Jorre S Mertens1, Manon C Zweers2, Wietske Kievit3, Hanneke K A Knaapen4, Martijn Gerritsen5, Timothy R D J Radstake6, Frank H J van den Hoogen4, Marjonne C W Creemers7, Elke M G J de Jong2. 1. Department of Dermatology, Radboud University Medical Centre, Nijmegen, the Netherlands2Laboratory of Translational Immunology, University Medical Centre Utrecht, Utrecht, the Netherlands. 2. Department of Dermatology, Radboud University Medical Centre, Nijmegen, the Netherlands. 3. Department of Epidemiology, Health Evidence, Radboud University Medical Centre, Nijmegen, the Netherlands. 4. Department of Rheumatology, Radboud University Medical Centre & Sint Maarstenskliniek, Nijmegen, the Netherlands. 5. Department of Rheumatology, Westfriesgasthuis, Hoorn, the Netherlands. 6. Laboratory of Translational Immunology, University Medical Centre Utrecht, Utrecht, the Netherlands6Department of Rheumatology and Clinical immunology, University Medical Centre Utrecht, Utrecht, the Netherlands. 7. Department of Rheumatology, Jeroen Bosch Hospital-Hertogenbosch, the Netherlands.
Abstract
Importance: Eosinophilic fasciitis (EF) is a connective tissue disorder in which conventional treatment leads to disappointing results in a proportion of patients. Therefore, we investigated high-dose intravenous (IV) pulse methotrexate (MTX) as a treatment for EF. Objective: To examine safety and effects of monthly high-dose IV pulse MTX in EF. Design, Setting, and Participants: For this prospective single-arm study, we recruited 12 patients diagnosed with biopsy specimen-proven EF between 2006 and 2009 from the Department of Dermatology and Rheumatology at the Radboud University Medical Centre. Interventions: Intravenous MTX (4 mg/kg) monthly for 5 months with folinic acid rescue 24 hours after MTX administration. Main Outcomes and Measures: The primary outcome was improvement of the modified skin score at month 5 vs baseline. Secondary outcomes were durometry, range of motion, visual analog scale scores for disease activity, and 36-Item Short Form Survey health questionnaires. Results: Overall, 12 patients (11 women between 37-69 years old) received a median (range) monthly dose of 288 (230-336) mg MTX. Median (range) modified skin score improved from 17.5 (8.0-24.0) at baseline to 8.5 (1.0-20.0) at month 5 (P = .001). Secondary outcome measures improved significantly, except for durometer scores and range of motion of the elbows. Adverse events included gastrointestinal symptoms (n = 9), mild stomatitis (n = 5), and alopecia (n = 4). Conclusions and Relevance: High-dose IV pulse MTX is a safe and effective treatment option in EF. Trial Registration: clinicaltrials.gov Identifier: NCT00441961.
RCT Entities:
Importance: Eosinophilic fasciitis (EF) is a connective tissue disorder in which conventional treatment leads to disappointing results in a proportion of patients. Therefore, we investigated high-dose intravenous (IV) pulse methotrexate (MTX) as a treatment for EF. Objective: To examine safety and effects of monthly high-dose IV pulse MTX in EF. Design, Setting, and Participants: For this prospective single-arm study, we recruited 12 patients diagnosed with biopsy specimen-proven EF between 2006 and 2009 from the Department of Dermatology and Rheumatology at the Radboud University Medical Centre. Interventions: Intravenous MTX (4 mg/kg) monthly for 5 months with folinic acid rescue 24 hours after MTX administration. Main Outcomes and Measures: The primary outcome was improvement of the modified skin score at month 5 vs baseline. Secondary outcomes were durometry, range of motion, visual analog scale scores for disease activity, and 36-Item Short Form Survey health questionnaires. Results: Overall, 12 patients (11 women between 37-69 years old) received a median (range) monthly dose of 288 (230-336) mg MTX. Median (range) modified skin score improved from 17.5 (8.0-24.0) at baseline to 8.5 (1.0-20.0) at month 5 (P = .001). Secondary outcome measures improved significantly, except for durometer scores and range of motion of the elbows. Adverse events included gastrointestinal symptoms (n = 9), mild stomatitis (n = 5), and alopecia (n = 4). Conclusions and Relevance: High-dose IV pulse MTX is a safe and effective treatment option in EF. Trial Registration: clinicaltrials.gov Identifier: NCT00441961.
Authors: Karmela Kim Chan; Cynthia Magro; Alexander Shoushtari; Charles Rudin; Veronica Rotemberg; Anthony Rossi; Cecilia Lezcano; John Carrino; David Fernandez; Michael A Postow; Arlyn Apollo; Mario E Lacouture; Anne R Bass Journal: Oncologist Date: 2019-10-15
Authors: Jorre S Mertens; Marieke M B Seyger; Rogier M Thurlings; Timothy R D J Radstake; Elke M G J de Jong Journal: Am J Clin Dermatol Date: 2017-08 Impact factor: 7.403