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rdf:type |
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lifeskim:mentions |
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pubmed:issue |
6
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pubmed:dateCreated |
2003-2-28
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pubmed:abstractText |
Allogeneic bone marrow transplantation (BMT) from HLA-identical siblings is an accepted treatment for both thalassemia and sickle cell disease (SCD). However, it is associated with decided risk of both transplant-related mortality (TRM) and chronic graft-versus-host disease (GVHD). We analyzed 44 patients (median age, 5 years; range, 1-20 years) given an allogeneic related cord blood transplant for either thalassemia (n = 33) or SCD (n = 11). Thirty children were given cyclosporin A (CsA) alone as GVHD prophylaxis, 10 received CsA and methotrexate (MTX), and 4 patients received other combinations of immunosuppressive drugs. The median number of nucleated cells infused was 4.0 x 10(7)/kg (range, 1.2-10 x 10(7)/kg). No patient died and 36 of 44 children remain free of disease, with a median follow-up of 24 months (range, 4-76 months). Only one patient with SCD did not have sustained donor engraftment as compared with 7 of the 33 patients with thalassemia. Three of these 8 patients had sustained donor engraftment after BMT from the same donor. Four patients experienced grade 2 acute GVHD; only 2 of the 36 patients at risk developed limited chronic GVHD. The 2-year probability of event-free survival is 79% and 90% for patients with thalassemia and SCD, respectively. Use of MTX for GVHD prophylaxis was associated with a greater risk of treatment failure. Related CBT for hemoglobinopathies offers a good probability of success and is associated with a low risk of GVHD. Optimization of transplantation strategies could further improve these results.
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pubmed:grant |
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pubmed:language |
eng
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pubmed:journal |
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pubmed:citationSubset |
AIM
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pubmed:chemical |
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pubmed:status |
MEDLINE
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pubmed:month |
Mar
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pubmed:issn |
0006-4971
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pubmed:author |
pubmed-author:BernaudinFrançoiseF,
pubmed-author:BlattJulieJ,
pubmed-author:BrichardBenedicteB,
pubmed-author:BrochsteinJoel AJA,
pubmed-author:ErtemMehmetM,
pubmed-author:Eurocord Transplant Group,
pubmed-author:GarnierFedericoF,
pubmed-author:GiorgianiGiovannaG,
pubmed-author:GluckmanElianeE,
pubmed-author:GrafakosSteliosS,
pubmed-author:HautPaul RPR,
pubmed-author:IonescuIrinaI,
pubmed-author:KurtzbergJoanneJ,
pubmed-author:LiXiaxinX,
pubmed-author:LocatelliFrancoF,
pubmed-author:LubinBertram HBH,
pubmed-author:LutzPatrickP,
pubmed-author:MinieroRobertoR,
pubmed-author:NaglerArnonA,
pubmed-author:NugentDiane JDJ,
pubmed-author:RajaThirumalairajT,
pubmed-author:ReedWilliamW,
pubmed-author:RobertsIreneI,
pubmed-author:RochaVandersonV,
pubmed-author:RubinCharles MCM,
pubmed-author:TannoiaNunziaN,
pubmed-author:VermylenChristianeC,
pubmed-author:WaltersMark CMC,
pubmed-author:WillAndrew MAM,
pubmed-author:WoodardPaulP,
pubmed-author:YanivIsaacI
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pubmed:issnType |
Print
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pubmed:day |
15
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pubmed:volume |
101
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pubmed:owner |
NLM
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pubmed:authorsComplete |
Y
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pubmed:pagination |
2137-43
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pubmed:dateRevised |
2007-11-14
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pubmed:meshHeading |
pubmed-meshheading:12424197-Acute Disease,
pubmed-meshheading:12424197-Anemia, Sickle Cell,
pubmed-meshheading:12424197-Blood Platelets,
pubmed-meshheading:12424197-Child,
pubmed-meshheading:12424197-Child, Preschool,
pubmed-meshheading:12424197-Chronic Disease,
pubmed-meshheading:12424197-Cord Blood Stem Cell Transplantation,
pubmed-meshheading:12424197-Cyclosporine,
pubmed-meshheading:12424197-Disease-Free Survival,
pubmed-meshheading:12424197-Female,
pubmed-meshheading:12424197-Graft Survival,
pubmed-meshheading:12424197-Graft vs Host Disease,
pubmed-meshheading:12424197-Humans,
pubmed-meshheading:12424197-Immunosuppressive Agents,
pubmed-meshheading:12424197-Infant,
pubmed-meshheading:12424197-Male,
pubmed-meshheading:12424197-Methotrexate,
pubmed-meshheading:12424197-Neutrophils,
pubmed-meshheading:12424197-Survival Rate,
pubmed-meshheading:12424197-Thalassemia,
pubmed-meshheading:12424197-Transplantation, Homologous,
pubmed-meshheading:12424197-Treatment Outcome
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pubmed:year |
2003
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pubmed:articleTitle |
Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease.
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pubmed:affiliation |
Oncoematologia Pediatrica, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo, Università di Pavia, Italy. f.locatelli@smatteo.pv.it
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pubmed:publicationType |
Journal Article,
Research Support, U.S. Gov't, P.H.S.,
Research Support, Non-U.S. Gov't
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