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Neurology 2002;59:1895-1904
© 2002 American Academy of Neurology

Severe neurologic complications after hematopoietic stem cell transplantation in children

M. Faraci, MD, E. Lanino, MD, G. Dini, MD, M.P. Fondelli, MD, G. Morreale, MD, S. Dallorso, MD, C. Manzitti, MD, M.G. Calevo, PhD, R. Gaggero, MD, E. Castagnola, MD and R. Haupt, MD

From the Department of Hematology/Oncology, Bone Marrow Transplant Unit (Drs. Faraci, Lanino, Dini, Morreale, Dallorso, and Manzitti), Neuroradiology (Dr. Fondelli), Scientific Directorate, Epidemiology and Biostatistics Section (Drs. Calevo and Haupt), Neurology (Dr. Gaggero), and Infectious Diseases Unit (Dr. Castagnola), G. Gaslini Children’s Research Institute, Genova, Italy.

Address correspondence and reprint requests to Dr. Maura Faraci, Department of Hematology/Oncology, Bone Marrow Transplant Unit, G. Gaslini Children’s Research Institute, Largo G. Gaslini, 5, 16147 Genova, Italy; e-mail: maurafaraci{at}ospedale-gaslini.ge.it

Objective: To describe and evaluate the incidence and risk factors of severe neurologic events (SNE) in pediatric recipients of allogeneic or autologous hematopoietic stem cell transplantation (HSCT) for hematologic or nonhematologic diseases.

Methods: Retrospective analysis of 272 consecutive children admitted to the G. Gaslini Children’s Research Institute and given HSCT (70 from unrelated donors, 115 from related donors, and 87 autologous) between June 1985 and January 2001.

Results: Thirty-seven children (13.6%) developed SNE after a median of 90 days (range, 5 days to 8.8 years) after HSCT. Cyclosporine A (CSA) neurotoxicity was the most frequent SNE (n = 21), followed by irradiation or chemotherapy injury (n = 7), CNS infections (n = 7), cerebrovascular events (n = 3), and immune-mediated etiology SNE (n = 2). Eleven patients (30%) died because of the neurologic complications. Type of HSCT, treatment with total body irradiation (TBI), acute graft-vs-host disease (GvHD), GvHD >grade 2, and treatment with CSA were associated with a significant increased risk of SNE.

Conclusions: Severe neurologic complications are frequent (14%) among children receiving HSCT, causing 8.5% of deaths after transplant. Transplant from allogeneic donor, especially if unrelated, the development of severe acute GvHD grade >2, and the use of TBI in the preparative regimen are the main risk factors for such complications.


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