Abstract
The use of bone marrow transplantation for the treatment of refractory autoimmune
diseases (AD) is a new concept that is starting to emerge based on many experimental
data and some clinical observations obtained in the past 10 years after either allogeneic
or, more recently, autologous bone marrow transplantation. Although experimental data
demonstrate that allogeneic bone marrow transplantation is effective in treating refractory
AD, the treatment-related mortality of such a procedure in humans even in the absence
of underlying malignancy, is such that it should only be proposed for patients with
refractory AD and an underlying hematological disorder requiring bone marrow transplantation.
Autologous bone marrow transplantation, or peripheral hematopoietic stem cell transplantation
(HSCT), is currently performed for the treatment of various malignancies including
leukemia, lymphomas, or several solid tumors, and this procedure is associated with
a low, short-term mortality (<3–5%). Therefore, on the basis of both experimental
and clinical data, it has recently been considered an alternative approach to treating
autoimmune diseases that are refractory to conventional treatment. Since 1996, an
international consensus has emerged and helped to develop some national protocols
with clear inclusion criteria, especially in cases of systemic sclerosis, vasculitis,
lupus erythematosus, inflammatory myositis, and rheumatoid arthritis that are refractory
to conventional treatment. The aim of these phase I–II pilot studies is to define
the feasibility of this new procedure, which should still be considered as experimental.
Data reporting is an integral part of the protocol. For the first 145 patients reported
to the Basel registry, the mortality rate associated with the procedure is 8% (similar
to that for non-Hodgkin's lymphoma), well below the estimated death probability at
6 months and 5 years for most of the diseases thus far treated.
Keywords
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Article info
Publication history
Accepted:
May 11,
1999
Identification
Copyright
© 1999 Elsevier Science B.V. Published by Elsevier Inc. All rights reserved.
