Document Type

Article

Publication Date

3-25-2025

Identifier

DOI: 10.1182/bloodadvances.2024014745

Abstract

Treatment options for patients with relapsed or refractory (R/R) anaplastic large cell lymphoma (ALCL) have increased in the era of targeted therapies such as brentuximab vedotin (BV) and anaplastic lymphoma kinase (ALK) inhibitors. However, there is no standard treatment and published data evaluating their use are limited. The goal of this retrospective study was to describe current real-world treatment and outcomes of pediatric, adolescent, and young adult patients with R/R ALK-positive ALCL. We conducted a retrospective, multi-institutional study identifying 81 patients with R/R ALK-positive ALCL aged ≤21 years at initial diagnosis treated between 2011 and 2022 across 18 institutions. Median time from diagnosis to relapse was 8.9 months (range, 2.6-131.9). Initial reinduction regimens included ALK-inhibitor monotherapy (n = 37, 46%), BV monotherapy (n = 19, 23%), chemotherapy without targeted therapy (n = 12, 15%), chemotherapy with targeted therapy (n = 9, 11%), or vinblastine monotherapy (n = 4, 5%), with 83% of patients achieving a complete response to initial reinduction regimen. Fifty-eight patients received a hematopoietic stem cell transplant (HSCT), 11 autologous and 48 allogeneic, with 1 receiving both. Duration of treatment for patients receiving BV or the ALK-inhibitor crizotinib (CZ) varied widely (BV, 1-11 years; CZ, 2-10 years). Five-year event-free survival was 63% (95% confidence interval [CI], 53-75) and 5-year overall survival was 91% (95% CI, 84-98). This is, to our knowledge, the largest collection of patients with R/R ALK-positive ALCL treated in the era of targeted therapy. Patients achieved excellent responses to ALK-inhibitor or BV monotherapy, but questions remain about duration of therapy and role of HSCT.

Journal Title

Blood Adv

Volume

9

Issue

6

First Page

1356

Last Page

1365

MeSH Keywords

Humans; Lymphoma, Large-Cell, Anaplastic; Child; Adolescent; Anaplastic Lymphoma Kinase; Female; Male; Child, Preschool; Retrospective Studies; Young Adult; Treatment Outcome; Molecular Targeted Therapy; Protein Kinase Inhibitors; Infant; Brentuximab Vedotin; Adult; Drug Resistance, Neoplasm; Recurrence; Antineoplastic Combined Chemotherapy Protocols; Hematopoietic Stem Cell Transplantation

PubMed ID

39841960

Keywords

Large-Cell Anaplastic Lymphoma; Anaplastic Lymphoma Kinase; Retrospective Studies; Treatment Outcome; Molecular Targeted Therapy; Protein Kinase Inhibitors; Infant; Brentuximab Vedotin; Adult; Drug Resistance, Neoplasm; Recurrence; Antineoplastic Combined Chemotherapy Protocols; Hematopoietic Stem Cell Transplantation

Comments

Grants and funding

Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved.

Publisher's Link: https://ashpublications.org/bloodadvances/article/9/6/1356/535241/Pediatric-relapsed-refractory-ALK-positive

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