title: Bone marrow transplantation in adults with thalassemia: Treatment and long-term follow-up.
abstract: Current regular blood transfusion programs and chelation treatment have considerably improved survival of patients with thalassemia, which resulted in a larger proportion of adult patients. However, disease- and treatment-related complications in these patients progress over time, causing severe morbidity and shortened life expectancy. Stem cell transplantation still remains the only cure currently available for patients with thalassemia. This study updates transplant outcomes in 107 adult patients with median age of 22 years (range, 17-35 years) who received bone marrow transplantation (BMT) from human leukocyte antigen (HLA)-identical related donors between 1988 and 1996 (group A) and describes the results of BMT in 15 adult patients with median age of 21 years (range, 17-31 years) who were treated with a new treatment protocol (Protocol 26) between 1997 and 2003 (group B). The probability of survival, event-free survival, nonrejection mortality, and rejection for group A patients were 66%, 62%, 37%, and 4%, respectively, with a median follow-up of 12 years (range, 8.3-16.2 years). Group B patients treated with the new protocol had some improvement in thalassemia-free survival (67%) and lower transplant-related mortality (27%) than that of previous protocols. However, transplant-related mortality in these high-risk patients remains elevated. Current myeloablative BMT in adult patients is characterized by higher transplant-related toxicity due to an advanced phase of disease. Although this new approach to transplant adult patients with a reduced-dose intensity-conditioning regimen has improved thalassemia-free survival, transplant-related mortality in these high-risk patients remains elevated.
authors.affiliation: Mediterranean Institute of Hematology, International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Policlinico Tor Vergata, Rome, Italy. j.gaziev@fondazioneime.org
authors.forename: Javid
authors.initials: J
authors.lastname: Gaziev
authors.forename: Pietro
authors.initials: P
authors.lastname: Sodani
authors.forename: Paola
authors.initials: P
authors.lastname: Polchi
authors.forename: Marco
authors.initials: M
authors.lastname: Andreani
authors.forename: Guido
authors.initials: G
authors.lastname: Lucarelli
mesh_terms: D000293:Adolescent; D000328:Adult; D001379:Azathioprine / Q000008:administration & dosage; D016026:Bone Marrow Transplantation / Q000009:adverse effects / Q000401:mortality / Q000706:statistics & numerical data*; D002066:Busulfan / Q000008:administration & dosage; D015913:Chelation Therapy; D002985:Clinical Protocols; D003131:Combined Modality Therapy; D015897:Comorbidity; D003676:Deferoxamine / Q000627:therapeutic use; D018572:Disease-Free Survival; D017707:Erythrocyte Transfusion; D004921:Erythropoietin / Q000008:administration & dosage; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000453:epidemiology / Q000209:etiology / Q000517:prevention & control; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage; D016298:Hematopoietic Cell Growth Factors / Q000008:administration & dosage; D006486:Hemosiderosis / Q000453:epidemiology / Q000209:etiology / Q000628:therapy; D006801:Humans; D006918:Hydroxyurea / Q000008:administration & dosage; D007166:Immunosuppressive Agents / Q000008:administration & dosage; D007502:Iron Chelating Agents / Q000627:therapeutic use; D015993:Life Tables; D008103:Liver Cirrhosis / Q000150:complications; D008297:Male; D018962:Phlebotomy; D011183:Postoperative Complications / Q000401:mortality; D016019:Survival Analysis; D013789:Thalassemia / Q000150:complications / Q000188:drug therapy / Q000401:mortality / Q000601:surgery* / Q000628:therapy; D065227:Transfusion Reaction; D019172:Transplantation Conditioning / Q000379:methods / Q000401:mortality / Q000706:statistics & numerical data; D014184:Transplantation, Homologous / Q000009:adverse effects / Q000401:mortality / Q000706:statistics & numerical data; D016896:Treatment Outcome; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives
chemical_list: D016298:Hematopoietic Cell Growth Factors; D007166:Immunosuppressive Agents; D007502:Iron Chelating Agents; D004921:Erythropoietin; D016179:Granulocyte Colony-Stimulating Factor; D014740:Vidarabine; D002066:Busulfan; D003676:Deferoxamine; D001379:Azathioprine; C024352:fludarabine; D006918:Hydroxyurea