Chronic Myeloid Leukemia, Philadelphia Positive
Conditions
Keywords
Chronic myeloid leukemia, Philadelphia positive, Compassionate use
Brief summary
This observational study aims at assessing the tolerability and safety profiles of Ponatinib, a drug used for Chronic Myeloid Leukemia patients who are Philadelphia positive. This drug is used during the chronic phase of the disease, according to the Italian national compassionate law 648/96.
Detailed description
This observational study aims at assessing the tolerability and safety profiles of Ponatinib, a drug used for Chronic Myeloid Leukemia patients who are Philadelphia positive. This drug is used during the chronic phase of the disease, according to the Italian national compassionate law 648/96.
Interventions
DRUGPonatinib
Observation of tolerability and safety profiles
Sponsors
Gruppo Italiano Malattie EMatologiche dell'Adulto
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* CML Ph+ patients in chronic phase * 18 years old or older * Patients have received either Dasatinib or Nilotinib and resulted resistant or not tolerant to the drugs or have developed the T3151 mutation * Patients have started Ponatinib at least 12 months before registration * Informed consent signed at registration
Exclusion criteria
* CML Ph+ patients in accelerated or blast phase with acute lymphoblastic leukemia
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of adverse events | Maximum of 60 months from treatment start | General adverse events and grade 3-4 adverse events according to NCI CTCAE version 4.0 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of days from treatment start till response | Maximum of 60 months from treatment start | — |
| Total number of patients alive | 12 months after patient enrollment in the study | Overall survival |
| Number of patients in event free survival | 12 months after patient enrollment in the study | Event free survival |
| Number of patients with hematological complete response | Maximum of 60 months from treatment start | Hematological complete response and related responses, such as major cytogenetic response, complete cytogenetic response and major molecular response. |
| Number of mutations | Maximum of 60 months from treatment start | Both at the beginning and at the end of treatment |
| Number of units of Ponatinib administered per units of time | Maximum of 60 months from treatment start | Chemotherapy dose intensity represents unit dose of chemotherapy administered per unit time. |
| Number of patients in progression free survival | 12 months after patient enrollment in the study | Progression free survival |
Countries
Italy
Outcome results
None listed