FormalPara Key Summary Points

Why carry out this study?

Data on the real-world effectiveness and tolerance of baricitinib for treatment of alopecia areata are limited.

This retrospective observational study assessed the effectiveness and tolerance of baricitinib for the treatment of alopecia areata in a tertiary university hospital.

What was learned from this study?

This real-life study assessed the effectiveness and tolerance of baricitinib in alopecia areata in a series of 19 adults with a median follow-up of 13 months.

Results were better than in clinical trials; patients with patchy alopecia areata had a better hair regrowth prognosis than those with universalis.

Safety profile was reassuring; induced lipid changes were the most frequent adverse events.

Introduction

Alopecia areata (AA) is an autoimmune disease, affecting approximately 2% of the general population [1, 2]. Although AA is not life-threatening, psychological comorbidities are common and result in major impacts on patients’ quality of life [3]. Response to conventional treatments is extremely variable and disappointing in most cases, with a high risk of relapse [1, 2, 4]. Recent clinical trials and real-life studies have demonstrated efficacy and safety of baricitinib (an oral Janus kinase 1 and 2 inhibitor) in AA, and it received European marketing authorization for the treatment of AA in June 2022 [5,6,7,8,9]. This study aimed to evaluate the effectiveness on hair regrowth and the tolerance of baricitinib in AA.

Methods

We retrospectively collected data from 1 January 2021 to 31 July 2023 from a monocentric cohort of adult patients with severe AA, with a Severity of Alopecia Tool (SALT) score of 50 or higher [range, 0 (no scalp hair loss) to 100 (complete scalp hair loss)], treated with baricitinib 4 mg orally daily. The primary outcome was evaluated by the percentage of patients who achieved a SALT score of ≤ 20 at the end of the follow-up. All adverse events were reported during the study. This study and data collection were conducted with the approval of the hospital and faculty institutional review board (Commission d’Ethique Biomédicale Hospitalo-Facultaire) of Université catholique de Louvain (UCLouvain), Belgium. This study was performed in accordance with the Helsinki Declaration of 1964 and its later amendments. The patients in this manuscript have given written informed consent to participate and for publication of their case details.

Results

Of the 19 patients included, 13 were female and the median age ± interquartile range (IQR) was 35 ± 12 years. The duration of the disease varied from 1 month to 63 years (median 13 years). Patients’ demographics, clinical characteristics, and treatment history are listed in Table 1. The median ± IQR follow-up duration of treatment with baricitinib was 13 ± 16.2 months (median follow-up duration was 23, 11.7, and 12 months for patchy, totalis, and universalis AA, respectively). Hair regrowth was observed in 17/19 (89.5%) patients between 4 and 16 weeks after initiation of treatment (median ± IQR of 6 weeks ± 2). At the end of the follow-up, 14/19 patients [73.7%–11/11 (100%) with patchy AA, 1/2 (50%) with totalis AA, 2/6 (33.3%) with universalis AA] reached the primary outcome (SALT score ≤ 20) (Fig. 1). Seven patients [36.8%–6/11 (54.5%) with patchy AA, 1/2 (50%) with totalis AA, 0/6 (0.0%) with universalis AA] showed complete hair regrowth (SALT score = 0). The median ± IQR treatment time required to obtain complete hair regrowth was 8.5 ± 10 months. Two patients with no signs of hair regrowth presented with long-lasting universalis AA. Baricitinib was discontinued in three patients of whom two relapsed (patients 5 and 13 relapsed 4 months and 1 month after discontinuation, respectively) and one (patient 6) maintained complete hair regrowth 12 months after discontinuation. Dosage was decreased to 2 mg daily in patient 11 after achieving complete hair regrowth; one small patch recurred 4 months after dose reduction (but SALT score remained ≤ 20).

Table 1 Patients’ demographics and clinical characteristics
Full size table
Fig. 1
figure 1

Clinical evolution of alopecia areata of patient 1 with baricitinib. At 1.5 months (A), at 3.5 months (B), at 5 months (C), at 7 months (D), at 15 months (E), and at 20 months (F)

Full size image

Adverse events observed were mild and included acne [n = 1 (5.3%)], headaches [n = 3 (15.8%)], altered lipid status [n = 5 (26.3%)], transaminitis [n = 2 (10.5%)] and increased creatine phosphokinase [n = 1 (5.3%)]. No major adverse events were reported.

Discussion

Although the present real-life study is limited by the sample size and the retrospective design, the results were better than those reported from clinical trials [5, 6]. In two phase III trials (BRAVE-AA1 and BRAVE-AA2), 40.9% of patients achieved a SALT score of ≤ 20 at 52 weeks, compared with the 73.7% in the present real-life study [6]. Our observation that all patients with patchy AA reached a SALT score of 20 or less is encouraging for this patient subgroup. However, this study also confirmed a poorer response for universalis AA, as well as the risk of relapse after treatment discontinuation or dose reduction [4]. In this young population, high dose of baricitinib was well tolerated. The reassuring safety profile is consistent with the recent data about the safety of baricitinib in different indications, including AA [10].

A major problem of the accessibility of baricitinib for the treatment of AA (particularly in terms of reimbursement) remains in most European countries. Presently, because AA remains a largely underrecognized condition, particularly in terms of quality of life and psychological impact, it is still not considered as a priority by the health authorities.

Conclusion

Baricitinib demonstrates good effectiveness for the treatment of AA, particularly for the patchy phenotype, and could substantially improve the management of patients with AA. However, a poorer regrowth prognosis observed for patients with totalis or universalis AA, as well as the risk of relapse after treatment discontinuation, need to be considered.