Peripheral blood stem cell transplantation in children with beta-thalassemia

• Published in: Bone marrow transplantation (Basingstoke) Vol. 28; no. 11; pp. 1037 - 1040
• Main Authors: YESILIPEK, M. A, HAZAR, V, KÜPESIZ, A, KIZILÖRS, A, UGUZ, A, YEGIN, O
• Format: Journal Article
• Language: English
• Published: Basingstoke Nature Publishing Group 01.12.2001
• Subjects: Adolescent; Anemias. Hemoglobinopathies; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy; beta-Thalassemia - therapy; Biological and medical sciences; Blood; Blood diseases; Bone marrow; Bone marrow transplantation; Bone marrow, stem cells transplantation. Graft versus host reaction; Child; Child, Preschool; Children; Disease-Free Survival; Diseases of red blood cells; Female; Follow-Up Studies; Graft vs Host Disease - drug therapy; Graft vs Host Disease - prevention & control; Graft-versus-host reaction; Hematologic and hematopoietic diseases; Hematopoietic Stem Cell Transplantation - statistics & numerical data; Hematopoietic stem cells; Histocompatibility antigen HLA; Humans; Infant; Leukocytes (neutrophilic); Male; Medical sciences; Nuclear Family; Patients; Peripheral blood; Prophylaxis; Recurrence; Stem cell transplantation; Stem cells; Thalassemia; Tissue Donors; Transfusion; Transfusions. Complications. Transfusion reactions. Cell and gene therapy; Transplantation; Transplants & implants; Human; Hemoglobinopathy; Indication; Stem cell; Treatment efficiency; Hematopoietic cell; Homograft; Hemopathy; Genetic disease; Blood cell; Hemolytic anemia; Treatment; β-Thalassemia; Graft; Child
• DOI: 10.1038/sj.bmt.1703284
• ISSN: 0268-3369

Fifteen patients with beta-thalassemia received an allogeneic peripheral blood stem cell transplant. Median age was 3.5 years (1-15 years). Six were class I, four class II and five class III according to the Pesaro criteria. All of the donors were HLA-phenotypically identical (13 siblings and two parents). Nine patients were given BU + CY and six BU + CY plus ATG as conditioning. All patients received MTX (+1, +3, +6) and CsA (9-12 months) post transplant for GVHD prophylaxis. The median neutrophil and platelet engraftment times were day 12 and day 16, respectively. cGVHD was observed in three patients. Two patients died. Thirteen patients are well, and transfusion-independent 2-30 months after PSCT. No recurrences of thalassemia have been seen. Overall and event-free survival were 86.6%. In conclusion, we suggest that PSCT can be considered a safe and effective treatment for children with beta- thalassemia.