Long-term persistency and costs associated with the use of iron chelation therapies in the treatment of Sickle cell disease within Medicaid programs.

OBJECTIVE: This retrospective study evaluated iron chelating therapy (ICT) discontinuation and costs in Sickle cell disease (SCD) Medicaid recipients using healthcare claims from 2006-2010. METHODS: Patients with ≥1 SCD diagnosis claim, ≥2 claims for deferoxamine (DFO) or deferosirox (DFX), and continuous enrollment ≥6 months prior to and 18 months following ICT initiation were included. Outcomes included treatment discontinuation, persistence (i.e., refill gaps ≥6 weeks), and total healthcare costs. RESULTS: The average age among 404 SCD patients meeting study inclusion criteria was 18.7 (±11.0) years, with 45.8% being males and 66.7% being Blacks. Switches or combinations from DFO at index occurred in 124 (74.7%) patients compared to 10 (4.2%) with DFX at index. The Cox regression model that assessed long-term medication persistence indicated a 1.30-times higher likelihood of treatment discontinuation with DFO compared to DFX (95% CI: 1.06-1.61). Some 19.7% of patient remained on DFX relative to 4.8% on DFO. Both inpatient and total costs were similar in DFX and DFO treatment groups. Following 1 year of treatment, 37.4% remained on DFX compared to 15.7% on DFO. Meaningful differences in treatment discontinuation between the two treatment groups did not occur until 220+ days during the study period. At 18-months, treatment discontinuation rates were high in both groups; 95% for DFO and 80% for DFX. CONCLUSION: This study of SCD Medicaid patients found more therapeutic switches from DFO to DFX and a higher medication persistency rate with DFX than DFO. The conclusions are limited by the study\u27s retrospective nature, which depends on multivariate statistics to account for patient heterogeneity and risk factors

Effectiveness of adjuvant imatinib in patients with gastrointestinal stromal tumor: results of a population-based, matched-cohort study

Data are limited on the use of adjuvant imatinib in patients with gastrointestinal stromal tumor (GIST) outside of clinical trials. This retrospective, population-based, matched-cohort study evaluated adjuvant imatinib treatment patterns, assessed impact on clinical outcomes, and estimated effectiveness based on number needed to treat (NNT). GIST-related claims from the PharMetrics claims database were included (2000-2010). A stepwise identification algorithm identified appropriate patients based on GIST-related ICD-9-CM codes, who were classified as 'imatinib (IM) patients' receiving imatinib within 84 days post-surgery and 'non-imatinib (non-IM) patients' undergoing surgery but not receiving imatinib during the study period. The primary composite outcome was based on incidence of a second GIST-related surgery and long-term follow-up in the matched cohorts. IM patients were matched with up to eight non-IM patients on age, gender, ICD-9-CM code, and first surgery date. A total of 118 IM and 4088 non-IM patients with possible GIST ICD-9s and surgery were included. The median duration between first and second surgeries was significantly longer in IM than non-IM patients (488 vs. 290 days; p = 0.0005). IM patients also had longer median follow-up from initial surgery to composite outcome (433 vs. 320 days; p = 0.002). Adherence to IM, measured by medication possession ratio, was 0.83 and 0.73 during the first and second years of treatment, respectively. IM patients were less likely to have the composite outcome compared with non-IM patients (hazard ratio = 0.501; p = 0.0005). The NNT to prevent one outcome was 4. Patients receiving adjuvant imatinib treatment were less likely to have second surgery or be lost to follow-up, and had a longer interval to second surgery. Although treatment with adjuvant imatinib in patients with primary GIST is effective, adherence to imatinib and treatment duration are less than recommended by current treatment guidelines