Liposomal daunorubicin (Daunoxome) and polyethylated glycol conjugated asparaginase (PEG-ASPA) in children with relapsed and refractory acute lymphoblastic leukemia treated on compassionate basis.

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.