Prioritized outcomes for AYA ALL: ASH guideline panels

An American Society of Hematology (ASH) study describing outcome prioritization for adolescents and young adults (AYAs) with acute lymphoblastic leukemia (ALL) was published in Blood Neoplasia by Saldanha et al. Two multidisciplinary guideline panels (N = 46) prioritized outcomes for 32 research questions covering first-line management, relapsed/refractory disease, or both using a three-step process. The objectives were to describe the outcome selection process, summarize prioritized outcomes, and assess how frequently those outcomes were reported in existing studies of AYAs with ALL.

Key data: Across 32 research questions, 34 unique outcomes were prioritized (median, 7 per question), spanning mortality/survival, physiological/clinical, functional, resource use, and adverse effect core areas. The most common outcomes were overall survival (OS; 84% of questions), relapse-/event-/disease-/progression-free survival or relapse (RFS/EFS/DFS/PFS/relapse; 84%), and quality of life (QoL; 81%). Short- and long-term toxicity, measurable residual disease (MRD)-negative status, allogeneic hematopoietic stem cell transplantation (allo-HSCT), and hospital admission were prioritized for 75%, 53%, 31%, and 31% of questions, respectively. Across 16 systematic reviews, each prioritized outcome was reported by a median of 25% of included studies. OS was the most frequently reported prioritized outcome (median, 79%), followed by RFS/EFS/DFS/PFS/relapse (median, 64%) and short- and long-term toxicity (median, 60%). Fertility (0 studies) and QoL (median, 0%; range, 0–6) were the least frequently reported prioritized outcomes.

Key learning: These findings highlight a gap between stakeholder-prioritized outcomes and current reporting practices in AYA ALL research, supporting the development and adoption of a core outcome set.