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

Abstract

Summary: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 $79 892 (95% CI $69 343 to $90 544) for patients receiving GM-CSF alone and $89 651 (95% CI $79 769 to $102 114) 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.

Calculation of quality adjusted life years in the published literature: a review of methodology and transparency

“…Even where missing data acknowledged as a problem it is rarely dealt with adequately. Studies not previously discussed [21][22][23][24][25][26][27][28][29][30][31] are listed for information.…”

Delaying treatment with granulocyte colony-stimulating factor after allogeneic bone marrow transplantation for hematological malignancies: a prospective randomized trial

“…[23][24][25] In a recent study, the addition of interleukin-3 starting on day 6 after initiating G-CSF on day 1 after autologous BMT seemed to be superior to a treatment schedule which starts with IL-3 only after G-CSF application from days 1 to 6. 26 Timing of treatment with G-CSF in the phase of expansion of the myeloid cell lines seems to be more effective, than just in the early phase after transplantation.…”

Clinical Economics in Clinical Trials: The Measurement of Cost and Outcomes in the Assessment of Clinical Services through Clinical Trials.

“…In multicenter (multicountry) clinical trials, economists are concerned about the possibili Another example of an economic study alongside a phase III clinical trial is the IL-3 bone-marrow trans plantation study, a multicenter (academic medical cen ters), randomized, controlled clinical trial. 51 The study assessed economic impact of a new cytokine therapy followed by GM-CSF that was being compared to stan dard therapy, GM-CSF alone as supportive care, in patients receiving autologous bone-marrow transplant (ABMT) for treatment of lymphoma. Economic analy sis of this study showed no significant impact of IL-3 on the costs of care for patients undergoing ABMT for a period of up to 13 months after the procedure.…”