Patient case: 4-year-old female with leukemia; genetic feature: ABL1

Clinical trial: Registration of Children With CML and Treatment With Imatinib

Summary: Newly diagnosed pediatric patients (age < 19 years) with bcr-abl-positive CML will be treated with imatinib. Serial monitoring of treatment response is performed in one month intervals during the first three months of treatment and in three months intervals thereafter. Patients with non-response, poor response (either molecular, cytogenetic, or hematologic non-/poor response) or progress of the disease while under imatinib treatment will stop imatinib and undergo stem cell transplantation. All responders to imatinib treatment with an HLA matched donor will undergo stem cell transplantation not later than 2 years after diagnosis.

Detailed description: Indication:
  Newly diagnosed pediatric patients with bcr-abl-positive CML.
  Design:
  Multicenter, non-randomized, open, prospective clinical trial.
  Objectives:
  Primary:
  - assessment of antileukemic activity of imatinib in children and adolescents with Philadelphia chromosome-positive chronic myeloid leukemia
  Secondary:
  -  assessment of the time-to event-efficacy variables
  -  correlation of the quality of haematological, cytogenetical and molecular remission in children and adolescents with CML on ongoing imatinib therapy with survival
  -  safety of imatinib
  Endpoints:
  Primary:
  - rate of haematological, cytogenetical and molecular remissions
  Secondary:
  -  time to progression
  -  duration of chronic phase
  -  time to loss of response
  -  overall survival
  -  assessment of treatment-related toxicities in children and adolescents.
  Inclusion criteria:
  Newly diagnosed Ph+ or bcr-abl-positive CML- Male and female patients aged 0 to 18 years- Written informed consent
  Exclusion criteria:
  -  CML without bcr-abl rearrangement detectable by PCR
  -  Pretreatment with Interferon alpha or any other cytostatic drug with the exception of hydroxyurea or anagrelide (Note: anagrelide is not approved in Germany for treatment of CML). However, these patients may be registered as observational patients.
  -  Any other severe underlying disease beside CML.
  -  Age > 18 years
  -  Pregnant or lactating women
  -  Subjects unlikely to comply with the requirements of the protocol
  Number of patients to be enrolled: 150
  Recruitment period: 5 years
  Treatment period: 2 years
  Planned start of study: 2007
  Planned end of study: 2013

Eligibility: Inclusion Criteria:
  -  Newly diagnosed Ph+ or bcr-abl-positive CML
  -  Male and female patients aged 0 to 18 years
  -  Written informed consent
  Exclusion Criteria:
  -  CML without bcr-abl rearrangement detectable by PCR
  -  Pretreatment with Interferon alpha or any other cytostatic drug with the exception of hydroxyurea or anagrelide (Note: anagrelide is not approved in Germany for treatment of CML). However, these patients may be registered as observational patients.
  -  Any other severe underlying disease beside CML.
  -  Age > 18 years- Pregnant or lactating women
  -  Subjects unlikely to comply with the requirements of the protocol

Relevance to patient case: definitely relevant