海曲泊帕治疗特发性多中心型Castleman病-TAFRO亚型患者血小板减少的疗效

Efficacy of Hetrombopag for Thrombocytopenia in Patients with Idiopathic Multicentric Castleman Disease-TAFRO Subtype

    摘要
  • 摘要:
    目的 研究海曲泊帕对特发性多中心型Castleman病(idiopathic multicentric Castleman disease, iMCD)-TAFRO血小板减少(thrombocytopenia)、全身水肿(anasarca)、发热(fever)、网状纤维化(reticulin fibrosis)、肾功能衰竭(renal failure)、器官肿大(organomegaly)亚型患者血小板减少的疗效。
    方法 回顾性纳入2022年10月至2025年12月于北京协和医院接受过海曲泊帕治疗的12例iMCD-TAFRO亚型患者。主要疗效指标为海曲泊帕治疗第4、8、16周时的血小板完全缓解(complete response，CR；血小板计数≥100×109/L且无出血)率和部分缓解(partial response，PR; 血小板计数≥50×109/L且较基线值升高，同时无出血)率。次要疗效指标包括起效时间、血小板计数达峰时间、血小板计数及Castleman病的生化缓解率。
    结果 患者中位年龄为51.5(38.0, 61.0)岁，重型占75%。12例iMCD-TAFRO亚型患者应用海曲泊帕治疗第4周和第8周时，血小板治疗总有效率均为75%(其中第4周、第8周时的CR率均为50%，PR率均为25%)；治疗第16周时，血小板治疗总有效率为100%(CR率75%，PR率25%)。使用海曲泊帕后中位起效时间为32.0(24.8, 72.3)d，血小板达峰时间为86.0(46.5, 126.8)d。海曲泊帕治疗第4周、第8周、第16周时血小板计数较治疗前基线水平显著升高，差异均有统计学意义P均＜0.017；第16周时：154.0 (97.5, 208.0)×109/L比28.5(19.5, 41.5)×109/L，Z=-3.059，P=0.002，血小板计数升至50×109/L和100×109/L时，Castleman病生化缓解率分别为50.0%、91.7%。海曲泊帕治疗期间未见严重不良事件。截至2026年3月31日，所有患者均已停用海曲泊帕，血小板计数均＞100×109/L。
    结论 海曲泊帕可有效提升iMCD-TAFRO亚型患者血小板计数，安全性较好，停药后血小板减少不易复发，可为原发病治疗提供支持。

     

    Abstract:
    Objective To investigate the efficacy of hetrombopag for thrombocytopenia in patients with idiopathic multicentric Castleman disease(iMCD)-TAFRO(thrombocytopenia, anasarca, fever, reticulin fibrosis, renal dysfunction, and organomegaly)subtype.
    Methods Twelve patients with iMCD-TAFRO subtype who received hetrombopag therapy at Peking Union Medical College Hospital from October 2022 to December 2025 were retrospectively included. The primary efficacy endpoints were the rates of complete response(CR; platelet count ≥100×109/L without bleeding)and partial response(PR; platelet count ≥50×109/L and at least twice the baseline value without bleeding)at week 4, 8 and 16 of hetrombopag treatment. Secondary efficacy endpoints included time to initial response, time to platelet peak, platelet count levels, and biochemical response rates of Castleman disease
    Results The median patient age was 51.5(38.0, 61.0)years, and 75% of patients had severe disease. Among the 12 patients with iMCD-TAFRO subtype, the overall platelet response rate was 75% at both week 4 and week 8(CR rate 50% and PR rate 25% at both time-points). At week 16, the overall platelet response rate reached 100%(CR rate 75%, PR rate 25%). The median time to initial response after hetrombopag initiation was 32.0(24.8, 72.3)d, and the median time to platelet peak was 86.0(46.5, 126.8)d. Platelet counts were significantly elevated at week 4, week 8 and week 16 compared with baselineall P < 0.017; at week 16: 154.0(97.5, 208.0)×109/L vs. 28.5(19.5, 41.5)×109/L, Z=-3.059, P=0.002.Biochemical response rates of Castleman disease were 50% and 91.7% when platelets rose to ≥50×109/L and ≥100×109/L, respectively. No serious adverse events were observed during hetrombopag treatment. As of March 31, 2026, all patients had discontinued hetrombopag, with platelet counts persistently > 100×109/L.
    Conclusions Hetrombopag effectively increases platelet counts in patients with iMCD-TAFRO subtype, with a favourable safety profile. Relapse of thrombocytopenia is uncommon after drug discontinuation, and hetrombopag can provide supportive conditions for the treatment of the primary disease.

     

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