New approach for bone marrow transplantation in patients with class 3 thalassemia aged younger than 17 years

• Published in: Blood Vol. 104; no. 4; pp. 1201 - 1203
• Main Authors: Sodani, Pietro, Gaziev, David, Polchi, Paola, Erer, Buket, Giardini, Claudio, Angelucci, Emanuele, Baronciani, Donatella, Andreani, Marco, Manna, Marisa, Nesci, Sonia, Lucarelli, Barbarella, Clift, Reginald A., Lucarelli, Guido
• Format: Journal Article
• Language: English
• Published: Washington, DC Elsevier Inc 15-08-2004; The Americain Society of Hematology
• Subjects: Adolescent; Age Factors; Anemias. Hemoglobinopathies; Antineoplastic Agents - administration & dosage; Biological and medical sciences; Bone Marrow Transplantation - methods; Bone Marrow Transplantation - mortality; Chelating Agents - administration & dosage; Child; Child, Preschool; Clinical Protocols; Deferoxamine - administration & dosage; Diseases of red blood cells; Erythrocyte Transfusion; Female; Growth Substances - administration & dosage; Hematologic and hematopoietic diseases; Hematopoiesis - drug effects; Humans; Immunosuppressive Agents - administration & dosage; Male; Medical sciences; Prognosis; Recurrence; Risk Assessment; Survival Analysis; Thalassemia - mortality; Thalassemia - therapy; Antineoplastic agent; Graft(material); HLA-System; Stem cell; Major histocompatibility system; Hematopoietic cell; Homograft; Hemopathy; Transplantation; Epidemiology; Incidence; Hemolytic anemia; HLA-A-Locus; Surgery; Bone marrow; Graft; Busulfan; Class I histocompatibility antigen; Human; Hemoglobinopathy; Purine nucleotide; Alkanediol; Thalassemia; Genetic disease; Rejection; Alkanesulfonic acid; Alkylating agent; Oxazaphosphinane derivatives; Cyclophosphamide; Treatment; Antimetabolic; Donor; Nitrogen mustard; Risk factor; Fluorine Organic compounds; Histocompatibility
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood-2003-08-2800

When prepared for transplantation with busulfan (BU) 14 mg/kg and cyclophosphamide (CY) 120 to 160 mg/kg, patients with thalassemia in risk class 3, aged younger than 17 years, who receive transplants from HLA-identical donors, had a 30% incidence of transplant rejection with recurrence of thalassemia. This, relatively poor, outcome was ascribed to insufficient immune suppression or to inadequate eradication of the thalassemic marrow, or both. In an attempt to enhance both immune suppression and eradication of the thalassemic clones, hydroxyurea, azathioprine, and fludarabine were added to the BU and CY. This regimen, called protocol 26, was applied to 33 consecutive patients with class 3 thalassemia aged younger than 17 years and was well tolerated with 93% survival. The incidence of recurrent thalassemia after the transplantation decreased from 30% to 8%. (Blood. 2004;104:1201-1203)