Humanized mouse model of glucose 6-phosphate dehydrogenase deficiency for in vivo assessment of hemolytic toxicity

Proc Natl Acad Sci U S A. 2013 Oct 22;110(43):17486-91. doi: 10.1073/pnas.1310402110. Epub 2013 Oct 7.

Abstract

Individuals with glucose 6-phosphate dehydrogenase (G6PD) deficiency are at risk for the development of hemolytic anemia when given 8-aminoquinolines (8-AQs), an important class of antimalarial/antiinfective therapeutics. However, there is no suitable animal model that can predict the clinical hemolytic potential of drugs. We developed and validated a human (hu)RBC-SCID mouse model by giving nonobese diabetic/SCID mice daily transfusions of huRBCs from G6PD-deficient donors. Treatment of SCID mice engrafted with G6PD-deficient huRBCs with primaquine, an 8-AQ, resulted in a dose-dependent selective loss of huRBCs. To validate the specificity of this model, we tested known nonhemolytic antimalarial drugs: mefloquine, chloroquine, doxycycline, and pyrimethamine. No significant loss of G6PD-deficient huRBCs was observed. Treatment with drugs known to cause hemolytic toxicity (pamaquine, sitamaquine, tafenoquine, and dapsone) resulted in loss of G6PD-deficient huRBCs comparable to primaquine. This mouse model provides an important tool to test drugs for their potential to cause hemolytic toxicity in G6PD-deficient populations.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Aminoquinolines / adverse effects
  • Aminoquinolines / therapeutic use
  • Anemia, Hemolytic / blood
  • Anemia, Hemolytic / chemically induced
  • Anemia, Hemolytic / diagnosis*
  • Animals
  • Antimalarials / adverse effects
  • Antimalarials / therapeutic use
  • Chloroquine / adverse effects
  • Chloroquine / therapeutic use
  • Combined Modality Therapy
  • Dapsone / adverse effects
  • Dapsone / therapeutic use
  • Dose-Response Relationship, Drug
  • Doxycycline / adverse effects
  • Doxycycline / therapeutic use
  • Drug Evaluation, Preclinical / methods
  • Erythrocyte Count
  • Erythrocyte Transfusion / methods*
  • Female
  • Glucosephosphate Dehydrogenase Deficiency / blood
  • Glucosephosphate Dehydrogenase Deficiency / therapy*
  • Humans
  • Mefloquine / adverse effects
  • Mefloquine / therapeutic use
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • Primaquine / adverse effects
  • Primaquine / therapeutic use*
  • Pyrimethamine / adverse effects
  • Pyrimethamine / therapeutic use
  • Reproducibility of Results
  • Sensitivity and Specificity
  • Transplantation, Heterologous

Substances

  • Aminoquinolines
  • Antimalarials
  • tafenoquine
  • Chloroquine
  • Dapsone
  • pamaquine
  • Primaquine
  • Doxycycline
  • Mefloquine
  • 8-aminoquinoline
  • Pyrimethamine