Results 41 to 50 of about 5,290,684 (266)
ABSTRACT Pediatric gastroenteropancreatic neuroendocrine neoplasms (GEP‐NENs) are extremely rare and clinically heterogeneous. Management has largely been extrapolated from adult practice. This European Standard Clinical Practice Guideline (ESCP), developed by the EXPeRT network in collaboration with adult NEN experts, provides (adult) evidence ...
Michaela Kuhlen +23 more
wiley +1 more source
ABSTRACT Background PIK3CA‐related overgrowth spectrum (PROS) includes several rare overgrowth disorders resulting from somatic gain‐of‐function mutations in PIK3CA. Despite treatment advances, including the recent approval of alpelisib for PROS in the United States, literature detailing the patient experience with PROS is limited.
Vamsi Bollu +8 more
wiley +1 more source
ABSTRACT Background Sickle cell disease (SCD) is a chronic, inherited hemoglobinopathy that requires frequent hospitalization for disease‐related complications. Canadian data on inpatient care is limited. This study compared caregiver‐reported hospital experiences of children with SCD to those with cystic fibrosis (CF), a chronic, autosomal recessive ...
Hailey M. Zwicker +11 more
wiley +1 more source
This is the first of a series of articles that will describe the different types of study design; considerations when choosing a study design; and the advantages and disadvantages of each type of study. This first article explains the importance of choosing an appropriate design and the decisions to be made when doing so.
openaire +2 more sources
ABSTRACT Claudin‐6 has emerged as a promising immunotherapeutic target, yet protein‐level data in atypical teratoid/rhabdoid tumors (AT/RTs) have been inconsistent. We analyzed 36 well‐characterized AT/RT samples and found membranous claudin‐6 protein expression in 58% of cases, with striking enrichment in the molecular subgroup AT/RT‐TYR (100%) and ...
Victoria E. Fincke +4 more
wiley +1 more source
AbstractCompactLight is a Design Study funded by the European Union under the Horizon 2020 research and innovation funding programme, with Grant Agreement No. 777431. CompactLight was conducted by an International Collaboration of 23 international laboratories and academic institutions, three private companies, and five third parties.
D'Auria G. +142 more
openaire +5 more sources
Study design synopsis: Observational study design in equine research
Abstract When planning to conduct research, thorough consideration of the study design is essential to enable valid results and purposeful conclusions. A good study design will provide a strong basis for robust conclusions that can contribute to the evidence base.
April Lawson, Gina Pinchbeck
openaire +2 more sources
ABSTRACT Background Wilms tumor (WT) treatment imposes a significant time burden on patients and their families. Time toxicity is a patient‐centered metric that quantifies the burden of healthcare interaction. We sought to define time toxicity in the first year after diagnosis of WT and hypothesized that it would increase as tumor stage and treatment ...
Caleb Q. Ashbrook +6 more
wiley +1 more source
ABSTRACT Background Central nervous system (CNS) involvement in childhood acute lymphoblastic leukemia (ALL) is assessed by cell counting and cytomorphology from cerebrospinal fluid (CSF) and is used for treatment stratification worldwide. The ratio of “CNS2” patients in clinical trials ranges from 3% to 40%, with unclear prognostic significance ...
Laura Almási +14 more
wiley +1 more source
ABSTRACT Immune effector cell‐associated hemophagocytic lymphohistiocytosis‐like syndrome (IEC‐HS) is a life‐threatening hyperinflammatory toxicity distinct from cytokine release syndrome (CRS) and neurotoxicity following chimeric antigen receptor T‐cell (CAR‐T) therapy. In a single‐institution retrospective cohort of pediatric and young adult patients
Thomas J. Galletta +6 more
wiley +1 more source

