Retrospective Evaluation of CML Patients in the National Compassionate Program

Study Overview
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Brief Summary
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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
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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.

Official Title
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Ponatinib Survey: Retrospective Evaluation of CML Patients in the National Compassionate Program According to the 648/96 Decree

Conditions
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Chronic Myeloid Leukemia, Philadelphia Positive

Intervention / Treatment
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* Drug: Ponatinib


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| CML ph+ patients<br>Chronic myeloid leukemia patients who are philadelphia positive | Drug: Ponatinib<br>* Observation of tolerability and safety profiles<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of adverse events | General adverse events and grade 3-4 adverse events according to NCI CTCAE version 4.0 | Maximum of 60 months from treatment start | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of patients with hematological complete response | Hematological complete response and related responses, such as major cytogenetic response, complete cytogenetic response and major molecular response. | Maximum of 60 months from treatment start | 
| Number of days from treatment start till response |  | Maximum of 60 months from treatment start | 
| Total number of patients alive | Overall survival | 12 months after patient enrollment in the study | 
| Number of patients in event free survival | Event free survival | 12 months after patient enrollment in the study | 
| Number of patients in progression free survival | Progression free survival | 12 months after patient enrollment in the study | 
| Number of mutations | Both at the beginning and at the end of treatment | Maximum of 60 months from treatment start | 
| Number of units of Ponatinib administered per units of time | Chemotherapy dose intensity represents unit dose of chemotherapy administered per unit time. | Maximum of 60 months from treatment start | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Chronic myeloid leukemia, Philadelphia positive, Compassionate use