Mapping the Clinical Development Trajectory of Cell and Gene Therapy Products
Clin Pharmacol Ther. 2024 Dec 10. doi: 10.1002/cpt.3512. Online ahead of print. ABSTRACT While cell and gene therapies (CGTs) have emerged as promising modalities to …
Paediatrics
Executive and adaptive function impacts long-term outcomes for adults with maple syrup urine disease
J Inherit Metab Dis. 2024 Dec 10. doi: 10.1002/jimd.12827. Online ahead of print. ABSTRACT Successful transition to independent adulthood requires intact executive and adaptive function. …
“You First Have to Check Out the Doctors”: Transition Expectations and Experiences of Survivors After Pediatric Cancer
Cancer Med. 2024 Dec;13(23):e70455. doi: 10.1002/cam4.70455. ABSTRACT PURPOSE: The shift from child-centered to adult-focused healthcare presents social and psychological challenges for adolescents and young adults …
Association of Unmet Social Needs With Disease-Related Outcomes in Pediatric Patients With Sickle Cell Disease
Pediatr Blood Cancer. 2024 Dec 9:e31478. doi: 10.1002/pbc.31478. Online ahead of print. ABSTRACT BACKGROUND: Social determinants of health (SDoH) are socioeconomic factors that influence health …
Current Surgical Approach to Pulmonary Metastasectomy
Pediatr Blood Cancer. 2024 Dec 9:e31468. doi: 10.1002/pbc.31468. Online ahead of print. ABSTRACT The lung is a common site of metastases for children and adolescents …
Feasibility of Utilizing a Brief Neurocognitive Battery in 3-Year-Old Patients During Treatment for Acute Lymphoblastic Leukemia
Pediatr Blood Cancer. 2024 Dec 9:e31469. doi: 10.1002/pbc.31469. Online ahead of print. ABSTRACT Treatment of acute lymphoblastic leukemia (ALL) is associated with neurocognitive deficits in …
Chemotherapy-Surgery Interval Effects on Tumor Necrosis and Outcome in Children and Young Adults With Osteosarcoma
Pediatr Blood Cancer. 2024 Dec 9:e31466. doi: 10.1002/pbc.31466. Online ahead of print. ABSTRACT BACKGROUND/OBJECTIVE: Osteosarcoma treatment incorporates chemotherapy and surgery. Resection of the primary tumor …
Salvage Therapy Efficacy is Modified by Risk Group at Diagnosis in Patients With Relapsed Rhabdomyosarcoma
Pediatr Blood Cancer. 2024 Dec 9:e31477. doi: 10.1002/pbc.31477. Online ahead of print. ABSTRACT BACKGROUND: Patients with relapsed rhabdomyosarcoma (RMS) are treated with varying approaches and …
Incidence of adverse events related to intravenous immunoglobulin therapy in children
Transfusion. 2024 Dec 9. doi: 10.1111/trf.18083. Online ahead of print. ABSTRACT BACKGROUND: Intravenous immunoglobulin (IVIG) therapy is used in the treatment of pediatric diseases, although …
Continuous Kidney Replacement Therapy in Children With Sinusoidal Obstruction Syndrome After Hematopoietic Cell Transplant: Outcome and Liberation
Pediatr Blood Cancer. 2024 Dec 9:e31473. doi: 10.1002/pbc.31473. Online ahead of print. ABSTRACT BACKGROUND: Sinusoidal obstruction syndrome (SOS), a serious complication after hematopoietic cell transplant …