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Reduced charges and costs associated with outpatient autologous stem cell transplantation |
Meisenberg B R, Ferran K, Hollenbach K, Brehm T, Jollon J, Piro L D |
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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 with autologous stem cell rescue (HDC/ASCR) delivered in the outpatient setting. The outpatient approach consisted of two strategies: subtotal outpatient transplant (STOT) involving inpatient chemotherapy with outpatient follow-up (4 days of high-dose chemotherapy in the hospital with discharge to a 7 day a week outpatient clinic the day after completion of chemotherapy or as soon as clinically stable), and total outpatient transplant (TOT) involving outpatient chemotherapy with outpatient follow-up (pre-hydration and antiemetics followed by chemotherapy administered via a 4 hour infusion into a central venous catheter daily for 4 days in the outpatient setting and discharge to either private home or a local hotel. Patients were seen daily by transplant nurses and physicians and had chemistry and hematology assessment. Patients in both of the outpatient groups were required to have a caregiver present to help monitor oral intake, urine output and temperature, and to record oral medication use.
Economic study type Cost-effectiveness analysis.
Study population Patients receiving HDC/ASCR.
Setting Hospital. The economic study was carried out in the USA.
Dates to which data relate Source of effectiveness data The evidence for the final clinical outcomes was derived from a single study.
Link between effectiveness and cost data Costing was prospectively undertaken on the same patient sample as that used in the effectiveness analysis.
Study sample Power calculations were not used to determine the sample size. The study sample consisted of 94 patients: 20 in the inpatient group (mean (SD) age of 49 (9.9) years), 46 in the STOT group (mean (SD) age of 45.4 (10.1) years), and 28 in the TOT group (mean (SD) age of 43.8 (11.2) years).
Study design This was a prospective non-randomized study with concurrent controls, carried out in a single centre. The duration of the follow-up appears to have been until 30 days after stem cell infusion unless still under care for transplant related complications. The study appears to have had no loss to follow-up. In both outpatient transplant programmes, specific criteria for re-admission were used including uncontrolled emesis, nausea, diarrhoea, rapidly rising creatinine or unstable hemodynamics. The chemotherapy doses and schedule were identical in all three treatment settings.
Analysis of effectiveness The principle used in the analysis of effectiveness was intention to treat since patients were analysed in their original assignment groups. The main health outcomes were response rate, disease-free survival, incidence of complications (proportion of patients with fever, and proportion of patients with toxicities defined as one or more toxicity scores greater than or equal to grade 3 using the CALGB Common Toxicity Criteria, days on antibiotics, number of transfusions received, and duration of neutropenia). Length of hospital stay was also compared across the groups. The patient groups were found comparable with regard to Karnofsky performance status (KPS), diagnosis and age. Borderline statistically significant differences were seen with regard to numbers of prior cycles of chemotherapy and pre-transplant albumin, but these were not considered clinically significant.
Effectiveness results There were no statistically significant differences in response rates or disease-free survival between patients treated in the three treatment groups (further details were not reported). There were no statistically significant differences with regard to incidence of fever(STOT - 65.2%, TOT - 64.3%, and inpatient group - 70%), median days on antibiotics (4.6 versus 3.4 versus 5.2), mean number of transfusions received (RBC: 3.3 versus 3 versus 3.9; platelets: 4.5 versus 3 versus 3.7), duration of neutropenia or incidence of life-threatening toxicity (4.3% versus 14.3% versus 10%). The mean (SD) length of stay including re-admissions was 17.3 (5.1) for the inpatient group, 8.2 (5.7) for the STOT group, and 2.7 (6.7) days for the TOT group, (p<0.001).
Clinical conclusions This study revealed that the type of treatment settings did not affect the main clinical outcomes associated with high-dose chemotherapy with autologous stem cell rescue.
Measure of benefits used in the economic analysis No summary benefit measure was identified in the economic analysis, and only separate clinical outcomes were reported. The economic analysis appears to have been conducted on a cost-minimisation basis because of the therapeutic equivalence of the different treatment regimens.
Direct costs Costs were not discounted due to the short time frame of the cost analysis. Some quantities were reported separately from the costs. Cost items were not reported separately but were reported in terms of mean, standard deviation (SD), minimum, and maximum values. Cost analysis covered the standardised costs for pre-transplant central venous catheter placement, mobilisation of autologous progenitor cells, leukapheresis and cryopreservation, believing these to be essential components of the transplant process. The charge data were based on hospital and clinic computerised patient charts and financial records. The perspective adopted in the cost analysis was not explicitly specified and the price year was not reported. Outpatient patients were charged on a sliding scale basis assigned by the treating nurse based upon the complexity of their case. Other charges, (pharmacy, blood bank, radiology, laboratory and physician fees) were based on actual utilisation by individual patients. The costs of local hotels or apartments and patient meals were added to their outpatient bills. Institution-specific cost-to-charge ratios were used to translate each category of resource use. The cost of induction chemotherapy and pre-transplant evaluation such as radiographic studies, blood tests, and baseline physiologic assessments were not included in the cost analysis and neither were out-of-pocket expenses of either the patients or their caregivers.
Statistical analysis of costs Statistical analysis was performed to compare mean costs among the study groups: analysis of variance and t tests for normally distributed and Kruskal-Wallis tests for non-normally distributed data were used but it was not specified which referred to the cost analysis.
Sensitivity analysis No sensitivity analysis was conducted.
Estimated benefits used in the economic analysis Cost results The mean charges were $74,417 (inpatient), $60,447 (STOT), and $48,874 (TOT), (p=0.002). The corresponding values in terms of mean costs were $39,703, $36,188, and $29,370, (p=0.029). In terms of subgroup analysis, no statistically significant differences were observed among the groups for patients with KPS of 60% or 70% at the time of treatment. For patients with a KPS greater than 70%, mean treatment costs were $36,199 (inpatient), $32,292 (STOT), and $24,030 (TOT), (p<0.001).
Synthesis of costs and benefits Costs and benefits were not combined given the therapeutic equivalence of the treatment options and so economic analysis was performed on a cost-minimisation basis.
Authors' conclusions High-dose chemotherapy and stem cell rescue can be safely administered in the outpatient setting and results in significant cost savings.
CRD COMMENTARY - Selection of comparators A justification was provided for the choice of the comparator (inpatient delivery of HDC/ASCR) as the traditional procedure in the context in question. You, as a database user, should consider whether this is a widely used health technology in your own setting.
Validity of estimate of measure of effectiveness The internal validity of the effectiveness results cannot be guaranteed due to the non-randomised nature of the study design, the relatively small sample size, and the possibility of self-selection bias (although it was deemed that the comparability of patient groups in terms of both pretreatment characteristics and treatment-related toxicities would suggest that self-selection was not a major factor). The patient groups were found comparable with regard to Karnofsky performance status (KPS), diagnosis and age, but not in terms of numbers of prior cycles of chemotherapy and pre-transplant albumin. The representativeness of the patient sample to the study population cannot be objectively assessed and the inclusion and exclusion criteria prospectively employed in the study were not reported.
Validity of estimate of measure of benefit The analysis of benefits was based upon the therapeutic equivalence of treatment alternatives. The economic analysis therefore included only costs.
Validity of estimate of costs Some quantities were reported separately from the costs and adequate details of the methods of cost estimation were given. The fact that out-of-pocket expenses of patients and/or their caregivers were not included may have had adverse effects on the internal validity of the cost results whilst the use of cost-to-charge ratios strengthened the internal validity of the effectiveness results. Cost results may not be generalisable to other settings or countries. The price year was not given. The effects of the alternative strategies on indirect costs (productivity loss) were not analysed. Statistical analyses were performed on some resource use data and charge/cost data for groups and subgroups of patients, although the sample size used in the subgroup analysis was extremely small.
Other issues In view of the non-randomised nature of the study design, the relatively small sample size, the possibility of self-selection bias, and the lack of sensitivity analysis, some degree of caution needs to be exercised in the interpretation of the study results. The issue of generalisability to other settings or countries was not addressed although appropriate comparisons were made with other studies. The degree to which the study sample was representative of the study population was not discussed.
Implications of the study While these data should not be used to coerce reluctant individual patients to undergo their therapy in the outpatient setting, it does demonstrate what is achievable when patients and families are able to participate in their own care in this fashion.
Bibliographic details Meisenberg B R, Ferran K, Hollenbach K, Brehm T, Jollon J, Piro L D. Reduced charges and costs associated with outpatient autologous stem cell transplantation. Bone Marrow Transplantation 1998; 21(9): 927-932 Indexing Status Subject indexing assigned by NLM MeSH Adult; Aged; Ambulatory Care /economics; Antineoplastic Agents /economics /therapeutic use; Combined Modality Therapy; Costs and Cost Analysis; Fees and Charges; Hematopoietic Stem Cell Transplantation /economics /methods; Hospital Charges; Hospital Costs; Humans; Length of Stay; Middle Aged; Neoplasms /drug therapy /therapy; Prospective Studies; Safety; Transplantation, Autologous; Treatment Outcome AccessionNumber 21998000703 Date bibliographic record published 30/11/2000 Date abstract record published 30/11/2000 |
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