Autoimmune Lymphoproliferative Syndrome (ALPS) Treatment

There currently is no standard cure for ALPS. The disorder can be managed by treating low blood-cell counts (cytopenias) and other autoimmune diseases that occur in people with ALPS. The disease also can be managed by monitoring for and treating the excessive production of a type of white blood cell called a lymphocyte (lymphoproliferation), an enlarged spleen, and cancer of the lymph nodes (lymphoma).

Doctors closely observe lymphoproliferation in people with ALPS. They may conduct routine imaging tests to observe the lymph nodes. If lymphoma is suspected, the doctor may perform a biopsy—the removal of a small piece of tissue. Lymphomas in people with ALPS can be successfully treated with standard cancer-treatment protocols. The defective apoptosis underlying ALPS does not seem to hinder response to cancer treatment.

When lymphoproliferation causes complications such as airway obstruction, doctors may prescribe corticosteroids and other immunosuppressive medications. ALPS-related autoimmunity also can be treated by suppressing the immune system with medication. The corticosteroid prednisone is often given for this purpose. However, low blood-cell counts frequently recur after a short course of treatment with prednisone, requiring repeated doses of corticosteroids or the use of different types of immune-suppressing medications. Chronic low blood-cell counts often can be managed successfully with steroid-sparing approaches, including medications such as mycophenolate mofetil and sirolimus. In most cases, rare autoimmune complications of ALPS, such as hepatitis, glomerulonephritis, and uveitis, can be treated effectively with immune-suppressing medications. 

Spleen removal, or splenectomy, may be necessary in rare cases of difficult-to-control cytopenias. Unfortunately, many patients still struggle with low blood-cell counts after splenectomy. Additionally, lack of a spleen increases the risk of sepsis, a potentially fatal response to severe infection with common bacteria called pneumococcus. NIAID scientists found in 2014 that sepsis is a major cause of severe illness and death among people with ALPS-FAS, the most common form of ALPS. 

Content last reviewed on November 30, 2017