Authors' recommendations: In two phase III RCTs, ulipristal was shown to reduce uterine bleeding in a greater percentage of patients than placebo in PEARL I and to a similar extent as GnRH agonist (i.e., leuprolide) therapy in PEARL II; hence, ulipristal was found to be non-inferior to leuprolide, based on the pre-specified non-inferiority margin of –20% in PEARL II. There were no clear differences between groups in quality of life or non-menstrual bleeding symptom control outcomes detected during 13 weeks of treatment in either study. A large proportion of surgeries were not completed as planned following preoperative study drug treatment, the reasons for which were not provided. Ulipristal treatment appeared generally well tolerated, with comparatively low incidence of WDAEs and SAEs. Of the two trials, headache and hot flashes were the most frequently presenting AEs for ulipristal-treated patients, but neither these nor any other AEs occurred more frequently than observed in the comparator group. However, long-term safety data (beyond three months) for ulipristal is lacking. Key limitations of the evidence included the lack of North American patients studied, which may reduce generalizability; the lack of pre-specified surgical end points, which limits the ability to fully evaluate ulipristal's potential place in therapy; and a lack of data demonstrating superiority over placebo on validated quality of life instruments — quality of life was identified as a patient-important outcome for this review.

Project Status: Completed

Year Published: 2014

URL for published report: http://www.cadth.ca/media/cdr/clinical/SR0326_Fibristal_CL_Report_e.pdf

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: Canada

Organisation Name: Canadian Agency for Drugs and Technologies in Health

Contact Address: 600-865 Carling Avenue, Ottawa, ON K1S 5S8 Canada. Tel: +1 613 226 2553; Fax: +1 613 226 5392;

Contact Name: requests@cadth.ca

Contact Email: requests@cadth.ca

Copyright: Canadian Agency for Drugs and Technologies in Health (CADTH)