First-line azathioprine plus prednisone treatment results in better hematological response and relapse-free duration than only prednisone in patients with immune thrombocytopenia (ITP) who are positive for antinuclear antibody (ANA). The combination treatment also leads to acceptable adverse events.
This is according to the results of a retrospective cohort study published in the journal Hematology.
Read more about the prognosis of ITP
Patients with ANA-positive ITP respond poorly to first-line treatment with corticosteroids and have a worse prognosis.
In order to compare the efficacy and safety of azathioprine plus prednisone to that of prednisone alone in these patients, a team of researchers from China retrospectively analyzed 33 patients. Of these 15 received azathioprine plus prednisone while 28 received prednisone only.
The results showed that the complete response rate of patients who received azathioprine plus prednisone was 60% while that of patients who received prednisone alone was only 22.2%.
Similarly, the overall response rate was higher in patients treated with azathioprine plus prednisone compared to those treated with prednisone alone (86.7% versus 55.6%). However, the difference was not statistically significant.
The researchers calculated that azathioprine plus prednisone treatment was independently associated with a higher chance of achieving a complete response.
Moreover, accumulating relapse-free duration was longer in patients treated with azathioprine plus prednisone at 7.8 months compared to those treated with prednisone alone for whom the accumulating relapse-free duration was only 3.4 months.
Similarly, azathioprine plus prednisone treatment was independently correlated with longer accumulating relapse-free duration.
There was no difference between the 2 groups of patients in terms of the incidence of adverse events. The most common adverse events in patients treated with the combination treatment were pneumonia, anemia, cough, nausea, and granulocytopenia. These were all tolerable and manageable.
These results may support the use of the combination therapy for the treatment of patients with ANA-positive ITP, however, the authors note that “further large-scale studies are required to validate the findings of this study”.
Su J, Xu M, Dong Z, et al. Efficacy and safety of azathioprine plus prednisone versus prednisone alone as first-line treatment for antinuclear antibody-positive immune thrombocytopenia: a retrospective cohort study. Hematology. Published online April 4, 2023. doi:10.1080/16078454.2023.2196864