PAS-positive lymphocyte vacuoles can be used as diagnostic screening test for Pompe disease

J Inherit Metab Dis. 2010 Apr;33(2):133-9. doi: 10.1007/s10545-009-9027-4. Epub 2010 Jan 27.

Abstract

Screening of blood films for the presence of periodic acid-Schiff (PAS)-positive lymphocyte vacuoles is sometimes used to support the diagnosis of Pompe disease, but the actual diagnostic value is still unknown. We collected peripheral blood films from 65 untreated Pompe patients and 51 controls. Lymphocyte vacuolization was quantified using three methods: percentage vacuolated lymphocytes, percentage PAS-positive lymphocytes, and a PAS score depending on staining intensity. Diagnostic accuracy of the tests was assessed using receiver operating characteristic (ROC) curves. All three methods fully discerned classic infantile patients from controls. The mean values of patients with milder forms of Pompe disease were significantly higher than those of controls, but full separation was not obtained. The area under the ROC curve was 0.98 for the percentage vacuolated lymphocytes (optimal cutoff value 3; sensitivity 91%, specificity 96%) and 0.99 for the percentage PAS-positive lymphocytes and PAS score (optimal cutoff value 9; sensitivity 100%, specificity 98%). Our data indicate that PAS-stained blood films can be used as a reliable screening tool to support a diagnosis of Pompe disease. The percentage of PAS-positive lymphocytes is convenient for use in clinical practice but should always be interpreted in combination with other clinical and laboratory parameters.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't
  • Validation Study

MeSH terms

  • Adult
  • Aged
  • Child
  • Child, Preschool
  • Enzyme Replacement Therapy
  • Eosine Yellowish-(YS)
  • Female
  • Glycogen / metabolism
  • Glycogen Storage Disease Type II / diagnosis*
  • Glycogen Storage Disease Type II / drug therapy
  • Humans
  • Infant
  • Infant, Newborn
  • Lymphocytes / metabolism
  • Lymphocytes / pathology*
  • Male
  • Mass Screening / methods*
  • Mass Screening / standards
  • Methylene Blue
  • Middle Aged
  • Periodic Acid-Schiff Reaction / methods*
  • Periodic Acid-Schiff Reaction / standards
  • ROC Curve
  • Sensitivity and Specificity
  • Vacuoles / metabolism
  • Vacuoles / pathology*
  • Young Adult

Substances

  • May-Grunwald Giemsa
  • Glycogen
  • Methylene Blue
  • Eosine Yellowish-(YS)