A European observational registry collecting efficacy and safety data in newly diagnosed pediatric Philadelphia-positive (Ph+) Acute Lymphoblastic Leukemia (ALL) patients treated with chemotherapy + imatinib ± hematopoietic stem cell treatment (±HSCT)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Effectiveness study (incl. comparative)
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Observational, multi-center registry

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (L01XE01) imatinib

  imatinib
• Medical condition to be studied:
  Philadelphia positive acute lymphocytic leukaemia

Population studied

• Short description of the study population:
  The study population included male and female pediatric patients (aged 1 to <18 years old) diagnosed with Philadelphia chromosome positive (Ph+) acute lymphoblastic leukemia (ALL) identified from hospital discharge files, clinical records and electronic medical records.; Inclusion Criteria:; 1. Male or female, pediatric patients aged greater than 1 year (≥365 days) and less than 18 years old (<17 years, 365 days) at diagnosis.; 2. Documented, newly diagnosed Philadelphia Chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL).;  Recorded presence of t(9;22)(q34;q11) is required e.g. determined via institutional cytogenetics or FISH and/ or of the presence of BCR-ABL fusion transcript identified by RT-PCR or FISH.; 3. Enrolled into this registry within 6 months of diagnosis or enrolled in a clinical trial within ; 6 months of diagnosis, although no earlier than Jan-2012.; 4. Previously treated or currently on treatment with any chemotherapy regimen + imatinib (of an HA-approved formulation or HA-approved Glivec generic) ± HSCT.; 5. Written informed consent obtained prior to any information being entered into the registry(parent / legal guardian consent, where applicable).;  Assent from a patient enrolled as a minor by parent / legal guardian consent must be obtained wherever possible. Obvious child dissent must be respected.;  A patient enrolled as a minor by parent / legal guardian consent must be re-consented as an adult upon reaching the legal age of maturity during the course of the registry(legal age of maturity defined by local regulations).;  Patients fulfilling the inclusion criteria, but who have died prior to registry opening and without the opportunity to give consent, may still be eligible for inclusion, subject to local requirements regarding the consent process.; The registry participating physician must assess whether there are any third-party agreements limiting the clinical trial patient’s data collection as part of the non-interventional study given that no identification of proprietary treatment information will take place.; Exclusion Criteria:; - There are no exclusion criteria for this non-interventional study.; - Patients may voluntarily withdraw from the registry at any time.</18></18>
• Age groups:
  Infants and toddlers (28 days – 23 months)

  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)
• Special population of interest:
  Other
• Special population of interest, other:
  Patients with Philadelphia chromosome positive (Ph+) acute lymphoblastic leukemia (ALL)
• Estimated number of subjects:
  65

Study design details

• Main study objective:
  The primary objective was to evaluate long-term clinical outcome measured by even-free survival (EFS).
• Outcomes:
  The primary outcomes relate to EFS, which is defined as time from diagnosis to relapse at any site, development of a second malignant neoplasm or death., Main secondary outcomes relate to overall survival (OS), defined as time from diagnosis to death from any cause and Safety, defined as adverse events and serious adverse events in the observation period. Other secondary outcomes relate to hematological remission status, MRD response, duration of complete remission, evaluation of time to transplantation, assessment of growth & development.
• Data analysis plan:
  All data analyses were performed by Novartis personnel and/ or designee. All data ; summaries and analyses have descriptive purposes only. Patient efficacy, safety and ; tolerability outcomes of patients treated with chemotherapy + imatinib were summarized ; overall, by HSCT (yes/ no) and risk group (good risk– induction responder/ poor risk– ; induction non-responder).

Documents

Study results

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