Treatment of visceral leishmaniasis patients outside clinical

trials: a case of the Leishmaniasis East Africa PlatformJoy Malongo1, Robert Kimutai1, Fabiana Alves2, Brima Musa3, Ermias Diro5, Mark Riongoita8, Patrick Sagaki9, Lawrence Okello9, Ahmed M. Mudawi3, Eltahir A.G. Khalil3, Asrat Hailu4, Jane Mbui7, Joseph Olobo6, Jorge Alvar2, Monique Wasunna1

1Drugs for Neglected Diseases initiative, Nairobi, Kenya, 2Drugs for Neglected Diseases initiative, Geneva, Switzerland, 3Institute of Endemic Disease, University of Khartoum, Sudan, 4Addis Ababa University, Ethiopia, 5University of Gondar, Ethiopia, 6Makerere University, Uganda, 7Kenya Medical Research Institute (KEMRI), Kenya, 8Kacheliba Sub-county Hospital, Kenya,

9Amudat Hospital, Uganda

BackgroundTreatment of patients outside clinical trials in the clinical research setting can be challenging. Evenmore so for neglected tropical diseases research, where trials are often conducted in constrained,poor, rural, and remote settings, and in vulnerable patients. The Leishmaniasis East Africa Platform(LEAP) conducts research in visceral leishmaniasis (VL) treatment centres in eastern Africa (Ethiopia,Kenya, Sudan and Uganda). LEAP aims to conduct clinical trials, build capacity and register newtreatments. With the support of the Drugs for Neglected Diseases initiative (DNDi) and otherpartners, LEAP has delivered one new VL treatment for the region, a sodium stibogluconate andparomomycin combination, with more studies for new treatments, geared towards development ofan oral drug for VL in the region, either ongoing or in the pipeline.

MethodsWe conducted a retrospective review of data for leishmaniasis patients at the LEAP clinical trial sites between 2010 and 2016. Five clinical trials were conductedduring the review period in Ethiopia, Kenya, Sudan, and Uganda. Data were obtained from the DNDi/LEAP data centre in Nairobi based on monthly reportsreceived from the VL treatment sites. We assessed the number of patients treated for leishmaniasis overall and categorized the patients according to whetherthey had participated in clinical trials or not, and further assessed other leishmaniasis cases by number treated for PKDL, cutaneous leishmaniasis and mucosalleishmaniasis. Ethically, it was obligatory to treat patients excluded from the clinical trials.

ResultsDuring the reporting period, 23,740 suspected cases were screened and 10,823 (45.59%) of these were positive for Leishmania. Out of the 10,823, 320 (3%)participated in phase II and III clinical trials, and 1,349 (12.5%) in a phase IV study. There was a total of 9,154 (84.6%) Leishmania cases treated outside the clinicaltrials, corresponding to: VL 8,409 out of 9,154 (91.9), PKDL 157 (1.7%), cutaneous leishmaniasis 491 (5.4.%) and mucosal leishmaniasis 97 (1%). These wereexcluded from the clinical trials due to stringent inclusion or exclusion criteria.

ConclusionsWhile most patients may not participate in clinical trials or be screened out of them, they benefit from a collaborative approach in which VL clinical research has beenintegrated with the treatment of leishmaniasis in patients excluded from participating in the trials. LEAP has implemented a new treatment regimen for VL. There have beenfew advances in the treatment of other forms of leishmaniasis. However, because of clinical trials, communities are offered treatments that would not otherwise beavailable. Resourcing activities, training and adoption of best practices, aimed at building capacity for clinical research, enhance the quality of care that both participants andnon-trial participants receive. Access to better health services, improved lives and livelihood, reduced pain and suffering, are, after all, the goal of research.

8409

491157 97

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Non Trial VL Non Trial CL PKDL Mucosal CL

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Patients treated out of trial

AimTo assess practice, and document treatment of visceral leishmaniasis patients outside clinical trials in LEAP sites.

WorldLeish Congress

16th – 20th May 2017, Toledo, Spain

Poster Number: CL1727

92%

1%5% 2%Patients Treated out of trial

Non Trial Mucosal CL Non Trial CL PKDL

54%

46%

patients Screened for VL

Negative Positive

Selection criteria Percentage

VL parasite negative 55

Abnormal laboratory values 12

Refusal to consent 2

Other eligibility criteria (age, recent VL treatment, concomitant conditions, pregnancy or lactating, malnutrition and undocumented reasons)

5

The research leading to these results has received funding from: the European Union Seventh Framework Programme under grant agreement nº3 05178; French Development Agency (AFD), France; Department for International Development (DFID), UK; Dutch Ministry of Foreign Affairs (DGIS), The Netherlands; Federal ministry of Education and Research (BMBF) through KFW, Germany; Ministry of Foreign Affairs and International Development, France; Medicor Foundation, Liechtenstein; Spanish Agency for International Development Cooperation (AECID), Spain; Médecins Sans Frontières; Swiss Agency for International Development and Cooperation (SDC), Switzerland; Other Private donors