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Severe muscular dystrophy (MD) has historically led to death in early adulthood, due to mainly cardiopulmonary complications. However, with newer methods of cardiac and respiratory management, survival has improved, and patients with MD are more frequently undergoing procedures requiring deep sedation or anesthesia. Respiratory management of these patients during procedures is challenging; safe and effective options for respiratory support are needed. In this report, we describe our experience using the laryngeal mask airway (LMA) to provide respiratory support during deep sedation or anesthesia for eight patients with severe MD during the following medical procedures: eight percutaneous endoscopic gastrostomy (PEG) placements, three lithotripsies, and placement of an implantable cardioverter-defibrillator. We also review the benefits and risks of the LMA in the context of other respiratory support options for people with MD, and the integral role of non-invasive positive pressure ventilation (NPPV) during induction of and recovery from deep sedation or general anesthesia.

We review the anesthetic implications of Joubert syndrome and report that spinal anesthesia under intravenous propofol sedation proved satisfactory for repair of an inguinal hernia in a spontaneously ventilating infant with this syndrome. We caution that anatomical peculiarities may complicate performance of a caudal epidural block and suggest consideration of the use of caffeine to ameliorate the apneic episodes seen in early infancy in this syndrome.

Background This phase I study investigated the maximum tolerated dose (MTD), safety, pharmacokinetics and antitumor activity of ganetespib in patients with solid malignancies. Methods Patients were enrolled in cohorts of escalating ganetespib doses, given as 1 hour IV infusion, once weekly for 3 weeks, followed by a 1-week rest until disease progression or unacceptable toxicity. Endpoints included safety, pharmacokinetic and pharmacodynamic parameters and preliminary clinical activity. Results Fifty-three patients were treated at doses escalating from 7 to 259 mg/m2. The most common adverse events were Grade 1 and 2 diarrhea, fatigue, nausea or vomiting. Dose-limiting toxicities (DLT) observed were: one Grade 3 amylase elevation (150 mg/m2), one Grade 3 diarrhea and one Grade 3 and one Grade 4 asthenia (259 mg/m2). The MTD was 216 mg/m2 and the recommended phase 2 dose was established at 200 mg/m2 given IV at Days 1, 8, and 15 every 4 weeks. There was a linear relationship between dose and exposure. Plasma HSP70 protein levels remained elevated for over a week post treatment. Disease control rate (objective response and stable disease at ≥ 16 weeks) was 24.4%. Conclusions Ganetespib is well tolerated as a weekly infusion for 3 of every 4 weeks cycle. The recommended phase II dose is 200 mg/m2, and is associated with an acceptable tolerability profile. Trial registration NCT00687934

Individuals with Duchenne muscular dystrophy may benefit from gastrostomy tube feeding due to progressive dysphagia and malnutrition. However, due to their severely impaired pulmonary function, these individuals are at risk of severe complications when they are sedated or undergo anesthesia for the procedure. We previously described a technique of noninvasive positive pressure ventilation to provide respiratory support during gastrostomy tube placement in such patients, but this technique had risks and limitations. In this case report, we examine two alternative techniques we used to provide respiratory support successfully to patients with severe muscular dystrophy and malnutrition who underwent percutaneous endoscopic gastrostomy tube placement. We then review the literature and discuss the potential benefits, risks, and limitations of the above techniques and of other options for gastrostomy placement in people with severe muscular dystrophy.

In medical practice, antibiotics are generally given empirically for the treatment of acute exacerbations of chronic bronchitis (AECB). To be effective, antibiotic therapy should be broad in spectrum, and it should also cover the common beta-lactamase—producing pathogens. In this multicenter, randomized, investigator-masked study, 469 patients with AECB were randomized (in a ratio of 2:1) to receive 400-mg oral ceftibuten capsules once daily or 500-mg amoxicillin-clavulanate tablets three times daily for 5 to 15 days. Patients receiving ceftibuten were further divided into those who took the capsule with a meal (fed) and those who took the capsule 1 hour before a meal (fasted). Clinical and microbiologic responses were evaluated after treatment at 0 to 6 days (end of treatment) and 7 to 21 days (follow-up). Overall clinical success was determined by cure/improvement of signs and symptoms of AECB at the end of treatment and at follow-up. Overall microbiologic assessment was graded as eradication, persistence, relapse, reinfection, colonization, superinfection, or unassessable. Tolerability was evaluated by grading observed adverse events. The mean duration of treatment was 10.4 days for patients who received ceftibuten and 10.1 days for patients who received amoxicillin-clavulanate. A total of 252 patients receiving ceftibuten and 117 patients receiving amoxicillin-clavulanate were evaluable for clinical efficacy, and 55 patients were evaluable for microbiologic response. Both treatments improved the signs and symptoms of bronchitis, and overall clinical success rates were equivalent for patients treated with ceftibuten (211 of 252 [84%]) and amoxicillin-clavulanate (93 of 117 [79%]) (95% confidence interval [CI], −4.5% to 13.6%). Overall microbiologic eradication rates were also similar for patients treated with ceftibuten (36 of 37 [97%]) and amoxicillin-clavulanate (12 of 14 [86%]) (95% CI, −5.2% to 21.2%). The most frequently reported treatment-related adverse events were gastrointestinal disturbances, which occurred in 15% (47 of 316) and 24% (36 of 152) of patients treated with ceftibuten and amoxicillin-clavulanate, respectively. No significant difference was observed in the ceftibuten-fed and ceftibuten-fasted groups in overall clinical assessments of the clinical efficacy population and safety population. In conclusion, 400 mg oral ceftibuten once daily has a similar clinical success rate to 500 mg amoxicillin-clavulanate three times daily, with a trend toward fewer gastrointestinal side effects, in the treatment of patients with AECB.

The role of percutaneous angioplasty in the management of chronic iliac artery occlusions is controversial. This article reviews 7 years of experience in treating patients with complete chronic iliac artery occlusions ( n = 99) by using thrombolysis and angioplasty. Patients were not excluded due to age or length of the lesion, or severity of underlying peripheral vascular disease. Thrombolysis was conducted with infusion of urokinase at a rate of 60,000–80,000 IU/h for 24 hours. Angioplasty was then performed irrespective of the results of thrombolysis. Seventy-nine percent of patients responded to urokinase, with complete lysis achieved in 55%. Angioplasty was successful in all patients with complete thrombolysis and in 88% of those with partial thrombolysis. The overall success rate was 86%, and more than 80% of surviving patients were symptom free at 5 years. There were seven complications, and the 30-day mortality rate was 2%. Our present results confirm the efficacy of thrombolysis/angioplasty for chronic arterial occlusions.

A 6-month-old infant presenting with pericardial effusion was found to have a malignant rhabdoid tumor of the heart. As visualized by two-dimensional echocardiography and subsequently by contrast-enhanced computerized axial tomographic scanning, the tumor arose from the left ventricular free wall and grew into the pericardial space. Despite two courses of single-agent chemotherapy with doxorubicin, the child died 3 months later. This is the first reported case of malignant rhabdoid tumor of the heart, and the fifth case of a primary cardiac malignancy in an infant.

Intracranial volume-pressure response was assessed in 6 children suffering from metabolic coma and ranging in age 2 months-13 years. No untoward pressure or infectious complications occurred. The relationship between baseline mean intracranial pressure (MICP) and volume-pressure response assessment (VPRA) in these patients seems to be exponential rather than linear. By itself, the test is no better than baseline MICP at identifying patients at greatest risk of developing significantly increased intracranial pressure (ICP). When used in conjunction with baseline MICP, this method of VPRA identifies a population with an 80% risk of developing serious ICP elevations within a 4 h time period. Patients with best overall prognosis had significantly lower mean VPRA values than those with poorest overall prognosis.