This study focused on patients with type I neurofibromatosis, who currently lack effective drug therapy and have a high recurrence rate after surgical resection. As a MEK inhibitor, Smetinib bisulfate capsule can induce tumor shrinkage by selectively binding mitogen-activated protein kinase (MEK) 1/2 protein, blocking the mitogen-activated protein kinase/extracellular signal regulatory kinase signaling pathway that regulates key cell responses. To create conditions for disease control, radical surgical resection, reducing postoperative recurrence and reducing complications. The purpose of this study was to provide treatment with Smetinib bisulfate for patients with type I neurofibromatosis, observe the therapeutic effect in stages, convert patients without surgical indications into patients with surgical indications, increase the proportion of surgical resection and reduce the recurrence rate. Objective tumor response rate (ORR) after drug treatment was used as the main outcome index in this study. The resectable scope, duration of remission (DOR), progression-free survival (PFS) were used as secondary outcome indicators to investigate the improvement of resectable rate, reduction of resectable scope and postoperative complications, tumor shrinkage effect, and the stability of curative effect of the use of smetinib bisulfate capsule on type I neurofibromatosis.
Age ≥18 years old
According to the National Institutes of Health (NIH) updated diagnostic criteria for NF1 in 2021, 6 or more CALMs: d>5 mm before puberty or d>15 mm after puberty; 2 or more neurofibromas of any type or 1 plexiform neurofibroma;
Before admission, the head and neck surgeon conducted pathological biopsy of solid tumors, confirmed pathological diagnosis and eliminated malignant peripheral schwannoma (MPNST).
There was at least one measurable tumor lesion according to the solid tumor efficacy evaluation criteria RECIST 1.1
The patient had abnormal blood indexes and abnormal liver, kidney and heart function, and could not tolerate the clinical study process after multidisciplinary consultation and evaluation
Patients have malignant peripheral schwannoma (MPNST) or other malignant tumors, heart disease and other serious complications, or have previously undergone anti-tumor therapy such as surgery, chemotherapy, radiotherapy
Unable to complete the entire clinical study due to personal, social and economic reasons
there is a serious systemic disease in the past and the disease cannot be cured or controlled by medicine at present
Single group
Selumetinib
DrugPatients without indications for surgical excision were evaluated with 6 cycles of daily oral smetinib capsules (20-50mg bid) for 30 days, individually calculated based on patient body surface area (BSA)
Objective response rate (ORR)
Percentage of patients with a partial response confirmed by analysis after 6 cycles of drug therapy. A partial response is defined as a reduction in the target neurofibroma volume of ≥ 20% from baseline. A confirmed partial response was defined as a partial response assessed by successive re-staging examinations at intervals of ≥ 3 months.
The scope of the operation can be resected
On the premise of not damaging the important organs and nerve functions that the tumor has invaded, according to the proportion of tumor resection, it can be divided into total resection/near-total resection (resection range ≥ 90%), subtotal resection (90% \> resection range ≥ 50%), and partial resection (resection range \< 50%).
Duration of response (DOR)
Refers to the time from the first evaluation of a tumor as CR or PR to the first evaluation as PD or death from any cause.
Progression-free survival (PFS)
Refers to the time from the start of treatment in the trial until tumor progression or death from any cause, whichever occurs first.
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