Neurologic-Event-Free Survival (NEFS): A New Endpoint for Severe Combined Immunodeficiency (SCID) Patients Diagnosed by Newborn Screening (NBS) and Treated with Hematopoietic Cell Transplantation (HCT)
Year: 2017
Type: article
Abstract: Background: In areas where TRECs have been added to the NBS program, patients with SCID are now typically diagnosed near the age of 1 month. Historically, patients with SCID who underwent allogeneic HCT before the age of 3.5 months had excellent overall survival (OS). Perhaps due to advances in supportive care, preliminary data from the PIDTC does not show superior 1-year OS for patients diagnosed by NBS compared to those diagnosed clinically due to infection. However, OS does not capture long-term quality of life.
Institution: UCSF Benioff Children's Hospital
Language: English
Subfield: Immunology
Field: Immunology and Microbiology
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $3,650
OpenAlex ID: W2591312730