Source:http://linkedlifedata.com/resource/pubmed/id/11260086
Switch to
| Predicate | Object |
|---|---|
| rdf:type | |
| lifeskim:mentions | |
| pubmed:issue |
3
|
| pubmed:dateCreated |
2001-3-22
|
| pubmed:abstractText |
Twenty-seven consecutive Italian patients with Fanconi's anaemia (FA) underwent stem cell transplantation (SCT) from an HLA-matched related donor in 10 Italian centres of the Associazione Italiana Ematologia ed Oncologia Pediatrica (AIEOP), Gruppo Italiano di Trapianto di Midollo Osseo (GITMO). Twenty-two patients (81.5%) were conditioned with low-dose (median 20 mg/kg) cyclophosphamide (Cy) and thoraco-abdominal or total body irradiation (median dose 500 cGy), five patients (18.5%) with high-dose Cy (median 120 mg/kg). Graft-vs.-host disease (GVHD) prophylaxis was carried out with cyclosporin A in 26 cases; methotrexate (MTX) was added in eight cases. One patient received MTX alone. The median follow-up was 36 months. Ninety-two percent of patients (25 out of 27) engrafted, grade II and III acute GVHD occurred in 28% and 8% of patients, respectively, with chronic GVHD in 12.5%. Conditioning-related toxicity was mild: 4% of patients had grade III mucositis, 7.4% had grade II haemorrhagic cystitis, 14.8% had grade III liver toxicity and 11.1% had grade III renal toxicity. Transplant-related mortality at 12 months was 19.2%, survival at 36 months was 81.5%, with a median Karnofsky score of 100%. No late tumours occurred after a mean follow-up of the survivors of 5 years. None of the studied variables significantly affected the survival, including conditioning regimen, acute GVHD and clinical non-haematological phenotype. Among the studied variables, only conditioning regimens containing high-dose Cy and the presence of genital abnormalities were significantly (P < 0.05) associated with an increased rate of acute GVHD. Our study demonstrates that the Italian FA patients undergoing SCT from an HLA-matched related donor have a very good outcome. These patients, when compared with others of different ethnic origin who underwent allogeneic bone marrow transplantation, showed a less severe non-haematological phenotype, raising the possibility that this milder phenotype may have, at least in part, contributed to the outcome. Our data may provide a useful tool for further studies aiming to correlate genotype with phenotype.
|
| pubmed:language |
eng
|
| pubmed:journal | |
| pubmed:citationSubset |
IM
|
| pubmed:status |
MEDLINE
|
| pubmed:month |
Mar
|
| pubmed:issn |
0007-1048
|
| pubmed:author |
pubmed-author:Associazione Italiana di Ematologia ed Oncologia Pediatrica (AIEOP),
pubmed-author:BacigalupoAA,
pubmed-author:BalduzziAA,
pubmed-author:BukerGG,
pubmed-author:Di BartolomeoPP,
pubmed-author:Di GirolamoGG,
pubmed-author:DiniGG,
pubmed-author:DufourCC,
pubmed-author:Gruppo Italiano Trapianto di Midollo Osseo (GITMO),
pubmed-author:IorioA PAP,
pubmed-author:LocatelliFF,
pubmed-author:MadonEE,
pubmed-author:MessinaCC,
pubmed-author:MianoMM,
pubmed-author:PessionAA,
pubmed-author:PortaFF,
pubmed-author:RondelliRR
|
| pubmed:issnType |
Print
|
| pubmed:volume |
112
|
| pubmed:owner |
NLM
|
| pubmed:authorsComplete |
Y
|
| pubmed:pagination |
796-805
|
| pubmed:dateRevised |
2007-11-15
|
| pubmed:meshHeading |
pubmed-meshheading:11260086-Adolescent,
pubmed-meshheading:11260086-Bone Marrow Transplantation,
pubmed-meshheading:11260086-Child,
pubmed-meshheading:11260086-Child, Preschool,
pubmed-meshheading:11260086-Congenital Abnormalities,
pubmed-meshheading:11260086-Fanconi Anemia,
pubmed-meshheading:11260086-Female,
pubmed-meshheading:11260086-Genotype,
pubmed-meshheading:11260086-Graft vs Host Disease,
pubmed-meshheading:11260086-Growth Disorders,
pubmed-meshheading:11260086-Hematopoietic Stem Cell Transplantation,
pubmed-meshheading:11260086-Histocompatibility Testing,
pubmed-meshheading:11260086-Humans,
pubmed-meshheading:11260086-Italy,
pubmed-meshheading:11260086-Male,
pubmed-meshheading:11260086-Phenotype,
pubmed-meshheading:11260086-Pigmentation Disorders,
pubmed-meshheading:11260086-Retrospective Studies,
pubmed-meshheading:11260086-Transplantation, Homologous,
pubmed-meshheading:11260086-Transplantation Conditioning,
pubmed-meshheading:11260086-Treatment Outcome
|
| pubmed:year |
2001
|
| pubmed:articleTitle |
Stem cell transplantation from HLA-matched related donor for Fanconi's anaemia: a retrospective review of the multicentric Italian experience on behalf of AIEOP-GITMO.
|
| pubmed:affiliation |
Department of Paediatric Haematology-Oncology and BMT Unit, IRCCS G. Gaslini, Largo G. Gaslini 5, 16148 Genoa, Italy. carlodufour@ospedale-gaslini.ge.it
|
| pubmed:publicationType |
Journal Article,
Comparative Study,
Multicenter Study
|