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Table 1 Phase II trials of sorafenib in DTC

From: Rationale and design of DECISION: a double-blind, randomized, placebo-controlled phase III trial evaluating the efficacy and safety of sorafenib in patients with locally advanced or metastatic radioactive iodine (RAI)-refractory, differentiated thyroid cancer

Trial Efficacy Results Grade ≥ 3 AEs in ≥ 5%
of Patients
Other
Single-arm trial
(N = 55 total;
n = 47 DTC) [1012]
mPFS = 84 weeks*
SD = 53.3%
PR = 23.3%
DCR = 76.6%
OS = 140 weeks
Hypertension (13%), hand-foot syndrome (10%), rash (10%), weight loss (10%), diarrhea (7%), elevated LFTs (7%) • Dose reduction (due to AEs) was required in 47% of patients (initial analysis)
• Median duration of treatment at initial analysis (30 of 55 patients accrued) = 27 weeks
• BRAF genotyping (n = 16): mPFS = 84+ weeks in DTC patients with BRAFV600E, compared with 54 weeks in those with BRAFwt (P = 0.028)
Single-arm component (chemotherapy-naive patients with DTC) of two-arm trial (N = 56 total; n = 41 DTC) [13] mPFS = 65 weeks*
SD = 57%*
PR = 15%*
DCR = 72%*
Fatigue, HSFR (11% each); weight loss, skin rash, hypertension, diarrhea, stomatitis, tongue/tooth pain, abdominal/rectal pain, proximal myopathy, back pain, general pain, hand/foot pain, arthralgia, colon perforation (5% each) • Dose reduction (due to AEs) was required in 52% of patients
Single-arm trial
(N = 26 total;
n = 14 DTC) [14, 15]
mPFS not reported
SD = 82%
PR = 18%
DCR = 100%
Hand-foot syndrome (19%), other skin toxicity (6%),
hypertension (6%), infection (8%)
• Dose reduction (due to AEs) was required in 62% of patients
• Five drug-related serious AEs were reported:
2 hospitalizations for non-neutropenic fever/infection, 1 for hypocalcemia, and 2 for fever/rash
Single-arm trial
(N = 31; all DTC) [16]
mPFS = 58 weeks
SD = 34%
PR = 25%
DCR = 59%
HFSR (22%), hypertension (16%), weight loss (9%) • Dose reduction (due to AEs) was required in 56% of patients
  1. *DTC cohort
  2. Total study sample
  3. One patient developed grade 3/4 LFT abnormalities at 8 weeks and died of liver failure 12 weeks later
  4. DTC = differentiated thyroid cancer; mPFS = median progression-free survival; SD = stable disease; PR = partial response;
  5. DCR = disease control rate; LFTs = liver function tests; HFSR = hand-foot skin reaction; AE = adverse event