Calcitriol and sirolimus combo effective in treating chronic primary immune thrombocytopenia: study

A study was conducted to investigate the clinical efficacy of calcitriol combined with sirolimus in the treatment of chronic primary immune thrombocytopenia (cITP) patients.

A total of 146 adult cITP patients treated in the First Affiliated Hospital of Hebei North University from March 2017 to March 2020 were randomly divided into observation group (73 cases) and control group (73 cases) according to random number table. The control group was treated with oral sirolimus capsule, the observation group was treated with oral calcitriol capsule combined with sirolimus capsule, and the curative effect of the 2 groups was evaluated after continuous treatment for 6 weeks. The changes of World Health Organization (WHO) bleeding grade, laboratory-related index, including peripheral blood regulatory T cell (Treg), serum 1,25-dihydroxy-vitamin D3 [1,25(OH)2D3], and serum level of vitamin D receptor (VDR), in the 2 groups before and after treatment were observed, and the scores of functional assessment of chronic illness therapy-figure (FACIT-F), ITP quality of life scale (ITP-PAQ) in the 2 groups were recorded and analyzed. Finally, the occurrence of side effects between the 2 groups was compared.

The Results of the study are:

The total effective rate of the observation group was 79.5% (58/73), which was significantly higher than 64.4% (47/73) of the control group (P<0.05). The revised WHO bleeding grades after treatment were significantly better than those before treatment in the 2 groups (P<0.05), but the observation group was improved more significantly than the control group (P<0.05). After treatment, platelet count (PLT), peripheral blood Treg cell ratio, and serum 1,25(OH)2D3 concentration in both the 2 groups significantly increased (P<0.05), while peripheral blood lymphocyte VDR significantly decreased (P<0.05). In addition, the ratio of PLT and Treg cells in peripheral blood and the level of 1,25(OH)2D3 in serum in the observation group were significantly higher than those in the control group (P<0.05) at the same period, while the content of VDR in peripheral blood was significantly lower (P<0.05). The scores of FACIT-F and ITP-PAQ after treatment in the two groups were significantly higher than those before treatment (P<0.05), but the scores in the observation group were increased more significantly than the control group (P<0.05). The side reaction rate of the observation group [11.0% (8/73)] had no significant difference compared with the control group [13.7% (10/73)] (P>0.05).

Thus, the researchers concluded that the overall efficacy of calcitriol combined with sirolimus in the treatment of cITP in adults is satisfactory, which can effectively alleviate patient's condition, improve the quality of life, further increase the platelet level and decrease the expression of VDR in peripheral blood lymphocyte, the mechanism may be related to increasing the level of serum 1,25(OH)2D3 and up-regulating the expression of Treg cells in peripheral blood.

Reference:

Clinical Study on Calcitriol Combined with Sirolimus in the Treatment of Chronic Primary Immune Thrombocytopenia Patients by Qi Li et al. published in the Zhongguo Shi Yan Xue Ye Xue Za Zhi.

https://pubmed.ncbi.nlm.nih.gov/34893133/