Praziquantel

TOKYO, March 4, 2025 /PRNewswire/ -- The Global Health Innovative Technology (GHIT) Fund is today announcing a significant milestone: the first administration of arpraziquantel to preschool-aged children in Uganda, in an implementation science setting. This new pediatric treatment for schistosomiasis, developed by the GHIT-funded Pediatric Praziquantel Consortium, is being introduced through the Consortium's ADOPT program, which focuses on integrating arpraziquantel into existing healthcare platforms in countries where schistosomiasis is prevalent. This achievement marks GHIT's first supported innovation to reach people in need since its establishment in 2013. "This isn't just a scientific achievement," said Osamu Kunii, CEO of the GHIT Fund. "It's a testament to the transformative power of global collaboration. By bringing together a diverse array of partners, each steadfastly contributing their unique expertise and sustaining momentum throughout a decade-long marathon of innovation to shepherd this breakthrough (from bench to bedside), arpraziquantel's development journey has unlocked solutions that once seemed impossible." Schistosomiasis, or bilharzia, affects 250 million people globally, including an estimated 50 million preschool-aged children, mainly in sub-Saharan Africa. Left untreated, this poverty-related parasitic disease can lead to anemia, stunted growth, and impaired learning ability, as well as chronic inflammation of the organs, which can be fatal. Until now, a child-friendly treatment specifically tailored for preschool-aged children was not available, leaving millions of preschoolers at risk. Early treatment of schistosomiasis in preschool-aged children is a crucial investment in public health and societal wellbeing. By intervening at this critical stage, we not only aim to shield young bodies from severe complications like organ damage and cognitive impairment but also alleviate the long-term burden on healthcare systems and hopefully better livelihoods. The Pediatric Praziquantel Consortium developed arpraziquantel to address this gap. Japan's Astellas Pharma Inc., as a founding member of the Pediatric Praziquantel Consortium, played a pivotal role by utilizing its proprietary technology to lead arpraziquantel's initial formulation development, resulting in water dispersible, climate-stable, child-friendly tablets with acceptable taste. The formulation was optimized by Merck in Germany; the manufacturing process served to produce clinical trial supply from Merck and Farmanguinhos in Brazil. Current manufacturing is done by Farmanguinhos; future large-scale production by Universal Corporation Ltd. in Kenya is planned in and for Africa. "Japanese innovation is a cornerstone of arpraziquantel's development," Dr. Kunii added, "embodying GHIT Fund's core mission: harnessing the power of international partnerships to revolutionize global health." GHIT Fund has supported arpraziquantel's development journey throughout the overall development program spanning over 10 years. GHIT has and continues to support the Pediatric Praziquantel Consortium in developing their access plans, with a particular focus on exploring new and sustainable access mechanisms. GHIT is also co-funding, with EDCTP, implementation via the Consortium's ADOPT Program, which is assessing different implementation platforms in three countries: Côte d'Ivoire, Uganda and Kenya. In addition, GHIT is coordinating, together with the consortium, in-country readiness preparations for introduction of arpraziquantel in additional countries such as Senegal and Tanzania. "Understanding what needs to be in place at country level in terms of policy framework, regulatory framework, implementation models and domestic resource mobilization is critical to the successful uptake and roll out of arpraziquantel. We hope the lessons learnt from these countries will inform other countries," said Dr Isaac Chikwanha, Senior Director of Access at the GHIT Fund. Arpraziquantel received European Medicines Agency's positive scientific opinion in December 2023 and was included into the World Health Organization (WHO)'s List of Prequalified Medicines in May 2024. Inclusion in the WHO's Essential Medicines List is expected in 2025. 1. About schistosomiasis Schistosomiasis (also known as bilharzia) is one of the most prevalent parasitic diseases worldwide and a very important one in terms of public health burden and economic impact. It is a poverty-related disease that is widespread in tropical and subtropical regions where large sections of the population have no access to clean water. Flatworms transmit the disease and people become infected with the parasite through contact with freshwater, for example, while working, swimming, fishing, or washing their clothes. The minuscule larvae penetrate human skin, enter the blood vessels, and attack internal organs. The infection rate is particularly high among children. Schistosomiasis is a chronic condition and is classified by the World Health Organization (WHO) as one of the 21 neglected tropical diseases (NTDs). 2. About arpraziquantel The current standard of care treatment for schistosomiasis is praziquantel. Praziquantel is already approved, and suitable for school-aged children and adults. Extending the range of options for the treatment of schistosomiasis, arpraziquantel is tailored for preschool-aged children against Schistosoma mansoni and Schistosoma haematobium. Tested in clinical development, under the responsibility of Merck, arpraziquantel is a 150mg dispersible tablet. The prototype of its pediatric formulation was developed by Astellas Pharma Inc. in Japan, and further optimized by Merck in Germany. In developing arpraziquantel, the Pediatric Praziquantel Consortium established a pediatric drug development program, divided into four major steps: preclinical development, clinical development, registration, and access. All details can be found on the Consortium website. 3. About the Pediatric Praziquantel Consortium The Pediatric Praziquantel Consortium is an international public-private partnership that aims to reduce the global disease burden of schistosomiasis and improve child health by addressing the medical needs of preschool-aged children. Its mission has been to develop, register, and provide access to a suitable pediatric drug for treating schistosomiasis in children 3 months to 6 years of age. For more information, and to see an overview of all Consortium partners, visit the Consortium website: The GHIT Fund is a Japan-based international public-private partnership (PPP) fund that was formed between the Government of Japan, multiple pharmaceutical companies, the Bill & Melinda Gates Foundation, Wellcome, and the United Nations Development Programme (UNDP). The GHIT Fund invests in and manages an R&D portfolio of development partnerships aimed at addressing neglected diseases, such as malaria, tuberculosis, and neglected tropical diseases, which afflict the world's vulnerable and underserved populations. In collaboration with global partners, the GHIT Fund mobilizes Japanese industry, academia, and research institutes to create new drugs, vaccines, and diagnostics for malaria, tuberculosis, and neglected tropical diseases. For more information, contact: Katy Lenard at +1-301-280-5719 or [email protected] Eriko Mugitani at +81-36441-2032 or [email protected] SOURCE GHIT Fund WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

无症状的寄生虫感染，虽然没有那么来势汹汹，但却在一定程度上掩盖了疾病的发现。 来源 | 医脉通 作者 | 昼辞 01 “病都是查出来的！”不可否认，在现实中有一类人群都有这样的认知错误。家住山区的蔡先生，就是这样的人。 虽然他到了知天命的年纪，但由于平时经常做户外活动，身体还算比较硬朗，只是烟不离手二十多年，家人频频督促他定期体检，但都被他置若罔闻。 直到十天前，在子女的轮番劝说下，蔡先生才在家人的陪同下进行了第一次体检。这一查，果然发现了大问题。 胸部CT显示：双肺多发结节、斑片影，考虑炎症；部分转移待除；食管下端增厚，考虑MT可能。 奇怪的是，患者本身却没有任何发热、畏寒、咳嗽、咳痰等症状，也正因为如此，蔡先生拒绝了医生进一步入院检查的建议。好在家属态度比较坚决，蔡先生见无力改变，才勉强答应住院。 02 很快，一项血检结果的异常升高引起了所有医生的注意。 血常规结果显示：白细胞计数9.68×10^9/L，中性分叶核粒细胞53.0%，嗜酸性粒细胞1.5×10^9/L，血红蛋白187g/L，血小板计数245×10^9/L。 嗜酸性粒细胞的正常值一般为0.02~0.52×10^9/L，很显然，患者的指标明显异常。 嗜酸性粒细胞是人体中比较常见的细胞颗粒，主要来自骨髓，其作用为杀伤细菌以及寄生虫，可帮助人体产生免疫反应。但是不同的仪器在测量过程中可能会存在一定误差，需结合其他的数值综合进行分析，包括白细胞、红细胞、中性粒细胞等。 那么，这究竟是误差，还是疾病呢？ 医生暂时给予患者“头孢曲松 2g QD”抗感染治疗，并辅以化痰治疗，但一切并没有想象中那么顺利。 03 几天后，复查胸部CT，结果显示：双肺上叶及右肺下叶斑片影，考虑感染；右侧胸腔少量积液。复查血常规，结果显示：白细胞计数30.23×10^9/L，中性分叶核粒细胞74.9%，嗜酸性粒细胞10.10x10^9/L，血小板计数280×10^9/L。 这个结果很奇怪，经过抗感染治疗后，炎症细胞、嗜酸性粒细胞不降反升，这到底是怎么回事？ 结合患者症状以及检查结果，医生又给患者使用了激素以抑制炎症，然而其肺部星星点点的病灶仍旧“顽固不化”。 胸部CT。图源：作者提供 住院治疗数日后，并没有任何好转，蔡先生本就沉不住气的脾气更加着急了，“我本来就没病，非让我住院，就这样治，我没病也得把我治出病来。” 说完就要闹着出院，幸亏家属明白事理，也担心是恶性肿瘤或其他疾病，所以在当地医生的建议下，转到了上级医院查明病因。 04 在上级医院，病史是这样写的： 患者系中年男性，以“体检发现‘双肺斑片影’10天”为主诉入院，无明显呼吸道、全身中毒症状，于外院住院期间多次复查血常规提示白细胞、嗜酸性粒细胞增高，查体：无特殊。胸部CT提示：双肺散在多发结节，较前新增，查血常规提示白细胞、嗜酸细胞升高，予以头孢曲松、甲泼尼龙抗炎后，未见明显缩小，故只能暂时诊断“肺部阴影待查、嗜酸性粒细胞增高”。 入院后嗜酸性粒细胞变化趋势。图源：作者提供 所以，上级医院的医生考虑的疾病有以下几种： 1.感染相关性疾病：包括寄生虫、真菌等，肺部病变呈游走性和多形性，医生询问患者职业史，对方表示，“我们靠山吃山，以前从事木板材相关工作，但平时工作环境没接触过什么粉尘，而且已经停工半年了。至于生吃河鲜，我平时都回家吃饭的，不存在这种情况。”但毕竟有野外接触的经历，必要时还需完善血清寄生虫抗体、支气管镜肺泡灌洗液查NGS等检查。 2.变态反应性疾病：包括嗜酸粒细胞性哮喘、变应性支气管肺曲霉菌病等，但患者无发热、畏寒，无咳嗽、咳痰等呼吸道症状，可完善血清Ig、肺功能等检查以辅助诊断。 3.结缔组织相关疾病：包括嗜酸性肉芽肿性多血管炎、IgG4相关性疾病等，不过患者无关节痛、肌无力、雷诺现象，可完善血清自身免疫抗体、体液免疫相关指标以明确。 4.肿瘤性疾病：包括淋巴瘤、白血病、肥大细胞增多症、实体瘤等可发生反应性嗜酸增高。患者多次复查血常规，提示白细胞、嗜酸性粒细胞进行性升高，予以完善骨髓穿刺涂片、细胞学计数、肿瘤标记物等协诊，治疗上暂时给予“莫西沙星”抗感染治疗。 05 血检结果回报，提示血清学IG4稍高，肿瘤标记物也有轻度异常。 短期内嗜酸性粒细胞升高如此之快，肿瘤性疾病的可能性也不容忽视，毕竟此前CT也有提示食管MT（转移瘤？）的可能。不过，在风湿免疫科及血液科医生会诊之后，均初步排除了结缔组织病和血液系统肿瘤的可能。 此外，医生还完善了胃镜检查，结果显示虽然食管部分增厚，胃内占位，但基本可以考虑是良性病变。于是，肿瘤的可能性也变小了。 那么，问题是患者外周嗜酸粒细胞增高和肺部的斑片淡薄影究竟是为什么呢？病毒、细菌、真菌、分枝杆菌均为阴性，那么剩下就应该考虑寄生虫感染的可能性了。 起初，患者是否认过生食河鲜等情况，但随着证据越来越指向寄生虫感染可能，患者也在努力回忆中。 电子支气管镜检查后，送检的肺泡灌洗液的寄生虫结果回报：肺吸虫IgG抗体、包虫IgG抗体阳性。于是医生再次追问患者病史，这才一步步接近真相。 “大概一个月前，我和朋友相约去爬山，遇到一条小溪，觉得水还挺清澈的，刚好口渴就喝了点溪水。然后我朋友捉到一只溪水里的螃蟹，说这个肯定很鲜，我就吃了，谁知道会把肺搞成这样呢？！” 值得注意的是，患者平素无咳嗽，胸部CT未见明显隧道样腔隙，但仍需警惕寄生虫引起继发性嗜酸性粒细胞增高从而导致肺嗜酸性粒细胞浸润症。所以医生还请了传染病科主任会诊，协助评估是否需进行抗寄生虫治疗。 随后传染病科主任医师前来会诊，诊疗后建议：1.吡喹酮治疗；2.查眼底镜及颅脑CT，排除寄生虫；3.定期复查血常规、肝、肾功能等。 尾记 无症状的寄生虫感染，虽然没有那么来势汹汹，但却在一定程度上掩盖了疾病的发现。 这次正因为蔡先生有家人的督促，才发现了其隐匿的疾患，如果没有这次“不情愿”的体检，后果将不堪设想。经过这次经历，蔡先生对“病都是查出来的”这一观念彻底扭转。 另外，这个病例也提醒所有人：健康无小事，在户外活动时，更要注意安全问题，即便溪水很清澈，也不代表全无危险，一定要慎之又慎。 同时也给医生提供了一个好的思路：当肺部渗出性病变在经抗生素治疗效果欠佳时，不要忘记寄生虫等特殊感染，嗜酸性粒细胞升高就是其“最好”的警钟。 参考资料： [1] Ben-David Yael, Bentur Lea, Gur Michal et al. Reverse butterfly pattern image with eosinophilia: Effective treatment with benralizumab-A case report [J]. Pediatr Pulmonol, 2021, 56: 2736-2739. 责编｜米子 封面图来源｜视觉中国 刚刚！复旦版《2023年度中国医院排行榜》发布，快来看看你们医院上榜了吗？ 索赔600余万！68岁男子术后死亡，到底是并发症，还是医生治疗不当？丨医眼看法 医脉通是专业的在线医生平台，“感知世界医学脉搏，助力中国临床决策”是平台的使命。医脉通旗下拥有「临床指南」「用药参考」「医学文献王」「医知源」「e研通」「e脉播」等系列产品，全面满足医学工作者临床决策、获取新知及提升科研效率等方面的需求。 ☟戳这里，更有料！