Multi-Stakeholder Qualitative Interviews to Inform Measurement of Patient Reported Outcomes After CAR-T.

Toxicities after chimeric antigen receptor T cell (CAR-T) therapy are well known, yet the patient experience during and after CAR-T therapy has not been well described outside of the trial setting. We explored the patient experience after CAR-T therapy to inform the patient-reported outcomes (PRO) measurement approach for the Center for International Blood and Marrow Transplant Research (CIBMTR). We recruited (1) adult patients diagnosed with a hematologic malignancy 14 days to 6 months after receiving a commercial CAR T cell product who had agreed to be contacted by the CIBMTR, (2) caregivers of those patients, and (3) clinical experts in CAR-T therapy.

Healthcare resource use and reimbursement amount by site of care in patients with diffuse large B-cell lymphoma receiving chimeric antigen receptor T-cell (CAR-T) therapy - a retrospective cohort study using CMS 100% Medicare claims database.

Chimeric antigen receptor T-cell (CAR-T) infusion settings may impact healthcare resource use (HRU) and reimbursement amounts. Adults with diffuse large B-cell lymphoma receiving CAR-T therapy were identified from the Centers for Medicare & Medicaid Services (CMS) 100% fee-for-service Medicare database and stratified into inpatient (IP;  = 380) and outpatient (OP;  = 50) cohorts based on CAR-T infusion setting. During the first month post-infusion, OP cohort had significantly fewer IP visits, IP days, intensive care unit (ICU) stays, ICU days, and significantly more OP, emergency room (ER) visits, than IP cohort.

A Retrospective Cohort Study of Treatment Outcomes of Adult Patients With Relapsed or Refractory Follicular Lymphoma (ReCORD-FL).

This study (ReCORD-FL) sought to construct a historical control cohort to augment single-arm trials in relapsed/refractory follicular lymphoma (r/r FL). A retrospective study in 10 centers across North America and Europe was conducted. Adults with grade 1-3A FL were required to be r/r after ≥2 therapy lines including an anti-CD20 and an alkylator.

Real-world healthcare resource utilization and costs associated with tisagenlecleucel and axicabtagene ciloleucel among patients with diffuse large B-cell lymphoma: an analysis of hospital data in the United States.

This study compared the real-world healthcare resource utilization (HRU), costs, adverse events (AEs), and AE treatments associated with the chimeric antigen receptor T-cell (CAR-T) therapies, tisagenlecleucel (tisa-cel) and axicabtagene ciloleucel (axi-cel), for relapsed/refractory diffuse large B-cell lymphoma (r/r DLBCL). Adults with DLBCL who received tisa-cel or axi-cel were identified in the Premier Healthcare Database (2017-2020). Non-CAR-T costs, HRU, and AE rates during the infusion and follow-up periods were compared between the tisa-cel and axi-cel cohorts.

Comparative efficacy of tisagenlecleucel and lisocabtagene maraleucel among adults with relapsed/refractory large B-cell lymphomas: an indirect treatment comparison.

This study compared overall survival (OS), progression-free survival (PFS), complete response rate (CRR), and overall response rate (ORR) of tisagenlecleucel (tisa-cel) and lisocabtagene maraleucel (liso-cel) in relapsed or refractory large B-cell lymphomas (r/r LBCL). Using matching-adjusted indirect comparison (MAIC), individual patient-level data from JULIET (tisa-cel) were weighted to match the patient population in TRANSCEND (liso-cel). The main analysis compared infused JULIET patients ( = 106) with the TRANSCEND efficacy-evaluable set (EES) ( = 256 [infused]).

Cost-Effectiveness Analysis of Tisagenlecleucel for the Treatment of Patients With Relapsed or Refractory Diffuse Large B-Cell Lymphoma in the United States.

Purpose: To assess the cost-effectiveness and cost-effective price of tisagenlecleucel, a novel, effective chimeric antigen receptor T-cell therapy, versus salvage chemotherapy (SC) for the treatment of relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL) using a willingness-to-pay (WTP) threshold of $150,000 per quality-adjusted life year (QALY) gained from a US third-party payer's perspective.

Methods: A three-state (progression-free survival, progressive disease, and death), responder-based partitioned survival model with a lifetime horizon and 3% annual discount rate was developed. Overall survival (OS) and progression-free survival of tisagenlecleucel were estimated separately for patients with and without an overall response (OR), using data from JULIET ( Study of Efficacy and Safety of CTL019 in Adult DLBCL Patients).

Health-Related Quality of Life Improvements in Moderate to Very Severe Chronic Obstructive Pulmonary Disease Patients on Nebulized Glycopyrrolate: Evidence from the GOLDEN Studies.

Symptoms of chronic obstructive pulmonary disease (COPD) may diminish patients' health-related quality of life (HRQoL). We report effects of Longhala™ Magnair™ (glycopyrrolate) Inhalation Solution, a drug/device combination of the long-acting antimuscarinic glycopyrrolate administered using the eFlow® closed system (eFlow CS) nebulizer, on HRQoL from the Glycopyrrolate for Obstructive Lung Disease Via Electronic Nebulizer (GOLDEN) clinical studies. Data consisted of a pooled analysis of 2 phase 3, 12-week efficacy studies (GOLDEN-3 and -4) of glycopyrrolate/eFlow CS (25 or 50 mcg twice daily [BID]) versus placebo, and a 48-week, open-label safety study (GOLDEN-5) of glycopyrrolate/eFlow CS 50 mcg BID versus tiotropium 18 mcg once daily in patients with moderate to very severe COPD.

Treatment patterns for patients hospitalized with chronic obstructive pulmonary disease.

Purpose: Medication treatment patterns for chronic obstructive pulmonary disease (COPD) in inpatient settings were examined, as were the characteristics of patients treated with long-acting bronchodilators (LABDs) during hospitalization.

Methods: This retrospective study was conducted using inpatient administrative data from hospitals and medical centers nationwide. All patients discharged from the hospital from January 1, 2010, through December 31, 2012, who were at least 40 years of age, had a primary discharge diagnosis of COPD or a secondary diagnosis of COPD with a primary diagnosis of a respiratory condition, and treatment with a bronchodilator were included.

Long-term health-related quality-of-life and symptom response profiles with arformoterol in COPD: results from a 52-week trial.

Background: Symptom severity is the largest factor in determining subjective health in COPD. Symptoms (eg, chronic cough, dyspnea) are associated with decreased health-related quality of life (HRQoL). We evaluated the impact of arformoterol on HRQoL in COPD patients, measured by St George's Respiratory Questionnaire (SGRQ).

Health Status of Patients with Moderate to Severe COPD after Treatment with Nebulized Arformoterol Tartrate or Placebo for 1 Year.

Purpose: Chronic obstructive pulmonary disease (COPD) is a progressive disease that impairs both objectively measured lung function and patient-reported health status. In a randomized clinical trial of patients with moderate to severe COPD, we compared changes in health status after adding arformoterol tartrate or placebo to patients' treatment regimens.

Methods: In this multicenter, double-blind trial, patients were randomized to receive nebulized arformoterol 15 µg BID (n = 420) or matched placebo (n = 421).

Readmission Risk in Chronic Obstructive Pulmonary Disease Patients: Comparative Study of Nebulized β-Agonists.

Background: Bronchodilators are used for managing the symptoms of chronic obstructive pulmonary disease (COPD) and minimizing the risk of hospitalization and readmission. Hospital readmission is predictive of morbidity and mortality.

Objective: The study objective was to compare all-cause readmission risk in COPD patients receiving nebulized long-acting β-agonists (neb-LABAs) versus nebulized short-acting β-agonists (neb-SABA) following COPD-related hospitalization discharge.

Exacerbations, health services utilization, and costs in commercially-insured COPD patients treated with nebulized long-acting β2-agonists.

Objective: This retrospective cohort study compared exacerbations, health services utilization, and costs among chronic obstructive pulmonary disease (COPD) patients who received nebulized arformoterol or nebulized formoterol therapy.

Methods: Using PharMetrics Plus health plan claims, 417 nebulized long-acting β2-agonist (LABA) users meeting the study inclusion criteria were identified: had ≥2 fills of nebulized arformoterol or nebulized formoterol from January 1, 2009, to December 31, 2011, adhered to using their index drug ≥60% of the days during 1 year post-index, were ≥35 years old and continuously enrolled 180 days pre- and 1 year post-index, and did not use a nebulized LABA or have an asthma diagnosis during the pre-index period. Descriptive and multivariate analyses were performed.

Clinical and economic burden of breakthrough seizures.

Purpose: The purpose of this study was to measure health-care resource utilization and costs in treatment-adherent, previously seizure-free patients with epilepsy who were treated in the inpatient/emergency room (ER) setting for new-onset seizures, compared with matched controls.

Methods: The study used a retrospective case/control study design using administrative claims from the IMS PharMetrics™ database. We identified adult patients with epilepsy with 1+ ER visit/hospitalization with primary diagnosis of epilepsy between 1/1/2006 and 3/31/2011, preceded by 6months of seizure-free activity and antiepileptic drug (AED) treatment adherence (≥80% of days covered by any AED); the first observed seizure defined the "breakthrough" seizure/index event.

Hospital readmissions following initiation of nebulized arformoterol tartrate or nebulized short-acting beta-agonists among inpatients treated for COPD.

Background: Inpatient admissions for chronic obstructive pulmonary disease (COPD) represent a significant economic burden, accounting for over half of direct medical costs. Reducing 30-day readmissions could save health care resources while improving patient care. Recently, the Patient Protection and Affordable Care Act authorized reduced Medicare payments to hospitals with excess readmissions for acute myocardial infarction, heart failure, and pneumonia.

Risk of all-cause hospitalization in COPD patients initiating long-acting or short-acting beta agonist therapy.

Objective: This retrospective claims study investigated the rates of all-cause hospitalization among chronic obstructive pulmonary disease (COPD) patients initiating treatment with short-acting beta agonists (SABA) or long-acting beta agonists (LABA).

Methods: Data from the 5% national sample of Medicare enrollees for 2006-2008 were used. Patients initiating COPD therapy were identified as those with no COPD therapy for ≥ 6-months prior to initiating SABA or LABA (administered via dry-powder inhalers, metered-dose inhalers, or nebulizer) treatment.

The economic burden of pleural effusions in patients with chronic myeloid leukemia treated with tyrosine kinase inhibitors.

Objective: Tyrosine kinase inhibitors (TKI), the standard of care for patients with chronic myeloid leukemia (CML) patients, may in some cases lead to the development of pleural effusion (PE). The purpose of this study is to compare healthcare resource utilization and costs associated with PE among CML patients treated with a TKI therapy.

Methods: Two large retrospective claims databases (1999-2009) were combined to identify adult CML patients who received ≥1 TKI prescription before the index date, which was defined as 30 days before the first PE diagnosis for patients with PE and a randomly selected date for PE-free patients.

Healthcare utilization and costs in patients beginning pharmacotherapy for generalized anxiety disorder: a retrospective cohort study.

Background: Patterns of healthcare utilization and costs in patients beginning pharmacotherapy for generalized anxiety disorder (GAD) have not been well characterized.

Methods: Using a large US health insurance database, we identified all patients with evidence of GAD (ICD-9-CM diagnosis code 300.02) who initiated pharmacotherapy with medications commonly used to treat GAD (eg, selective serotonin reuptake inhibitors [SSRIs], venlafaxine, benzodiazepines) between 1/1/2003 and 12/31/2007.

Comparative efficacy of nilotinib and dasatinib in newly diagnosed chronic myeloid leukemia: a matching-adjusted indirect comparison of randomized trials.

Objective: Nilotinib and dasatinib have not been directly compared in a randomized trial for the treatment of newly diagnosed chronic myeloid leukemia in the chronic phase (CML-CP). The purpose of this study was to indirectly compare rates of major molecular response (MMR), progression-free survival (PFS) and overall survival by month 12 with nilotinib and dasatinib treatment of newly diagnosed CML-CP.

Methods: Individual patient data from a randomized trial of nilotinib vs.

Epidemiology, survival, and costs of localized gastrointestinal stromal tumors.

Purpose: The aim of this study is to examine the epidemiologic and economic burden in surgically resected localized gastrointestinal stromal tumor (GIST) patients versus age- and gender-matched controls.

Method: Two data sources were used to conduct a series of complementary analyses. First, the Surveillance, Epidemiology, and End Results (SEER) cancer registry was used to identify diagnosed GIST patients from 1993 to 2002 and determine incidence, prevalence, and 3-year survival.

Retrospective real-world comparison of medical visits, costs, and adherence between nilotinib and dasatinib in chronic myeloid leukemia.

Objective: To compare healthcare resource utilization, costs, and treatment adherence associated with dasatinib versus nilotinib treatment as second-line therapies in chronic myeloid leukemia (CML) patients.

Methods: Two large retrospective claims databases (01/1999-06/2009) were combined to identify CML patients (ICD-9 code 205.1x) who received one or more prescriptions of dasatinib or nilotinib.

Budgetary impact of treatment with adjuvant imatinib for 1 year following surgical resection of Kit-positive localized gastrointestinal stromal tumors.

Background: Imatinib mesylate, an orally administered kinase inhibitor that targets the Kit (CD117) protein, currently has 10 approved indications including treatment of chronic myelogenous leukemia and metastatic gastrointestinal stromal tumors (GIST). Treatment with adjuvant imatinib following surgical resection of localized Kit-positive GIST, the most recent FDA-approved indication (December 2008), has been shown to significantly improve recurrence-free survival (RFS) compared with surgical resection alone. Although adjuvant imatinib has proven effective in clinical trials, it is important to consider the economic impact to health plans of introducing imatinib in accordance with its new labeled indication.

Healthcare resource utilization and costs associated with non-adherence to imatinib treatment in chronic myeloid leukemia patients.

Background: Patients with chronic myeloid leukemia (CML) who do not adhere to treatment may experience suboptimal outcomes.

Objective: To examine the association between adherence with imatinib and direct healthcare costs and resource utilization in a large group of privately insured CML patients.

Patients And Methods: CML patients under age 65 were identified with ICD-9 code 205.