A prospective economic evaluation of basiliximab (Simulect) therapy following renal transplantation.

BACKGROUND: Immunoprophylaxis with basiliximab (Simulect), an anti-interleukin-2-receptor (anti-IL-2R; CD25) chimeric monoclonal antibody, has been demonstrated to significantly reduce the incidence of acute cellular rejection in adult renal allograft recipients (32% vs. placebo, p < 0.01). METHODS: An economic evaluation was conducted as part of a U.S. multi-center, randomized, double-blind, placebo-controlled clinical trial comparing basiliximab plus dual immunosuppressive therapy (cyclosporine modified [Neoral] and corticosteroids) to dual therapy alone. Healthcare resources utilized by the 346 subjects in the 'intent-to-treat' population were prospectively collected over the 1-yr study period. Direct medical costs were determined for all hospitalizations, outpatient provider visits, procedures (excluding the initial transplant procedure), laboratory and diagnostic tests, and immunosuppressants, including basiliximab when administered. RESULTS: Total first-year medical costs were lower for the basiliximab group than for the placebo group ($28 927 vs. $32 300, difference = $3373). although this difference was not statistically significant. First-year hospital costs for treating acute rejection were also lower for the basiliximab group ($9328 vs. $10761, difference = $1433); however, this difference did not achieve statistical significance. Importantly, the efficacy analysis demonstrated a significant reduction in the incidence of acute rejection (38 vs. 55%, p < 0.01) in the basiliximab arm, and this was accomplished without increasing the overall cost of care. Fewer basiliximab-treated patients (8 vs. 15%,, p = 0.03) were hospitalized. This observation suggested less serious illness and reduced treatment costs among basiliximab-treated patients, because the overall incidence of infection was similar between the groups. The adverse event profile of patients receiving basiliximab was clinically and economically indistinguishable from that of those treated with placebo. CONCLUSION: Induction immunosuppression with basiliximab, combined with cyclosporine modified and corticosteroids, was therapeutically beneficial and contained medical costs during the initial post-transplant year.

Cost of treating an episode of variceal bleeding in a VA setting.

OBJECTIVES: The specific aims of this study were to develop a demographic description of a sample of patients presenting with bleeding esophageal varices and determine the direct health care costs of variceal bleeding. METHODS: This was a retrospective evaluation of patients who underwent esophagogastroduodenoscopy at the Portland VA Medical Center between January 1993 and May 1997. Data sources included both electronic databases and patient medical charts. The primary unit of analysis was an episode of care, defined as an index bleed plus 6 months of follow-up or death, whichever came first. RESULTS: The total inpatient direct cost was $1,566,904 and outpatient direct cost was $104,611, for a total of $1,671,515 for 100 bleeding episodes in 79 patients. Episodes of care for patients receiving < or =2 units of packed red blood cells were approximately a third as costly as those receiving >2 units of packed red blood cells (n = 17, $6,470 and n = 83, $17,553). The difference in costs was statistically significant (p < 0.05), and primarily attributable to hospital bed costs. CONCLUSIONS: There is a substantial financial burden associated with this illness, primarily attributable to inpatient costs. In addition to severity of bleeding, Child's class, endoscopic findings, and the timing of pharmacological therapy seem to influence the overall cost of managing esophageal varices.

Prospective economic evaluation accompanying a trial of GM-CSF/IL-3 in patients undergoing autologous bone marrow transplantation for Hodgkin's and non-Hodgkin's lymphoma. IL-3 BMT Study Team.

Our objective was to assess the economic impact of a new cytokine therapy that was being compared to standard therapy as supportive care in patients receiving autologous bone marrow transplantation for treatment of lymphoma. We performed an economic study accompanying a multicenter, randomized, controlled clinical trial in academic medical centers. One hundred and fifteen patients consented to participate in a parallel economic study of a randomized controlled trial of sequential IL-3 followed by GM-CSF vs GM-CSF alone after autologous bone marrow transplantation. We measured costs and quality-adjusted survival over a 13-month follow-up period. For the 13-month study period, the total cost estimates were $79892 (95% CI $69343 to $90544) for patients receiving GM-CSF alone and $89651 (95% CI $79769 to $102114) for patients receiving IL-3/GM-CSF. The difference was not statistically significant. During the 13-month study period, the total number of quality-adjusted life-months in the GM-CSF arm was 6.67 (95% CI 5.75 to 7.56) months, while the total number of quality-adjusted life-months in the IL-3 arm was 6.26 (95% CI 5.34 to 7.15) months. The difference in quality-adjusted life-months between the two treatment arms was not statistically significant. We conclude that economic analysis of a phase III clinical trial of IL-3/GM-CSF compared with GM-CSF alone showed no significant effect of IL-3 on the costs of care for patients undergoing bone marrow transplantation for a period of up to 13 months after the procedure. This study demonstrates the feasibility of prospective economic evaluation within phase III trials of new cancer therapies. Data from this type of economic protocol could be used to help physicians, patients and managed care organizations understand the effect of new treatments from both a clinical and an economic perspective.

Quality of life in persons with irritable bowel syndrome: development and validation of a new measure.

How irritable bowel syndrome (IBS) and its treatment affect quality of life (QOL) is important. To develop a quality-of-life measure specific to irritable bowel syndrome, items were generated using a conceptual model and qualitative interviews with persons diagnosed using the Rome criteria. Symptom frequency and bothersomeness indices were created. Psychometric evaluation methods involved an initial cross-sectional survey followed by a repeat survey. The resulting 34-item measure demonstrated high internal consistency (Cronbach's alpha = 0.95) and high reproducibility (ICC = 0.86) with average time of seven days (SD = 1). For discriminant validity: number of symptoms (P < 0.05), self-reported severity of symptoms (P < 0.001), and the functional bowel disorder severity index (P < 0.001) significantly predicted IBS-QOL scores. Convergent validity and analyses confirmed predictions that scores are more closely related to psychological well-being (0.45) than to function (0.36). We conclude this measure meets established psychometric criteria for reliability and validity; testing of its responsiveness is warranted.