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

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.

• Authors: Patrick Hines, George J Dover, Linda M S Resar
• Journal: Pediatric blood & cancer (Pediatr Blood Cancer) Vol. 50 Issue 2 Pg. 357-9 (Feb 2008) ISSN: 1545-5017 [Electronic] United States
• PMID: 17253639 (Publication Type: Case Reports, Journal Article)
• Copyright: (c) 2007 Wiley-Liss, Inc.

Chemical References

• Phenylbutyrates
• Fetal Hemoglobin

Topics

• Administration, Oral
• Anemia, Sickle Cell (blood, drug therapy)
• Child
• Drug Administration Schedule
• Fetal Hemoglobin (metabolism)
• Humans
• Male
• Phenylbutyrates (administration & dosage)