An Observational Real-Life Study with a New Infant Formula in Infants with Functional Gastro-Intestinal Disorders.

Functional gastro-intestinal disorders (FGIDs) impair the quality of life of many infants and their families. A formula with partial whey hydrolysate, starch, high magnesium content, prebiotic fructo-oligosaccharide and galacto-oligosaccharide and the probiotic Lactobacillus reuteri DSM 17938 was given during two weeks to 196 infants with at least two FGIDs. The efficacy was evaluated with the Cow Milk-associated Symptom Score (CoMiSS®) and quality of life with the QUALIN score. The formula was shown to decrease FGIDs within three days (decrease of CoMiSS -1.29 (3.15) (mean (SD), p < 0.0001) followed by an improvement of quality of life after seven days (increase QUALIN +1.4 (7.8); p: 0.008). Constipation decreased from 18.8% to 6.5% within three days. In combination with reassurance and guidance, the nutritional intervention was shown to be effective in infants with FGIDS in real-life circumstances.

Real-world data confirming the efficacy and safety of decitabine in acute myeloid leukaemia - results from a retrospective Belgian registry study.

Objectives: Currently, there is no standard treatment for patients with acute myeloid leukaemia (AML) ineligible for standard induction chemotherapy (IC). This study aimed to report real-world evidence data on the efficacy and safety of decitabine in this patient group.Methods: This study was a Belgian, retrospective, non-interventional, multicentre registry of patients ≥ 65 years, with newly-diagnosed de novo or secondary AML ineligible for IC. Patients were treated according to routine clinical practice. Overall survival (OS), progression-free survival (PFS) and transfusion independence for ≥8 consecutive weeks were evaluated.Results: Forty-five patients were enrolled, including 67% (n = 30) with secondary AML. Median OS and PFS were 7.3 months (95% CI: 2.2-11.1) and 4.1 months (95% CI: 2.1-7.6) respectively. A subpopulation analysis showed that patients treated with ≥4 cycles (n = 21) had significantly better outcomes compared to patients receiving <4 cycles (median OS 17.5 vs 1.6 months; median PFS 17.5 vs. 1.4 months). Twenty-five percent and 58% of patients that were respectively RBC or platelet transfusion-dependent at baseline became transfusion independent during treatment.Conclusion: This real-world data confirms that decitabine can lead to transfusion independence and longer OS in AML patients, particularly after administering ≥4 cycles, as indicated in the summary of product characteristics.

Photodynamic therapy using methyl aminolevulinate in the management of primary superficial basal cell carcinoma: clinical and health economic outcomes.

BACKGROUND: Basal cell carcinoma (BCC) is the most common form of skin cancer worldwide. Different treatment options exist. The efficacy of photodynamic therapy with methyl aminolevulinate (MAL-PDT) has been established in several randomized controlled trials (RCTs). Real life data can differ greatly from data derived from randomized controlled trials (RCTs). OBJECTIVES: To describe the results of a Belgian observational study concerning superficial BCC (sBCC) vis-a-vis clinical and health economic outcomes in order to evaluate the real-life practice of MAL-PDT. METHODS: This study was a prospective, single-arm, open study conducted at eight dermatological institutions during six months after the first MAL-PDT treatment. Eligible patients had to present with lesions, suitable for MAL-PDT according to Belgian reimbursement criteria. Resource use was collected during the study period. Clinical Response (CR) and Cosmetic Outcome (CO), as well as cost of care were evaluated. A subset analysis of patients with sBCC only was conducted. RESULTS: Ninety patients were identified for the analysis (mean age 65 years; 61% female). The mean number of lesions per patient was 1.6, mostly located on the face, the back and the chest. For the entire period, the mean number of visits to a dermatologist was 4 per patient including two MAL-PDT sessions. The average, cumulative amount of MAL used per treatment was 1,256 mg. Two patients experienced adverse events at the application site, none of them serious; all resolved completely. The CR rate was 89% at the end of the study. The CO was "excellent" or "good" in 96% of the patients. Total cost of care was Euro 289 ($414 U.S.) per patient. Cost per lesion was Euro 195 ($280 U.S.). CONCLUSION: The results from the real-life practice study confirm the efficacy found in prior, prospective randomized trials. About four visits and less than one tube of MAL are needed for the full treatment of sBCC in one patient.

Real-life practice study of the clinical outcome and cost-effectiveness of photodynamic therapy using methyl aminolevulinate (MAL-PDT) in the management of actinic keratosis and basal cell carcinoma.

Clinical trials have shown that photodynamic therapy using methyl aminolevulinate (MAL-PDT) is an effective treatment for actinic keratosis (AK), and nodular and superficial basal cell carcinoma (nBCC and sBCC) unsuitable for other available therapies. Economic evaluation models have shown that it is a cost effective intervention as well. The objectives of this prospective, observational, one arm study were (i) to verify in a real-life practice study the results obtained in previous clinical trials with MAL-PDT in the treatment of AK, nBCC and sBCC; (ii) to calculate the real-life cost of treatment and validate predictions from an economic evaluation model. Patients with AK and/or BCC were selected according to Belgian reimbursement criteria for treatment with MAL-PDT. Clinical response, cosmetic outcome and tolerability were assessed. MAL-PDT cost was calculated and compared to published model cost data. Data were collected from 247 patients (117 AK, 130 BCC). A complete clinical response was obtained for 83% of AK (85/102) and BCC (97/116) patients. A good or excellent cosmetic outcome was obtained for 95% of AK patients and 93% of BCC patients. Tolerability was good: only 2 patients withdrew for adverse events. Clinical results were similar to previous studies. Total cost of care per patient was euro 381 for AK, euro 318 for nBCC, and euro 298 for sBCC. Total cost per lesion was euro 58 for AK (identical to model prediction), euro 316 for nBCC and euro 178 for sBCC (both within 20% of model prediction). The clinical results of MAL-PDT in this real-life practice study confirm those demonstrated in previous clinical trials. Costs calculated from this study confirm predicted cost-effectiveness in the original model for MAL-PDT in the management of AK and BCC.

Resource use and cost of hepatitis C-related care.

BACKGROUND: Chronic hepatitis because of the hepatitis C virus (CHC) is a major health problem that can lead to decompensated cirrhosis, hepatocellular carcinoma, and eventually death, all of which are associated with significant healthcare costs. AIM: To update the cost of care of CHC according to the different severity stages of the disease in a west European country (Belgium). METHODS: Medical records of 157 patients, who were referred to the medical specialist at different stages of disease, were reviewed to identify the medical costs over a follow-up period of 3 years or 2 years in the case of liver transplantation (LT). Six disease stages were defined on the basis of histology (Metavir classification) and/or clinical data. RESULTS: In comparison with mild disease, the cost increased 1.6 times in the case of decompensated cirrhosis, 1.9 times in the case of hepatocellular carcinoma, and 3.4 in the case of LT. The costs for medication, hospitalization, and ambulatory care were, respectively, on the one hand, 81, 8, and 11% for mild disease and, on the other, 18, 79, and 3% for LT. In the case of a sustained viral response, the cost of follow-up within 3 years decreased by 45% for patients with mild and moderate disease. CONCLUSION: Antiviral treatment is the most important factor governing cost in mild and moderate disease, but once complications of CHC occur, hospitalization costs far exceed the cost of antiviral therapy. Already during the first 3 years of follow-up, sustained viral response decreased the cost significantly. Treatment of patients with CHC in an early stage has the potential to be cost-effective.

Cost effectiveness of darunavir/ritonavir 600/100 mg bid in protease inhibitor-experienced, HIV-1-infected adults in Belgium, Italy, Sweden and the UK.

BACKGROUND: Two phase II trials (POWER 1 and 2) have demonstrated that darunavir co-administered with low-dose ritonavir (DRV/r) provides significant clinical benefit compared with control protease inhibitors (PIs) in highly treatment-experienced, HIV-1-infected adults, when co-administered with optimized background therapy (OBR). OBJECTIVE: To determine whether DRV/r is cost effective compared with control PIs, from the perspective of Belgian, Italian, Swedish and UK reimbursement authorities, when used in treatment-experienced patients similar to those included in the POWER 1 and 2 trials. METHODS: An existing Markov model containing health states defined by CD4 cell count ranges (> 500, 351-500, 201-350, 101-200, 51-100 and 0-50 cells/mm³) and death was adapted for use in four European healthcare settings. Baseline demographics, CD4 cell count distribution and antiretroviral drug usage reflected those reported in the POWER 1 and 2 trials. Virological/immunological response rates and matching transition probabilities over the patient's lifetime were based on results from the POWER trials and published data. After treatment failure, patients were assumed to switch to a tipranavir-containing regimen plus OBR. For each CD4 cell count range, utility values and HIV-related mortality rates were obtained from the published literature. National all-cause mortality data and published data on the increased risk of non HIV-related mortality in HIV-infected individuals were taken into account in the model. Data from observational studies conducted in each healthcare setting were used to determine resource-use patterns and costs associated with each CD4 cell count range. Unit costs were derived from official local sources; a lifetime horizon was taken and discount rates were selected based on local guidelines. RESULTS: In the base-case analysis, quality-adjusted life-year (QALY) gains of up to 1.397 in Belgium, over 1.171 in Italy, 1.142 in Sweden and 1.091 in the UK were predicted when DRV/r-based therapy was used instead of control PI-based treatment. The base-case analyses predicted an incremental cost-effectiveness ratio (ICER) of €11,438/QALY in Belgium, €12,122/QALY in Italy,€10,942/QALY in Sweden and €16,438/QALY in the UK. Assuming an acceptability threshold of €30,000/QALY, DRV/r-based therapy remained cost effective over all parameter ranges tested in extensive one-way sensitivity analyses. Probabilistic sensitivity analysis revealed a 95% (Belgium), 97% (Italy), 92% (Sweden) or 78% (UK) probability of attaining an ICER below this threshold. CONCLUSION: From four European payer perspectives, DRV/r-based antiretroviral therapy is predicted to be cost effective compared with currently available control PIs, when both are used with an OBR in treatment-experienced, HIV-1-infected adults who failed to respond to more than one PI-containing regimen.

Cost-effectiveness analysis of bicalutamide (Casodex) for adjuvant treatment of early prostate cancer.

OBJECTIVES: To assess the cost-effectiveness of bicalutamide (Casodex) as adjuvant treatment in early prostate cancer (EPC). METHODS: A Markov state transition model was developed, using disease progression rates from a large (N = 8113) clinical trial program comparing bicalutamide in addition to standard care with standard care alone. Utility scores for different disease stages were obtained from published reports. Costs of disease progression were obtained from a retrospective patient chart analysis in six Belgian centers (n = 60). The time horizon was 15 years and the analysis was conducted from the public payer perspective. RESULTS: The model showed good validity in predicting clinical outcomes. At a time horizon of 15 years, an incremental cost-effectiveness of 27,059 euros/QALY was obtained. The main factors influencing conclusions included the time horizon, the duration of bicalutamide treatment, which was set at a maximum (5 years) in the base case, and possible differences in prognosis of metastatic cancer between comparators. Also the discounting of health effects significantly altered cost-effectiveness ratios. Many of these influences are inherently associated with any cost-effectiveness analysis related to treatment of early, slowly progressing malignancies because such an analysis requires a sufficient time horizon to include not only the treatment costs but its benefits as well. CONCLUSION: Based on the current data, bicalutamide appears to be a cost-effective option for adjuvant treatment of EPC.