Valproic Acid (Depakote[Registered Trademark]) to Treat Autoimmune Lymphoproliferative Syndrome (ALPS)
Status:
Completed
Trial end date:
2011-09-01
Target enrollment:
Participant gender:
Summary
This study will test whether valproic acid (Depakote[Registered Trademark]) can shrink
enlarged lymph glands and spleen in patients with autoimmune lymphoproliferative syndrome
(ALPS). Depakote has been used for more than 30 years for treating various medical disorders
in adults and children, including migraine headaches, seizures and psychiatric disorders. In
animal studies, it was effective in shrinking both lymph nodes and spleen in animals with
conditions similar to ALPS.
People with ALPS who are between 2 and 70 years of age and who have had an enlarged spleen or
lymph glands for at least 1 year may be eligible for this study.
Participants take Depakote as a tablet or liquid or sprinkled on food twice a day for 16
weeks. The drug dose is increased slowly over the first 3 to 4 weeks until the maximum
tolerated dose is reached. Blood tests are done at 2, 4, 6, 8 and 10 weeks after starting the
drug and 1 week after the drug is stopped to check for treatment side effects. Valproic acid
blood levels will be checked during drug escalation, half way through therapy, and just
before the end of treatment. A physical examination and CT scan (or ultrasound of the abdomen
for patients who cannot undergo CT) are done before starting treatment and at the end of the
16-week treatment period to evaluate the response to treatment.
Patients who tolerate the treatment well and show shrinkage of the lymph glands or spleen may
be offered extended treatment for up to 1 year in consultation with their primary physician.
During the extended treatment period, blood tests are done at home every 6 to 8 weeks to
monitor for drug side effects. Follow up evaluation visits are scheduled at the NIH Clinical
Center every 3 months during the extended treatment period and 3, 6, and 12 months after
treatment has ended.