- In relapsed/refractory T-acute lymphoblastic leukaemia/lymphoma (R/R T-ALL/T-LBL), salvage therapy with nelarabine is an effective option in terms of overall response rate.
- Nelarabine is also effective in bridging patients to allogeneic haematopoietic stem cell transplantation (Allo-SCT).
- The safety profile is acceptable.
Why this matters
- Outcomes for adult patients with R/R T-ALL/T-LBL are poor (5-year survival
- No standard of care is available in this setting and data from clinical trials are limited.
- There’s a need of data supporting the use of nelarabine, specifically approved as an orphan drug for these patients.
- 118 adult patients with R/R T-ALL/T-LBL treated with at least one complete cycle of nelarabine (1,500 mg/sqm; Days 1,3,5) as salvage therapy in 27 haematologic sites were included in the observational, phase 4 study.
- The overall response rate (ORR) and the overall survival (OS) were primary endpoints.
- Additional endpoints included Allo-SCT rate after nelarabine, post-transplant OS and safety.
- ORR in the whole population was 50%, with 36% and 14% of patients achieving complete remission (CR) and partial remission (PR), respectively.
- OS for the entire population was 38% at 1 year (median survival 8 months).
- Relapsing patients showed an advantage in OS compared to refractory patients.
- 40% of patients (68% complete or partial response) underwent Allo-SCT after nelarabine and a better 1-year OS was observed in this population compared to non-transplanted patients (54% vs 22 %; log-rank P=0.0001; median survival 14.5 vs 4.8 months).
- 2- and 5-year OS after transplant were 46% and 38%, respectively
- Response and Allo-SCT after treatment were the only factors affecting post-nelarabine OS.
- A 5-year OS of 40% and a median survival of 22.5 months were observed in responders who also received Allo-SCT.
- Nelarabine showed an acceptable safety profile with grade III/IV neurological events reported in 9% of patients.
“Waiting for new treatments for R/R T-ALL/T-LBL, nelarabine can be considered an effective option and a good bridge to transplant. At the moment, no clear and complete data on mutational status are available in our population, but this is a very important issue and we think we will be able to collect these data in the future”. Anna Candoni, Division of Haematology and SCT, University of Udine, Udine, Italy.