How is autoimmune lymphoproliferative syndrome (ALPS) treated?

Updated: Jul 29, 2019
  • Author: Akaluck Thatayatikom, MD, RhMSUS; Chief Editor: Harumi Jyonouchi, MD  more...
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Answer

The treatment key of ALPS depends upon the patient’s manifestations and the disease complications. Upon confirmation of a diagnosis of ALPS, patients should undergo counseling aimed at specifically addressing the natural history, the disease manifestations, and the risks and complications associated with ALPS and its treatment.

The patient and the family should be educated on the risks associated with significant cytopenias (anemia, thrombocytopenia, neutropenia) and other autoimmune diseases that can develop and require immediate medical attention, such as systemic constitutional symptoms, petechiae, or mucosal bleeding. The increased risk of lymphomas and other malignancies should be addressed, especially in ALPS with FAS mutations, and patients should be encouraged to seek further evaluation for any severe or sudden fluctuations in a lymph node or spleen size. The increased risk of severe infections, especially pneumococcal sepsis associated with splenectomy, which is compounded by lack of memory B cells and autoimmune neutropenia, should be discussed.

The massive lymphadenopathy seen in children with ALPS can evoke considerable anxiety in patients and families, and as a result, clinicians may feel inclined to treat these patients for cosmetic purposes alone. However, corticosteroids or immunosuppressive drugs (e.g., azathioprine, cyclosporine, or mycophenolate mofetil) do not consistently shrink the lymph nodes and spleens of ALPS patients, and their use to treat these patients for solely cosmetic purposes is not indicated. [45]

The surgical role in ALPS is limited to lymph node biopsies. Splenectomy for chronic refractory cytopenias should be avoided since it increases significant risk of sepsis and is often ineffective and rarely leads to permanent remission. Hematopoietic stem cell transplantation (HSCT), the only curative treatment, is considered only in severe clinical phenotypes with no response to immune suppressive medications. [6]   

Patients with ALPS are admitted to the hospital on a limited basis—generally, only to treat a critical care emergency (e.g., fever, sepsis, mucocutaneous bleeding, profound anemia, or thrombocytopenia or systemic autoimmune diseases). Conditions associated with ALPS, such as Hodgkin lymphoma and non-Hodgkin lymphoma, may require more extensive hospitalization. Other treatments are usually provided in an outpatient or home setting.

Outpatient care must be individualized. A team approach with appropriate consultants (see Consultations) should emphasize activities of daily living (to include school attendance), proper nutrition, and a healthy and positive attitude. Treatment teams should monitor medications used, as well as medication compliance, and admit patients to the hospital only for definitive medical or surgical treatment. Patients who have undergone splenectomy should wear medical alert bracelets or necklaces or carry wallet cards outlining their risk for sepsis.


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