Newborn screening for six lysosomal storage disorders in a cohort of Mexican patients: Three-year findings from a screening program in a closed Mexican health system


Por: Navarrete-Martínez J.I., Limón-Rojas A.E., Gaytán-García M.D.J., Reyna-Figueroa J., Wakida-Kusunoki G., Delgado-Calvillo M.D.R., Cantú-Reyna C., Cruz-Camino H., Cervantes-Barragán D.E.

Publicada: 1 ene 2017
Resumen:
Objective To evaluate the results of a lysosomal newborn screening (NBS) program in a cohort of 20,018 Mexican patients over the course of 3 years in a closed Mexican Health System (Petróleos Mexicanos [PEMEX] Health Services). Study design Using dried blood spots (DBS), we performed a multiplex tandem mass spectrometry enzymatic assay for six lysosomal storage disorders (LSDs) including Pompe disease, Fabry disease, Gaucher disease, mucopolysaccharidosis type I (MPS-I), Niemann–Pick type A/B, and Krabbe disease. Screen-positive cases were confirmed using leukocyte enzymatic activity and DNA molecular analysis. Results From July 2012 to April 2016, 20,018 patients were screened; 20 patients were confirmed to have an LSD phenotype (99.9 in 100,000 newborns). Final distributions include 11 Pompe disease, five Fabry disease, two MPS-I, and two Niemann–Pick type A/B patients. We did not find any Gaucher or Krabbe patients. A final frequency of 1 in 1001 LSD newborn phenotypes was established. Discussion NBS is a major public health achievement that has decreased the morbidity and mortality of inborn errors of metabolism. The introduction of NBS for LSD presents new challenges. This is the first multiplex Latin–American study of six LSDs detected through NBS. © 2017 Elsevier Inc.

Filiaciones:
Navarrete-Martínez J.I.:
 Department of Genetics, Hospital Central Sur de Alta Especialidad, PEMEX, Mexico City, Mexico

Limón-Rojas A.E.:
 General Dictatorate, Hospital Central Sur de Alta Especialidad, PEMEX, Mexico City, Mexico

Gaytán-García M.D.J.:
 Department of Genetics, Hospital Central Sur de Alta Especialidad, PEMEX, Mexico City, Mexico

Reyna-Figueroa J.:
 Department of Medical Education and Research, Hospital Central Sur de Alta Especialidad, PEMEX, Mexico City, Mexico

Wakida-Kusunoki G.:
 General Dictatorate, Hospital Central Sur de Alta Especialidad, PEMEX, Mexico City, Mexico

Delgado-Calvillo M.D.R.:
 Department of Pediatrics, Hospital Regional de Villahermosa, PEMEX, Villahermosa, Tabasco, Mexico

Cantú-Reyna C.:
 Genomi-k SAPI de CV. MonterreyNuevo León, Mexico

 Escuela de Medicina Tecnológico de Monterrey, Monterrey, Nuevo León, Mexico

Cruz-Camino H.:
 Genomi-k SAPI de CV. MonterreyNuevo León, Mexico

 Escuela de Biotecnología y Ciencias de la Salud, Tecnológico de Monterrey, Monterrey, Nuevo León, Mexico

Cervantes-Barragán D.E.:
 Department of Genetics, Hospital Central Sur de Alta Especialidad, PEMEX, Mexico City, Mexico

 Facultad Mexicana de Medicina, Universidad La Salle, Mexico City, Mexico
ISSN: 10967192
Editorial
ACADEMIC PRESS INC ELSEVIER SCIENCE, 525 B ST, STE 1900, SAN DIEGO, CA 92101-4495 USA, Estados Unidos America
Tipo de documento: Article
Volumen: 121 Número: 1
Páginas: 16-21
WOS Id: 000402024100003
ID de PubMed: 28302345