title: Long-term results of survival in patients with thalassemia major treated with bone marrow transplantation.
abstract: Allogeneic bone marrow transplantation (BMT) is the only available curative approach for thalassemia major, although long-term morbidity and mortality are not established. The aim of this study was to assess the long-term clinical and hematological results in children and adults with thalassemia major treated with BMT. We analyzed the outcome of 115 patients (median age 9 years, range 11 months to 28 years) with thalassemia major undergoing BMT from a related donor between 1983 and 2006. All patients received the same protocol, consisting of busulfan and cyclophoshamide as conditioning therapy and cyclosporin (CSA) alone or CSA and methotrexate for graft-versus-host disease (GvHD) prophylaxis. The cumulative probability of graft rejection was 6.7%. The transplant-related mortality at 1 year was 8.7%. The 20-year Kaplan-Meier estimate of overall survival and disease-free survival was 89.2% and 85.7%, respectively. Ninety-nine patients out of 103 survivors were in excellent clinical and hematological conditions at last visit following a median follow-up of 15 years (range, 1-24 years) with the exception of two patients who had invalidating chronic GvHD. This study conducted with a large cohort of patients and covering a long period of observation time, showed BMT to be curative for the majority of patients with thalassemia major. The impact of long-term transplant-related sequelae was very limited.
authors.affiliation: Unità di Terapia Intensiva Ematologica per il Trapianto Emopoietico, Dipartimento di Ematologia, Ospedale Civile, Pescara, Italy. pescaratmo@virgilio.it
authors.forename: Paolo
authors.initials: P
authors.lastname: Di Bartolomeo
authors.forename: Stella
authors.initials: S
authors.lastname: Santarone
authors.forename: Erminia
authors.initials: E
authors.lastname: Di Bartolomeo
authors.forename: Paola
authors.initials: P
authors.lastname: Olioso
authors.forename: Pasqua
authors.initials: P
authors.lastname: Bavaro
authors.forename: Gabriele
authors.initials: G
authors.lastname: Papalinetti
authors.forename: Paolo
authors.initials: P
authors.lastname: Di Carlo
authors.forename: Franco
authors.initials: F
authors.lastname: Papola
authors.forename: Antonio
authors.initials: A
authors.lastname: Nicolucci
authors.forename: Marta
authors.initials: M
authors.lastname: Di Nicola
authors.forename: Antonio
authors.initials: A
authors.lastname: Iacone
mesh_terms: D000293:Adolescent; D000328:Adult; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D015996:Survival Rate; D013789:Thalassemia / Q000453:epidemiology / Q000601:surgery; D013997:Time Factors