Lupus Immunosuppressive/Immunomodulatory Therapy or Stem Cell Transplant (LIST)
Status:
Withdrawn
Trial end date:
1969-12-31
Target enrollment:
Participant gender:
Summary
Systemic lupus erythematosus, also known as lupus or SLE, is a chronic, multisystem,
autoimmune disease in which the body's internal system of defense attacks its own normal
tissues. This abnormal autoimmune response can result in damage to many parts of the body,
especially the skin, joints, lungs, heart, brain, intestines, and kidneys. Both genetic and
environmental risk factors are involved in the development of lupus, but these are poorly
understood.
SLE has an overall 10-year survival between 80 and 90%. However, we estimate that severe
lupus not responding to the usual available treatments has a 50% mortality rate in 10 years.
Kidney problems occur in 30% to 50% of lupus patients and may progress to kidney failure.
Kidney disease due to lupus occurs more frequently in African-Americans and Hispanics. Lupus
can affect many parts of the body and cause damage, but the severe form can result in death
from kidney disease; cardiovascular disease, specifically atherosclerosis; central nervous
system disease; and infections.
Currently, no single standard therapy for treatment of severe SLE exists. Usually physicians
prescribe an aggressive regimen of one or a combination of immunosuppressive/immunomodulatory
treatments. This approach to therapy for all forms of severe SLE derives largely from studies
of lupus nephritis. Current treatment, although effective in many people, are not effective
in all patients and are associated with drug-induced morbidity. The design of the control arm
for this study reflects the current status of treatment of SLE in the academic setting.
Investigators may choose from a list of commonly used and currently available
immunosuppressive/immunomodulatory treatments to optimize the treatment of their patients,
based on their past treatment history and response to those treatments. Study treatments may
consist of corticosteroids, cyclophosphamide (CTX), azathioprine, methotrexate, cyclosporine,
mycophenolate mofetil (MMF), plasmapheresis, intravenous immunoglobulin (IVIG), rituximab,
and leflunomide. Treatment may be changed as frequently and as necessary within the first
year of the study to control the manifestations of SLE in each patient. New therapies that
become available during the course of this trial may be added to the list of approved
medications for this study.
In response to the absence of a uniformly effective treatment for severe lupus, autologous
hematopoietic stem cell transplantation (HSCT) has been proposed as a potential therapy.
Hematopoietic stem cells are immature blood cells that can develop into all of the different
blood and immune cells the body uses. Researchers believe that resetting the immune system
may stop or slow down the progression of the disease. The main purpose of this study is to
compare two ways of treating SLE: 1) high-dose immunosuppressive therapy (HDIT) followed by
HSCT and 2) currently available immunosuppressive/immunomodulatory therapies.
Phase:
Phase 2
Details
Lead Sponsor:
National Institute of Allergy and Infectious Diseases (NIAID)