PF692 COMBINATION OF MYCOPHENOLATE MOFETIL AND DEXAMETHASONE IN THE PATIENTS OF CHRONIC ITP
Authors: N. Wasekar, G. badarkhe, V. Patil, S. bamborde
Journal: HemaSphere (2019)

Background: MMF (Mycophenolate mofetil) is recommended as a second line therapy in immune thrombocytopenia (ITP). Dexamethasone is used as first‐line treatment for ITP; also it is used in combination with Rituximab for refractory cases of ITP. Retrospective analysis of 23 ITP patients treated with MMF (1000 mg/day) and Dexamethasone (40 mg/day for four days per month for 6 months) was done in order to assess its efficacy and safety. The overall response rate was 52% (n = 12, with a complete response in 30%) with a median time to response of 29 days (24–41 days). Aims: We aimed to assess the efficacy and safety of MMF along with Dexamethasone in a retrospective monocentric study in adult ITP. Methods: Cases of severe ITP who had received MMF and Dexamethasone were retrospectively identified from HCG Hospital. All of the included patients were requiring second‐line therapy beyond steroids or IVIg. Bleeding manifestations ranged from oral mucosal bleeding, petechiae, per rectal bleeding (n = 2) to intracerebral hemorrhage (n = 1). We were analyzing the response, duration of response and side effects. Response criteria/definitions Responses to therapy were categorized in keeping with published guidance [7]. In short, a response to therapy in ITP was the ability to maintain a platelet count sufficient to prevent clinically significant bleeding. Results: The total cohort of 23 patients included 14 males and 9 females. The median age at the time of initiating MMF therapy was 37 years (range 18–92 years). The median duration of follow‐up of all patients was 12 months (range 2–40 months), measured in months from the time of starting MMF and Dexamethasone to the last documented follow‐up at the time of data collection. The median time between diagnosis of ITP and initiation of MMF and Dexamethasone was 25 months (range 1–184 months). Fifteen had primary ITP and 8 had secondary ITP. Eight of 23 had an associated autoimmune disorder. The previous treatment used before MMF and Dexamethasone were documented and are summarized in Table [1]. The median number of previous treatments was 2 (n = 15), with a range of 1 (n = 7) to 7. The overall response rate was 52% (n = 12, with a complete response in 30%) with a median time to response of 29 days (24–41 days). Summary/Conclusion: In conclusion, from our cohort of 23 patients, we have demonstrated a response to MMF and Dexamethasone in more than 50% of cases, including patients refractory to multiple lines of therapy, and many years from diagnosis. Emphasis should be given to the limited side effects and the relatively low therapeutic dose needed to achieve and maintain the response. There is potential promise for those patients who have unfortunately failed to sustain remission with first‐line agents. Even if MMF and Dexamethasone do not induce a complete remission, it may at least help to reduce steroid burden, or indeed be used in combination with other drugs (e.g. rituximab). As an easily available and economical choice, MMF and Dexamethasone should be considered in the patient therapeutic pathway and formal trials documenting its response in a randomized prospective setting are called for. A formal prospective randomized trial is necessary to confirm these findings.