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Purpose <p>Selumetinib is a MEK inhibitor indicated for pediatric patients with neurofibromatosis type 1 (NF1) and symptomatic inoperable plexiform neurofibromas (PNs).</p> Methods <p>This retrospective study collected data from 70 patients (aged 3 − 18 years) with NF1 and symptomatic inoperable PNs treated with selumetinib as part of compassionate use at 11 Italian centers between October 2018 and October 2024. Assessments included the clinical benefit rate (CBR) after 24 months and at the last observation. Major response (MR) was defined as a ≥ 50% reduction from baseline in tumor volume. Adverse events (AEs), patient-reported pain and quality of life (QoL), and Eastern Cooperative Oncology Group performance status (ECOG PS), were also evaluated.</p> Results <p>Of 45/70 patients with available natural history data at C0, 33/45 (73.3%) had progressive disease (PD). Radiological evaluation at C6 in 17/33 patients showed 16 (94.1%) had stable disease (SD) or partial response (PR). 52/58 patients (91.5%) had SD or PR/MR at C12; final response at last radiological follow-up was PD (7.7%), SD (42.3%), PR (30.8%) and MR (19.2%). CBR was 83.3% (24/70) at C24 and 91.5% (43/47) at last radiological follow-up. Selumetinib significantly reduced pain perception and improved QoL and ECOG PS. The type of response at C6 seems to predict response at C12 and at last observation. Adverse events were generally mild (78% grade ≤ 2).</p> Conclusion <p>Our findings suggest that the response after 6 and 12 selumetinib cycles may predict long-term outcomes, providing clinicians with an early indicator for therapeutic decision-making.</p> Trial registration number <p>Not applicable.</p>

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Real-world experience with selumetinib in children with neurofibromatosis type 1: a multicentric retrospective study

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年8月13日

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  • Claudia Santoro,
  • Mariachiara Servedio,
  • Maria Cristina Diana,
  • Irene Russo,
  • Elena Arkhangelskaya,
  • Gianluca Piccolo,
  • Andrea Santangelo,
  • Angela Mastronuzzi,
  • Antonella Cacchione,
  • May El Hachem,
  • Carmela Russo,
  • Mario Cirillo,
  • Ilaria Cecconi,
  • Antonio Grasso,
  • Mariateresa Loiotine,
  • Nicola Santoro,
  • Mariachiara Resta,
  • Carmela De Meco,
  • Consolata Soddu,
  • Eugenia Spreafico,
  • Bartolomeo Rossi,
  • Chiara Fossati,
  • Chiara Leoni,
  • Silverio Perrotta,
  • Teresa Perillo

摘要

Purpose

Selumetinib is a MEK inhibitor indicated for pediatric patients with neurofibromatosis type 1 (NF1) and symptomatic inoperable plexiform neurofibromas (PNs).

Methods

This retrospective study collected data from 70 patients (aged 3 − 18 years) with NF1 and symptomatic inoperable PNs treated with selumetinib as part of compassionate use at 11 Italian centers between October 2018 and October 2024. Assessments included the clinical benefit rate (CBR) after 24 months and at the last observation. Major response (MR) was defined as a ≥ 50% reduction from baseline in tumor volume. Adverse events (AEs), patient-reported pain and quality of life (QoL), and Eastern Cooperative Oncology Group performance status (ECOG PS), were also evaluated.

Results

Of 45/70 patients with available natural history data at C0, 33/45 (73.3%) had progressive disease (PD). Radiological evaluation at C6 in 17/33 patients showed 16 (94.1%) had stable disease (SD) or partial response (PR). 52/58 patients (91.5%) had SD or PR/MR at C12; final response at last radiological follow-up was PD (7.7%), SD (42.3%), PR (30.8%) and MR (19.2%). CBR was 83.3% (24/70) at C24 and 91.5% (43/47) at last radiological follow-up. Selumetinib significantly reduced pain perception and improved QoL and ECOG PS. The type of response at C6 seems to predict response at C12 and at last observation. Adverse events were generally mild (78% grade ≤ 2).

Conclusion

Our findings suggest that the response after 6 and 12 selumetinib cycles may predict long-term outcomes, providing clinicians with an early indicator for therapeutic decision-making.

Trial registration number

Not applicable.