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, SY2
Chik, KW5
Ling, SC3
Lee, A1
Luk, CW4
Lam, C5
Ng, I2
Chan, G2
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
2011 Impact Factor: 1.304
2011 SCImago Journal Rankings: 0.131
DOIhttp://dx.doi.org/10.1080/03630260600642617
ISI Accession Number IDWOS:000237623200016
ReferencesReferences in Scopus
DC Field
Value
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
2011 Impact Factor: 1.304
2011 SCImago Journal Rankings: 0.131
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
Author Affiliations
  1. Tuen Mun Hospital
  2. The University of Hong Kong
  3. Princess Margaret Hospital Hong Kong
  4. Queen Elizabeth Hospital Hong Kong
  5. Prince of Wales Hospital Hong Kong