Efficacy and Safety of Tofacitinib in Paediatric Alopecia Aerata - A Prospective Observational Study

ABSTRACT

Background:

Alopecia areata (AA) is an autoimmune condition marked by non-scarring hair loss.

Materials and Method:

In this prospective observational study, we enrolled 50 pediatric patients with moderate-to-severe alopecia areata. Participants received oral tofacitinib for 24 weeks. Clinical efficacy was assessed using the Severity of Alopecia Tool (SALT) score. Secondary outcomes included time to hair regrowth, patient/parent satisfaction, and Paediatric Quality of Life Inventory (PedsQL) scores.

Result:

Forty-five participants completed the 24-week study. A significant decrease in SALT score from baseline (mean 66.2 ± 8.1) to week 24 (mean 25.7 ± 9.2) was observed (P < 0.001). Complete or near-complete hair regrowth was achieved in 42.2% of patients, with partial regrowth in 37.8%.

Conclusion:

Tofacitinib demonstrated a favorable risk–benefit profile for treating moderate-to-severe pediatric alopecia areata over 24 weeks. These findings support the potential of JAK inhibitors as a therapeutic option in children with recalcitrant disease, although long-term safety and relapse rates warrant further investigation.

INTRODUCTION

Alopecia areata (AA) is a T-cell mediated autoimmune disorder that typically presents with patchy, non-scarring hair loss on the scalp and, occasionally, other hair-bearing areas of the body.[1] Pediatric alopecia areata is particularly distressing due to the profound impact on a child’s self-esteem, quality of life, and psychosocial development.[2] Management of alopecia areata in pediatric populations typically involves topical corticosteroids, topical immunotherapy such as diphencyprone, and intralesional corticosteroids for localized disease.[3] Systemic therapy, including corticosteroids and immunosuppressives such as methotrexate, is less commonly used and is limited by safety concerns, side effects, and variable efficacy.[4] More recently, Janus kinase (JAK) inhibitors have been identified as a promising class of therapy for moderate-to-severe alopecia areata in adults, with tofacitinib and ruxolitinib showing encouraging clinical results.[5]

This research aims to assess the effectiveness and safety of tofacitinib in children with moderate-to-severe alopecia areata.

MATERIALS AND METHODS

This was a prospective observational study conducted at a tertiary care dermatology center between January 2022 and June 2023. Prior to study approval from ethical approval obtained from concerned authority and informed consent from participants. Eligible participants were children aged 6–16 years diagnosed with moderate-to-severe alopecia areata (SALT score ≥50), who had an inadequate response or intolerance to at least one conventional therapy (topical or intralesional corticosteroids). After taking into account the inclusion and exclusion criteria, the study was completed. The hospital’s Institutional Review Board (IRB) gave its approval to the study. All parents provided written informed permission.

Intervention

Tofacitinib was administered orally at a weight-based dose:

• For participants weighing <30 kg, a tofacitinib dose of 2 mg twice daily.

• For participants weighing ≥30 kg, a tofacitinib dose of 5 mg twice daily.

All patients continued to receive standard supportive care, including scalp hygiene measures and nutritional support. Concomitant use of other immunomodulatory agents was not permitted.

Outcome measures

Primary efficacy was assessed using the Severity of Alopecia Tool (SALT) score at baseline and at weeks 12 and 24. A reduction in SALT scores ≥ 50% from baseline was defined as a clinically meaningful improvement. Complete or near-complete hair regrowth was noted if the SALT score was ≤ 5 at week 24. Secondary endpoints included time to initial hair regrowth, percentage of patients achieving ≥ 75% SALT improvement, patient/parent satisfaction on a 10-point Likert scale, and changes in the Paediatric Quality of Life Inventory (PedsQL) at baseline, week 12, and week 24.

Safety assessments

Safety evaluations comprised recording of all adverse events, physical examinations, and laboratory investigations, including complete blood count, liver and renal function tests, and lipid profile at baseline, week 12, and week 24. Any major or unexpected adverse events were reported to the IRB within 24 hours.

The obtained data were analyzed using SPSS version 26.0 (IBM Corp., Armonk, NY, USA) using Paired t-tests or Wilcoxon signed-rank tests used for within-group comparisons at P value < 0.05.

RESULTS

Efficacy outcomes

Table 1 shows the progression of hair regrowth and percentage improvements in SALT scores over the study period. (P < 0.001)

Table 1.

Change in SALT scores and response rates

Time Point	Mean SALT Score±SD	≥50% Reduction (n, %)	≥75% Reduction (n, %)	SALT ≤5 (n, %)
Baseline	66.2±8.1	–	–	–
Week 12	41.2±10.3	25 (55.5%)	15 (33.3%)	0
Week 24	25.7±9.2	38 (84.4%)	31 (68.9%)	19 (42.2%)

Table 2 shows the change in PedsQL domain scores. Figure 1 indicates the trends in Patient/Parent Satisfaction (Likert Scale) and PedsQL Total Scores from Baseline to Week 24. Figure 2 indicates the images before and after applying Tofacitinib to children. Safety and Tolerability: Most side effects were mild to moderate in intensity and did not require stopping the research or changing the dosage.

Table 2.

Changes in pediatric quality of life inventory (PedsQL) scores

Domain	Baseline (Mean±SD)	Week 24 (Mean±SD)	P
Physical	68.1±9.3	75.0±8.8	<0.001
Emotional	55.2±7.6	71.6±8.2	<0.001
Social	63.5±8.0	79.2±7.5	<0.001
School	61.0±8.7	72.8±8.4	<0.001
Total	62.5±8.4	77.8±7.9	<0.001

Figure 1.

Trends in patient/parent satisfaction (Likert scale) and PedsQL total scores from baseline to week 24

Figure 2.

Images before and after applying Tofacitinib to children

DISCUSSION

This is the first prospective observational study and provides evidence that facitinib can be both effective and safe for children with moderate to severe alopecia areata, extending findings from earlier studies in adults.[6] Most interestingly, the entire study population almost showed a complete or nearly complete regrowth; this observation fits with the small pediatric case series, though it has never been previously reported in such a large population.[7]

Restoration of hair has significant implications: not only is it cosmetic, but it also impacts self-esteem, social interaction, and school performance in children. This overall benefit underlines the importance of dealing with clinical and psychosocial components of management in the case of pediatric alopecia areata.[3]

Despite these promising findings, some limitations must be acknowledged. As an observational study without a control group, definitive conclusions about causality are less robust than those from randomized controlled trials. Furthermore, the relatively short follow-up period of 24 weeks does not address longer-term safety, the durability of hair regrowth, or relapse rates upon discontinuation. Future studies may involve randomized controlled designs with longer follow-up to determine the optimal dosage, maintenance approach, and risk-reduction strategy for this susceptible population.[8]

CONCLUSION

Tofacitinib demonstrated a favourable risk–benefit profile for treating moderate-to-severe paediatric alopecia areata over a 24-week period. These findings support the potential of JAK inhibitors as a therapeutic option in children with recalcitrant disease, although long-term safety and relapse rates warrant further investigation.

Declaration of patient consent

The authors certify that they have obtained all appropriate patient consent forms. In the form, the patient(s) has/have given his/her/their consent for his/her/their images and other clinical information to be reported in the journal. The patients understand that their names and initials will not be published and due efforts will be made to conceal their identity, but anonymity cannot be guaranteed.

Conflicts of interest

There are no conflicts of interest.

Funding Statement

Nil.