Are there any peer reviewed articles to support use of infliximab for Bechet's disease?

Comment by InpharmD Researcher

The use of infliximab for the management of Behçet's disease is supported by multiple meta-analyses and clinical trials, as well as an abundance of observational data. Infliximab has been found to help improve symptoms of Behçet's uveitis, neuro-Behçet's, and intestinal Behçet's. American guidelines recommend the use of anti-TNF therapy, with infliximab or adalimumab, as a first- or second-line corticosteroid-sparing therapy for patients with ophthalmic manifestations of Behçet's disease. Infliximab may also be considered as a first- or second-line treatment for acute exacerbations of pre-existing Behçet's disease. Of note, European guidelines further recommend the use of anti-TNF therapy for refractory venous thrombosis or arterial involvement, severe and/or refractory gastrointestinal involvement, and severe nervous system involvement. Clinical data have observed similar outcomes when comparing adalimumab or interferon-alfa-2a and infliximab; however, one study found infliximab to be superior to cyclophosphamide in severe Behçet's disease (see Table 1).

Background

A 2014 guidance statement published by the Executive Committee of the American Uveitis Society provided recommendations for the management of Behçet's disease in uveitis. Based on the available evidence, the expert panel recommends the use of anti-tumor-necrosis factor (anti-TNF) therapy, with infliximab or adalimumab, as a first- or second-line corticosteroid-sparing therapy for patients with ophthalmic manifestations of Behçet's disease. Infliximab may also be considered as a first- or second-line treatment for acute exacerbations of pre-existing Behçet's disease. The use of etanercept may be considered for Behçet's patients with uveitis who are intolerant to infliximab and adalimumab, although the quality of evidence is relatively lower. These recommendations are based on meta-analyses and comparative studies that have demonstrated the efficacy of anti-TNF agents, particularly infliximab, in managing the various manifestations of Behçet's disease. These studies have reported response rates exceeding 90% for patients with disease inadequately controlled by traditional immunomodulatory treatments. Infliximab has shown superior outcomes compared to conventional immunosuppressants, with significant reductions in disease exacerbations and improved visual acuity in patients with ocular involvement. [1]

According to 2018 European League Against Rheumatism (EULAR) guidelines for the management of Behcet’s syndrome, patients presenting with an initial or recurrent episode of acute sight-threatening uveitis should be treated with high-dose glucocorticoids, infliximab, or interferon-alpha. This recommendation is primarily based on observational data that observed a rapid response and improvement in visual acuity with infliximab. Choice of treatment is recommended to depend on patient factors such as risk of infections, tolerability, physician’s experience, and reimbursement policies. Monoclonal anti-TNF antibodies should also be considered in patients with refractory venous thrombosis or arterial involvement of Behcet’s syndrome. For patients with severe and/or refractory gastrointestinal involvement, monoclonal anti-TNF antibodies and/or thalidomide should be considered. Similarly, monoclonal anti-TNF antibodies should be considered in severe nervous system involvement as first-line or in refractory patients. [2]

A 2024 systematic review and meta-analysis evaluated the long-term efficacy, safety, and cumulative retention rate of anti-TNF therapy in patients with Behçet's uveitis (BU). This meta-analysis included data from 12 clinical studies with a total of 1,156 patients diagnosed with BU, nine of which were retrospective and three prospective. Anti-TNF therapy achieved a pooled remission rate of ocular inflammation of 85.0% (95% confidence interval [CI] 78.7% to 90.5%) and visual acuity improvement in 77.4% of cases (95% CI 57.5% to 92.5%). Moreover, glucocorticoid dose reduction was significant, with a pooled decrease of 11.08 mg (95% CI -13.34 mg to -8.83 mg). The cumulative drug retention rate was determined to be 67.3% across studies, with subgroup analysis showing higher retention rates for adalimumab (70.3%) compared to infliximab (66.4%). Serious adverse events occurred in 5.8% of patients, with the most common being infusion or injection reactions (2.7%), tuberculosis (1.3%), and bacterial pneumonia (1.3%). Adalimumab exhibited a slightly higher ocular inflammation remission rate (89.3%) and lower incidence of severe infusion or injection reactions (2.2%) compared to infliximab (83.7% and 3.5%, respectively), which may reflect variations in immunogenicity and administration routes. Significant reductions in central macular thickness (CMT) were also observed, with a pooled decrease of 135.72 mcm in studies with follow-up of at least one year. Importantly, while the long-term safety profile of anti-TNF agents was favorable, the incidence of severe adverse events underscores the need for careful monitoring during treatment. [3]

A 2023 systematic review and meta-analysis evaluated the effectiveness of infliximab, a chimeric monoclonal anti-TNF antibody, for treating refractory central neuro-Behçet's disease. The analysis incorporated 21 studies conducted between 2000 and 2020, collectively involving 64 patients. Infliximab was administered in standard dosing regimens, commonly 5 mg/kg at weeks 0, 2, and 6, followed by maintenance dosing every 8 weeks. Across the pooled studies, 93.7% of patients exhibited a favorable response to therapy, defined as clinical improvement, stabilization or regression of radiographic findings, and biochemical normalization in cerebrospinal fluid (CSF) where available. Temporal trends suggested improved outcomes with earlier recognition and management of refractory central nervous system (CNS) involvement in Behçet's disease. Regression analysis confirmed that age, sex, or disease duration did not significantly confound treatment outcomes. Notably, infliximab displayed a steroid-sparing effect, with corticosteroid doses reduced or discontinued in several patients. Reported adverse events included mild hypersensitivity reactions, opportunistic infections, and headache, with few requiring discontinuation. These findings underscore infliximab as a highly effective treatment option for refractory neuro-Behçet's disease, although further validation in randomized controlled trials is warranted to address limitations in study design and reporting consistency. [4]

A 2022 meta-analysis and systematic review synthesized data from 13 single-arm cohort studies involving 739 patients with intestinal Behçet's disease to evaluate the efficacy and safety of anti-TNF agents, including infliximab and adalimumab. Study participants, predominantly from East Asia, had moderate to severe disease or were refractory to conventional therapies such as corticosteroids and immunomodulators. Pooled efficacy outcomes demonstrated clinical remission rates of 61%, 51%, 57%, and 38% at 3, 6, 12, and 24 months following treatment initiation, respectively. Endoscopic remission, a critical outcome indicating mucosal healing, was achieved in 66%, 82%, 65%, and 69% of patients at the same time points. Subgroup analysis showed comparable efficacy between infliximab and adalimumab, although moderate heterogeneity was noted across studies. Adverse drug reactions (ADRs) related to infliximab were reported in 22% of patients, with infusion reactions and infections being the most common events, occurring in 12% and 21% of cases, respectively. Adalimumab-associated ADRs were reported at rates of 30.7 and 28.7 events per 100 patient-years. Severe adverse events, including two cases of severe infection leading to hospitalization, were infrequent, and no deaths related to therapy were reported. These findings emphasize the promising role of anti-TNF agents as an effective therapeutic strategy for achieving remission and mucosal healing in patients with intestinal Behçet's disease, especially in those refractory to conventional treatments. However, limitations such as reliance on observational studies and heterogeneous follow-up protocols warrant further randomized controlled trials to establish definitive guidance. [5]

A 2022 single-center, retrospective study evaluated the efficacy of infliximab and adalimumab, two anti-TNF agents, in the management of refractory uveitis associated with Behçet's syndrome and sarcoidosis. The analysis included 131 patients: 68 in the infliximab group and 63 in the adalimumab group, treated between 2007 and 2019 at Tokyo Medical University Hospital. Infliximab was administered intravenously at a dose of 5 mg/kg at specified intervals, while adalimumab was delivered subcutaneously at a dose of 40 mg biweekly. The findings underscored significant visual acuity (VA) improvement (0.72 to 0.40 logMAR; p<0.001) in patients with Behçet's syndrome-related uveitis receiving infliximab, while adalimumab stabilized VA without significant change (0.42 to 0.36 logMAR; p= 0.11). Both agents significantly reduced CMT in eyes with uveitic macular edema (UME; 33 eyes in the infliximab group and 40 in the adalimumab group), with adalimumab showing greater efficacy in corticosteroid reduction for sarcoidosis patients. Infliximab achieved a complete discontinuation of corticosteroids in all treated patients, while adalimumab achieved a reduction or discontinuation in Behçet's syndrome and sarcoidosis to a lesser extent. Safety profiles revealed adverse events like infusion reactions in both groups, though adalimumab was associated with a higher overall frequency of adverse events (40.6% vs. 17.6%). The results suggest that while both agents are effective in controlling inflammation and UME in Behçet's syndrome, the pathogenesis of sarcoidosis may influence the comparative efficacy of adalimumab. [6]

A 2012 retrospective analysis evaluated the therapeutic impact of infliximab in seven patients presenting with severe vascular manifestations of Behçet's disease. The cohort consisted of five male and two female patients, with a mean age at diagnosis of 32.7 years. These patients exhibited significant vascular complications, including thoracoabdominal aortic dissection, retinal vasculitis, recurrent venous thromboses, and internal carotid artery dissection. Therapy with infliximab (3-5 mg/kg) was initiated as either a first-line intervention in three individuals or as an adjunct after failure of conventional immunosuppressive regimens in four patients. Existing immunosuppressive therapies included agents such as azathioprine, methotrexate, cyclosporine, and low-dose glucocorticoids. Treatment intervals with infliximab varied between four to eight weeks, tailored to disease severity and response. [7]

The analysis documented rapid suppression of inflammatory activity within 1-5 days of initiating infliximab therapy, evidenced by a decrease in mean C-reactive protein levels from 89 mg/L to 9 mg/L. Vascular stabilization was achieved, with grafts remaining patent and inflamed aortic walls demonstrating structural healing over six months. Notably, both patients with retinal vasculitis experienced significant visual improvement within two days of treatment initiation. Long-term outcomes included the cessation of infliximab in two patients after achieving sustained remission and the extension of infusion intervals in four additional cases. Concomitant immunosuppression regimens were well-tolerated, and no infliximab-related adverse effects were reported. These findings underscore the efficacy and safety of infliximab as a potential first-line therapeutic agent in the management of severe vascular complications associated with Behçet's disease. [7]

A 2019 prospective, large-scale, long-term postmarketing surveillance study (BRIGHT) evaluated the safety and efficacy of infliximab in 656 Japanese patients with refractory uveoretinitis in Behçet's disease (RUBD). The BRIGHT study encompassed a 24-month follow-up period and included patients who initiated infliximab therapy between January 2007 and January 2010. Infliximab was administered at a standard dose of 5 mg/kg body weight at weeks 0, 2, and 6, followed by maintenance infusions every eight weeks. The surveillance revealed that 32.32% of patients experienced ADRs, with infections being the most frequently reported (11.89%). Serious ADRs, such as tuberculosis and opportunistic infections, occurred in 6.10% of patients, particularly in those with comorbid respiratory or allergic conditions or concomitant glucocorticoid use. Efficacy findings demonstrated a sustained response, with 80.7% of participants achieving improvement or slight improvement by physician global assessment (PGA) at 24 months. Moreover, the median frequency of ocular attacks per six-month period dropped significantly from 2.0 at baseline to 0.0, while corrected VA was maintained throughout the study period. Additionally, the use of adjunctive therapies, including glucocorticoids and cyclosporine, decreased over time. These results underscore the tolerability and long-term efficacy of infliximab as a viable treatment option for RUBD in real-world clinical settings. [8]

A 2020 retrospective analysis examined the long-term efficacy and safety of infliximab monotherapy compared with combination therapies, including colchicine or corticosteroids, in patients with refractory uveitis due to Behçet's syndrome. The study evaluated 50 eyes of 27 patients, with detailed subgroup analyses involving 30 eyes treated with infliximab monotherapy and 20 eyes treated with combination therapies. The retention rate of infliximab exceeded 70% after 10 years, with no significant differences observed between monotherapy and combination approaches (p= 0.86). The frequency of ocular inflammatory attacks decreased from 4.2 episodes per year before treatment to 0.1 episodes per year by the sixth year, with no relapses observed after seven years of treatment. Best-corrected VA showed sustained improvement from a mean of 0.38 at baseline to 0.07 at the 10-year mark (p<0.001), indicating the treatment's long-term impact on preserving vision. The 10-year corticosteroid-sparing effect was substantial, with a mean daily dose reduction of 77.5% (from 11.1 mg to 2.5 mg). Adverse events, including infusion reactions and serious infections, were reported predominantly during the first five years of treatment; no adverse events occurred after the sixth year. Notably, the overall safety profile appeared consistent across both treatment modalities. The findings underscore that infliximab monotherapy is not inferior to combination therapy in terms of either efficacy or safety outcomes over a decade. These results provide robust evidence supporting infliximab as a critical option for the long-term management of refractory uveitis in Behçet's syndrome. [9]

A 2023 retrospective cohort analysis assessed the efficacy and safety of infliximab in vascular involvement among 127 patients with Behçet's syndrome over an 18-year span. Patients included in the cohort had various types of vascular involvement, including pulmonary artery thrombosis or aneurysms, non-pulmonary arterial lesions, extensive venous thrombosis, and venous ulcers. Nearly 87% of patients received infliximab for induction of remission, most of whom had been refractory to conventional immunosuppressive therapies such as azathioprine, cyclophosphamide, or mycophenolate mofetil. The remission rates at six and twelve months were assessed based on the absence of new or worsening vascular symptoms, CRP levels <10 mg/L, and stability or improvement of primary vascular lesions on imaging. [10]

The findings demonstrated remission rates of 73% at six months and 63% at twelve months across the cohort. Pulmonary artery involvement, accounting for 29% of cases, showed the most favorable response, with remission rates of 84% and 68% at six and twelve months, respectively, followed by venous thrombosis with rates of 82% and 70%. Conversely, patients with venous ulcers exhibited minimal improvement, with only 8% achieving remission at six months. During the mean follow-up period of 37 months, 50% of patients remained on infliximab, while adverse events, such as infusion reactions and serious infections, led to discontinuation in 11%. The relapse rate among those who achieved remission was remarkably low at 17%, highlighting infliximab's potential superiority for long-term maintenance in refractory vascular Behçet's syndrome, particularly in cases of pulmonary artery or severe venous involvement. [10]

References:

[1] Levy-Clarke G, Jabs DA, Read RW, Rosenbaum JT, Vitale A, Van Gelder RN. Expert panel recommendations for the use of anti-tumor necrosis factor biologic agents in patients with ocular inflammatory disorders. Ophthalmology. 2014;121(3):785-96.e3. doi:10.1016/j.ophtha.2013.09.048
[2] Hatemi G, Christensen R, Bang D, et al. 2018 update of the EULAR recommendations for the management of Behçet's syndrome. Ann Rheum Dis. 2018;77(6):808-818. doi:10.1136/annrheumdis-2018-213225
[3] Guan X, Zhao Z, Xin M, Xia G, Yang Q, Fu M. Long-term efficacy, safety, and cumulative retention rate of antitumor necrosis factor-alpha treatment for patients with Behcet's uveitis: A systematic review and meta-analysis. Int J Rheum Dis. 2024;27(2):e15096. doi:10.1111/1756-185X.15096
[4] Mohammed RHA, Woldeamanuel YW. The effectiveness of the anti-tumor necrosis factor therapy infliximab in neuro-Behçet's disease: a systematic review and meta-analysis. J Int Med Res. 2023;51(5):3000605231169895. doi:10.1177/03000605231169895
[5] Zhang M, Liu J, Liu T, et al. The efficacy and safety of anti-tumor necrosis factor agents in the treatment of intestinal Behcet's disease, a systematic review and meta-analysis. J Gastroenterol Hepatol. 2022;37(4):608-619. doi:10.1111/jgh.15754
[6] Kunimi K, Usui Y, Asakage M, et al. Anti-TNF-α Therapy for Refractory Uveitis Associated with Behçet's Syndrome and Sarcoidosis: A Single Center Study of 131 Patients. Ocul Immunol Inflamm. 2022;30(1):223-230. doi:10.1080/09273948.2020.1791346
[7] Adler S, Baumgartner I, Villiger PM. Behçet's disease: successful treatment with infliximab in 7 patients with severe vascular manifestations. A retrospective analysis. Arthritis Care Res (Hoboken). 2012;64(4):607-611. doi:10.1002/acr.21557
[8] Ohno S, Umebayashi I, Matsukawa M, Goto T, Yano T. Safety and efficacy of infliximab in the treatment of refractory uveoretinitis in Behçet's disease: a large-scale, long-term postmarketing surveillance in Japan. Arthritis Res Ther. 2019;21(1):2. Published 2019 Jan 5. doi:10.1186/s13075-018-1793-7
[9] Horiguchi N, Kamoi K, Horie S, et al. A 10-year follow-up of infliximab monotherapy for refractory uveitis in Behçet's syndrome. Sci Rep. 2020;10(1):22227. Published 2020 Dec 17. doi:10.1038/s41598-020-78718-z
[10] Hatemi G, Tukek NB, Esatoglu SN, et al. Infliximab for vascular involvement in Behçet's syndrome. Clin Immunol. 2023;253:109682. doi:10.1016/j.clim.2023.109682

Literature Review

A search of the published medical literature revealed 4 studies investigating the researchable question:

Are there any peer reviewed articles to support use of infliximab for Bechet's disease?

Level of evidence

A - Multiple high-quality studies with consistent results  Read more→



Please see Tables 1-4 for your response.


 

Infliximab versus Cyclophosphamide for Severe Behcet’s Syndrome

Design

Phase 2, Bayesian, multicenter randomized controlled trial

N= 52

Objective

To evaluate whether infliximab improved complete response rates at 22 weeks in patients with severe Behcet’s syndrome compared with cyclophosphamide

Study Groups

Cyclophosphamide (n= 25)

Infliximab (n= 27)

Inclusion Criteria

Aged 12 years or older; met International Study Group’s criteria for Behcet’s syndrome; major vascular or CNS involvement; active disease at the time of enrollment

Exclusion Criteria

Evidence of active tuberculosis, HIV or active hepatitis B virus infection, pregnancy or lactation, taking an oral daily dose of glucocorticoid of more than 20 mg prednisone equivalent for more than 6 weeks continuously prior to incision visit or taking more than 3000 mg methylprednisolone 4 weeks prior to inclusion visit, severe renal impairment or pre-existing hemorrhagic cystitis or liver insufficiency, heart failure stage 3 or 4, history of malignancy within 5 years other than carcinoma in situ of the cervix or excised basal cell or squamous cell carcinoma of the skin, history of multiple sclerosis and/or demyelinating disorder

Methods

Patients were randomized 1:1 to infliximab (5 mg/kg intravenously at weeks 0, 2, 6, 12 and 18) or cyclophosphamide (0.7 g/m2 intravenously at weeks 0, 4, 8, 12, 16, and 20; maximum dose 1.2 g/infusion). All patients received an oral prednisone equivalent 1 mg/kg/day (up to 80 mg/day), as well as premedication (acetaminophen and dexchlorpheniramine for infliximab; ondansetron and uromitexan for cyclophosphamide). Complete response was defined as clinical, biological, and radiological remission with a daily prednisone dose ≤0.1 mg/kg.

Duration

May 2018 to April 2021

Treatment: 22 weeks

Outcome Measures

Primary: Complete response at week 22

Secondary: Vascular complete response, CNS complete response, relapse

Baseline Characteristics

 

Cyclophosphamide (n= 25)

Infliximab (n= 27)

 

Age, years (IQR)

40.9 (34.7 to 48.2) 38.1 (29.3 to 46.0)  

Female

10 (40%) 12 (44%)  

Behcet’s syndrome assessment scores (IQR)

Behcet’s Disease Current Activity Form index

Physician’s Global Assessment

 

2 (2 to 3)

70 (50 to 80)

 

3 (1 to 4)

60 (40.2 to 77.5)

 

Previous treatments

Glucocorticoids

Colchicine

Synthetic immunosuppressants

 

10 (40%)

4 (16%)

4 (16%)

 

14 (52%)

5 (18%)

4 (15%)

 

Abbreviations: IQR= interquartile range

Results

Endpoint

Cyclophosphamide (n= 25)

Infliximab (n= 27)

Estimated difference (95% CrI)

Overall complete response

Vascular complete response

CNS complete response

14/25 (56%)

10/18 (56%)

4/7 (57%)

22/27 (81%)

17/19 (94%)

5/8 (71%)

29.8 (6.6 to 51.7)

35.2 (9.7 to 59.2)

11.4 (-31.9 to 52.3)

Relapse

4 (16%) 1 (4%) -12.3 (-29.6 to 4.8)

Any mild-to-moderate AE

Mild-to-moderate infection, n

16 (64%)

29

8 (30%)

13

-

Any severe AE

3 (12%) 4 (15%) -

Abbreviations: AE= adverse event; CrI= credible interval

Serious AEs that required hospitalization were mainly due to infections; no differences between groups.

Adverse Events

See Results; no deaths were recorded. Four cyclophosphamide vs two infliximab patients discontinued treatment regimens.

Study Author Conclusions

Among patients with severe Behçet’s syndrome, induction therapy with infliximab had a superior complete response rate at 22 weeks and fewer adverse events than induction with cyclophosphamide.

InpharmD Researcher Critique

The study was open-label, which may introduce bias in the results, and use of a small sample size in both infliximab and the comparator arm increases possibility of type 2 error. Clinical manifestations of Behcet's syndrome among patients also varied.



References:

Saadoun D, Maalouf G, Vieira M, et al. Infliximab versus Cyclophosphamide for Severe Behçet's Syndrome. NEJM Evid. 2024;3(11):EVIDoa2300354. doi:10.1056/EVIDoa2300354

 

Efficacy and safety of infliximab or adalimumab in severe mucocutaneous Behçet’s syndrome refractory to traditional immunosuppressants: a 6-month, multicentre, randomised controlled, prospective, parallel group, single-blind trial

Design

Phase 3, multi-center, prospective, randomised, active-controlled, parallel-group study

N= 41

Objective

To evaluate the efficacy and safety of infliximab (IFX) or adalimumab (ADA) in patients with Behcet’s syndrome (BS) with mucocutaneous manifestations, refractory to the standard of care (SoC) with azathioprine (AZA) or cyclosporine A (CsA)

Study Groups

ADA (n= 18)

IFX (n= 22)

Inclusion Criteria

Age between 18 and 65 years old; diagnosed with BS according to the 1990 International Study Group criteria; presenting active mucocutaneous manifestations which occurred while on treatment with either AZA or CsA for ≥3 months

Exclusion Criteria

"End-stage" BS or other severe BS manifestations; infection at screening or frequent acute or chronic infections within 3 months; prior history of anti-TNF alpha agents or other monoclonal antibody treatments

Methods

Patients were randomized 1:1 to either IFX (5 mg/kg intravenously [IV] administered at weeks 0, 2 and 6 and then q6-8 weeks) or ADA (40 mg subcutaneously [SC] q2 weeks). Prior and concomitant therapies allowed were AZA (maximum 2 mg/kg/day) or CsA (maximum 5 mg/kg/day for month 1, then tapered 0.5 mg/kg q2 weeks until maintenance at 3 mg/kg/day). Medium-to-high doses of glucocorticoids were allowed on a taper schedule (6-methylprednisolone via 0.5 g for <50 kg or 1 g for >50 kg IV x3 days; then tapered to 40 mg/day intramuscularly [IM] x3 days, then to 20 mg/day orally [PO] x3 days, then to 4 mg/weekly until stop). 

Duration

January 2018 to January 2020

Treatment: 24 weeks

Follow-up: 12 weeks

Outcome Measures

Primary: Time to response

Secondary: Proportion of BS subjects who had a relapse

Baseline Characteristics

 

ADA (n= 18)

IFX (n= 22)

 

Age, years

47.7 ± 10.20 45.73 ± 11.88  

Female

5 (27.78%) 7 (31.82%)  

White

16 (88.89%) 18 (81.82%)  

Results

Endpoint

ADA (n= 18)

IFX (n= 22)

p-value

Time to response

Median time to response, days

17 (94%)

42

14 (64%)

152

0.023

0.001

Ocular, mucocutaneous or neurological manifestations

Active manifestations at day 0

5 (27.8%)

4 (22.2%)

9 (40.9%)

5 (22.7%)

0.510

1.000

Disease flare

2 (11.1%)

7 (31.8%)

0.149

Abbreviations: CI= confidence interval; HR= hazard ratio

There was no difference between groups regarding isolated or associated mucocutaneous involvement. Additionally, no differences were observed between arms for time to first relapse (p= 0.156). however, when adjusting for variables significantly different between groups (i.e., smoking habits and glucocorticoid use), higher risk of relapse was observed in IFX (adjusted HR 7.57; 95% CI 1.14 to 50.19; p= 0.036).

Significant differences were observed between the two groups, with the risk of non-response being higher with IFX use (adjusted HR 2.5; 95% CI 1.22 to 5.26; p= 0.013).

Adverse Events

N/A

Study Author Conclusions

The overall results of this study confirm the effectiveness of both IFX and ADA in achieving remission in patients with BS affected by mucocutaneous involvement.

InpharmD Researcher Critique

Due to COVID-19, the study duration was extended beyond 36 months for data analysis. Limitations include use of a small sample size, as well as the use of standard of care therapy prior to selection of infliximab or adalimumab.



References:

Talarico R, Italiano N, Emmi G, et al. Efficacy and safety of infliximab or adalimumab in severe mucocutaneous Behçet's syndrome refractory to traditional immunosuppressants: a 6-month, multicentre, randomised controlled, prospective, parallel group, single-blind trial. Ann Rheum Dis. Published online October 17, 2024. doi:10.1136/ard-2024-226113

 

Infliximab vs interferon-α in the treatment of Behcet’s syndrome: clinical data from the BIO-BEHCET’S randomized controlled trial

Design

Randomized, controlled, head-to-head clinical trial

N= 79

Objective

To compare infliximab and interferon-α, the two most frequently used biologics in active refractory Behcet’s, after first-line treatment failure

Study Groups

Intention-to-treat analysis:

Infliximab (n = 37)

Interferon-α2a (n = 37)

Inclusion Criteria

Aged over 18 years, have not previously received a biologic agent, diagnosed to have active Behçet’s Syndrome by International Study Group for Behcet’s Disease (ISG) criteria, refractory disease as defined by the UK Centres of Excellence criteria (failure to respond to steroid and/or immunomodulatory therapy with significant or major organ-threatening disease) and therefore qualified for biologic therapy with either infliximab or interferon-α2a

Exclusion Criteria

Contraindication to either infliximab or interferon-α2a, severe heart failure that would contraindicate the use of infliximab, diagnosis of multiple sclerosis, evidence of infection with HIV, active tuberculosis

Methods

Eligible patients were randomized to receive to receive either infliximab or interferon-α2a. Infliximab was administered at 5 mg/kg infusions at weeks 0, 2, and 6, then every 8 weeks (or every 6 weeks for active ocular or vascular disease). Patients on infliximab also received concurrent immunomodulators (methotrexate or azathioprine) to reduce the risk of anti-drug antibodies. IFN-α2a was given via subcutaneous injection with a standardized dose reduction protocol. In the absence of adverse events, immunomodulators were tapered every 4 weeks per routine clinical practice.

Duration

June 2016 to February 2020

Outcome Measures

Primary: Behcet’s Disease Activity Index (BDAI) at 12 weeks

Secondary: BDAI at 24 weeks and significant improvement in individual organ systems, including ocular symptoms, oral and genital ulcers, arthritis pain, quality of life, disease activity and steroid use

Baseline Characteristics

 

Infliximab (n = 37)

Interferon-α2a (n = 37)

 

Age, years

38.9  39.3  

Female

65% 70%   

White

92% 81%   

Steroid use

49% 51%  

Primary ocular

27% 19%  

Primary oral 

32% 41%  

Primary genital

30% 38%  

Primary musculoskeletal

27% 27%  

Malignancy

3% 0  

Heart failure

0 0  

Cutaneous activity

38% 38%  

Results

Change in BDAI between baseline and 12 weeks (data presented as figures):

  • Mean difference: 0.13; 80% credible interval: –0.19 to 0.46
  • Median BDAI change for infliximab: –1.5 (interquartile range [IQR] –4 to 0)

BDAI at 24 weeks:

  • Regression analysis of 24-week data against baseline BDAI showed a significant average score reduction of 30% (p<0.001).
  • Median BDAI change for IFN-α2a: –3 (IQR: –5.25 to –1).
  • Regression against baseline indicated a significant 24-week average reduction of 39% (p<0.001).

Improvement in organ systems was evaluated at 12 and 24 weeks:

  • No significant differences between groups were observed for ulcers or arthritis pain.
  • Both conditions showed clinically significant reductions within each group over time.

Adverse Events

Eight serious adverse events (SAEs) were reported among five patients:
  • Three infliximab patients reported six SAEs, including four from one patient (two hypertension events, bacterial UTI, and uncontrolled blood pressure).
  • Two IFN-α2a patients reported two SAEs.

No suspected drug interactions or unexpected serious adverse reactions (SUSARS) occurred.


More IFN-α2a patients (n=11) switched treatments compared to infliximab (n=3; p=0.010). Infliximab switches: two due to clinician decision (not AE) and one for inadequate response. IFN-α2a switches: four for unacceptable AEs, four for inadequate response, and four unrecorded reasons.

Study Author Conclusions

In this first reported, high-quality, head-to-head trial of two biologics in Behcet’s, both infliximab and IFN-α2a showed comparable short-term clinical efficacy and safety in refractory active disease of all subtypes. 

InpharmD Researcher Critique

The study faced challenges, including slow recruitment, limited follow-up, and early cessation of IFN-α2a production, which restricted long-term evaluation and access to treatment. Concerns about adverse effects deterred participation, though no significant mental health impacts were observed.  



References:

Moots RJ, Fortune F, Jackson R, et al. Infliximab versus interferon-alpha in the treatment of Behçet's Syndrome: Clinical data from the BIO-BEHÇET'S randomised controlled trial. Rheumatology (Oxford). Published online October 21, 2024. doi:10.1093/rheumatology/keae585

 

Infliximab Therapy for Intestinal, Neurological, and Vascular Involvement in Behcet disease: Efficacy, Safety, and Pharmacokinetics in a Multicenter, Prospective, Open-label, Single-arm Phase 3 Study

Design

Multicenter, prospective, open-label, single-arm phase 3 study

N= 18 participants

Objective

To evaluate the efficacy, safety, and pharmacokinetics of infliximab in Behcet disease (BD) patients with serious complications

Study Groups

Intestival BD (n=11)

Neurological BD (n= 3)

Vascular BD (n= 4)

Inclusion Criteria

Patients aged 16-75 with complete or incomplete BD with intestinal, neurological, or vascular involvement, and insufficient response or intolerance to conventional therapy

Exclusion Criteria

Conditions not differentiated from other diseases, history of certain treatments, serious infections, active hepatitis, HIV, heart failure, demyelinating diseases, or recent malignancy

Methods

Infliximab 5mg/kg was administered at weeks 0, 2, 6, and every 8 weeks until week 46; with the dose increased to 10mg/kg if an inadequate response after week 30.

Duration

54 weeks

Outcome Measures

Primary: Percentage of complete responders at week 30

Secondary: Complete responders at weeks 14 and 54, VAS score, SF-36 score, oral steroid dosage

Baseline Characteristics

 

Intestinal BD (n= 11)

Neurological BD (n= 3)

Vascular BD (n= 4) 

Age, years

35.0 ± 13.4 38.3 ± 8.5 44.0 ± 2.9

Male

45% 67% 75%

Weight, kg

63.0 ± 16.9 64.3 ± 11.0 83.8 ± 18.5

Time since diagnosis of BD, months

76.1 ± 78.0 64.0 ± 61.9 153.5 ± 85.5 

Results

Endpoint

Intestinal BD (n= 11)

Neurological BD (n= 3)

Vascular BD (n= 4) 

Complete responders

Week 14

Week 30 

Week 54

Final point of 5mg/kg therapy

 

55%

55%

60%

64% 

 

33%

33%

50%

33% 

 

100%

100%

100%

100% 

Adverse Events

Adverse events were reported in 94% of patients and infections were the most common (61% overall, 64% intestinal BD, 33% neurological BD, and 75% in vascular BD). No serious infections or infusion reactions were reported.

Study Author Conclusions

Infliximab is effective and well-tolerated for treating intestinal, neurological, and vascular BD in patients with poor response or intolerance to conventional therapy.

InpharmD Researcher Critique

Study limitations include a small sample size, lack of a control group, and limited generalizability due to single-arm design.



References:

Hibi T, Hirohata S, Kikuchi H, et al. Infliximab therapy for intestinal, neurological, and vascular involvement in Behcet disease: Efficacy, safety, and pharmacokinetics in a multicenter, prospective, open-label, single-arm phase 3 study [published correction appears in Medicine (Baltimore). 2016 Aug 07;95(31):e5074. doi: 10.1097/01.md.0000490009.39850.74]. Medicine (Baltimore). 2016;95(24):e3863. doi:10.1097/MD.0000000000003863