Acute Promyelocytic Leukemia
Conditions
Keywords
Acute promyelocytic leukemia, Adults, Arsenic trioxide
Brief summary
The therapeutic advantage of the association of ATRA + Arsenic Trioxide is more favorable and manageable as compared to ATRA + chemotherapy. Nevertheless, at present, there is not enough information on the incidence of long-term side effects. This study, as well as other similar studies conducted around Europe, will focus on following patients treated with this therapy on a long-term basis. Once all studies in Europe will be concluded, all data will be analyzed together.
Detailed description
Considering the clear therapeutic advantage associated with ATRA+ATO combination therapy and the more favorable and overall manageable safety profile compared to ATRA+chemotherapy, the benefits of the combination in the first-line indication do appear to overweigh the risks. However, no information regarding the actual adverse event (AE) incidence and the long-term toxicity of ATRA+ATO is available at present and therefore, as a postmarketing commitment, TEVA (the Company holding the Marketing Authorisation of Trisenox® (Arsenic trioxide)) is setting up a long-term safety cohort study of Trisenox in newly diagnosed low- to intermediate-risk APL patients retrospectively analyzing data from APL disease registries all around Europe. As a result, this observational study is part of the retrospective PASS Study (A Post-Authorisation Long-Term Retrospective Safety Cohort Study of Arsenic Trioxide in First Line Low-to Intermediate-Risk Acute Promyelocytic Leukaemia Patients) that will use data from multinational APL disease registries in Europe. The present study will focus on Italian patients.
Interventions
DRUGArsenic Trioxide
This is an observational study. Patients who have received or are receiving all trans retinoic acid (ATRA) + Arsenic Trioxide will be followed and analyzed.
Sponsors
Gruppo Italiano Malattie EMatologiche dell'Adulto
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* APL diagnosis based on cytological criteria and confirmed by the presence of the (15;17) translocation and/or the presence of the PML/RARA rearrangement (with the determination of the breakpoint subtype). * Newly diagnosed low- to intermediate-risk APL (white blood cells \[WBC\] count ≤10x103/µL) * First line treatment with ATRA+ATO * Aged 18 years or above * Signed informed consent, if applicable
Exclusion criteria
* High risk APL (WBC count \> 10x103/µL) * APL relapse
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of grade III/IV (Common Terminology Criteria for Adverse Events (CTCAE) v4.03) adverse events of special interest (AESI). | At a maximum of 5 years from study entry | AESI are, among others: differentiation syndrome, creatinine, bilirubin, neurotoxicity, aspartate amino transferase/alanine amino transferase (ASAT/ALAT) ratio, haemorrhage, sepsis, QTc prolongation, cardiac events including congestive heart failure (CHF). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of unexpected serious adverse events (SAEs). | At a maximum of 5 years from study entry | Including grading and relationship as documented in the study. |
| Number of patients developing secondary malignancies. | At a maximum of 5 years from study entry | — |
| Number of patients developing therapy-related myelodysplastic syndrome (tMDS). | At a maximum of 5 years from study entry | — |
| Number of patients developing acute myeloid leukemia (tAML). | At a maximum of 5 years from study entry | — |
| Number of patients who die. | At a maximum of 5 years from study entry | — |
Countries
Italy
Contacts
Primary ContactPaola Fazi
Backup ContactEnrico Crea
Outcome results
None listed