Impact of a risk-sharing agreement in rheumatoid arthritis in Spain.

CONTEXT AND OBJECTIVE: Risk-sharing agreements(RSA) allow decision-makers to manage the uncertainty associated with effectiveness and costs of treatments. Our objective was to estimate the economic impact of RSA implementation on treatment of patients diagnosed with rheumatoid arthritis(RA) with certolizumab pegol(CZP) and assess the potential impact of alternative RSA. METHODS: Under original RSA, treatment with CZP was reimbursed when the response was optimal (DAS28 score <3.2) or satisfactory (DAS28 score ≥3.2 and reduction from baseline ≥1.2) at 12 weeks. Alternative RSA would additionally include a 50 % reimbursement for moderate responders(DAS28 score >3.2 and ≤5.1, and reduction from baseline between 0.6 and 1.2). We estimated average savings per patient for hospital's pharmacy service(HPS) at 12 weeks, taking into account the pharmacological cost of CZP. Uncertainty associated with effectiveness of CZP was assessed through 1000 Monte Carlo simulations. RESULTS: After 12 weeks of treatment, 57.8 % (n = 52) and 22.2 %(n = 20) of patients had optimal and satisfactory responses, respectively, and average disease activity improved by 1.77 points. Average savings for HPS amounted to 876.9€ and 706.4€ per patient under original and alternative RSA, respectively. Savings in simulated cohort reached 846.2€ and 681.8€ per patient, respectively, leading to estimated net savings for HPS of 846,209€ and 681,790€, respectively. CONCLUSIONS: RSA implementation on patients with RA treated with CZP has generated savings and improved efficiency within HPS.

Value of α-fetoprotein as an early biomarker for treatment response to sorafenib therapy in advanced hepatocellular carcinoma.

Sorafenib is an oral multikinase inhibitor with antiangiogenic and antiproliferative properties, and is used as the first-line treatment for patients with advanced hepatocellular carcinoma (HCC). Previous studies have identified an improvement in overall survival and progression-free survival in patients with a manageable toxicity profile. α-fetoprotein (AFP) has been revealed to be of great diagnostic and predictive value for tumour staging in multiple studies; however, its role as a predictive factor of response to treatment with sorafenib is not entirely clear. The present study aimed to determine the effectiveness of sorafenib and investigate the value of AFP as a predictive factor of early response to sorafenib in patients with HCC. Effectiveness was analysed based on median overall survival (mOS) time, while to analyse the possible predictive value of AFP, patients were classified into two groups: Non-responders (≤20% AFP reduction) and responders (>20% AFP reduction) at 6-8 weeks of treatment when compared with basal AFP level. For assessment of toxicity, any adverse effects were recorded. A total of 167 patients were included, who collectively exhibited a mOS time of 11 months with a median treatment duration of 5 months. The mOS time was significantly higher for patients with better hepatic function (12 months in cases of Child-Pugh score A vs. 8 months in cases of Child-Pugh score B; P=0.03) and with basal AFP values ≤200 ng/ml (14 months vs. 8 months in patients with AFP levels >200 ng/ml; P=0.01). A >20% reduction of AFP at 6-8 weeks was determined to be a positive predictive factor upon multivariate analysis (P=0.002), obtaining, for the responder patients, an mOS of 18 months compared with 10 months (P=0.004) for the non-responders. The main adverse reactions were hand-foot syndrome (35/167; 21%), diarrhoea (39/167; 23.4%), anorexia (29/167; 17.4%) and arterial hypertension (30/167; 18%). In conclusion, a >20% drop in AFP at 6-8 weeks may be useful as a predictive factor of response to sorafenib, as indicated by its association with longer survival times in patients with advanced HCC following treatment with sorafenib in the present study.

Study of the effectiveness of first-line treatment in renal cell carcinoma.

The emergence of novel drugs corresponds with the determination of the effectiveness of the current treatments used in clinical practice. A retrospective observational study was conducted to evaluate the effectiveness of first-line treatments and to test the influence of the prognostic factors established using the Memorial Sloan-Kettering Cancer Center (MSKCC) and the analysis of Mekhail's study for two or more metastatic sites. The primary endpoints were median progression-free survival (mPFS) and median overall survival (mOS) times. A total of 65 patients were enrolled and the mPFS and mOS of the patients treated with sunitinib (n=51) were 9.0 and 20.1 months, respectively, and for the patients treated with temsirolimus (n=14) these were 3.0 and 6.2 months, respectively. In the poor-prognosis (PP) group, a difference of 1.2 months (P=0.049) was found in mPFS depending on the first-line treatment. A difference of 4.1 months (P=0.023) was also found in mPFS when classified by histology (clear verses non-clear cell) in the sunitinib-treatment group. When stratified by the prognostic group, differences of >7 months (P<0.001) were found between the groups. Therefore, it was concluded that the effectiveness of the treatments was reduced compared to previous studies and differences were found in the PP group when classified by first-line drug and histology. Additionally, the influence of prognostic factors on OS and the value of stratifying patients using these factors have been confirmed.

Effectiveness of the bevacizumab-irinotecan regimen in the treatment of recurrent glioblastoma multiforme: Comparison with other second-line treatments without this regimen.

A retrospective cohort study was conducted to analyse the effectiveness of bevacizumab and irinotecan (BVZ/CPT-11) as a second-line treatment in patients with primary glioblastoma multiforme (GBM) in comparison with a control group that were not administered BVZ/CPT-11 at the first recurrence. The difference in overall survival (OS) between the two groups was used as a predictor of effectiveness. OS was calculated according to prognostic factors and gender. A total of 28 and 32 patients were enrolled in the BVZ/CPT-11 cohort and control group, respectively. The median OS was 17.94 months (95% CI, 14.91-20.96) in the BVZ/CPT-11 treatment cohort and 10.97 months (95% CI, 7.65-14.30) in the control cohort. The results obtained on the effectiveness of BVZ/CPT-11 treatment in patients with primary GBM are consistent with data from previous studies. No significant differences were identified in OS based on prognostic factors; therefore, the latter cannot be used to select patients who would incur the greatest benefits from BVZ/CPT-11 treatment.

Good clinical and cost outcomes using dexrazoxane to treat accidental epirubicin extravasation.

A 75-year-old man diagnosed with lower esophageal adenocarcinoma suffered from epirubicin extravasation during the second cycle of neoadjuvant chemotherapy with epirubicin and oxaliplatin. A full recovery was achieved after treatment with dexrazoxane (Cardioxane® ). This is the first time in our hospital that extravasation of an anthracycline has been treated with dexrazoxane. We used Cardioxane® , approved for the prevention of anthracycline-induced cardiotoxicity, while Savene® is indicated for the treatment of anthracycline extravasation. The treatment was effective, and the selection of Cardioxane® (seven-fold cheaper than Savene® ) yielded a cost saving. Consequently, Cardioxane® has been included in our guidelines for anthracycline extravasation.