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Bone marrow transplantation today.

Abstract

Today, bone marrow transplantation (BMT) is an established therapy. This statement is best verified by the number of BMTs performed. Between January 1990 and December 1992, 172 European teams in 26 countries carried out a total of 14,334 transplants. There were 6642 allogeneic transplants: 5513 BMT from an HLA-identical sibling donor, 370 from a non-identical family member, 88 from an identical twin donor and 671 from an unrelated volunteer donor. There were 7692 autologous transplants: 6577 autologous bone marrow, 777 peripheral-blood stem-cell and 338 combined bone-marrow and peripheral-blood stem-cell transplants. Indications were: leukaemias in 52% (7479), lymphoproliferative disorders in 29% (4125), solid tumours in 11% (1540), aplastic anaemia and thalassaemia in 3% (487) and inborn errors an miscellaneous disorders in the remaining 5% (703). The results of these transplants are not yet known. From previous analyses it can be expected that more than 50% of patients will be alive and well 10 years after BMT. The main factors influencing outcome are known; they depend on type, sub-type, stage of disease at time of transplant, the time from diagnosis to transplant and the conditioning regimen for all transplants. For allogeneic BMT, donor source, donor and recipient age, sex, donor/recipient sex combination, donor and recipient viral status, graft-versus-host disease prevention method and region are additional factors. Knowledge of these factors enables us today to estimate the potential risk and adjust the therapy for an individual patient.

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BibTeXRIS

A Gratwohl. 1994. Bone marrow transplantation today.. https://doi.org/10.1007/bf00355237

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A syndrome of irreversible leukoencephalopathy following pediatric allogeneic bone marrow transplantation.

BACKGROUND: Despite decreases in overall mortality following bone marrow transplantation (BMT), a number of complications such as neurotoxicity have been described and often associated with immunosuppressive agents. The syndrome of reversible posterior leukoencephalopathy has been described in patients receiving cyclosporin and FK-506. We report here a subset of children who developed a syndrome of previously undescribed irreversible leukoencephalopathy following allogeneic BMT. PATIENTS AND METHODS: Between 1996 and 2002, 138 pediatric patients received an allogeneic BMT at Lucile Salter Packard Children's Hospital at Stanford. Six cases of irreversible leukoencephalopathy were observed. Cases were defined as children who exhibited progressive and continued, severe neurologic deterioration lasting greater than 2 weeks and consistent with non-localizing, central nervous system abnormalities. Medical records and magnetic resonance images (MRIs) were reviewed. RESULTS: Median age of the affected patients at BMT was 7.8 years. All six received cyclosporine, and [corrected] one had elevated drug levels. Encephalopathy occurred at a median of 53 days (range 14-77) following BMT. Symptoms at onset of leukoenceophalopathy included confusion and altered mental status, sluggish pupillary responses, abnormal movements, and seizures. Two patients died during their neurologic decline. Four patients remain alive with persistent encephalopathy. MRI showed abnormalities in all patients including periventricular or subcortical white matter involvement in all, and basal ganglia lesions in three. CONCLUSIONS: We report a syndrome of irreversible neurologic deficits and cerebral white matter abnormalities following allogeneic BMT, yet not associated with elevated cyclosporin levels. A precise mechanism for this syndrome is lacking and warrants further consideration.

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