Clinical Efficacy of Selumetinib in Alleviating Neuropathic Pain Associated with Plexiform Neurofibroma: A Case Series

<b>Background</b>: Selumetinib, an MEK inhibitor, was approved by the U.S. Food and Drug Administration in April 2020 and became reimbursable in Italy in January 2020, for treating patients aged ≥3 years with neurofibromatosis type 1 (NF1) complicated by symptomatic, inoperable plexiform neurofibromas (PNs). Selumetinib has been shown to effectively reduce the volume of target PNs and alleviate neuropathic pain even in long-term-treated patients. We report the impact of Selumetinib on pain in three NF1 cases with inoperable symptomatic plexiform neurofibromas. <b>Case Series</b>: Three patients with NF1 (aged 13-27 years) presented with symptomatic, inoperable plexiform neurofibromas (PNs) associated with severe neuropathic pain. Following selumetinib administration, a marked reduction in or disappearance of neuropathic pain was observed within a few weeks, allowing the complete discontinuation of pain therapy. Interestingly, pain recurred whenever selumetinib was temporarily suspended, requiring patients to resume analgesic therapy. <b>Conclusions</b>: Selumetinib treatment has been shown to effectively reduce neuropathic pain in patients with NF1. These findings represent a significant advancement in managing pain related to PNs and support its potential application in treating other forms of neuropathic pain.