Background: Daunoxome (DNX) is an encapsulated form of daunorubicin in liposomal vesicles with suggested better pharmacokinetics, pharmacodynamics and lesser cardio toxicity than the free form. Polyethyl glycol asparaginase (PEG-ASPA), a modified form of L-asparaginase, has better activity and lesser immunogenicity than its native molecule.
Aim: To evaluate on a compassionate basis, the combination of Daunoxome and PEG-ASPA, as regard to efficacy and toxicity, as a salvage treatment in refractory/relapsed childhood ALL.
Methods: The combination of these 2 drugs were used in 9 multiple relapsed or refractory ALL children on the basis of: DNX weekly 100 mg/m2 D1, 8, 15. PEG-ASPA single, 2500IU/m2 dose D15. Vinca alkaloids and corticosteroids were associated.
Results: Median hospital stay was 4 days (0-55).
Complications: Neutropenia grade IV n=4, grade III infection n=6, grade II cardiac toxicity n=1, grade III allergy, grade III hemostasis disorders, thrombosis, n=1 respectively. All patients achieved complete remission (CR) except one who died from disease progression. 8 patients were subjected to hematopoietic stem cell transplantatation (HSCT). Two patients of them are alive and well, 4 died from transplant related causes and 2 from disease progression.
Conclusion: Salvage treatment containing DNX and PEG-ASPA, of small sample childhood ALL with very advanced disease, is feasible as regard toxicity and response rate allowing to HSCT and possible cure.