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Scaled-Up Preparation of an Intermediate of Upatinib, ACT051-3
Published on: April 7, 2023
Treatment of primary cutaneous amyloidosis with tofacitinib: preliminary observations from a single-arm clinical
Pan Zou1, Yuxin Du2, Yuchun Cao3
1Department of Dermatology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Background:
Primary cutaneous amyloidosis (PCA) lacks consensus-based therapeutic guidelines, with conventional modalities demonstrating suboptimal clinical responses.
Objectives:
To assess the efficacy and safety profile of oral tofacitinib, a Janus kinase 1/3 inhibitor, in PCA management.
Methods:
This single-centre, open-label, single-arm prospective interventional study enrolled 20 patients with PCA receiving tofacitinib 5 mg twice daily. Demographic parameters, disease characteristics [measured using the pruritus numeric rating scale (p-NRS), Dermatology Life Quality Index (DLQI) and Lesion Severity (LS) scores], and adverse events were systematically recorded at baseline and weeks 2, 6 and 10 post-treatment.
Results:
At week 10, significant reductions were observed across all endpoints: p-NRS from 6.9 (SD 2.9) to 0.4 (SD 0.6, P < 0.001); DLQI from 11.8 (SD 5.6) to 2.6 (SD 2.5, P < 0.001); and LS from 13.3 (SD 5.2) to 4.9 (SD 2.4, P < 0.001). Patients with lichen amyloidosis (8 of 15) and biphasic amyloidosis (3 of 3), but neither of the 2 patients with macular amyloidosis, achieved the primary efficacy threshold. Two treatment-emergent adverse events occurred; these were transient hepatobiliary enzyme elevations resolving with hepatoprotectants and one patient with a self-limiting upper respiratory infection.
Conclusions:
Tofacitinib demonstrates rapid antipruritic effects and sustained lesion clearance in PCA, with favourable tolerability. Study limitations, including the small sample size (n = 20), 10-week duration and uncontrolled design, which may limit generalizability, warrant phase III trials for validation.
Insights
Oral tofacitinib effectively treats primary cutaneous amyloidosis (PCA), rapidly reducing itch and clearing lesions. This JAK inhibitor shows promise for PCA management, though larger trials are needed.
Area of Science:
- Dermatology
- Pharmacology
- Immunology
Background:
- Primary cutaneous amyloidosis (PCA) lacks established treatment guidelines.
- Current therapies offer limited efficacy for PCA patients.
Purpose of the Study:
- To evaluate the efficacy and safety of oral tofacitinib in managing PCA.
- To assess tofacitinib's impact on pruritus, quality of life, and lesion severity.
Main Methods:
- A single-center, open-label study involving 20 PCA patients.
- Patients received oral tofacitinib 5 mg twice daily for 10 weeks.
- Outcomes measured included pruritus, DLQI, and lesion severity scores.
Main Results:
- Significant reductions in pruritus (p < 0.001), DLQI (p < 0.001), and lesion severity (p < 0.001) were observed.
- 55% of patients met the primary efficacy endpoint of ≥60% improvement in lesion severity.
- Adverse events were mild, including transient enzyme elevations and a respiratory infection.
Conclusions:
- Tofacitinib demonstrates rapid and sustained efficacy in PCA, significantly improving symptoms and appearance.
- The drug exhibits a favorable safety profile in this patient cohort.
- Further validation through phase III trials is recommended due to study limitations.
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