A multicenter observational cohort study in survivors of Down Syndrome-associated acute leukemia (ALTE22C1): a report from the Children’s Oncology Group

Gramatges MM, Sanclemente LN, Hall L, Taylor OA, Nuño MM, Bhatia S, Chow EJ, Getz KD, Hitzler JK, Li AM, McCloskey K, Nathan PC, O’Brien MM, Patel S, Verma A, Yarbrough AR, Richard MA, Rosser TC, Jacola LM, Lupo PJ, Rabin KR. A multicenter observational cohort study in survivors of Down Syndrome-associated acute leukemia (ALTE22C1): a report from the Children’s Oncology Group. BMC Cancer. 2025 Oct 20;25(1):1611. doi: 10.1186/s12885-025-14898-z. PMID: 41116168; PMCID: PMC12538926.

Down syndrome (DS) is a common genetic disorder resulting from an extra copy of genetic material from all or part of chromosome 21. Individuals with DS have a higher burden of co-occurring structural birth defects, neurocognitive delay, and chronic health conditions when compared to those without DS, as well as a 10 to 20-fold excess risk for acute leukemia (AL). Few studies have reported the late effects of cancer treatment in DS-AL survivors, and even fewer have compared outcomes to children with DS and no cancer history. The Children’s Oncology Group study ALTE22C1 was developed to address this knowledge gap.