Feasibility and Efficacy of Lorlatinib in Japanese Patients With Relapsed/Refractory <i>ALK</i> ‐Aberrant Neuroblastoma
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.
Authors
- Kagehiro Kouzuki
- Shuro Nishio (ORCID: https://orcid.org/0009-0007-2200-4685)
- Masahiro Irie (ORCID: https://orcid.org/0000-0002-9446-6244)
- Yusuke Imanaka (ORCID: https://orcid.org/0000-0002-7261-7080)
- Kimiyoshi Sakaguchi (ORCID: https://orcid.org/0000-0002-3665-401X)
- Satoshi Saida (ORCID: https://orcid.org/0000-0002-8930-3539)
- Yoshinori Uchihara (ORCID: https://orcid.org/0000-0001-6006-4362)
- Itaru Kato (ORCID: https://orcid.org/0000-0002-2932-4960)
- Ryo Akazawa (ORCID: https://orcid.org/0000-0002-8770-0493)
- Katsutsugu Umeda (ORCID: https://orcid.org/0000-0002-6844-2011)
- J Takita
- Hirohito Kubota (ORCID: https://orcid.org/0000-0002-6935-5120)
- Yoshiyuki Takahashi (ORCID: https://orcid.org/0000-0001-8644-171X)
- Ryunosuke Tojo
- Takashi Imazu (ORCID: https://orcid.org/0009-0002-1300-1734)
Institutions
- University of Fukui (JP)
- Hiroshima University (JP)
- Tohoku University (JP)
- Kyoto University (JP)
- Hamamatsu University School of Medicine (JP)
- Nagoya University (JP)
Publication Details
- Journal
- Pediatric Blood & Cancer
- Published
- 2026-07-25
- DOI
- https://doi.org/10.1002/1545-5017.70511
- Primary Topic
- Neuroblastoma Research and Treatments
- Type
- article
- Field-Weighted Citation Impact
- 0.00