Ruxolitinib cream for treatment of vitiligo: a randomised, controlled, phase 2 trial
Vitiligo is a chronic autoimmune disease resulting in skin depigmentation and reduced quality of life. There is no approved treatment for vitiligo repigmentation and current off-label therapies have limited efficacy, emphasising the need for improved treatment options. We investigated the therapeuti...
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| Veröffentlicht in: | The Lancet (British edition) 2020-07, Vol.396 (10244), p.110-120 |
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| creator | Rosmarin, David Pandya, Amit G Lebwohl, Mark Grimes, Pearl Hamzavi, Iltefat Gottlieb, Alice B Butler, Kathleen Kuo, Fiona Sun, Kang Ji, Tao Howell, Michael D Harris, John E |
| description | Vitiligo is a chronic autoimmune disease resulting in skin depigmentation and reduced quality of life. There is no approved treatment for vitiligo repigmentation and current off-label therapies have limited efficacy, emphasising the need for improved treatment options. We investigated the therapeutic potential of ruxolitinib cream in patients with vitiligo and report the efficacy and safety results up to 52 weeks of double-blind treatment.
We did a multicentre, randomised, double-blind, phase 2 study for adult patients with vitiligo in 26 US hospitals and medical centres in 18 states. Patients with depigmentation of 0·5% or more of their facial body surface area (BSA) and 3% or more of their non-facial BSA were randomly assigned (1:1:1:1:1) by use of an interactive response technology system to receive ruxolitinib cream (1·5% twice daily, 1·5% once daily, 0·5% once daily, or 0·15% once daily) or vehicle (control group) twice daily on lesions constituting 20% or less of their total BSA for 24 weeks. Patients in the control group in addition to patients in the 0·15% once daily group who did not show a 25% or higher improvement from baseline in facial Vitiligo Area Scoring Index (F-VASI) at week 24 were re-randomised to one of three higher ruxolitinib cream doses (0·5% once daily, 1·5% once daily, 1·5% twice daily). Patients in the 0·5% once daily, 1·5% once daily, or 1·5% twice daily groups remained at their original dose up to week 52. Patients, investigators, and the study sponsor (except members of the interim analysis and primary endpoint analysis data monitoring teams) remained masked to treatment assignment throughout the study. The primary endpoint was the proportion of patients achieving a 50% or higher improvement from baseline in F-VASI (F-VASI50) at week 24, assessed in the intention-to-treat population. The study is registered with ClinicalTrials.gov, NCT03099304.
Between June 7, 2017, and March 21, 2018, 205 patients were screened for eligibility, 48 were excluded and 157 patients (mean age, 48·3 years [SD 12·9]; 73 [46%] male and 84 [54%] female) were randomly assigned to either an intervention group or the control group. 32 (20%) of 157 were assigned to the control group, 31 (20%) to the 0·15% once daily group, 31 (20%) to the 0·5% once daily group, 30 (19%) to the 1·5% once daily group, and 33 (21%) to the 1·5% twice daily group. F-VASI50 at week 24 was reached by significantly more patients given ruxolitinib cream at 1·5% twice daily (15 [45 |
| doi_str_mv | 10.1016/S0140-6736(20)30609-7 |
| format | Article |
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We did a multicentre, randomised, double-blind, phase 2 study for adult patients with vitiligo in 26 US hospitals and medical centres in 18 states. Patients with depigmentation of 0·5% or more of their facial body surface area (BSA) and 3% or more of their non-facial BSA were randomly assigned (1:1:1:1:1) by use of an interactive response technology system to receive ruxolitinib cream (1·5% twice daily, 1·5% once daily, 0·5% once daily, or 0·15% once daily) or vehicle (control group) twice daily on lesions constituting 20% or less of their total BSA for 24 weeks. Patients in the control group in addition to patients in the 0·15% once daily group who did not show a 25% or higher improvement from baseline in facial Vitiligo Area Scoring Index (F-VASI) at week 24 were re-randomised to one of three higher ruxolitinib cream doses (0·5% once daily, 1·5% once daily, 1·5% twice daily). Patients in the 0·5% once daily, 1·5% once daily, or 1·5% twice daily groups remained at their original dose up to week 52. Patients, investigators, and the study sponsor (except members of the interim analysis and primary endpoint analysis data monitoring teams) remained masked to treatment assignment throughout the study. The primary endpoint was the proportion of patients achieving a 50% or higher improvement from baseline in F-VASI (F-VASI50) at week 24, assessed in the intention-to-treat population. The study is registered with ClinicalTrials.gov, NCT03099304.
Between June 7, 2017, and March 21, 2018, 205 patients were screened for eligibility, 48 were excluded and 157 patients (mean age, 48·3 years [SD 12·9]; 73 [46%] male and 84 [54%] female) were randomly assigned to either an intervention group or the control group. 32 (20%) of 157 were assigned to the control group, 31 (20%) to the 0·15% once daily group, 31 (20%) to the 0·5% once daily group, 30 (19%) to the 1·5% once daily group, and 33 (21%) to the 1·5% twice daily group. F-VASI50 at week 24 was reached by significantly more patients given ruxolitinib cream at 1·5% twice daily (15 [45%] of 33) and 1·5% once daily (15 [50%] of 30) than were treated with vehicle (one [3%] of 32). Four patients had serious treatment-emergent adverse events (one patient in the 1·5% twice daily group developed subdural haematoma; one patient in the 1·5% once daily group had a seizure; one patient in the 0·5% once daily group had coronary artery occlusion; and one patient in the 0·5% once daily group had oesophageal achalasia), all of which were unrelated to study treatment. Application site pruritus was the most common treatment-related adverse event among patients given ruxolitinib cream (one [3%] of 33 in the 1·5% twice daily group; three [10%] of 30 in the 1·5% once daily group; three [10%] of 31 in the 0·5% once daily group; and six [19%] of 31 in the 0·15% once daily group)with three [9%] of 32 patients showing application site pruritis in the control group. Acne was noted as a treatment-related adverse event in 13 (10%) of 125 patients who received ruxolitinib cream and one (3%) of 32 patients who received vehicle cream. All treatment-related adverse events were mild or moderate in severity and similar across treatment groups.
Treatment with ruxolitinib cream was associated with substantial repigmentation of vitiligo lesions up to 52 weeks of treatment, and all doses were well tolerated. These data suggest that ruxolitinib cream might be an effective treatment option for patients with vitiligo.
Incyte.</description><identifier>ISSN: 0140-6736</identifier><identifier>EISSN: 1474-547X</identifier><identifier>DOI: 10.1016/S0140-6736(20)30609-7</identifier><identifier>PMID: 32653055</identifier><language>eng</language><publisher>England: Elsevier Ltd</publisher><subject>Achalasia ; Acne ; Adult ; Autoimmune diseases ; Biomarkers ; Blood ; Brain ; Case-Control Studies ; Clinical trials ; Coronary artery ; Double-Blind Method ; Double-blind studies ; Dura mater ; Esophagus ; Female ; Health care facilities ; Health services ; Hematoma ; Humans ; Inhibitor drugs ; Interactive systems ; Janus Kinase Inhibitors - administration & dosage ; Janus Kinase Inhibitors - adverse effects ; Janus Kinase Inhibitors - therapeutic use ; Lesions ; Light therapy ; Male ; Meninges ; Middle Aged ; Occlusion ; Patients ; Population studies ; Pruritus ; Pyrazoles - administration & dosage ; Pyrazoles - adverse effects ; Pyrazoles - therapeutic use ; Quality of life ; Randomization ; Seizures ; Skin ; Skin Cream - administration & dosage ; Skin diseases ; Treatment Outcome ; Vitiligo ; Vitiligo - drug therapy</subject><ispartof>The Lancet (British edition), 2020-07, Vol.396 (10244), p.110-120</ispartof><rights>2020 Elsevier Ltd</rights><rights>Copyright © 2020 Elsevier Ltd. All rights reserved.</rights><rights>2020. Elsevier Ltd</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c445t-931a5f2dae5f2797594643bfc1bcba6c5c4a0c986636c7e7e25710a544ea9c443</citedby><cites>FETCH-LOGICAL-c445t-931a5f2dae5f2797594643bfc1bcba6c5c4a0c986636c7e7e25710a544ea9c443</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://www.proquest.com/docview/2424137496?pq-origsite=primo$$EHTML$$P50$$Gproquest$$H</linktohtml><link.rule.ids>314,780,784,3550,27924,27925,45995,64385,64389,72469</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/32653055$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Rosmarin, David</creatorcontrib><creatorcontrib>Pandya, Amit G</creatorcontrib><creatorcontrib>Lebwohl, Mark</creatorcontrib><creatorcontrib>Grimes, Pearl</creatorcontrib><creatorcontrib>Hamzavi, Iltefat</creatorcontrib><creatorcontrib>Gottlieb, Alice B</creatorcontrib><creatorcontrib>Butler, Kathleen</creatorcontrib><creatorcontrib>Kuo, Fiona</creatorcontrib><creatorcontrib>Sun, Kang</creatorcontrib><creatorcontrib>Ji, Tao</creatorcontrib><creatorcontrib>Howell, Michael D</creatorcontrib><creatorcontrib>Harris, John E</creatorcontrib><title>Ruxolitinib cream for treatment of vitiligo: a randomised, controlled, phase 2 trial</title><title>The Lancet (British edition)</title><addtitle>Lancet</addtitle><description>Vitiligo is a chronic autoimmune disease resulting in skin depigmentation and reduced quality of life. There is no approved treatment for vitiligo repigmentation and current off-label therapies have limited efficacy, emphasising the need for improved treatment options. We investigated the therapeutic potential of ruxolitinib cream in patients with vitiligo and report the efficacy and safety results up to 52 weeks of double-blind treatment.
We did a multicentre, randomised, double-blind, phase 2 study for adult patients with vitiligo in 26 US hospitals and medical centres in 18 states. Patients with depigmentation of 0·5% or more of their facial body surface area (BSA) and 3% or more of their non-facial BSA were randomly assigned (1:1:1:1:1) by use of an interactive response technology system to receive ruxolitinib cream (1·5% twice daily, 1·5% once daily, 0·5% once daily, or 0·15% once daily) or vehicle (control group) twice daily on lesions constituting 20% or less of their total BSA for 24 weeks. Patients in the control group in addition to patients in the 0·15% once daily group who did not show a 25% or higher improvement from baseline in facial Vitiligo Area Scoring Index (F-VASI) at week 24 were re-randomised to one of three higher ruxolitinib cream doses (0·5% once daily, 1·5% once daily, 1·5% twice daily). Patients in the 0·5% once daily, 1·5% once daily, or 1·5% twice daily groups remained at their original dose up to week 52. Patients, investigators, and the study sponsor (except members of the interim analysis and primary endpoint analysis data monitoring teams) remained masked to treatment assignment throughout the study. The primary endpoint was the proportion of patients achieving a 50% or higher improvement from baseline in F-VASI (F-VASI50) at week 24, assessed in the intention-to-treat population. The study is registered with ClinicalTrials.gov, NCT03099304.
Between June 7, 2017, and March 21, 2018, 205 patients were screened for eligibility, 48 were excluded and 157 patients (mean age, 48·3 years [SD 12·9]; 73 [46%] male and 84 [54%] female) were randomly assigned to either an intervention group or the control group. 32 (20%) of 157 were assigned to the control group, 31 (20%) to the 0·15% once daily group, 31 (20%) to the 0·5% once daily group, 30 (19%) to the 1·5% once daily group, and 33 (21%) to the 1·5% twice daily group. F-VASI50 at week 24 was reached by significantly more patients given ruxolitinib cream at 1·5% twice daily (15 [45%] of 33) and 1·5% once daily (15 [50%] of 30) than were treated with vehicle (one [3%] of 32). Four patients had serious treatment-emergent adverse events (one patient in the 1·5% twice daily group developed subdural haematoma; one patient in the 1·5% once daily group had a seizure; one patient in the 0·5% once daily group had coronary artery occlusion; and one patient in the 0·5% once daily group had oesophageal achalasia), all of which were unrelated to study treatment. Application site pruritus was the most common treatment-related adverse event among patients given ruxolitinib cream (one [3%] of 33 in the 1·5% twice daily group; three [10%] of 30 in the 1·5% once daily group; three [10%] of 31 in the 0·5% once daily group; and six [19%] of 31 in the 0·15% once daily group)with three [9%] of 32 patients showing application site pruritis in the control group. Acne was noted as a treatment-related adverse event in 13 (10%) of 125 patients who received ruxolitinib cream and one (3%) of 32 patients who received vehicle cream. All treatment-related adverse events were mild or moderate in severity and similar across treatment groups.
Treatment with ruxolitinib cream was associated with substantial repigmentation of vitiligo lesions up to 52 weeks of treatment, and all doses were well tolerated. These data suggest that ruxolitinib cream might be an effective treatment option for patients with vitiligo.
Incyte.</description><subject>Achalasia</subject><subject>Acne</subject><subject>Adult</subject><subject>Autoimmune diseases</subject><subject>Biomarkers</subject><subject>Blood</subject><subject>Brain</subject><subject>Case-Control Studies</subject><subject>Clinical trials</subject><subject>Coronary artery</subject><subject>Double-Blind Method</subject><subject>Double-blind studies</subject><subject>Dura mater</subject><subject>Esophagus</subject><subject>Female</subject><subject>Health care facilities</subject><subject>Health services</subject><subject>Hematoma</subject><subject>Humans</subject><subject>Inhibitor drugs</subject><subject>Interactive systems</subject><subject>Janus Kinase Inhibitors - administration & dosage</subject><subject>Janus Kinase Inhibitors - adverse effects</subject><subject>Janus Kinase Inhibitors - therapeutic use</subject><subject>Lesions</subject><subject>Light therapy</subject><subject>Male</subject><subject>Meninges</subject><subject>Middle Aged</subject><subject>Occlusion</subject><subject>Patients</subject><subject>Population studies</subject><subject>Pruritus</subject><subject>Pyrazoles - administration & dosage</subject><subject>Pyrazoles - adverse effects</subject><subject>Pyrazoles - therapeutic use</subject><subject>Quality of life</subject><subject>Randomization</subject><subject>Seizures</subject><subject>Skin</subject><subject>Skin Cream - administration & dosage</subject><subject>Skin diseases</subject><subject>Treatment Outcome</subject><subject>Vitiligo</subject><subject>Vitiligo - drug 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cream for treatment of vitiligo: a randomised, controlled, phase 2 trial</title><author>Rosmarin, David ; Pandya, Amit G ; Lebwohl, Mark ; Grimes, Pearl ; Hamzavi, Iltefat ; Gottlieb, Alice B ; Butler, Kathleen ; Kuo, Fiona ; Sun, Kang ; Ji, Tao ; Howell, Michael D ; Harris, John E</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c445t-931a5f2dae5f2797594643bfc1bcba6c5c4a0c986636c7e7e25710a544ea9c443</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2020</creationdate><topic>Achalasia</topic><topic>Acne</topic><topic>Adult</topic><topic>Autoimmune diseases</topic><topic>Biomarkers</topic><topic>Blood</topic><topic>Brain</topic><topic>Case-Control Studies</topic><topic>Clinical trials</topic><topic>Coronary artery</topic><topic>Double-Blind Method</topic><topic>Double-blind studies</topic><topic>Dura mater</topic><topic>Esophagus</topic><topic>Female</topic><topic>Health care 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Korea</collection><collection>British Nursing Index (BNI) (1985 to Present)</collection><collection>Health Research Premium Collection</collection><collection>Health Research Premium Collection (Alumni)</collection><collection>ProQuest Central Student</collection><collection>Research Library Prep</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>SciTech Premium Collection</collection><collection>British Nursing Index</collection><collection>Consumer Health Database (Alumni Edition)</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Nursing & Allied Health Database (Alumni Edition)</collection><collection>ProQuest Newsstand Professional</collection><collection>ProQuest Biological Science Collection</collection><collection>Consumer Health Database</collection><collection>Health & Medical Collection (Alumni Edition)</collection><collection>Healthcare Administration Database</collection><collection>Medical Database</collection><collection>Psychology Database</collection><collection>Research Library</collection><collection>Science Database</collection><collection>Algology Mycology and Protozoology Abstracts (Microbiology C)</collection><collection>Biological Science Database</collection><collection>Research Library (Corporate)</collection><collection>Nursing & Allied Health Premium</collection><collection>ProQuest One Academic Eastern Edition (DO NOT USE)</collection><collection>ProQuest One Academic</collection><collection>ProQuest One Academic UKI Edition</collection><collection>ProQuest One Psychology</collection><collection>ProQuest Central Basic</collection><collection>SIRS Editorial</collection><jtitle>The Lancet (British edition)</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Rosmarin, David</au><au>Pandya, Amit G</au><au>Lebwohl, Mark</au><au>Grimes, Pearl</au><au>Hamzavi, Iltefat</au><au>Gottlieb, Alice B</au><au>Butler, Kathleen</au><au>Kuo, Fiona</au><au>Sun, Kang</au><au>Ji, Tao</au><au>Howell, Michael D</au><au>Harris, John E</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Ruxolitinib cream for treatment of vitiligo: a randomised, controlled, phase 2 trial</atitle><jtitle>The Lancet (British edition)</jtitle><addtitle>Lancet</addtitle><date>2020-07-11</date><risdate>2020</risdate><volume>396</volume><issue>10244</issue><spage>110</spage><epage>120</epage><pages>110-120</pages><issn>0140-6736</issn><eissn>1474-547X</eissn><abstract>Vitiligo is a chronic autoimmune disease resulting in skin depigmentation and reduced quality of life. There is no approved treatment for vitiligo repigmentation and current off-label therapies have limited efficacy, emphasising the need for improved treatment options. We investigated the therapeutic potential of ruxolitinib cream in patients with vitiligo and report the efficacy and safety results up to 52 weeks of double-blind treatment.
We did a multicentre, randomised, double-blind, phase 2 study for adult patients with vitiligo in 26 US hospitals and medical centres in 18 states. Patients with depigmentation of 0·5% or more of their facial body surface area (BSA) and 3% or more of their non-facial BSA were randomly assigned (1:1:1:1:1) by use of an interactive response technology system to receive ruxolitinib cream (1·5% twice daily, 1·5% once daily, 0·5% once daily, or 0·15% once daily) or vehicle (control group) twice daily on lesions constituting 20% or less of their total BSA for 24 weeks. Patients in the control group in addition to patients in the 0·15% once daily group who did not show a 25% or higher improvement from baseline in facial Vitiligo Area Scoring Index (F-VASI) at week 24 were re-randomised to one of three higher ruxolitinib cream doses (0·5% once daily, 1·5% once daily, 1·5% twice daily). Patients in the 0·5% once daily, 1·5% once daily, or 1·5% twice daily groups remained at their original dose up to week 52. Patients, investigators, and the study sponsor (except members of the interim analysis and primary endpoint analysis data monitoring teams) remained masked to treatment assignment throughout the study. The primary endpoint was the proportion of patients achieving a 50% or higher improvement from baseline in F-VASI (F-VASI50) at week 24, assessed in the intention-to-treat population. The study is registered with ClinicalTrials.gov, NCT03099304.
Between June 7, 2017, and March 21, 2018, 205 patients were screened for eligibility, 48 were excluded and 157 patients (mean age, 48·3 years [SD 12·9]; 73 [46%] male and 84 [54%] female) were randomly assigned to either an intervention group or the control group. 32 (20%) of 157 were assigned to the control group, 31 (20%) to the 0·15% once daily group, 31 (20%) to the 0·5% once daily group, 30 (19%) to the 1·5% once daily group, and 33 (21%) to the 1·5% twice daily group. F-VASI50 at week 24 was reached by significantly more patients given ruxolitinib cream at 1·5% twice daily (15 [45%] of 33) and 1·5% once daily (15 [50%] of 30) than were treated with vehicle (one [3%] of 32). Four patients had serious treatment-emergent adverse events (one patient in the 1·5% twice daily group developed subdural haematoma; one patient in the 1·5% once daily group had a seizure; one patient in the 0·5% once daily group had coronary artery occlusion; and one patient in the 0·5% once daily group had oesophageal achalasia), all of which were unrelated to study treatment. Application site pruritus was the most common treatment-related adverse event among patients given ruxolitinib cream (one [3%] of 33 in the 1·5% twice daily group; three [10%] of 30 in the 1·5% once daily group; three [10%] of 31 in the 0·5% once daily group; and six [19%] of 31 in the 0·15% once daily group)with three [9%] of 32 patients showing application site pruritis in the control group. Acne was noted as a treatment-related adverse event in 13 (10%) of 125 patients who received ruxolitinib cream and one (3%) of 32 patients who received vehicle cream. All treatment-related adverse events were mild or moderate in severity and similar across treatment groups.
Treatment with ruxolitinib cream was associated with substantial repigmentation of vitiligo lesions up to 52 weeks of treatment, and all doses were well tolerated. These data suggest that ruxolitinib cream might be an effective treatment option for patients with vitiligo.
Incyte.</abstract><cop>England</cop><pub>Elsevier Ltd</pub><pmid>32653055</pmid><doi>10.1016/S0140-6736(20)30609-7</doi><tpages>11</tpages></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0140-6736 |
| ispartof | The Lancet (British edition), 2020-07, Vol.396 (10244), p.110-120 |
| issn | 0140-6736 1474-547X |
| language | eng |
| recordid | cdi_proquest_journals_2424137496 |
| source | MEDLINE; Elsevier ScienceDirect Journals Complete; ProQuest Central UK/Ireland |
| subjects | Achalasia Acne Adult Autoimmune diseases Biomarkers Blood Brain Case-Control Studies Clinical trials Coronary artery Double-Blind Method Double-blind studies Dura mater Esophagus Female Health care facilities Health services Hematoma Humans Inhibitor drugs Interactive systems Janus Kinase Inhibitors - administration & dosage Janus Kinase Inhibitors - adverse effects Janus Kinase Inhibitors - therapeutic use Lesions Light therapy Male Meninges Middle Aged Occlusion Patients Population studies Pruritus Pyrazoles - administration & dosage Pyrazoles - adverse effects Pyrazoles - therapeutic use Quality of life Randomization Seizures Skin Skin Cream - administration & dosage Skin diseases Treatment Outcome Vitiligo Vitiligo - drug therapy |
| title | Ruxolitinib cream for treatment of vitiligo: a randomised, controlled, phase 2 trial |
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