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Table 2 Information about included clinical trials

From: Polyamines and related signaling pathways in cancer

  Disease Population Population characteristics Observation target Groups Dosage Duration Outcomes
Age Gender (M/F)
Lewis 2020 [90] Neuroblastoma Patients with HRNB who completed standard upfront therapy without progression were eligible for enrollment Mean = 4.0 96 (61.1%)/60 (38.2%) Event‐free survival (EFS) and overall survival (OS) DFMO/No treatment 750±250 mg/m2/dose, twice daily 5 years 2 year EFS: DFMO:86.4% [95% CI, 79.3–94.2%]; No treatment:78.3% [95% CI, 69.5–88.3%]. 5 year EFS: DFMO: 85.2% [95% CI, 77.8–93.3%]; No treatment:65.6% [95% CI, 55.5–77.7%]. 2 year OS: DFMO:8.8% [95% CI, 96.4–100%]; No treatement:94.4%[95% CI, 89.3–99.9%. 5 year OS: DFMO:95.1% [95% CI, 90.5–99.9%]; No treatment:81.6%[95% CI, 73.0–91.2%]
Lynch 2016 [93] Familial adenomatous polyposis Study population was eligible for FAP diagnosis, requiring an evaluable colon and/or rectal segment and five or more colon polyps at baseline Mean = 38 (18–63) 60 (54%) /52 (46%) Average percentage change in adenoma polyp counts; Average percentage change in adenoma polyp burden; Video scores. DFMO+CXB/CXB CXB, 400 mg orally twice a day; DFMO 0.5 g/m2/day 6 months Average percentage change in adenoma polyp counts:CXB + DFMO:− 13.0%; CXB:− 1.0%; average percentage change in adenoma polyp burden:CXB + DFMO:− 40%; CXB:− 27%; video scores:CXB + DFMO:− 0.80; CXB:− 0.33
Jeter 2016 [96] Sun-damaged skin Individuals over 40 years of age with visible sun damage to their skin; Individuals with a history of non-melanoma skin cancer received specific cancer treatment at least 30 days prior to enrollment (outside the forearm) or 6 months prior to enrollment (above the forearm) Mean = 60.4±10.2 44 (28%) /112 (72%) Average nuclear abnormality (ANA); Proportion of nuclei assigned to the baseline; Histological score DFMO+Diclofenac/Diclofenac/DFMO 10% DFMO cream (designated 5 cm×5 cm area on the left forearm twice a day); 3% diclofenac gel (designated 5 cm×5 cm area on the left upper arm once a day) 90 days Average nuclear abnormality (ANA): higher than baseline; Responders with at least a 30% reduction in the nuclear ratio assigned to baseline: DFMO+Diclofenac: 13/45 (29%), Diclofenac: 16/49 (33%), DFMO: 12/50 (24%); histological score: No significant difference from baseline
Bailey 2010 [92] Non-melanoma skin cancer (NMSC) Participants had a history of basic or squamous skin cancer, had been treated for basal cell carcinoma or squamous cell carcinoma (stage 0–2), were over 21 years of age, had ECOG status of 0 or 1, and were more than four weeks from prior major surgery or cancer chemotherapy, radiotherapy, or hormone therapy Mean = 60.9 175 (60%)/116 (40%) Number of new NMSC events; probability of NMSC occurrence DFMO/Placebo DFMO:0.5 g/m2/day (0.2 g/ml DFMO liquid or 250 mg DFMO tablet) 3–5 years Number of new NMSCs: DFMO: 260; placebo: 363; Probability of new NMSCS (/person/year): DFMO: 0.44; placebo: 0.61
Bartels2009 [97] Actinic skin keratoses Participants must have at least 2 actinic keratosis on the posterolateral forearm and moderate to severe sunburn as assessed by a dermatologist Not shown. But there was no difference in the distribution of population characteristics between groups Percentage of nuclei with ANA>1.0; Percentage of nuclei assigned to the baseline data set DFMO+triamcilone/DFMO+Eucerin/triamcilone+Eucerin/Eucerin+Eucerin 10% DFMO; 1%Triamcinolone; A dose (1-inch) of the test article or placebo to each posterolateral forearm once daily. 6 months Changes in percentage of nuclei with ANA>1.0:DFMO+triamcilone:− 6.3%; DFMO+Eucerin:− 2.9%; triamcilone+Eucerin:− 18.7%; Eucerin+Eucerin:4.5%; Changes in percentage of nuclei assigned to the baseline data set:DFMO+triamcilone:− 15.9% vs Eucerin+Eucerin: − 6.3%; DFMO+Eucerin:− 17.9% vs Eucerin+Eucerin:− 10.8%; triamcilone+Eucerin:− 25.6% vs Eucerin+Eucerin:4.8%
Messing 2006 [91] Low risk superficial bladder cancer Patients with stage 1 or stage 2 TA or T1 urothelial carcinoma who have undergone complete endoscopic resection or occasional recurrence (less than 1 recurrence per year and no more than 3 total recurrences) Mean = 64.9 348 (76.7%)/106 (23.3%) Tumor recurrence DFMO/Placebo 1 gm daily 12 months (42 months follow-up) At least 1 case of tumor recurrence:DFMO:103 (46%); Placebo:97 (43%); Progress to TIS or level 3: DFMO:10 (4.4%); Placebo:9(3.9%)
Vlastos 2005 [94] Cervical intraepithelial neoplasia Women over 18 years of age who are not pregnant and have biopsy proven squamous intraepithelial neoplasia (CIN 2 or 3, or CIS) Mean = 31 (18–75) 0/100% Histopathologic response Placebo/DFMO 0.125 gm/m2/ DFMO 0.5 gm/m2 DFMO:0.125 and 0.5 gm/m2 28 days Histopathologic response:0.125 gm/m2 DFMO:Complete regression:4; Partial regression:13; No change/progression:30; 0.5 gm/m2 DFMO:Complete regression:2; Partial regression:14; No change/progression:31; Placebo:Complete regression:4; Partial regression:13; No change/progression:30