1. Enduring Clinical Remission in Refractory Celiac Disease Type II With Tofacitinib: An Open-Label Clinical Study.
- Author
-
Dieckman T, Schumann M, Beaumont H, Bontkes HJ, Koning F, and Bouma G
- Subjects
- Humans, Male, Female, Prospective Studies, Pilot Projects, Middle Aged, Adult, Remission Induction, Treatment Outcome, Protein Kinase Inhibitors therapeutic use, Aged, Netherlands, Pyrimidines therapeutic use, Piperidines therapeutic use, Celiac Disease drug therapy
- Abstract
Refractory celiac disease type 2 (RCDII) is a rare condition with high mortality because of a lack of effective treatment strategies. RCDII is caused by clonal expansion of intraepithelial lymphocytes (IELs). Gain-of-function JAK1 mutations are frequently found in these cells.
1,2 In a previous in vitro study,3 we demonstrated the potential of tofacitinib, a small-molecule JAK1/JAK3 inhibitor, to control activity of the aberrant IEL population. Here, we report on an open-label prospective pilot study with tofacitinib in patients with therapy-refractory RCDII (EudraCT 2018-001678-10; Dutch Trial Registry [LTR] NL7313). Between November 2019 and February 2022, 4 patients with an established diagnosis of RCDII4 who had failed previous therapies were treated in the Netherlands with tofacitinib 10 mg twice-daily for 12 weeks (Methods; Supporting Documents). Two patients in Germany who fulfilled the inclusion criteria received similar treatment outside this protocol., (Copyright © 2024 The Author(s). Published by Elsevier Inc. All rights reserved.)- Published
- 2024
- Full Text
- View/download PDF