MICA: Hydroxyurea - Pragmatic Reduction In Mortality and Economic burden (H-PRIME)
MICA:羟基脲 - 务实降低死亡率和经济负担 (H-PRIME)
基本信息
- 批准号:MR/S004904/1
- 负责人:
- 金额:$ 570.73万
- 依托单位:
- 依托单位国家:英国
- 项目类别:Research Grant
- 财政年份:2019
- 资助国家:英国
- 起止时间:2019 至 无数据
- 项目状态:未结题
- 来源:
- 关键词:
项目摘要
Sickle cell anaemia (SCA) is a common inherited condition that affects around 1% of all children born in much of sub-Saharan Africa. Without early diagnosis and appropriate treatment under-5 mortality in those affected is between 50 and 90%. As a result, through much of the continent SCA is responsible for between 5 and 16% of total under-5 mortality. These high levels of mortality could be reduced dramatically with simple treatments that include educating parents to recognise danger signs and seek emergency care, and by measures that protect against acute bacterial and malarial infections using vaccines and prophylactic antibiotic and anti-malarial drugs. Nevertheless, without specific treatments that modify the course of the disease, many of those affected will live lives that are characterized by frequent and recurrent bouts of severe illness that include acute and chronic pain and progressive multi-organ deterioration. Unlike diseases like HIV, malaria and tuberculosis, SCA does not enjoy a high profile in the eyes of the international community and remains widely neglected by ministries of health through much of sub-Saharan Africa. Through H-PRIME, we will address three key questions in the management of children with SCA in Africa today through a single large, efficient and pragmatic clinical trial. First, we will determine whether hydroxyurea, a common and effective treatment for SCA in resource-rich regions, could be a useful option in parts of Africa with limited access to medical care. In most countries hydroxyurea is administered and monitored in a way that will not be achievable in most of Africa and our primary question, therefore, will be whether the drug can be used safely and effectively to reduce mortality and improve the quality of life in survivors when administered pragmatically following a weight-band-based dosing strategy with minimal clinical and laboratory monitoring. Second, we will investigate whether better protection from bacterial infections in children with SCA can reduce all cause hospital admission and further reduce mortality. The current approach to the prevention of bacterial infections is through the use of oral penicillin. However, this is only effective against a narrow range of bacterial organisms and we will therefore investigate whether the addition of a second agent, co-trimoxazole, could bring further benefits in the absence of harm. Finally, the current approach to the prevention of malaria infections relies on drugs that are associated with high levels of resistance. We will therefore investigate whether malaria prevention with a more modern and highly effective drug - dihidroartemisinin-piperaquine - could be used as an alternative, and that this will not cause harm in terms of side effects and the development of further drug resistance.
镰状细胞贫血(SCA)是一种常见的遗传性疾病,影响了撒哈拉以南非洲大部分地区出生的所有儿童的约1%。如果没有早期诊断和适当治疗,5岁以下儿童的死亡率在50%至90%之间。因此,在非洲大陆的大部分地区,SCA占5岁以下儿童死亡总数的5%至16%。通过简单的治疗方法,包括教育父母识别危险信号并寻求紧急护理,以及通过使用疫苗和预防性抗生素和抗疟疾药物预防急性细菌和疟疾感染的措施,可以大大降低这些高死亡率。然而,如果没有改变病程的具体治疗,许多受影响的人将过上以频繁和反复发作严重疾病为特征的生活,包括急性和慢性疼痛以及进行性多器官退化。与艾滋病毒、疟疾和结核病等疾病不同,SCA在国际社会眼中并不引人注目,在撒哈拉以南非洲的大部分地区,它仍然被卫生部广泛忽视。通过H-PRIME,我们将通过一项大型、高效和务实的临床试验,解决当今非洲SCA儿童管理中的三个关键问题。首先,我们将确定在资源丰富的地区普遍有效的SCA治疗方法-在大多数国家,羟基脲的给药和监测方式在非洲大部分地区是无法实现的,因此,我们的主要问题是,当按照基于体重带的给药策略务实地给药时,该药物是否可以安全有效地用于降低死亡率和改善幸存者的生活质量,并进行最少的临床和实验室监测。其次,我们将研究更好地保护SCA儿童免受细菌感染是否可以减少全因住院并进一步降低死亡率。目前预防细菌感染的方法是使用口服青霉素。然而,这只对一小部分细菌有效,因此我们将研究添加第二种药物复方新诺明是否会在没有危害的情况下带来更多益处。最后,目前预防疟疾感染的方法依赖于与高耐药性有关的药物。因此,我们将研究是否可以使用一种更现代和更有效的药物-双氢青蒿素-哌喹-来预防疟疾,并确保这不会造成副作用和进一步产生抗药性方面的伤害。
项目成果
期刊论文数量(6)
专著数量(0)
科研奖励数量(0)
会议论文数量(0)
专利数量(0)
Characterising demographics, knowledge, practices and clinical care among patients attending sickle cell disease clinics in Eastern Uganda.
描述乌干达东部镰状细胞病诊所患者的人口统计、知识、实践和临床护理特征。
- DOI:10.12688/wellcomeopenres.15847.2
- 发表时间:2020
- 期刊:
- 影响因子:0
- 作者:Olupot-Olupot P
- 通讯作者:Olupot-Olupot P
Red Blood Cell Deformability Demonstrated with the Oxygenscan: Exploring the Association with Hydroxyurea Treatment, Co-Inherited a-Thalassemia, and Frequency of Pain in Children with Sickle Cell Anemia
使用 Oxygenscan 证明红细胞变形能力:探索与羟基脲治疗、共同遗传性 α-地中海贫血以及镰状细胞性贫血儿童疼痛频率的关系
- DOI:10.1182/blood-2019-129836
- 发表时间:2019
- 期刊:
- 影响因子:20.3
- 作者:Nardo-Marino A
- 通讯作者:Nardo-Marino A
Filling the data gaps on sickle cell anaemia in sub-Saharan Africa.
- DOI:10.1016/s2352-3026(22)00042-4
- 发表时间:2022-03
- 期刊:
- 影响因子:0
- 作者:Williams TN
- 通讯作者:Williams TN
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Thomas Williams其他文献
UVAE: Integration of Heterogeneous Unpaired Data with Imbalanced Classes
UVAE:异构不成对数据与不平衡类的集成
- DOI:
10.1101/2023.12.18.572157 - 发表时间:
2023 - 期刊:
- 影响因子:0
- 作者:
Mike Phuycharoen;Verena Kaestele;Thomas Williams;Lijing Lin;Tracy Hussell;John Grainger;Magnus Rattray - 通讯作者:
Magnus Rattray
BronchStart Study Extended Data
BronchStart 研究扩展数据
- DOI:
- 发表时间:
2021 - 期刊:
- 影响因子:0
- 作者:
Thomas Williams - 通讯作者:
Thomas Williams
Évaluation de la longueur du tendon du semi-tendineux en fonction de paramètres cliniques. Analyse et application clinique
- DOI:
10.1016/j.rcot.2013.09.148 - 发表时间:
2013-11-01 - 期刊:
- 影响因子:
- 作者:
Thomas Williams;Aude Griffart;Philippe Colombet - 通讯作者:
Philippe Colombet
3122 – DYNAMIC REGULATION OF HIERARCHICAL HETEROGENEITY IN ACUTE MYELOID LEUKAEMIA, SERVES AS A TUMOUR IMMUNOEVASION MECHANISM.
- DOI:
10.1016/j.exphem.2020.09.131 - 发表时间:
2020-08-01 - 期刊:
- 影响因子:
- 作者:
Constandina Pospori;William Grey;Shayin Gibson;Sara Gonzalez-Anton;Thomas Williams;Christiana Georgiou;Flora Birch;Myriam Haltalli;Maria-Nefeli Skoufou-Papoutsaki;Georgia Stevens;Katherine Sloan;Reema Khorshed;Francesca Hearn-Yeates;Jack Hopkins;Chrysi Christodoulidou;Dimitrios Stampoulis;Hans Stauss;Ronjon Chakraverty;Dominique Bonnet;Cristina Lo Celso - 通讯作者:
Cristina Lo Celso
Investigating the relationship between thalamic iron concentration and disease severity in secondary progressive multiple sclerosis using quantitative susceptibility mapping: Cross-sectional analysis from the MS-STAT2 randomised controlled trial
- DOI:
10.1016/j.ynirp.2024.100216 - 发表时间:
2024-09-01 - 期刊:
- 影响因子:
- 作者:
Thomas Williams;Nevin John;Alberto Calvi;Alessia Bianchi;Floriana De Angelis;Anisha Doshi;Sarah Wright;Madiha Shatila;Marios C. Yiannakas;Fatima Chowdhury;Jon Stutters;Antonio Ricciardi;Ferran Prados;David MacManus;Francesco Grussu;Anita Karsa;Becky Samson;Marco Battiston;Claudia A.M. Gandini Wheeler-Kingshott;Karin Shmueli - 通讯作者:
Karin Shmueli
Thomas Williams的其他文献
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{{ truncateString('Thomas Williams', 18)}}的其他基金
Tracing the origin and diversification of a morphological trait through transcriptional regulators and their target genes
通过转录调节因子及其靶基因追踪形态性状的起源和多样化
- 批准号:
2211833 - 财政年份:2022
- 资助金额:
$ 570.73万 - 项目类别:
Continuing Grant
CAREER: Cognitively-Informed Memory Models for Language-Capable Robots
职业:具有语言能力的机器人的认知信息记忆模型
- 批准号:
2044865 - 财政年份:2021
- 资助金额:
$ 570.73万 - 项目类别:
Standard Grant
CHS: Small: Collaborative Research: Role-Based Norm Violation Response in Human-Robot Teams
CHS:小型:协作研究:人机团队中基于角色的规范违规响应
- 批准号:
1909847 - 财政年份:2019
- 资助金额:
$ 570.73万 - 项目类别:
Standard Grant
S&AS: FND: Context-Aware Ethical Autonomy for Language Capable Robots
S
- 批准号:
1849348 - 财政年份:2019
- 资助金额:
$ 570.73万 - 项目类别:
Standard Grant
CHS: Small: Collaborative Research: APERTURE: Augmented Reality based Perception-Sensitive Robotic Gesture
CHS:小型:协作研究:APERTURE:基于增强现实的感知敏感机器人手势
- 批准号:
1909864 - 财政年份:2019
- 资助金额:
$ 570.73万 - 项目类别:
Standard Grant
CRI: II-New: Infrastructure for Robust Interactive Underground Robots
CRI:II-新:强大的交互式地下机器人基础设施
- 批准号:
1823245 - 财政年份:2018
- 资助金额:
$ 570.73万 - 项目类别:
Standard Grant
Collaborative Research: Resolving the gene regulatory network alterations responsible for the repeated evolution of a Hox-regulated trait
合作研究:解决导致 Hox 调控性状重复进化的基因调控网络改变
- 批准号:
1555906 - 财政年份:2016
- 资助金额:
$ 570.73万 - 项目类别:
Standard Grant
Collaborative Research: The structure, function, and evolution of a regulatory network controlling sexually dimorphic fruit fly development
合作研究:控制性二态性果蝇发育的调控网络的结构、功能和进化
- 批准号:
1146373 - 财政年份:2012
- 资助金额:
$ 570.73万 - 项目类别:
Continuing Grant
相似海外基金
Identifying Mechanisms Involved in Hydroxyurea-Mediated Reduction in Vaso-occlusive Adhesive Events in Sickle Cell Disease
确定羟基脲介导的镰状细胞病血管闭塞性粘附事件减少机制
- 批准号:
10724590 - 财政年份:2023
- 资助金额:
$ 570.73万 - 项目类别:
ADHERE (Applying Directly observed therapy to HydroxyurEa to Realize Effectiveness)
ADHERE(对 HydroxyurEa 应用直接观察疗法以实现有效性)
- 批准号:
10698769 - 财政年份:2023
- 资助金额:
$ 570.73万 - 项目类别:
mAnaging siCkle CELl disease through incReased AdopTion of hydroxyurEa in Nigeria (ACCELERATE)
在尼日利亚通过增加羟基脲的使用来控制镰状细胞病(加速)
- 批准号:
10638598 - 财政年份:2023
- 资助金额:
$ 570.73万 - 项目类别:
Developmental Pharmacology of Hydroxyurea Across the Age Span for the Treatment of Sickle Cell Anemia
不同年龄段羟基脲治疗镰状细胞性贫血的发育药理学
- 批准号:
10382052 - 财政年份:2022
- 资助金额:
$ 570.73万 - 项目类别:
Developmental Pharmacology of Hydroxyurea Across the Age Span for the Treatment of Sickle Cell Anemia
不同年龄段羟基脲治疗镰状细胞性贫血的发育药理学
- 批准号:
10551233 - 财政年份:2022
- 资助金额:
$ 570.73万 - 项目类别:
Model-Informed Evaluation of Hydroxyurea Exposure in Special Populations
特殊人群羟基脲暴露的模型知情评估
- 批准号:
10653016 - 财政年份:2022
- 资助金额:
$ 570.73万 - 项目类别:
Model-Informed Evaluation of Hydroxyurea Exposure in Special Populations
特殊人群羟基脲暴露的模型知情评估
- 批准号:
10427738 - 财政年份:2022
- 资助金额:
$ 570.73万 - 项目类别:
Promoting Utilization and Safety of Hydroxyurea Using Precision in Africa
在非洲精准推广羟基脲的利用和安全
- 批准号:
10209231 - 财政年份:2021
- 资助金额:
$ 570.73万 - 项目类别:
Molecular determinants of fetal hemoglobin induction by hydroxyurea to treat sickle cell disease
羟基脲诱导胎儿血红蛋白治疗镰状细胞病的分子决定因素
- 批准号:
10561728 - 财政年份:2021
- 资助金额:
$ 570.73万 - 项目类别:
Molecular determinants of fetal hemoglobin induction by hydroxyurea to treat sickle cell disease
羟基脲诱导胎儿血红蛋白治疗镰状细胞病的分子决定因素
- 批准号:
10899194 - 财政年份:2021
- 资助金额:
$ 570.73万 - 项目类别: