Periodic Reporting for period 1 - OPTIC-TB (OPTIMIZING THE IMPLEMENTATION AND SCALE-UP OF THE WHO TB TREATMENT DECISION ALGORITHMS FOR CHILDREN WITH PULMONARY TUBERCULOSIS IN SUB-SAHARAN AFRICA)
Periodo di rendicontazione: 2024-04-01 al 2025-09-30
Each year, nearly one million children develop TB, and a quarter die. Over 60% of cases go undiagnosed, especially in sub-Saharan Africa, where diagnostic challenges and limited laboratory capacity hinder detection. In 2022, WHO issued an interim recommendation for using Treatment-Decision Algorithms (TDAs) to diagnose TB in children under ten, based on limited evidence. Our project will assess the effectiveness, cost-effectiveness, feasibility, and acceptability of implementing TDAs in routine care settings in Tanzania, Uganda, and the Democratic Republic of Congo. The goal is to increase case detection, reduce delays, and improve treatment outcomes, while strengthening research capacity and regional collaboration.
Pathways toward impact
The project will be implemented in 120 primary health facilities and will involve screening of 60,000 children across the three countries. Healthcare workers will be trained to improve clinical practice, and we will engage policymakers, District Health Management Teams, communities, and other key stakeholders to support uptake. Evidence from the project will inform integration of TDAs into national TB guidelines. The project will also promote technology transfer, knowledge sharing, and stronger South–South and North–South collaboration. Empowering primary facilities to provide timely pediatric TB care with the new TDAs will reduce dependence on unavailable diagnostic services such as chest X-ray.
Integration of social sciences and humanities
We will use social sciences and humanities to answer five broad research questions: 1) What are the barriers and facilitators to the uptake and implementation of the TDAs in routine healthcare settings, and how can these be addressed? 2) What strategies can be employed to train healthcare workers on the effective use of TDAs, considering limited resources, diverse cultural contexts, and varying levels of healthcare infrastructure? 3) How can community engagement and involvement be effectively incorporated into the implementation of the TDA strategy to enhance acceptability, ownership, and sustainability? 4) What financing models and policy interventions can be developed or strengthened to support the availability, affordability, and accessibility of TDAs? 5) How can the research findings from the project be effectively communicated to key stakeholders to promote its adoption and integration into clinical practice?
Pathways toward impact
The project will be implemented in 120 primary health facilities and will involve screening of 60,000 children across the three countries. Healthcare workers will be trained to improve clinical practice, and we will engage policymakers, District Health Management Teams, communities, and other key stakeholders to support uptake. Evidence from the project will inform integration of TDAs into national TB guidelines. The project will also promote technology transfer, knowledge sharing, and stronger South–South and North–South collaboration. Empowering primary facilities to provide timely pediatric TB care with the new TDAs will reduce dependence on unavailable diagnostic services such as chest X-ray.
Integration of social sciences and humanities
We will use social sciences and humanities to answer five broad research questions: 1) What are the barriers and facilitators to the uptake and implementation of the TDAs in routine healthcare settings, and how can these be addressed? 2) What strategies can be employed to train healthcare workers on the effective use of TDAs, considering limited resources, diverse cultural contexts, and varying levels of healthcare infrastructure? 3) How can community engagement and involvement be effectively incorporated into the implementation of the TDA strategy to enhance acceptability, ownership, and sustainability? 4) What financing models and policy interventions can be developed or strengthened to support the availability, affordability, and accessibility of TDAs? 5) How can the research findings from the project be effectively communicated to key stakeholders to promote its adoption and integration into clinical practice?
Objective 1: Compare the effectiveness of TDA vs. Standard of Care
Ethical approvals have been secured in all four countries, and the trial is registered in PACTR (PACTR202501677523721). An Independent Ethics Advisor provided input that strengthened the ethical dimension of the project. Baseline assessments and randomization of 120 health facilities are complete. A robust digital data system (REDCap and KoboToolbox) is fully operational, enabling secure, real-time, multilingual data entry. Recruitment is progressing well: 7,456 children have been screened, 3,519 identified as presumptive TB, and 527 initiated on treatment. A seven-member Scientific Advisory Committee (DSMB) with defined Terms of Reference has been established and has met twice. The Scientific Advisory Committee is active and has convened twice to monitor project implementation.
Objective 2: Identify implementation processes and contextual factors
A baseline assessment with 280 respondents across Tanzania, Uganda, and DR Congo found strong stakeholder support for TDAs as effective, sustainable, and well integrated into existing systems. Ongoing supervisory visits show gaps in algorithm adherence—particularly clinicians’ reluctance to initiate treatment at the recommended TDA threshold, and caregivers’ hesitancy to start treatment based solely on TDA results. A protocol acceptability/feasibility studies have been developed and submitted to peer-reviewed journals.
Objective 3: Assess costs, cost-effectiveness, and population-level impact
Costing and economic evaluation studies have been designed alongside the trial. A comprehensive economic evaluation protocol has been developed and submitted for publication. Data collection for patient costs and Health-Related Quality of Life (HRQL) is ongoing alongside the main trial across all countries. To date, 321 caregivers have been interviewed for cost data, and 160 children have completed the initial HRQL assessments in Tanzania and Uganda.
Objective 4: Validate diagnostic performance of TDAs
A detailed protocol and validation checklist have been developed to evaluate TDA sensitivity, specificity, and predictive values. A total of 424 children who met the TDA treatment threshold but were not started on treatment are being closely followed to determine their true TB status. These efforts lay the foundation for comprehensive performance analysis across diverse clinical and demographic settings.
Ethical approvals have been secured in all four countries, and the trial is registered in PACTR (PACTR202501677523721). An Independent Ethics Advisor provided input that strengthened the ethical dimension of the project. Baseline assessments and randomization of 120 health facilities are complete. A robust digital data system (REDCap and KoboToolbox) is fully operational, enabling secure, real-time, multilingual data entry. Recruitment is progressing well: 7,456 children have been screened, 3,519 identified as presumptive TB, and 527 initiated on treatment. A seven-member Scientific Advisory Committee (DSMB) with defined Terms of Reference has been established and has met twice. The Scientific Advisory Committee is active and has convened twice to monitor project implementation.
Objective 2: Identify implementation processes and contextual factors
A baseline assessment with 280 respondents across Tanzania, Uganda, and DR Congo found strong stakeholder support for TDAs as effective, sustainable, and well integrated into existing systems. Ongoing supervisory visits show gaps in algorithm adherence—particularly clinicians’ reluctance to initiate treatment at the recommended TDA threshold, and caregivers’ hesitancy to start treatment based solely on TDA results. A protocol acceptability/feasibility studies have been developed and submitted to peer-reviewed journals.
Objective 3: Assess costs, cost-effectiveness, and population-level impact
Costing and economic evaluation studies have been designed alongside the trial. A comprehensive economic evaluation protocol has been developed and submitted for publication. Data collection for patient costs and Health-Related Quality of Life (HRQL) is ongoing alongside the main trial across all countries. To date, 321 caregivers have been interviewed for cost data, and 160 children have completed the initial HRQL assessments in Tanzania and Uganda.
Objective 4: Validate diagnostic performance of TDAs
A detailed protocol and validation checklist have been developed to evaluate TDA sensitivity, specificity, and predictive values. A total of 424 children who met the TDA treatment threshold but were not started on treatment are being closely followed to determine their true TB status. These efforts lay the foundation for comprehensive performance analysis across diverse clinical and demographic settings.
Preliminary results indicate that the new TDAs facilitate earlier diagnosis and treatment of children with presumptive TB, which can significantly reduce TB-related morbidity and mortality. Under the new TDA approach, 18% of children with presumptive TB have been started on treatment, compared to 11% in the control arm. This proportion could be even higher if the WHO recommendation to initiate treatment when the TDA score is above 10 was consistently followed.