Skip to main content
. Author manuscript; available in PMC: 2024 Sep 1.
Published in final edited form as: Pediatr Blood Cancer. 2023 Jul 21;70(Suppl 6):e30577. doi: 10.1002/pbc.30577

Table 1:

Cellular Therapy Specific Trials

First
Author
COG
Study/Collaboration
Reference Findings Outcome/Follow-up
Study
Yanik ASCT0521 57 Phase 2 study of TNF blockade with etanercept for the treatment of idiopathic pneumonia syndrome (IPS). 28 IPS patients were treated and a high rate of complete responses, 71%. Etanercept is standard of care for pediatric IPS
Pulsipher ASCT 0431 – Ancillary analysis 7 IgH-V(D)J NGS-MRD predicted relapse and survival post-HCT better than flow MRD. Relapse 0 vs 16%, 2 yr OS 96% vs 77% PTCTC ONC1701 (EndRAD Trial) to test radiation free conditioning in NGS MRD- patients
Wagner BMT CTN 0501 17 Survival similar for both single vs double cord blood transplants (73% vs 65%) but better platelet recovery and less grade 3-4 GVHD with single cord Single cord blood unit HCT preferred for children
Dvorak ASCT1221 13 Tested BuCyMel vs BuFlu regimens in JMML patients. More complications with BuCyMel but more relapses with BuFlu. BuCyMel remains standard of care despite more complications