Effectiveness of Nifurtimox Eflornithine Combination Therapy (NECT) in T. b. gambiense second stage sleeping sickness patients in the Democratic Republic of Congo: Report from a field study

<h4>Background</h4> Nifurtimox-eflornithine combination therapy (NECT) for the treatment of second stage gambiense human African trypanosomiasis (HAT) was added to the World Health Organization’s Essential Medicines List in 2009 after demonstration of its non-inferior efficacy compared t...

Descripción completa

Guardado en:

Detalles Bibliográficos
Autores principales:	Andrea Kuemmerle, Caecilia Schmid, Sonja Bernhard, Victor Kande, Wilfried Mutombo, Medard Ilunga, Ismael Lumpungu, Sylvain Mutanda, Pathou Nganzobo, Digas Ngolo Tete, Mays Kisala, Christian Burri, Severine Blesson, Olaf Valverde Mordt
Formato:	article
Lenguaje:	EN
Publicado:	Public Library of Science (PLoS) 2021
Materias:	Arctic medicine. Tropical medicine RC955-962 Public aspects of medicine RA1-1270
Acceso en línea:	https://doaj.org/article/97cd5f838f9b406bbc6d7a30a0545e1c
Etiquetas:	Agregar Etiqueta Sin Etiquetas, Sea el primero en etiquetar este registro!

id	oai:doaj.org-article:97cd5f838f9b406bbc6d7a30a0545e1c
record_format	dspace
institution	DOAJ
collection	DOAJ
language	EN
topic	Arctic medicine. Tropical medicine RC955-962 Public aspects of medicine RA1-1270
spellingShingle	Arctic medicine. Tropical medicine RC955-962 Public aspects of medicine RA1-1270 Andrea Kuemmerle Caecilia Schmid Sonja Bernhard Victor Kande Wilfried Mutombo Medard Ilunga Ismael Lumpungu Sylvain Mutanda Pathou Nganzobo Digas Ngolo Tete Mays Kisala Christian Burri Severine Blesson Olaf Valverde Mordt Effectiveness of Nifurtimox Eflornithine Combination Therapy (NECT) in T. b. gambiense second stage sleeping sickness patients in the Democratic Republic of Congo: Report from a field study
description	<h4>Background</h4> Nifurtimox-eflornithine combination therapy (NECT) for the treatment of second stage gambiense human African trypanosomiasis (HAT) was added to the World Health Organization’s Essential Medicines List in 2009 after demonstration of its non-inferior efficacy compared to eflornithine therapy. A study of NECT use in the field showed acceptable safety and high efficacy until hospital discharge in a wide population, including children, pregnant and breastfeeding women, and patients with a HAT treatment history. We present here the effectiveness results after the 24-month follow-up visit. <h4>Methodology/Principal findings</h4> In a multicenter, open label, single arm phase IIIb study, second stage gambiense HAT patients were treated with NECT in the Democratic Republic of Congo. Clinical cure was defined 24 months after treatment as survival without clinical and/or parasitological signs of HAT. Of the 629 included patients, 619 (98.4%) were discharged alive after treatment and were examined for the presence of trypanosomes, white blood cell count in cerebro-spinal fluid, and disease symptoms. The clinical cure rate of 94.1% was comparable for all subpopulations analyzed at the 24-month follow-up visit. Self-reported adverse events during follow-up were few and concerned mainly nervous system disorders, infections, and gastro-intestinal disorders. Overall, 28 patients (4.3%) died during the course of the trial. The death of 16 of the 18 patients who died during the follow-up period was assessed as unlikely or not related to NECT. Within 24 months, eight patients (1.3%) relapsed and received rescue treatment. Sixteen patients were completely lost to follow-up. <h4>Conclusions/Significance</h4> NECT treatment administered under field conditions was effective and sufficiently well tolerated, no major concern arose for children or pregnant or breastfeeding women. Patients with a previous HAT treatment history had the same response as those who were naïve. In conclusion, NECT was confirmed as effective and appropriate for use in a broad population, including vulnerable subpopulations. <h4>Trial registration</h4> The trial is registered at ClinicalTrials.gov, number NCT00906880. Author summary The advanced stage of the neglected tropical disease human African trypanosomiasis was, until relatively recently, treated with an old toxic arsenical drug and there was little investment in an improved treatment option. Eflornithine alone was efficacious, but difficult to administer as it required four two-hour infusions a day for 14 days. Nifurtimox-eflornithine combination therapy (NECT) was developed as a simplified and easier to use treatment and was shown to be effective and sufficiently well tolerated in a randomized clinical trial. The present study was conducted to assess the overall effectiveness, including the feasibility of implementation of NECT under field conditions in a wider population than in the randomized clinical trial. We found that NECT can be implemented under field conditions and in remote areas, with the necessary logistical support and staff training for treatment administration. Adverse events, although very frequent, were considered acceptable given the severity of the disease. Less than 10% of patients showed severe adverse events. Over 24 months, the case fatality rate was 4.5% and relapses were rare (1.3%). The effectiveness of NECT was confirmed in a broad spectrum of second stage gambiense HAT patients, including children, pregnant and breastfeeding women, and patients who had been previously treated for HAT.
format	article
author	Andrea Kuemmerle Caecilia Schmid Sonja Bernhard Victor Kande Wilfried Mutombo Medard Ilunga Ismael Lumpungu Sylvain Mutanda Pathou Nganzobo Digas Ngolo Tete Mays Kisala Christian Burri Severine Blesson Olaf Valverde Mordt
author_facet	Andrea Kuemmerle Caecilia Schmid Sonja Bernhard Victor Kande Wilfried Mutombo Medard Ilunga Ismael Lumpungu Sylvain Mutanda Pathou Nganzobo Digas Ngolo Tete Mays Kisala Christian Burri Severine Blesson Olaf Valverde Mordt
author_sort	Andrea Kuemmerle
title	Effectiveness of Nifurtimox Eflornithine Combination Therapy (NECT) in T. b. gambiense second stage sleeping sickness patients in the Democratic Republic of Congo: Report from a field study
title_short	Effectiveness of Nifurtimox Eflornithine Combination Therapy (NECT) in T. b. gambiense second stage sleeping sickness patients in the Democratic Republic of Congo: Report from a field study
title_full	Effectiveness of Nifurtimox Eflornithine Combination Therapy (NECT) in T. b. gambiense second stage sleeping sickness patients in the Democratic Republic of Congo: Report from a field study
title_fullStr	Effectiveness of Nifurtimox Eflornithine Combination Therapy (NECT) in T. b. gambiense second stage sleeping sickness patients in the Democratic Republic of Congo: Report from a field study
title_full_unstemmed	Effectiveness of Nifurtimox Eflornithine Combination Therapy (NECT) in T. b. gambiense second stage sleeping sickness patients in the Democratic Republic of Congo: Report from a field study
title_sort	effectiveness of nifurtimox eflornithine combination therapy (nect) in t. b. gambiense second stage sleeping sickness patients in the democratic republic of congo: report from a field study
publisher	Public Library of Science (PLoS)
publishDate	2021
url	https://doaj.org/article/97cd5f838f9b406bbc6d7a30a0545e1c
work_keys_str_mv	AT andreakuemmerle effectivenessofnifurtimoxeflornithinecombinationtherapynectintbgambiensesecondstagesleepingsicknesspatientsinthedemocraticrepublicofcongoreportfromafieldstudy AT caeciliaschmid effectivenessofnifurtimoxeflornithinecombinationtherapynectintbgambiensesecondstagesleepingsicknesspatientsinthedemocraticrepublicofcongoreportfromafieldstudy AT sonjabernhard effectivenessofnifurtimoxeflornithinecombinationtherapynectintbgambiensesecondstagesleepingsicknesspatientsinthedemocraticrepublicofcongoreportfromafieldstudy AT victorkande effectivenessofnifurtimoxeflornithinecombinationtherapynectintbgambiensesecondstagesleepingsicknesspatientsinthedemocraticrepublicofcongoreportfromafieldstudy AT wilfriedmutombo effectivenessofnifurtimoxeflornithinecombinationtherapynectintbgambiensesecondstagesleepingsicknesspatientsinthedemocraticrepublicofcongoreportfromafieldstudy AT medardilunga effectivenessofnifurtimoxeflornithinecombinationtherapynectintbgambiensesecondstagesleepingsicknesspatientsinthedemocraticrepublicofcongoreportfromafieldstudy AT ismaellumpungu effectivenessofnifurtimoxeflornithinecombinationtherapynectintbgambiensesecondstagesleepingsicknesspatientsinthedemocraticrepublicofcongoreportfromafieldstudy AT sylvainmutanda effectivenessofnifurtimoxeflornithinecombinationtherapynectintbgambiensesecondstagesleepingsicknesspatientsinthedemocraticrepublicofcongoreportfromafieldstudy AT pathounganzobo effectivenessofnifurtimoxeflornithinecombinationtherapynectintbgambiensesecondstagesleepingsicknesspatientsinthedemocraticrepublicofcongoreportfromafieldstudy AT digasngolotete effectivenessofnifurtimoxeflornithinecombinationtherapynectintbgambiensesecondstagesleepingsicknesspatientsinthedemocraticrepublicofcongoreportfromafieldstudy AT mayskisala effectivenessofnifurtimoxeflornithinecombinationtherapynectintbgambiensesecondstagesleepingsicknesspatientsinthedemocraticrepublicofcongoreportfromafieldstudy AT christianburri effectivenessofnifurtimoxeflornithinecombinationtherapynectintbgambiensesecondstagesleepingsicknesspatientsinthedemocraticrepublicofcongoreportfromafieldstudy AT severineblesson effectivenessofnifurtimoxeflornithinecombinationtherapynectintbgambiensesecondstagesleepingsicknesspatientsinthedemocraticrepublicofcongoreportfromafieldstudy AT olafvalverdemordt effectivenessofnifurtimoxeflornithinecombinationtherapynectintbgambiensesecondstagesleepingsicknesspatientsinthedemocraticrepublicofcongoreportfromafieldstudy
_version_	1718413696967901184
spelling	oai:doaj.org-article:97cd5f838f9b406bbc6d7a30a0545e1c2021-11-25T06:33:06ZEffectiveness of Nifurtimox Eflornithine Combination Therapy (NECT) in T. b. gambiense second stage sleeping sickness patients in the Democratic Republic of Congo: Report from a field study1935-27271935-2735https://doaj.org/article/97cd5f838f9b406bbc6d7a30a0545e1c2021-11-01T00:00:00Zhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC8601604/?tool=EBIhttps://doaj.org/toc/1935-2727https://doaj.org/toc/1935-2735<h4>Background</h4> Nifurtimox-eflornithine combination therapy (NECT) for the treatment of second stage gambiense human African trypanosomiasis (HAT) was added to the World Health Organization’s Essential Medicines List in 2009 after demonstration of its non-inferior efficacy compared to eflornithine therapy. A study of NECT use in the field showed acceptable safety and high efficacy until hospital discharge in a wide population, including children, pregnant and breastfeeding women, and patients with a HAT treatment history. We present here the effectiveness results after the 24-month follow-up visit. <h4>Methodology/Principal findings</h4> In a multicenter, open label, single arm phase IIIb study, second stage gambiense HAT patients were treated with NECT in the Democratic Republic of Congo. Clinical cure was defined 24 months after treatment as survival without clinical and/or parasitological signs of HAT. Of the 629 included patients, 619 (98.4%) were discharged alive after treatment and were examined for the presence of trypanosomes, white blood cell count in cerebro-spinal fluid, and disease symptoms. The clinical cure rate of 94.1% was comparable for all subpopulations analyzed at the 24-month follow-up visit. Self-reported adverse events during follow-up were few and concerned mainly nervous system disorders, infections, and gastro-intestinal disorders. Overall, 28 patients (4.3%) died during the course of the trial. The death of 16 of the 18 patients who died during the follow-up period was assessed as unlikely or not related to NECT. Within 24 months, eight patients (1.3%) relapsed and received rescue treatment. Sixteen patients were completely lost to follow-up. <h4>Conclusions/Significance</h4> NECT treatment administered under field conditions was effective and sufficiently well tolerated, no major concern arose for children or pregnant or breastfeeding women. Patients with a previous HAT treatment history had the same response as those who were naïve. In conclusion, NECT was confirmed as effective and appropriate for use in a broad population, including vulnerable subpopulations. <h4>Trial registration</h4> The trial is registered at ClinicalTrials.gov, number NCT00906880. Author summary The advanced stage of the neglected tropical disease human African trypanosomiasis was, until relatively recently, treated with an old toxic arsenical drug and there was little investment in an improved treatment option. Eflornithine alone was efficacious, but difficult to administer as it required four two-hour infusions a day for 14 days. Nifurtimox-eflornithine combination therapy (NECT) was developed as a simplified and easier to use treatment and was shown to be effective and sufficiently well tolerated in a randomized clinical trial. The present study was conducted to assess the overall effectiveness, including the feasibility of implementation of NECT under field conditions in a wider population than in the randomized clinical trial. We found that NECT can be implemented under field conditions and in remote areas, with the necessary logistical support and staff training for treatment administration. Adverse events, although very frequent, were considered acceptable given the severity of the disease. Less than 10% of patients showed severe adverse events. Over 24 months, the case fatality rate was 4.5% and relapses were rare (1.3%). The effectiveness of NECT was confirmed in a broad spectrum of second stage gambiense HAT patients, including children, pregnant and breastfeeding women, and patients who had been previously treated for HAT.Andrea KuemmerleCaecilia SchmidSonja BernhardVictor KandeWilfried MutomboMedard IlungaIsmael LumpunguSylvain MutandaPathou NganzoboDigas Ngolo TeteMays KisalaChristian BurriSeverine BlessonOlaf Valverde MordtPublic Library of Science (PLoS)articleArctic medicine. Tropical medicineRC955-962Public aspects of medicineRA1-1270ENPLoS Neglected Tropical Diseases, Vol 15, Iss 11 (2021)

Effectiveness of Nifurtimox Eflornithine Combination Therapy (NECT) in T. b. gambiense second stage sleeping sickness patients in the Democratic Republic of Congo: Report from a field study

Ejemplares similares