Reduced inotropic reserve is predictive of further degradation in left ventricular ejection fraction in patients with Duchenne muscular dystrophy.

AIMS: Duchenne muscular dystrophy (DMD), an inherited X-linked muscular disease, is associated with dilated cardiomyopathy that is responsible for death in 40% of patients. Our objective was to determine whether inotropic reserve is predictive of LV trend over time. METHODS AND RESULTS: A total of 69 DMD patients (age 12.2±2.3 years) were investigated. At baseline, LVEF and the presence of inotropic reserve (defined as an increase in LVEF >10% during dobutamine infusion) were investigated using radionuclide ventriculography. During follow-up (FU), LVEF was remeasured after a mean 29±19 months delay. In the whole population, mean LVEF was 58±8% at baseline and declined to 54±11% during FU (P =0.004). At baseline, 21 patients (30.4%) had LVEF <55% and 38 had no LV inotropic reserve. LVEF declined in the 38 patients (55.1%) without LV inotropic reserve (58±8% to 52±10%, P =0.001), and not in the other patients (58±8% to 57±11%, P =0.516) (P =0.042 for trends in LVEF between groups after adjustment for age, FU duration, and baseline LVEF). Fewer patients with vs. without inotropropic reserve at baseline show a depressed LVEF <55% during follow-up(35.5% vs. 63.2%, respectively, P =0.030). Similar findings were observed in the subgroups of patients with LVEF >45% or 55% at baseline. CONCLUSION: Inotropic reserve assessment allows the distinction of DMD patients who will vs. those who will not show a deterioration in LVEF, thus offering a sensitive approach for delineating the presence and progression of cardiovascular disease in these patients.

Upper limb strength and function changes during a one-year follow-up in non-ambulant patients with Duchenne Muscular Dystrophy: an observational multicenter trial.

INTRODUCTION: Upper limb evaluation of patients with Duchenne Muscular Dystrophy is crucially important to evaluations of efficacy of new treatments in non-ambulant patients. In patients who have lost ambulation, there are few validated and informative outcome measures. In addition, longitudinal data demonstrating sensitivity to clinical evolution of outcome measures over short-term periods are lacking. PATIENTS AND METHODS: We report here the results of a one-year multicenter study using specifically designed tools to assess grip, pinch strength, and hand function in wheelchair-bound patients. Our study assessed 53 non-ambulant patients with Duchenne muscular dystrophy aged 17.1 ± 4.8 years (range: 9 - 28.1 years). The average Brooke functional score of these patients was 4.6 ± 1.1. The average forced vital capacity was 44.5% predicted and 19 patients used non-invasive ventilation. Patients were assessed at baseline, 6 months, and one year using the Motor Function Measure and innovative devices (namely the MyoSet composed of MyoGrip, MyoPinch, and MoviPlate). RESULTS: Our study confirmed preliminary data previously reported regarding feasibility of use and of reliability of the MyoSet and the correlation at baseline between distal strength and clinical outcomes such as FVC, Brooke score, age, and duration since loss of ambulation. A significant correlation was observed between the distal upper limb strength and clinical variables. The sensitive dynamometers (MyoGrip and MyoPinch) and MoviPlate captured a 12-month change in non-ambulant Duchenne muscular dystrophy patients of all ages. TRIAL REGISTRATION: ClinicalTrials.gov NCT00993161 NCT00993161.

[Foot deformities in children]. / Podologie de l'enfant en pratique quotidienne.

The child comes in specialist consultation very often. Only after a good examination, we can quiet parents. Flatfoot, cavus foot toewalking can be alone symptom for a general disease. Then it is important to think to it and to do some others checkup. It may be necessary to have some opinion from different paediatric specialists like orthopaedic, neuroiogic, myologic. The follow-up of child allows to find a good evolution or some disease.

[Conservative treatment of clubfoot: the Functional Method and its long-term follow-up]. / Carpik ayagin konservatif tedavisi: Fonksiyonel Yöntem ve uzun dönemli izlem.

Despite the presence of a considerable number of papers published in the international literature, talipes equinovarus keeps its secrets. Conservative methods of treatment are currently accepted as advantageous over surgery. Prediction of the future of a clubfoot after treatment is not possible, for this reason, a long-term follow-up is needful. A true Functional Method based solely on gentle manipulations has been used for more than thirty years. Its aim is to correct the deformity and to allow the child to walk without delay. The principle of this method is to distract joint contractures prior to progressive correction of the deformity. No selection is made regarding the severity of the deformity and its etiology. Family plays an important role in performing routine manipulations at home. Concerning this Functional Method, three main series have been published by our team, with excellent-good results accounting for up to 77%. We believe that our Functional Method of conservative treatment of clubfoot is an appropriate way for correction of the deformity and its stabilization.

Functional treatment of clubfoot: a new series of 350 idiopathic clubfeet with long-term follow-up.

A new series of 350 previously unreported cases of clubfoot treated by the functional method are reported. One hundred and eighteen clubfeet were unilateral and 116 were bilateral. The follow-up ranged from 11 to 18 years, with an average of 14 years. The improvements in the technique of physical therapy increased the rate of good results up to 77% compared with our previously published series. In an effort to objectively compare these results with other series, the authors assessed all clubfeet using the "at birth classification" published in 1995. Outcome evaluation was performed when the patients were 6 years old, and final evaluation at the end of growth of the foot. This is the first major series to be assessed according to such objective criteria, as much before the inception of treatment as at the end of growth. The results of this new series have been compared with those obtained in our former series.