Faculty Collaboration Database

Pulmonary, gonadal, and central nervous system status after bone marrow transplantation for sickle cell disease. Biol Blood Marrow Transplant 2010 Feb;16(2):263-72

Date

10/14/2009

Pubmed ID

19822218

Pubmed Central ID

PMC2919571

DOI

10.1016/j.bbmt.2009.10.005

Scopus ID

2-s2.0-75149170038 (requires institutional sign-in at Scopus site)   155 Citations

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.

Author List

Walters MC, Hardy K, Edwards S, Adamkiewicz T, Barkovich J, Bernaudin F, Buchanan GR, Bunin N, Dickerhoff R, Giller R, Haut PR, Horan J, Hsu LL, Kamani N, Levine JE, Margolis D, Ohene-Frempong K, Patience M, Redding-Lallinger R, Roberts IA, Rogers ZR, Sanders JE, Scott JP, Sullivan KM, Multicenter Study of Bone Marrow Transplantation for Sickle Cell Disease

Author

David A. Margolis MD Chair, Professor in the Pediatrics department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Adolescent
Anemia, Sickle Cell
Bone Marrow Transplantation
Central Nervous System Diseases
Child
Donor Selection
Female
Follow-Up Studies
Gonadal Disorders
Graft Survival
Graft vs Host Disease
HLA Antigens
Health Status
Hematopoietic Stem Cell Transplantation
Histocompatibility Testing
Humans
Lung Diseases, Obstructive
Male
Siblings
Survival Analysis
Transplantation Chimera
Treatment Outcome