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In-hospital safety in field conditions of nifurtimox eflornithine combination therapy (NECT) for T.b. gambiense sleeping sickness

Schmid, C. and Kümmerle, A. and Blum, J. and Ghabri, S. and Kande, V. and Mutombo, W. and Ilunga, M. and Lumpungu, I. and Mutanda, S. and Nganzobo, P. and Tete, D. and Mubwa, N. and Kisala, M. and Blésson, S. and Mordt, O. V.. (2012) In-hospital safety in field conditions of nifurtimox eflornithine combination therapy (NECT) for T.b. gambiense sleeping sickness. PLoS Neglected Tropical Diseases, 6 (11). e1920.

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Abstract

BACKGROUND: Trypanosoma brucei (T.b.) gambiense Human African trypanosomiasis (HAT; sleeping sickness) is a fatal disease. Until 2009, available treatments for 2(nd) stage HAT were complicated to use, expensive (eflornithine monotherapy), or toxic, and insufficiently effective in certain areas (melarsoprol). Recently, nifurtimox-eflornithine combination therapy (NECT) demonstrated good safety and efficacy in a randomised controlled trial (RCT) and was added to the World Health Organisation (WHO) essential medicines list (EML). Documentation of its safety profile in field conditions will support its wider use.METHODOLOGY: In a multicentre, open label, single arm, phase IIIb study of the use of NECT for 2(nd) stage T.b. gambiense HAT, all patients admitted to the trial centres who fulfilled inclusion criteria were treated with NECT. The primary outcome was the proportion of patients discharged alive from hospital. Safety was further assessed based on treatment emergent adverse events (AEs) occurring during hospitalisation.PRINCIPAL FINDINGS: 629 patients were treated in six HAT treatment facilities in the Democratic Republic of the Congo (DRC), including 100 children under 12, 14 pregnant and 33 breastfeeding women. The proportion of patients discharged alive after treatment completion was 98.4% (619/629; 95%CI [97.1%; 99.1%]). Of the 10 patients who died during hospitalisation, 8 presented in a bad or very bad health condition at baseline; one death was assessed as unlikely related to treatment. No major or unexpected safety concerns arose in any patient group. Most common AEs were gastro-intestinal (61%), general (46%), nervous system (mostly central; 34%) and metabolic disorders (26%). The overall safety profile was similar to previously published findings.CONCLUSIONS/SIGNIFICANCE: In field conditions and in a wider population, including children, NECT displayed a similar tolerability profile to that described in more stringent clinical trial conditions. The in-hospital safety was comparable to published results, and long term efficacy will be confirmed after 24 months follow-up. REGISTRATION: The trial is registered at ClinicalTrials.gov, number NCT00906880.
Faculties and Departments:09 Associated Institutions > Swiss Tropical and Public Health Institute (Swiss TPH) > Former Units within Swiss TPH > Medical Practice Föhre (Blum)
UniBasel Contributors:Blum, Johannes A. and Schmid, Christoph
Item Type:Article, refereed
Article Subtype:Research Article
Bibsysno:Link to catalogue
Publisher:Public Library of Science
ISSN:1935-2727
e-ISSN:1935-2735
Note:Publication type according to Uni Basel Research Database: Journal article
Language:English
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Last Modified:16 Feb 2018 13:52
Deposited On:19 Jul 2013 07:38

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