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Conference Paper: A randomized controlled study evaluating the safety and efficacy of deferiprone treatment in thalassemia major patients from Hong Kong
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TitleA randomized controlled study evaluating the safety and efficacy of deferiprone treatment in thalassemia major patients from Hong Kong
 
AuthorsHa, SY1
Chik, KW5
Ling, SC2
Lee, A3
Luk, CW4
Lam, C5
Ng, I1
Chan, G1
 
KeywordsCombined therapy
Deferiprone (L1)
Desferrioxamine (DFO)
Efficacy
Safety
Thalassemia major
 
Issue Date2006
 
PublisherInforma Healthcare. The Journal's web site is located at http://www.tandf.co.uk/journals/titles/03630269.asp
 
CitationHemoglobin, 2006, v. 30 n. 2, p. 263-274 [How to Cite?]
DOI: http://dx.doi.org/10.1080/03630260600642617
 
AbstractA controlled, open-label and randomized study was conducted to evaluate the safety and efficacy of the oral iron chelator deferiprone (L1) in thalassemia major patients from Hong Kong. Forty-nine patients were recruited in total (median age: 20 years; range: 8 to 40 years). The division of the patients was determined based on liver iron content and put into either the poorly-chelated (Group I) or well-chelated (Group II) groups. In Group I, 20 patients received combined therapy of L1 daily plus desferrioxamine (DFO), in a reduced frequency of twice weekly, while the control group consisted of 16 patients who were treated with DFO alone. In Group II, six patients received L1 only, while the control group consisted of seven patients treated with DFO alone. Only patients who participated for longer than 6 months were analyzed for efficacy (n = 44). The median study period was 18 months. Transient and mild gastrointestinal upset (31%), joint pain (15%) and liver enzyme elevation (23%) were the most common side effects noted for L1. No case of neutropenia was observed in this study. Serum ferritin (SF) levels showed significant decline in the poorly-chelated patients using combined therapy (L1 and reduced frequency DFO) as compared to those on DFO alone. However, their pre- and post-study liver iron content was not significantly different. Evaluation of the well-chelated group demonstrated no significant change in SF or liver iron content in both the study and control arms. We conclude that the short-term use of L1, with or without DFO, was safe and efficacious in our Chinese patient cohort. The long-term efficacy of reducing iron overload by treatment regimens including L1 requires further study. Copyright © Taylor & Francis Group, LLC.
 
ISSN0363-0269
2013 Impact Factor: 0.955
 
DOIhttp://dx.doi.org/10.1080/03630260600642617
 
ISI Accession Number IDWOS:000237623200016
 
ReferencesReferences in Scopus
 
DC FieldValue
dc.contributor.authorHa, SY
 
dc.contributor.authorChik, KW
 
dc.contributor.authorLing, SC
 
dc.contributor.authorLee, A
 
dc.contributor.authorLuk, CW
 
dc.contributor.authorLam, C
 
dc.contributor.authorNg, I
 
dc.contributor.authorChan, G
 
dc.date.accessioned2010-09-06T08:02:22Z
 
dc.date.available2010-09-06T08:02:22Z
 
dc.date.issued2006
 
dc.description.abstractA controlled, open-label and randomized study was conducted to evaluate the safety and efficacy of the oral iron chelator deferiprone (L1) in thalassemia major patients from Hong Kong. Forty-nine patients were recruited in total (median age: 20 years; range: 8 to 40 years). The division of the patients was determined based on liver iron content and put into either the poorly-chelated (Group I) or well-chelated (Group II) groups. In Group I, 20 patients received combined therapy of L1 daily plus desferrioxamine (DFO), in a reduced frequency of twice weekly, while the control group consisted of 16 patients who were treated with DFO alone. In Group II, six patients received L1 only, while the control group consisted of seven patients treated with DFO alone. Only patients who participated for longer than 6 months were analyzed for efficacy (n = 44). The median study period was 18 months. Transient and mild gastrointestinal upset (31%), joint pain (15%) and liver enzyme elevation (23%) were the most common side effects noted for L1. No case of neutropenia was observed in this study. Serum ferritin (SF) levels showed significant decline in the poorly-chelated patients using combined therapy (L1 and reduced frequency DFO) as compared to those on DFO alone. However, their pre- and post-study liver iron content was not significantly different. Evaluation of the well-chelated group demonstrated no significant change in SF or liver iron content in both the study and control arms. We conclude that the short-term use of L1, with or without DFO, was safe and efficacious in our Chinese patient cohort. The long-term efficacy of reducing iron overload by treatment regimens including L1 requires further study. Copyright © Taylor & Francis Group, LLC.
 
dc.description.natureLink_to_subscribed_fulltext
 
dc.identifier.citationHemoglobin, 2006, v. 30 n. 2, p. 263-274 [How to Cite?]
DOI: http://dx.doi.org/10.1080/03630260600642617
 
dc.identifier.doihttp://dx.doi.org/10.1080/03630260600642617
 
dc.identifier.epage274
 
dc.identifier.hkuros116179
 
dc.identifier.isiWOS:000237623200016
 
dc.identifier.issn0363-0269
2013 Impact Factor: 0.955
 
dc.identifier.issue2
 
dc.identifier.openurl
 
dc.identifier.pmid16798652
 
dc.identifier.scopuseid_2-s2.0-33745568162
 
dc.identifier.spage263
 
dc.identifier.urihttp://hdl.handle.net/10722/80098
 
dc.identifier.volume30
 
dc.languageeng
 
dc.publisherInforma Healthcare. The Journal's web site is located at http://www.tandf.co.uk/journals/titles/03630269.asp
 
dc.publisher.placeUnited Kingdom
 
dc.relation.ispartofHemoglobin
 
dc.relation.referencesReferences in Scopus
 
dc.rightsHemoglobin. Copyright © Informa Healthcare.
 
dc.subject.meshAdolescent
 
dc.subject.meshAdult
 
dc.subject.meshArthralgia - chemically induced
 
dc.subject.meshBiopsy, Needle
 
dc.subject.meshBlood Transfusion - adverse effects
 
dc.subject.meshChelation Therapy - adverse effects
 
dc.subject.meshChild
 
dc.subject.meshCombined Modality Therapy
 
dc.subject.meshDeferoxamine - administration & dosage - adverse effects - therapeutic use
 
dc.subject.meshDrug Eruptions - etiology
 
dc.subject.meshDrug Therapy, Combination
 
dc.subject.meshFemale
 
dc.subject.meshGastrointestinal Diseases - chemically induced
 
dc.subject.meshHong Kong
 
dc.subject.meshHumans
 
dc.subject.meshIron - analysis
 
dc.subject.meshIron Chelating Agents - administration & dosage - adverse effects - therapeutic use
 
dc.subject.meshIron Overload - drug therapy - etiology
 
dc.subject.meshLiver - chemistry - pathology
 
dc.subject.meshMale
 
dc.subject.meshPyridones - administration & dosage - adverse effects - therapeutic use
 
dc.subject.meshbeta-Thalassemia - drug therapy
 
dc.subjectCombined therapy
 
dc.subjectDeferiprone (L1)
 
dc.subjectDesferrioxamine (DFO)
 
dc.subjectEfficacy
 
dc.subjectSafety
 
dc.subjectThalassemia major
 
dc.titleA randomized controlled study evaluating the safety and efficacy of deferiprone treatment in thalassemia major patients from Hong Kong
 
dc.typeConference_Paper
 
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<contributor.author>Chik, KW</contributor.author>
<contributor.author>Ling, SC</contributor.author>
<contributor.author>Lee, A</contributor.author>
<contributor.author>Luk, CW</contributor.author>
<contributor.author>Lam, C</contributor.author>
<contributor.author>Ng, I</contributor.author>
<contributor.author>Chan, G</contributor.author>
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<subject>Combined therapy</subject>
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<subject>Desferrioxamine (DFO)</subject>
<subject>Efficacy</subject>
<subject>Safety</subject>
<subject>Thalassemia major</subject>
<subject.mesh>Adolescent</subject.mesh>
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<subject.mesh>Arthralgia - chemically induced</subject.mesh>
<subject.mesh>Biopsy, Needle</subject.mesh>
<subject.mesh>Blood Transfusion - adverse effects</subject.mesh>
<subject.mesh>Chelation Therapy - adverse effects</subject.mesh>
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<subject.mesh>Drug Eruptions - etiology</subject.mesh>
<subject.mesh>Drug Therapy, Combination</subject.mesh>
<subject.mesh>Female</subject.mesh>
<subject.mesh>Gastrointestinal Diseases - chemically induced</subject.mesh>
<subject.mesh>Hong Kong</subject.mesh>
<subject.mesh>Humans</subject.mesh>
<subject.mesh>Iron - analysis</subject.mesh>
<subject.mesh>Iron Chelating Agents - administration &amp; dosage - adverse effects - therapeutic use</subject.mesh>
<subject.mesh>Iron Overload - drug therapy - etiology</subject.mesh>
<subject.mesh>Liver - chemistry - pathology</subject.mesh>
<subject.mesh>Male</subject.mesh>
<subject.mesh>Pyridones - administration &amp; dosage - adverse effects - therapeutic use</subject.mesh>
<subject.mesh>beta-Thalassemia - drug therapy</subject.mesh>
<title>A randomized controlled study evaluating the safety and efficacy of deferiprone treatment in thalassemia major patients from Hong Kong</title>
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Author Affiliations
  1. The University of Hong Kong
  2. Princess Margaret Hospital Hong Kong
  3. Tuen Mun Hospital
  4. Queen Elizabeth Hospital Hong Kong
  5. Prince of Wales Hospital Hong Kong