Bone marrow transplant options and preferences in a sickle cell anemia cohort on chronic transfusions

• Published in: Pediatric blood & cancer 2012-04, Vol.58 (4), p.611-615
• Main Authors: Hansbury, Eileen N., Schultz, William H., Ware, Russell E., Aygun, Banu
• Format: Article
• Language: English
• Subjects: Adolescent; Anemia, Sickle Cell - therapy; Blood Transfusion; Bone Marrow Transplantation; Child; Child, Preschool; decision-making; Female; Histocompatibility Testing; Humans; Infant; Male; Retrospective Studies; sickle cell; transplantation; Transplantation, Homologous
• ISSN: 1545-5009; 1545-5017
• DOI: 10.1002/pbc.23304

Background Bone marrow transplantation (BMT) using human leukocyte antigen (HLA)‐matched sibling donors can be curative for children with sickle cell anemia (SCA). However, minimal data exist regarding availability of HLA‐identical matched siblings for transplant‐eligible children, and family interest in pursuing transplantation. Methods We retrospectively analyzed a pediatric SCA cohort receiving chronic transfusions between July 2004 and January 2011. Data were analyzed regarding the number of full siblings and half‐siblings, availability, and family interest in HLA testing the full siblings, and interest in proceeding with HLA‐matched transplantation. Results Among 113 patients, 46 (41%) had at least 1 full sibling and 40 (35%) had an unaffected full sibling who could serve as a BMT donor. The families of 23 of these patients (58%) agreed to HLA‐type sibling, 8 of whom (35%) were matched. Transfusion indications for families agreeing to HLA typing included stroke (46%) abnormal TCD (29%), acute chest syndrome (21%), and other CNS reasons (4%). Common reasons to decline HLA typing or transplantation included fear of the process, toxicities of the procedure, and comfort with current quality of life on transfusions. Only 8 of 113 (7%) were eligible for matched BMT, and only 3 (3%) underwent HLA‐matched transplantation. Two unmatched children received haploidentical transplantation. Conclusions Most families of children with SCA on chronic transfusions choose to proceed with HLA typing. However, when a matched sibling was identified, most families declined to proceed with matched‐sibling transplantation. Discussing BMT as a treatment option, offering HLA typing and identifying barriers may improve acceptance of this treatment modality. Pediatr Blood Cancer 2012; 58: 611–615. © 2011 Wiley Periodicals, Inc.