title: Management of CML in the Pediatric Age Group: Imatinib Mesylate or SCT.
abstract: 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 imatinib mesylate 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 imatinib mesylate at a dose of 340mg÷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 eventfree 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 imatinib arm, while adverse events were more severe and common in the SCT group (55% had GVHD and 78% had infection).
  
  
  CONCLUSIONS
  Imatinib mesylate has a superior OS and EFS than SCT in children. It is generally safe and well tolerated. Imatinib mesylate should be the 1st line treatment of pediatric patients with CML in the chronic phase.
  
  
  BACKGROUND
  CML- Imatinib- SCT- Pediatrics.
authors.affiliation: The Department of Pediatrics, Faculty of Medicine, Ain Shams University.
authors.forename: Mohsen S
authors.initials: MS
authors.lastname: El-Alfy
authors.forename: Alaa M
authors.initials: AM
authors.lastname: Al-Haddad
authors.forename: Ahmed A
authors.initials: AA
authors.lastname: Hamed