Improving results of autologous stem cell transplantation for Philadelphia-positive acute lymphoblastic leukaemia in the era of tyrosine kinase inhibitors: a report from the Acute Leukaemia Working Party of the European Group for Blood and Marrow Transplantation.

Background: Outcome of Philadelphia-positive acute lymphoblastic leukaemia (Ph+ ALL) improved significantly with the introduction of tyrosine kinase inhibitors (TKIs). Autologous stem cell transplantation (ASCT) has never been considered a standard of care in this setting. The aim of our study was to analyse if results of ASCT improved in the era of TKIs.

Patients And Methods: One-hundred and seventy-seven adults with Ph+ ALL treated with ASCT in first complete remission were analysed for the impact of year of transplantation on outcome. Additional analysis was performed including 32 patients for whom detailed data on the use of TKIs and the status of minimal residual disease were collected.

Results: The probability of the overall survival (OS) at 3 years increased from 16% for transplants performed between 1996 and 2001 to 48% between 2002 and 2006 and 57% between 2007 and 2010 (P<.0001). Leukaemia-free survival (LFS) was 11%, 39% and 52%, respectively (P<.0001). Relapse incidence decreased from 70% to 45% and 45% (P=.01), respectively, while non-relapse mortality was 19%, 15% and 3% (P=.08). In a multivariate analysis, year of ASCT was the only independent factor influencing the risk of treatment failure (hazard ratio (HR)=0.37; P<.001). In a subgroup of 22 patients actually treated with TKIs and being in complete molecular remission at the time of ASCT, the LFS rate at 3 years was 65%.

Conclusions: Results of ASCT for Ph+ ALL improved significantly over time. Prospective, innovative studies are needed to verify the role of ASCT in this patient setting.