New Research Highlights Drivers of Outcomes in AYA With CLL

According to the researchers, their study is the first to assess the impact of oral targeted therapies among a large number of adolescents and young adults (AYA) patients with chronic lymphocytic leukemia (CLL).

With more understanding needed on the disease characteristics and outcomes of adolescents and young adults (AYAs) with chronic lymphocytic leukemia (CLL), researchers have published their findings on biomarkers and treatment patterns and their associated outcomes among these patients.

According to the researchers, their study is the first to assess the outcomes of a large number of AYA patients with CLL taking oral targeted therapies.

“The outcomes and treatment patterns of AYA patients with CLL have not been thoroughly explored since the approval of several oral targeted therapies, which have supplanted CIT in most settings,” wrote the researchers. “In this single institution retrospective analysis, we evaluated the outcomes of a cohort of AYA patients diagnosed in the last two decades; this is the first article of its kind to include a large number of patients treated with BTK, BCL2 or PI3K inhibitors.”

Over 200 patients from the institution were evaluated throughout the study, from which the researchers determined several factors being associated with outcomes. For example, age was associated with both overall survival (OS) and time to Richer transformation—the transformation of CLL to an aggressive lymphoma.

When comparing patients aged under 30 years with patients aged 35-39 years, the hazard ratio (HR) for OS was 1.72. Meanwhile, when comparing patients aged under 30 years with patients aged 30-34 years, the HR for OS was 2.33. The probability of Richter transformation was 14%, 8%, and 1% for patients aged under 30 years, 30-34 years, and 35-39 years, respectively.

Median 10-year OS was 78% and median time to first treatment (TTFT) was 2.2 years across the patients. Having pretreatment elevated beta 2-microglubin (≥3.5 mg/L), advanced Rai stage (Stage 1-4), del(11q) or del(17p) by FISH, unmutated IGHV, and CD38 positivity were all associated with shorter OS and TTFT.

There were 72 patients who received oral targeted therapy at any time throughout their disease course, del(11q) or del(17q), as well as complex karyotype were associated with inferior OS. Notably, the first therapy patients received as treatment was significantly associated with their time to second treatment (TTST).

“Validated prognostic disease biomarkers are useful for predicting outcomes for AYA patients. First-line oral targeted therapy has not been the standard of care for long enough to detect a difference in TTST compared to [chemoimmunotherapy] regimens, such as [fludarabine, cyclophosphamide and rituximab] for these patients,” explained the researchers. “While experience with oral targeted therapy continues to mature, clinicians must still consider other factors, such as quality of life, toxicity, time on therapy and cost of care when making treatment decisions for patients.”


Cherng H, Jammal N, Paul S, et al. Clinical and molecular characteristics and treatment patterns of adolescent and young adult patients with chronic lymphocytic leukaemia. B J Haematol. Published online May 10, 2021. doi:10.1111/bjh.17498

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