Patients' preferences of cutaneous leishmaniasis treatment outcomes: Findings from an international qualitative study

Astrid C. Erber (1, 2) , Byron Arana (3) , Afif Ben Salah (4, 5) , Issam Bennis (6, 7) , Aicha Boukthir (4) , María del Mar Castro Noriega (8, 9) , Mamoudou Cissé (10) , Gláucia Fernandes Cota (11) , Farhad Handjani (12) , Liliana López-Carvajal (13) , Kevin Marsh (1) , Dalila Martínez Medina (14) , Emma Plugge (1, 15) , Trudie Lang (1) , Piero Olliaro (1, 16)

1 University of Oxford
2 Medizinische Universität Wien = Medical University of Vienna
3 Drugs for Neglected Diseases Initiative
4 Institut Pasteur de Tunis
5 Arabian Gulf University
6 ENSP - Ecole Nationale de Santé Publique - National School of Public Health [Rabat]
7 ITM - Institute of Tropical Medicine [Antwerp]
8 CIDEIM - Centro Internacional de Entrenamiento e Investigaciones Medicas
9 ICESI - Universidad Icesi
10 Centre Muraz [Bobo-Dioulasso, Burkina Faso]
11 Fiocruz Minas - René Rachou Research Center / Instituto René Rachou [Belo Horizonte, Brésil]
12 SUMS - Shiraz University of Medical Sciences [Iran]
13 Universidad de Antioquia = University of Antioquia [Medellín, Colombia]
14 UPCH - Universidad Peruana Cayetano Heredia
15 Public Health England [London]
16 TDR - UNICEF/UNDP/World Bank/WHO Special Programme for Research and Training in Tropical Diseases

Background Cutaneous leishmaniasis (CL) is a disease that often affects exposed skin areas and may heal leaving lifelong scars. Patients’ expectations from treatment are rarely considered in drug development for CL. An initiative aiming to address shortcomings in clinical trial design and conduct for CL treatments involving the researchers’ community is on-going. This manuscript presents patient-preferred outcomes for CL and an assessment on how to consider these in the conduct of future trials. Methodology/Principal findings We report preferred treatment outcomes by 74 patients with confirmed CL in endemic regions of Brazil, Burkina Faso, Colombia, Iran, Morocco, Peru and Tunisia during individual in-depth interviews. Beyond outcomes customarily considered in trials (such as lesion appearance and adverse events), patients talked about a large number of outcomes related to quality of life, such as pain, scar formation, and others affecting their work and daily activities. They also reported fears around getting rid of the parasite, disease recurrence, and possible sequelae. Conclusions/Significance The study results provide a rich insight into important outcomes for CL treatments, as well as related topics, from the perspective of a diverse patient population. Among the outcomes identified, we argue that those related to quality of life as well as recurrence should be included to a greater extent for assessment in clinical trials, and discuss the suitability of measurement instruments such as the Dermatology Quality of Life Index (DLQI). Interviews also point out the potential need to address concerns related to parasitological cure or scar formation, such as social stigmatization and disability. In addition, patients should be given information in order to clarify reported misconceptions. This study therefore suggests a methodology for consulting CL patients on outcomes as elements of clinical trial design, and how to incorporate these outcomes in trials. It also discusses how reported outcomes could be addressed in clinical care.

Domaines: Sciences du Vivant [q-bio]

Astrid C. Erber, Byron Arana, Afif Ben Salah, Issam Bennis, Aicha Boukthir, et al.. Patients' preferences of cutaneous leishmaniasis treatment outcomes: Findings from an international qualitative study. PLoS Neglected Tropical Diseases, 2020, 14 (2), pp.e0007996. ⟨10.1371/journal.pntd.0007996⟩. ⟨pasteur-03561572⟩