First Author COG Study Reference Findings Outcome/Follow-up Study
Pollard AAML1031 31 Sorafenib for HAR FLT3/ITD+ patients during chemo and 1 yr of maintenance (post-HCT if donor available). Sorafenib exposed patients had improved EFS, DFS and relapse rates. AAML1831 – Test gilteritinib, a second generation TKI, that may be less toxic than sorafenib.
Davies AAML05P1 28 Prospective study of KIR typing of unrelated donors prior to donor selection for patients with AML. No difference in OS, DFS or RR between KIR matched and mismatched. Acute GVHD rates were lower in the mismatched group. PTCTC ONC1401 trial – testing whether KIR mismatches are beneficial with haploidentical donors
Reddy ACNS0333 24 Intensified treatment for CNS AT/RT patients improved 4 yr EFS from 6% to 35%. Planned ACNS2232 – add tazemetostat to ACNS0333 schema to decrease relapse.
Park ANBL0532 21 Randomized 355 HR NB to 1 (CEM) vs 2 (Thio/Cy then dose reduced CEM) HCT. Tandem recipients had improved 3 yr EFS 62% vs 48%. ANBL1531 – testing whether 131I-MIBG treatment during induction improves EFS.
Andolina AYA 36 Retrospective analysis of GVHD, relapse, and survival in 188 pts transplanted on 3 COG protocols for acute leukemia (ASCT0431, AAML0531, AAML1031). Compared rates of acute and chronic GVHD, relapse and survival based on younger child (ages 2-12) vs AYA (13-21). AYA were more likely to experience grade II-IV acute GVHD compared to younger children (56% vs 32%, p=0.006) and less likely to relapse (20% vs 35%, p=0.032). No difference by age for chronic GVHD (26% vs 20%, p=0.72), EFS (43% vs 52%, p=0.18) or OS (49% vs 52%, p=0.56). These results formed the basis of the age stratification in the current ASCT2031 study.
Schofield 58 Joint Cellular Therapy and Behavioral Sciences task force developed evidence based guidelines for monitoring neurocognitive outcomes following cellular therapy treatments. Guidelines inform COG CT trial design.