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Background: Fremanezumab, a fully humanized monoclonal antibody (mAb; IgG2Δa) that selectively targets calcitonin gene-related peptide (CGRP), is approved for the preventive treatment of migraine in adults. The efficacy and safety of fremanezumab for migraine prevention have been demonstrated in randomized, double-blind, placebo-controlled trials. Real-world effectiveness data are needed to complement clinical trial data. This study assessed the effectiveness of fremanezumab across different subgroups of adult patients with episodic migraine (EM), chronic migraine (CM), or difficult-to-treat (DTT) migraine in real-world clinical settings. Methods: This retrospective, panel-based online chart review used electronic case report forms. Patient inclusion criteria were a physician diagnosis of EM or CM; age ≥ 18 years at the time of first fremanezumab initiation; ≥ 1 dose of fremanezumab treatment; ≥ 1 follow-up visit since first initiation; and ≥ 2 measurements of monthly migraine days (MMD; with 1 within a month before or at first initiation and ≥ 1 after first initiation). Changes in MMD and monthly headache days were assessed during the follow-up period. These endpoints were evaluated in subgroups of patients by migraine type (EM/CM) and in subgroups with DTT migraine (diagnosis of medication overuse [MO], major depressive disorder [MDD], generalized anxiety disorder [GAD], or prior exposure to a different CGRP pathway–targeted mAb [CGRP mAb]). Results: Data were collected from 421 clinicians and 1003 patients. Mean (percent) reductions from baseline in MMD at Month 6 were − 7.7 (77.0%) in EM patients, − 10.1 (68.7%) in CM patients, − 10.8 (80.6%) in the MO subgroup, − 9.9 (68.3%) in the MDD subgroup, − 9.5 (66.4%) in the GAD subgroup, and − 9.0 (68.7%) in the prior CGRP mAb exposure subgroup. Improvements in MDD or GAD severity were reported by 45.5% and 45.8% of patients with comorbid MDD or GAD, respectively. Conclusions: In this real-world study, fremanezumab demonstrated effectiveness for migraine regardless of migraine type or the presence of factors contributing to DTT migraine (MO, GAD, MDD, or prior exposure to a different CGRP mAb). [ABSTRACT FROM AUTHOR]

Background: The efficacy and tolerability of fremanezumab, a fully humanized monoclonal antibody (IgG2Δa) that selectively targets calcitonin gene-related peptide (CGRP) and is approved for the preventive treatment of migraine in adults, have been demonstrated in randomized, double-blind, placebo-controlled trials. Real-world data can further support those clinical trial data and demonstrate the full clinical benefits of fremanezumab. This chart review assessed the effectiveness of fremanezumab for improving clinical outcomes in adult patients with migraine treated according to real-world clinical practice. Methods: This retrospective, panel-based, online physician chart review study used electronic case report forms with US physicians. Patient inclusion criteria were a physician diagnosis of migraine, fremanezumab treatment initiation at ≥ 18 years of age after US Food and Drug Administration approval, ≥ 1 dose of fremanezumab treatment, and ≥ 2 assessments of monthly migraine days (MMD; 1 within 30 days before treatment initiation and ≥ 1 after initiation). Changes from baseline in MMD, monthly headache days (MHD), and Migraine Disability Assessment (MIDAS) and 6-item Headache Impact Test (HIT-6) scores were assessed over 6 months. These endpoints were evaluated in the overall population and subgroups divided by dosing schedule and number of prior migraine preventive treatment failures. Results: This study included data from 421 clinicians and 1003 patients. Mean age at fremanezumab initiation was 39.7 years, and most patients were female (75.8%). In the overall population, mean baseline MMD and MHD were 12.7 and 14.0, respectively. Mean (percent) reductions from baseline in MMD and MHD, respectively, were − 4.6 (36.2%) and − 4.7 (33.6%) at Month 1, − 6.7 (52.8%) and − 6.8 (48.6%) at Month 3, and − 9.2 (72.4%) and − 9.8 (70.0%) at Month 6. Mean (percent) reductions from baseline in MIDAS and HIT-6 scores also increased over the 6-month study period, from − 6.2 (21.6%) and − 8.4 (14.0%) at Month 1 to − 18.1 (63.1%) and − 16.2 (27.0%) at Month 6, respectively. Improvements in these outcomes over 6 months were observed across all evaluated subgroups. Conclusions: This real-world study demonstrated effectiveness of fremanezumab treatment for up to 6 months, irrespective of dosing regimen or number of prior migraine preventive treatment failures, reflecting ongoing, clinically meaningful improvements in patient outcomes. [ABSTRACT FROM AUTHOR]

Background: This retrospective longitudinal study compared the effectiveness of dexamethasone+lenalidomide (Rd)-based triplet regimens containing proteasome inhibitors (PIs) ixazomib (IRd), carfilzomib (KRd), and bortezomib (VRd) or monoclonal antibodies (MABs) elotuzumab (ERd) and daratumumab (DRd) in patients with relapsed/refractory multiple myeloma (RRMM)-including those with high cytogenetic risk-primarily treated at community oncology clinics in the United States., Methods: Electronic health records of adult RRMM patients in a deidentified real-world database (01/01/2014-09/30/2020) who initiated IRd, KRd, VRd, ERd, or DRd in the second or later line of therapy (LOT) were analyzed. The index date was the date of initiation of each LOT and baseline was the 6-month pre-index period. Duration of therapy (DOT), time to next therapy (TTNT), progression-free survival (PFS), and overall survival (OS) were compared across regimens with multivariable Cox proportional hazards models., Results: Of the 1,185 patients contributing 1,332 LOTs, 985 had standard cytogenetic risk (median age, 71 years) and 180 had high risk (median age, 69 years). Compared with other regimens, DRd was associated with longer DOT overall (adjusted hazard ratio [95 % confidence interval]: 1.84 [1.42, 2.38] vs. KRd, 1.65 [1.20, 2.28] vs. ERd, 1.58 [1.23, 2.04] vs. IRd, and 1.54 [1.18, 2.00] vs. VRd), and longer TTNT and PFS. KRd was associated with shorter OS compared with DRd (1.45 [1.01, 2.08]) and VRd (1.32 [1.01, 1.73]). High-risk patients had similar outcomes with all triplet regimens., Conclusion: Although DRd improved clinical outcomes overall, Rd-based triplet regimens containing a PI or MAB are similarly effective in high-risk RRMM., Competing Interests: Declaration of competing interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: MC, MD, AH, JC, and MSD are employees of Analysis Group, a consulting firm that received research funding from Takeda Development Center Americas, Inc., to conduct this study. DC and DMS are employees of Takeda Development Center Americas, Inc. LS and SA have received consulting funds from Takeda Development Center Americas, Inc., (Copyright © 2024. Published by Elsevier Inc.)

This study compared medication use, healthcare resource utilization (HRU), and exacerbations among individuals with chronic obstructive pulmonary disease (COPD) who initiated glycopyrrolate/eFlow Closed System nebulizer 25 mcg/mL glycopyrrolate (hereafter GLY) in a real-world setting before and after treatment initiation. Retrospective claims and hospital charge master data were used to identify individuals ≥ 40 years of age diagnosed with COPD who initiated GLY between 1 April 2018 and 28 February 2019 (first prescription claim = index date). Patients were excluded if they had ≥1 asthma diagnosis in the 6-month pre-index period. The proportion of patients with COPD-related medications, other outpatient HRU, hospitalizations, and exacerbations were compared between the 6-month pre-index and 6-month follow-up periods. Among patients utilizing the service, per-person utilization rates were compared between the two periods. Among patients initiating GLY (n = 767), the mean age was 71.4 years, 56.1% were female, and the mean Charlson Comorbidity Index score was 2.0. The mean number of GLY claims per person was 3.8 during the follow-up period. Compared to the pre-index period, a lower proportion of patients had claims for COPD medications including oral corticosteroids (62.1% vs. 69.1%, p =.0001) and fixed-dose SAMA/SABA (26.1% vs. 33.0%, p <.0001) and a higher proportion of patients had claims for LABA (29.7% vs. 22.6%, p <.0001) during the follow-up period. Fewer patients had ≥1 COPD-related physician office visit (42.4% vs. 49.8%, p <.0001), radiology test (40.7% vs. 46.5%, p =.005), or moderate exacerbation (48.0% vs. 53.2%, p =.01) after initiating GLY. Among patients with linkage to inpatient data (n = 316), fewer were hospitalized (7.9% vs. 13.0%, p =.037) and hospital length of stay was shorter (1.9 vs. 3.6 days, p =.017) after initiating GLY/eFlow. Among patients initiating GLY in a real-world setting, COPD medications, hospitalizations, other HRU, and exacerbations decreased after treatment initiation compared with the 6-month pre-index period. [ABSTRACT FROM AUTHOR]

Purpose: Lenvatinib was approved for the treatment of patients with radioiodine-refractory differentiated thyroid cancer (RAI-R DTC) in the United States (US) in 2015. The main objective of the current study was to assess real-world clinical effectiveness in RAI-R DTC patients treated with first line lenvatinib monotherapy in the US., Methods: A retrospective chart review was conducted in RAI-R DTC patients who initiated lenvatinib monotherapy as first line treatment between February 2015 and September 2020. Anonymized data were abstracted by prescribing physicians from individual patient's electronic health records. Clinical outcomes included provider-reported real-world best overall response (rwBOR), real-world progression-free survival (rwPFS), and overall survival (OS). Time-to-event endpoints were assessed using Kaplan-Meier methods., Results: Our study included 308 RAI-R DTC patients treated with first line lenvatinib. At lenvatinib initiation, patients' median age was 60 years, 51.6% were female, and 26.0% of patients had an ECOG performance score of ≥2. Over the follow-up period, 32.5% of patients discontinued first line lenvatinib permanently, with others remaining on treatment. The median duration of lenvatinib therapy was 17.5 months overall. Provider-reported rwBOR (complete or partial response) to lenvatinib was 72.4%. Median rwPFS was 49.0 months. Estimated rwPFS rates at 24 and 48 months were 68.5% and 55.0%, respectively. Estimated OS rates at 24 and 72 months were 78.4% and 57.0%, respectively; median OS was not reached., Conclusion: The current study reinforces the clinical effectiveness of first line lenvatinib as standard of care in patients with RAI-R DTC in real-world clinical practice in the US., (© 2023. The Author(s).)

Background: Sir Archibald Cochrane and Sir Austin Bradford Hill requested 80 years ago answers to three questions (Can it work? Does it work? Is it worth it?) before a new intervention is implemented in day-to-day healthcare. A critical analysis of currently accepted answers identified a terminology conflict that inherits the risk of misleading interpretations and inappropriate decisions., Content: For detection of the terminology problem and the development of the solutions was possible through three recommendations (the "Ulm heritage"): (1) Albert Einstein (*1879 in Ulm/Germany) predicted, "A problem cannot be solved with the way of thinking that caused it"; (2) The "hochschule für gestaltung ulm (Academy of Design, 1953-1968)" disseminated the rules of American designers and architects e.g., "Form Follows Function"; and (3) requested to "design not only individual but universally valid solutions with socio-political relevance"., Summary: Our data confirms the power of the FFF rule, the pros and cons of value judgments, the complexity of healthcare decisions, the difference between scientific statements and political decisions, and the effects of interprofessional collaboration., Outlook: "Efficacy" offers promising "Proofs of Principle (PoP)" while "effectiveness" describes "Real-World Effectiveness (RWE)". That is why the results obtained are always more important than hypotheses., (© 2023 Walter de Gruyter GmbH, Berlin/Boston.)

Summary: This study demonstrates real-world effectiveness of teriparatide in reducing the risk of hip and other fragility fractures. Fracture incidence significantly decreased as adherence and persistence increased for any clinical, vertebral, nonvertebral, and hip fractures among patients who were observed for 2 years after teriparatide initiation. Introduction: Examine the relationship of treatment adherence and persistence to teriparatide with hip and other fractures. Methods: Truven MarketScan Research Databases, 2004 through 2014, provided teriparatide users ≥18 years old with continuous coverage 12 months pre- and 24 months post-teriparatide prescription. Adherence (medication possession ratio, MPR) groups were defined as high (≥0.80), medium (0.50 ≤ MPR < 0.80), and low (<0.50). Persistence, allowing for ≤90-day gaps between prescriptions, was defined as 1-6, 7-12, 13-18, and 19-24 months. Fracture incidence was summarized and compared by using ANOVA and logistic regression models; the effects of adherence were examined with Cox proportional hazard models with time-dependent covariates for teriparatide exposure. Results: Among 14,284 teriparatide subjects, mean age was 68.4 years, 89.8% were female, and 29.6% had a fracture in the previous year; these characteristics were similar across MPR and persistence groups. The effects of adherence and persistence to teriparatide were statistically significant ( P < .001) for all fracture types except wrist ( P ≥ .125). By logistic regression, high vs low adherence was associated with reduced risk for any (OR = 0.67; P < .001); vertebral (OR = 0.64; P < .001); nonvertebral (OR = 0.71; P < .001); and hip fractures (OR = 0.52; P < .001) and longer (19-24 months) vs shorter persistence (1-6 months) was associated with reduced risk for any (OR = 0.63, P < .001); vertebral (OR = 0.56, P < .001); nonvertebral (OR = 0.69, P < .001); and hip fractures (OR = 0.48, P < .001). Cox models revealed a significantly reduced risk between high and low adherence for any (OR = 0.69, P < .001); vertebral (OR = 0.60, P < .001); nonvertebral (OR = 0.77, P < .001); and hip fractures (OR = 0.55, P < .001). Conclusion: Fracture incidence significantly decreased as persistence and adherence to teriparatide increased. [ABSTRACT FROM AUTHOR]

Background: Ulcerative colitis (UC) treatment aims to induce response and maintain steroid-free remission. For patients with moderate to severe UC and/or nonresponse to conventional treatment, advanced therapies (immunosuppressants and biologics) are available. We assessed real-world effectiveness of advanced UC therapies., Methods: This retrospective analysis of claims data included adult patients with UC initiating immunosuppressant or biologic therapy, with 12 months' continuous enrollment pre- and postinitiation. Patients had no prescription for biologic therapy (and/or immunosuppressant if initiating immunosuppressant) in the previous 12 months. Proportion of patients remaining steroid-free (excluding 14-week tapering period), hospitalizations, and costs in the 12 months postinitiation were assessed., Results: In total, 3562 patients were included in the analysis. Most patients (83.0%) used steroids in the 12 months before initiating advanced therapy. Overall, 47.8% remained steroid-free after 12 months (excluding tapering). After adjusting for patient characteristics, remaining steroid-free was significantly more likely with infliximab (43.9%) than with adalimumab (39.4%; P < 0.05); golimumab (38.2%) and vedolizumab (41.4%) were not significantly different vs adalimumab. Overall, 12.2% of patients had a UC-related hospitalization within 12 months of initiation, with a mean (SD) total length of stay of 8.2 (8.9) days and no significant differences between biologic therapies. Mean, unadjusted, UC-related costs in the 12 months postinitiation were $42,579 and were similar between therapies., Conclusions: Patients with UC initiating advanced therapy frequently continued using steroids for at least a year. Some patients experienced extended UC-related hospitalizations, with high UC-related costs overall. This suggests an ongoing challenge in managing patients with moderate to severe UC., (© 2019 Crohn’s & Colitis Foundation. Published by Oxford University Press on behalf of Crohn’s & Colitis Foundation.)