Universal Newborn Screening For Sickle Cell Disease In Mozambique
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 6,750
- 主要终点
- Percentage of eligible population screened for Sickle Cell Disease
研究概览
简要总结
The overarching goal of this study is to evaluate the feasibility of a new methodology that combines three multi-level implementation strategies to optimize the population-level uptake of essential evidence-based, standard of care treatments for infants with sickle cell disease (SCD) in low-resource settings. The study will be done in Mozambique.
详细描述
This prospective mixed-methods hybrid effectiveness-implementation feasibility study will evaluate the feasibility and effectiveness of an implementation strategy package designed to improve early diagnosis and care entry for children born with sickle cell disease (SCD) in rural, low-resource settings. The implementation strategy package includes three components: 1) integration of essential products into national supply chain systems, 2) integration of systematic newborn screening using point-of-care-testing into clinical site workflows and 3) linkage of children who screen positive to a PEN-Plus Non-Communicable Disease (NCD) clinic for longitudinal care. Participants will be tracked longitudinally to evaluate protocol adoption over time and clinical outcomes among participating children at 2 years of age.
Primary Objective
- To test if combining three multi-level implementation strategies can facilitate systematic birth diagnosis and timely linkage to evidence-based care interventions for infants with SCD in under-resourced settings.
Secondary Objectives (Micro level):
- To test the clinical effectiveness of implementing the BB-SCD through the three combined implementation strategies to prevent excess mortality of children with SCD under the age of 2 years.
- To evaluate the effectiveness of combining the three implementation strategies to promote care retention among infants with SCD by 2 years of age.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Other
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Children participants: All infants between birth and 6.0 months of age who are born or receive care at secondary-level facilities involved in the UNIQUE study.
- •Children participants will fall into one of two categories:
- •Patient participants: All infants between birth and 6.0 months of age who were screened through the UNIQUE study and tested positive for SCD (HbSS, HbSC, or other form of SCD) or had indeterminate results.
- •Healthy control participants: Infants between birth and 6.0 months of age who screen negative for SCD (HbAA) through the UNIQUE study or tested positive for sickle cell trait (HbAS).
- •Health facility staff participants: Healthcare staff ages ≥18 years working at secondary-level facilities involved in the UNIQUE study.
- •Supply chain expert participants: Administrative professionals with experience working in or around the national supply chain systems in Mozambique to support procurement, importation, customs clearance, storage, and in-country distribution of medical products.
- •National public health system expert participants: Administrative professionals with experience working in the national public health system (e.g., Ministry of Health, MISAU) who oversee the delivery of health services to infants in-country, such as neonatal testing and vaccination programs.
排除标准
- •Children participants:
- •Stillbirths.
- •Children who received an erythrocyte (blood) transfusion within 3 months of testing. Exogenous (transfused) HbA could artificially lower the sickle hemoglobin concentration, thus causing false-negative results.
- •Patient participants: none
- •Healthy control participants:
- •Stillbirths.
- •Children who received an erythrocyte (blood) transfusion within 3 months of testing. Exogenous (transfused) HbA could artificially lower the sickle hemoglobin concentration, thus causing false-negative results.
- •Health facility staff participants: none.
- •Supply chain expert participants: none.
- •National public health system expert participants: none.
结局指标
主要结局
Percentage of eligible population screened for Sickle Cell Disease
时间窗: 3 years
Percentage of new SCD cases linked to care
时间窗: 3 years
次要结局
- Comparison of under-2 mortality between participants with SCD whom received longitudinal care and participants who screened negative for SCD(2 years post-screening and therapy)
- Percentage of SCD cases retained in care by 2 years of age(2 years post-screening and therapy)
- Percentage of eligible secondary-level clinical wards implementing systematic screening and linkage to longitudinal care for new SCD cases(3 years)
- Percentage of eligible health facility staff participants in secondary-level clinical wards implementing systematic screening and linkage to longitudinal care for new SCD cases(3 years)
- Comparison of SCD Screening Performance Results from Initial point-of-care (POCT), Gazelle, and Hemoglobin Fractionation(3 years)
- Cost-Effectiveness of Integrating SCD Consumables into National Supply Chains(2 to 5 years)
- Cost-Effectiveness of POCT for Early SCD Diagnosis(2 to 5 years)
- Cost-Effectiveness of Decentralized PEN-Plus SCD Management(2 to 5 years)
- Frequency of SCD Supply Stockouts at Study Sites(5 years)