Study Design and Participants
This multicentre, investigator-initiated, single-arm, phase 2 trial was conducted in two academic medical centres in the USA. The trial aimed to determine the rate of MRD undetectable response of the BOVen regimen in patients with CLL or SLL. Inclusion criteria included age at least 18 years, Eastern Cooperative Oncology Group performance status up to 2, and diagnosis of CLL or SLL, among others. Patients with certain conditions or treatments were excluded.
Procedures
BOVen was administered in 28-day cycles, with specific dosages and schedules for each drug. Treatment duration was determined by prespecified MRD criteria, with patients undergoing bone marrow biopsy and aspiration to assess MRD status. Dose adjustments were permitted for adverse events.
Outcomes
The primary endpoint was undetectable MRD, achieved by 89% of patients in the per-protocol analysis. Secondary endpoints included time to undetectable MRD, treatment-free survival, and safety. The study also explored the use of ΔMRD400 as a predictive biomarker for treatment duration.
Results
Between March 14, 2019, and Oct 10, 2019, 39 patients initiated treatment. The median age was 62 years, with a majority being male. Of these, 33 patients (89%) reached the prespecified MRD endpoint and discontinued therapy after a median of 10 cycles. The study reported a low rate of grade 3 or worse neutropenia and one episode of febrile neutropenia.
Discussion
The trial's results are highly encouraging, with BOVen showing a favourable safety profile and high efficacy in achieving undetectable MRD. The study suggests that ΔMRD400 could serve as a biomarker to guide treatment duration, identifying patients who may benefit from an abbreviated treatment course. Further research is needed to validate these findings and explore the regimen's potential in other patient populations.
This study represents a significant step forward in the treatment of CLL/SLL, offering a promising new approach that could improve patient outcomes and quality of life.
