title: Bone marrow transplantation for childhood hematological disorders: a global pediatric approach in a twelve year single center experience.
abstract: One hundred and 43 consecutive pediatric patients (June 1985-December 1996) with at least 18 months of follow-up, were considered: most of the patients (111/143, 77.6%) underwent allogeneic BMT. The median follow-up was 5.7 years. Overall survival and 5 years EFS were 48.6% and 46.9%, respectively. For patients who underwent allogeneic BMT from HLA-identical siblings, the 5 years EFS for ALL was 75% in 1st CR, 60.4% in 2nd CR, 22.3% in > 2nd CR and 86.7% for AML in 1st CR. The EFS for Allo-BMT in "good" and "poor" prognosis patients was 68.6% and 21.8%, respectively (p value = 0.001). Early mortality in Allo-BMT patients was 17.7% between 1985-1990 and 10.3% between 1991-1996. Early treatment-related organ complications occurred mostly in patients who underwent BMT from an unrelated or a mismatched family donor. Late toxicity was evaluated in 57 patients (median follow-up of 82 months): none of the patients complained of significant late cardiac or respiratory dysfunction. With regards to growth, 18/57 patients (31.6%) lost more than two height centile channels. Three cases of thyroid neoplasms were observed. Evaluation of psychosocial functioning, studied in 39 patients who had at least 2 years of follow-up in CR, did not reveal any evident quality of life impairment. The possibility of curing childhood hematological malignancies is based on a global pediatric and multidisciplinary approach. A continuous need to improve results in terms of EFS and quality of life suggests that further multicenter prospective studies should be carried out.
authors.affiliation: Clinica Pediatrica, Ospedale San Gerardo di Monza, Italy.
authors.forename: C
authors.initials: C
authors.lastname: Uderzo
authors.forename: E
authors.initials: E
authors.lastname: Biagi
authors.forename: A
authors.initials: A
authors.lastname: Rovelli
authors.forename: A
authors.initials: A
authors.lastname: Balduzzi
authors.forename: R
authors.initials: R
authors.lastname: Schirò
authors.forename: D
authors.initials: D
authors.lastname: Longoni
authors.forename: C
authors.initials: C
authors.lastname: Arrigo
authors.forename: B
authors.initials: B
authors.lastname: Nicolini
authors.forename: L
authors.initials: L
authors.lastname: Placa
authors.forename: A
authors.initials: A
authors.lastname: Da Prada
authors.forename: L
authors.initials: L
authors.lastname: Mascaretti
authors.forename: G
authors.initials: G
authors.lastname: Giltri
authors.forename: S
authors.initials: S
authors.lastname: Galimberti
authors.forename: M G
authors.initials: MG
authors.lastname: Valsecchi
authors.forename: A
authors.initials: A
authors.lastname: Locasciulli
authors.forename: G
authors.initials: G
authors.lastname: Masera
mesh_terms: D000293:Adolescent; D000328:Adult; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000453:epidemiology; D006402:Hematologic Diseases / Q000628:therapy*; D006801:Humans; D007223:Infant; D008297:Male; D013997:Time Factors