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Observational Study
. 2025 Jul 15;15(1):25540.
doi: 10.1038/s41598-025-09802-5.

Long-term efficacy of therapeutic drug monitoring-guided optimization of ustekinumab maintenance therapy for Crohn's disease patients

Haixia Ren  1   2   3 , Juan Su  1   2   3 , Anning Yin  1   2   3 , Xinsheng Gao  4 , Wei Wang  5 , Jiao Li  1   2   3 , Jian Kang  1   2   3 , Jing Wang  1   2   3 , Qian Zhou  1   2   3 , Jing An  1   2   3 , Zhishun Tang  6 , Xinxin Wang  6 , Huipeng Wan  1   2   3 , Wei Chen  1   2   3 , Pengbo Wu  1   2   3 , Ping An  7   8   9
Affiliations
Observational Study

Long-term efficacy of therapeutic drug monitoring-guided optimization of ustekinumab maintenance therapy for Crohn's disease patients

Haixia Ren et al. Sci Rep. .

Abstract

Long-term remission in Crohn's disease (CD) remains challenging. While ustekinumab effectively induces remission, strategies to enhance its maintenance efficacy are urgently needed. This study evaluated therapeutic drug monitoring (TDM)-guided ustekinumab optimization for sustained CD management. A retrospective observational study was conducted involving 158 patients [TDM: n = 87, age(16 39), male = 64; Non-TDM: n = 71, age(16-40), male = 57] with moderate-to-severe CD who achieved clinical remission following ustekinumab therapy, between October 2020 and November 2024, sourced from three inflammatory bowel disease centers. All patients received 8 weekly ustekinumab maintenance treatment with or without therapeutic drug monitoring (TDM)-guided optimization. The clinical outcomes and disease relapse were evaluated at year 1 and 2. The non-TDM group had a slightly higher endoscopic response and mucosal healing rate at baseline, there were no statistically significant differences between two cohorts at baseline with respect to demographic and clinical characteristics. In this multicenter retrospective study of 158 CD patients in clinical remission, TDM-guided dosing (n = 87) significantly improved 1 year (83.9% vs. 70.4%, p = 0.042) and 2 year remission rates (71.3% vs. 46.5%, p = 0.002) compared to standard therapy (n = 71). Subgroup analyses confirmed benefits in endoscopic responders and mucosal healing cohorts. TDM patients exhibited higher ustekinumab trough levels (3.00 vs. 1.46 μg/mL at year 1, p < 0.001) and lower relapse rates (p = 0.003). Neither the TDM nor the non-TDM cohorts reported any severe adversative events. TDM-guided optimization of ustekinumab maintenance treatment is an efficacious and safe strategy for CD patients with ustekinumab induced clinical remission.

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Conflict of interest statement

Declarations. Competing interests: The authors declare no competing interests. Patient consent for publication: Obtained. Ethics approval: This study was approved by the ethics committee of Renmin Hospital of Wuhan University (Wuhan, China; WDRY2021-Y076).

Figures

Fig. 1
Fig. 1
Study design and flow chart. (A) Study design and cases illustration of ustekinumab maintenance treatment guided by TDM (TDM group) or not (non-TDM group). (B) Study flowchart.
Fig. 2
Fig. 2
Long-term clinical remission for patients in TDM and non-TDM cohorts. (A) clinical remission for patients achieved clinical remission at baseline. (B) Clinical remission for patients reached endoscopic response at baseline. (C) Clinical remission for patients with mucosal healing at baseline. TDM group, patients received optimized ustekinumab maintenance treatment guided by TDM; non-TDM group, patients received routinely subcutaneous ustekinumab maintenance treatment.
Fig. 3
Fig. 3
Long-term relapse for patients in TDM and non-TDM cohorts. (A) Non-relapse rate for patients achieved clinical remission at baseline. (B) Non-relapse rate for patients reached endoscopic response at baseline. (C) Non-relapse rate for patients with mucosal healing at baseline. TDM group, patients received optimized ustekinumab maintenance treatment guided by TDM; non-TDM group, patients received routinely subcutaneous ustekinumab maintenance treatment.
Fig. 4
Fig. 4
Serum ustekinumab trough levels since ustekinumab maintenance treatment. TDM group, patients received optimized ustekinumab maintenance treatment guided by TDM; non-TDM group, patients received routinely subcutaneous ustekinumab maintenance treatment. (*, p < 0.05; **, p < 0.01; ***, p < 0.001).
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References

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