Scholarlink

<p>Large granular lymphocytic leukemia (LGLL) is characterized by the clonal proliferation of cytotoxic T lymphocytes or NK cells. Standard first-line immunosuppressive treatments have limitations, achieving complete remission (CR) rates of up to 50%. Immune system dysregulation is implicated in LGLL. Promising results for thalidomide, an immunomodulatory drug, combined with prednisone and methotrexate (TPM), were observed in our pilot study. This multicenter study evaluated the efficacy and safety of a thalidomide, prednisone, and methotrexate (TPM) regimen in 52 symptomatic, methotrexate- and thalidomide-naive LGLL patients from June 2020 to August 2022. Thalidomide (100 mg daily for up to 24 months), prednisone (0.5–1.0 mg/kg every other day, tapered after 3 months), and methotrexate (10 mg/m<sup>2</sup> weekly for up to 12 months) were administered. The primary objective was to determine the CR rate. The median follow-up duration was 29.0 months (range: 4.0–42.0). Forty-seven patients (90.4%) achieved hematological and symptomatic responses. Thirty-nine patients (75.0%) achieved CR. The median time to response was 3.0 months (range: 3.0–9.0). The median progression-free survival was 40.0 months (95% confidence interval (CI): 38.0–42.0), and the median duration of response was 39.0 months (95% CI: 36.1–41.9). The most common adverse event was peripheral neuropathy (24.1%), most of which (84.6%) were grades 1–2. Four patients experienced grade ≥3 adverse events. In conclusion, the TPM regimen was an effective and safe treatment for symptomatic LGLL patients, with a particularly high CR rate. This trial was registered at <a href="http://www.clinicaltrials.gov">www.clinicaltrials.gov</a> (#NCT04453345).</p>

错误：搜索内容不能为空，请输入英文关键词
错误：关键词超出字数限制，请精简
高级检索

Thalidomide-based regimen shows promising efficacy in large granular lymphocytic leukemia: a multicenter phase II study

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2025年3月12日

查看全文
  • Ying Yu,
  • Yuxi Li,
  • Rui Cui,
  • Yuting Yan,
  • Fei Li,
  • Yan Chen,
  • Tingyu Wang,
  • Xiaoli Hu,
  • Yaqing Feng,
  • Tengteng Yu,
  • Yanshan Huang,
  • Jingwen Sun,
  • Rui Lyu,
  • Wenjie Xiong,
  • Qi Wang,
  • Wei Liu,
  • Gang An,
  • Weiwei Sui,
  • Yan Xu,
  • Wenyang Huang,
  • Dehui Zou,
  • Huijun Wang,
  • Zhijian Xiao,
  • Jianxiang Wang,
  • Lugui Qiu,
  • Shuhua Yi

摘要

Large granular lymphocytic leukemia (LGLL) is characterized by the clonal proliferation of cytotoxic T lymphocytes or NK cells. Standard first-line immunosuppressive treatments have limitations, achieving complete remission (CR) rates of up to 50%. Immune system dysregulation is implicated in LGLL. Promising results for thalidomide, an immunomodulatory drug, combined with prednisone and methotrexate (TPM), were observed in our pilot study. This multicenter study evaluated the efficacy and safety of a thalidomide, prednisone, and methotrexate (TPM) regimen in 52 symptomatic, methotrexate- and thalidomide-naive LGLL patients from June 2020 to August 2022. Thalidomide (100 mg daily for up to 24 months), prednisone (0.5–1.0 mg/kg every other day, tapered after 3 months), and methotrexate (10 mg/m2 weekly for up to 12 months) were administered. The primary objective was to determine the CR rate. The median follow-up duration was 29.0 months (range: 4.0–42.0). Forty-seven patients (90.4%) achieved hematological and symptomatic responses. Thirty-nine patients (75.0%) achieved CR. The median time to response was 3.0 months (range: 3.0–9.0). The median progression-free survival was 40.0 months (95% confidence interval (CI): 38.0–42.0), and the median duration of response was 39.0 months (95% CI: 36.1–41.9). The most common adverse event was peripheral neuropathy (24.1%), most of which (84.6%) were grades 1–2. Four patients experienced grade ≥3 adverse events. In conclusion, the TPM regimen was an effective and safe treatment for symptomatic LGLL patients, with a particularly high CR rate. This trial was registered at www.clinicaltrials.gov (#NCT04453345).