Melphalan and cytarabine as a salvage therapy in children with relapsed
or refractory acute leukemia
Abstract
Background: Leukemia in children has a good prognosis with an overall
cure rate of 85% in pediatric patients with acute lymphoblastic
leukemia and 50-60% in pediatric patients with acute myeloid leukemia.
Nevertheless in patients with refractory or relapsed leukemia the
prognosis is limited and can only be cured by a salvage chemotherapy, in
most cases followed by an allogeneic hematopoietic stem cell
transplantation. Methods: In this retrospective case cohort ananlysis we
investigated the outcome of eight patients with relapsed or refractory
acute myeloid (n=2), lymphoblastic (n=4), biphenotypic (n=1) leukemia or
T-lymphoblastic lymphoma (n=1) who failed to respond to standard salvage
regimens. They received a salvage therapy with melphalan and cytarabine
at our institution between 2015 and 2019. Results: After salvage
chemotherapy with melphalan and cytarabine 63% of the patients achieved
a remission of the disease and qualified for subsequent allogeneic
hematopoietic stem cell transplantation. The one year overall survival
rate was 50%, the three year overall survival rate was 29%. 25% of
patients experienced a temporary period of fever and SIRS. Conclusions:
The reported results of our case cohort analysis indicate that a salvage
therapy with melphalan and cytarabine in relapsed or refractory leukemia
could represent a curative approach with the possibility of achieving
remission and subsequent allogeneic hematopoietic stem cell
transplantation. Future multicentre studies are needed to verify the
here presented results.