Sunday, November 8, 2020

Fw: (LML) Clinical Trials need clinical outcomes

 

 


Leprosy Mailing List – November 8,  2020

 

Ref.:  (LML) Clinical Trials need clinical outcomes 

 

From:  Diana Lockwood, London, UK


 

Dear Pieter,

 

We thank Dr David Scollard for this viewpoint (1) in Plos NTD discussing the different approaches taken by ourselves and Bhushan Kumar to evaluating new MDT regimens. (2) (3)   We agree that clinical trials are needed to identify new antimicrobial regimes for leprosy multi-drug therapy (MDT).  However, it is vital that these trials have clinical outcomes. The clinical course of leprosy means that relapses will occur after many years.

 

Large randomised controlled trials have been performed with long periods of follow up. The Brazilian Uniform-MDT trial with 613 participants with multibacillary (MB) leprosy had a median follow up time of 4.87 years. This trial yielded data on relapse and new molecular evidence suggests that a significant proportion of such cases were in fact due to reinfection. (4) (5)  The observational study from Bangladesh of 1612 MB patients treated with six month MB MDT by Butlin et al   had a mean duration of follow up of more than seven years. (6) 

 

We would welcome innovative outcome measures with the potential to reduce the follow up period for clinical trials of the efficacy of new MDT regimens, but these must have proven clinical validity. (7) 

 

The molecular assays to determine the viability of M. leprae are a mouse model and using these to screen drug regimens would provide useful but limited information. Scollard states that any newly proposed treatment regimens need to be evaluated in a "robust manner" and this would require clearly defined patient-centred clinical outcomes measured over an adequate period of time.  

 

The organisations that provide global leadership in leprosy and other stakeholders need to promote the identification of potential new treatment regimens and have the determination to test their safety and efficacy in well-designed clinical trials.  

 

 

Diana NJ Lockwood 

Stephen L Walker  

London School of Hygiene & Tropical Medicine, 

London WC1E 7HT 

 

1.      Scollard DM (2020) Leprosy treatment: Can we replace opinions with research? PLoS Negl Trop Dis 14(10): e0008636. https://doi.org/10.1371/journal.pntd.0008636 

 

2.      Lockwood DNJ, Lambert S, Srikantam A, Darlong J, Pai VV, Butlin CR, et al. 2020 Three drugs are unnecessary for treating paucibacillary leprosy-A critique of the WHO guidelines. PLoS Negl Trop Dis. 22019; 13: e0007671. https://doi.org/10.1371/journal.pntd.0007671 PMID: 31671087 

 

3.      Kumar B, Thakur V, Narang T, Dogra S. 2020 Response to "Three drugs are unnecessary for treating paucibacillary leprosy—A critique of the WHO guidelines". PLoS Negl Trop Dis. 14(6): e0008169. 3https://doi.org/10.1371/journal.pntd.0008169 PMID: 32497033 

 

4.      Penna GO, Bührer-Sékula S, Kerr LRS, Stefani MMA, Rodrigues LC, de Araújo MG, Ramos AMC, de Andrade ARC, Costa MB, Rosa PS, Gonçalves HS, Cruz R, Barreto ML, Pontes MAA, Penna MLF. 2017. 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. PLoS Negl Trop Dis 11(7):e0005725 

 

5.      Stefani MMA, Avanzi C, Bührer-Sékula S, Benjak A, Loiseau C, Singh P, Pontes MAA, Gonçalves HS, Hungria EM, Busso P, Piton J, Silveira MIS, Cruz R, Schetinni A, Costa MB, Virmond MCL, Diorio SM, Dias-Baptista IMF, Rosa PS, Matsuoka M, Penna MLF, Cole ST, Penna GO. 2017. Whole genome sequencing distinguishes between relapse and reinfection in recurrent leprosy cases. PLoS Negl Trop Dis 11(6):e0005598. 

 

6.      Butlin CR, Pahan D, Maug AKJ, Withington S, Nicholls P, Alam K, Salim MDAH. 2016 Outcomes of 6 months MB-MDT in MB patients in Bangladesh_preliminary results. Leprosy Rev 87(2) 171-182 

 

7.      Davis GL, Ray NA, Lahiri R, Gillis TP, Krahenbuhl JL, Williams DL, Adams LB. 2013. Molecular assays for determining Mycobacterium leprae viability in tissues of experimentally infected mice. PLoS Negl 

Trop Dis. 7:e2404. https://doi.org/10.1371/journal.pntd.0002404 PMID: 24179562 

 

 

Diana NJ Lockwood

Emeritus professor of Tropical Medicine

London School of Hygiene & Tropical Medicine

Keppel St

London WC1E 7HT

Visit my blog http://dnjtravels.blogspot.co.uk/

 


LML - S Deepak, B Naafs, S Noto and P Schreuder

LML blog link: http://leprosymailinglist.blogspot.it/

Contact: Dr Pieter Schreuder << editorlml@gmail.com

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