Document Detail

Desensitization to hydroxycarbamide following long-term treatment of thalassaemia intermedia as observed in vivo and in primary erythroid cultures from treated patients.
MedLine Citation:
PMID:  20955403     Owner:  NLM     Status:  MEDLINE    
Hydroxycarbamide (HC) is a pharmacological agent capable of stimulating fetal haemoglobin (HbF) production during adult life. High levels of HbF may ameliorate the clinical course of β-thalassaemia and sickle cell disease. The efficacy of HC for the treatment of thalassaemia major and thalassaemia intermedia is variable. Although an increase of HbF has been observed in most patients, only some patients experience significant improvement in total haemoglobin levels. This study aimed to determine the effectiveness and safety of short- (1 year) and long-term (mean follow-up 68 months) HC treatment in 24 thalassaemia intermedia patients. Additionally, we evaluated if primary erythroid progenitor cells cultured from treated patients responded to HC treatment in a manner similar to that observed in vivo. Our results confirm a good response to HC after a short-term follow-up in 70% of thalassaemia intermedia patients and a reduction of clinical response in patients with a long follow-up. Erythroid cultures obtained from patients during treatment reproduced the observed in vivo response. Interestingly, haematopoietic stem cells from long-term treated patients showed reduced ability to develop into primary erythroid cultures some months before the reduction of the 'in vivo' response. The mechanism of this loss of response to HC remains to be determined.
Paolo Rigano; Alice Pecoraro; Roberta Calzolari; Antonio Troia; Santina Acuto; Disma Renda; Gaetano Restivo Pantalone; Aurelio Maggio; Rosalba Di Marzo
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Publication Detail:
Type:  Clinical Trial; Journal Article; Research Support, Non-U.S. Gov't     Date:  2010-10-19
Journal Detail:
Title:  British journal of haematology     Volume:  151     ISSN:  1365-2141     ISO Abbreviation:  Br. J. Haematol.     Publication Date:  2010 Dec 
Date Detail:
Created Date:  2010-11-12     Completed Date:  2011-01-03     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  0372544     Medline TA:  Br J Haematol     Country:  England    
Other Details:
Languages:  eng     Pagination:  509-15     Citation Subset:  IM    
Copyright Information:
© 2010 Blackwell Publishing Ltd.
U.O.C. Ematologia II, Ospedali Riuniti Villa Sofia-Cervello, Palermo, Italy.
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MeSH Terms
Cells, Cultured
Drug Administration Schedule
Drug Tolerance
Erythroid Precursor Cells / drug effects*
Erythropoiesis / drug effects
Fetal Hemoglobin / biosynthesis
Follow-Up Studies
Hematopoiesis, Extramedullary / drug effects
Hemoglobins / metabolism
Hydroxyurea / administration & dosage,  adverse effects,  therapeutic use*
Middle Aged
Treatment Outcome
Young Adult
beta-Thalassemia / blood,  drug therapy*,  genetics
Reg. No./Substance:
0/Hemoglobins; 127-07-1/Hydroxyurea; 9034-63-3/Fetal Hemoglobin

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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