
Response assessment.

death, malignancy, Treatment-related, PD, PRES
INFILTRATION, DISEASE, SECONDARY, REMISSION, ADVERSE EVENT
Before the first DB cycle, 25/68 patients (36.8%) were in CR, 35 (51.5%) were in PR, and eight (11.8%) had NR/MR/PD (Table Response to trial treatment: Two patients were ineligible because of screening failure or substantial PD with impaired general condition (Lansky score < 20 before the first antibody cycle). The remaining 68 patients were included in this analysis. (A) The intention-to-treat population (all trial patients), ie, last antibody cycle refers to the last administered cycle of DB received by the respective patient. (B) Patients receiving full protocol treatment (per-protocol population), that is, response represents the remission status after completion of trial treatment, defined as six full cycles of DB treatment. Dropout patients are listed in the upper part of B (PD before treatment completion and dropout side effects or AE). Percentages in the lower part of B refer to patients treated according to protocol (n = 39) and to the whole cohort of 68 patients (intention-to-treat population). CR maintained: patients who started DB treatment in CR; CR improved: patients who achieved CR during/after DB treatment. Event-free survival and overall survival were calculated from start of trial treatment (first antibody cycle in this trial, ie, first day of first DB cycle after haplo-SCT). AE, adverse event; CR, complete remission; DB, dinutuximab beta; EFS, event-free survival; haplo-SCT, haploidentical stem-cell transplantation; MR, mixed response; NR, nonremission; OS, overall survival; PD, progressive disease; PR, partial remission; SD, stable disease.A total of 39 patients (57.4%) completed six cycles: 13/68 (19.1%) maintained CR, 15 (22.1%) achieved CR, six (8.8%) had PR, two (2.9%) SD, and three (4.4%) PD at the end of treatment (Fig At the last follow-up in October 2021, at a median follow-up of 7.8 years, 35/68 (51.5%) patients were alive. The 5-year EFS and OS rates from start of trial treatment of the whole cohort were 53% (95% CI, 41 to 65) and 43% (95% CI, 31 to 55), respectively (Fig (A) OS and EFS of whole cohort; (B) CI of relapse; (C) EFS: remission status before first DB cycle; (D) OS: remission status before first DB cycle; (E) EFS: BM infiltration before first DB cycle; (F) OS: BM infiltration before first DB cycle; (G) EFS: age at study entry; and (H) OS: age at study entry. Event-free survival and overall survival were calculated from start of trial treatment (first antibody cycle in this trial, ie, first day of first DB cycle after haplo-SCT). BM, bone marrow; CI, cumulative incidence; CR, complete remission; DB, dinutuximab beta; EFS, event-free survival; haplo-SCT, haploidentical stem-cell transplantation; MR, mixed response; NR, nonremission; OS, overall survival; PD, progressive disease; PR, partial remission; SD, stable disease.Causes of death were relapse in 29 patients, infections in three patients, PRES, and secondary malignancy in one patient each. Treatment-related mortality (TRM) at day 100 and after one year was 1.5% [95% CI, 0 to 7] and 7.4% [95% CI, 3 to 15], respectively.In MVAs, we studied the impact of possible risk factors at relevant time points of the treatment (Table Multivariate Analysis of Risk Factors for OS and EFS at Various Treatment Time Points