Prognostic relevance of TLX3 (HOX11L2) expression in childhood T‐cell acute lymphoblastic leukaemia treated with Berlin–Frankfurt–Münster (BFM) protocols containing early and late re‐intensification elements

Summary TLX3 expression ( TLX3+ ) in childhood T‐cell acute lymphoblastic leukaemia (T‐ALL) seems to be associated with a poor prognosis when treated with regimens that lack early and/or late re‐intensification therapy elements. Because such elements are essential components of the ALL‐BFM (Berlin–Frankfurt–Münster) protocols, we evaluated whether TLX3+ T‐ALL patients benefit from this type of therapy. Thirty‐one/131 childhood T‐ALL cases (24%) enrolled into four population‐based Austrian ALL‐BFM therapy studies were TLX3+. The male to female ratio was 3·5:1 and median age and leucocyte count at diagnosis were 8·7 years and 58·9 × 10 9 /l, respectively. Twenty‐four patients (77%) were good responders to prednisone. All were in complete remission after induction therapy. After a median observation time of 4·9 years (range 0·4–16·1 years) 28/31 TLX3 + cases remained in first complete remission after chemotherapy with one after additional stem cell transplantation. Although molecular disease was frequently present after a 4‐drug induction therapy, final treatment outcome was excellent indicating that TLX3 + T‐ALL cases may benefit from a BFM‐type of ALL therapy with early and late re‐intensification elements. Moreover, the fact that 2/3 relapses were also NUP214‐ABL1 + suggests that these cases might represent the particular risk‐prone TLX3 + subgroup that could benefit from a targeted tyrosine kinase inhibitor therapy.