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Table 1 Study Characteristics of Clinical Trials focused on management of Cancer Cachexia (2005–2016)

From: Pharmacological management of cachexia in adult cancer patients: a systematic review of clinical trials

Author, year, Study design Number of patients Cancer type Definition of cachexia Intervention Study duration Outcome measures Results1 (Weight) (Anorexia-Cachexia Score) (LBM) Results 2 (Appetite) Results 3 (Functioning) Results 4 (QOL-indicators) Survival Attrition/Completion Rate (%)
Group 1: Appetite Stimulants
 Strasser (2006) [22] RCT 243 Multiple ≥5% loss of body weight Arm1: CE
Arm2: THC
Arm3: PL
6 weeks Appetite (VAS)
QOL (EORTC QLQ-C30)
Body Weight
No differences on body weight or or Anorexia-Cachexia EORTC
QLQ-C30 scores (p = NA)
No differences in appetite (VAS) scores between 3 groups (p = 0.46 and 0.95 respectively) No significant differences on physical, role, emotional, cognitive, and social functioning No differences in QOL indicators (p = 0.80 and 0.43 respectively) N/A 68
 Del Fabbro (2013) [24] RCT 48 Lung/GI ≥5% loss of body weight Arm1: Melatonin
Arm2: Placebo
28 days Appetite change (ESAS)
Body weight
FACIT-F, Functional Assessment for Chronic Illness Therapy–Fatigue
NS (p = 0.17) NS (p = 0.8) No significant difference on FACIT Scale (p = 0.65) No differences in scores on depression, wellbeing, pain and insomnia N/A 66
 Garcia (2015) [25] RCT 82 Multiple (mostly colon and lung) ≥5% loss of body weight Arm1: Anamorelin (50 mg)
Arm2: Placebo
12 weeks LBM
Handgrip strength
ASAS score
Improvement in both lean body mass (p = 0.0006) and appendicular body mass (p = 0.006) in anamorelin as compared to placebo. No differences in fat mass. Non-significant improvement (p = 0.36) Handgrip Strength improved in A group as compared to P group (p = 0.014) ASAS score improved in Anamorelin as compared to Placebo Group (p = 0.03) N/A 56
 Takayama (2016) [26] RCT 181 NSCLC ≥5% loss of body weight Arm1: Anamorelin (50 mg)
Arm2: Anamorelin (100 mg)
Arm3: Placebo
12 weeks LBM
Handgrip strength
QOL-ACD
Improved weight in anamorelin 50 and 100 mg (p = 0.02 and 0.0002) respectively as compared to placebo.
Improvement in lean body mass (LBM) in both groups (P=NS)
Significant improvement in QOL-ACD appetite score for Anamorelin 100 mg as compared to placebo (p = 0.03) No differences in handgrip strength across all groups. (p = NS) Significant improvement in QOL-ACD total score in Anamorelin 100 mg as compared to placebo (p = 0.01) NS (=0.08, for 100 mg, =0.70 for 50 mg) 96
 Temel (2016) [27] RCT ROMANA1: 484
ROMANA2: 495
NSCLC ≥5% loss of body weight or BMI < 20Kg/m2 Arm1: Anamorelin (100 mg)
Arm2: Placebo
3 months LBM
Handgrip strength
Anorexia-cachexia score
ROMANA1
Arm1: + 0.99 kg
Arm2: -0.47 kg
P = < 0.0001
ROMANA2
Arm1:+ 0.65 kg
Arm2:-0.98 kg
P = < 0.0001
N/A ROMANA1
Arm1:-1.10 kg
Arm2:=1.58 kg
P = 0.15
ROMANA2
Arm1:-1.49 kg
Arm2:-0.95 kg
P = 0.65
ROMANA1
Arm1:4.12
Arm2:1.92
P = 0.0004
ROMANA2
Arm1:3.48
Arm2:1.34
P = 0.0016
NS (p = 0.47) 85
 Katakami (2018) [29] RCT 172 NSCLC ≥5% loss of body weight Arm 1: Anamorelin (100 mg)
Arm 2: Placebo
3 months LBM (DEXA)
Body weight
Appetite
Handgrip Strengh (Dynamometer)
QOL (QOL-ACD)
Arm 1: + 1.06 kg
Arm 2: −0.50 kg
P < 0.0001
Arm 1: + 0.7
Arm 2: + 0.3
P = 0.0005
NS (p = 0.08) Significant improvement in QOL in Arm 1 vs Arm 2(p = 0.02) NS (p = 0.37) 68
 Turcott (2018) [23] RCT 47 NSCLC Based on AC/S of the Functional Assessment of Anorexia Cachexia Therapy (FAACT) tool Arm 1: Nalibone (0.5 mg)
Arm 2: Placebo
2 weeks Anorexia (FAACT)
Appetite (VAS)
QOL (EORTC-QOL-C30)
No difference (p = 0.72) No difference (p = 0.21) N/A NS NA 70
Group 2: Cytokine Modulators
 Jatoi (2007) [32] RCT 63 Multiple (mostly lung and GI, exclude brain tumors) 1.Weight loss (> 2.27 kg in 2 months)
2. Caloric intake< 20 cal/Kg of bw/day
Arm1: Etanercept 25 mg subcutaneous twice wkly
Arm2: placebo
24 weeks 1. > 10% non-fluid weight gain from baseline
2. Appetite
Arm1: (0%)
Arm2: (0%) (p = 0.35)
Appetite: (p = 0.87) N/A NS (p = NA) NS (p = 0.82) 95
 Jatoi (2010) [33] RCT 61 NSCLC Age ≥ 65 years
If < 65 years ECOG PS of 2
Weekly docetaxel+
Arm1: Infliximab 5 mg/kg IV on day 1 and weeks 1, 3, 5
Arm2: placebo
8 weeks Non-fluid weight gain≥10% of baseline weight Arm1 (0%)
Arm2 (0%)
NS (p = 0.17)
N/A N/A NS (p = NA) NS (p = 0.88) 65
 Gordon (2005) [34] RCT 50 Inoperable Pancreatic cancer > 10% weight loss over 6 months Arm1: Thalidomide
Arm2: Placebo
24 weeks 1. Weight change(4wks)
2. Muscle mass
3. grip strength
4.QOL
5. Survival
Arm1:(+ 0.37Kg)
Arm2:(−2.2Kg) (p = 0.005)
N/A Grip strength
Arm1:-0.88
Arm2:-1.00 (p = 0.9)
QOL: No difference (p = NA NS (p = 0.45) 66
 Yennurajalingam (2012) [35] RCT 31 Multiple (Mostly GI, GU) ≥5% loss of body weight Arm1: Thalidomide
Arm2: Placebo
2 weeks Symptom assessment
Functional assessment
NS (p = NA) NS (p = NA) N/A NS (p = NA) N/A 60
 Mehrzad (2016) [36] RCT 70 Multiple ≥5% loss of body weight Arm1: Pentoxifylline
Arm2: Placebo
2 months Body weight
QOL
Arm1: Decreased
Arm2: Decreased
(p = 0.80)
N/A N/A Arm1: Decreased
Arm2: Decreased
(P = 0.037)
N/A 91
Group 3: Anabolic Agents
 Lundholm (2007) [37] RCT 138 GI 1. Weight loss (2–3% of referral weight)
2.Albumin< 36 g/L
Arm1:Insulin (0.11+/−0.05 units/kg/d) + BSC
Arm2:BAS (no placebo)
Variable 1. Body composition
2. Physical activity
3. QOL
NS (p = NA) NS (p = NA) NS (p = NA) NS (p = NA) Significant Arm1: 181 days
Arm2:128 days
P < 0.03
NA
 Dobs (2013) [38] RCT 159 Multiple
(Mostly lung, colorectal)
≥2% loss of body weight Arm1: Enobosarm 1 mg
Arm2: Enobsoarm 3 mg
Arm3: Placebo
113 days LBM
Physical activity (Stair climb time)
QOL (FAACT)
Arm1: Increased (p = 0.0012)
Arm2: Increased
(p = 0.046)
Arm3: No change (p = 0.88)
N/A Arm1:Improved(p = 0.0019)
Arm2: Improved(p = 0.0065)
Arm3: No change (p = 0.11)
Arm1: (P = 0.007)
Arm2: (p = 0.14)
Arm3: (p-0.41)
NS 63
Group 4: Combination Agents
 Wen (2012) [41] RCT 102 Multiple (Mostly lung, gastric) ≥5% loss of body weight Arm1:MA + thalidomide
Arm2: MA
8 weeks Body weight
Fatigue
QOL
Grip strength
NS (=p = 0.05) NS (p = 0.12) NS (p = 0.05) Sig (p < 0.01) N/A 91
 Kanat (2013) [42] RCT 62 Multiple ≥5% loss of body weight Arm1:MA + Meloxicam
Arm2:MA + Meloxicam+EPA
Arm3: Meloxicam+EPA
3 months LBM
BMI
QOL
NS (p = 0.61) N/A N/A NS (p = NA) N/A TBD
 Mantovani (2010) [43] RCT 332 Multiple (Mostly lung, breast, colon) Weight loss > 5% Arm1: MPA or MA
Arm2: EPA supplement
Arm3: L-Carnitine
Arm4: Thalidomide
Arm5: combination
4 months 1.LBM (DEXA)
Grip strength QOL (EQ-5D)
Arm3:(−0.7 kg)
Arm4:(−0.8 kg)
Arm5: (2.1 kg)
(P .007)
Significant improvement in arm 5.(p = 0.0037) NS (p = NA) NS (p = NA) NS (p = NA) 100
 Madeddu (2012) [44] RCT 60 Multiple (Mostly head & neck, lung) ≥5% loss of body weight Arm1: L-Carnitine + Celecoxib
Arm2: L-Carnitine + Celecoxib + MA
4 months LBM
Physical activity
NS(p = 0.33) NS (=0.25) NS(p = 0.08) NS (p = 0.51) NS (p = NA) 93
 Kouchaki (2018) [45] RCT 60 GI > = 5% loss of body weight, 2 years of life expectancy Arm 1: Celecoxib+MA
Arm 2: MA
2 months Body Weight
(> = 5%)
Appetite (VAS)
Grip strength
QOL (EORCT QOL-C30)
NS (0.83) NS (p = 0.05) NS (p = 0.36) NS (p = 0.25) NS (p = NA) 37
  1. CE Cannabis extract, THC Tetrahydrocannabinol, PL Placebo, VAS Visual analog scale, MPA Medroxyprogesterone acetate, MA Megesterol acetate, RCT Randomized controlled trial, QOL Quality of life, NS Not significant, N/A Not available, ESAS Edmonton symptom assessment scale, FAACT Quality of life by the functional assessment of anorexia/cachexia therapy (FAACT) questionnaire, LBM Lean body mass, ASAS Anderson symptom assessment scale, NSCLC Non-small cell lung cancer, BAS Best available support, QOL-ACD Quality of life-anti cancer drugs