Pulmonary, Gonadal, and Central Nervous System Status after Bone Marrow Transplantation for Sickle Cell Disease

Autor: James Barkovich, Lewis L. Hsu, J. Paul Scott, Jean E. Sanders, Zora R. Rogers, Melinda Patience, Irene Roberts, Paul R. Haut, Kwaku Ohene-Frempong, John T. Horan, Naynesh Kamani, Roger Giller, Mark C. Walters, Karen A. Hardy, Keith M. Sullivan, Sandie Edwards, David A. Margolis, Rupa Redding-Lallinger, Roswitha Dickerhoff, George R. Buchanan, John E. Levine, Thomas V. Adamkiewicz, Françoise Bernaudin, Nancy Bunin
Jazyk: angličtina
Rok vydání: 2010
Předmět:
Male
medicine.medical_specialty
Adolescent
Anemia
medicine.medical_treatment
Health Status
Graft vs Host Disease
chemical and pharmacologic phenomena
Hematopoietic stem cell transplantation
Anemia
Sickle Cell
Article
Pulmonary function testing
Donor Selection
Central Nervous System Diseases
HLA Antigens
immune system diseases
Internal medicine
hemic and lymphatic diseases
Medicine
Humans
Lung Diseases
Obstructive
Child
Stroke
Long-term follow-up
Bone Marrow Transplantation
Transplantation
Transplantation Chimera
Lung
business.industry
Donor selection
Histocompatibility Testing
Siblings
Gonadal Disorders
Graft Survival
Hematopoietic Stem Cell Transplantation
hemic and immune systems
Hematology
medicine.disease
Survival Analysis
Sickle cell anemia
medicine.anatomical_structure
Treatment Outcome
surgical procedures
operative
Immunology
Female
business
Follow-Up Studies
DOI: 10.17615/pj8b-5j59
Popis: 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.
Databáze: OpenAIRE
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