Hematology

Systematic review: many RWD indirect comparisons lack causal transparency

This systematic review of 55 single-arm drug trials (1987–2024) found many indirect comparisons to real-world controls lacked rationale and threatened validity. Only 29% of studies justified the indirect comparison and control recruitment often spanned earlier, longer time windows, compromising positivity. Studies using balancing-based methods were more recent and reported larger sample sizes and more post-adjustment balance checks. Authors recommend adopting transparent causal-inference frameworks and balancing-based estimators for unanchored indirect comparisons with real-world controls. 1

ACCESS trial broadened alloHCT access and increased participant diversity

The ACCESS cohort (n=208) enrolled adults lacking matched donors using mismatched unrelated donors and showed greater racial, ethnic, and socioeconomic diversity than comparator cohorts. ACCESS had higher Medicaid representation and more non-White participants versus the PRO Protocol and BMT CTN 1703 cohorts. The findings support that broadened eligibility and alternative donor strategies can expand transplant access and reduce disparities. 2

ICU outcomes for acute leukemia improved over time but remain poor with ventilation

Individual-participant meta-analysis of 2003 adults from 55 ICUs found overall ICU mortality 45% and 66% for ventilated patients. Age >65 (OR 1.98), AML diagnosis, relapsed disease, and mechanical ventilation (OR 6.46) independently increased mortality. Year of ICU admission associated with improved survival only among ventilated patients, suggesting temporal gains concentrated in the sickest group. 3