Diurnal ventilation via mouthpiece: survival in end-stage Duchenne patients

ABSTRACT: The present study aimed to assess the impact of diurnal mouthpiece intermittent positive pressure ventilation (MIPPV) as the extension of the nasal intermittent positive pressure ventilation (NIPPV) in Duchenne muscular dystrophy (DMD). In total, 42 DMD patients aged 15-33 yrs, normocapnic at night with NIPPV and receiving MIPPV since end-diurnal hypercapnia, were studied. Transcutaneous CO 2 tension (Pt,CO 2 ) was prospectively monitored at the end of the day, before and after MIPPV initiation. Vital capacity (VC), breathing pattern and maximal inspiratory strength were measured. Patients were asked to score the presence (1 point) or absence (0 point) of seven respiratory-linked symptoms before and after MIPPV establishment. Survival rates reached 88, 77, 58 and 51% after 1, 3, 5 and 7 yrs, respectively. The mean survival rate was 31 yrs. VC stabilised during 5 yrs with MIPPV. Symptom scores significantly decreased and Pt,CO 2 normalised during the day (8.17¡2.22 to 5.78¡0.73 kPa). No accident and minor sideeffects were observed in this 184 cumulated patient-yrs study. In conclusion, daytime mouthpiece ventilation is safe, prolongs survival and stabilises vital capacity in Duchenne muscular dystrophy patients. It is recommended on the condition that patients are equipped with a self-supporting harness

Lung volume restriction in patients with chronic respiratory muscle weakness: the role of microatelectasis

BACKGROUND--It is well established that patients with longstanding weakness of the respiratory muscles have a reduction in lung distensibility. Although this occurs in most patients without any radiographic changes suggesting parenchymal lung disease, it has been attributed to the development of microatelectasis. METHODS--A high resolution computed tomographic (CT) scanner was used in eight patients with traumatic tetraplegia and six patients with generalised neuromuscular disorders to look for areas of atelectasis. With the patient in the supine posture scans of 1 mm thickness were obtained at total lung capacity at intervals of 1 cm from the apex to the base of the lung. RESULTS--Vital capacity, total lung capacity, and inspiratory muscle strength were reduced to a mean of 59.5%, 73.9%, and 51.1% of predicted values, respectively. Static expiratory lung compliance was decreased in 12 of the 14 patients and averaged 69.1% of the predicted value. The CT scans revealed only small areas of atelectasis in one tetraplegic patient and in one patient with a generalised neuromuscular disorder; no parenchymal abnormality was seen in the other 12 patients. CONCLUSIONS--In many patients with chronic weakness of the respiratory muscles the reduced lung distensibility does not appear to be caused by microatelectasis. It might be related to alterations in elasticity of the lung tissue.Journal ArticleResearch Support, Non-U.S. Gov'tinfo:eu-repo/semantics/publishe

Reduced pulmonary compliance in patients with chronic respiratory muscle weakness: the role of microatelactasis

info:eu-repo/semantics/publishe

Clinical phase I and pharmacokinetic study of S 16020, a new olivacine derivative: report on three infusion schedules.

S 16020, a new 9-OH olivacine derivative, is a novel topoisomerase II inhibitor with activity in cell lines presenting the classical multidrug resistance phenotype. This report summarizes, in addition to pharmacokinetic data, the whole phase I clinical experience of S 16020 using three different infusion schedules. Asthenia and skin toxicity were the main side effects. In an attempt to understand the skin toxicity mechanism, experiments in animals were performed, the results of which are reported. S 16020 showed rapid tumor necrotizing activity in some patients, with soft tissue metastases of epidermoïd tumors and pain at the tumor site. To document the side effects of S 16020 and tumor site reactions (pain, edema, inflammatory signs), inflammatory parameters and some cytokines were measured. In our patients there was no hemolysis and no detection of anti-S 16020 antibodies, confirming the absence of immunogenicity of the compound. Based on the overall data of the three infusion schedules of S 16020, the dose of 100 mg/m(2) over 3 h every 3 weeks was selected for phase II studies.Clinical TrialClinical Trial, Phase IJournal ArticleMulticenter Studyinfo:eu-repo/semantics/publishe