Phase II clinical trial of pazopanib for patients with unresectable or metastatic malignant peripheral nerve sheath tumors.
Yoshihiro NishidaHiroshi UrakawaRobert NakayamaEisuke KobayashiToshifumi OzakiKeisuke AeYoshihiro MatsumotoHiroyuki TsuchiyaTakahiro GotoHiroaki HiragaNorifumi NakaShunji TakahashiYuichi AndoMasahiko AndoYachiyo KuwatsukaShunsuke HamadaTakafumi UedaAkira KawaiPublished in: International journal of cancer (2020)
Malignant peripheral nerve sheath tumor (MPNST) often does not respond well to chemotherapy and develops against a background of NF1. The purpose of our study was to examine the efficacy of pazopanib against MPNST. Our study was designed as a physician-initiated phase II clinical trial in patients with advanced MPNST. Patients were registered from 11 large hospitals. The primary endpoint was set to clarify the clinical benefit rate (CBR) at 12 weeks according to response evaluation criteria in solid tumors (RECIST). Progression-free survival (PFS), overall survival (OS) and the CBR based on modified Choi evaluation at week 12 were set as secondary endpoints along with treatment-related safety. The study enrolled 12 patients. Median age was 49 years. Seven had Grade 2 and five Grade 3 according to the FNCLCC evaluation. Median follow-up period was 10.6 months. CBR at 12 weeks was both 50.0% (RECIST and Choi). The median PFS was 5.4 months for both RECIST and Choi, and the median OS was 10.6 months. Of special interest, the median PFS was 2.9 months for patients with FNCLCC Grade 2 and 10.2 months for Grade 3 (both RECIST and Choi). Grade 4 adverse events of neutropenia and lipase elevation were noted in one patient each. The results of this pazopanib therapy were generally better than those of any of the other single molecular targeted therapies reported previously. Although accumulation of more cases remains necessary, we conclude pazopanib treatment for MPNST to be a safe and promising treatment after doxorubicin-based chemotherapy.
Keyphrases
- phase ii
- clinical trial
- peripheral nerve
- end stage renal disease
- open label
- free survival
- ejection fraction
- chronic kidney disease
- newly diagnosed
- locally advanced
- emergency department
- small cell lung cancer
- phase iii
- squamous cell carcinoma
- peritoneal dialysis
- healthcare
- randomized controlled trial
- double blind
- case report
- cell proliferation
- radiation therapy
- gestational age
- replacement therapy
- patient reported outcomes