[Cystic fibrosis in Morroco : what do words mean without action ?] / Mucoviscidose au Maroc : que valent les mots sans les actes ?

It is estimated that in highly medicalised countries, median life expectancy for most newborns with cystic fibrosis now exceeds 70 years, approaching that of the general population. However, socio-economic disparities between countries continue to have a devastating impact on the prognosis of patients in Eastern Europe, Africa, India and South America. In Morocco, very limited genetic data suggest that the prevalence of this disease is at least of the same order as in Belgium. But as it is not really recognised by the national health system, patients are denied access even to symptomatic treatment. As a result, their outcome is tragic, similar to what it was 60 years ago in the most medicalised countries. A pilot project for a first paediatric reference centre in Casablanca is currently being set up. If properly resourced, this project can only be a success and should be the first step on the road towards cystic fibrosis care in this country. In a very humble way, several Belgian stakeholders are trying to support this project.

Dans les pays les plus médicalisés, l'espérance de vie médiane de la plupart des nouveau-nés atteints de mucoviscidose excède aujourd'hui 70 ans et se rapproche de celle de la population générale. Ailleurs, en Europe de l'Est comme en Afrique, en Inde ou en Amérique du Sud, les disparités socio-économiques des pays continuent à impacter très durement le pronostic des patients. Au Maroc, des données génétiques très fragmentaires suggèrent que la prévalence de la mucoviscidose est au moins du même ordre qu'en Belgique. Mais la maladie n'y est pas réellement reconnue par le système de santé, de telle sorte que même le traitement symptomatique reste inaccessible aux patients et leur pronostic est tragique, similaire à ce qu'il était il y a 60 ans dans les pays les plus médicalisés. À Casablanca, le projet pilote d'un premier Centre pédiatrique de Référence est en train de se mettre en place. S'il bénéficie d'un support adéquat, ce projet ne peut être qu'un succès et doit constituer un tout premier pas sur le chemin vers une prise en charge des patients dans ce pays. Très modestement, plusieurs intervenants belges tentent d'y apporter leur soutien.

Impact of PEGylation on the mucolytic activity of recombinant human deoxyribonuclease I in cystic fibrosis sputum.

Highly viscous mucus and its impaired clearance characterize the lungs of patients with cystic fibrosis (CF). Pulmonary secretions of patients with CF display increased concentrations of high molecular weight components such as DNA and actin. Recombinant human deoxyribonuclease I (rhDNase) delivered by inhalation cleaves DNA filaments contained in respiratory secretions and thins them. However, rapid clearance of rhDNase from the lungs implies a daily administration and thereby a high therapy burden and a reduced patient compliance. A PEGylated version of rhDNase could sustain the presence of the protein within the lungs and reduce its administration frequency. Here, we evaluated the enzymatic activity of rhDNase conjugated to a two-arm 40 kDa polyethylene glycol (PEG40) in CF sputa. Rheology data indicated that both rhDNase and PEG40-rhDNase presented similar mucolytic activity in CF sputa, independently of the purulence of the sputum samples as well as of their DNA, actin and ions contents. The macroscopic appearance of the samples correlated with the DNA content of the sputa: the more purulent the sample, the higher the DNA concentration. Finally, quantification of the enzymes in CF sputa following rheology measurement suggests that PEGylation largely increases the stability of rhDNase in CF respiratory secretions, since 24-fold more PEG40-rhDNase than rhDNase was recovered from the samples. The present results are considered positive and provide support to the continuation of the research on a long acting version of rhDNase to treat CF lung disease.

Short-Term Effect of Autogenic Drainage on Ventilation Inhomogeneity in Adult Subjects With Stable Non-Cystic Fibrosis Bronchiectasis.

BACKGROUND: Lung clearance index (LCI), a measure of ventilation inhomogeneity derived from a multiple-breath washout test, is a promising tool for assessing airway function in patients with non-cystic fibrosis bronchiectasis. However, it is unknown whether ventilation inhomogeneity could improve after successful elimination of excessive secretions within bronchiectasis. The objective of this work was to assess the short-term effects of lung secretion clearance using the autogenic drainage technique on standard lung function tests and LCI in subjects with non-cystic fibrosis bronchiectasis. METHODS: Nitrogen-based multiple-breath washout, spirometry, and body plethysmography tests were performed 30 min before autogenic drainage in adults with stable non-cystic fibrosis bronchiectasis. The autogenic drainage session was followed by a 5-min break, after which the tests were repeated in the same order. Sputum expectorated during autogenic drainage was quantified as dry weight and correlated with change between post- and pre-measurements (Δ). Paired t test or Wilcoxon signed-rank tests were used to compare pre- and post-autogenic drainage measurement outcomes. A P value of ≤.05 was considered as statistically significant. RESULTS: Twenty-four subjects were studied (18 females, median age [range]: 65 [21-81] y). Mean ± SD LCI significantly improved after autogenic drainage (10.88 ± 2.62 vs 10.53 ± 2.35, P = .042). However, only 20% of subjects with mucus hyperproduction during autogenic drainage had a ΔLCI that exceeded measurement variability. The percent of predicted slow vital capacity (SVC%) also slightly improved (88.7 ± 19.3% vs 90 ± 19.1%, P = .02). ΔLCI was inversely related to dry sputum weight (r = -.48, P = .02) and ΔSVC% (r = -.64, P = .001). ΔSVC% also correlated with dry sputum weight (r = 0.46, P = .02). CONCLUSIONS: In adults with non-cystic fibrosis bronchiectasis and mucus hypersecretion, autogenic drainage improved ventilation inhomogeneity. LCI change may be the result of the maximum recruited lung volume and the amount of cleared mucus secretion. (ClinicalTrials.gov registration NCT02411981.).

Objective study of sensor relevance for automatic cough detection.

The development of a system for the automatic, objective, and reliable detection of cough events is a need underlined by the medical literature for years. The benefit of such a tool is clear as it would allow the assessment of pathology severity in chronic cough diseases. Even though some approaches have recently reported solutions achieving this task with a relative success, there is still no standardization about the method to adopt or the sensors to use. The goal of this paper is to study objectively the performance of several sensors for cough detection: ECG, thermistor, chest belt, accelerometer, contact, and audio microphones. Experiments are carried out on a database of 32 healthy subjects producing, in a confined room and in three situations, voluntary cough at various volumes as well as other event categories which can possibly lead to some detection errors: background noise, forced expiration, throat clearing, speech, and laugh. The relevance of each sensor is evaluated at three stages: mutual information conveyed by the features, ability to discriminate at the frame level cough from these latter other sources of ambiguity, and ability to detect cough events. In this latter experiment, with both an averaged sensitivity and specificity of about 94.5%, the proposed approach is shown to clearly outperform the commercial Karmelsonix system which achieved a specificity of 95.3% and a sensitivity of 64.9%.