Pyrimethamine and Sulfadoxine for Treatment of Autoimmune Lymphoproliferative Syndrome
Status:
Completed
Trial end date:
2003-03-01
Target enrollment:
Participant gender:
Summary
This study will evaluate the safety and effectiveness of an antibiotic called Fansidar on
autoimmune lymphoproliferative syndrome (ALPS). Patients with ALPS have enlarged lymph
glands, spleen and/or liver, abnormal blood cell counts and overactive immune function.
Current treatments are aimed at suppressing the immune system and improving symptoms, such as
anemia (low red blood cell count) and low white blood cell and platelet counts. These
treatments, however, are only partially effective and may have complications. Fansidar is a
combination of two drugs, sulfadoxine and pyrimethamine, that is used to treat or prevent
parasitic infections such as malaria. Recently a child with ALPS who was treated with
Fansidar for a different illness had a marked shrinkage of the lymph organs. This study will
examine whether Fansidar can shrink the lymph glands or spleen in patients with ALPS.
Patients with ALPS between the ages of 4 and 70 years who have had lymph gland enlargement
for at least 1 year and are not allergic to sulfa drugs may be eligible for this study.
Candidates will be screened with a medical history and physical examination and blood tests.
Females of reproductive age will have a urine pregnancy test.
Participants will be evaluated at the NIH Clinical Center in Bethesda, MD, with blood tests
and a computed tomography (CT) scan of the lymph nodes. For the CT scan, the patient lies on
a table during an X-ray scan of the neck, part of the chest, and, if the spleen has not been
removed, the stomach area.
When these baseline tests are completed, patients will be given Fansidar pills to take once a
week for 12 weeks. The dosage will be increased after 2 weeks and again after 4 weeks. At 2,
4, 6, 8 and 10 weeks after starting the treatment and 2 weeks after the last dose, patients
will have blood drawn to check for possible side effects of therapy. Women will have a repeat
urine pregnancy test at week 6 of treatment.
Within a week before completing treatment or after completing treatment, patients will return
to NIH for a history, physical examination, blood tests and CT scan. Patients who responded
well to treatment will be offered to return to NIH again 3, 6 and 12 months later to repeat
the evaluations. If ALPS symptoms recur during this time, patients will be offered another
12-week course of Fansidar and the procedure, including the 3, 6 and 12-month evaluations
will be repeated again. If symptoms recur again, patients will be asked to resume Fansidar
for 6 months or longer, with doses adjusted as needed. During this time, patients will be
seen at NIH every 12 weeks for evaluation and blood will be drawn by the patient's private
physician every 6 weeks or 2 and 4 weeks after the dose is increased to check for side
effects.
Phase:
Phase 1
Details
Lead Sponsor:
National Institute of Allergy and Infectious Diseases (NIAID)
Treatments:
Fanasil, pyrimethamine drug combination Pyrimethamine Sulfadoxine