UCSF Benioff Children’s Hospital Oakland formerly known as Children's Hospital Oakland is a pediatric acute care hospital located in Oakland, California. The hospital has 191 beds and is affiliated with the UCSF School of Medicine. The hospital provides comprehensive pediatric specialties and subspecialties to infants, children, teens, and young adults aged 0–21 throughout Northern California. UCSF Benioff Children’s Hospital Oakland also features a Level 1 Pediatric Trauma Center, 1 of 5 in the state.It has an affiliated research organization, the Children's Hospital Oakland Research Institute, or CHORI, and is involved in research and treatment for a variety of children's health issues, such as pediatric obesity, cancers, sickle cell disease, AIDS/HIV, hemophilia and cystic fibrosis.
This is a revision of the previous joint policy statement titled "Pediatric Readiness in the Emergency Department." This is a joint policy statement from the American Academy of Pediatrics, the American College of Emergency Physicians, the American College of Surgeons, and the Emergency Nurses Association. These updated recommendations are intended to serve as a resource for clinical and administrative leadership of emergency departments as they strive to improve their readiness for the emergency care of children of all ages.
In 2018, the International Niemann-Pick Disease Alliance (INPDA) and the International Niemann-Pick Disease Registry (INPDR) developed and published comprehensive clinical management guidelines to support inclusive and standardized care pathways in Niemann-Pick disease type C (NPC)-an ultra-rare, autosomal recessive, neurovisceral lysosomal disorder. Since then, advances in diagnostics, care, and the approval of two novel disease-modifying agents have underscored the need to revise these guidelines to ensure safe, consistent, and high-quality care for those affected by NPC. In response, the INPDA and INPDR convened a multidisciplinary Guidelines Development Group (GDG) comprising individuals with NPC expertise from 14 countries across five continents, representing a broad range of specialties, as well as patients and families involved in NPC care. Informed by a comprehensive literature review and two meetings, the GDG systematically reviewed, revised, and updated the 2018 guideline statements, re-evaluating the level of evidence, strength of recommendations, and expert agreement for each. The resulting 2025 consensus clinical management guidelines constitute a timely, up-to-date, and internationally applicable resource for the diagnosis, treatment, and holistic management of individuals with NPC. These guidelines serve as a critical resource for specialist centers, hospital-based medical teams, staff involved in NPC patient care, family physicians and other primary caregivers, and, importantly, patients and their families.
The advancement of cell and gene therapies (CGT) represents a pivotal shift in the treatment model for cancers and a broad spectrum of other diseases, particularly rare and orphan disorders. Despite major advances, information regarding global regulatory pathways is scarce and often perplexing. Essential technicalities to approval, such as current good manufacturing practice, good laboratory practices, critical process parameters and pathways that accelerate the approval process (like orphan drug designation), are often inadvertently marginalized or overcomplicated. Furthermore, the globalization of these therapies is crucially hampered by fragmented, economically unsustainable and unreliable regulatory pathways. These regulatory challenges and the high cost of the traditional clinical trial model create significant bottlenecks for developers. We analyze the current frameworks in major jurisdictions, highlighting the technicalities in each and propose a new, harmonized global blueprint for development and approval. Key components of this proposed framework include a shift toward advanced preclinical models, adaptive trial design and patient-centric endpoints. Along with simplifying the processes for CGT developers (whether industry or academia), adopting this framework could increase the efficiency of CGT approvals, and lower the development costs. Such changes would help in achieving the goal of bench to bedside to globalization, directly translating into a more robust research atmosphere that benefits all by increasing the efficiency of CGT trial and product approvals in both developed and developing countries.
Abstract: In utero hematopoietic cell transplantation (IUHCT) has the potential to treat patients who have hemoglobinopathies by harnessing the unique period of fetal tolerance to maternal cells, thereby enabling semiallogeneic transplantation without conditioning or immunosuppression. We conducted a phase 1 clinical trial of IUHCT in fetuses with α-thalassemia major (ATM), a diagnosis that requires serial in utero transfusions (IUT) for survival. We also analyzed outcomes in fetuses with ATM treated with IUT alone in the same time period. Six fetuses underwent transplantation with maternal CD34+ cells (1.33 × 108 ± 2.67 × 107 cells per kg + 1% T cells) at 23.1 ± 1.1 weeks’ gestation. All received serial IUT and were delivered at, or near, term. Perinatal outcomes were broadly similar to those undergoing IUT alone. Two mothers received prophylactic antibiotics because of late positive cultures of harvested cells (without fetal adverse events). Low-level maternal microchimerism was detected in all recipients; T-cell hyporeactivity to maternal antigens was detected in 2 of 6 offspring but persisted in only 1 child. Cord blood from ATM-affected pregnancies demonstrated marked expansion of host hematopoietic stem and progenitor cells compared with healthy controls, suggesting a competitive disadvantage for donor cells in this disease context. Neurodevelopmental assessments were largely reassuring, with high quality-of-life scores. Overall, this protocol was safe and feasible but demonstrates the limitations of IUHCT for ATM without concurrent bone marrow conditioning. The favorable perinatal and neurologic outcomes in most offspring underscore the benefits of prenatal transfusions and the unmet medical need for developing definitive therapies for ATM. This trial was registered at www.clinicaltrials.gov as NCT02986698.
