Economic burden during remission and after relapse among older patients with newly diagnosed acute myeloid leukemia without hematopoietic stem cell transplant: A retrospective study using the SEER-Medicare database.

Acute myeloid leukemia (AML) is associated with a substantial clinical and economic burden. This study characterized the magnitude of this burden following initial treatment with standard or less intensive therapies (hypomethylating agents [HMAs]) and throughout different treatment phases post-remission. The Surveillance, Epidemiology, and End Results (SEER) cancer registry (2007-2016) linked with Medicare beneficiary claims (2007-2015) was analyzed.

Indirect treatment comparison of oral versus injectable azacitidine as maintenance therapy for acute myeloid leukemia.

Evaluate the relative efficacy of oral versus injectable azacitidine (AZA) maintenance therapy in acute myeloid leukemia (AML) after complete remission. Systematic literature review identified QUAZAR AML-001, HOVON 97 AML, UK NCRI AML16 and QoLESS-AZA-AMLE (sensitivity analysis) trials. Network meta-analysis and matching-adjusted indirect comparisons assessed survival outcomes.

Economic burden in US patients with newly diagnosed acute myeloid leukemia receiving intensive induction chemotherapy.

This retrospective, observational study assessed healthcare resource utilization (HCRU) and costs for newly diagnosed acute myeloid leukemia (AML) patients receiving intensive induction chemotherapy. Adult AML patients with inpatient hospitalization or hospital-based outpatient visit receiving intensive induction chemotherapy (CPX-351 or 7 + 3 treatments) were identified from the Premier Healthcare Database (US). All 642 patients had inpatient hospitalizations (median number = 2; median length of stay = 16 days); 22.

Real-world clinical outcomes with enasidenib in relapsed or refractory acute myeloid leukemia.

Enasidenib was approved by the Food and Drug Administration in 2017 for the treatment of patients with relapsed or refractory (RR) acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation. Given limited data in clinical practice, this study assessed real-world clinical outcomes and healthcare resource use in patients with RR AML. Physicians performed chart abstraction of patients with RR IDH2-mutated AML treated with enasidenib (between 1/2018 and 6/2019) or other first-line (1 L) RR therapy (between 1/2016 and 7/2017).

The impact of remission duration on the long-term economic burden of acute myeloid leukemia among patients without hematopoietic stem cell transplant in the United States.

Background And Aims: Acute myeloid leukemia (AML) prognosis is poor, with sustained remission occurring in <35% of young adults and <15% of older adults. This descriptive study examined the potential benefit of prolonged remission on the economic burden of AML.

Methods: Using the IBM MarketScan Commercial and Medicare Supplemental databases, we identified newly diagnosed patients with AML without hematopoietic stem cell transplantation from January 1, 2012 to December 31, 2018; AML diagnosis was the index date.

Treatment Patterns, Health Care Resource Utilization, and Cost in Patients with Myelofibrosis in the United States.

Background: This study analyses treatment patterns, health care resource utilization (HCRU), and costs in patients with myelofibrosis (MF) and a subgroup treated with ruxolitinib (RUX).

Materials And Methods: Treatment patterns, all-cause and MF-related HCRU, and costs were analyzed in adults with MF with continuous enrollment in a commercial or the Medicare Advantage health plan in the pre-index period, defined as the 12 months immediately prior to the index date (date of primary or secondary MF diagnosis), and the post-index period, defined as ≥6 months following the index date. In a subgroup analysis, outcomes were analyzed in patients treated with optimal RUX (OPT RUX, ≥30 mg) and suboptimal RUX (SUB RUX, <30 mg) in the pre-index RUX period, defined as the 3 months immediately prior to the index RUX date (first date for an RUX claim), and the post-index RUX period, defined as ≥6 months following the index RUX date.

Real-World Treatment Patterns Among Patients Initiating Small Molecule Kinase Inhibitor Therapies for Thyroid Cancer in the United States.

Introduction: Little is known about real-world use of small molecule kinase inhibitors (SMKI) for advanced thyroid cancer in the United States. This study examined prescribing patterns of SMKI agents recommended by the National Comprehensive Cancer Center (NCCN).

Methods: This retrospective study used a national health insurance database to identify patients diagnosed with thyroid cancer during 1/1/2006-6/30/2016 and with prescription claims for NCCN-recommended SMKI during 1/1/2010-5/31/2016 whose first claim date was the index date.

Cost-effectiveness analysis of lenvatinib treatment for patients with unresectable hepatocellular carcinoma (uHCC) compared with sorafenib in Japan.

Background: Lenvatinib demonstrated a treatment effect on overall survival by the statistical confirmation of non-inferiority to sorafenib for the first-line treatment of uHCC. The objective of this study was to evaluate the cost-effectiveness of lenvatinib compared with sorafenib for patients with uHCC in Japan.

Methods: A partitioned-survival model was developed to estimate the cost-effectiveness of lenvatinib versus sorafenib when treating uHCC patients over a lifetime horizon and considering total public healthcare expenditure.

Clinical benefit of treatment with eribulin mesylate for metastatic triple-negative breast cancer: Long-term outcomes of patients treated in the US community oncology setting.

Introduction: Real-world data are critical to demonstrate the consistency of evidence and external generalizability of randomized controlled trials (RCTs). This study examined characteristics and outcomes of metastatic triple-negative breast cancer (mTNBC) patients treated with eribulin mesylate at community oncology practices in the United States.

Methods: Physicians identified mTNBC patients initiating eribulin between 1 January 2011 and 1 January 2014 and abstracted data into an electronic case report form (eCRF).

Number needed to treat in indirect treatment comparison.

Aim: For dichotomous outcomes, odds ratio (OR) is one of the usual summary measures of indirect treatment comparison. A corresponding number needed to treat (NNT) estimate may facilitate understanding of the treatment effect.

Methods: We show how to estimate NNT based on OR results of a matching adjusted indirect comparison.

Measuring the Value of New Drugs: Validity and Reliability of 4 Value Assessment Frameworks in the Oncology Setting.

Background: Several organizations have developed frameworks to systematically assess the value of new drugs.

Objective: To evaluate the convergent validity and interrater reliability of 4 value frameworks to understand the extent to which these tools can facilitate value-based treatment decisions in oncology.

Methods: Eight panelists used the American Society of Clinical Oncology (ASCO), European Society for Medical Oncology (ESMO), Institute for Clinical and Economic Review (ICER), and National Comprehensive Cancer Network (NCCN) frameworks to conduct value assessments of 15 drugs for advanced lung and breast cancers and castration-refractory prostate cancer.

Validity and Reliability of Value Assessment Frameworks for New Cancer Drugs.

Background: Several organizations have developed frameworks to systematically assess the value of new drugs. These organizations include the American Society of Clinical Oncology (ASCO), the European Society for Medical Oncology (ESMO), the Institute for Clinical and Economic Review (ICER), and the National Comprehensive Cancer Network (NCCN).

Objectives: To understand the extent to which these four tools can facilitate value-based treatment decisions in oncology.

Health-care costs and utilization related to long- or short-acting antiepileptic monotherapy use.

Purpose: This study aimed to compare health-care utilization and costs in patients treated with long-acting (LA) vs. short-acting (SA) antiepileptic drug (AED) monotherapy.

Methods: We conducted a cross-sectional study of claims from the OptumInsight™ database.

Patient preferences and treatment adherence among women diagnosed with metastatic breast cancer.

Background: Given the various profiles (eg, oral vs intravenous administration, risk of hot flashes vs fatigue) of treatment options (eg, endocrine therapy, chemotherapy) for metastatic breast cancer (mBC), how patients value these attributes of their medications has implications on making treatment decisions and on adherence.

Objectives: To understand how patients trade off medication side effects with improved effectiveness and/or quality of life, to provide estimates of nonadherence among women with mBC, and to quantify the association of medication nonadherence with health outcomes.

Methods: The study was a cross-sectional, Internet-based survey of 181 women diagnosed with mBC who were recruited from cancer-specific online panels (response rate, 7%).

Effectiveness of antiepileptic drug combination therapy for partial-onset seizures based on mechanisms of action.

Importance: To our knowledge, the current study is the first to describe antiepileptic drug (AED) combination therapy patterns according to their mechanism of action (MOA) in a real-world setting and to evaluate the differences in outcomes comparing different-MOA combination therapy with same-MOA combination therapy for patients with partial-onset seizure.

Objective: To compare treatment persistence and health care use with AED combinations categorized by MOA in patients with partial-onset seizures.

Design, Setting, And Participants: Using the Truven Health MarketScan Commercial Claims Database containing 96 million covered lives from July 1, 2004, through March 31, 2011, adults with concomitant use of 2 different AEDs and a recent partial-onset seizure diagnosis were selected.

Healthcare utilization and costs in children with stable and uncontrolled epilepsy.

Objective: Epilepsy adversely affects childhood development, possibly leading to increased economic burden in pediatric populations. We compared annual healthcare utilization and costs between children (<12 years old) with stable and uncontrolled epilepsy treated with antiepileptic drugs (AEDs).

Methods: Children (<12 years old) with epilepsy (ICD-9-CM 345.

Healthcare utilization and costs in adults with stable and uncontrolled epilepsy.

Despite the availability of numerous antiepileptic drugs (AEDs), some epilepsies remain resistant to treatment. We compared utilization and costs in patients with uncontrolled epilepsy to those with stable epilepsy. Claims data (2007-2009) were used to identify adults with epilepsy requiring additional AED therapy (having uncontrolled epilepsy) and those not requiring additional AED therapy (having stable epilepsy).

Retrospective analysis of variation in heavy menstrual bleeding treatments by age and underlying cause.

Objective: To describe treatment patterns associated with heavy menstrual bleeding (HMB) in US practice.

Study Design: A retrospective claims-based analysis of organic (ICD-9 codes 218.x, 621.

Treatment patterns and healthcare system burden of managed care patients with suspected pulmonary arterial hypertension in the United States.

Objectives: To describe treatment patterns and healthcare burden among individuals with suspected pulmonary arterial hypertension (PAH), as identified through a practice guideline-based healthcare claims algorithm.

Methods: Adults with evidence of PAH from 1 January 2004 (commercial and Medicaid) or 1 July 2006 (Medicare Advantage) through 30 June 2008 were identified. Given the lack of an ICD-9 code for PAH, an algorithm was developed requiring: (1) ≥ 1 claim for PAH medication (index date); (2) ≥ 1 claim with a pulmonary hypertension diagnosis code in the 6-month pre-index period (baseline) or within 90 days post-index; (3) a right heart catheterization or pulmonary hypertension-related inpatient stay during baseline or within 90 days post-index; and (4) continuous health plan enrollment for 6 months pre-index and ≥ 6 months post-index.