Oral Miltefosine as Salvage Therapy for Refractory Acanthamoeba Keratitis

Praneetha Thulasi, Hajirah N. Saeed, Christopher J. Rapuano, Joshua H. Hou, Alpheus B. Appenheimer, James Chodosh, Joann J. Kang, Amber M. Morrill, Neil Vyas, Michael E. Zegans, Richard Zuckerman, Elmer Y. Tu

Research output: Contribution to journalArticlepeer-review


Purpose: To report a case series of patients with treatment-resistant Acanthamoeba keratitis (AK) using oral miltefosine, often as salvage therapy. Design: Descriptive, retrospective multicenter case series. Methods: We reviewed 15 patients with AK unresponsive to therapy who were subsequently given adjuvant systemic miltefosine between 2011 and 2017. The main outcome measures were resolution of infection, final visual acuity, tolerance of miltefosine, and clinical course of disease. Results: All patients were treated with biguanides and/or diamidines or azoles without resolution of disease before starting miltefosine. Eleven of 15 patients retained count fingers or better vision, and all were considered disease free at last follow-up. Eleven of 15 patients had worsening inflammation with miltefosine, with 10 of them improving with steroids. Six patients received multiple courses of miltefosine. Most tolerated oral miltefosine well, with mild gastrointestinal symptoms as the most common systemic side effect. Conclusions: Oral miltefosine is a generally well-tolerated treatment adjuvant in patients with refractory AK. The clinician should be prepared for a steroid-responsive inflammatory response frequently encountered during the treatment course.

Original languageEnglish (US)
Pages (from-to)75-82
Number of pages8
JournalAmerican journal of ophthalmology
StatePublished - Mar 2021

Bibliographical note

Funding Information:
Funding/support: This work was supported by an unrestricted departmental grant from Research to Prevent Blindness , United States grant (E.Y.T.).

Funding Information:
Financial disclosures: H.S. has received grant support from the National Eye Institute , United States. C.R. has been a Board Member of American Academy of Ophthalmology , United States and Ophthalmic Mutual Insurance Company; and has been consultant and/or lecturer for Glaukos/Avedro, Bio-Tissue, Dompé, Novartis/Shire, Sun Ophthalmics, and TearLab. J.H. holds a patent with Brightstar Therapeutics. J.C. has been a consultant for the U.S. Food and Drug Administration; has received travel funds from Santen; and has received grant support from the National Eye Institute. M.Z. has received grant support and intramural funding from the National Eye Institute. R.Z. has been a Data and Safety Monitoring Board member for AiCuris for the drug Pritelivir. E.T. has been a consultant for Dompé, Okogen, Kedrion, and Avellino Labs; and has been a member of the medical advisory board of Eversight Eye Bank. The other authors have reported that they have no relationships relevant to the contents of this paper to disclose.

PubMed: MeSH publication types

  • Journal Article

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