Table (E) T-1. Summary of clinical and radiologic response to therapy with pirfenidone
Pt# / Months
of therapy / Best response to therapy (^^) / Last measured response (^^) / % tumor volume change from baseline (^) / Neurologic deficit
(scale 1-10) / Subjective symptoms (scale 1-10) / Dose
mg/day / Tumor location and symptoms / Treatment termination/
Toxicity/
Comments
Months / Months / Months
6 / 12 / 18 / 24 / 6 / 12 / 18 / 24 / 6 / 12 / 18 / 24
1 / 24 / Minor response
(sustained)^ / Minor response / 0 / -3 / - / -17 / 10 / 8 / 7 / 7 / 8 / 8 / 8 / 8 / 2,400 / Pelvis, pain and weakness in legs / Nausea**
2 / 24 / Progression
(sustained) / Progression / +28 / +40 / +40 / +38 / - / - / - / - / 6 / 6 / 6 / 6 / 2,400 / Pelvis, hip pain / Nausea**
3 / 12 / Stable / Stable / - / +11 / - / - / - / - / - / - / - / - / - / - / 2,400 / Neck, post-surgical residual tumor, risk for re-grow / Non-compliance with follow-ups*
4 / 24 / Progression
(non-sustained) / Stable / +3 / +26 / - / +13 / 8 / 5 / 5 / 5 / 7 / 3 / 3 / 3 / 2,400 / Brachial plexus, pain and weakness in arms / Upset stomach, fatigue, gastritis**
5 / 12 / Minor response (non-sustained) / Stable / -20 / -14 / - / - / 9 / 7 / - / - / 7 / 6 / - / - / 1,200-1,800 / Paraspinal tumors, cord compression, quadriparesis, spasticity / Toxicity (nausea, lightheadedness, nasal congestion)***
6 / 24 / Stable / Stable / - / +9 / - / -2 / - / - / - / - / 6-7 / 4-5 / 4-5 / 4-5 / 1,200 / Chest tumor, pain, weakness and numbness in hands / Nausea and fatigue with full dose**
7 / 12 / Partial response (non-sustained) / Partial response / -4 / -37 / - / - / 7 / 8 / - / - / 8 / 8 / - / - / 2,400 / Neck and chest tumors, back pain, legs weakness / Cochlear implant (hereditary hearing loss), no follow-up scans *
8 / 24 / Stable / Stable / - / +3 / +1 / +2 / 8 / 8 / - / - / 8 / 8 / - / - / 2,400 / Pelvis, pressure to bladder, dysuria
9 / 24 / Stable / Stable / +2 / -7 / -3 / +12 / - / - / - / - / 3 / 2 / 2 / 2 / 2,400 / Pelvis, leg pain / Nausea, dizziness, upset stomach**
10 / 24 / Stable / Stable / - / - / -9 / -7 / 2 / 2 / 2 / 2 / 8-9 / 7-8 / 5 / 4-5 / 2,400 / Pelvis, pains, leg weakness
11 / 24 / Stable / Stable / +1 / +2 / -4 / -5 / - / - / - / - / 8-9 / 9 / 8 / 8 / 2,400 / Neck/chest tumor, arm weakness
12 / 24 / Progression (non-sustained) / Stable / -2 / +10 / +18 / +13 / - / - / - / - / 5 / 4 / 4 / 4 / 1200-2400 / Face and neck tumor, sense of pressure, obstructed ear canal / Reduced dose due young age (15)*
13 / 18 / Stable / Stable / +2 / +4 / -3 / - / - / - / - / - / 5-6 / 5-6 / 5-6 / - / 2,400 / Pelvis, leg pain / Persistent pain*
14 / 24 / Progression (non-sustained) / Stable / +34 / +12 / +37 / +14 / - / - / - / - / 3 / 3 / 3 / 3 / 2,400 / Chest tumor, arm numbness and pain
15 / 24 / Partial response (non-sustained) / Progression / -39 / +12 / +58 / +274 / 3 / 3 / 3 / 3 / 6-7 / 3 / 2-3 / 8-9 / 2,400 / Pelvis, leg weakness, pain / Dizziness**
Developed MPNST
16 / 24 / Progression (non-sustained) / Stable / +13 / +2 / +17 / -1 / - / - / - / - / 6 / 5-6 / 4-5 / 4-5 / 2,400 / Back and pelvis tumors, severe back and leg pain / Grade 2 skin rash and photosensitivity**
17 / 24 / Minor response (sustained) / Minor response / -22 / -27 / -24 / -22 / 3 / 3 / 3 / 3 / 4 / 3 / 3 / 3 / 2,400 / Leg, swelling and pain of the leg
18 / 24 / Stable / Stable / +6 / +7 / +13 / +8 / 2 / 3 / 3 / 3 / - / - / - / - / 2,400 / Lumbar plexus tumor, gait problem, leg weakness
19 / 24 / Stable / Stable / +10 / -8 / +8 / -1 / - / - / - / - / 4 / 3 / 3 / 3 / 2,400 / Face, swelling, sense of pressure / Nausea**
20 / 24 / Minor response (sustained) / Minor response / -16 / - / -28 / -15 / 3 / 2 / 2 / 2 / 5 / 5-6 / 5-6 / 6 / 2,400 / Leg, pain and weakness / Dizziness and nausea**
21 / 24 / Minor response (non-sustained)^ / Progression / 0 / +5 / -16 / +15 / 3 / 3-4 / 3-4 / 3-4 / 10 / 5 / 5-6 / 6 / 2,400 / Chest, pain and weakness in arms / New skin tumors*
22 / 18 / Stable / Stable / +8 / +8 / +12 / - / 4-5 / 4-5 / 4-5 / - / 8-9 / 7-8 / 8-9 / - / 2,400 / Pelvis, leg pain / Persistent pain*
23 / 6 / Stable / Stable / +13 / - / - / - / 4-5 / - / - / - / 5 / - / - / - / 2,400 / Paraspinal tumors, spasticity, back pain / Toxicity (persistent nausea)***
24 / 6 / Stable / Stable / +8 / - / - / - / 3-4 / - / - / - / - / - / - / - / 2,400 / Abdominal tumor, pain, potential for vascular obstruction with tumor growth / Toxicity (upset stomach, non-specific abdominal complaints)***
^For two patients (# 1 and #21), the 6-months measurement was used as the baseline (Volume 0), since the baseline scans were not technically comparable. Some patients missed scans in at least one assessment time point (-)
^^Response criteria were defined based on tumor volume change on follow-up 3D-MRI scans compared to the pretreatment volume (see legend below the Figure 1).
*** Treatment terminating toxicity
** Toxicity not requiring treatment termination
* Other comments