1Abstract 10500: COG AALL0434: Phase 3 Results in Newly diagnosed T-cell malignancy
COG AALL0434 is the largest trial ever conducted for newly diagnosed T-ALL and T-LL, and showed outstanding overall outcomes (4 yr. DFS and OS is 84.1% and 90.2% respectively). Nelarabine improves DFS for children and young adults with T-ALL and should become a new standard of care for this population
2Abstract 10525: KEYNOTE-051: Updated Phase 2 Results in Advanced melanoma or a PD-L1+ advanced, relapsed or refractory solid tumor or lymphoma
Pembrolizumab demonstrated durable anti-tumor activity in various tumors specially Hodgkin Lymphoma (ORR: 50%, mPFS: 12.2 months, 12 month PFS rate: 59.7%). The results indicate further study of pembrolizumab in pediatric patients with particulate interest in Lymphoma
3Abstract 10503: SPRINT: Phase 2 Results in Neurofibromatosis type 1 (NF1) and inoperable Plexiform neurofibromas (PN)
Phase 2 Study results confirms PR rate of 72% with that of phase 1 (71%). Most responses were sustained ≥6 months. Improvements in PN related pain and motor impairment demonstrate that selumetinib can provide clinical benefit
4Abstract 10504: MEK116540: Phase I/IIa Results in Neurofibromatosis type 1 (NF-1) associated plexiform neurofibroma
Trametinib demonstrated ORR of 46% and Disease control rate of 81%. Data support continued study of trametinib in pediatric pts. with NF-1 associated PN who currently have very limited treatment options
5Abstract 10536: STARTRK-NG: Phase 1 Results in ATRK, ROS1, and ALK inhibitor, in recurrent or refractory solid tumors
The RP2D of entrectinib in children, adolescents, and young adults with solid tumors is 550 mg/m2 daily. Preliminary antitumor activity has been seen in gene fusion-positive patients.
6Abstract 10537: MEK116540: Phase 1 Results in Relapsed solid tumors or neurofibromatosis type 1 (NF1) progressive plexiform neurofibromas (PN)
Age-specific dose, safety, and tolerability of trametinib alone and combined with dabrafenib were defined for children and adolescents (TRAEs Trametinib vs Trametinib + Dabrafenib are Paronychia (58% vs 17%) and rash (25% vs 50%). More than 50% of pts continued on Tx
7Abstract 10541: JGDN: Phase 1 (Part A) Results in Relapsed or Refractory Solid Tumors
Based on Part A results, olaratumab in combination chemotherapy (doxorubicin/ Vincristine/Irinotecan / High-dose ifosfamide) was tolerable in pediatric patients. However, investigation is still on as to which chemo combination should be preferred
8Abstract 10506: NCT01677741: Phase I/IIa (part 2) Results in BRAF V600–mutant relapsed refractory low-grade glioma
Dabrafenib demonstrated clinical activity (ORR: 44%; mPFS: 35 mo) with tolerability in pediatric pts with relapsed, refractory, or progressive BRAF V600E mutation–positive LGG
9Abstract 10505: NCT01677741: Phase 1 Results in BRAF V600–positive high-grade glioma (HGG)
Dabrafenib was well tolerated in pediatric pts with BRAF V600–positive HGG, and a number of durable objective responses demonstrate the potential for clinical benefit (ORR:45%; mDoR: 7.7 months)
10Abstract 10540: Cediranib: NCT00942877 - Phase 2 Results in Alveolar soft part Sarcoma (ASPS):
Cediranib did not reach the target RR. However, prolonged SD was observed in 5 pts, but given the indolent nature of ASPS, SD cannot be clearly attributed to cediranib
11Abstract 10542: CUDC-907: NCT02909777 - Phase 1 Results in Solid tumors, CNS tumors, and lymphomas
CUDC-907 was found to be safe and tolerable with RP2D dose is 45 mg/m2 administered orally on a 5/2 schedule
12Abstract 10556: Entinostat: ADVL1513 - Phase 1 Results in Solid tumors and CNS tumors
Entinostat was found to be safe and effective with RP2D dose is 4 mg/m2 Oral, once weekly
13Abstract 10536: Survival among children, adolescent and young adults with Acute Lymphoblastic Leukemia (ALL) receiving In-patient Cancer Care throughout therapy
78% of children and 19% of adolescents and young adults (AYAs) received all their treatment at Specialized Cancer Centres. In both age groups, worse survival was associated with older age, Hispanic and African American race/ethnicity (vs non-Hispanic white), public insurance (vs private) and comorbidities. Therefore, children and AYAs should be referred to and treated at SCCs for better survival outcomes