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2016 ; 7
(51
): 85306-85317
Nephropedia Template TP
gab.com Text
Twit Text FOAVip
Twit Text #
English Wikipedia
Sunitinib for the treatment of benign and malignant neoplasms from von
Hippel-Lindau disease: A single-arm, prospective phase II clinical study from the
PREDIR group
#MMPMID27852035
Oudard S
; Elaidi R
; Brizard M
; Le Rest C
; Caillet V
; Deveaux S
; Benoit G
; Corréas JM
; Benoudiba F
; David P
; Gaudric A
; Hammel P
; Joly D
; Timsit MO
; Méjean A
; Richard S
Oncotarget
2016[Dec]; 7
(51
): 85306-85317
PMID27852035
show ga
Von Hippel-Lindau (VHL) disease is an autosomal dominant hereditary cancer
syndrome that predisposes affected individuals to the development of multiple
benign and malignant tumors. One of the main manifestations of VHL is renal cell
carcinoma (RCC). RCC is increasingly being treated with targeted therapies, which
offer an alternative treatment option for patients with VHL disease. This study
investigated the effectiveness of sunitinib in VHL patients with advanced tumors
or tumors unsuitable for surgery.This multicenter, phase II, open-label study
from the PREDIR VHL network, treated patients with genetically-confirmed advanced
VHL disease with oral sunitinib (50 mg/day for 28 days then a 2-week rest period)
until progression. Lesions were performed using magnetic resonance imaging (MRI)
and computed tomographic (CT) scan. The primary endpoint was objective response
rate; secondary endpoints included tolerability and overall survival.All five
patients showed stable disease as best response at 6 months. Two patients showed
impressive transitory clinical improvement during early cycles. No patient died
during sunitinib treatment. Reasons for discontinuing sunitinib therapy were
disease progression (n=1), unacceptable toxicity (n=3) and lack of clinical
improvement after 7 cycles (10.5 months) with unacceptable toxicity (n=1).In
conclusion, sunitinib was of limited benefit in patients with advanced VHL
disease, but had better efficacy against metastatic RCC than other VHL-related
lesions. Treatment-related toxicity is an important limiting factor in this frail
patient population. New agents with different mechanisms of action are required
to treat this disease.