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Background: Immune thrombotic thrombocytopenic purpura (iTTP) is an acute and life-threatening rare disease. While plasma exchange (PEX) and steroids have improved patient survival, mortality and relapse rates remain high.
Methods: We performed a retrospective analysis of newly diagnosed iTTP patients admitted to the Second Affiliated Hospital of Anhui Medical University from May 2009 to May 2025. The treatment group received first-line rituximab combined with PEX and steroids, while the control group received PEX and steroids alone. Primary efficacy endpoints included clinical remission rate and 3-month mortality. Secondary efficacy endpoints comprised relapse rate, number of PEX sessions, ADAMTS13 activity recovery rate, cellular immune function, and overall survival (OS). The primary safety outcomes were infusion-related reactions, allergic reactions, and infection rates.
Results: The clinical remission rates were 91.7% vs. 40% (p = 0.037) in the treatment and control groups, respectively. The 3-month mortality rates were 8.3% vs. 46.7% (p = 0.043). Relapse rates were 9.1% vs. 62.5% (p = 0.041). The median number of PEX sessions was 10 vs. 18 (p = 0.049). ADAMTS13 activity recovery rates were 75% vs. 26.7% (p = 0.021). During rituximab treatment, B cells showed a progressive decline, reaching 0.8% (0% - 3.12%) after the fourth infusion. By the follow-up cutoff in August 2025, 75% of patients had normalized B-cell counts. No effects were observed on CD4+ T cells, CD8+ T cells, or immunoglobulin (IgG, IgA, IgM) levels. The 5-year OS rates were 70.7% vs. 26.7%. Median survival times were 54.1 months (95% CI: 36.791 - 71.369) vs. 39.9 months (95% CI: 8.11 - 71.69) (p = 0.007). No increased infection rates were recorded in the treatment group during hospitalization or follow-up.
Conclusions: First-line rituximab treatment can reduce mortality and relapse rates in iTTP patients, improve sur-vival outcomes, and demonstrates good safety.
DOI: 10.7754/Clin.Lab.2025.250905
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