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Economic analysis of a randomized clinical trial to compare filgrastim-mobilized peripheral-blood progenitor-cell transplantation and autologous bone marrow transplantation in patients with Hodgkins and non-Hodgkins lymphoma |
Smith T J, Hillner B E, Schmitz N, Linch D C, Dreger P, Goldstone A H, Boogaerts M A, Ferrant A, Link H, Zander A, Yanovich S, Kitchin R, Erder M H |
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Record Status This is a critical abstract of an economic evaluation that meets the criteria for inclusion on NHS EED. Each abstract contains a brief summary of the methods, the results and conclusions followed by a detailed critical assessment on the reliability of the study and the conclusions drawn. Health technology High-dose chemotherapy (HDC) with either filgrastim-mobilized peripheral-blood progenitor-cell transplantation (PBPCT) or autologous bone marrow transplantation (ABMT).
Economic study type Cost-effectiveness analysis.
Study population Patients with Hodgkin's Disease (HD) and non-Hodgkin's lymphoma (NHL), aged between 16 and 60 years and with relapsed HD and NHL, an Eastern Cooperative Oncology Group performance status 0 to 2, an absolute neutrophil count (ANC) >
Setting Cancer centres in Germany, the United Kingdom and Belgium. Resource utilisation data were used to project costs to one cancer centre in the United States.
Dates to which data relate The years during which the effectiveness and resource use data were collected were not stated; results of the trial were reported elsewhere in 1996.1995 costs were used.
Source of effectiveness data The evidence for final outcomes was derived from a single study.
Link between effectiveness and cost data The costing was undertaken retrospectively on a different patient sample to that used in the effectiveness study.
Study sample 72 candidate patients were registered at six sites in England, Germany and Belgium.58 patients were given HDC and received stem-cell support (31 ABMT and 27 PBPCT). Power calculations were not used to determine the sample size.
Study design The study was a phase III, randomised, open-label multicentre study. The trial was performed in six cancer centres in Germany, the United Kingdom and Belgium.
Analysis of effectiveness It is not clear from this paper whether the analysis of effectiveness was based on intention to treat or treatment completers only. The primary study end-points were the number of days on which platelet transfusion was required, duration of hospitalisation, mortality rates, time to platelet recovery, and time to neutrophil recovery.
Effectiveness results All clinical study end points were improved for PBPCT patients compared to ABMT patients. The median number of days on which platelet transfusions were required was 6 for PBPCT versus 10 for ABMT patients (p<.001). The duration of hospitalisation was significantly shorter, with a median of 17 days for PBPCT and 23 days for ABMT patients (p=.002). The median time to platelet recovery was 16 for PBPCT versus 23 for ABMT patients (p<.02). The median time to neutrophil recovery was 11 for PBPCT versus 14 for ABMT patients (p<.005). One patient in the ABMT arm died following transplant during the course of the study. A total of 7 of the 58 transplanted patients died (ABMT group, 4 and PBPCT group, 3): 4 from relapse, 1 from hepatitis C, one 1 from respiratory insufficiency, and 1 from a suspected pulmonary embolism.
Clinical conclusions PBCT is as safe and more effective than ABMT for HD and NHL in the short term.
Measure of benefits used in the economic analysis No single measure of benefit was produced within the economic evaluation as the effectiveness analysis revealed similarity in terms of effectiveness between the intervention and the comparator.
Direct costs Actual resource utilisation was determined from data gathered prospectively during the clinical trial. A data set was created of charges and costs based on similar patients at one centre in the United States using both ABMT and PBPCT; this included 50 HD and NHL patients treated since 1992 and included all charges, ratios of cost to charge, and costs for all inpatient and selected outpatient services used in the trial. Analysis was based on patients who actually underwent HDC, as the full costs of those who relapsed or left the study were unknown. Both quantities and costs were reported separately. Costs were not discounted as the study included only short term results. Price dates refer to 1995.
Sensitivity analysis One-way simple sensitivity analysis was performed to determine the consequences of alternative assumptions about variation in autograft procurement costs, intensive care unit use, and costs of BEAM chemotherapy.
Estimated benefits used in the economic analysis Cost results The overall projected cost saving associated with the use of PBPCT instead of ABMT was $13,521, (23% of the total). Autograft collection was less costly in the PBCBT arm ($5,760 versus $8,531) due primarily to the absence of operating room costs; overall autograft collection accounted for only 14% to 15%. The resource utilisation for the PBCBT and ABMT arms was similar during BEAM chemotherapy as both groups received identical conditioning regimens ($10,019 versus $10,030). The largest difference was in the mean length of hospitalisation and use of supportive care after BEAM chemotherapy until discharge ($30,013 versus $40,752). Under all the sensitivity analyses which were performed PBCBT remained less expensive than ABMT (range 19% to 24%).
Synthesis of costs and benefits Synthesis was not undertaken by the authors since it was considered irrelevant given that PBPCT was shown to be the dominant strategy with better clinical outcomes and lower costs.
Authors' conclusions The cost savings from less hospitalisation and supportive care far outweigh the initial cost of filgrastim used to collect the peripheral stem cells. These significant cost savings may make HDC even more cost-effective than reported in previous studies.
CRD COMMENTARY - Selection of comparators The reason for the choice of comparator is clear.
Validity of estimate of measure of benefit The estimate of the measure of benefit is likely to be internally valid (based on a multicentre randomised controlled trial); the results are detailed separately in another paper. The authors noted that the trial establishes only short-term efficacy and that the sample size was too small to detect long-term differences in survival.
Validity of estimate of costs Adequate details of the methods of cost estimation were given, and the authors acknowledged the potential shortcomings of using separate clinical and economic data.
Other issues Issues of generalisability were addressed and appropriate comparisons with other studies were made. The authors' conclusions and caveats were justified.
Source of funding Supported in part by a grant from the Agency for Health Care Policy and Research, Bethesda, MD (R01 HS 07614) (TJS, BEH), a grant from the Office of Cancer Communications, National Cancer Institute, Bethesda, MD (RFP CO 94388-63) ( TJS), an unrestricted research award from Amgen Inc, Thousand Oaks, CA (TJS, BEH) a Career Development Award (TJS) and a Faculty Research Award (BEH) from the American Cancer Society, Atlanta, GA, and a Faculty Scholar Award, Project on Death in America, Open Society, New York (TJS).
Bibliographic details Smith T J, Hillner B E, Schmitz N, Linch D C, Dreger P, Goldstone A H, Boogaerts M A, Ferrant A, Link H, Zander A, Yanovich S, Kitchin R, Erder M H. Economic analysis of a randomized clinical trial to compare filgrastim-mobilized peripheral-blood progenitor-cell transplantation and autologous bone marrow transplantation in patients with Hodgkins and non-Hodgkins lymphoma. Journal of Clinical Oncology 1997; 15(1): 5-10 Indexing Status Subject indexing assigned by NLM MeSH Adolescent; Adult; Bone Marrow Transplantation /economics; Cancer Care Facilities /economics /utilization; Combined Modality Therapy /economics; Costs and Cost Analysis; Filgrastim; Granulocyte Colony-Stimulating Factor /economics /therapeutic use; Health Resources /utilization; Health Services Research /methods; Hematopoietic Stem Cell Transplantation /economics; Hodgkin Disease /therapy; Hospital Costs /statistics & Humans; Length of Stay /economics; Lymphoma, Non-Hodgkin /therapy; Middle Aged; Prospective Studies; Recombinant Proteins; Sensitivity and Specificity; Virginia; numerical data AccessionNumber 21997000223 Date bibliographic record published 30/11/1998 Date abstract record published 30/11/1998 |
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