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July 27, 2026

Feasibility and Efficacy of Lorlatinib in Japanese Patients With Relapsed/Refractory ALK ‐Aberrant Neuroblastoma

TITakashi ImazuKyoto University HKHirohito KubotaKyoto University SSSatoshi SaidaKyoto University

Puntos clave

This study aims to evaluate the feasibility and efficacy of lorlatinib in Japanese patients with relapsed or refractory ALK-aberrant neuroblastoma.
Five heavily pretreated patients received off-label lorlatinib.
Patients exhibited various ALK alterations, including F1174L and MYCN amplification.
Adverse events were monitored for tolerability assessment.
Three patients achieved partial responses, while two experienced disease progression.
The longest progression-free survival was 6.7 months in a patient with F1174L and non-amplified MYCN.
Lorlatinib was generally well tolerated with manageable adverse events.

Resumen

Lorlatinib, a third-generation ALK inhibitor, was administered off-label to five heavily pretreated patients with relapsed or refractory ALK-aberrant neuroblastoma. ALK alterations included F1174L, R1275Q, BEND5::ALK fusion, and ALK amplification; three patients had MYCN amplification. Best responses were three partial responses and two disease progressions. The longest progression-free survival (6.7 months) occurred in a patient with F1174L and non-amplified MYCN, whereas rapid progression was observed in two MYCN-amplified cases. Lorlatinib was generally well tolerated with manageable adverse events. These findings suggest that lorlatinib is a feasible therapeutic option in ALK-aberrant neuroblastoma and that clinical heterogeneity in treatment response warrants further investigation.

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