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Table 2 Approaches to inducing HbF

From: Genomic approaches to identifying targets for treating β hemoglobinopathies

Method Drug or locus Features References
Pharmacologic Hydroxyureaa Regulatory approval, inexpensive, ineffective in severe β thalassemia, inconsistent response [141]
  HQK-1001 (sodium 2,2-dimethylbutyrate) HbF increase of 5-21 % in HbE-β0 thalassemia [142]
  Decitabine ? Epigenetic modification, possible oral route [143]
  Pomalidomide Immunomodulatory agent [144]
  Scriptaid ? Epigenetic modification [145]
  SAHA ? Epigenetic modification [146]
  BCL11A See text See text
  MYB See text See text
  Direct repeats (DRED) repressors Point mutations cause HPFH, forced expression increases HbF in sickle mice [147, 148]
  LCR/HBG promoters See text See text
  1. aHydroxyurea is the sole agent with widespread regulatory approval. Decitabine, scriptaid and SAHA are histone deacetylase inhibitors and other drugs of this general class have also been associated with HbF induction