Considerations in the design of clinical trials for multibacillary leprosy treatment

Abstract

In 1991, the World Health Assembly adopted a resolution to attain the goal of global elimination of leprosy as a public health problem by the year 2000, based on universal access to multidrug therapy established by the WHO. The WHO multidrug therapy recommendation was not based on clinical trial results, but was based on all scientific knowledge available at the time and incorporated some financial constraints, such as the impossibility of daily rifampin use. Further decisions regarding treatment duration were needed to allow the World Health Assembly resolution target to be reached, which was set without a deep epidemiological evaluation. At present there are many uncertainties about leprosy treatment, including for instance, its ideal duration including decisions concerning daily or intermittent use of drugs, the use of immune modulators with antibacterial drugs as prophylaxis, and treatment of reactions during and after chemotherapy. To advance our knowledge of leprosy treatment, knowledge revision of the available research would be the first step to develop comprehensive Bayesian clinical trials designed to shed light on these uncertainties.

Uniform multidrug therapy for leprosy patients in Brazil (U-MDT/CT-BR): Results of an open label, randomized and controlled clinical trial, among multibacillary patients

“…e ., prevention and effective treatment of reactions, with effective prevention of further neurological damage after diagnosis. Clinical trials, including those with a Bayesian design [ 19 ], should address the main triggers of disability progression rate.…”