Fenretinide (4-HPR): A Preventive Chance for Women at Genetic and Familial Risk?

Table 2

Clinical trials with fenretinide.

Study	Design	Treatment	End points	Outcomes	References
Costa et al. (1989)	Phase I, R, PC (60)	Orally: 100, 200, and 300 mg × 6 months subsequently at 200 mg for another 6 months	Tolerability	Recommended dose for chemoprevention trials of HPR is 200 mg/die.	[38]
Formelli et al. (1989)	Phase II, R, PC (60)	Orally: 100, 200, and 300 mg × 6 months subsequently at 200 mg for another 6 months	Pharmacokinetic	HPR treatment lowers retinol and RPB plasma concentrations. This effect is related to HPR levels and is reversible on cessation of HPR administration.	[39]
Veronesi et al. (1999)	Phase III R (2867)	Orally 200 mg versus no treatment × 5 years	Second breast cancer prevention	No statistically significant effect but a possible benefit in premenopausal women.	[40]
Veronesi et al. (2006)	Phase III, R, 15-year followup (1879)	Orally 200 mg versus no treatment × 5 years; 15-year followup	Second breast cancer prevention	4-HPR induces a significant risk reduction of second breast cancer in premenopausal women, which is remarkable at younger ages, and persists several years after treatment cessation.	[41]

HPR: Fenretinide; PC: placebo controlled; R: randomized; RBP: retinol-binding protein.