Retrospective Evaluation of CML Patients in the National Compassionate Program

Gruppo Italiano Malattie EMatologiche dell'Adulto38 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Chronic Myeloid Leukemia Philadelphia Positive

Interventions

PonatinibDRUG

Observation of tolerability and safety profiles

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (10)

UO Ematologia con trapianto-Università degli Studi di Bari Aldo Moro

Bari, Italy

IRCCS_AOU San Martino-IST.Clinica Ematologica

Genova, Italy

ASL Le/1 P.O. Vito Fazzi - U.O. di Ematologia ed UTIE

Lecce, Italy

Outcomes

Primary Outcomes

Number of adverse events

General adverse events and grade 3-4 adverse events according to NCI CTCAE version 4.0

Time frame: Maximum of 60 months from treatment start

Secondary Outcomes

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.

Time frame: Maximum of 60 months from treatment start

Number of days from treatment start till response

Time frame: Maximum of 60 months from treatment start

Total number of patients alive

Overall survival

Time frame: 12 months after patient enrollment in the study

Number of patients in event free survival

Event free survival

Time frame: 12 months after patient enrollment in the study

Number of patients in progression free survival

Progression free survival

Time frame: 12 months after patient enrollment in the study

Number of mutations

Both at the beginning and at the end of treatment

Time frame: 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.

Data from ClinicalTrials.gov

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