Pulmonary, Gonadal, and Central Nervous System Status after Bone Marrow Transplantation for Sickle Cell Disease
Year: 2009
Type: article
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.
Authors: Mark C. Walters, Karen Hardy, Sandie Edwards, Thomas Adamkiewicz, James Barkovich, Francoise Bernaudin, George R. Buchanan, Nancy Bunin, Roswitha Dickerhoff, Roger Giller, Paul R. Haut, John Horan, Lewis L. Hsu, Naynesh Kamani, John E. Levine, David Margolis, Kwaku Ohene-Frempong, Melinda Patience, Rupa Redding-Lallinger, Irene A.G. Roberts, Zora R. Rogers, Jean E. Sanders, J. Paul Scott, Keith M. Sullivan
Institutions: UCSF Benioff Children's Hospital, Emory University, University of California, San Francisco, Centre Hospitalier Universitaire Henri-Mondor, Hôpitaux Universitaires Henri-Mondor, The University of Texas Southwestern Medical Center, University of Pennsylvania, University of Bonn, University of Colorado Denver, Riley Hospital for Children, University of Rochester, St. Christopher's Hospital for Children, Children's National, Howard University, George Washington University, University of Michigan, Medical College of Wisconsin, University of North Carolina at Chapel Hill, Imperial College London, University of Washington, Fred Hutch Cancer Center, Duke University, Duke Medical Center
Language: English
Subfield: Genetics (clinical)
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $3,650
Funders: Yale University, University of Southern California, University of Miami, University of Pennsylvania, George Washington University, University of Minnesota, University of Washington, University of Rochester, Rheinische Friedrich-Wilhelms-Universität Bonn, National Institutes of Health, University of California, San Francisco, National Heart, Lung, and Blood Institute
OpenAlex ID: W1987823938