Primary immune thrombocytopenia (ITP) is an acquired autoimmune disorder causing isolated thrombocytopenia. Driven by the rapid emergence of novel therapies and new clinical evidence, the Thrombosis and Hemostasis Group of the Chinese Society of Hematology has released the 2025 edition of its guideline for adult ITP.1 This update incorporates major advances, including new roles for thrombopoietin receptor agonists (TPO-RAs) and the introduction of spleen tyrosine kinase (SYK) inhibitors, Bruton’s tyrosine kinase (BTK) inhibitors, and neonatal Fc receptor (FcRn) antagonists. The key recommendations emphasize a shift toward individualized, goal-directed therapy aimed at achieving sustained, treatment-free remission. Diagnostic Evaluation The diagnosis of ITP remains one of exclusion. Key diagnostic criteria include at least two consecutive blood tests showing a platelet count 60%; evidence level Ib, Grade A recommendation). The choice of agent depends on administration preference (oral vs. subcutaneous) and dietary restrictions. Switching between different TPO-RAs is recommended if the first choice is ineffective. Rituximab B-cell depletion therapy with rituximab is an established second-line option. Both the standard dose (375 mg/m2 × 4 weeks) and low dose (100 mg/week × 4 weeks or 375 mg/m2 once) are effective (evidence level IIa/IIb, Grade B recommendation). It is particularly suitable for patients seeking long-term drug-free remission, though the onset of action is slower than TPO-RAs. SYK inhibitors The 2025 guideline now includes SYK inhibitors, which inhibit SYK‑mediated signaling downstream of Fcγ receptors and B‑cell receptors, thereby reducing antibody‑mediated platelet destruction and potentially limiting autoantibody production. Fostamatinib shows an ORR of 43–73% with a time to response (TTR) of approximately 2 weeks (evidence level Ib). In a phase III trial, sovleplenib, an agent developed in China, demonstrated an ORR of 80%, a median TTR of 8 days, and a 6-month sustained response rate (SRR) of 48% (evidence level Ib), providing a potent oral option for refractory patients. BTK inhibitors BTK inhibitors suppress key pathogenic pathways in ITP, including B-cell activation and macrophage phagocytosis. Rilzabrutinib demonstrated an ORR of 64% with a median TTR of 15 days and a 24-week SRR of 23% (evidence level Ib). A Chinese phase II trial of orelabrutinib showed an ORR of 40% and a median TTR of 9 days (evidence level III), with improved efficacy observed in patients who had previously responded to initial therapy. FcRn antagonists Efgartigimod is a FcRn antagonist that selectively targets and promotes the degradation of immunoglobulin G antibodies. This leads to a rapid reduction in the levels of pathogenic immunoglobulins G. Efgartigimod has demonstrated an ORR of 46% with a median TTR of 7 days (evidence level Ib). It is generally well tolerated, with common adverse events including headache and hematuria. Decitabine Low-dose decitabine is an option for refractory ITP (evidence level IIa, Grade B recommendation). Its mechanism is thought to involve both restoring normal megakaryocyte apoptosis and modulating immune responses. The therapy has shown efficacy and a favorable safety profile in patients unresponsive to other treatments. Subsequent combination therapies The guideline explicitly recommends combining agents with different mechanisms for refractory patients to achieve synergistic effects and improve sustained response. Key evidence-based combinations include rituximab plus rhTPO, rituximab plus all-trans retinoic acid (ATRA), eltrombopag plus diacerein, tacrolimus plus danazol, and ATRA plus danazol (evidence level Ib). Clinical trials Enrollment in phase II/III clinical trials investigating novel agents, repurposed drugs, and combination therapies is recommended for eligible patients. This provides access to novel investigational agents and contributes to advancing the treatment of ITP. Splenectomy Splenectomy remains a curative option but is generally deferred until medical therapies have failed and the disease duration exceeds 12 months (Grade C recommendation). It is less commonly performed due to the availability of effective pharmacological alternatives but remains valuable for patients desiring a permanent drug-free solution. Other agents For patients failing the above treatments, other immunosuppressive or immunomodulatory agents such as cyclosporine A, danazol, azathioprine, and vincristine are also options, but are typically reserved for refractory cases due to limited evidence and side effect profiles. In conclusion, the 2025 Chinese guideline marks a paradigm shift in ITP management, moving beyond platelet count normalization to focus on preventing bleeding and improving quality of life. The introduction of novel agents—including SYK, BTK, and FcRn inhibitors—alongside refined combination strategies has significantly expanded the therapeutic toolkit. By standardizing diagnosis and stratifying treatment, this guideline aims to optimize outcomes for adults with ITP in China. Future research should prioritize identifying biomarkers to further personalized therapy. This should be coupled with a greater emphasis on patient-reported outcomes, ensuring that improvements in HRQoL are a fundamental goal of treatment. Conflicts of interest None.
Liu et al. (Fri,) studied this question.