Rituximab may be considered medically necessary for the following off-label indications:
• the following autoimmune hemolytic anemias (AIHA):
o warm AIHA in glucocorticoid-refractory or glucocorticoid-dependent patients;
o cold agglutination syndrome;
• thrombotic thrombocytopenic purpura in patients with refractory disease or relapse (ie, lack of response to plasma exchange therapy and glucocorticoids);
• Churg-Strauss syndrome (eosinophilic granulomatosis with polyangiitis):
o first-line treatment in combination with glucocorticoids for patients with severe (organ-threatening) disease;
o add-on therapy for treatment-refractory disease;
• factor inhibitors in patients with hemophilia who are refractory to conventional first-line treatments (eg, immune tolerance induction, glucocorticoids with or without cyclophosphamide), preferably as add-on therapy
• add-on therapy for patients with hepatitis C virus-associated cryoglobulinemic vasculitis who have:
o active disease resistant to antiviral drugs; or
o severe or life-threatening cryoglobulinemic vasculitis;
• multicentric Castleman disease (first- or second-line therapy);
• primary Sjögren syndrome that is refractory to glucocorticoids and other immunosuppressive agents;
• add-on therapy for systemic lupus erythematosus refractory to standard first-line treatment;
• add-on therapy for lupus nephritis refractory to standard first-line treatment regimens;
• systemic sclerosis (scleroderma) in patients refractory to first-line treatment;
• neuromyelitis optica for relapse prevention;
• idiopathic membranous nephropathy;
• glucocorticoid-refractory chronic graft-versus-host disease; and
• desensitization of human leukocyte antigen-sensitized renal transplant candidates before transplantation.
• Moderate to severe Pemphigus Vulgaris (PV) in adult patients
Rituximab is investigational for all other nononcologic uses, including but not limited to:
• idiopathic thrombocytopenic purpura in patients who do not respond to first-line treatments;
• paroxysmal cold hemoglobinuria;
• mixed connective tissue disease;
• multiple sclerosis;
• treatment of myasthenia gravis;
• treatment of minimal change disease;
• prophylaxis for graft-versus-host disease;
• induction immunosuppressive therapy for kidney transplantation;
• induction immunosuppressive therapy for heart transplantation
• treatment of antibody-mediated rejection in solid organ transplant recipients; and
• treatment of antibody-mediated rejection after pancreatic islet transplantation.
