Long-term follow up of pediatric Philadelphia positive acute lymphoblastic leukemia treated with the EsPhALL2004 study: high white blood cell count at diagnosis is the strongest prognostic factor
Abstract
Approximately 3-5% of children with acute lymphoblastic leukemia (ALL) present with Philadelphia chromosome (Ph+). In the pre-tyrosine kinase inhibitors (TKIs) era, Ph+ ALL was associated with a poor prognosis.The outcome improved with the use of TKIs.11-14 Results of the EsPhALL2004 study (EudraCT 2004-001647-30 and clinicaltrials.gov identifier 00287105) with the use of imatinib mesylate were published with a median follow-up time of 3.1 years;we now report on long-term outcomes and the impact of prognostic factors in this study.
This update shows that event-free survival (EFS) is stable after about 4 years, and that white blood cell (WBC) count at diagnosis is the most relevant prognostic factor, in a setting where the large majority of patients receives imatinib and hematopoietic stem cell transplantation (HSCT).