Appeal type: Experimental/Investigational
Decision: Upheld

Diagnosis: Sarcoidosis
Treatment: Xeljanz
The insurer denied Xeljanz.
The determination is overturned.

This is a female patient with a medical history of lymph node, osseous and symptomatic pulmonary involvement of sarcoidosis. The patient has tried and failed multiple therapies including Remicade, CellCept, methotrexate, Imuran and prednisone. Based on the failure of first, second- and third-line therapies for her sarcoidosis, the provider is requesting Xeljanz for treatment in this refractory case. At issue is whether the Xeljanz 11 milligrams (mg) is likely to be more beneficial than any standard treatment or treatments for the insured's life threatening or disabling condition or disease.

The proposed health service or treatment Xeljanz 11 mg is likely to be more beneficial than any standard treatment or treatments for the insured's life threatening or disabling condition or disease.

As per the clinical documentation, the patient has lymph node, osseous and symptomatic pulmonary involvement of sarcoidosis. The patient has tried and failed multiple therapies including Remicade, CellCept, methotrexate, Imuran and prednisone. Sarcoidosis is a multi-system disease of unknown etiology characterized by granuloma formation in various organs, such as the lung, lymph and heart in this patient. Corticosteroids are the first-line treatment, but some patients may not respond or tolerate them. That is when second-line treatments, immunosuppressants (steroids sparing medications), should be considered. The current American Thoracic Society (ATS), the European Respiratory Society (ERS) and the World Association of Sarcoidosis and Other Granulomatous Disorders (WASOG) guidelines recommend that on the basis of safety and efficacy, the immunosuppressants Methotrexate and Azathioprine (Imuran) as the preferred agents for most patients. Third-line treatment for refractory sarcoidosis, a rare condition, usually requires biologic therapy, such as Remicade, for treatment. There are currently no guidelines or recommendations regarding refractory sarcoidosis. At this time based on the clinical documentation provided, the patient has tried and failed first, second- and third-line therapies for her sarcoidosis and the provider is requesting Xeljanz for treatment in this refractory case.

Over the past few years, Janus kinase (JAK) inhibitors, such as Xeljanz, have emerged as a promising new treatment approach for patients with both cutaneous and internal organ sarcoidosis. Xeljanz has been shown in multiple case studies to be safe and efficacious for use in sarcoidosis patients. A recent case study by Damsky et al demonstrated the efficacious use of Xeljanz as a strategy to treat recalcitrant sarcoidosis. In addition, Friedman et al in an open-label, proof-of-concept study, demonstrated the addition of Xeljanz allowed 60 percent (%) of patients with pulmonary sarcoidosis to successfully taper corticosteroids and act as a steroid-sparing agent. In another retrospective study by Moursi et al, showed that Xeljanz is a promising strategy for cutaneous and pulmonary sarcoidosis treatment. Therefore, based on the available peer reviewed literature for this orphan disease (refractory to treatment sarcoidosis), the proposed health service or treatment Xeljanz 11 mg is likely to be more beneficial than any standard treatment or treatments for the insured's life threatening or disabling condition or disease.