Development of a Core Outcome Measure Instrument; “LeishCOM_LCL”, for Localised Cutaneous Leishmaniasis

Abstract

AbstractBackgroundLocalized cutaneous leishmaniasis (LCL) is a chronic ulcerating disease. A literature review identified inconsistencies in clinical trials. The aims of this study were to reach a consensus on the most important domains to measure when assessing LCL, agree on parameters to measure the domains, and develop a tool representing a Core Outcome Set (COS), for use in clinical assessment of LCL.Methodology & Principal findingsA literature review was conducted to identify any existing COS for LCL embracing agreed Outcome Domains, i.e. what to measure and any Outcome Measurement Instruments (OMIs). As no COS was available, potential outcome domains for assessment of LCL were identified through an international collaborative approach using e-consultations and virtual discussions with expert stakeholders (n=20) from geographically different LCL endemic countries. Subsequent judgmental validation process included a face-to-face multidisciplinary stakeholders’ meeting adopting the Nominal Group Technique. A final consensual agreement on outcome domains and items required to measure these domains was established. “Clinical Cure” was defined as the ideal overall "General Concept". The five Core Outcome Domains includedSignscapturing clinical morphology, diameter, and induration of an index lesion with the aid of a palpability score,Treatment Efficacy assessing percentage change in size of the lesion and re-epithelialization compared to baseline,Treatment Impactwhich included an investigator and patient visual analogue score, andClinical Sequelaerating pigment change, atrophic and hypertrophic/keloid scars. It was agreed that two open-ended questions should be included to capture some aspects ofHealth-Related Quality of Lifeas a means of capturing a patient-focused approach.ConclusionLeishCOM_LCL was generated to reflect a COS for LCL. This captured demographic details, agreed outcome domains and measures to assess these domains. Validation of LeishCOM_LCL will be reported in a separate paper. Development of a Patient Reported Outcome Measure will be considered in the future.Author SummaryLocalized cutaneous leishmaniasis (LCL) is a chronic ulcerating disease caused by the parasiteLeishmaniaspp. Literature review identified inconsistencies in methods and parameters used to evaluate treatment/alternative-interventions resulting in difficulties in comparing new treatment/interventions in clinical trials. In our international consensual study, we adopted the face-to-face nominal group technique and a judgment process to identify domains key to assessment of LCL. Subsequent measures for each domain were used to form a Core Outcome Set (COS). LeishCOM_LCL was developed as an outcome measure instrument (OMI) to capture the COS incorporating existing and newly developed tools. “Clinical Cure” was agreed as the “General Concept” to be captured through five domains. It was agreed that “Signs” domain should capture clinical morphology, diameter, and induration of an index lesion with the aid of a palpability score. “Treatment Efficacy” was assessed by recording percentage change in size of the lesion and re-epithelialization compared to baseline. “Treatment Impact” was reflected through an investigator and the patient visual analogue score and “Clinical Sequelae” rated pigment change, atrophic/hypertrophic scars. Two open-ended questions were included to capture some aspects of “Health-Related Quality of Life”. CutLeishCOM also records patient demographic details and was validated in a small cohort of patients.