Pediatric Oncology – Key Results from ASCO 2018

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

Innovative Compounds in Development for Pediatric Oncology

Sakshi Saini
Sakshi Saini
Sakshi has a passion for healthcare and innovation in medical techs (Rx and devices). Her ability lies in providing an "outside-in" perspective to colleagues involved in the design, development and commercialization of medical innovation. She comes with a wide range of experience in the oncology space pertaining to both solid tumors and blood malignancies (Lung, Colorectal, Melanoma,AML and MDS).She works with RAS clients to provide activities related to market research and competitive intelligence. She is also a member of European Society of Clinical Oncology.

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