Autoimmune Lymphoproliferative Syndrome Medication

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

Agents used to treat autoimmune lymphoproliferative syndrome (ALPS) include immunosuppressants and immune globulins.


Immunosuppressant Agents

Class Summary

Initial therapy for autoimmune hemolytic anemia (AIHA) or idiopathic thrombocytopenic purpura (ITP) includes corticosteroids. In refractory autoimmune cytopenias necessitating long-term steroid therapy, mycophenolate mofetil and tacrolimus have been shown to be effective steroid-sparing agents.

Prednisone (Deltasone, Rayos)

Prednisone is useful for treating inflammatory and allergic reactions; it may decrease inflammation by reversing increased capillary permeability and suppressing polymorphonuclear leukocyte (PMN) activity. It is the drug of choice for all adult patients with platelet counts below 50,000/µL. Asymptomatic patients with platelet counts higher than 20,000/µL or patients with counts of 30,000-50,000/µL with only minor purpura may not need therapy; withholding medical therapy may be appropriate for asymptomatic patients, regardless of platelet count.

Methylprednisolone (Solu-Medrol, Depo-Medrol, Medrol)

Methylprednisolone decreases inflammation by suppressing migration of PMNs and reversing increased permeability. It is used as the alternative glucocorticoid of choice for all patients with severe life-threatening bleeding or children with platelet counts below 30,000/µL. Careful observation without medical treatment may be appropriate in some asymptomatic children.

Prednisolone (Orapred, Pediapred, Millipred)

Corticosteroids act as potent inhibitors of inflammation. They may cause profound and varied metabolic effects, particularly in relation to salt, water, and glucose tolerance, in addition to their modification of the immune response of the body. Alternative corticosteroids may be used in equivalent dosage. It is used in all patients with severe life-threatening bleeding or children with platelet counts below 30,000/µL. Careful observation without medical treatment may be appropriate in some asymptomatic children.

Mycophenolate (CellCept, Myfortic)

Mycophenolate inhibits inosine monophosphate dehydrogenase (IMPDH) and suppresses de novo purine synthesis by lymphocytes, thereby inhibiting their proliferation. It inhibits antibody production. Two formulations are available; they are not interchangeable. The original formulation, mycophenolate mofetil (CellCept) is a prodrug that, once hydrolyzed in vivo, releases the active moiety, mycophenolic acid. A newer formulation, mycophenolic acid (Myfortic), is an enteric-coated product that delivers the active moiety.

Sirolimus (Rapamune)

Sirolimus inhibits a mammalian target of rapamycin (mTOR), a kinase that play a fundamental role in regulating the progression of the cell cycle and disrupts proliferation of T cells. Sirolimus monotherapy is a safe and effective steroid-sparing agent with rapid improvement of lymphoproliferation, autoimmunity and normalized biomarkers including Vit B12, IL-10, and soluble FASLG. A dose of 2.5mg/m2 daily can achieve a trough level of 5-15ng/ml. The well-known side effects are oral mucositis, hyperlipidemia, decreased renal function, myelosuppression, and drug-drug interaction.


Immune Globulin

Class Summary

High-dose (1-2 g/kg IV) immune globulin may be considered for concomitant use with pulse-dose corticosteroids in those with severe AIHA.

Immune globulin intravenous (Bivigam, Carimune NF, Octagam, Gammaplex, Gammagard, Privigen, Panzyga, Gamunex-C)

Immune globulin intravenous is given as a temporary measure to increase platelets. It neutralize circulating myelin antibodies through anti-idiotypic antibodies; down-regulates proinflammatory cytokines, including interferon gamma; blocks Fc receptors on macrophages; suppresses inducer T and B cells while augmenting suppressor T cells; blocks the complement cascade; promotes remyelination; and may increase immunoglobulin G (IgG) in cerebrospinal fluid (10% of cases).