Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Chronic immune thrombocytopenic purpura.
Treatment: Promacta 25 mg (milligram) OR (oral) tabs
The insurer denied Promacta 25 mg OR tabs.
The determination is overturned.

The patient has a diagnosis of chronic immune thrombocytopenic purpura (ITP). She initially presented with petechiae and easy bruising and was found to be thrombocytopenic with a platelet count of 1000. She was initially treated with intravenous immunoglobulin (IVIG) at 1 gram per kilogram (g/kg) and had a minimal response with a platelet count of 14,000. Subsequently, she presented with wet purpura and the platelet count of 0 and was treated with steroids at 2 milligrams/kilograms (mg/kg) every 12 hours for 7 days.
She has intermittently been treated with both IVIG and/or steroids. Her activities of daily living and quality of life have been suboptimal. The provider is currently recommending therapy with Promacta. This is denied as the patient had not failed the preferred treatment with rituximab. The provider states that Promacta would be preferred over rituximab, as there is high-priority consideration to avoid immunosuppression in children in this situation. The use of Promacta is currently under review for medical necessity.

The requested health service/treatment of Promacta 25 milligrams (mg) tablets (tabs) is medically necessary for this patient.

This patient has chronic ITP, and requires second line therapy. The patient has failed both IVIG and steroids. The provider is currently recommending Promacta, stating that this is preferred over rituximab in this particular case. They have provided medical literature to support their stance. As per recent review from Grace et al, Promacta is the only product that resulted in a median improvement in treatment outcomes including platelet count, bleeding, and quality of life scoring using the kids ITP tool (KIT). Patients treated with Promacta had a median improvement at 1 month that met the minimal important difference for the review.

As per the American Society of Hematology, and the 2019 recommendations for the management of ITP, the American Society of Hematology (ASH) guideline panel suggested the use of product such as Promacta rather than rituximab, noting that the second line treatments should be individualized based on the duration of ITP, the frequency of bleeding episodes requiring hospitalization or rescue medication, comorbidities, age of the patient, medication adherence, medical and social support networks, patient values and preferences, cost, and availability. Patient education and shared decision making are encouraged. Specifically noted is that patients who placed a high value on achieving durable response may prefer medication such as Promacta over products such as rituximab.

As per a review in UpToDate, medications such as Promacta are appropriate for use for patients with ITP lasting over 3 months to 6 months when they require pharmacologic intervention, and while there are notes that rituximab is preferred for those patients with autoimmune disorders or autoantibodies, otherwise the thrombopoietin receptor products are preferred. Consideration should be given to the age of the patient, presence of autoimmune disorders, ease of administration, and expected adherence to treatment regimen, as well as possibility of long-term remission, when deciding which product to use a second line therapy. For this young patient, with no autoimmune component, the use of Promacta appears to be the most appropriate choice.

Therefore, based on the clinical information provided for review, to include this individual patient's clinical characteristics, the request for Promacta is clinically appropriate and medically necessary.

The rituximab products (to include Ruxience, Truxima, or Riabni) are not the preferred method of treatment for this patient's specific condition.