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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

TitleA randomized controlled study evaluating the safety and efficacy of deferiprone treatment in thalassemia major patients from Hong Kong
Authors
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
Citation
Hemoglobin, 2006, v. 30 n. 2, p. 263-274 How to Cite?
Abstract
A 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.
Persistent Identifierhttp://hdl.handle.net/10722/80098
ISSN
2013 Impact Factor: 0.955
ISI Accession Number ID
References

 

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
DC FieldValueLanguage
dc.contributor.authorHa, SYen_HK
dc.contributor.authorChik, KWen_HK
dc.contributor.authorLing, SCen_HK
dc.contributor.authorLee, Aen_HK
dc.contributor.authorLuk, CWen_HK
dc.contributor.authorLam, Cen_HK
dc.contributor.authorNg, Ien_HK
dc.contributor.authorChan, Gen_HK
dc.date.accessioned2010-09-06T08:02:22Z-
dc.date.available2010-09-06T08:02:22Z-
dc.date.issued2006en_HK
dc.identifier.citationHemoglobin, 2006, v. 30 n. 2, p. 263-274en_HK
dc.identifier.issn0363-0269en_HK
dc.identifier.urihttp://hdl.handle.net/10722/80098-
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.en_HK
dc.languageengen_HK
dc.publisherInforma Healthcare. The Journal's web site is located at http://www.tandf.co.uk/journals/titles/03630269.aspen_HK
dc.relation.ispartofHemoglobinen_HK
dc.rightsHemoglobin. Copyright © Informa Healthcare.en_HK
dc.subjectCombined therapyen_HK
dc.subjectDeferiprone (L1)en_HK
dc.subjectDesferrioxamine (DFO)en_HK
dc.subjectEfficacyen_HK
dc.subjectSafetyen_HK
dc.subjectThalassemia majoren_HK
dc.subject.meshAdolescenten_HK
dc.subject.meshAdulten_HK
dc.subject.meshArthralgia - chemically induceden_HK
dc.subject.meshBiopsy, Needleen_HK
dc.subject.meshBlood Transfusion - adverse effectsen_HK
dc.subject.meshChelation Therapy - adverse effectsen_HK
dc.subject.meshChilden_HK
dc.subject.meshCombined Modality Therapyen_HK
dc.subject.meshDeferoxamine - administration & dosage - adverse effects - therapeutic useen_HK
dc.subject.meshDrug Eruptions - etiologyen_HK
dc.subject.meshDrug Therapy, Combinationen_HK
dc.subject.meshFemaleen_HK
dc.subject.meshGastrointestinal Diseases - chemically induceden_HK
dc.subject.meshHong Kongen_HK
dc.subject.meshHumansen_HK
dc.subject.meshIron - analysisen_HK
dc.subject.meshIron Chelating Agents - administration & dosage - adverse effects - therapeutic useen_HK
dc.subject.meshIron Overload - drug therapy - etiologyen_HK
dc.subject.meshLiver - chemistry - pathologyen_HK
dc.subject.meshMaleen_HK
dc.subject.meshPyridones - administration & dosage - adverse effects - therapeutic useen_HK
dc.subject.meshbeta-Thalassemia - drug therapyen_HK
dc.titleA randomized controlled study evaluating the safety and efficacy of deferiprone treatment in thalassemia major patients from Hong Kongen_HK
dc.typeConference_Paperen_HK
dc.identifier.openurlhttp://library.hku.hk:4550/resserv?sid=HKU:IR&issn=0363-0269&volume=30&issue=2&spage=263&epage=274&date=2006&atitle=A+randomized+controlled+study+evaluating+the+safety+and+efficacy+of+deferiprone+treatment+in+thalassemia+major+patients+from+Hong+Kongen_HK
dc.identifier.emailNg, I:iolng@hkucc.hku.hken_HK
dc.identifier.emailChan, G:gcfchan@hkucc.hku.hken_HK
dc.identifier.authorityNg, I=rp00335en_HK
dc.identifier.authorityChan, G=rp00431en_HK
dc.description.naturelink_to_subscribed_fulltext-
dc.identifier.doi10.1080/03630260600642617en_HK
dc.identifier.pmid16798652en_HK
dc.identifier.scopuseid_2-s2.0-33745568162en_HK
dc.identifier.hkuros116179en_HK
dc.relation.referenceshttp://www.scopus.com/mlt/select.url?eid=2-s2.0-33745568162&selection=ref&src=s&origin=recordpageen_HK
dc.identifier.volume30en_HK
dc.identifier.issue2en_HK
dc.identifier.spage263en_HK
dc.identifier.epage274en_HK
dc.identifier.isiWOS:000237623200016-
dc.publisher.placeUnited Kingdomen_HK
dc.identifier.scopusauthoridHa, SY=7202501115en_HK
dc.identifier.scopusauthoridChik, KW=35580472400en_HK
dc.identifier.scopusauthoridLing, SC=7102701299en_HK
dc.identifier.scopusauthoridLee, A=7405631431en_HK
dc.identifier.scopusauthoridLuk, CW=7005748744en_HK
dc.identifier.scopusauthoridLam, C=7402527629en_HK
dc.identifier.scopusauthoridNg, I=7102753722en_HK
dc.identifier.scopusauthoridChan, G=16160154400en_HK

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