Scholarlink

Purpose <p>To evaluate the hypothesis that breakthrough acromegaly symptom exacerbation frequency would be reduced after switching biochemically controlled patients from depot somatostatin receptor ligand (SRL) injections to once-daily oral paltusotine.</p> Methods <p>Biochemical disease control (insulin-like growth factor 1 [IGF-I]) and Acromegaly Symptom Diary (ASD) data were analyzed from PATHFNDR-1, a 36-week, randomized, placebo-controlled, double-blind, phase 3 trial of paltusotine in patients with acromegaly biochemically controlled (IGF-I ≤ 1.0× ULN) with SRLs. The ASD measured the daily severity of 7 core and 2 exploratory acromegaly symptoms, each rated on a scale from 0 to 10 based on 24-hour recall. Breakthrough acromegaly symptom exacerbations were defined as ≥ 2-point increases for any individual symptom score, comparing a 2-day average with the prior 2-day average.</p> Results <p>Despite no significant changes in IGF-I levels or overall core symptom severity scores, mean (SE) symptom exacerbation frequencies declined progressively after switching to paltusotine, from 30.2% (5.6%) of days during screening (SRL treatment) to 6.2% (1.6%) of days (<i>n</i> = 22, <i>p</i> &lt; 0.0001). The reduction in individual symptom exacerbation frequencies with paltusotine treatment was consistent across all acromegaly symptoms assessed. IGF-I levels did not correlate significantly with symptom severity scores or symptom variability.</p> Conclusion <p>In patients with acromegaly biochemically controlled with injected SRLs, switching to once-daily oral paltusotine was associated with stable biochemical control, stable symptom severity, and a significantly reduced frequency of symptom exacerbations. A simple daily symptom assessment tool provided important information pertaining to acromegaly disease control that was not apparent from IGF-I measurements.</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

Reduced breakthrough symptom exacerbations in patients with biochemically controlled acromegaly switched from injected depot somatostatin receptor ligands to once-daily oral paltusotine in the PATHFNDR-1 clinical trial

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2026年7月23日

查看全文
  • David R. Clemmons,
  • Tiffany P. Quock,
  • Alessandra Casagrande,
  • Yang Wang,
  • Alan Krasner

摘要

Purpose

To evaluate the hypothesis that breakthrough acromegaly symptom exacerbation frequency would be reduced after switching biochemically controlled patients from depot somatostatin receptor ligand (SRL) injections to once-daily oral paltusotine.

Methods

Biochemical disease control (insulin-like growth factor 1 [IGF-I]) and Acromegaly Symptom Diary (ASD) data were analyzed from PATHFNDR-1, a 36-week, randomized, placebo-controlled, double-blind, phase 3 trial of paltusotine in patients with acromegaly biochemically controlled (IGF-I ≤ 1.0× ULN) with SRLs. The ASD measured the daily severity of 7 core and 2 exploratory acromegaly symptoms, each rated on a scale from 0 to 10 based on 24-hour recall. Breakthrough acromegaly symptom exacerbations were defined as ≥ 2-point increases for any individual symptom score, comparing a 2-day average with the prior 2-day average.

Results

Despite no significant changes in IGF-I levels or overall core symptom severity scores, mean (SE) symptom exacerbation frequencies declined progressively after switching to paltusotine, from 30.2% (5.6%) of days during screening (SRL treatment) to 6.2% (1.6%) of days (n = 22, p < 0.0001). The reduction in individual symptom exacerbation frequencies with paltusotine treatment was consistent across all acromegaly symptoms assessed. IGF-I levels did not correlate significantly with symptom severity scores or symptom variability.

Conclusion

In patients with acromegaly biochemically controlled with injected SRLs, switching to once-daily oral paltusotine was associated with stable biochemical control, stable symptom severity, and a significantly reduced frequency of symptom exacerbations. A simple daily symptom assessment tool provided important information pertaining to acromegaly disease control that was not apparent from IGF-I measurements.