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

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. RobertsZora R. Rogers, Jean E. Sanders, J. Paul Scott, Keith M. Sullivan

Research output: Contribution to journalArticle

104 Scopus 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.

Original languageEnglish (US)
Pages (from-to)263-272
Number of pages10
JournalBiology of Blood and Marrow Transplantation
Volume16
Issue number2
DOIs
StatePublished - Feb 2010

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Keywords

  • Bone marrow transplantation
  • Long-term follow-up
  • Sickle cell anemia

ASJC Scopus subject areas

  • Transplantation
  • Hematology

Cite this

Walters, M. C., Hardy, K., Edwards, S., Adamkiewicz, T., Barkovich, J., Bernaudin, F., Buchanan, G. R., Bunin, N., Dickerhoff, R., Giller, R., Haut, P. R., Horan, J., Hsu, L. L., Kamani, N., Levine, J. E., Margolis, D., Ohene-Frempong, K., Patience, M., Redding-Lallinger, R., ... Sullivan, K. M. (2010). Pulmonary, Gonadal, and Central Nervous System Status after Bone Marrow Transplantation for Sickle Cell Disease. Biology of Blood and Marrow Transplantation, 16(2), 263-272. https://doi.org/10.1016/j.bbmt.2009.10.005