Objective To conduct a meta-analysis assessing the efficacy and safety of cyclosporine-based combinations for primary immune thrombocytopenia (ITP). Methods Randomized controlled clinical trials were collected by systematically searching databases (PubMed®, MEDLINE®, EMBASE, The Cochrane Library, China National Knowledge Infrastructure) from inception to June 2022. All studies included patients with ITP who received cyclosporine-based regimens. We performed comprehensive analyses of the overall response rate (ORR), complete response (CR) rate, partial response (PR) rate, relapse rate, platelet count, and adverse drug reaction (ADR) rate. Results Seven studies (n = 418) were ultimately included. According to a fixed-effects model, cyclosporine-based combinations improved the ORR and CR rate and reduced the relapse rate. The ADR rate was not increased in the cyclosporine-based combination group. Cyclosporine-based regimens effectively increased the platelet count. Subgroup analysis illustrated that cyclosporine-based combinations were linked to higher ORRs in both children (odds ratio [OR] = 5.74, 95% confidence interval [CI] = 1.79–18.41) and adults (OR = 5.46, 95% CI = 2.48–12.02) and a higher CR rate in adults (OR = 2.97, 95% CI = 1.56–5.63). Conclusion Cyclosporine exhibited efficacy in the treatment of ITP without increasing the risk of ADRs.

Background: Lower-risk myelodysplastic syndrome (MDS) is characterized by the presence of dysplasia, low bone marrow blast percentage, low number and depth of cytopenia(s), and relatively good-risk karyotpic and molecular abnormalities. Lower-risk MDS patients have primarily received supportive care, immunomodulators, and immunosuppressants. New effective regimens or drugs are urgently needed for treatment of lower-risk MDS due to the unsatisfactory clinical efficacy. Sanzi-huangshi pill is an arsenic-containing Chinese medicine as a promising drug used for MDS for decades whose efficacy is not yet proven by high-quality clinical trial. Methods: SPIRIT guidelines were followed in drafting this protocol for a randomized controlled multicenter trial. Ninety-two adults with lower-risk MDS are randomly assigned in a 1:1 ratio to Sanzi-huangshi pill group or control group, and all participants are required to receive 6 months of intervention and 18 months of observation. The primary outcome is Overall Response Rate defined as the sum of complete remission, partial remission, cytogenetic complete remission and hematologic improvement accounting for the proportion of total evaluable cases. Discussion: This is the first randomized controlled trial to evaluate the efficacy and safety of arsenic-containing Chinese medicine as an adjuvant therapy in lower-risk MDS with serum erythropoietin (sEPO) >500 IU/ml. The purpose of this study is to correctly evaluate the position of traditional Chinese medicine in the treatment plan of MDS and to formulate an effective regimen of integrated Traditional Chinese and Western Medicine to provide preliminary clinical evidence. Clinical Trial Registration: http://www.chictr.org.cn, identifier: ChiCTR2200064230, Registered September 30, 2022.

Background: Lower-risk myelodysplastic syndrome (MDS) is characterized by the presence of dysplasia, low bone marrow blast percentage, low number and depth of cytopenia(s), and relatively good-risk karyotpic and molecular abnormalities. Lower-risk MDS patients have primarily received supportive care, immunomodulators, and immunosuppressants. New effective regimens or drugs are urgently needed for the treatment of lower-risk MDS owing to the unsatisfactory clinical efficacy. Sanzi-huangshi pill is an arsenic-containing Chinese medicine that has been used as a promising drug for MDS for decades, but its efficacy has not yet been proven by high-quality clinical trial. Methods: SPIRIT guidelines were followed in drafting this protocol for a randomized controlled multicenter trial. Ninety-two adults with lower-risk MDS are randomly assigned in a 1:1 ratio to Sanzi-huangshi pill group or control group, and all participants are required to receive 6 months of intervention and 18 months of observation. The primary outcome is Overall Response Rate defined as the sum of complete remission, partial remission, cytogenetic complete remission and hematologic improvement accounting for the proportion of total evaluable cases. Discussion: This is the first randomized controlled trial to evaluate the efficacy and safety of arsenic-containing Chinese medicine as an adjuvant therapy in lower-risk MDS with serum erythropoietin (sEPO) >500 IU/ml. The purpose of this study is to correctly evaluate the position of traditional Chinese medicine in the treatment plan of MDS and to formulate an effective regimen of integrated Traditional Chinese and Western Medicine to provide preliminary clinical evidence. Clinical Trial Registration: http://www.chictr.org.cn, identifier: ChiCTR2200064230, Registered September 30, 2022.