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

Abstract EN

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 100mg/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 Journal of the Egyptian Nat.

Cancer Inst., Vol.

20, No.

1, March: 55-62, 2008 Liposomal Daunorubicin (Daunoxome) and Polyethylated Glycol Conjugated Asparaginase (PEG-ASPA) in Children with Relapsed and Refractory Acute Lymphoblastic Leukemia Treated on Compassionate Basis MOHAMED SEDKI, M.D.1; JEAN-PIERRE VANNIER, M.D.2; GUY LEVERGER, M.D.3; KARIMA YAKOUBEN, M.D.1; DALILA ADJAOUD, M.D.1; ETIENNE VILMER, M.D.1 and ANDRÉ BARUCHEL, M.D.1 The Departments of 1Service d’Hématologie-Immunologie Pédiatrique, Hôpital Robert Debré, Paris, France.

2Service d’Hématologie-Oncologie Pédiatrique Hôpital Charles Nicolle CHU Rouen, France and 3Service d’Hématologie-Oncologie Pédiatrique, Hôpital d’Enfants Armand Trousseau 75012 Paris, France.

55 advanced disease, is feasible as regard toxicity and response rate allowing to HSCT and possible cure.