Prophylaxis versus Episodic Treatment to Prevent Joint Disease in Boys with Severe Hemophilia
This randomized trial involving young boys with severe hemophilia showed that prophylaxis with regular infusions of recombinant factor VIII was associated with clinically and statistically significant reductions in joint damage, as compared with episodic infusions at the time of a clinically evident hemarthrosis. Because of the high cost of recombinant factor VIII, its widespread use for prophylaxis may be impractical.
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Rituximab for the Treatment of Severe Pemphigus
In this study of 21 patients with severe pemphigus whose disease was refractory to or dependent on systemic corticosteroids or who had contraindications to corticosteroids, 18 patients (86%) had a complete remission after a single cycle of rituximab treatment. Two patients had serious infections, one of which resulted in death. The efficacy of rituximab for pemphigus must be weighed against the risk of severe adverse events.
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Complement C3 and Age-Related Macular Degeneration
A variant on complement factor 3 is associated with age-related macular degeneration, with a population attributable risk of 22%. This finding underlines the importance of complement activation in the pathogenesis of the disease.
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NXY-059 for Acute Ischemic Stroke
Two phase 3 clinical trials (SAINT I and SAINT II) evaluated the free-radical–trapping agent NXY-059 for the treatment of acute ischemic stroke. The SAINT I trial, reported last year, suggested that NXY-059 might be effective. The authors now report the results of the SAINT II trial, which clearly shows that NXY-059 is not effective for ischemic stroke. The discrepancy in the findings of the two trials is best explained by chance false positive findings in the SAINT I trial.
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Acute Ischemic Stroke
A 62-year-old man has sudden weakness of the left arm and leg and slurred speech. Except for untreated hypertension, his medical history is unremarkable. He is a current smoker with a smoking history of 45 pack-years. On arrival at the emergency department 1 hour 15 minutes after the onset of symptoms, he reports no headache or vomiting. His blood pressure is 180/100 mm Hg; his pulse is 76 beats per minute and is regular. How should this patient be evaluated and treated in the short term?
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Current Concepts: Drug-Induced Immune Thrombocytopenia
Drug-induced thrombocytopenia should be suspected in any patient with acute thrombocytopenia of unknown cause. Although the incidence is low, more than 100 drugs have been implicated in thrombocytopenia, including quinine, sulfonamides, abciximab, carbamazepine, and vancomycin, as well as herbal remedies and several nonprescription drugs. This review summarizes the current understanding of pathogenesis and provides a guide for diagnosis and management of this potentially dangerous disorder.
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A Pregnant Woman with Altered Mental Status
A 20-year-old pregnant woman was admitted at 26 weeks' gestation because of dizziness, confusion, and difficulty walking. Six weeks earlier, she was confused and had odd head movements. Four days before admission, she had dizziness and weakness; she began to fall to her left and vomited. On admission, examination of the cerebrospinal fluid showed a lymphocytic pleocytosis with mildly elevated protein and normal glucose levels. During the next 18 days, her condition worsened.
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Needing to Improve Care versus Knowing How to Do It
The authors argue that interventions to improve health care quality should be held to the same standards that we apply to medical treatments. They believe quality-improvement initiatives should not be widely disseminated unless studies have demonstrated that they are safe and effective.
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