Prioritized health outcomes for adolescents and young adults with acute lymphoblastic leukemia

The American Society of Hematology convened 2 multidisciplinary panels to develop guidelines for the management of acute lymphoblastic leukemia (ALL) among adolescents and young adults (AYAs). The objectives of this study are to (1) describe the process for selecting and prioritizing outcomes for 32 research questions relevant to the guideline development, (2) summarize the prioritized outcomes, and (3) describe the frequency of reporting of those outcomes in existing studies of AYAs with ALL. The panels prioritized outcomes for each of the 19 questions on frontline management, 9 on the management of relapsed/refractory disease, and 4 related to both topics. A 3-step process was used: (1) initial identification of outcomes, (2) online survey of all panel members to rate each outcome's importance, and (3) iterative discussions among the panels to finalize the prioritized outcomes. We examined the frequency with which each prioritized outcome was reported for systematically reviewed research questions. The panels prioritized 34 unique outcomes across questions (median, 7 outcomes per question). The most common outcomes were overall survival (27 questions [84% of questions]); relapse-, event-, disease-, or progression-free survival/relapse (27 questions [84%]); and quality of life (26 questions [81%]). Across 16 systematic reviews for which we found at least 1 study, each prioritized outcome was reported by a median of 25% (interquartile range, 20-48; range 0-79) of the studies. The outcomes prioritized by multidisciplinary guideline panels can inform future primary studies, systematic reviews, and guidelines on ALL, other blood cancers, and AYAs. Future research should involve the development and adoption of a core outcome set for ALL.