Feasibility of a New Risk Stratification System in Pediatric Anaplastic Large Cell Lymphoma: Result of a Prospective Open-Label Multiple-Institution Study

Abstract

Abstract Background The risk stratification of pediatric anaplastic large cell lymphoma (ALCL) has not been unified. Purpose: In this study, new risk factors were included to establish a new risk stratification system for ALCL, and its feasibility in clinical practice was explored. Methods On the basis of the non-Hodgkin’s lymphoma Berlin–Frankfurt–Munster 95 (NHL-BFM-95) protocol, patients with minimal disseminated disease (MDD), high-risk tumor site (multiple bone, skin, liver, and lung involvement), and small cell/lymphohistiocytic (SC/LH) pathological subtype were enrolled in risk stratification. Patients were treated with a modified NHL-BFM-95 protocol combined with an anaplastic lymphoma kinase inhibitor or vinblastine. Results A total of 136 patients were enrolled in this study. The median age was 8.8 years. The 3-year event-free survival (EFS) and overall survival of the entire cohort were 77.7% and 92.3%, respectively. The 3-year EFS rates of R1, R2, and R3 patients were 100%, 89.5%, and 67.9%, respectively. The prognosis of patients with MDD (+), stage IV cancer, SC/LH lymphoma, and high-risk sites was poor, and the 3-year EFS rates were 45.3%, 65.7%, 55.7%, and 67.9%, respectively. Conclusions This study established a new risk stratification system for the precise treatment of pediatric ALCL that is convenient and feasible in clinical practice. Among high-risk patients, patients with SC/LH lymphoma and MDD (+) at diagnosis still need to receive better treatment.