Telitacicept in refractory juvenile generalized myasthenia gravis: a case report and collective analysis.

BACKGROUND

Refractory or corticosteroid-dependent juvenile generalized myasthenia gravis (gMG) remains a clinical challenge, given the scarcity of available regimens and prominent corticosteroid-related adverse effects. Telitacicept, a dual inhibitor of B-cell activating factor (BAFF) and a proliferation-inducing ligand (APRIL), has demonstrated efficacy in adult gMG, whereas clinical data in pediatric patients remains limited.

OBJECTIVE

To evaluate the clinical response, tolerability, and potential corticosteroid-sparing benefit of telitacicept in juvenile refractory gMG.

METHODS

We report one new case of juvenile refractory gMG treated with telitacicept and review three previously reported pediatric gMG cases. Collective analysis was performed to evaluate key outcomes, including changes in Myasthenia Gravis Activities of Daily Living (MG-ADL) score, Quantitative Myasthenia Gravis (QMG) score, corticosteroid dose reduction, and adverse events.

RESULTS

In the index case, telitacicept reduced the MG-ADL score from 3 to 0 and the QMG score from 10 to 3 by week 12. By week 32, the MG-ADL score remained at 0, while the QMG score further decreased to 2, accompanied by a 41.6% reduction in corticosteroid dose. The patient developed mild injection-site erythema and pruritus on only one occasion, which resolved spontaneously. She also had a mild upper respiratory tract infection that resolved within one week following symptomatic treatment. Collective analysis of the four included patients (age range: 7-17 years) showed that three cases (Cases 1, 2, and 4) achieved marked improvement (≥3-point reduction in the MG-ADL score) at 3 months. By 5-6 months, all patients presented sustained clinical improvement with a ≥2-point reduction in the MG-ADL score. No severe adverse events were reported.

CONCLUSION

Telitacicept may be of clinical value and well-tolerated, and may exert a corticosteroid-sparing benefit in juvenile refractory gMG, providing preliminary real-world clinical observations for this understudied population. Further large-scale prospective studies are warranted to confirm these findings.