Patients present nonmalignant lymphadenopathy and splenomegaly, autoimmune phenomena and in some cases even malignancy. Autoimmune manifestations include hemolytic anemia and thrombocytopenia. In rare cases, there are systemic manifestations like vasculitis, arthritis and glomerulonephritis. ALPS 0 patients have the severe form of disease, characterized by neonatal or prenatal onset with marked hyperlymphocytosis. Most cases of ALPS Ia are with delayed onset.
Diagnosis
Diagnostic recommendations
Additional Information
Diagnostic laboratories
Clinical:
Genetic:
Therapeutic options
- In patients with massive lymphoproliferation, chemotherapy with prednisone, cyclophosphamide and vincristine has been unsuccessful. Allogenic bone-marrow transplantation is the only cure for complete Fas deficiency. In case of hypersplenism, splenectomy is often performed. Severe autoimmune manifestation can be treated with steroids and cyclophosphamide.
- Lymphoproliferative disorders, eMedicine
Research programs, clinical trials
- European Initiative for Primary Immunodeficiencies
- APLSbase, Jennifer Puck, National Human Genome Research Institute
- Pyrimethamine to Treat Autoimmune Lymphoproliferative Syndrome, ClinicalTrial.gov
- Study of Autoimmune Lymphoproliferative Syndrome (ALPS), ClinicalTrial.gov
- Genetic Analysis of Immune Disorders, ClinicalTrial.gov