Reliability and validity of a patient self-administered daily questionnaire to assess impact of oral mucositis (OM) on pain and daily functioning in patients undergoing autologous hematopoietic stem cell transplantation (HSCT).

Oral mucositis (OM) is a frequent complication of myeloablative therapy and HSCT. We evaluated the feasibility, reliability, and validity of a new patient self-reported daily questionnaire on OM and its impact on daily functions. This OM Daily Questionnaire (OMDQ), containing 10 items, was developed for use in palifermin clinical trials. In a phase 3 study, 212 patients received palifermin or placebo for three consecutive days before conditioning and three consecutive days after HSCT. Compliance rates were consistently >80% for most patients. Mouth and throat soreness (MTS) and MTS-Activity Limitations (MTS-AL) (swallowing, drinking, eating, talking, and sleeping) scores on consecutive days were highly correlated (days 7,8 = 0.70-0.86; test-retest reliability). Correlations among items measuring the same construct ranged between 0.5 and 0.8 (internal consistency reliability). The WHO Oral Toxicity scale was the clinical comparator to assess the criterion, discriminative, and evaluative validities of MTS-related questions. Most correlation coefficients between the WHO and MTS ranged between 0.45 and 0.55. Patients with more severe WHO OM grades had higher MTS mean scores. Changes in MTS scores were similar, but patients detected changes 1-3 days earlier than clinicians. In conclusion, the OMDQ is a feasible, reliable, valid, and responsive patient-reported measure of OM severity.

Platelet transfusions: utilization and associated costs in a tertiary care hospital.

We implemented a prospective study to evaluate platelet transfusion utilization, resource use, and costs in a tertiary care hospital over a 6-month period. All hospitalized patients receiving platelet transfusions between July and December 1996 were followed prospectively to determine platelet use and costs. Clinical and financial data were collected, evaluated, and compared to identify trends in resource utilization based on admitting service and platelet-refractory status. One thousand nine hundred forty-four platelet units were transfused to 245 hospitalized patients (50.6% male, mean age 49 years) during the study period. The majority of platelet units transfused were single donor (N = 1,460, 75%) and administered to bone marrow patients and patients with a hematological malignancy/disorder. Median hospitalization costs per admission were $27,750, ranging from a high of $58,729 for admission to the Bone Marrow Transplant service to $13,856 per admission to the Internal Medicine/Other service. Patients were refractory to platelet transfusions during 21.6% of hospitalizations. Hospital stays were longer (35.0 days vs. 14.4 days, P < 0.001) and inpatient hospital costs ($103,956 vs. $37,817, P < 0.001) were more than two and a half times higher for patients refractory to platelet transfusions. Platelet utilization, resource use, and costs vary by admitting service. Refractoriness to platelet transfusion was associated with significantly greater costs and lengths of stay. Monitoring platelet transfusion practices, particularly for patients refractory to platelet transfusions, may be beneficial for limiting costs and improving efficacy.

Summary measures and statistics in the analysis of quality of life data: an example from an EORTC-NCIC-SAKK locally advanced breast cancer study.

Quality of Life (QL) is now included as an endpoint in many phase III cancer clinical trials. Numerous statistical techniques have been presented in the literature to analyse QL data but there is still no agreement as to what is the optimal approach of analysis. In this paper we, therefore, present and compare various techniques which have all appeared in the literature and which may be globally described as summary measures and summary statistics. These techniques are illustrated using data from an EORTC clinical trial in locally advanced breast cancer (EORTC trial 10921). It is also explained in this paper how and when these techniques may be used in other cancer settings. For EORTC trial 10921, it is shown that by choosing different techniques different conclusions may be drawn concerning the QL outcome. This highlights the importance of choosing an appropriate primary statistical method and for describing it a priori in the protocol and analysis plan. In this paper, we show the importance of performing sensitivity or supportive analysis to support conclusions drawn from the primary analysis.

Assessing the value of a new pharmaceutical. A feasibility study of contingent valuation in managed care.

OBJECTIVES: The authors assessed the feasibility and construct validity of the contingent valuation method for measuring the monetary value to healthy enrollees in a health maintenance organization of a new drug, filgrastim, as prophylaxis against febrile neutropenia after chemotherapy treatment for cancer. METHODS: A random sample of 220 enrollees from a closed-panel staff-model health maintenance organization who did not have cancer were interviewed. Chemotherapy, febrile neutropenia and filgrastim were described by video and decision board. Questions were asked in two different scenarios: (1) User-based: Assuming they were at the point of consumption and about to receive chemotherapy, what is the maximum they would be willing to pay to receive filgrastim? and (2) Insurance-based: Given they were at risk of cancer in the future, what is the maximum they would be willing to pay in additional monthly insurance premiums to add filgrastim to the plan? In a second insurance scenario where respondents were told that filgrastim was covered, what is the minimum reduction in premium that persons were willing to accept to relinquish coverage of the drug? A 2 x 2 factorial design was used to contrast two bidding algorithms to test for starting point bias and two 5-yearly prior risks of cancer, 1/200 versus 1/100. Main effects were tested by ANCOVA controlling for age, sex, health, and income. RESULTS: Demographics of experimental cells were similar. No evidence was found of significant starting point bias. For user-based questions, as expected, willingness-to-pay increases with febrile neutropenia risk reduction, but at a declining marginal rate. Despite careful presentation of information to respondents, willingness-to-pay for insurance was higher in the lower prior risk group. Consistent with previous contingent valuation studies, the authors of the present study found evidence that willingness-to-accept exceeds willingness-to-pay for coverage of the same benefit. CONCLUSIONS: An insurance-based contingent valuation study is feasible in a health maintenance organization. Construct validation evidence was encouraging, with the exception of the test for prior risk of cancer; however, this was a between-person contrast and may have been confounded by other factors.