Kantilal M. Patel

CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy

We tested the efficacy and safety of glutamine (0.6gm/kg/day) and creatine (5gm/day) in 50 ambulant boys with Duchenne muscular dystrophy in a 6‐month, double‐blind, placebo‐controlled clinical trial. Drug efficacy was tested by measuring muscle strength manually (34 muscle groups) and quantitatively (10 muscle groups). Timed functional tests, functional parameters, and pulmonary function tests were secondary outcome measures. Although there was no statistically significant effect of either therapy based on manual and quantitative measurements of muscle strength, a disease‐modifying effect of creatine in older Duchenne muscular dystrophy and creatine and glutamine in younger Duchenne muscular dystrophy cannot be excluded. Creatine and glutamine were well tolerated. Ann Neurol 2005;58:151–155

Parental Literacy Level and Understanding of Medical Information

Objective.u2003To ascertain the impact of literacy level on parents understanding of medical information and ability to follow therapy prescribed for their children. Design/Methods.u2003A prospective cohort of parents accompanying their children for acute care. Parents were interviewed about demographic status, their childs health, and use of pediatric preventive services. The Rapid Estimate of Adult Literacy in Medicine (REALM) test was used to assess parental literacy. The same parent was interviewed 48 to 96 hours later and asked to recall the childs diagnosis, any medication prescribed, and instructions. Results.u2003A total of 633 patients were enrolled. Follow-up was obtained in 543 patients (85.8%). Mean parental age was 32.43 years (SD = 9.07). Mean REALM score was 57.6 (SD = 10.9), corresponding to a 7th- to 8th-grade reading level, with a mean parental educational level of 13.43 years (SD = 2.09). Low REALM score was significantly correlated with young parental age and parental education. African-American race was associated with lower REALM scores. After controlling for these variables, REALM score significantly correlated with parental perception of how sick the child was, but not with use of preventive services, comprehension of diagnosis, medication name and instructions, or ability to obtain and administer prescribed medications. Conclusions.u2003Parental literacy level did not correlate with use of preventive services or parental understanding of or ability to follow medical instructions for their children.

Combining physician's subjective and physiology-based objective mortality risk predictions

ObjectiveNone of the currently available physiology-based mortality risk prediction models incorporate subjective judgements of healthcare professionals, a source of additional information that could improve predictor performance and make such systems more acceptable to healthcare professionals. Thi

Decision support issues using a physiology based score

Objective: As physiology based assessments of mortality risk become more accurate, their potential utility in clinical decision support and resource rationing decisions increases. Before these prediction models can be used, however, their performance must be statistically evaluated and interpreted in a clinical context. We examine the issues of confidence intervals (as estimates of survival ranges) and confidence levels (as estimates of clinical certainty) by applying Pediatric Risk of Mortality III (PRISM III) in two scenarios: (1) survival prediction for individual patients and (2) resource rationing. Design: A non-concurrent cohort study. Setting: 32 pediatric intensive care units (PICUs). Patients: 10 608 consecutive patients (571 deaths). Interventions: None. Measurements and results: For the individual patient application, we investigated the observed survival rates for patients with low survival predictions and the confidence intervals associated with these predictions. For the resource rationing application, we investigated the maximum error rate of a policy which would limit therapy for patients with scores exceeding a very high threshold. For both applications, we also investigated how the confidence intervals change as the confidence levels change. The observed survival in the PRISM III groups > 28, > 35, and > 42 were 6.3, 5.3, and 0 %, with 95 % upper confidence interval bounds of 10.5, 13.0, and 13.3 %, respectively. Changing the confidence level altered the survival range by more than 300 % in the highest risk group, indicating the importance of clinical certainty provisions in prognostic estimates. The maximum error rates for resource allocation decisions were low (e. g., 29 per 100 000 at a 95 % certainty level), equivalent to many of the risks of daily living. Changes in confidence level had relatively little effect on this result. Conclusions: Predictions for an individual patients risk of death with a high PRISM score are statistically not precise by virtue of the small number of patients in these groups and the resulting wide confidence intervals. Clinical certainty (confidence level) issues substantially influence outcome ranges for individual patients, directly affecting the utility of scores for individual patient use. However, sample sizes are sufficient for rationing decisions for many groups with higher certainty levels. Before there can be widespread acceptance of this type of decision support, physicians and families must confront what they believe is adequate certainty.

Certainty and mortality prediction in critically ill children

Objectives: The objective of this study is to investigate the relationship between a physician’s subjective mortality prediction and the level of confidence with which that mortality prediction is made. Design and participants: The study is a prospective cohort of patients less than 18 years of age admitted to a tertiary Paediatric Intensive Care Unit (ICU) at a University Children’s Hospital with a minimum length of ICU stay of 10 h. Paediatric ICU attending physicians and fellows provided mortality risk predictions and the level of confidence associated with these predictions on consecutive patients at the time of multidisciplinary rounds within 24 hours of admission to the paediatric ICU. Median confidence levels were compared across different ranges of mortality risk predictions. Results: Data were collected on 642 of 713 eligible patients (36 deaths, 5.6%). Mortality predictions greater than 5% and less than 95% were made with significantly less confidence than those predictions <5% and >95%. Experience was associated with greater confidence in prognostication. Conclusions: We conclude that a physician’s subjective mortality prediction may be dependent on the level of confidence in the prognosis; that is, a physician less confident in his or her prognosis is more likely to state an intermediate survival prediction. Measuring the level of confidence associated with mortality risk predictions (or any prognostic assessment) may therefore be important because different levels of confidence may translate into differences in a physician’s therapeutic plans and their assessment of the patient’s future.