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Aims: Treatment of acute myeloid leukemia (AML) requires significant healthcare resource utilization (HRU), including lengthy hospitalizations. In a phase 3 study (NCT01696084), CPX-351 (Vyxeos) sh...

A Phase-3 study of defibrotide compared with historical controls demonstrated a 23% improvement in 100-day survival post-hematopoietic stem cell transplantation (HSCT) among patients with veno-occlusive disease with multi-organ dysfunction (VOD with MOD).To estimate the budget impact and cost-effectiveness of introducing defibrotide to a transplant center.The authors developed a budget impact model from the perspective of a bone-marrow transplant center. It was estimated that 2.3% of adults and 4.2% of children would develop VOD with MOD following HSCT based on a retrospective hospital database analysis and the effect that treating patients with defibrotide would have on costs for adult and pediatric centers was estimated. A cost-utility analysis (CUA) was also developed to capture the long-term cost-effectiveness of defibrotide. Projected life expectancies in the two groups were estimated based on trial data, transplant registry data, studies of long-term survival among HSCT patients, and US population life-tables.There was an estimated 3% increase ($330,706) per year in total adult transplantation center costs associated with adopting defibrotide, and a1% increase ($106,385) for pediatric transplant centers, assuming 100 transplants per year. In the CUA, the lifetime increase in cost per patient was $106,928, life expectancy increased by 3.74 years, and quality-adjusted life-years (QALYs) increased by 2.24. The incremental cost-effectiveness ratio (ICER) was $47,736 per QALY gained; 88% probability defibrotide was cost-effective at a $100,000/QALY threshold.The budget impact of defibrotide for a transplant center is relatively modest compared to the overall cost of transplantation. Defibrotide provides an important survival advantage for VOD with MOD patients, and the life years gained lead to defibrotide being highly cost-effective.

Sinusoidal obstruction syndrome (SOS) is a life-threatening complication of hematopoietic stem cell transplantation (HSCT) associated with significant morbidity and mortality. Healthcare utilization, costs, and mortality were assessed in HSCT patients diagnosed with SOS, with and without multi-organ dysfunction (MOD).This retrospective observational study identified real-world patients undergoing HSCT between January 1, 2009 and May 31, 2014 using the Premier Healthcare Database. In absence of a formal ICD-9-CM diagnostic code, SOS patients were identified using a pre-specified definition adapted from Baltimore and Seattle criteria and clinical practice. Severe SOS (SOS/MOD) and non-severe SOS (SOS/no-MOD) were classified according to clinical evidence for MOD in the database.Of the 5,418 patients with a discharge diagnosis of HSCT, 291 had SOS, with 134 categorized as SOS/MOD and 157 as SOS/no-MOD. The remaining 5,127 patients had HSCT without SOS. Overall SOS incidence was 5.4%, with 46% having evidence of MOD. Distribution of age, gender, and race were similar between the SOS cohorts and non-SOS patients. After controlling for hospital profile and admission characteristics, demographics, and clinical characteristics, the adjusted mean LOS was 31.0 days in SOS/MOD compared to 23.9 days in the non-SOS cohort (medians = 26.9 days vs 20.8 days, p .001). The adjusted mean cost of SOS/MOD patients was $140,653, which was $41,702 higher than the non-SOS cohort (medians = $105,749 vs $74,395, p .001). An almost 6-fold increased odds of inpatient mortality was associated with SOS/MOD compared to the non-SOS cohort (odds ratio = 5.88; 95% CI = 3.45-10.33).Limitations of retrospective observational studies apply, since the study was not randomized. Definition for SOS was based on ICD-9 diagnosis codes from a hospital administrative database and reliant on completeness and accuracy of coding.Analysis of real-world data shows that SOS/MOD is associated with significant increases in healthcare utilization, costs, and inpatient mortality.

To investigate adherence to dornase alfa therapy among commercially-insured patients with cystic fibrosis (CF) and to examine the impact of adherence on health and economic outcomes.This retrospective cohort analysis included CF patients with ≥1 dornase alfa (Pulmozyme) pharmacy claim between 1 October 2006 and 30 September 2008 and with continuous enrollment in the health insurance plan at least 1 year before and 1 year after their index dornase alfa claim. Adherence was measured with the medication possession ratio (MPR). Multivariate models were used to estimate the relationship between adherence and exacerbations, utilization, and cost.Nine hundred and seven patients met the inclusion criteria. The mean age was 19.5 years (SD = 11.5) and 49.1% were female. Overall MPR was 0.59 and by age was 0.66 for patients of 5-12 years, 0.57 for 13-20 years, 0.54 for 21-30 years, and 0.56 for patients ≥31 years. Adherence was better in fall and winter than in spring and summer. There was no statistically significant difference in the proportion of patients with inpatient respiratory exacerbations across groups with low (0.5), moderate (0.5-0.79), and high (≥0.8) adherence (24.5%, 22.3%, and 19.1%, respectively, p = 0.250). There was a trend toward higher total charges in more-adherent patients (mean $58,612 in the least-adherent group and mean $69,427 in the most adherent group, p = 0.107). In multivariate models, MPR was not significantly associated with the risk of inpatient respiratory exacerbations (hazard ratio = 1.16 for MPR0.5 vs ≥0.8; 95% CI = 0.83-1.61).Study data were derived from insurance claims; adherence measures were based on prescription fills, not observed medication use.Adherence to dornase alfa was generally low, but varied by age and season. Adherence was not found to be significantly associated with respiratory exacerbations or total charges, but was associated with shorter hospital length of stay.