Survival outcomes for patients with multiple myeloma in France: A retrospective cohort study using the Système National des Données de Santé national healthcare database.

OBJECTIVE: Evaluate the overall survival (OS) of patients with multiple myeloma (MM) at different treatment stages in France. METHODS: This retrospective observational cohort study used data from the French National Health Insurance database to study patients with MM (diagnosis 2013-2019). Patient outcomes included OS (all-cause mortality), time-to-next treatment (TTNT), and duration of therapy (DoT) from initial diagnosis, the start of different lines of therapy (LOTs), triple-class exposure (TCE), and subsequent treatment following TCE. The Kaplan-Meier method analyzed "time-to-event" data. RESULTS: From diagnosis, death rates increased from 1% at 1 month to 24% at 2 years; median OS was 63.8 months (N = 14 309). Median OS from the start of LOTs declined from 61.0 months (LOT1) to 14.8 months (LOT4). Median OS from TCE start was 14.7 months. There was a large variation in TTNT within LOTs (e.g., LOT1: bortezomib + lenalidomide: TTNT = 26.4 months, OS = 61.7 months; lenalidomide: TTNT = 20.0 months, OS = 39.6 months); DoT was similar for LOT1 and LOT2, then progressively declined at LOT4. Patients with stem cell transplant, younger age, and less comorbidity had better survival outcomes. CONCLUSIONS: Patients with MM face a poor prognosis after relapse to multiple LOTs and TCE, demonstrating a worsening of survival outcomes. Access to novel therapies may improve outcomes.

Societal preferences for adjuvant melanoma health states: UK and Australia.

BACKGROUND: No studies have measured preference-based utility weights for specific toxicities and outcomes associated with approved and investigational adjuvant treatments for patients with resected high-risk melanoma. METHODS: A cross-sectional study was conducted in the United Kingdom and Australia to obtain utilities for 14 adjuvant melanoma health states. One-on-one interviews were conducted using standard gamble; utility weights range from 0.0, dead, to 1.0, full health. Supplemental risk questions also were asked. RESULTS: Among 155 participants (52% male; mean age, 46 years) "adjuvant treatment no toxicities" (0.89) was most preferred, followed by "induction treatment" (0.88), and "no treatment" (0.86). Participants least preferred "cancer recurrence" (0.62); the utility for "cancer recurrence and 10-year survival with treatment" was 0.70. Disutilities for grade 2 toxicities ranged from -0.06 for fatigue to -0.13 for hypophysitis. The mean maximum acceptable risk of a life-threatening event ranged from 30% for a 6% increase in the chance of remaining cancer free over 3 years to 40% for an 18% increase; Australian respondents were willing to take higher risks. CONCLUSION: Reproducible health utilities for adjuvant melanoma health states were obtained from the general population in two countries. These utilities can be incorporated into treatment-specific cost-effectiveness evaluations.

Treatment patterns and overall survival of patients with double-class and triple-class refractory multiple myeloma: a US electronic health record database study.

Patients with relapsed/refractory multiple myeloma (RRMM) resistant to multiple drug classes remain a high unmet need population. This longitudinal retrospective cohort study assessed real-world treatment patterns and outcomes in adults with RRMM. Patients who had three or more prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent (double-exposed) were further categorized as refractory to a PI and an immunomodulatory agent (double-class refractory, n = 381) or additionally to an anti-CD38 monoclonal antibody (triple-class refractory, n = 173). Treatment options are limited for patients with double-class or triple-class refractory disease. Retreatment is a part of standard of care. Bortezomib and lenalidomide had the highest retreatment rates among double-class and triple-class refractory patients. Survival outcomes remain poor among RRMM patients with median overall survival (OS) of 22.3 and 11.6 months for double-class refractory and triple-class refractory patients, respectively. This study highlights the need for novel efficacious therapies in this heavily pretreated RRMM population.

Daclatasvir combined with asunaprevir is a cost-effective and cost-saving treatment for hepatitis C infection in China.

Aim: To evaluate the cost-effectiveness of the novel all-oral direct-acting antiviral regimen daclatasvir + asunaprevir (DUAL), versus interferon-based regimens for the treatment of chronic hepatitis C virus genotype 1b infection. Methods: Inputs for a lifetime Markov model were sourced from clinical trials and published literature. Outputs include disease management costs, life expectancy, quality-adjusted life-years and cost-effectiveness. Sensitivity analyses assessed the drivers of cost-effectiveness and sustained virologic response thresholds at which DUAL is cost-saving. Results: DUAL was associated with discounted incremental quality-adjusted life-years of 1.29-3.85 and incremental life-years of 0.85-2.59 per patient, with discounted lifetime cost savings of USD$1415-8525. Associated sustained virologic response rates could fall to 45.1-84.8%, while remaining dominant. Conclusion: Treatment with DUAL provides significant clinical benefit, while accruing lower lifetime costs.

Resource utilization and costs of blood management services associated with knee and hip surgeries in US hospitals.

This article assesses the use and costs of blood transfusion during knee and hip surgery through a retrospective observational study of 92,223 discharged inpatients who had undergone knee or hip surgery from July 1, 2003, through June 30, 2004; a sample of US hospitals that participated in the Perspective Comparative Database (Premier Inc., Charlotte, NC) was used. Descriptive and multivariate analyses were performed to determine the use and costs of allogeneic blood transfusion (ABT). The average cost of ABT per user ranged from $387 (SD=$952) for red blood cells to $6585 (SD=$11,162) for cryoprecipitate. Utilization rates in the sample were as follows: antifibrinolytics, 0.14%; topical sealants, 3.24%; volume expanders, 3.89%; erythropoietin agents, 5.08%; and hypotensive anesthesia, 22.28%. Patients who were given volume expanders ($133.73, SD=$23.00, P<.01) or erythropoietin ($177.72, SD=$34.61, P<.01) had higher costs associated with ABT than did those who did not use volume expanders or erythropoietin. Patients who received hypotensive anesthesia (odds ratio [OR]=1.96; 95% confidence interval [CI], 1.87-2.06), a volume expander (OR=1.71; 95% CI, 1.57- 1.85), a topical sealant (OR=1.61; 95% CI, 1.45-1.79), or an erythropoietic agent (OR=2.30; 95% CI, 2.06-2.57) had a greater likelihood of ABT. Investigators concluded that most transfusion reduction techniques are underused, or they do not reduce the burden of ABT associated with knee or hip surgery.

Treatment patterns and clinical outcomes in patients with chronic immune thrombocytopenia (ITP) switched to eltrombopag or romiplostim.

This observational study aimed to assess real-world treatment patterns and clinical outcomes for patients with chronic immune thrombocytopenia (ITP) currently being treated with eltrombopag or romiplostim after switching from corticosteroids, rituximab, or the alternate thrombopoietin receptor agonist (TPO-RA). The study examined the rationale for switching to TPO-RA therapy using aided responses. Dosing patterns were also analyzed before and after switching. Treatment outcomes were assessed through platelet counts at multiple time points including treatment initiation and after switching at the last office visit. A total of 280 patients were enrolled whose active therapy for ITP was replaced with either eltrombopag (n = 130) or romiplostim (n = 150). Efficacy-related issues (desired platelet count not achieved and/or lack of response to prior therapy) were the main drivers for therapy switching among all patients (54 % for eltrombopag vs. 57 % for romiplostim). Platelet counts at the last office visit showed improvement compared with counts at the initiation of either eltrombopag or romiplostim treatment. No significant differences were noted when comparing clinical outcomes between the eltrombopag and romiplostim treatment cohorts. Our results suggest that switching to the other TPO-RA may be beneficial if there is inadequate response to treatment with the initial TPO-RA.

Trends in the prevalence of thrombocytopenia among individuals infected with hepatitis C virus in the United States, 1999-2008.

BACKGROUND: Thrombocytopenia is associated with the natural history of hepatitis C virus (HCV) infection and anti-viral therapy. Recent, national estimates of the clinical burden of thrombocytopenia among HCV-infected individuals in the United States are unavailable. Bi-yearly data from the 1999-2000 to 2007-2008 National Health and Nutrition Examination Surveys (NHANES) were used to examine the prevalence of thrombocytopenia among HCV-infected individuals in the United States. RESULTS: Among 467 HCV-infected individuals in the survey (weighted population = 3,597,039), mean weighted age was 46.7 years (standard deviation = 15.5) and 61.7% were male. Overall, 7.6% met the study definition of TCP at the 150 × 10(9)/L threshold; 4.5%, 2.0%, and 0.8% had platelet counts below 125, 100, and 75 × 10(9)/L, respectively. The 2-year weighted prevalences of thrombocytopenia (150 × 10(9)/L threshold) from 1999-2008 were 4.9%, 8.6%, 6.5%, 4.1%, and 12.9%. The unadjusted biannual time trend (odds ratio) was 1.16 (95% confidence interval = 0.82-1.64). In the two adjusted models, the odds by time ranged from 1.24-1.40, depending on whether the model included demographic or laboratory variables or both, but did not reach statistical significance. Age was positively and significantly related to thrombocytopenia status. CONCLUSIONS: As the HCV-infected population ages, the prevalence of thrombocytopenia is expected to rise. This study provides limited evidence of such an effect at the national level.

Treatment patterns and metastasectomy among mCRC patients receiving chemotherapy and biologics.

BACKGROUND: Several long-standing chemotherapy regimens are available to treat metastatic colorectal cancer (mCRC) including: oxaliplatin plus 5-fluorouracil (5-FU) and leucovorin (FOLFOX); and irinotecan plus 5-FU and leucovorin (FOLFIRI). More recently, new biologic therapies were approved for use in mCRC. OBJECTIVES: This study examined treatment patterns and trends in metastasectomy among newly diagnosed mCRC patients after the introduction of capecitabine (CAP) in 2001 and the biologic therapies in 2004. METHODS: Using a large, US-based administrative medical claims database of a national commercially insured population, patients with newly diagnosed mCRC were identified from 2001 to 2005. At least 6 months of patient history prior to mCRC diagnosis were required to confirm patients were newly diagnosed. Patients were followed from initial mCRC diagnosis to death, disenrollment, or 12/31/2006. Chemotherapy and biologic treatments and rates of metastasectomy over time were analyzed. RESULTS: A total of 3781 mCRC patients met the study criteria. The average time from mCRC diagnosis to initiation of systemic treatment decreased from 134.4 days (SD 261.2) to 61.7 days (SD 89.3) in 2001-2005 (p < 0.001). The most common first-line regimens were FOLFIRI (40%), 5-FU/LV (31%), and capecitabine (21%) in 2001, and FOLFOX plus bevacizumab (22%), FOLFOX alone (15%), 5-FU/LV (15%), and capecitabine (15%) in 2005. Among patients with ≥1 year of follow-up, the use of biologics increased from 37.3% in 2004 to 52.0% in 2005 (p < 0.001). The percentage of patients who underwent resection after systemic treatment increased from 2.9% to 5.6% in 2001-2005 (p = 0.169). CONCLUSIONS: Over time the standard of care chemotherapy for 1st-line mCRC has changed from FOLFIRI to FOLFOX, and the use of biologics has become common. The percentage of patients who underwent resection after systemic treatment almost doubled during the study period.

Systemic Therapy for Metastatic Colorectal Cancer: Patterns of Chemotherapy and Biologic Therapy Use in US Medical Oncology Practice.

PURPOSE: With the emergence of new chemotherapies and biologic agents in the treatment of metastatic colorectal cancer (mCRC), the optimal combination and sequencing of these therapies are yet to be determined. This study examined the extent and pattern of chemotherapy and biologic therapy use by line of treatment. Biologic continuation and dose escalation were also examined. METHODS: This study used an integrated electronic medical record database of 91 US oncology practices. Records were analyzed for 1,655 adult patients with mCRC who were treated from January 1, 2004 to January 31, 2008 with systemic therapy and could be observed for ≥ 3 months beyond their diagnosis of metastatic disease. Combination and sequence of individual drugs and regimens were examined. RESULTS: For first-line therapy, the most common chemotherapy backbone was infused fluorouracil, leucovorin, and oxaliplatin (FOLFOX; 40.5% of patients), and the most common treatment regimen was FOLFOX plus bevacizumab (26.2%). For second-line therapy, fluorouracil, leucovorin, and irinotecan (FOLFIRI) was the most common chemotherapy backbone (25.7%), and FOLFIRI plus bevacizumab was the most common treatment regimen (18.3%). Across the study period, 68.6%, 22%, and 7% of patients received bevacizumab, cetuximab, and panitumumab, respectively. Among 412 patients receiving bevacizumab-containing regimens as first-line therapy who then received second-line therapy, 58% continued receiving bevacizumab, with dose escalation observed in 44%. CONCLUSION: The most commonly used chemotherapy backbones for mCRC treatment were first-line FOLFOX and second-line FOLFIRI. Bevacizumab was the most frequently administered biologic therapy. Continuation and dose escalation with bevacizumab were frequently observed across lines of therapy.