Are there long-term effects of combination therapy for newly diagnosed adults with ITP?

In newly diagnosed (ND) adults with immune thrombocytopenia, standard first-line treatment (steroids ± IVIG) effectively raises platelet counts and mitigates bleeding or the risk thereof. In difficult-to-treat patients who fail first-line treatment, combination therapy may be appropriate; however, first- and second-line treatments could be combined at diagnosis to further improve initial platelet count and sustained off-treatment response in ND patients following the hypothesis that autoimmune responses are more malleable at diagnosis than later. Three randomised controlled trials (2 dexamethasone (dex) vs. dexamethasone + rituximab (ritux), 1 mycophenolate mofetil (MMF) + steroids vs. steroids alone) and six single-arm trials (dex + ritux ± ciclosporin and dex + TPO-RA ± ritux) were analysed to determine the effects of combining these therapies. Across the studies, initial and short-term responses were robust (50%-84%) with responses past 1 year ranging from 47% to 77%. While short-term responses were promising, many studies lacked a longer follow-up past 1-1.5 years. The amount of ND patients who would have entered sustained response off therapy long term with no treatment/steroids only, as well as with combination therapy, is thus unknown and remains to be investigated.

VIVO Weill Cornell Medical College