Management of CML in the pediatric age group : imatinib mesylate or SCT

Abstract EN

Background: Management of CML has changed markedly since the introduction of tyrosine kinase inhibitors (TKIs).

However stem cell transplantation (SCT) remains a valid therapeutic modality especially in developing countries due to its relatively lower cost.

We aim to compare between imagine maculate and SCT as regard outcome in CML in the pediatric age group.

Methods : Forty-eight patients with newly diagnosed CML in the chronic phase, aged 3 to 18 years were enrolled in this prospective study.

Patients without a matched donor (Group I ; N=30) were assigned to receive imagine maculate at a dose of 340 mg / m2 / day, while patients with a fully matched related donor (Group II ; N = 18), were offered SCT.

Response (hematologic, cytogenetic and molecular) side effects and survival were analyzed.

Results : complete hematologic response was obtained in 97 % of the patients in group I and 94 % in group II.

Major cytogenetic response (CyR) was obtained in 80 % of patients in group I and 100% in group II.

Complete CyR was 57% in group I and 64% in group II.

Major molecular response (MMR) was 36 % in group I and 50 % in group II with no significant difference between both groups.

Six years overall survival (OS) was 87 % in the 1st group and 61 % in the 2nd group (p < 0.01), while event free survival (EFS) was 66 % in the 1st group and 50 % in the 2nd group with a highly significant difference between both groups (p < 0.01).

Side effects were generally rare and mild in the imagine arm, while adverse events were more severe and common in the SCT group (55 % had GVHD and 78 % had infection).

Conclusion : imagine maculate has a superior OS and EFS than SCT in children.

It is generally safe and well tolerated.

Imagine maculate should be the 1st line treatment of pediatric patients with CML in the chronic phase.