TY - JOUR
T1 - Pulmonary, Gonadal, and Central Nervous System Status after Bone Marrow Transplantation for Sickle Cell Disease
AU - Walters, Mark C.
AU - Hardy, Karen
AU - Edwards, Sandie
AU - Adamkiewicz, Thomas
AU - Barkovich, James
AU - Bernaudin, Francoise
AU - Buchanan, George R.
AU - Bunin, Nancy
AU - Dickerhoff, Roswitha
AU - Giller, Roger
AU - Haut, Paul R.
AU - Horan, John
AU - Hsu, Lewis L.
AU - Kamani, Naynesh
AU - Levine, John E.
AU - Margolis, David
AU - Ohene-Frempong, Kwaku
AU - Patience, Melinda
AU - Redding-Lallinger, Rupa
AU - Roberts, Irene A G
AU - Rogers, Zora R.
AU - Sanders, Jean E.
AU - Scott, J. Paul
AU - Sullivan, Keith M.
N1 - Funding Information:
Financial disclosure: This investigation was supported by NIH Grants HL 36444 and HL 68091 .
PY - 2010/2
Y1 - 2010/2
N2 - 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.
AB - 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.
KW - Bone marrow transplantation
KW - Long-term follow-up
KW - Sickle cell anemia
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U2 - 10.1016/j.bbmt.2009.10.005
DO - 10.1016/j.bbmt.2009.10.005
M3 - Article
C2 - 19822218
AN - SCOPUS:75149170038
SN - 1083-8791
VL - 16
SP - 263
EP - 272
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
IS - 2
ER -