Post-kala-azar dermal leishmaniasis (PKDL) drug efficacy study landscape: a systematic scoping review of clinical trials and observational studies to assess the feasibility of establishing an individual participant-level data (IPD) platform

Author:

Singh-Phulgenda SaumanORCID,Kumar Rishikesh,Dahal Prabin,Munir Abdalla,Rashan Sumayyah,Chhajed Rutuja,Naylor Caitlin,Maguire Brittany J.,Siddiqui Niyamat Ali,Harriss Eli,Rahi Manju,Alves Fabiana,Sundar Shyam,Stepniewska Kasia,Musa Ahmed,Guerin Philippe J,Pandey Krishna

Abstract

AbstractBackgroundPost-kala-azar dermal leishmaniasis (PKDL) is a dermatosis which can occur after successful treatment of visceral leishmaniasis (VL) and is a public health problem in VL endemic areas. We conducted a systematic scoping review to assess the characteristics of published PKDL clinical studies, understand the scope of research and explore the feasibility and value of developing a PKDL individual patient data (IPD) platform.MethodsA systematic review of published literature was conducted to identify PKDL clinical studies by searching the following databases: PubMed, Scopus, Ovid Embase, Web of Science Core Collection, WHO Global Index Medicus, PASCAL, Clinicaltrials.gov, Ovid Global Health, Cochrane Database and CENTRAL, and the WHO International Clinical Trials Registry Platform. Only prospective studies in humans with PKDL diagnosis, treatment, and follow-up measurements between January 1973 and March 2023 were included. Extracted data includes variables on patient characteristics, treatment regimens, diagnostic methods, geographical locations, efficacy endpoints, adverse events and statistical methodology.ResultsA total of 3,418 records were screened, of which 56 unique studies (n=2,486 patients) were included in this review. Out of the 56 studies, 36 (64.3%) were from India (1983-2022), 12 (21.4%) from Sudan (1992-2021), 6 (10.7%) were from Bangladesh (1991-2019), and 2 (3.6%) from Nepal (2001-2007). Five (8.9%) studies were published between 1981-1990 (n=193 patients), 10 (17.9%) between 1991-2000 (n=230 patients), 10 (17.9%) between 2001-2010 (n=198 patients), and 31 (55.4%) from 2011 onwards (n=1,865 patients). Eight (14.3%) were randomised clinical trials, and 48 (85.7%) were non-randomised studies. The median post-treatment follow-up duration was 365 days (range: 90-540 days) in 8 RCTs and 360 days (range: 28-2,373 days) in 48 non-randomised studies. Disease diagnosis was based on clinical criterion in 3 (5.4%) studies, a mixture of clinical and parasitological methods in 47 (83.9%) and was unclear in 6 (10.7%) studies. Major drugs used for treatment were miltefosine (n=636 patients), liposomal amphotericin B (L-AmB) (n=508 patients), and antinomy regimens (n=454 patients). Ten other drug regimens were tested in 270 patients with less than 60 patients per regimen.ConclusionsOur review identified studies with very limited sample size for the three major drugs (miltefosine, L-AmB, and pentavalent antimony), while the number of patients combined across studies suggest that the IPD platform would be valuable. With the support of relevant stakeholders, the global PKDL community and sufficient financing, a PKDL IPD platform can be realised. This will allow for exploration of different aspects of treatment safety and efficacy, which can potentially guide future healthcare decisions and clinical practices.PROSPEROCRD42021295848Author summaryPost-kala-azar dermal leishmaniasis (PKDL) is a dermatosis which mostly manifests after successful treatment of visceral leishmaniasis (VL) and is characterised by macular, papular, nodular, erythematous, or polymorphic rashes. PKDL is a public health problem in VL endemic areas, as recent infectivity studies show thatL. donovaniparasites can be found in PKDL lesions and remain infectious to sandfly vectors. There are numerous gaps in our existing knowledge of PKDL, including its pathology, immunology, and risk factors associated with therapeutic outcomes. Currently recommended treatments are either expensive (liposomal amphotericin-B), have raised safety concerns (especially for antimony regimens), or require long treatment duration (e.g. miltefosine). In order to scope the measure of evidence supporting therapeutic efficacy recommendations for PKDL patients, we conducted a systematic literature review. Our systematic review identified 56 PKDL studies describing 2,486 patients, with a majority of the studies (31 studies and 1,865 patients) published from 2010 onwards. The Infectious Diseases Data Observatory (IDDO) already have an established data platform for VL, and theIDDO VL data platformcurrently hosts a critical mass of data from efficacy trials in VL conducted over the past 20 years. Based on the identified volume of data, with a substantial number of studies being relatively recent, we believe that the establishment of a PKDL data platform is feasible. Creating a platform to facilitate the sharing of the datasets would enable in-depth IPD meta-analyses with existing data to address several knowledge gaps of PKDL and guide future research priorities. With the help of relevant stakeholders, the global PKDL community and sufficient resources, a PKDL data platform can be realised and help address key research gaps.

Publisher

Cold Spring Harbor Laboratory

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