Y Benoit MD, PhD

Juvenile myelomonocytic leukaemia: the quest for more specific therapies

Summary

Juvenile myelomonocytic leukaemia is a very rare, aggressive stem cell disorder predominantly affecting infants and young children. Current survival rates are disappointing and the only available curative therapy is haematopoietic stem cell transplantation. Over the last years, intensive research efforts elucidated a plethora of molecular aberrations involved in the pathogenesis of juvenile myelomonocytic leukaemia. Current investigations are mainly directed towards the complete unravelling of the molecular biology behind the disease in order to find more specific drugs. This review will focus on the diagnosis, genomic characterisation and the use of experimental therapies in juvenile myelomonocytic leukaemia.

(BELG J HEMATOL 2014; 5(4): 119–24)

Minimal residual disease quantification by PCR in childhood acute lymphoblastic leukaemia

Summary

In Belgium approximately 70 children are diagnosed with acute lymphoblastic leukaemia annually. For these children, the monitoring of minimal residual disease has an important prognostic value. The level of minimal residual disease during the first three months of therapy is used to recognise subgroups that differ substantially in outcome. Two techniques are used for minimal residual disease monitoring: the Genescan method and the allele specific oligonucleotide polymerase chain reaction. The Genescan method is a less sensitive method (10⁻³) but is fast and less expensive. The allele specific oligonucleotide polymerase chain reaction requires more time and budget but has a sensitivity of 10⁻⁴–10⁻⁵. Both techniques have proven their value in minimal residual disease monitoring in childhood acute lymphoblastic leukaemia.

(BELG J HEMATOL 2014;5(3):81–8)

P2.09 Impact of minimal residual disease monitoring on therapy in Belgian childhood acute lymphoblastic leukaemia

PEG-asparaginase in the treatment of childhood acute lymphoblastic leukaemia

Summary

Asparaginase is an essential compound of combination chemotherapy in acute lymphoblastic leukaemia in children and adults. Essentially, three preparations of asparaginase are used in childhood acute lymphoblastic leukaemia: native Escherichia coli asparaginase, Erwinia chrysanthemi asparaginase and PEG-asparaginase. Although PEG-asparaginase seems to have some advantages over the other asparaginase preparations, its clinical use in Europe is limited to second-line therapy after allergic reactions to native asparaginase. This is in contrast to the United States, where PEG-asparaginase has been approved as first-line treatment of children with acute lymphoblastic leukaemia. This report describes the properties, clinical benefits and side effects of PEG-asparaginase.

(BELG J HEMATOL 2013;4(4): 138–143)

Cost-minimisation analysis of PEG-L-Asparaginase versus native L-Asparaginase for the treatment of children with acute lymphoblastic leukaemia in Belgium

Summary

PEG-L-Asparaginase (Oncaspar®) is a major compound of antineoplastic combination therapy for reinduction in acute lymphoblastic leukaemia in children and adults with known hypersensitivity. In the United States, it has been approved for many years as first-line treatment of children with acute lymphoblastic leukaemia. Its clinical benefits have been extensively described. In this report, a cost-minimisation analysis comparing the direct cost of PEG-L-asparaginase with that of native E. coli and Erwinia L-asparaginase treatment is described.

(BELG J HEMATOL 2013; 4(4): 144–147)

P.02 H3K27me3 erasers, novel targets in T-cell acute lymphoblastic leukaemia

P.03 Extending the functional redundant cooperative miRNA tumour suppressor and oncogene network in T-ALL