Registration of Children With CML and Treatment With Imatinib

Protocol for Standardized Diagnostic Procedures, Registration, and Treatment Recommendations in Children and Adolescents With Philadelphia Chromosome-positive Chronic Myeloid Leukemia (CML)

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.

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

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