1995
DOI: 10.1182/blood.v85.1.43.bloodjournal85143
|View full text |Cite
|
Sign up to set email alerts
|

Oral sodium phenylbutyrate therapy in homozygous beta thalassemia: a clinical trial

Abstract: Butyrate analogues have been shown to increase fetal hemoglobin (HbF) production in vitro and in vivo. Sodium phenylbutyrate (SPB), an oral agent used to treat individuals with urea-cycle disorders, has been shown to increase HbF in nonanemic individuals and in individuals with sickle cell disease. We have treated eleven patients with homozygous beta thalassemia (three transfusion dependent) and one sickle-beta- thalassemia patient with 20 g/d (forty 500-mg tablets) of SPB for 41 to 460 days. All patients show… Show more

Help me understand this report

Search citation statements

Order By: Relevance

Paper Sections

Select...
2
1

Citation Types

0
96
0
1

Year Published

1997
1997
2012
2012

Publication Types

Select...
10

Relationship

0
10

Authors

Journals

citations
Cited by 228 publications
(97 citation statements)
references
References 32 publications
0
96
0
1
Order By: Relevance
“…18,19 Several short-chain fatty acids (SCFAs) specifically induce transcription from the γ-globin gene promoter and in some patients can increase the efficiency of γ-globin mRNA translation. [20][21][22][23][24][25][26][27][28][29][30][31] These effects do not require histone deacetylase (HDAC) activity, but HDAC inhibitors often are potent γ-globin inducers. 32 However, those SCFADs that inhibit HDACs also inhibit cell proliferation, which is a limiting factor in thalassemia.…”
Section: Introductionmentioning
confidence: 99%
“…18,19 Several short-chain fatty acids (SCFAs) specifically induce transcription from the γ-globin gene promoter and in some patients can increase the efficiency of γ-globin mRNA translation. [20][21][22][23][24][25][26][27][28][29][30][31] These effects do not require histone deacetylase (HDAC) activity, but HDAC inhibitors often are potent γ-globin inducers. 32 However, those SCFADs that inhibit HDACs also inhibit cell proliferation, which is a limiting factor in thalassemia.…”
Section: Introductionmentioning
confidence: 99%
“…The next approach for the pharmacotherapy of thalassemia intermedia is to use drugs that stimulate the synthesis of fetal hemoglobin (HbF), including hydroxyurea and derivatives of butyric acid (10)(11)(12)(13)(14)(15)(16)(17)(18). Hydroxyurea is a well-known chemotherapeutic agent that has been used largely for the treatment of various myeloproliferative conditions.…”
mentioning
confidence: 99%
“…HDAC inhibitors increase acetylation of histones, thereby promoting transcriptional activation. Of the five classes of HDAC inhibitors, the butyrates are the most developed for clinical use in humans (Cremer et al 1977;Collins et al 1995;Vigushin and Coombes 2002). Because they penetrate the blood-brain barrier, the butyrates are candidates for HDAC inhibition therapy of disorders of the brain and spinal cord.…”
mentioning
confidence: 99%