Identification of risk features for complication in Gaucher's disease patients: a machine learning analysis of the Spanish registry of Gaucher disease.

Journal: Orphanet journal of rare diseases
Published Date:

Abstract

BACKGROUND: Since enzyme replacement therapy for Gaucher disease (MIM#230800) has become available, both awareness of and the natural history of the disease have changed. However, there remain unmet needs such as the identification of patients at risk of developing bone crisis during therapy and late complications such as cancer or parkinsonism. The Spanish Gaucher Disease Registry has worked since 1993 to compile demographic, clinical, genetic, analytical, imaging and follow-up data from more than 400 patients. The aims of this study were to discover correlations between patients' characteristics at diagnosis and to identify risk features for the development of late complications; for this a machine learning approach involving correlation networks and decision trees analyses was applied.

Authors

  • Marcio M Andrade-Campos
    Grupo Español de Enfermedades de Depósito Lisosomal, Sociedad Española de Hematología y Hemoterapia, (GEEDL), Zaragoza, Spain.
  • Laura López de Frutos
    Grupo Español de Enfermedades de Depósito Lisosomal, Sociedad Española de Hematología y Hemoterapia, (GEEDL), Zaragoza, Spain.
  • Jorge J Cebolla
    Grupo de Investigación en Enfermedades Metabólicas y Hematológicas Raras (GIIS-012), Instituto Investigación Sanitaria Aragón, Zaragoza, Spain.
  • Irene Serrano-Gonzalo
    Fundación Española para el Estudio y Terapéutica de la Enfermedad de Gaucher y otras lisosomales (FEETEG), Zaragoza, Spain.
  • Blanca Medrano-Engay
    Fundación Española para el Estudio y Terapéutica de la Enfermedad de Gaucher y otras lisosomales (FEETEG), Zaragoza, Spain.
  • Mercedes Roca-Espiau
    Fundación Española para el Estudio y Terapéutica de la Enfermedad de Gaucher y otras lisosomales (FEETEG), Zaragoza, Spain.
  • Beatriz Gomez-Barrera
    Kampal Solutions, Universidad de Zaragoza, Zaragoza, Spain.
  • Jorge Pérez-Heredia
    Instituto de Biocomputación y Física de Sistemas Complejos (BIFI), Zaragoza, Spain.
  • David Iniguez
    Kampal Solutions, Universidad de Zaragoza, Zaragoza, Spain.
  • Pilar Giraldo
    Grupo Español de Enfermedades de Depósito Lisosomal, Sociedad Española de Hematología y Hemoterapia, (GEEDL), Zaragoza, Spain. giraldocastellano@gmail.com.