St. Jude Children's Research Hospital, Inc.

近期，美国USnews《美国新闻与世界报道》发布了“2024~2025最佳儿童医院排行榜”，其中丹娜—法伯/波士顿儿童医院位列第二，连续 18 年荣登全美最佳儿童癌症项目榜单，自 2007 年排名开始以来，每年都被评为全美前三的儿童癌症中心，是世界上当之无愧最好的儿科医院之一！ 波士顿儿童医院血液学/肿瘤学部主任、哈佛医学院儿科 Leland Fikes 教授医学博士David A. Williams表示：“丹娜法伯/波士顿儿童医院的项目非常独特，因为它将世界一流癌症中心和国际知名儿童医院的专业知识结合在一起，为患有癌症和血液疾病的儿童提供专业护理。这项排名认可了我们能够为所有患者提供的个性化治疗方法。” 波士顿儿童医院介绍 丹娜—法伯/波士顿儿童医院（Boston Children's Hospital）汇集了两家国际知名的研究和教学机构，自 1947 年以来一直为儿科肿瘤和血液病患者提供全面的护理。哈佛医学院的附属医疗机构共享一支临床人员队伍，在波士顿儿童医院提供住院护理和手术，在丹娜—法伯癌症研究所提供大部分门诊护理。 波士顿儿童医院始于1869年，一家非盈利医疗机构。150 年以来，一直是多学科儿科护理和疑难杂症治疗领域的全球领先者，享誉全球的医生和护士团队每年会安排超过 650,000 次患者就诊并实施超过 27,000 例外科手术，同时为来自超过 100 个不同国家/地区的患者提供服务，这使其成为全球最大的儿童医疗中心之一。 波士顿儿童医院（Boston Children's Hospital）是全球知名的儿童医院，多年蝉联全美儿童医院综合排名第一/第二，小儿癌症全美第一/第二。此外，在儿童心脏、儿童神经、儿童骨科、儿童内分泌、儿童泌尿、儿童肺科等领域也稳居全美前列。 排名第一--神经内科和神经外科 《美国新闻与世界报道》将波士顿儿童医院的神经病学/神经外科部门评为全美最佳部门之一。 作为全球规模最大、历史最悠久、经验最丰富的神经内科和神经外科部门，波士顿儿童医院治疗的神经系统疾病比世界上任何其他儿科项目都要多。可以为患有神经系统疾病的儿童提供专业的临床护理，并提供广泛而深入的专科护理，以提供最准确的诊断和最先进的治疗方法。 排名第一--胃肠道科和胃肠外科 胃肠病学、肝病学和营养学科排名第一。团队由儿科胃肠病学家、肝病学家、营养师、心理学家、临床医生和护士组成，致力于帮助患有常见或复杂的胃肠道、肝脏和营养问题的儿童，例如： 乳糜泻 慢性腹痛 便秘和腹泻 克罗恩病 胃食管反流 营养不良 运动障碍 短肠综合征 等等，无论孩子刚确诊，还是正在寻找针对新症状或持续症状的新方案，专家团队都可以为您提供帮助。 排名第二--肿瘤科 波士顿儿童医院丹娜法伯癌症研究院联合成立了丹娜法伯/波士顿儿童医院癌症中心，共同创建了综合性儿童癌症和血液病项目，提供世界一流癌症中心和国际知名儿童医院的综合专业护理。丹娜法伯癌症研究所和波士顿儿童医院两家医院通过人行天桥连接，丹娜法伯/波士顿儿童医院提供的专家、治疗方案和临床试验比世界上几乎任何其他治疗癌症和血液病的儿科中心都要多。为患有各种癌症和血液病的儿童提供无缝、综合的医疗团队，包括最罕见和最复杂的病例。 儿童肾病 波士顿儿童医院被公认为治疗婴儿、儿童和青少年肾功能障碍的顶级中心之一，并被《美国新闻与世界报道》评为最好的儿童医院之一，在护理患有慢性肾病的幼儿方面享有国际声誉。对于患有复杂泌尿道异常、侵袭性肾病综合征或慢性肾炎、复杂肾结石以及导致电解质失衡的罕见遗传性肾病的儿童方面也拥有丰富的经验。此外，对于需要长期透析或肾移植的终末期肾病 (ESRD) 儿童方面也处于领先地位。 项目和服务 终末期肾病项目 普通肾脏病治疗计划 高血压计划 肾脏遗传学诊所 肾结石计划 肾脏移植计划 中主动脉综合征和肾血管性高血压中心 研究与创新 波士顿儿童肾脏病科拥有规模最大、历史最悠久的以儿科肾脏病为中心的研究项目之一。最新的研究包括： 肾脏移植研究项目，开展实验室研究，旨在开发更安全、更有效的免疫抑制方案 一项强有力的研究计划，旨在调查肾病综合征、终末期肾病和肾结石的遗传原因，目的是为每位患者制定个性化的治疗方案 一项多学科项目，引领开发针对中主动脉综合征和其他复杂高血压病因的新疗法 再生生物学团队致力于了解导致大多数慢性肾病的发育异常 药物基因组学小组，开发针对儿童基因差异的药物 波士顿儿童医院享誉全球,它的出色表现不仅在于其手术创新以及新的治疗方法的发现，还在于为儿童提供高质量的医疗并给予儿童高度的关注和关爱。 一、被全球公认为儿童肿瘤最好的医院 二、拥有两个世界上最大的儿童癌症和血液病研究项目 三、拥有众多世界知名的医师和科学家 四、基于研究的基础之上的治疗，为患者带来最新的治疗技术 五、为患有疑难和病情反复的患者提供众多临床试验、特殊治疗及高科技资源，以提高其治疗效果 六、多学科治疗，包括儿童血液学家、肿瘤学家、外科手术专家、影像学专家、病理学家、干细胞移植医生和心理学家共同会诊 七、制定儿童综合性的医疗护理计划，照顾到患有癌症和血液疾病的儿童各个方面的健康，给予儿童高程度的关注和关爱。 2024~2025世界最佳儿童医院排名（按字母顺序） 无论孩子患了什么病，这些医院都是全球治疗中名列前茅的！--美国USnews 为了能够给孩子们提供优质医疗照护，帮助迷茫的父母们寻求更为合适的医疗机构，美国三大新闻杂志之一的《美国新闻与世界报道》（U.S News & World Report）每年都会发布年度美国最佳儿童医院排行榜，至今已连续18年。全球肿瘤医生网医学部大家整理编译，希望给迷茫中的父母照亮希望之光。 2024-25年的排名是通过向近88家医院发送的临床调查收集的数据创建的，并向超过数千名儿科专家的医生进行了声誉调查。 波士顿儿童医院 在新生儿科、神经内科和神经外科以及泌尿科领域名列前茅。 辛辛那提儿童医院 在癌症、胃肠病学和胃肠外科以及肺病学和肺外科方面名列前茅。 奥罗拉科罗拉多儿童医院 在糖尿病和内分泌科领域排名第一。 洛杉矶儿童医院 在骨科和泌尿科领域名列前茅。 费城儿童医院 在糖尿病与内分泌学以及骨科领域名列前茅。 华盛顿特区儿童国家医院 在新生儿科领域排名第一。 俄亥俄州哥伦布市全国儿童医院 在胃肠病学和胃肠外科以及肾脏病学方面名列前茅。 圣地亚哥拉迪儿童医院 在心脏病学和心脏外科领域排名第一。 华盛顿州西雅图儿童医院 在肾脏病学领域排名第一。 休斯顿德克萨斯儿童医院 在心脏病学、心脏外科和肾脏病学方面名列前茅。 2024~2025世界最佳儿童癌症医院排名TOP10 排名 医院名称 1 辛辛那提儿童医院医疗中心 2 波士顿儿童医院 3 费城儿童医院 4 德克萨斯儿童医院 5 儿童国家医院 6 洛杉矶儿童医院 7 圣犹达儿童研究医院 8 科罗拉多儿童医院 9 全国儿童医院 10 西雅图儿童医院 2024~2025十大儿童专科世界排名TOP1 专科 医院名称 癌症 辛辛那提儿童医院 心脏病学和心脏外科 德克萨斯儿童医院 糖尿病和内分泌 费城儿童医院 胃肠病学和胃肠外科 辛辛那提儿童医院 新生儿科 波士顿儿童医院 肾脏科 德克萨斯儿童医院 神经科和神经外科 波士顿儿童医院 骨科 费城儿童医院 肺外科 辛辛那提儿童医院 泌尿科 波士顿儿童医院 参考资料：https://health.usnews.com/health-news/best-childrens-hospitals/articles/best-childrens-hospitals-honor-roll-and-overview

Ongoing international oncology collaboration works to empower health care workers and save lives across diverse resource environments. Accra, GHANA, Oct. 29, 2024 /PRNewswire/ -- International oncology experts are gathering in Accra, Ghana for a series of meetings beginning today, to update cancer treatment recommendations in the NCCN Harmonized Guidelines™ for Sub-Saharan Africa. This is the latest event from a longstanding collaboration between the African Cancer Coalition (ACC), American Cancer Society (ACS), and National Comprehensive Cancer Network® (NCCN®), and the Clinton Health Access Initiative (CHAI) that collectively form the group Allied Against Cancer. Together, the organizations are focused on providing context-appropriate, evidence-based cancer care strategies that address the unique challenges faced across the region. Continue Reading NCCN Harmonized Guidelines for Sub-Saharan Africa available for free at NCCN.org/harmonized. The NCCN Harmonized Guidelines™ for Sub-Saharan Africa are free resources for optimizing and standardizing cancer care across the region. They are available at NCCN.org/harmonized or via the Virtual Library of NCCN Guidelines® App. "The Sub-Saharan Africa region is dealing with a rising rate of cancer incidences. Standardizing care based on the latest evidence-based expert consensus-driven recommendations helps ensure consistency in treatment and enables better planning for how to use available resources," said Dr. Henry Ddungu, Senior Consultant, Hematology/Oncology; African Cancer Coalition. "We can ensure people with cancer receive the most effective treatment by harnessing local expertise combined with global best practices." During the meeting, the updates to the NCCN Harmonized Guidelines™ for Sub-Saharan Africa will focus on caring for blood malignancies and children's cancers, including: Acute Lymphoblastic Leukemia Acute Myeloid Leukemia B-cell Lymphomas Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma Chronic Myeloid Leukemia Hodgkin Lymphoma Multiple Myeloma Pediatric Acute Lymphoblastic Leukemia Pediatric Aggressive B-Cell Lymphomas Pediatric Central Nervous System Cancers Waldenstrom's Macroglobulinemia/Lymphoplasmacytic Lymphoma "Since the launch of Allied Against Cancer and publication of the first NCCN Harmonized Guidelines™ for Sub-Saharan Africa in 2017, we have grown to include guidelines covering more than 90% of adult cancer incidences, and most pediatric cancers," said Anu Agrawal, MD, Vice President, Global Cancer Support, American Cancer Society (ACS). "We are proud of the work we've accomplished collaboratively and excited to see continued progress in cancer care across the region." "As we strive for health equity across the globe, it is vital that health care providers are equipped with the best tools and resources to deliver optimal care for a variety of different settings," said Crystal S. Denlinger, MD, Chief Executive Officer, NCCN. "These guidelines are a critical step for helping cancer care professionals stay current on the latest advancements in treatment. They provide a roadmap for ideal options while also taking into account the challenges that exist when delivering care in resource limited settings." Dr. Denlinger was joined by NCCN's Chief Medical Officer, Wui-Jin Koh, MD, along with the following NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) Panel leadership: Ranjana H. Advani, MD, Stanford Cancer Institute Jessica K. Altman, MD, Robert H. Lurie Comprehensive Cancer Center of Northwestern University Hiroto Inaba, MD, PhD, St. Jude Children's Research Hospital/The University of Tennessee Health Science Center The NCCN Harmonized Guidelines™ offer color-coded tiers of treatment recommendations based on resource availability. They cover more than 40 different cancer types, plus screening, supportive care, and specific population concerns. They have been utilized nearly 70,000 times since 2017 and have been endorsed by the Federal Ministries of Health and leading cancer centers in Ethiopia, Liberia, Malawi, Nigeria, Tanzania, Uganda, and Zambia. Learn more about Allied Against Cancer's collaborative work to improve cancer care in Sub-Saharan Africa at alliedagainstcancer.org. Visit NCCN.org/global for other free international cancer prevention and treatment resources. About the National Comprehensive Cancer Network The National Comprehensive Cancer Network® (NCCN ®) is a not-for-profit alliance of leading cancer centers devoted to patient care, research, and education. NCCN is dedicated to improving and facilitating quality, effective, equitable, and accessible cancer care so all patients can live better lives. The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines ®) provide transparent, evidence-based, expert consensus recommendations for cancer treatment, prevention, and supportive services; they are the recognized standard for clinical direction and policy in cancer management and the most thorough and frequently-updated clinical practice guidelines available in any area of medicine. The NCCN Guidelines for Patients® provide expert cancer treatment information to inform and empower patients and caregivers, through support from the NCCN Foundation®. NCCN also advances continuing education, global initiatives, policy, and research collaboration and publication in oncology. Visit NCCN.org for more information. Media Contact: Rachel Darwin 267-622-6624 [email protected] Photo - Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

iStock/ YuLi4ka In the wake of Pfizer’s voluntary market withdrawal of the popular sickle cell disease therapy, BioSpace looks at five investigational drugs currently making their way through the pipeline. A recent fumble has left the sickle cell disease community reeling . Last month, Pfizer abruptly pulled Oxbryta from all global markets after new data showed a higher risk of deaths and complications in treated patients. “[Oxbryta] was one of four medications which we would describe as disease-modifying,” Clifford Takemoto, director of clinical hematology at St. Jude Children’s Research Hospital, told BioSpace . Since the approval of Bristol Myers Squibb’s hydroxyurea for sickle cell disease (SCD) in 1998, only three disease-modifying medications have entered the market, he said. Oxbryta, which won FDA accelerated approval in November 2019, was one of these. Another, Novartis’ Adakveo, has also been called into question after failing to reduce vaso-occlusive crises (VOC) in people with SCD in a Phase III trial last year. EU regulators formally revoked Adakveo’s approval in August 2023, but it remains available in the U.S. “We were optimistic that things were changing,” Takemoto said. “People had hoped that these medications would make a difference.” SCD affects more than 100,000 people in the U.S. and 8 million worldwide. With so few treatment options, it remains a seriously underserved population . Takemoto said the Oxbryta withdrawal has left some patients a “bit angry” and distrustful of new medications, and the question for the community remains: “What’s next?” For Takemoto, the ultimate hope for SCD treatment lies in next-gen transplantation and gene therapy. With the approval of bluebird bio’s Lyfgenia and Vertex and CRISPR Therapeutics’ Casgevy, the potential for gene therapies in SCD is there, even if the rollout is slow and for most, not very accessible, he said. St. Jude is developing its own gene therapy, which the research hospital hopes to move into clinical trials sometime next year. Takemoto also pointed to work being done in bone marrow transplantation intended to reduce toxicity and improve success rates. St. Jude is working on a process that will utilize a “half match,” where a parent’s bone marrow can be used instead of a sibling. These treatments could be transformative, Takemoto said. Here, BioSpace looks at five investigational therapies making their way through the SCD pipeline. Pfizer’s Inclacumab and Osivelotor Oxbryta is not the only SCD asset Pfizer brought onboard with its 2022 acquisition of Global Blood Therapeutics . Two other investigational therapies, inclacumab and osivelotor, were also part of the deal. Inclacumab, a P-selectin inhibitor, is currently being assessed in a Phase III trial to determine its safety and efficacy in reducing VOC. The drug works by binding to P-selectin, a cell adhesion molecule found on platelets. According to Pfizer, this prevents platelets from aggregating, which in turn, is expected to help maintain normal blood flow. Like Oxbryta, osivelotor is a sickle hemoglobin polymerization inhibitor, but with a next-generation design. The asset is currently being studied in a Phase II/III study slated for completion in 2028. While Pfizer has not stated whether the recent findings related to Oxbryta will impact the development of osivelotor, Leerink Partners noted that a Phase II study raised safety concerns, with treatment-emergent adverse events seen in 8 out of 35 patients. Agios Pharmaceuticals’ Mitapivat Agios Pharmaceuticals is developing mitapivat, a pyruvate kinase (PK) activator that is already approved for the treatment of hemolytic anemia in adults with PK deficiency, for SCD. Red blood cells require the PK enzyme for energy, Takemoto explained. In December 2023, the company presented positive data from the Phase II portion of its pivotal RISE UP study. The annualized rate of sickle cell pain crises for patients in the higher dose treatment arm was 0.51 compared to 1.71 for participants in the placebo arm. Takemoto said the oral medication seems to benefit red blood cell health in general and noted that the data look favorable for patients with SCD and other hemolytic anemias. But not everyone is as convinced. Leerink Partners analysts wrote last month of concerns that similar physiological effects could be seen with both Oxbryta and mitapivat. A Phase III readout is expected next year, and the company is targeting a label expansion in 2026. Novo Nordisk’s Etavopivat In 2022, Novo Nordisk paid $1.1 billion to acquire Forma Therapeutics, gaining a lead SCD asset, etavopivat, in the deal. Like mitapivat, etavopivat is also a PK activator, but Novo’s candidate comes with a potential advantage —once daily dosing, versus mitapivat’s twice daily regimen. Etavopivat is currently being studied in a Phase III trial slated for completion in 2026. Phase I data presented by Forma at the American Society of Hematology’s annual meeting in 2021 showed a “sustained increase in hemoglobin and improvement in biomarkers of hemolysis and red blood cell health.” The results, from 15 patients dosed for up to 12 weeks, also showed a trend toward decreasing VOC compared to the 12 months prior to their entering the trial. Takemoto said the candidate “looks very promising” so far but acknowledged that “there are things we don’t know about it. We may learn new side effects as it’s used.” St. Jude will serve as a site for the Phase III trial. Fulcrum Therapeutics’ Pociredir Fulcrum Therapeutics ’ pociredir, a polycomb repressive complex 2 (PRC2) inhibitor, is designed to induce expression of fetal hemoglobin to compensate for the mutated hemoglobin that occurs in SCD. The development path for pociredir has not been without bumps. In February 2023, the FDA slapped Fulcrum with a clinical hold based on previously submitted preclinical data and non-clinical and clinical evidence of hematological malignancies observed with other PRC2 inhibitors. The hold was released six months later. The small molecule, which is currently being studied in a Phase Ib trial, has been well-tolerated in people with SCD with no treatment-related adverse events reported after up to three months exposure, according to a press release announcing the lifted clinical hold.