Risk-adjusted monitoring of veno-occlusive disease following Bayesian individualization of busulfan dosage for bone marrow transplantation in paediatrics.

In order to assess the performance of Bayesian individualization of busulfan (BU) dosage regimens, veno-occlusive disease (VOD) rate was monitored for paediatric patients undergoing allogeneic bone marrow transplantation (BMT). Consecutive patients undergoing allogeneic BMT with BU as conditioning regimen during 5-years period (January 2000-February 2006) were reviewed. VOD was a major outcome variable. Preconditioning risk of VOD was estimated for each patient using a scoring system that included type of transplant, recipient CMV-positive status and total parenteral nutrition (TPN) provided pretransplantation. A risk-adjusted cumulative sum method was used to compare observed versus predicted outcome by assigning a risk score, based on log-likelihood ratios, to each patient. These cumulative scores were sequentially plotted with preset control limits for 'signalling' where results were substantially different than expected (doubling or halving of odds ratio). Sixty-six children received BMT after oral busulfan-based conditioning regimen with median age 3.9 years, 63.6% of male. Median preconditioning risk of VOD was 0.34 ranging from 0.23 to 0.84. Observed VOD rate was 16.7% (n = 11) which was 60.7% (17) fewer than the expected number estimated by the risk score. The resulting risk-adjusted score for each patient was plotted sequentially. This plot adopted early a negative slope, crossing the lower control limit twice, after 27 and 66 patients, indicating improved results compared to those expected. Bayesian individualization of oral busulfan dosage regimens is useful to reduce the VOD rate in children undergoing allogeneic BMT.

Asthmatic patients' poor awareness of inadequate disease control: a pharmacy-based survey.

BACKGROUND: Many asthmatic patients fail to perceive their level of disease control. OBJECTIVE: To investigate whether patients' ability to identify asthma control varied with personal characteristics or factors related to disease management. METHODS: Asthmatic patients were consecutively recruited at 348 pharmacies. They completed a questionnaire (regarding personal characteristics and asthma management) linked to pharmacies records of dispensed medications. The Asthma Control Test questionnaire includes 4 dimensions of asthma control (activity limitations, dyspnea, nocturnal awakenings, and rescue medication use) and assesses patients' perception of control ("How would you rate your asthma control during the past 14 days?"). Analyses were restricted to patients with inadequate control. Patients' perception of control was compared across the other dimensions of the questionnaire. The correlates of patients' failure to perceive inadequate asthma control were investigated. RESULTS: Seven hundred eighteen (68.5%) of the 1,048 patients with inadequate asthma control and documented perception of control considered themselves to be "completely" or "well" controlled. Patients' perception of control did not vary with each dimension of inadequate control. High rates of failure to perceive poor control were observed in patients with at least weekly dyspneas (60%) or nocturnal symptoms (60%). Failure to perceive inadequate control was more likely in patients aged 41 to 50 years (odds ratio, 1.51; 95% confidence interval, 1.05-2.15). No significant effect of factors related to asthma management was observed. CONCLUSIONS: Patients with most uncontrolled asthma have difficulty in properly perceiving their level of disease control regardless of their personal characteristics or disease management. The reasons for this poor perception should be investigated. Education programs should be created that focus on knowledge of asthma miscontrol criteria.