Pulsed-dosing with oral sodium phenylbutyrate increases hemoglobin F in a patient with sickle cell anemia

Research output: Contribution to journalArticlepeer-review

Abstract

Increasing hemoglobin F (HbF) appears to be beneficial for patients with sickle cell anemia. We previously demonstrated that daily, oral sodium phenylbutyrate (OSPB) induces HbF synthesis in pediatric and adult patients with hemoglobin SS (HbSS). The high doses and need for daily therapy, however, have limited its use. Here, we report a patient treated with pulsed-dosing of OSPB for over 3 years. This patient developed a modest, but sustained elevation in HbF over the course of therapy without side effects. Although larger studies are needed, this case demonstrates that pulsed-dosing with OSPB enhances HbF synthesis.

Original languageEnglish (US)
Pages (from-to)357-359
Number of pages3
JournalPediatric Blood and Cancer
Volume50
Issue number2
DOIs
StatePublished - Feb 1 2008

Keywords

  • Hemoglobin F
  • Pulsed-dosing
  • Sickle cell anemia
  • Sodium phenylbutyrate

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

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