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Titolo:
TRANSPLANTATION FOR FANCONI ANEMIA - LONG-TERM FOLLOW-UP OF 50 PATIENTS TRANSPLANTED FROM A SIBLING DONOR AFTER LOW-DOSE CYCLOPHOSPHAMIDE AND THORACOABDOMINAL IRRADIATION FOR CONDITIONING
Autore:
SOCIE G; DEVERGIE A; GIRINSKI T; PIEL G; RIBAUD P; ESPEROU H; PARQUET N; MAAREK O; NOGUERA MH; RICHARD P; BRISON O; GLUCKMAN E;
Indirizzi:
HOP ST LOUIS,SERV GREFFE MOELLE,1 AVE CLAUDE VELLEFAUX F-75475 PARIS 10 FRANCE HOP ST LOUIS,SERV HEMATOL GREFFE MOELLE PARIS FRANCE HOP ST LOUIS,UNITE RECH BIOL CELLULES SOUCHES PARIS FRANCE INST GUSTAVE ROUSSY,DEPT RADIOTHERAPIE VILLEJUIF FRANCE UNIV PARIS 07,CTR BIOINFORMAT,INSERM,U444 PARIS FRANCE HOP ST LOUIS,CENT HEMATOL LAB PARIS FRANCE INST GUSTAVE ROUSSY,LAB GENET ONCOL,CNRS,URA 1967 VILLEJUIF FRANCE
Titolo Testata:
British Journal of Haematology
fascicolo: 1, volume: 103, anno: 1998,
pagine: 249 - 255
SICI:
0007-1048(1998)103:1<249:TFFA-L>2.0.ZU;2-Y
Fonte:
ISI
Lingua:
ENG
Soggetto:
BONE-MARROW TRANSPLANTATION; SEVERE APLASTIC-ANEMIA; THORACOABDOMINAL IRRADIATION; TRANSFORMATION; MALIGNANCIES; EXPERIENCE; RADIATION; LEUKEMIA; SEATTLE; REGIMEN;
Keywords:
FANCONIS ANEMIA; TRANSPLANTATION; LATE EFFECTS;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Science Citation Index Expanded
Citazioni:
32
Recensione:
Indirizzi per estratti:
Citazione:
G. Socie et al., "TRANSPLANTATION FOR FANCONI ANEMIA - LONG-TERM FOLLOW-UP OF 50 PATIENTS TRANSPLANTED FROM A SIBLING DONOR AFTER LOW-DOSE CYCLOPHOSPHAMIDE AND THORACOABDOMINAL IRRADIATION FOR CONDITIONING", British Journal of Haematology, 103(1), 1998, pp. 249-255

Abstract

We describe the long-term follow-up of 50 Fanconi's anaemia patients who were transplanted from a related donor with a median follow-up of >6 years. The survival estimate was 74.4% at 54 months and 58.5% at 100 months, All patients were conditioned with low-dose cyclophosphamideand thoraco-abdominal irradiation. Acute graft-versus-host disease (GVHD) of grade II or more developed in 26 patients and chronic GvHD developed in 30/43 (69.9%) patients, The survival of patients without chronic GVHD (n = 13) was 100%. In addition to chronic GVHD, 20 pre-transplant transfusions was shown to have an adverse impact on survival by multivariate analysis (relative risk = 7.08, P=0.0003). Prospective follow-up of growth and endocrine function could be performed in 31 patients. Of 20 boys, six have already reached normal puberty within the expected time. Among the 11 girls, three were at the pubertal age at the time of analysis. Growth retardation was common, whereas late complications (e.g. peripheral hypothyroidism, cataract) were rare. However,the most important long-term complication was the occurrence of cancer in seven patients (8-year projected incidence 24%), Among the 32 survivors, 27 (84.5%) had a normal and four a moderately reduced performance status, and all achieved complete engraftment with donor cells. Therefore transplantation was able to cure these patients who remain at high risk for developing late complications. Clearly, a genetic predisposition and chronic GvHD could have led to the development of these cancers, However, we cannot completely rule out irradiation as a cofactor in the genesis of these cancers, and therefore no longer use irradiation for the conditioning of Fanconi's anaemia patients.

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Documento generato il 03/06/20 alle ore 00:09:35