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age
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•Prescrire International Special Edition 2015/Volume 24 N°158
A
New Products
About 5% of patients with acute
lymphoblastic leukaemia have the
abnormal “Philadelphia” chromosome
associated with a poor prognosis.
Initial chemotherapy takes place in
several phases and lasts a total of 2 to
3 years. Many Philadelphia chromosome-
positive patients subsequently undergo
allogeneic haematopoietic stem cell
transplantation.
Imatinib (Glivec°, Novartis Pharma), a
tyrosine kinase inhibitor, is authorised in
the European Union for the treatment of
children with Philadelphia chromosome-
positive acute lymphoblastic leukaemia,
in combination with first-line chemo-
therapy. It was already authorised in the
same conditions in adults.
Two non-comparative trials of imati-
nib in children showed a roughly 40%
to 50% improvement in the 5-year sur-
vival rate by comparison with historical
controls who had received chemo-
therapy alone, an average of 10 years
previously. In these two trials imatinib
was introduced either during or imme-
diately after the induction phase.
The impact of imatinib on the sub-
sequent need for transplantation has
not been established, and neither has
the optimal dosage.
Imatinib has potentially serious
adverse effects, which add to those of
chemotherapy. The long-term safety
of imatinib in children is poorly docu-
mented.
Imatinib tablets may be dispersed in
water or apple juice, but the lack of a
specific paediatric form hinders precise
dose adjustment for young children.
Translated from
Rev Prescrire
September 2014; 34 (371): 650-651
imatinib
(G
livec
°)
and acute lymphoblastic leukaemia in children
Prolonged survival in Philadelphia chromosome-positive cases
imatinib
tablets
G
livec
°
• 100 mg
of
imatinib
per tablet
•
400 mg
of
imatinib
per tablet
Novartis Pharma
tyrosine kinase inhibitor
New indication: “
(…) paediatric patients
with newly diagnosed Philadelphia-
chromosome-positive acute lympho-
blastic leukaemia (Ph+ ALL) integrated
with chemotherapy
”.
[EU marketing authorisation, centralised
procedure]
A REAL ADVANCE
In children with acute
lymphoblastic leukae
mia harbouring the
Philadelphia chromo-
some, historical com-
parisons, although representing
relatively weak evidence, suggest
that adding
imatinib
to chemo-
therapy significantly increases
the 5-year overall survival rate. It
remains to be seen whether
imati-
nib
avoids the need for transplan-
tation.
Imatinib
has potentially seri-
ous adverse effects that must be
taken into account, and long-term
safety is inadequately document-
ed.
Imatinib
must continue to be
evaluated in this setting but clearly
represents a therapeutic advance.
The lack of a paediatric form under-
mines the quality of care.
Rev Prescrire
2014; 34 (371): 650-651.
In practice, imatinib should be used
to treat children with Philadelphia
chromosome-positive acute lympho
blastic leukaemia. As this is a rare
condition, all imatinib-treated patients
should be included in a registry.
©Prescrire
In response to our request for infor-
mation, Novartis Pharma provided us
with no documentation on its product.
Abstract
Prescrire Int • February 2015
