Data Highlight Efficacy, Safety of Potential Pharmacologic Treatment for Ptosis

Oxymetazoline, 0.1% may hold potential as a promising treatment option for individuals with acquired blepharoptosis, according to results of a pooled analysis published in JAMA Ophthalmology.

Oxymetazoline, 0.1%, may hold potential as a promising treatment option for individuals with acquired blepharoptosis, according to results of a pooled analysis published in JAMA Ophthalmology. However, future studies are needed to better understand the treatment’s long-term efficacy.

Blepharoptosis, or ptosis, is the abnormal drooping of the upper eyelid when one’s eye is in primary gaze. In some cases, the condition can lead to superior visual deficits if the upper eyelid obstructs the pupil.

Ptosis can be either congenital or acquired. Among those who acquire the condition, the underlying cause could be neurogenic, myogenic, traumatic, or mechanical. “Transient or persistent ptosis can also occur following ocular procedures, such as glaucoma or cataract surgery, and has been associated with contact lens wear,” the researchers wrote.

In addition to reduced visual function, patients with blepharoptosis report high levels of anxiety, depression, and appearance-related distress. The current standard of care for the condition is surgery targeting the upper eyelid retractor muscles; however, those who undergo surgery face the risk of overcorrection, undercorrection, infection, and asymmetry.

“An effective, noninvasive, pharmacologic treatment option might enable treatment of a wider range of affected individuals,” the authors wrote. Oxymetazoline hydrochloride, 0.1%, ophthalmic solution is approved for the treatment of acquired ptosis and is thought to stimulate receptors on the Müller muscle, causing the contraction and lifting of the upper eyelid.

To examine the efficacy and safety of oxymetazoline, 0.1%, for the treatment of acquired ptosis, the researchers combined data from 2 randomized, double-masked, placebo-controlled, multicenter phase 3 clinical trials. All participants were 9 years and older and had both acquired ptosis and superior visual field deficit. RVL-1201-201 and RVL-1201-202 included 304 patients randomized 2:1 to receive treatment with oxymetazoline, 0.1%, or a vehicle (ie, placebo).

Participants self-administered a single drop per eye once daily for 42 days. Follow-up visits occurred at days 1, 14, and 42. The researchers measured efficacy based on change from baseline to day 14 in the number of points seen in the top 4 rows on the Leicester Peripheral Field Test (LPFT).

One hundred and ninety-eight of 203 participants who received the treatment and 98 of 101 participants who received the vehicle completed the studies.

Analyses revealed:

  • Oxymetazoline, 0.1%, was associated with a significant increase in the mean (SD) number of points seen on the LPFT vs vehicle (day 1: 5.9 [6.4] vs 1.8 [4.1]; mean difference, 4.07; 95% CI, 2.74-5.39; P < .001; day 14: 7.1 [5.9] vs 2.4 [5.5]; mean difference, 4.74; 95% CI, 3.43-6.04; P < .001)
  • Oxymetazoline, 0.1%, was associated with a significant increase in marginal reflex distance 1 from baseline (mean [SD]: day 1: 0.96 [0.89] mm vs 0.50 [0.81] mm; mean difference, 0.47 mm; 95% CI, 0.27-0.67; P < .001; day 14: 1.16 [0.87] mm vs 0.50 [0.80] mm; mean difference, 0.67 mm; 95% CI, 0.46-0.8,8; P < .001)
  • After 42 days treatment-emergent adverse events (TEAEs) occurred in 31.0% (63 of 203) of participants receiving oxymetazoline, 0.1%, and 35.6% (36 of 101) receiving vehicle
  • Among participants receiving oxymetazoline, 0.1%, with a TEAE, 81% (51 of 63) had a maximum TEAE intensity of mild, and 62% (39 of 63) had no TEAE suspected of being treatment related
  • No mean clinically relevant shifts from baseline in vital signs or intraocular pressure, Snellen visual acuity, pupil diameter, slitlamp, or ophthalmoscopy/fundus examination results in either eye were found after 42 days

Because of the lack of literature independently validating the clinical relevance of LFPTs, the researchers noted the broader clinical implications of the improvements observed require further evaluation. “Future studies of longer duration of the effect of oxymetazoline, 0.1%, on activities of daily living and psychosocial factors typically affected by ptosis in conjunction with improvements on the LPFT might support the clinical benefit of oxymetazoline, 0.1%,” the authors wrote.

In addition, future studies could compare outcomes with oxymetazoline, 0.1%, treatment vs surgical intervention to better understand long-term treatment efficacy.


Slonim CB, Foster S, Jaros M, et al. Association of oxymetazoline hydrochloride, 0.1%, solution administration with visual field in acquired ptosis. JAMA Ophthalmol. Published online October 1, 2020. doi:10.1001/jamaophthalmol.2020.3812