Pentoxifylline as a rescue treatment for DMD: a randomized double-blind clinical trial.

OBJECTIVE: To determine whether pentoxifylline (PTX) slows the decline of muscle strength and function in ambulatory boys with Duchenne muscular dystrophy (DMD). METHODS: This was a multicenter, randomized, double-blinded, controlled trial comparing 12 months of daily treatment with PTX or placebo in corticosteroid-treated boys with DMD using a slow-release PTX formulation (~20 mg/kg/day). The primary outcome was the change in mean total quantitative muscle testing (QMT) score. Secondary outcomes included changes in QMT subscales, manual muscle strength, pulmonary function, and timed function tests. Outcomes were compared using Student t tests and a linear mixed-effects model. Adverse events (AEs) were compared using the Fisher exact test. RESULTS: A total of 64 boys with DMD with a mean age of 9.9 ± 2.9 years were randomly assigned to PTX or placebo in 11 participating Cooperative International Neuromuscular Research Group centers. There was no significant difference between PTX and the placebo group in total QMT scores (p = 0.14) or in most of the secondary outcomes after a 12-month treatment. The use of PTX was associated with mild to moderate gastrointestinal or hematologic AEs. CONCLUSION: The addition of PTX to corticosteroid-treated boys with DMD at a moderate to late ambulatory stage of disease did not improve or halt the deterioration of muscle strength and function over a 12-month study period. CLASSIFICATION OF EVIDENCE: This study provides Class I evidence that treatment with PTX does not prevent deterioration in muscle function or strength in corticosteroid-treated boys with DMD.

Video-assisted thoracoscopic thymectomy in juvenile myasthenia gravis.

As video-assisted thoracoscopic surgery for thymectomy has been reported to be as effective as traditional open surgical approaches in predominantly adult patients with myasthenia gravis, we applied this procedure to juvenile patients with this condition. Six patients underwent total thymectomy using the video-assisted technique (1997-98). Six patients in whom a median transsternal approach was used (1989-95) formed the comparison group. The two patient groups were similar in terms of age at thymectomy and preoperative clinical severity. There were no serious perioperative complications in either group. Minimum post-thymectomy duration of follow-up in the video-assisted thoracoscopic surgery patients was 2.3 years (mean 2.7 years), with all patients clinically improved over their baseline status. Preliminary results suggest that video-assisted thymectomies are comparably effective to transsternal procedures in treating generalized juvenile myasthenia gravis and can be safely performed in children as young as 20 months of age. In addition, video-assisted surgeries are less invasive than transsternal approaches, significantly shorten the postoperative hospital stay, and have superior cosmetic results.

Thoracoscopic thymectomy in juvenile myasthenia gravis.

BACKGROUND: Although transsternal thymectomy is an effective method in the treatment of juvenile myasthenia gravis (JMG) it is traumatic in pediatric patients. Thoracoscopic thymectomy offers an effective and less traumatic approach with respect to cosmesis and postoperative recovery. METHODS: A retrospective analysis of 6 consecutive patients treated with thoracoscopic thymectomy was performed. Perioperative parameters and cost analysis were compared with those of 6 consecutive open procedures performed before the study. RESULTS: Thoracoscopic thymectomy can be performed in patients as young as 1.6 years. There was no conversion to open procedure and no perioperative morbidity and mortality. The length of operating time and the surgical cost of thoracoscopic procedure were not significantly different from those of open procedure. The length of hospitalization, however, was significantly shorter with thoracoscopic procedure, and hence the overall cost was significantly reduced (P < .05). An intermediate follow-up shows that outcome after thoracoscopic procedure is equally as effective as open procedure. CONCLUSIONS: Thoracoscopic thymectomy offers an equally effective but cosmetically more acceptable approach than sternotomy. It has a quicker recovery period and appears to be a less costly alternative to transsternal thymectomy.

Etiology of acute childhood encephalitis at The Hospital for Sick Children, Toronto, 1994-1995.

Of 145 patients admitted to our hospital because of encephalitis-like illness, 50 patients hospitalized for > or =72 hours underwent standardized microbiological investigations. A confirmed or probable etiologic agent was identified in 20 cases (40%), including Mycoplasma pneumoniae (9 cases). M. pneumoniae and enterovirus (2), herpes simplex virus (4), Epstein-Barr virus (1), human herpes-virus 6 (HHV-6) (1), HHV-6 and influenza virus type A (1), influenza virus type A (1), and Powassan virus (1). In 13 cases (26%), a possible pathogen was identified, including M. pneumoniae in nine cases. Presenting features included fever (80% of patients), seizures (78%), focal neurological findings (78%), and decreased consciousness (47%). The frequency of findings at the time of admission vs. later in hospitalization was as follows: pleocytosis, 59% vs. 63%; electroencephalogram abnormalities, 87% vs. 96%; and neuroimaging abnormalities, 37% vs. 69%, respectively. The outcomes at the time of discharge were as follows: normal results of physical examination, 32% (16) of the patients; death, 2% (1); motor difficulties, 26% (13); global neurological deficits, 16% (severe, 6; mild, 2); mental status changes, 14% (7); visual defects, 8% (4); and hearing impairment, 2% (1).

[Randomized ergometric study of vasodilators combined with betablockaders]. / Etude ergométrique randomisée des vasodilatateurs associés aux bêtabloqueurs.

The ergometric effects of different vasodilator drugs in 5 series of 10 patients with stable angina and persistent effort ischaemia despite beta-offckade, were compared two by two in a random, single blind cross-over study under basal conditions on betablocker therapy and at the peak of their action, the second measurement being performed after a 2 to 7 day interval. The principal criteria of assessment were the work required to induce 1 mm ST depression (WST1), and the maximum ST depression (ST max) at comparable work loads. Molsidomine (2 mg), Risordan 20 mg) and Nifedipine (10 mg) significantly improved both parameters (p less than 0.001). Lenitral (7.5 mg), Langoran (40 mg), Trinitrin skin patch (10 mg) did not produce a significant improvement. Corditrine improved WST1 (p less than 0.05) and slow release Trinitrin (2.5 and 5 mg) improved WST1 at 3 hours (p less than 0.05) and ST max at 15 minutes (p less than 0.001) and 3 hours (p less than 0.05). The fall in resting blood pressure was parallel to the ergometric changes. These results suggest that Molsidomine, Nifedipine, Risordan and slow release Trinitrin (2.5 mg) are the most effective vasodilators when used in association with betablockers.

[Long-term clinical and hemodynamic results of the treatment of refractory cardiac failure with molsidomine]. / Résultats à long terme, cliniques et hémodynamiques, du traitement de l'insuffisance cardiaque réfractaire par la molsidomine.

Molsidomine, one of the sydnonimine group of drugs; the object of this study was to evaluate its efforts in refractory cardiac failure. In the first part of the study, the haemodynamic effects of a single oral dose of 2 or 4 mg of molsidomine were compared with placebo controls in 23 patients. This showed molsidomine to be an active venous vasodilator reducing pulmonary artery and right atrial pressures without changing cardiac index or systemic pressures. The peak effect was observed after 1 to 1,5 hours. In the second phase, molsidomine was used in 9 patients aged 32 to 71 years (mean 47 +/- 12 years) over an average period of 19 months (3,5 to 42 months). The maintenance dose varied from 8 to 24 mg/24 hours. These patients had refractory cardiac failure secondary to primary cardiomyopathy with dilatation (6 cases) or ischemic heart disease (3 cases). The 9 patients were in functional classes IV (5 cases) or III (4 cases). Four patients were theoretically good indications for transplantation. Haemodynamic control was performed 1,8 +/- 5 months after a washout period of 8 hours, and after initial right heart catheterisation, the measurements were repeated 1 hour after oral administration of a 4 mg dose of molsidomine. Two patients did not respond initially to molsidomine; one died, the other remained in functional Class III. Another patient who responded initially was improved for over two years but died in cardiac failure after 42 months' treatment. The other six patients have been significantly improved and were in functional Class II at their last control.(ABSTRACT TRUNCATED AT 250 WORDS)