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

Aim: This retrospective, observational study assessed healthcare resource utilization (HCRU) and costs for newly diagnosed acute myeloid leukemia (AML) patients receiving intensive induction chemotherapy. Materials & methods: 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). Results: All 642 patients had inpatient hospitalizations (median number = 2; median length of stay = 16 days); 22.4% had an ICU admission. Median total outpatient hospital cost was US$2904 per patient, inpatient hospital cost was $83,440 per patient, and ICU cost was $16,550 per patient. Discussion: In the US hospital setting, substantial HCRU and costs associated with intensive induction chemotherapy for AML were driven by inpatient hospitalizations.

The economic burden of caregiving in epilepsy: An estimate based on a survey of US caregivers.

OBJECTIVE: The burden of caregiving for persons with epilepsy (PWEs) has not been examined previously in the United States. We assessed the clinical impact and direct and indirect economic costs for caregivers of PWEs. METHODS: An internet survey of 500 caregivers of PWEs was conducted from May to July 2015 using a combination of validated instruments and questions designed specifically for this survey. Caregivers were stratified by PWE age (adult/child) and disease severity (low: 0 vs high: 1 + seizures in the prior month). Annual self-reported direct and indirect costs were reported per caregiver and extrapolated to all US caregivers. The economic burden of caregiving for PWEs was defined as the difference between costs for caregivers and the general population. RESULTS: Caregivers reported that PWEs averaged 11.4 seizures in the prior month. Eighty percent of respondents were female and the average age was 44.3. Since becoming a caregiver, many reported anxiety (52.8%), depression (41.0%), and insomnia (30.8%). Annual mean direct medical costs for caregivers of children with low vs high seizure frequency were $4344 and $10 162, respectively. Costs for caregivers of adult PWEs were $4936 and $8518. Mean indirect costs associated with caregiving for a child with low vs high seizure frequency were $20 529 and $40 137; those for caregivers of an adult were $13 981 and $28 410. The cost estimates are higher vs the general US population; annual per-person healthcare utilization costs were $2740 and productivity loss costs were $5015. When extrapolating to the US population of PWE caregivers, annual costs exceeded $62 billion vs $14 billion for the general population, resulting in a caregiver burden of nearly $48 billion. SIGNIFICANCE: The clinical and economic burden of caregivers for PWE were substantial, and greatest for those caring for children with frequent seizures. The impact on caregivers should be considered when estimating the value of interventions that control epilepsy.

Risk of hospitalization among patients with epilepsy using long versus short half-life adjunctive antiepileptic drugs.

INTRODUCTION: While antiepileptic drugs (AEDs) remain the primary treatment for epilepsy, many patients continue to have seizures. Uncontrolled seizures may be related to AED half-life, since short half-life (SHL) AEDs require more frequent dosing compared with the simplified regimens of long half-life (LHL) AEDs. Long half-life AEDs may also improve seizure control by extending missed dose forgiveness periods. The value of LHL AEDs may be assessed as reduced healthcare utilization. The study's objective was to examine the impact of adding an LHL versus SHL adjunctive AED on the risk of hospitalizations in patients with uncontrolled epilepsy. METHODS: This was a retrospective, longitudinal cohort study using the Symphony Health Solution Patient Integrated Dataverse. Patients ≥12 years old with uncontrolled epilepsy (≥2 medical claims ≥30 days apart) were identified during a study period (8/1/2012-7/31/2017). Patients were selected if they were subsequently initiated an adjunctive AED (excluding modified release formulations), and the prescription date served as the index. Patients were stratified into two mutually exclusive cohorts based on the index AED half-life (≤20 versus >20 h). Poisson regressions with robust error variances were performed for the relative risks (RRs) of all-cause, epilepsy-related, and injury-related hospitalizations. RESULTS: A total of 4984 patients were identified (2705 in the LHL and 2279 in the SHL cohort). Compared with those in the SHL cohort, patients in the LHL cohort were significantly younger [mean (SD, years): 43.9 (18.5) versus 49.2 (17.2), p < 0.001] and were less comorbid [mean (SD) of Charlson comorbidity index: 1.2 (1.8) versus 1.8 (2.2), p < 0.001]. In the one-year postindex date, adjusting for group differences, the risks of both all-cause and epilepsy-related hospitalizations were significantly lower in the LHL cohort than in the SHL cohort [all-cause: 0.84 (95% CI: 0.76-0.93), p = 0.0006; epilepsy-related: 0.83 (0.73-0.94), p = 0.0046].Injury-related hospitalizations did not differ between LHL and SHL cohorts. CONCLUSION: In patients with uncontrolled epilepsy who were initiated on an adjunctive AED, the choice of an LHL versus SHL was associated with significantly lower risks of all-cause and epilepsy-related hospitalizations.

Observational study of clinical outcomes of eribulin mesylate in metastatic breast cancer after cyclin-dependent kinase 4/6 inhibitor therapy.

Aim: To examine the effectiveness of eribulin mesylate for metastatic breast cancer post cyclin-dependent kinase inhibitor (CDKi) 4/6 therapy. Materials & methods: US community oncologists reviewed charts of patients who had received eribulin from 3 February 2015 to 31 December 2017 after prior CDKi 4/6 therapy and detailed their clinical/treatment history, clinical outcomes (lesion measurements, progression, death) and toxicity. Results: Four patient cohorts were created according to eribulin line of therapy: second line, third line, per US label and fourth line with objective response rates/clinical benefit rates of 42.2%/58.7%, 26.1%/42.3%, 26.7%/54.1% and 17.9%/46.4%, respectively. Median progression-free survival/6-month progression-free survival (79.5% of all patients censored) by cohort was: 9.7 months/77.3%, 10.3 months/71.3%, not reached/70.4% and 4.0 months/0.0%, respectively. Overall occurrence of neutropenia = 23.5%, febrile neutropenia = 1.3%, peripheral neuropathy = 10.1% and diarrhea = 11.1%. Conclusion: Clinical outcome and adverse event rates were similar to those in clinical trials and other observational studies. Longer follow-up is required to confirm these findings.

Understanding the Association between Red Blood Cell Transfusion Utilization and Humanistic and Economic Burden in Patients with ß-Thalassemia from the Patients' Perspective.

We assessed the humanistic and economic burden of chronic red blood cell (RBC) transfusions on patients with ß-thalassemia. This cross-sectional, US-based study included adults (≥18 years) who self-reported a ß-thalassemia physician diagnosis and had received ≥1 RBC transfusion in the past 6 months. The outcomes included the Functional Assessment of Cancer Therapy-Anemia (FACT-An), Patient Health Questionnaire-9, Generalized Anxiety Disorder-7, and ad hoc questions about treatment experience, side effects, direct/indirect costs, and psychological burden. Overall, 100 patients completed the survey, of whom 70% experienced "moderate" to "extremely high" burden due to RBC transfusions, 81% reported iron overload, 42% reported compromised social lives. The mean FACT-An score was 132 (higher score indicates better outcomes; 0-188). Mean scores were 33/52 for fatigue and 20/28 for anemia symptoms in the previous 7 days. Health-related quality of life (HRQoL) temporarily improved after RBC transfusion, although patients continued to experience mild-to-severe depression and anxiety, substantial direct costs, compromised employment, and suboptimal quality of life. Over 6 months, patients dedicated a mean of 173 h to transfusion requirements and incurred out-of-pocket costs of USD 2239 for transfusions and USD 896 for additional care costs. These findings highlight the need for new treatment options to improve patient HRQoL and economic outcomes.

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). Progression-free survival (PFS) and overall survival (OS) were estimated by the Kaplan-Meier method, and adjusted risk of progression and death were estimated by multivariable Cox proportional hazard models. Among 124 patients treated with enasidenib and 76 patients treated with other 1 L RR therapy, overall response rate was higher among patients treated with enasidenib vs. other 1 L RR therapies (77% vs. 52%, p < 0.01). After a median follow-up of 9 and 6 months, median PFS was 8 months in enasidenib-treated patients and 5 months in patients receiving other 1 L RR therapy, respectively (adjusted HR=0.36, 95% CI: 0.23-0.57, p < 0.01). Median OS was 11 and 6 months in enasidenib-treated patients and patients receiving other 1 L RR therapy, respectively (adjusted HR=0.37, 95% CI: 0.22-0.60, p < 0.01). Fewer enasidenib-treated patients were hospitalized during 1 L RR therapy vs. those receiving other therapies (14% vs. 46%, p < 0.01). Results from this real-world study confirm the effectiveness of enasidenib among patients with IDH2-mutated RR AML and demonstrate that hospitalizations were significantly lower vs. other 1 L RR treatment in clinical practice.

Real-world analysis of hospitalizations in patients with epilepsy and treated with perampanel.

OBJECTIVES: (1) To evaluate risk of hospitalization following initiation of perampanel (pre- and post-analysis) and (2) to compare hospitalization rates following initiation of perampanel vs lacosamide. METHODS: Patients were identified from Symphony Health's Patient Integrated Database if they had a prescription for perampanel (July 1, 2014-June 30, 2016). Patients 4-11 years of age with any partial-onset seizure (POS) or ≥12 years of age with any POS or primary generalized tonic-clonic seizure (GTCS) (pre-post); or ≥12 years of age (perampanel vs lacosamide). The first fill of perampanel ("index date") marked the start of the analysis period. Patients had ≥1 additional fill for perampanel and ≥2 diagnoses for epilepsy or nonfebrile convulsion diagnosis during pre-index (based on ICD-9/ICD-10 codes). Patients were matched using a 1:1 propensity scoring method for the perampanel vs lacosamide analysis. Primary outcome was hospitalization during the one year following medication initiation. RESULTS: Pre- and post-perampanel: N = 1771 (mean age 34 years, 55% female). One-year all-cause hospitalization risk ratio was 0.76 (P < .05) and 36.2% with hospitalization during the pre-period vs 29.5% in the follow-up. One-year epilepsy-related inpatient hospitalization risk ratio was 0.72 (P < .05) and 30.8% with hospitalization during the pre-period vs 23.9% during follow-up. In the perampanel and lacosamide cohorts, N = 1717 per cohort after matching, most baseline demographics were balanced. A higher percentage of subjects were prescribed ≥3 anti-seizure medications for perampanel vs lacosamide (60.5% vs 57.7%, P < .001). The perampanel cohort had a 9.6% reduction in all-cause hospitalizations vs 5.8% for the lacosamide cohort (P < .05). Epilepsy-related hospitalizations decreased from the pre-index rate by 9.9% for perampanel and 8.3% for lacosamide (P < .05). Among those with baseline hospitalizations, perampanel was associated with a 59.9% reduction in all-cause hospitalizations vs 48.6% for lacosamide (P < .05), and for epilepsy-related hospitalizations, a reduction of 65.0% vs 58.9%, respectively (P < .05). SIGNIFICANCE: Perampanel was associated with a significant reduction in one-year hospitalization risk.

The effect of medication adherence on health care utilization in bipolar disorder.

A retrospective analysis of electronic prescription and medical claims representing approximately 1.4 million managed care commercial health plan members with mental health benefits was conducted. The effect of patient adherence to traditional mood-stabilizer therapy (lithium, valproate, carbamazepine, lamotrigine, or oxcarbazepine) for bipolar disorder on mental health-related hospitalization was assessed among 1,399 patients (mean age, 42.9 yr; 66.3% female) studied. Reduced adherence to traditional mood-stabilizing therapy (< 80%) in patients with bipolar disorder was associated with significantly greater risk of mental health-related, emergency room visits (odds ratio, 1.98; 95% confidence interval, 1.38-2.84) and inpatient hospitalizations (odds ratio, 1.71; 95% confidence interval, 1.27-2.32), even after adjusting for age, gender, and comorbidity.

Effect of inadequate response to treatment in patients with depression.

OBJECTIVES: To assess the effects of inadequate response to antidepressant treatment on healthcare resource utilization and on work productivity in patients diagnosed as having major depressive disorder (MDD). STUDY DESIGN: This study used data from the 2006 US National Health and Wellness Survey, a cross-sectional survey of adults 18 years and older. METHODS: Patients who self-reported a confirmed diagnosis of depression and were currently taking antidepressant medication were included in the analyses. Adequacy of antidepressant treatment response was determined from responses to the mental health domain of the 8-Item Short Form Health Survey (SF-8). Logistic regression analyses adjusted for demographics, comorbidity, and component scores on the SF-8 were used to determine the associations between inadequacy of treatment response and health outcomes. RESULTS: Of 5988 patients who met the inclusion criteria for the study, 30.9% were classified as antidepressant treatment responders, 31.2% were partial responders, and 37.9% were nonresponders. Partial response and nonresponse to treatment were associated with greater likelihood of emergency department utilization (odds ratios [ORs], 1.26 and 1.54, respectively; P <.01 for both) and hospitalization (OR, 1.23; P = .05 and OR, 1.39; P <.01, respectively). Similarly, partial response and nonresponse were associated with lower likelihood of current employment (OR, 0.83; P = .01 and OR, 0.63; P <.01, respectively) and with greater likelihood of work productivity loss among the employed (ORs, 1.42 and 1.99, respectively; P <.01 for both). CONCLUSIONS: Patients with MDD who failed to respond to antidepressant treatment as evidenced by poor self-reported mental health status used more healthcare resources, were less likely to be employed, and had more work productivity loss than those who responded to antidepressant therapy.

Assessment of psychological functioning in adolescent earthquake victims in Colombia using the MMPI-A.

The earthquake that hit Armenia, Colombia, on January 25, 1999, dramatically impacted the lives of thousands of people, including children and adolescents. This study used the Hispanic MMPI-A to clinically assess for ongoing psychopathology in a group of 59 adolescent earthquake victims. Their scores on the basic, content, and supplementary scales of the instrument were compared to those of a control group of 62 Colombian adolescents with similar socioeconomic, educational, and ethnic backgrounds. The results showed no clinically significant elevations for the earthquake victims indicating that the disaster had not resulted in diagnosable psychopathology. When compared to controls, earthquake victims showed significant elevations on D, Pt, and Sc indicating that they were mildly affected (but within the normal range) by the earthquake. The results are discussed in the context of cultural factors and the contemporary disaster and resiliency literature.