This study focused on patients with type I neurofibromatosis undergoing surgical treatment, who currently lack effective drug therapy and have a high recurrence rate after surgical resection. For patients with small solid tumors, limited space, and no invasion of the brain, spine and other important organs, surgical treatment is the main treatment. As a MEK inhibitor, Smetinib bisulfate capsule can induce tumor shrinkage and reduce postoperative recurrence by selectively binding mitogen-activated protein kinase (MEK) 1/2 protein to block the mitogen-activated protein kinase/extracellular signal regulatory kinase signaling pathway that regulates key cell responses. The purpose of this study was to treat patients with type I neurofibromatosis with indications of surgery with the drug smetinib bisulfate after surgical treatment, observe the therapeutic effect of the drug in stages, consolidate the postoperative effect and reduce the recurrence rate. In this study, progression-free survival (PFS) after postoperative drug treatment was used as the main outcome index, and duration of remission (DOR) and objective response rate (ORR) were used as secondary outcome indicators to investigate the efficacy of the use of Smetinib hydrosulfate capsule on tumor control, reduction of recurrence rate and stability of efficacy in patients with type I neurofibromatosis after surgery.
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
The patient has become malignant peripheral schwannoma (MPNST) or is accompanied by other malignant tumors, heart disease and other serious complications, or has previously received chemotherapy, radiotherapy and other anti-tumor therapy
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
DrugAfter surgical resection，based on the patient's body surface area (BSA), the patient was given an oral dose of Simetinib bisulfate capsule (20-50mg bid) daily for 30 days for 6 cycles
2-year progression-free survival (2-year PFS rate)
Refers to the rate of postoperative patients who are free of tumor progression or death from any cause (whichever occurs first) within 2 years from the start of treatment in the trial.
Duration of remission
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
Objective response rate
The proportion of patients whose tumors shrink by ≥ 20% for a certain period of time, including complete response (CR) and partial response (PR) cases. ORR was defined as the proportion of patients whose tumor size decreased by ≥ 20% in the shortest time.
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