Pulmonary, gonadal, and central nervous system status after bone marrow transplantation for sickle cell disease.

Publication Type:

Journal Article

Source:

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, Volume 16, Issue 2, p.263-72 (2010)

Keywords:

2010, Adolescent, Anemia, Sickle Cell, Bone Marrow Transplantation, Center-Authored Paper, Central Nervous System Diseases, Child, Clinical Research Division, Donor Selection, Female, Follow-Up Studies, Gonadal Disorders, Graft Survival, Graft vs Host Disease, Health Status, hematopoietic stem cell transplantation, Histocompatibility Testing, HLA Antigens, Humans, Lung Diseases, Obstructive, Male, Siblings, Survival Analysis, Transplantation Chimera, Treatment Outcome

Abstract:

We conducted a prospective, multicenter investigation of human-leukocyte antigen (HLA) identical sibling bone marrow transplantation (BMT) in children with severe sickle cell disease (SCD) between 1991 and 2000. To determine if children were protected from complications of SCD after successful BMT, we extended our initial study of BMT for SCD to conduct assessments of the central nervous system (CNS) and of pulmonary function 2 or more years after transplantation. In addition, the impact on gonadal function was studied. After BMT, patients with stroke who had stable engraftment of donor cells experienced no subsequent stroke events after BMT, and brain magnetic resonance imaging (MRI) exams demonstrated stable or improved appearance. However, 2 patients with graft rejection had a second stroke after BMT. After transplantation, most patients also had unchanged or improved pulmonary function. Among the 11 patients who had restrictive lung changes at baseline, 5 were improved and 6 had persistent restrictive disease after BMT. Of the 2 patients who had obstructive changes at baseline, 1 improved and 1 had worsened obstructive disease after BMT. There was, however, significant gonadal toxicity after BMT, particularly among female recipients. In summary, individuals who had stable donor engraftment did not experience sickle-related complications after BMT, and were protected from progressive CNS and pulmonary disease.