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. 2024 Apr 10;8(2):rkae020. doi: 10.1093/rap/rkae020

Table 2.

Clinical trials and comparative effectiveness studies in IgG4-related disease

Study/trial Study design a Study arms Primary outcome Follow-up time Results/status
Glucocorticoids
Masaki et al. (2017) [72] Single-arm open-label trial PSL 0.6 mg/kg/day tapered to maintenance ≤10 mg (median 7 mg/day), n = 61 CR 12 months

  • 62% complete remission

  • 5% ultimately determined not to have IgG4-RD

  • 29 of 44 (66%) with definite IgG4-RD had complete remission at 1 year
Wu et al. (2017) [73] Open-label RCT

  • Group 1: high-dose prednisone: 0.8–1.0 mg/kg/day tapered to maintenance 7.5–10 mg/day, n = 21 (1 w/d)

  • Group 2: medium-dose prednisone: 0.5–0.6 mg/kg/day tapered to maintenance 7.5–10 mg/day, n = 20 (1 w/d)

 CR 24 months

  • CR at 12 weeks: 95% (group 1) vs 95% (group 2), P = 1.00

  • CR at 24 weeks: 95% (group 1) vs 80% (group 2), P = 0.157
Masamune et al. (2017) [74] Open-label RCT (AIP)

  • Group 1: PSL 0.6 mg/kg/day tapered over 12 weeks to maintenance 5–10 mg/day, continued for 26 weeks, n = 19

  • Group 2: PSL (as in group 1) followed by maintenance 5–7.5 mg/day for additional 2.5 years, n = 30

 Relapse 36 months Relapse rate 61% (group 1) vs 24% (group 2), P = 0.007
Conventional synthetic DMARDs
Yunyun et al. (2017) [75] Non-randomized clinical trial

  • Group 1: PDN 0.5–1.0 mg/kg/day for 1 month then tapered by 5 mg/day to maintenance 5–10 mg/day, n = 52

  • Group 2: PDN (as in group 1) + CYC 50–100 mg/day for 3 months then 50 mg/day or 50 mg every other day, n = 50

 Relapse rate 12 months

  • Relapse rate 39% (group 1) vs 12% (group 2)

  • Median time to relapse 7 months (group 1) vs 11 months (group 2), P = 0.018
Yunyun et al. (2019) [76] Open-label RCT

  • Group 1: PDN 0.6–0.8 mg/kg/day tapered to maintenance ≤10 mg/day, n = 35

  • Group 2: PDN (as in group 1) + MMF 1–1.5 g/day for 6 months then tapered to 0.5–1.0 g/day, n = 34

 CR, PR 12 months

  • No statistical difference in CR or PR at 12 weeks

  • Relapses: 40% (group 1) vs 21% (group 2), P = 0.06
Wang et al. (2020) [77] Open-label RCT

  • Group 1: PSL 0.5–0.8 mg/kg/day tapered to 10 mg over 6 months, n = 33

  • Group 2: PSL (as in group 1) + LEF 20 mg daily p.o., n = 33

 Relapse 12 months Hazard ratio for time to relapse 0.35 (0.13, 0.90, P = 0.23), favouring group 2
Biologic DMARDs
Carruthers et al. (2015) [78] Single-arm open-label trial RTX 1 g × 2 doses, either monotherapy (n = 26) or with concomitant glucocorticoids tapered off over 2 months (n = 4) At 6 months: decline in IgG4-RD RI, no relapses, and no GC use after 2 months 12 months

  • Primary outcome achieved in 77%

  • 47% in CR at 6 months, 40% at 12 months
Ebbo et al. (2017) [79] Retrospective cohort study

  • Group 1: no RTX maintenance, n = 21

  • Group 2: RTX administered before relapse (regimens ranging from 300 mg to 1 g, every 1 month to 17 months), n = 12

 Relapse Mean 25 months

  • Mean time to relapse 21 months (group 1) vs 41 months (group 2)

  • Hazard ratio for time to relapse 0.10 (0.02, 0.69, P = 0.02), favouring group 2
Majumder et al. (2018) [80] Retrospective cohort study (pancreaticobiliary IgG4-RD)

  • Group 1: RTX induction only (375 mg/m2 weekly × 4 or 1000 mg biweekly × 2), n = 14

  • Group 2: RTX induction (as in group 1) and maintenance (375 mg/m2 or 1 g every 2–6 months), n = 29

 Relapse rate Median 34 months (group 1), 27 months (group 2)

  • 86% CR or PR and off GCs at 6 months after induction

  • Relapse rate 45% (group 1) vs 11% (group 2), P = 0.034
Campochiaro et al. (2020) [81] Retrospective cohort study

  • Group 1: RTX induction only (1 g × 2 separated by 15 days), n = 7

  • Group 2a: RTX induction (as in group 1) + maintenance (1 g × 2 every 6 months), n = 4

  • Group 2b: RTX induction (as in group 1) + maintenance (1 g × 1 every 6 months), n = 3

 Relapse at 18 months Median 26 months (group 1), 19 months (group 2a), 21 months (group 2b) 71% relapse (group 1) vs 0% relapse (group 2), P = 0.006
Matza et al. (2022) [82] Single-arm open-label trial Abatacept 125 mg s.c. weekly, n = 10 CR 6 months

  • CR at 12 weeks in 30%

  • 60% PR at 12 weeks, 50% at week 24
Perugino et al. (2023) [83] Single-arm, open-label trial Obexelimab 5 mg/kg i.v. every 2 weeks with GCs discontinued within 2 months, n = 15 Decline in IgG4-RD RI 6 months

  • 80% met primary endpoint, 93% with any response

  • Median time to response 15 days
Other
Zhang et al. (2019) [84] Single-arm open-label trial (mild disease) One i.m. injection of 5 mg betamethasone dipropionate with 2 mg betamethasone sodium phosphate + iguratimod 25 mg p.o. twice daily, n = 30 CR, PR 6 months

  • Week 12: 33% CR, 53% PR

  • Week 24: 30% CR, 57% PR
a

Studies specific to AIP were limited to randomized controlled trials only.

AIP: autoimmune pancreatitis; CR: complete remission/response; GC: glucocorticoid; PDN: prednisone; PR: partial remission/response; PSL: prednisolone; RCT: randomized controlled trial; RI: responder index; RTX: rituximab; w/d: withdrawal.