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.


OBJECTIVE
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).


RESULTS
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.


CONCLUSIONS
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.
The Department of Service d'Hématologie-Immunologie Pédiatrique, Hôpital Robert Debré, Paris, France. msedky64@yahoo.com
Mohamed
M
Sedki
Jean-Pierre
JP
Vannier
Guy
G
Leverger
Karima
K
Yakouben
Dalila
D
Adjaoud
Etienne
E
Vilmer
André
A
Baruchel
D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001215:Asparaginase / Q000008:administration & dosage / Q000009:adverse effects; D002648:Child; D057176:Compassionate Use Trials; D004317:Doxorubicin / Q000008:administration & dosage / Q000009:adverse effects; D005260:Female; D006801:Humans; D008297:Male; D011092:Polyethylene Glycols / Q000008:administration & dosage / Q000009:adverse effects; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000188:drug therapy*; D012008:Recurrence; D012189:Retrospective Studies; D016879:Salvage Therapy
D011092:Polyethylene Glycols; C042705:pegaspargase; D004317:Doxorubicin; D001215:Asparaginase