Identifying patients with chronic lymphocytic leukemia without need of treatment: End of endless watch and wait?
Christian Brieghel, Veerle Galle, Rudi Agius, Caspar da Cunha‐Bang, Michael Asger Andersen, Philip Vlummens, Mattias Mattsson, Richard Rosenquist, Karin E. Smedby, Carmen Herling, Jasmin Bahlo, Michael Hallek, Jens Lundgren, Fritz Offner, Carsten Utoft Niemann
Abstract
INTRODUCTION: Early-stage chronic lymphocytic leukemia (CLL) challenges specialized management and follow-up. METHODS: We developed and validated a prognostic index to identify newly diagnosed patients without need of treatment (CLL-WONT) by a training/validation approach using data on 4708 patients. Composite scores derived from weighted hazards by multivariable analysis defined CLL-WONT risk groups. RESULTS: /L; 2 points), β2-microglobulin (>4 mg/L: 1 point), IGHV mutation status (unmutated: 1 point), and 11q or 17p deletion (1 point) were independently associated with shorter time to first treatment (TTFT). Low-risk patients demonstrated 5-year TTFT of 2% by internal validation, but 7-19% by external validation. Including all patients with complete scores, the 5-year TTFT was 10% for the 756 (39%) CLL-WONT low-risk patients, and the 704 (37%) patients who were both CLL-WONT and CLL-IPI low risk demonstrated even lower 5-year TTFT (8%). CONCLUSION: We have adopted the CLL-WONT at an institution covering 1 800 000 individuals to allow patients with both low-risk CLL-WONT and CLL-IPI to be managed by primary healthcare providers, thereby prioritizing specialized hematology services for patients in dire need.