Stem Cell Transplant in the Treatment of Childhood Biphenotypic Acute Leukemia

Background: Many studies have found that biphenotypic acute leukemia (BAL) is associated with a poor outcome.

Methods: We retrospectively reviewed the medical records and analyzed clinicopathological data on 25 children with BAL, and correlated outcomes with prognostic factors.

Results: BAL constituted 4.4% of all acute childhood leukemia cases. In terms of immunophenotype, 14 patients had leukemia with myeloid plus B-lymphoid (M + B) marker, 7 with myeloid plus T-lymphoid (M + T) marker, and 4 with myeloid plus B-lymphoid and T-lymphoid (M + B + T) markers. Overall survival was superior in patients with the M + B immunophenotype (P = 0.004). Hematopoietic stem cell transplantation (HSCT) did not improve either overall survival or event-free survival compared to chemotherapy alone (hazard ratio 0.98, 95% CI 0.35-2.76, P = 0.966; hazard ratio 1.07, 95% CI 0.41-2.78, P = 0.88). Each of four patients with high-hyperdiploidy (>50 chromosomes) displayed a good treatment response and long-term overall survival even though these patients were treated with chemotherapy alone.

Conclusions: Treatment outcomes in childhood BAL patients differed by immunophenotype and cytogenetics. HSCT did not offer a significantly greater survival advantage compared to chemotherapy. While these data suggest that treatment should be individualized and stratified according to biologic characteristics and prognostic factors in BAL, prospective trial data are still needed.