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Buruli ulcer (BU) is a cutaneous neglected tropical disease caused by Mycobacterium ulcerans. Synthesizing the evidence on their efficacy of antibiotic in the management of BU can help to better define their roles, identify weaknesses and inform clinicians on relevant measures than can be used to control BU. Our objectives is to assess the clinical efficacy of Rifampicin-Streptomycin given for 8 weeks of treatment of early M. ulcerans infection. We searched the following electronic databases from January 2005 to July 2012: Medline, EMBASE (Excerpta Medica Database), The Cochrane Library, Google Scholar, CINAHL (Cumulative Index to Nursing and Allied Health Literature), WHOLIS (World Health Organization Library Database), LILACS (Latin American and Caribbean Literature on Health Sciences) and contacted experts in the field. There were no restrictions to language or publication status. All study designs that could provide the information we sought for were eligible provided the studies were conducted in the third world. Critical appraisal of all identified citations was done independently by three authors to establish the possible relevance of the articles for inclusion in the review. Of the 115 studies, 09 papers met the inclusion criteria. The duration of treatment ranged from 8 to 48 weeks depending on the severity. Oral chemotherapy alone obtained a curative rate of 50%. The "dual" mode of treatment (surgery + chemotherapy) reduced hospital admission period from 90 to 39.8 days, thats to 44.2%. This treatment for early stages could therefore replace surgery and in severe cases, is an indispensable aid before surgery. These results confirmed that the daily administration of Rifampicin and Streptomycin is an effective treatment for M. ulcerans infection in an early stage. Subsequent systematic reviews should be conducted to determine if antibiotics could heal injuries without resorting to surgery and to compare different treatment durations.

Abstract: Fabry disease is a X-linked lysosomal storage disorder. Two preparations of the enzyme α-galactosidase A are available in Europe since 2001: agalsidase alpha and agalsidase beta. Clinical evidence of efficacy are mandatory considering the absence of a robust biomarker. A literature review was performed to assess the clinical efficacy of these two enzyme replacement therapies. Only open or randomised controlled trials were considered. No unflawed direct comparison exists between the two drugs. Significant clinical benefits have been demonstrated with enzyme replacement therapy (ERT), mainly at an early phase of the disease, with positive effects on heart, kidneys, pain, and quality of life. Further prospective studies are required to confirm the long term clinical benefits of ERT. More specific studies are also needed in women or with ERT earlier in the course of Fabry disease to assess prevention of organ damage. [Copyright &y& Elsevier]

Résumé La maladie de Fabry, maladie lysosomale liée à l’X, résulte d’un déficit enzymatique (α-galactosidase A). Deux traitements enzymatiques recombinants différents (algalsidase alpha, agalsidase bêta) sont disponibles depuis 2001, pour traiter une maladie qui touche principalement les hommes, mais aussi les femmes. L’enzymothérapie substitutive favorise la clairance cellulaire du susbtrat et améliore, comparativement au placebo, certains paramètres cliniques (cœur, atteinte rénale, douleurs, qualité de vie). Cependant, il n’y a pas d’efficacité démontrée à ce jour sur l’atteinte du système nerveux central, sur la morbimortalité cardiaque, ni même sur l’atteinte rénale au-delà d’un certain stade (protéinurie supérieure à 1 g/j, débit de filtration glomérulaire estimé inférieur à 60 mL/min/1,73 m 2 ). Nous évoquons dans cette revue le bénéfice potentiel d’une intervention précoce, les mesures protectrices vasculaires devant être associées à l’enzymothérapie et leur rationnel, ainsi que les traitements alternatifs en cours de développement, tels que les molécules chaperons et les inhibiteurs de substrat. Fabry disease, an X-linked lysosomal storage disease, results from α-galactosidase A deficiency. Two different recombinant enzyme treatments (algalsidase alpha agalsidase beta) have been available since 2001 to treat a disease that affects not only men but also women. Enzyme replacement therapy promotes cell clearance of susbtrate, and improves some clinical parameters (heart, kidney damage, pain, quality of life). However, there is no proven efficacy to date on central nervous system lesions, on cardiac morbidity and mortality, nor on renal damage beyond a certain stage (proteinuria > 1 g/day and/or estimated glomerular filtration rate < 60 mL/min/1.73 m 2 ). In this review, we discuss the potential benefit of an early intervention, the vascular protective measures to be associated with enzyme therapy and their rationale, and some alternative treatments under development, such as chaperones and substrate molecules inhibitors. [ABSTRACT FROM AUTHOR]

Fabry disease, an X-linked lysosomal storage disease, results from α-galactosidase A deficiency. Two different recombinant enzyme treatments (algalsidase alpha agalsidase beta) have been available since 2001 to treat a disease that affects not only men but also women. Enzyme replacement therapy promotes cell clearance of susbtrate, and improves some clinical parameters (heart, kidney damage, pain, quality of life). However, there is no proven efficacy to date on central nervous system lesions, on cardiac morbidity and mortality, nor on renal damage beyond a certain stage (proteinuria>1g/day and/or estimated glomerular filtration rate<60mL/min/1.73m(2)). In this review, we discuss the potential benefit of an early intervention, the vascular protective measures to be associated with enzyme therapy and their rationale, and some alternative treatments under development, such as chaperones and substrate molecules inhibitors., (Copyright © 2016 Association Société de néphrologie. Published by Elsevier SAS. All rights reserved.)