Engraftment Failure Following Bone Marrow Transplantation in Children with Thalassemia Major Using Busulfan and Cyclophosphamide Conditioning

• Published in: Pediatric hematology and oncology Vol. 14; no. 1; pp. 73 - 77
• Main Authors: Solh, H., Rao, K., Martins da Cunha, A., Padmos, A., Giri, N., Spence, D., Clink, H., Ernst, P.
• Format: Journal Article
• Language: English
• Published: Philadelphia, PA Informa UK Ltd 1997; Taylor & Francis
• Subjects: Anemias. Hemoglobinopathies; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy; beta-Thalassemia - therapy; Biological and medical sciences; Bone Marrow Transplantation; Bone marrow, stem cells transplantation. Graft versus host reaction; Busulfan - therapeutic use; Child; Child, Preschool; Cyclophosphamide - therapeutic use; Diseases of red blood cells; engraftment failure; Female; Graft Rejection; Hematologic and hematopoietic diseases; Humans; Immunosuppressive Agents - therapeutic use; Male; Medical sciences; Retrospective Studies; Statistics, Nonparametric; thalassemia; Transfusions. Complications. Transfusion reactions. Cell and gene therapy; Transplantation Conditioning - methods; Human; Hemoglobinopathy; Treatment efficiency; Adjuvant treatment; Alkanediol; Thalassemia; Hemopathy; Transplantation; Genetic disease; Alkanesulfonic acid; Cyclophosphamide; Oxazaphosphinane derivatives; Hemolytic anemia; Surgery; Nitrogen mustard; Bone marrow; Busulfan; Child
• ISSN: 0888-0018; 1521-0669
• DOI: 10.3109/08880019709030887

Thirteen children older than 3 years of age with β-thalassemia major underwent allogeneic bone marrow transplantation (BMT) from a full human leukocyte antigen (HLA) matched sibling donor in a single institution. These patients received busulfan (Bu) 16 mg/kg followed by cyclophosphamide (Cy) 200 mg/kg for conditioning. Eight of the 13 patients (Group 1) engrafted and have a median age of 13 years (range 5-15 years). The five patients (Group 2) who failed to engraft have a median age of 6 years (range 3-8 years). The association with the following factors was found to be statistically significant: age (older in Group 1), duration of nadir of white blood count (WBC) of . 1 × 109/L (longer in Group 1), and the dose of Bu administered to each patient calculated on the basis of body surface area (higher dose in Group 1). The high rate of engraftment failure (5 out of 13) may be related to the suboptimal systemic exposure of Bu in younger children leading to inadequate bone marrow ablation when the standard dose of 16 mg/kg is used.