دورية أكاديمية
أعرض في EDS

Hematopoietic stem cell transplantation for people with β-thalassaemia.

التفاصيل البيبلوغرافية
العنوان: Hematopoietic stem cell transplantation for people with β-thalassaemia.
المؤلفون: Sharma A; Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, Tennessee, USA., Jagannath VA; Department of Paediatrics, American Mission Hospital, Manama, Bahrain., Puri L; St. Jude Children's Research Hospital, Memphis, Tennessee 38105, USA.
المصدر: The Cochrane database of systematic reviews [Cochrane Database Syst Rev] 2021 Apr 21; Vol. 4. Cochrane AN: CD008708. Date of Electronic Publication: 2021 Apr 21.
نوع المنشور: Journal Article; Research Support, Non-U.S. Gov't; Systematic Review
اللغة: English
بيانات الدورية: Publisher: Wiley Country of Publication: England NLM ID: 100909747 Publication Model: Electronic Cited Medium: Internet ISSN: 1469-493X (Electronic) Linking ISSN: 13616137 NLM ISO Abbreviation: Cochrane Database Syst Rev Subsets: MEDLINE
أسماء مطبوعة: Publication: 2004- : Chichester, West Sussex, England : Wiley
Original Publication: Oxford, U.K. ; Vista, CA : Update Software,
مواضيع طبية MeSH: Hematopoietic Stem Cell Transplantation*, beta-Thalassemia/*therapy, Humans
مستخلص: Background: Thalassaemia is an autosomal recessive blood disorder, caused by mutations in globin genes or their regulatory regions, resulting in a reduced rate of synthesis of one of the globin chains that make up haemoglobin. In β-thalassaemia there is an underproduction of β-globin chains combined with excess of free α-globin chains. The excess free α-globin chains precipitate in red blood cells, leading to their increased destruction (haemolysis) and ineffective erythropoiesis. The conventional treatment is based on the correction of haemoglobin through regular red blood cell transfusions and treating the iron overload that develops subsequently with iron chelation therapy. Although, early detection and initiations of such supportive treatment has improved the quality of life for people with transfusion-dependent thalassaemia, allogeneic hematopoietic stem cell transplantation is the only widely available therapy with a curative potential. Gene therapy for β-thalassaemia has recently received conditional authorisation for marketing in Europe, and may soon become widely available as another alternative therapy with curative potential for people with transfusion-dependent thalassaemia. This is an update of a previously published Cochrane Review.
Objectives: To evaluate the effectiveness and safety of different types of hematopoietic stem cell transplantation, in people with transfusion-dependent β-thalassaemia.
Search Methods: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Haemoglobinopathies Trials Register comprising references identified from comprehensive electronic database searches and handsearches of relevant journals and abstract books of conference proceedings. We also searched online trial registries. Date of the most recent search: 07 April 2021.
Selection Criteria: Randomised controlled trials and quasi-randomised controlled trials comparing hematopoietic stem cell transplantation with each other or with standard therapy (regular transfusion and chelation regimen).
Data Collection and Analysis: Two review authors independently screened trials and had planned to extract data and assess risk of bias using standard Cochrane methodologies and assess the quality using GRADE approach, but no trials were identified for inclusion in the current review.
Main Results: No relevant trials were retrieved after a comprehensive search of the literature.
Authors' Conclusions: We were unable to identify any randomised controlled trials or quasi-randomised controlled trials on the effectiveness and safety of different types of hematopoietic stem cell transplantation in people with transfusion-dependent β-thalassaemia. The absence of high-level evidence for the effectiveness of these interventions emphasises the need for well-designed, adequately-powered, randomised controlled clinical trials.
(Copyright © 2021 The Cochrane Collaboration. Published by John Wiley & Sons, Ltd.)
التعليقات: Update of: Cochrane Database Syst Rev. 2016 Nov 30;11:CD008708. (PMID: 27900772)
References: Cochrane Database Syst Rev. 2020 Jul 3;7:CD007001. (PMID: 32617981)
Biol Blood Marrow Transplant. 2006 Jun;12(6):683-7. (PMID: 16737942)
Pediatr Rep. 2011 Jun 22;3 Suppl 2:e13. (PMID: 22053275)
Bull World Health Organ. 2008 Jun;86(6):480-7. (PMID: 18568278)
BMC Health Serv Res. 2013 Feb 05;13:45. (PMID: 23379888)
Transfusion. 2016 May;56(5):1038-45. (PMID: 27041389)
Nat Rev Genet. 2001 Apr;2(4):245-55. (PMID: 11283697)
Cochrane Database Syst Rev. 2016 Nov 30;11:CD008708. (PMID: 27900772)
Annu Rev Med. 2005;56:157-71. (PMID: 15660507)
Biol Blood Marrow Transplant. 2011 Jun;17(6):861-6. (PMID: 20870029)
Bone Marrow Transplant. 2008 Sep;42(5):319-27. (PMID: 18560410)
Bone Marrow Transplant. 2000 Aug;26(4):445-9. (PMID: 10982293)
Biol Blood Marrow Transplant. 2017 Apr;23(4):552-561. (PMID: 28065838)
Biol Blood Marrow Transplant. 2003 Aug;9(8):519-28. (PMID: 12931121)
N Engl J Med. 2014 Nov 13;371(20):1908-16. (PMID: 25390741)
Haematologica. 2004 Oct;89(10):1187-93. (PMID: 15477202)
Haematologica. 2014 May;99(5):811-20. (PMID: 24790059)
Drug Metab Dispos. 2001 Mar;29(3):264-7. (PMID: 11181493)
Chimerism. 2011 Jan;2(1):21-2. (PMID: 21547033)
Iran Red Crescent Med J. 2013 Feb;15(2):93-100. (PMID: 23682320)
Ann N Y Acad Sci. 2010 Aug;1202:149-54. (PMID: 20712786)
Int J Dermatol. 2009 Oct;48(10):1057-61. (PMID: 19785086)
Ann N Y Acad Sci. 2005;1054:78-91. (PMID: 16339654)
Bone Marrow Transplant. 2016 Apr;51(4):536-41. (PMID: 26752139)
Blood. 2013 Aug 8;122(6):1072-8. (PMID: 23692854)
Haematologica. 1995 Jul-Aug;80(4):300-4. (PMID: 7590497)
Bone Marrow Transplant. 2005 Nov;36(10):839-45. (PMID: 16151422)
Cold Spring Harb Perspect Med. 2012 May;2(5):a011825. (PMID: 22553502)
Lancet. 1982 Jul 31;2(8292):227-9. (PMID: 6124668)
Nan Fang Yi Ke Da Xue Xue Bao. 2012 May;32(5):691-4. (PMID: 22588927)
Bone Marrow Transplant. 1999 Jul;24(1):5-11. (PMID: 10435727)
Curr Opin Hematol. 2004 Nov;11(6):386-91. (PMID: 15548992)
Blood. 2004 Sep 1;104(5):1574-7. (PMID: 15142875)
N Engl J Med. 1999 Jul 8;341(2):99-109. (PMID: 10395635)
Hum Gene Ther. 2019 Jun;30(6):753-761. (PMID: 30700149)
BMJ. 1997 Sep 13;315(7109):629-34. (PMID: 9310563)
Am J Hematol. 2008 Jul;83(7):528-30. (PMID: 18383328)
Cochrane Database Syst Rev. 2011 Oct 05;(10):CD008708. (PMID: 21975785)
Biol Blood Marrow Transplant. 2013 Jan;19(1):62-8. (PMID: 22892550)
Pediatr Blood Cancer. 2013 Aug;60(8):1345-9. (PMID: 23424175)
Blood. 2013 Sep 26;122(13):2262-70. (PMID: 23958950)
Blood Rev. 2008 Mar;22(2):53-63. (PMID: 18039551)
Am J Hematol. 2017 Dec;92(12):1303-1310. (PMID: 28850704)
Blood. 1999 Feb 15;93(4):1164-7. (PMID: 9949158)
An Pediatr (Barc). 2013 Aug;79(2):75-82. (PMID: 23402775)
Blood. 2011 Sep 29;118(13):3479-88. (PMID: 21813448)
Lancet. 2004 Jul 10-16;364(9429):156-62. (PMID: 15246728)
Ann N Y Acad Sci. 2005;1054:186-95. (PMID: 16339665)
J Pak Med Assoc. 2008 Mar;58(3):107-10. (PMID: 18517111)
Cochrane Database Syst Rev. 2014 Oct 15;(10):CD008708. (PMID: 25316103)
Am J Hematol. 2013 May;88(5):409-15. (PMID: 23475638)
BMJ. 2003 Sep 6;327(7414):557-60. (PMID: 12958120)
تواريخ الأحداث: Date Created: 20210421 Date Completed: 20210531 Latest Revision: 20220423
رمز التحديث: 20231215
مُعرف محوري في PubMed: PMC8078520
DOI: 10.1002/14651858.CD008708.pub5
PMID: 33880750
قاعدة البيانات: MEDLINE
الوصف
تدمد:1469-493X
DOI:10.1002/14651858.CD008708.pub5