Retrospective Evaluation of CML Patients in the National Compassionate Program

Terminated

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.

Recruitment & Eligibility

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

Arm && Interventions

Group	Intervention	Description
CML ph+ patients	Ponatinib	Chronic myeloid leukemia patients who are philadelphia positive

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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
Total number of patients alive	12 months after patient enrollment in the study	Overall survival
Number of days from treatment start till response	Maximum of 60 months from treatment start
Number of patients in event free survival	12 months after patient enrollment in the study	Event free survival
Number of patients in progression free survival	12 months after patient enrollment in the study	Progression free survival
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.

Locations (10): ASL Le/1 P.O. Vito Fazzi - U.O. di Ematologia ed UTIE
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Lecce, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico UOC Oncoematologia- Padiglione Marcora 2° piano
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Milano, Italy
Azienda Ospedaliera "S.Gerardo"
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Monza, Italy
Dipartimento Emato-Oncologia A.O."Bianchi-Melacrino-Morelli"
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Reggio Calabria, Italy
UO Ematologia con trapianto-Università degli Studi di Bari Aldo Moro
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Bari, Italy
IRCCS_AOU San Martino-IST.Clinica Ematologica
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Genova, Italy
S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro
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Novara, Italy
Dip. di Scienze Cliniche e Biologiche - Ospedale S. Luigi Gonzaga-Medicina Interna 2 - Orbassano
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Orbassano, Italy
S.C. Ematologia - Fondazione IRCCS Policlinico S. Matteo
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Pavia, Italy
U.O.C. Ematologia - Ospedale S. Eugenio
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Roma, Italy