Refractory Pure Red Cell Aplasia Manifesting as Deficiency of Adenosine Deaminase 2

J Pediatr Hematol Oncol. 2017 Jul;39(5):e293-e296. doi: 10.1097/MPH.0000000000000805.

Abstract

Primary progress has been made in the last 2 years, particularly in finding novel disease-causing genes for a number of autoinflammatory diseases and primary immunodeficiencies. Whole-exome sequencing has dramatically increased the pace at which causative genes are being discovered. CECR1 (Cat eye syndrome chromosome region, candidate 1) gene encodes adenosine deaminase 2 (ADA2) protein. Patients who carry CECR1 mutation(s) suffer from deficiency of ADA2 (DADA2). Here, we describe a patient with pure red cell aplasia discovered to have DADA2. We also review the literature on DADA2. This report will help raise awareness of physicians for this complex disease.

Publication types

  • Case Reports

MeSH terms

  • Adenosine Deaminase / deficiency*
  • Bone Marrow Transplantation / methods
  • Child, Preschool
  • Diagnosis, Differential
  • Humans
  • Intercellular Signaling Peptides and Proteins / deficiency*
  • Male
  • Red-Cell Aplasia, Pure / complications
  • Red-Cell Aplasia, Pure / diagnosis*
  • Sequence Analysis, DNA
  • Severe Combined Immunodeficiency / diagnosis*

Substances

  • Intercellular Signaling Peptides and Proteins
  • ADA2 protein, human
  • Adenosine Deaminase