autologous blood and marrow transplantation - Blog Science ...

Autologous Stem Cell Transplantation
for Autoimmune Diseases:
Clinical Indications
Edward C. Keystone
The Center for Advanced Therapeutics, University of Toronto,
Toronto, Ontario, Canada
A number of autoimmune rheumatic diseases are known to cause substantial
morbidity and mortality despite current therapy. With significant improvements in
the technology of hematopoietic stem cell transplantation (HSCT), it is
increasingly possible to apply this modality to the treatment of these disorders. A
consensus report written on behalf of the European League Against Rheumatism
(EULAR) and the European Group for Blood and Marrow Transplantation
(EBMT) 1
have suggested that only diseases with an increased risk of mortality
should be considered. This list of rheumatologic disorders included progressive
systemic sclerosis (scleroderma) (PSS), autoimmune pulmonary hypertension,
necrotizing vasculitis, rheumatoid arthritis (RA), systemic lupus erythematosus
(SLE), antiphospholipid antibody syndrome, cryoglobulinemia, and several
pediatric disorders, such as scleroderma, dermatomyositis, and necrotizing
vasculitis. I will confine my remarks to patients with RA, SLE, and PSS.
The rationale for considering HSCT in the rheumatologic diseases listed above
stems not only from their considerable morbidity and their substantial economic
burden, on society but also their mortality. 2
RA has a significant morbidity with
almost 60% of patients unable to work within 10 years of disease. Life expectancy
is reduced on average about 10 years. With SLE, the 10-year survival rate is
approximately 75-90%. PSS has a 5-year mortality rate of 40%. With a mortality
rate now associated with autologous stem cell transplantation (autoSCT) of approximately
5% and a morbidity of less than 15%, the time is right for examining this
form of treatment for these serious life-threatening rheumatic disorders.
Until now there are only a few case reports 3
published concerning autoSCT in
RA, SLE, and PSS utilizing either peripheral blood or bone marrow stem cells. The
results were promising, with improvement or remission lasting in some cases as
long as 30 months. In almost all patients, improvement was temporary. The problem
with data generated to date is that, in most cases, autoSCT was performed with
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