Abstract
BackgroundThe treatment of acute promyelocytic leukemia (APL) has been revolutionized in the past two decades by the advent of all-trans retinoic acid (ATRA) and arsenic trioxide (ATO). It suggests that non-high-risk APL patients can be cured without chemotherapy. However, ATRA plus chemotherapy is still the standard therapy for the high-risk patients. Central nervous system (CNS) relapse remains a significant cause of treatment failure in high-risk patients. However, increasing the ATO concentration in cerebrospinal fluid (CSF) may reduce CNS relapse in high-risk patients. Mannitol can allow ATO to penetrate the blood-brain barrier (BBB) and reach therapeutically effective levels in the CSF. It is used for the treatment of CNS relapse in patients APL. We compare ATRA-ATO with ATRA-ATO plus chemotherapy in both high-risk and non-high-risk patients with APL.MethodsThis study was designed as a multicenter randomized controlled trial. Patients with APL were randomly assigned into two groups: the ATRA-ATO group (experimental group) and the ATRA-ATO plus chemotherapy group (control group). The experimental group receives therapy with ATRA-ATO for induction, consolidation and maintenance therapy. In the high-risk patients, mannitol will be used with ATO in the consolidation and maintenance therapy. Hydroxyurea will be used in patients who developed leukocytosis in the induction therapy. The control group receives therapy with ATRA-ATO plus chemotherapy for induction and consolidation therapy.DiscussionIn this study, a randomized clinical trial design is described. It aims to compare the efficacy of ATRA-ATO versus ATRA-ATO plus chemotherapy in all-risk patients with APL.Trial registrationChinese Clinical Trials Registry, ID: ChiCTR-IPR-15006821. Registered on 27 July 2015.
Highlights
The treatment of acute promyelocytic leukemia (APL) has been revolutionized in the past two decades by the advent of all-trans retinoic acid (ATRA) and arsenic trioxide (ATO)
Acute promyelocytic leukemia (APL) is a unique subtype of acute myeloid leukemia (AML) which accounts for 10–15% of acute myeloid leukemia
Wang et al [9] showed that a mannitol infusion can cause a transient increase in blood-brain barrier (BBB) permeability to ATO, thereby increasing the ATO concentration in the cerebrospinal fluid (CSF) and curing most patients with Central nervous system (CNS) relapse
Summary
Study hypothesis To prove that ATRA-ATO is not inferior, and may be superior to, ATO-ATRA with conventional chemotherapy, for non-high-risk and for high-risk APL patients. All study participants will sign a written informed consent before participation. All participants will go through a standardized interview process and receive more information about the study and the treatments. The purpose, procedures, potential risks and benefits of the study will be explained thoroughly to the participants. The participants will be able to withdraw from the study at any time without consequence. Inclusion criteria Participants meeting the following criteria will be included: 1. Patients who can complete the entire treatment process 4. Serum total bilirubin ≤ 3.0 mg/dL (≤ 51 μmol/L) 5. Serum creatinine ≤ 3.0 mg/dL (≤ 260 μmol/L) 6. White blood cell (WBC) count at diagnosis < 40 × 109/L
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Free) 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call
