Appeal type: Formulary Exception
Decision: Overturned

Diagnosis:  Immune Thrombocytopenia (ITP)

Treatment:  Promacta

The insurer denied Promacta.  The denial was reversed.   

This is a female patient with a history of anemia and immune thrombocytopenia (ITP).  Record reports that the patient's platelet have been dropping despite medical intervention; prednisone and dexamethasone therapy.  A bone marrow biopsy was performed that revealed normal a platelet count.  It was reported that the patient was experiencing bruising.  A platelet count was obtained revealing results of 23K.  The treating provider has recommended Intravenous immunoglobulin (IVIG) and Promacta.
Promacta is unlikely to cause the patient to have an adverse reaction.  There is no information listed in the file to show that the patient is at an increased risk of having a reaction to the requested medication.  Additionally, the documentation reviewed has shown that the patient has failed both prednisone and dexamethasone treatment.  The patient continues to have a low platelet count, and she is now having increased bruising.  Current treatment with prednisone and dexamethasone has not been effective for this patient.   
  
Promacta is likely to be more effective than the formulary medication.  The patient had already failed prednisone and dexamethasone, both yielding very little positive response.  The patient's platelet count remained at 23K, and she is now having busing.  In addition, the patient has an ITP diagnosis.  Promacta is medically necessary for this patient in this case.

According to Wong, R. S. M., et al. (2017), "In phase 2/3 trials, eltrombopag treatment of 6 months or less in patients with chronic/persistent immune thrombocytopenia (ITP) increased platelet counts and reduced bleeding.  The open-label EXTEND study evaluated long-term safety and efficacy of eltrombopag in adults with ITP who had completed a previous eltrombopag study.  For the 302 patients enrolled, median duration of eltrombopag treatment was 2.37 years (2 days-8.76 years).  Median platelet counts increased to 50  109/L or more by week 2 and were sustained throughout the treatment period.  Overall, 259 patients (85.8%) achieved a response (platelet count 50  109/L at least once in the absence of rescue), and 133 (52%) of 257 patients achieved a continuous response of 25 weeks or longer.  Responses in patients with platelet counts lower than 15  109/L, more previous therapies, and/or splenectomy were somewhat lower.  Thirty-four (34%) of 101 patients receiving concomitant ITP medication discontinued 1 or more medication. In patients with assessments, bleeding symptoms (World Health Organization grades 1-4) decreased from 57% at baseline to 16% at 1 year.  Forty-one patients (14%) withdrew because of adverse events.  Hepatobiliary adverse events (n = 7), cataracts (n = 4), deep vein thrombosis (n = 3), cerebral infarction (n = 2), headache (n = 2), and myelofibrosis (n = 2) occurred in more than 1 patient; the remaining adverse events occurred only once.  Rates of thromboembolic events (6%) and hepatobiliary adverse events (15%) did not increase with treatment duration past 1 year.  EXTEND demonstrated that long-term use of eltrombopag was effective in maintaining platelet counts of 50  109/L or more and reducing bleeding in most patients with ITP of more than 6 months' duration.  Important adverse events (e.g., thrombosis, hepatobiliary, and bone marrow fibrosis) were infrequent. (ClinicalTrials.gov:NCT00351468)."

Lastly, according to Cooper, N., & Ghanima, W. (2019), "Immune thrombocytopenia in patients who present with serious bleeding is treated with platelet transfusions, glucocorticoids, and intravenous immune globulin.  In patients without serious bleeding, treatment decisions are guided by the platelet count and other factors.  Commonly used agents include glucocorticoids, thrombopoietin-receptor agonists, and rituximab."                                                       

Based on the above, the denial for a request for a Formulary Exception for Promacta is reversed.