proteasome Inhibitor and/or Immunomodulatory Drugs.

Background/Aim: Thrombocytopenia is a poor prognostic factor in patients with myeloma; however, the factors associated with thrombocytopenia have not been extensively discussed. This study aimed to investigate the clinical significance of thrombocytopenia, defined as 130×103/μl or less, in patients with newly diagnosed multiple myeloma (NDMM) treated with proteasome inhibitors and/or immunomodulatory drugs. This is a retrospective review of medical records of myeloma patients treated between 2000 and 2021. A total of 241 patients were included in this study, with a median age of 72 years. Overall survival (OS) and time to next treatment (TTNT) were assessed using Kaplan-Meier analysis and Cox regression analysis. Prognostic factors were evaluated by univariate and multivariate analyses. The incidence of thrombocytopenia was 17.8%. In the median follow-up period of 46.6 months, OS and TTNT in the thrombocytopenia group were significantly shorter than those in the non-thrombocytopenia group using multivariate analysis (p<0.001 and p<0.001). C-reactive protein (CRP) level was not associated with thrombocytopenia, and high CRP predicted short OS and TTNT independently from thrombocytopenia. When the low (neither thrombocytopenia nor high CRP), intermediate (either thrombocytopenia or high CRP), and high (thrombocytopenia and high CRP) risk groups were defined, the OS and TTNT among these groups showed significant differences; the hazard ratios for survival in the high and intermediate risk groups were 7.022 and 2.598, and for TTNT, they were 4.216 and 1.887, respectively, compared to the low-risk group. Thrombocytopenia was associated with the activity of NDMM and predicted prognosis in NDMM. When combined with high CRP levels, thrombocytopenia serves as a new indicator of poor prognosis in these patients.

Time to Next Treatment Following Sub-Ablative Progression Directed Radiation Therapy for Oligoprogressive Non-Small-Cell Lung Cancer

We aimed to evaluate whether progression-directed radiation therapy (PDRT) can prolong the initiation of a subsequent systemic therapy regimen in a cohort of patients with oligoprogressive NSCLC. A retrospective analysis was conducted on NSCLC patients who underwent PDRT for extracranial oligoprogressive NSCLC, defined as limited (up to five) progressing lesions following initial complete, partial, or stable response to systemic therapy according to REC1ST 1.1 and/or PERCIST 1.0 criteria. Cox proportional hazard regressions were performed to identify factors influencing time to next treatment (TTNT), which was considered the primary endpoint. Forty patients were analyzed. First, second, and ≥3 lines of systemic therapy were administered in 22 (58.2%), 14 (27.2%), and 4 (14.6%) cases, respectively. The median total dose was 36 Gy (range: 12–60) in five fractions (1–10), with a median biological effective dose for tumor control (BED10) of 52 Gy (26.4–151.2). After a median follow-up of 11 months (2–50), PDRT delayed further systemic therapy in 32 (80.0%) treatments. Median TTNT was not reached at 8 months (1–47) with a one-year Kaplan–Meier estimate of 81.4% (95% CI: 75.0% to 87.8%). No &gt;grade 3 adverse event was observed. On multivariate analysis, patients with ≥3 lines of systemic therapy and/or with larger CTV volumes did not benefit from PDRT. Despite the use of sub-ablative doses, our findings show that PDRT represents an effective, safe, and viable option for oligoprogressive NSCLC. Patients irradiated early during their systemic treatment course, with a low volume of disease and nonmetastatic oligoprogression, could derive substantial benefits from PDRT.

Outcomes following treatment for progression in patients treated with durvalumab consolidation in LA-NSCLC

Introduction: PACIFIC 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. Methods: Our analysis included patients from a US nationwide database with LA-NSCLC who received consolidative durvalumab between 2017-2023 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 post-durvalumab therapy was analyzed using Kaplan Meier methodology. Results: Our cohort included 751 patients, median age 68 (IQR 61-74), 53% female, 80% White, 91% ECOG 0-1, 90% smoking history, and 53% non-squamous histology. The most common post-durvalumab 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<0.001)]. Conclusion: Patients treated with systemic therapy after consolidative durvalumab, particularly those requiring chemotherapy-based treatment, have poor outcomes and are in need of improved treatment strategies.

Changing Treatment and Metastatic Disease Patterns in Patients with EGFR Mutated Non-Small Cell Lung Cancer: An ATOMIC Registry Analysis

IntroductionOsimertinib is now a standard 1L therapy for EGFR-mutated advanced non-small cell lung cancer (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 (ATOMIC) were included; demographic and clinical data including treatment patterns were described. Analyses of overall survival (OS), time to next treatment (TTNT), and incident brain and liver metastasis were performed on patients who started 1L therapy in ≥2015 using Kaplan-Meier method, Cox regression, and cumulative incidence functions. ResultsThe full cohort included 1,132 patients; mean age 63.4 years, 53% White, 68% female, 67% nonsmoker; 830 patients received 1L systemic therapy in ≥2015. The predominant first EGFR-TKI was erlotinib (65%) prior to 2018 and osimertinib (81%) after 2018. The median TTNT after start of 1L therapy was 13.9 months overall, and 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, 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 brain MRIs for patients with EGFRm NSCLC, which are not currently included in guidelines.

Real-World Evaluation of Treatment Patterns, Healthcare Costs, and Healthcare Resource Utilization Among Patients with Non-small Cell Lung Cancer in the US Receiving Sotorasib.

Sotorasib was the first drug approved for adults with Kirsten rat sarcoma G12C-mutated locally advanced/metastatic non-small cell lung cancer (NSCLC) who received prior systemic therapy in the US. This study aimed to provide initial real-world evidence on patient characteristics, treatment patterns, healthcare resource utilization (HCRU), and healthcare costs (HCC) associated with sotorasib in US clinical practice. A retrospective observational study was conducted using the Optum Clinformatics® Data Mart US claims database spanning January 2016 to March 2023. The study population included adults with a diagnosis of lung cancer (diagnosis (Dx) date), claims for sotorasib on/post-Dx date (index date), Continuous enrollment for medical/pharmacy benefits from 180 days pre-Dx date to≥30 days post-index date was required. Patients receiving treatments for small-cell lung cancer (SCLC) pre-index were excluded. Outcomes were analyzed for patients receiving sotorasib as second or subsequent line (2L+) treatment and included adherence [proportion of days covered (PDC)], treatment duration, time to next treatment (TTNT), HCRU, and HCC during sotorasib treatment. Among 169 patients with lung cancer that met all inclusion criteria, 140 patients received sotorasib as 2L+ treatment (mean age: 71 years; 67.1% females). Mean PDC for sotorasib was 94.9%. Kaplan-Meier median treatment duration was 4.3 months. Median TTNT in patients with subsequent treatment (n=31) was 6.8 months. During sotorasib treatment, patients had a mean 3.87 outpatient, 0.09 inpatient, and 0.11 emergency visits per month. Mean monthly HCC during sotorasib treatment were US$23,063 versus $25,541 during the 180-day pre-index period. Patients in the US receiving sotorasib as 2L+ therapy for NSCLC in real-world clinical practice showed high adherence, TTNT comparable to progression-free survival observed in clinical trials, and HCC similar to those immediately prior to treatment demonstrating real-world benefits with no additional impact on healthcare resources with sotorasib.

Adjuvant Use of Pembrolizumab for Stage III Melanoma in a Real-World Setting in Europe.

Although data on patients treated with pembrolizumab are available from clinical trials and single-country real-world reports, to our knowledge no multi-country real-world studies have investigated the use of pembrolizumab as an adjuvant treatment for stage III melanoma. We used the European Melanoma Registry (EUMelaReg), a disease entity-based registry specific for melanoma, to examine treatment and outcomes for adult patients with stage III melanoma with lymph node involvement who had complete resection and received adjuvant treatment with pembrolizumab. The primary objectives were to describe the demographic and clinical characteristics of the included patients as well as time on adjuvant pembrolizumab treatment (TOT), real-world recurrence-free survival (RFS) and distant metastasis-free survival (DMFS) from adjuvant pembrolizumab initiation. Secondary objectives were time to next treatment (TTNT) after adjuvant use of pembrolizumab, next-line therapy for stage III and unresectable stage IV melanoma and overall survival (OS) from initiation of pembrolizumab. Patients were stratified according to age, sex, BRAF status, number of positive lymph nodes and disease substage. Median TOT was 11.1 (9.2-11.5) months, median RFS was 29.6 [18.7-not reached (NR)] months and median DMFS was 32.4 (22.7-NR) months. TTNT was 29.9 (22.2-NR) months, while median OS was not reached. The results of this study offer insights into the real-world use of pembrolizumab as an adjuvant therapy for melanoma in Europe.

Impact of chromosomal aberrations detected by chromosome banding analysis in symptomatic Waldenström's macroglobulinemia.

The clinicopathologic features and prognostic impact of MYD88 L265P (MYDL265P) and CXCR4 mutations (CXCR4Mut) have been well reported, although little is known regarding the impact of chromosomal aberrations (CA) detected by chromosome banding analysis (CBA) in symptomatic Waldenström's macroglobulinemia (sWM). Thus, we investigated the clinicopathologic features and prognostic impact in sWM with CAs identified by CBA. We retrospectively analyzed the clinicopathologic results and genomic alterations by droplet digital PCR, fluorescence in situ hybridization (FISH), and CBA using the G-banding method in bone marrow samples from sWM patients collected between April 2010 and March 2024 at our institute. The relationship between CAs and clinicopathologic features was evaluated, as well as the time to next treatment (TTNT). Thirty-five patients were enrolled. The median age was 71 years, and the median hemoglobin level was 10.1g/dL. The median serum IgM and M-protein levels were 3,009mg/dL and 2.95g/dL, respectively. MYDL265P was found in 30/35 patients (85.7%), whereas CXCR4Mut was found in 3/35 patients (8.6%). Deletion 6q identified by FISH in 5/18 patients (28%), and CAs using CBA in 9/34 patients (26%), including 4/34 (12%) complex karyotypes. sWM with CAs had more anemia (p = 0.04) and hypoalbuminemia (p = 0.007), in addition to higher serum M-protein and IgM levels (p = 0.03). With a median follow-up of 73 months, the median TTNT in patients with and without CAs was 27 and 68 months, respectively. CAs with CBA may be associated with clinical aggressiveness and shorter TTNT in sWM.

Real-world clinical outcomes among patients with non-squamous advanced non-small cell lung cancer (aNSCLC) treated with docetaxel-based regimens post initial standard of care (SOC) in the United States.

392 Background: Immunotherapy (IO) or targeted therapy used separately or in combination with platinum-based chemotherapy (PBC) are SOC among treatment naïve aNSCLC patients. After progression on SOC, patients have limited treatment options. Previous US-based real-world studies have shown docetaxel ± ramucirumab as the most used line of treatment (LOT) post-SOC. This study sought to assess clinical outcomes associated with docetaxel+ramucirumab (DTX-R) or docetaxel monotherapy (DTX) received post SOC among patients with non-squamous aNSCLC independent of genomic alteration status. Methods: This retrospective study, using electronic medical record data from the ConcertAI Patient360 NSCLC data product (01/2015–09/2022), included adult patients (≥18 years) with stage IIIB-IV non-squamous aNSCLC who received docetaxel-based regimens (DTX-R or DTX) as subsequent LOT (index LOT) after discontinuation of the prior qualifying SOC LOT (IO + PBC or targeted therapy + PBC). Kaplan-Meier analysis was used to estimate median time to discontinuation (TTD), time to next treatment (TTNT), real-world progression-free survival (rwPFS), and real-world overall survival (rwOS) on docetaxel-based index regimens. Results: Out of 656 non-squamous aNSCLC patients which discontinued prior SOC therapy, 165 (25.2%) received a docetaxel-based regimen (n = 96 [DTX-R]; 69 [DTX]) which was the most commonly received index regimen. Other patients received a variety of other treatments, most commonly, pembrolizumab monotherapy (11.4%), pemetrexed + pembrolizumab (8.4%), pemetrexed monotherapy (4.4%), carboplatin + pemetrexed (4.1%), and osimertinib (3.8%). Among patients with index DTX-R and DTX, median age was 66 and 65 years; 36.5% and 60.9% were female; 74.0% and 63.8% were White; 86.5% and 75.4% received care in community setting; 15.1% and 31.8% had brain metastasis; 63.6% and 65.2% had ECOG 0-1; 1 and 2 median prior LOT; and median follow-up from index was 10 and 11 months, respectively. Median TTD, TTNT, rwPFS, and rwOS for docetaxel-based regimens are reported in the table. Conclusions: Docetaxel ± ramucirumab is the most used regimen in a fragmented treatment landscape post SOC therapy among patients with non-squamous aNSCLC. Observed rwOS, rwPFS, and treatment durations on docetaxel-based regimens in this real-world setting suggested the limited clinical benefit associated with these therapies. Overall, there is a need for more effective treatment options post SOC therapies. Real-world outcomes for DTX-R and DTX treated patients post SOC. Outcome, months (median, 95% CI) Docetaxel+Ramucirumab Docetaxel Monotherapy rwOS 6.37 (4.4, 9.3) 4.73 (2.3, 7.8) rwPFS 3.88 (2.8, 6.0) 3.22 (2.0, 5.7) TTD 3.06 (2.4, 3.5) 1.71 (1.7, 2.4) TTNT 3.45 (2.9, 4.5) 3.02 (1.9, 4.6)

Characteristics, efficacy, and prognosis analysis of newly diagnosed marginal zone lymphoma.

To retrospectively analyze the characteristics of newly diagnosed marginal zone lymphoma (MZL) patients, evaluate the efficacy of different treatment regimens, and explore prognostic factors in the era of immunotherapy. We reviewed the clinical data of newly diagnosed MZL patients treated at the Department of Hematology, The First Hospital of Jilin University, from October 2013 to October 2023. Survival differences between groups were analyzed using the log-rank test, and prognostic factors were identified. A total of 265 newly diagnosed MZL patients were included, with a median age of 59 years (range 22-90). The most common pathological type was mucosa-associated lymphoid tissue (MALT) lymphoma, accounting for 66.0% of cases. Among the 147 MZL patients included in the efficacy analysis, the median follow-up was 43.4 months. Both the median progression-free survival (PFS) and overall survival (OS) were not reached. The 5-year PFS and OS rates were 76.0% and 86.6%, respectively. Patients who achieved complete response (CR) after induction therapy had significantly better PFS (P=0.0045), OS (P<0.001), and time to next treatment (TTNT) (P=0.0045) compared to those who did not achieve CR. A subgroup analysis was conducted on 51 MZL patients with high tumor burden who received ≥4 cycles of treatment. It was found that the CR rate (CRR) in patients receiving obinutuzumab (G) ± chemotherapy was significantly higher than in those receiving rituximab (R) ± chemotherapy (93.8% vs. 48.6%, P=0.002). Multivariate analysis revealed that disease progression or death within 24 months of initial treatment (POD24) was an independent risk factor affecting OS (P<0.001). Patients who experienced POD24 had a median survival of only 19.7 months, with a 3-year OS rate of just 37.6%, whereas those without POD24 had a 3-year OS rate of 97.3%. MZL is predominantly seen in middle-aged and elderly patients and is a specific indolent B-cell lymphoma, with MALT lymphoma being the most common subtype. Achieving CR after induction therapy significantly prolongs survival in MZL patients. Compared to R ± chemotherapy, G ± chemotherapy achieves a higher CRR in high tumor burden MZL patients. In the era of immunotherapy, POD24 is an independent prognostic factor for MZL.

Real-World First-Line Maintenance Niraparib Monotherapy Use Following Chemotherapy Plus Bevacizumab: The SW1TCH Study.

Clinical trials have demonstrated prolonged survival associated with niraparib first-line maintenance (1LM) therapy, compared with placebo, for patients with ovarian cancer (OC). However, data are limited on real-world 1LM niraparib monotherapy use, particularly as switch 1LM, following first-line (1L) combination chemotherapy plus bevacizumab. This real-world study aimed to describe patient demographics, clinical characteristics, and clinical outcomes of patients with OC receiving 1LM niraparib monotherapy following 1L combination chemotherapy plus bevacizumab. This retrospective observational study used data from a US-based nationwide database of deidentified, electronic health record-derived data. Patients diagnosed with OC during the study period (1 January 2011-30 November 2022, inclusive) were eligible if they received 1L chemotherapy plus bevacizumab treatment followed by 1LM niraparib monotherapy, initiated between 1 January 2017 (inclusive) and 2 September 2022. Patients were followed from index date (initiation of niraparib 1LM) until the first occurrence of death, end of follow-up, or end of study. Clinical outcomes were time to treatment discontinuation (TTD) and time to next treatment (TTNT). Kaplan-Meier curves were used to estimate TTD, TTNT, and 95% confidence intervals (CIs). Among 93 patients selected, median age at index was 67years (interquartile range [IQR] 60-72 years). Most patients had BRCA wild-type/homologous recombination (HR)-proficient or BRCA wild-type/HR unknown disease (75.3%). In all, 18 (19.4%) patients had HR-deficient disease. Five (5.4%) patients had unknown test results for both BRCA and HR deficiency status. Median follow-up time was 16.3months (IQR 8.7-25.4months), and median time from end of 1L therapy to 1LM initiation was 35.0days (IQR 25.0-53.9days). Median TTD was 9.3months (95% CI 6.1-11.3months). Median TTNT was 12.9months (95% CI 11.5-19.0months). This real-world study provided insights into switch maintenance with 1LM niraparib monotherapy, which may be a viable treatment option for patients with advanced OC.

CLL-479 6-Year Time to Next Treatment (TTNT) Extrapolation Curve for GLOW Study: First-Line Ibrutinib Plus Venetoclax (I+V) Offers Long Treatment-Free Period for Older Adult/Unfit Patients With Chronic Lymphocytic Leukemia (CLL)

CLL-479 6-Year Time to Next Treatment (TTNT) Extrapolation Curve for GLOW Study: First-Line Ibrutinib Plus Venetoclax (I+V) Offers Long Treatment-Free Period for Older Adult/Unfit Patients With Chronic Lymphocytic Leukemia (CLL)

Real-World Evaluation of Treatment Pattern, Time to Next Treatment (TTNT), Health Care Resource Utilization (HCRU), and Cost of Care in Follicular Lymphoma (FL)

Real-World Evaluation of Treatment Pattern, Time to Next Treatment (TTNT), Health Care Resource Utilization (HCRU), and Cost of Care in Follicular Lymphoma (FL)

IBCL-516 Real-World Evaluation of Treatment Pattern, Time to Next Treatment (TTNT) Health Care Resource Utilization (HCRU), and Cost of Care in Follicular Lymphoma (FL)

IBCL-516 Real-World Evaluation of Treatment Pattern, Time to Next Treatment (TTNT) Health Care Resource Utilization (HCRU), and Cost of Care in Follicular Lymphoma (FL)

6-Year Time to Next Treatment (TTNT) Extrapolation Curve for GLOW Study: First-Line Ibrutinib Plus Venetoclax (I+V) Offers Long Treatment-Free Period for Older Adult/Unfit Patients With Chronic Lymphocytic Leukemia (CLL)

6-Year Time to Next Treatment (TTNT) Extrapolation Curve for GLOW Study: First-Line Ibrutinib Plus Venetoclax (I+V) Offers Long Treatment-Free Period for Older Adult/Unfit Patients With Chronic Lymphocytic Leukemia (CLL)

Patient Characteristics Associated with Time to Next Treatment in Patients with Ovarian Cancer Treated with Niraparib: The PRED1CT Real-World Study.

Niraparib first-line maintenance (1LM) therapy has demonstrated clinical benefit for patients with ovarian cancer (OC) in clinical trial and real-world settings, but data on factors associated with real-world patient outcomes remain limited. This analysis identified patient characteristics associated with time to next treatment (TTNT), a proxy for real-world progression-free survival, in patients with OC treated with 1LM niraparib monotherapy. This retrospective observational study used a USA nationwide electronic health record-derived deidentified database and included adult patients diagnosed with OC who initiated 1LM niraparib monotherapy after first-line platinum-based chemotherapy. Patients were followed until the earliest occurrence of last clinical activity, death, or end of study period. TTNT was measured from 1LM niraparib initiation to the start of second-line treatment or death. Cox proportional hazards models assessed univariable and multivariable associations between baseline characteristics and TTNT. Of 7872 patients diagnosed with OC, 526 met the eligibility criteria and were included in this analysis. Median (IQR) duration of follow-up was 14.1 (7.4-23.6) months. In univariable analyses, age, BRCA/homologous recombination deficiency (HRD) status, socioeconomic status, stage at initial diagnosis, cytoreductive surgery type, and residual disease status were significantly associated with observed TTNT and were introduced into the multivariable model with other clinically relevant variables. In the multivariable analysis, BRCA/HRD status, cytoreductive surgery type, and residual disease status were significantly associated with observed TTNT after covariate adjustment. Conversely, age, Eastern Cooperative Oncology Group performance status, disease stage, niraparib starting dose status, and first-line bevacizumab use were not associated with observed TTNT. This real-world, retrospective, observational analysis offers valuable insights on prognostic factors associated with TTNT in patients with OC treated with 1LM niraparib monotherapy after first-line platinum-based chemotherapy. Future studies are needed to examine how additional patient characteristics associated with clinical outcomes may guide treatment decisions and improve outcomes.

Talquetamab Versus Real-World Physician's Choice Treatment: Comparative Effectiveness in Patients With Triple-Class Exposed Relapsed/Refractory Multiple Myeloma

BackgroundTalquetamab, a first-in-class GPRC5D×CD3 bispecific antibody, is approved for treatment of triple-class exposed (TCE) patients with relapsed/refractory multiple myeloma (RRMM) based on MonumenTAL-1 results. We evaluated the comparative effectiveness of talquetamab in MonumenTAL-1 versus real-world physician's choice (RW) treatment. Materials and MethodsAn external control arm for MonumenTAL-1 was created from eligible patients in the Flatiron Health database who satisfied key MonumenTAL-1 eligibility criteria (n = 629 with 1169 eligible lines of therapy). Patient-level data from MonumenTAL-1 were included for patients who received subcutaneous talquetamab 0.4 mg/kg QW (n = 143) and 0.8 mg/kg Q2W (n = 145). After adjusting for baseline covariate imbalances, comparative effectiveness was assessed for progression-free survival (PFS), time to next treatment (TTNT), and overall survival (OS). ResultsBaseline covariates were comparable across cohorts after adjustment. Talquetamab 0.4 mg/kg QW and 0.8 mg/kg Q2W cohorts, respectively, showed significant improvement in PFS (HR, 0.55 [95% CI, 0.44-0.69; P<0.0001; median, 7.5 vs. 4.0 months] and 0.40 [95% CI, 0.31-0.53; P<0.0001; median, 14.2 vs. 4.0 months]), TTNT (HR, 0.59 [95% CI, 0.47-0.74; P<0.0001; median, 9.1 vs. 5.1 months] and 0.45 [95% CI, 0.35-0.59; P<0.0001; median, 13.3 vs. 5.1 months]), and OS (HR, 0.56 [95% CI, 0.40-0.78; P=0.0007; median, NR vs. 16.5 months] and 0.48 [95% CI, 0.33-0.70; P=0.0002; median NR vs. 15.9 months]) versus RW treatment. ConclusionBoth talquetamab schedules demonstrated superior effectiveness over RW treatment for all outcomes assessed. These data suggest talquetamab as an effective novel immunotherapy option in patients with TCE RRMM. MicroabstractTalquetamab has been evaluated in the single-arm MonumenTAL-1 trial in patients with relapsed/refractory multiple myeloma. Effectiveness of talquetamab was assessed against outcomes of real-world treatments using indirect treatment comparison methodology. Talquetamab led to better outcomes compared to standard treatments, suggesting that novel immunotherapies such as talquetamab may be a superior treatment option versus previous standard regimens.

The EMMY longitudinal, cohort study: real-world data to describe multiple myeloma management and outcomes as more therapeutic options emerge

The therapeutic management of patients with multiple myeloma (MM) is complex. Despite substantial advances, MM remains incurable, and management involves cycles of treatment response, disease relapse, and further therapy. Currently, evidence to support the therapeutic decision is limited. Thus, the EMMY longitudinal, real-world study was designed to annually assess therapeutic management of MM in France to provide evidence to support physicians. During an annual prespecified 3-month recruitment period, eligible patients will be identified from their medical records. Adults aged ≥18 years diagnosed with symptomatic MM and requiring systemic treatment will be eligible. The primary objective, the evolution of MM therapeutic management, will be described, as well as the impact on the following outcomes: time-to-next treatment (TTNT), progression-free survival (PFS), and overall survival (OS). The study plans to recruit 5000 patients over 6 years: 700 to 900 patients annually. EMMY is a unique opportunity to collect real-world data to describe the evolving MM therapeutic landscape and record outcomes in France. These data will provide annual snapshots of various aspects of MM management. This knowledge will provide physicians with real-life, evidence-based data for therapeutic decision-making and ultimately improve treatment for MM patients.

A retrospective observational study of ibrutinib in chronic lymphocytic leukaemia in a real-life setting in France using the national claims database (OSIRIS)

BackgroundIbrutinib is a Bruton’s tyrosine kinase inhibitor indicated for the first-line treatment and relapse of chronic lymphocytic leukaemia (CLL), Waldenström’s macroglobulinemia (WM) and mantle cell lymphoma (MCL). This study aimed to describe the characteristics of CLL patients treated with ibrutinib and its effectiveness, safety, and treatment pattern in real life.MethodsAll patients covered by the general health scheme (approximately 80% of the French population) with a first ibrutinib dispensation from August 1, 2017 (date of reimbursement in France) to December 31, 2020, were identified in the French National Health Insurance database (SNDS). An algorithm was developed to identify the disease (CLL, MCL or WM) for which ibrutinib was prescribed. This article focused on CLL patients. The time to next treatment (TTNT) was plotted using Kaplan‒Meier curves.ResultsDuring this period, 6,083 patients initiated ibrutinib, among whom 2,771 (45.6%) patients had CLL (mean age of 74 years; 61% of men). At ibrutinib initiation, 46.6% of patients had a cardiovascular comorbidity. Most patients (91.7%) were not hospitalized during the exposure period for one of the cardiovascular or bleeding events studied. Hospitalizations were more frequent in patients with a cardiovascular comorbidity (5.9% versus 11.0%, p-value < 0.0001) and aged over 70 (5.9% versus 9.4%, p-value < 0.0001). The median TTNT was not reached.ConclusionThis is one of the largest cohorts of ibrutinib-treated patients in the world. The profile of CLL patients treated with ibrutinib was in accordance with the marketing authorization and reimbursement. This study confirmed effectiveness and safety data.

Real-world evaluation of treatment pattern, time to next treatment (TTNT), healthcare resource utilization (HCRU), and cost of care in follicular lymphoma (FL).

e19044 Background: FL often becomes relapsed or refractory (R/R) to treatment. This study aimed to analyze TTNT, HCRU, and cost of care in FL. Methods: A retrospective study was conducted using Optum Clinformatics Data Mart to identify patients ≥18 years with ≥1 diagnosis for FL initiating 1L, 2L, 3L, or 4L treatment from 1/2019 to 02/2023. Patients were required to be continuously enrolled for 30 days pre- and post-index date, defined as the date of treatment initiation. Patients were categorized into mutually exclusive cohorts based on treatment regimen. TTNT was measured for patients who had a subsequent treatment within the study period. Total cost of care included both plan- and patient-paid claims, adjusted for inflation (2023 USD). HCRU included outpatient visits and inpatient admissions. Costs and HCRU were reported per patient per month over the treatment duration. Results: There were 4,525 patients initiating 1L treatment and 1,053, 304, and 97 patients initiating 2L, 3L, and 4L treatment, respectively. Median follow-up was 487 days (1L), 430 days (2L), 357 days (3L), and 276 days (4L). The mean age at the index was similar across cohorts (range: 69.6 [1L] to 71.9 [4L]). Rituximab monotherapy (R-mono) was the most utilized treatment in 1L and 2L, followed by bendamustine + rituximab (BR) and R-CHOP (Table). Lenalidomide + rituximab (R2) was the most utilized regimen in 3L and 4L. Obinutuzumab monotherapy (O-mono) use increased by line of therapy. The median TTNT across all regimens decreased by line of therapy (Table). BR and bendamustine + obinutuzumab (BO) had longer TTNT in 1L, while BR and R2 had longer TTNT in 3L. Inpatient admissions and outpatient visits increased with the line of therapy. Mean (SD) outpatient visits were 5.3 (3.2) in 1L, 5.8 (3.7) in 2L, 6.3 (4.0) in 3L, and 6.1 (3.3) in 4L, while inpatient admissions ranged from 0.1 (0.3) in 1L to 0.3 (0.4) in 4L. The mean total cost of care ranged from $40,538-$74,466, increasing by line of therapy. The mean total cost of care was consistently lowest in R-mono ($31,704-$36,197), while CAR-T had the highest total cost ($501,493-$522,378). R-mono had the lowest while R-CHOP had the highest mean outpatient visits across lines of therapy. Conclusions: Real-world TTNT decreased while costs and HCRU increased with each subsequent line of treatment, indicating a high disease burden in FL patients, particularly for those with R/R FL.These findings suggest the need for better treatment options for patients with FL, especially in 3L and 4L. [Table: see text]