Study of Low-Dose Cytarabine and Etoposide With or Without All-Trans Retinoic Acid in Older Patients Not Eligible for Intensive Chemotherapy With Acute Myeloid Leukemia and NPM1 Mutatio
- Conditions
- C92.0Acute myeloblastic leukaemia [AML]
- Registration Number
- DRKS00000783
- Lead Sponsor
- niversity of Ulm
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- All
- Target Recruitment
- 144
Patients with confirmed diagnosis of acute myeloid leukemia according to the World Health Organization (WHO) classification (including de novo AML, t-AML and s-AML)
- Presence of NPM1 mutation as assessed in one of the central AMLSG reference laboratories.
- Age > 60 years. There is no upper age limit.
- No prior chemotherapy for leukemia except hydroxyurea to control hyperleukocytosis if needed for up to 10 days during the diagnostic screening phase.
- Signed written informed consent
- Men must give their informed consent that they do not father a baby and must use a latex condom during any sexual contact with women of childbearing potential, even if they have undergone a successful vasectomy. (while on therapy and for 3 month after the last dose of chemotherapy)
- WHO performance status = 3
- Patients not eligible for intensive chemotherapy according to at least one of the following criteria:
- HCT-CI Score >2
- Patient's decision
- age = 75 years
- All other AML subtypes, in particular those AML with other recurrent genetic changes (according to WHO 2008):
o AML with t(8;21)(q22;q22); RUNX1-RUNX1T1
o AML with inv(16)(p13.1q22) or t(16;16)(p13.1;q22); CBFB-MYH11
o AML with t(15;17)(q22;q12); PML-RARA (or other translocations involving RARA)
o AML with t(9;11)(p22;q23); MLLT3-MLL (or other translocations involving MLL)
o AML with t(6;9)(p23;q34); DEK-NUP214
o AML with inv(3)(q21q26.2) or t(3;3)(q21;q26.2); RPN1-EVI1
- No consent for registration, storage and processing of the individual disease-characteristics and course as well as information of the family physician and all other treating physicians about study participation
- Bleeding disorder independent of leukemia
- Uncontrolled infection
- Known positive for HIV, HBV or HCV
- Organ insufficiency (creatinine >1.5x upper normal serum level; bilirubin, AST or ALP >2.5x upper normal serum level, not attributable to AML; heart failure NYHA III/IV; severe obstructive or restrictive ventilation disorder)
- Severe neurological or psychiatric disorder interfering with ability of giving an informed consent
- Patients with a currently active second malignancy other than non-melanoma skin cancers. Patients are not considered to have a currently active malignancy if they have completed therapy and are considered by their physician to be at less than 30% risk of relapse within one year.
Study & Design
- Study Type
- interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method overall survival over 2 years
- Secondary Outcome Measures
Name Time Method - Rate of Complete remission (CR)<br>- cumulative incidence of relapse (CIR)<br>- cumulative incidence of death in complete remission (CID)<br>- event-free survival<br>;<br>Safety Endpoints:<br>- Rate of early deaths/ hypoplastic deaths<br>- Type, frequency, severity, timing and relatedness of adverse events (AEs) and laboratory abnormalities observed during different treatment cycles<br>- Incidence of infection after each treatment cycle<br>- Duration of neutropenia after each treatment cycle<br>- Duration of thrombocytopenia after each treatment cycle<br>- Duration of hospitalization after each treatment cycle<br>;<br>Quality of life (assessed by the EORTC Quality of Life Core Questionnaire (QLQ-C30), supplemented by information on self-assessed concomitant diseases, late treatment effects, and demographics