Oral cyclophosphamide monotherapy in advanced resistant ocular cicatricial pemphigoid

PURPOSE: The purpose of the study was to study the efficacy of oral cyclophosphamide monotherapy in advanced resistant ocular cicatricial pemphigoid (OCP). MATERIALS AND METHODS: This retrospective case series examines patients diagnosed with advanced resistant OCP at a uveitis tertiary care center...

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Main Authors: Arash Maleki, Allison Richey, Hasan Bashir, Soheila Asgari, Tate Valerio, Yasmin Massoudi, Andrew Dolinko, Peter Y. Chang, C. Stephen Foster, Stephen D. Anesi
Format: Article
Language:English
Published: Wolters Kluwer Medknow Publications 2025-04-01
Series:Taiwan Journal of Ophthalmology
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Online Access:https://journals.lww.com/10.4103/tjo.TJO-D-24-00057
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author Arash Maleki
Allison Richey
Hasan Bashir
Soheila Asgari
Tate Valerio
Yasmin Massoudi
Andrew Dolinko
Peter Y. Chang
C. Stephen Foster
Stephen D. Anesi
author_facet Arash Maleki
Allison Richey
Hasan Bashir
Soheila Asgari
Tate Valerio
Yasmin Massoudi
Andrew Dolinko
Peter Y. Chang
C. Stephen Foster
Stephen D. Anesi
author_sort Arash Maleki
collection DOAJ
description PURPOSE: The purpose of the study was to study the efficacy of oral cyclophosphamide monotherapy in advanced resistant ocular cicatricial pemphigoid (OCP). MATERIALS AND METHODS: This retrospective case series examines patients diagnosed with advanced resistant OCP at a uveitis tertiary care center who were treated with oral cyclophosphamide therapy. RESULTS: Seventeen patients were included in this study. The average age of the participants was 74.3 ± 10.3 years, ranging from 60 to 99 years. The gender distribution was 8 females to 9 males. Oral cyclophosphamide-induced remission in 7 patients (41.1%) and was prematurely discontinued in 10 patients (58.9%), with 7 (63.7%) citing ineffectiveness and 3 (36.3%) experiencing side effects as reasons for discontinuation. The average therapy dose of cyclophosphamide administered was 116.1 ± 47.5 mg. The average duration required for the induction of remission was 150 ± 128 days. The overall therapy duration averaged 306 ± 189 days. Conducting univariate logistic regression with generalized estimating equations (GEEs) on variables did not reveal statistically significant differences between the two groups (responsive and nonresponsive to oral cyclophosphamide therapy), except for the maximum dose of oral cyclophosphamide therapy (P = 0.007). CONCLUSION: The prognosis of advanced OCP in patients is unlikely to be altered by the stepladder approach and step-up strategy. While oral cyclophosphamide can be efficacious for remission induction in resistant and aggressive OCP cases, a step-down strategy employing less potent agents with safer side effect profiles should be contemplated for subsequent treatment.
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spelling doaj-art-ca0f45cc685c4cab9db9f60404cdf27e2025-08-20T03:16:11ZengWolters Kluwer Medknow PublicationsTaiwan Journal of Ophthalmology2211-50562211-50722025-04-0115229029610.4103/tjo.TJO-D-24-00057Oral cyclophosphamide monotherapy in advanced resistant ocular cicatricial pemphigoidArash MalekiAllison RicheyHasan BashirSoheila AsgariTate ValerioYasmin MassoudiAndrew DolinkoPeter Y. ChangC. Stephen FosterStephen D. AnesiPURPOSE: The purpose of the study was to study the efficacy of oral cyclophosphamide monotherapy in advanced resistant ocular cicatricial pemphigoid (OCP). MATERIALS AND METHODS: This retrospective case series examines patients diagnosed with advanced resistant OCP at a uveitis tertiary care center who were treated with oral cyclophosphamide therapy. RESULTS: Seventeen patients were included in this study. The average age of the participants was 74.3 ± 10.3 years, ranging from 60 to 99 years. The gender distribution was 8 females to 9 males. Oral cyclophosphamide-induced remission in 7 patients (41.1%) and was prematurely discontinued in 10 patients (58.9%), with 7 (63.7%) citing ineffectiveness and 3 (36.3%) experiencing side effects as reasons for discontinuation. The average therapy dose of cyclophosphamide administered was 116.1 ± 47.5 mg. The average duration required for the induction of remission was 150 ± 128 days. The overall therapy duration averaged 306 ± 189 days. Conducting univariate logistic regression with generalized estimating equations (GEEs) on variables did not reveal statistically significant differences between the two groups (responsive and nonresponsive to oral cyclophosphamide therapy), except for the maximum dose of oral cyclophosphamide therapy (P = 0.007). CONCLUSION: The prognosis of advanced OCP in patients is unlikely to be altered by the stepladder approach and step-up strategy. While oral cyclophosphamide can be efficacious for remission induction in resistant and aggressive OCP cases, a step-down strategy employing less potent agents with safer side effect profiles should be contemplated for subsequent treatment.https://journals.lww.com/10.4103/tjo.TJO-D-24-00057cyclophosphamideimmunomodulatory therapyocular cicatricial pemphigoidstep downstep upstepladder approach
spellingShingle Arash Maleki
Allison Richey
Hasan Bashir
Soheila Asgari
Tate Valerio
Yasmin Massoudi
Andrew Dolinko
Peter Y. Chang
C. Stephen Foster
Stephen D. Anesi
Oral cyclophosphamide monotherapy in advanced resistant ocular cicatricial pemphigoid
Taiwan Journal of Ophthalmology
cyclophosphamide
immunomodulatory therapy
ocular cicatricial pemphigoid
step down
step up
stepladder approach
title Oral cyclophosphamide monotherapy in advanced resistant ocular cicatricial pemphigoid
title_full Oral cyclophosphamide monotherapy in advanced resistant ocular cicatricial pemphigoid
title_fullStr Oral cyclophosphamide monotherapy in advanced resistant ocular cicatricial pemphigoid
title_full_unstemmed Oral cyclophosphamide monotherapy in advanced resistant ocular cicatricial pemphigoid
title_short Oral cyclophosphamide monotherapy in advanced resistant ocular cicatricial pemphigoid
title_sort oral cyclophosphamide monotherapy in advanced resistant ocular cicatricial pemphigoid
topic cyclophosphamide
immunomodulatory therapy
ocular cicatricial pemphigoid
step down
step up
stepladder approach
url https://journals.lww.com/10.4103/tjo.TJO-D-24-00057
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