Table of Contents

Fabry disease is a rare, X-linked lysosomal storage disorder that can cause early death from renal, cardiac, and cerebrovascular involvement. In this double-blind multicenter trial, 82 adults with kidney dysfunction from Fabry disease were randomly assigned to bimonthly infusions of enzyme replacement with agalsidase beta or placebo for up to 35 months. Agalsidase beta reduced the frequency of and time to renal, cardiac, and cerebrovascular events and death and caused infusion reactions more often than placebo.

The expected survival of HIV-infected patients has been difficult to measure by comparing selected HIV samples and control groups. However, because Denmark carefully tracks each of its residents' vital status, it is possible to accurately compare survival of HIV-infected persons and uninfected persons. In 2000 to 2005, life expectancy at 25 years of age was 51 years in the general population and 39 years for HIV-infected persons without hepatitis C virus infection. The corresponding mortality rate ratio (relative to the general population) was 11.5.

Early studies showed that benzodiazepine use was associated with increased hip fracture rates. New York has since adopted legislation to control overprescribing of benzodiazepines; New Jersey has not. The authors observed benzodiazepine prescribing and hip fractures in Medicaid patients for 12 months before and 21 months after New York began to track physicians' benzodiazepine prescribing rates. Prescribing rates fell abruptly by 60%, but hip fracture rates did not change. In New Jersey, concurrent rates did not change.

It is often unclear how the quality of evidence underlying decision analytic models affects the precision and accuracy of their results. Braithwaite and colleagues sought to test the feasibility of an approach to incorporate quality of evidence into decision analytic modeling. They created a simple 10-parameter probabilistic Markov model to estimate the cost-effectiveness of directly observed therapy for persons with recently diagnosed HIV infection. They concluded that the accuracy and precision of decision model results may vary dramatically depending on the quality of the evidence used in the model.

This year's Update in Neurology includes discussions of stroke, diabetic neuropathy, cancer neurology, headache, and multiple sclerosis. Changes to clinical practice emerging from these articles are also discussed.

Human growth hormone is widely sold and used as an antiaging drug, although its use for this purpose is controversial and has not been approved by the U.S. Food and Drug Administration. In this study, the researchers reviewed all clinical trials of growth hormone to determine whether it is safe and effective in healthy elderly persons. They found that human growth hormone had no important effects on body composition but led to frequent adverse effects, most notably soft tissue edema and arthralgias.

Patients with low back pain often request pain medication, and many physicians prescribe opioids despite concerns about drug dependence. This study found that opioid prescribing rates in 11 studies varied widely (3% to 66%). In 4 short-term randomized trials, pain relief was similar with opioids and either active treatment or placebo. In several poor-quality, heterogeneous studies, the prevalence of substance abuse disorders in patients taking long-term opioids for back pain varied from 5% to 24%.

In this issue, Banikazemi and colleagues' landmark trial on Fabry disease provides a crucial path toward lowering the morbidity of this disease. Among other findings, they determined that hemizygous male patients with the classic form of Fabry disease and symptomatic patients with milder variants should receive enzyme replacement therapy, with the particular goal of preserving renal function.

“But you're a doctor.” They don't say it, but it's in their eyes. My office manager tells me she doesn't understand it. I tell her they don't start off by hitting you.

He sits there, a skeletal silhouette, his body reminiscent of a Holocaust victim, his eyes closed halfway to death. I want to tell him that everything is okay, everything will be fine. But it isn't, and it it won't be. At least not in the mortal confines of his earthly body.