Nelarabine-containing salvage therapy and conditioning regimen in
transplants for pediatric T-cell acute lymphoblastic leukemia and
Background. Therapy for relapsed or refractory (r/r)
T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic
lymphoma (T-LBL) in children is challenging, and new treatment methods
are needed. Previous studies have shown a promising response to the
addition of nelarabine to chemotherapy for r/r T-ALL and T-LBL.
Methods. We retrospectively analyzed the therapy of
nelarabine in combination with etoposide, cyclophosphamide, and
intrathecal therapy in eight pediatric patients with r/r T-ALL and
T-LBL. The treatment regimen consisted of five consecutive days each of
nelarabine (650mg/m 2/dose) and etoposide (100mg/m
2/dose) separated by at least three days.
Results. Five patients had T-ALL, and three patients had
T-LBL. Of all patients, five achieved complete response, and the other
three achieved partial response. All the patients underwent
hematopoietic stem cell transplantation (HSCT) after two cycles of the
treatment, except for one case with one course. Three patients who had
previously received HSCT were treated with reduced-intensity
conditioning regimens, including fludarabine, melphalan, and nelarabine;
one of whom is still alive over five years after the second HSCT. Grade
2 neuropathy occurred in one patient, and other severe toxicities
commonly associated with nelarabine were not observed during
nelarabine-containing salvage therapy. With a median follow-up of 900
days for survivors, the 2-year overall survival and event-free survival
rates were 60.0% and 36.5%, respectively. Conclusion.
The addition of nelarabine to reinduction chemotherapy was useful for
HSCT in remission and did not lead to excessive toxicity. In addition, a
conditioning regimen including nelarabine appeared to be effective in
previous HSCT patients.