A review study to evaluate mannitol-assisted prophylaxis and treatment for acute promyelocytic leukemia

ISRCTN	ISRCTN94954912
DOI	https://doi.org/10.1186/ISRCTN94954912
Secondary identifying numbers	97-01-CHN

Submission date: 14/12/2013
Registration date: 20/01/2014
Last edited: 26/06/2014

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Cancer

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Plain English summary of protocol

Background and study aims
In acute promyelocytic leukemia (APL), central nervous system (CNS) relapse occurs due to a lack of sufficient medication in the brain. The aim of our study is to enrol medium to high risk APL patients and patients with APL CNS relapse and to study whether mannitol-assisted prophylaxis (protective treatment) helps drugs penetrate the blood-brain barrier, thereby increasing the amount of the drugs in the CNS.

Who can participate?
Any APL patients (all age groups) at risk of CNS relapse or diagnosed with CNS relapse.

What does the study involve?
Our mannitol-assisted treatment strategy includes intravenous infusion (i.e., administered into a vein) of mannitol and arsenic trioxide (ATO). Patients at risk of CNS relapse will receive prophylaxis of mannitol and ATO; patients who have been diagnosed with CNS relapse will receive intrathecal chemotherapy (i.e., administered into the spine) plus mannitol and ATO. Long-term follow-up of the patients will be carried out.

What are the possible benefits and risks of participating?
Benefits are expected for the patients who will receive mannitol-assisted prophylaxis or treatment. Mannitol should help the ATO cross the blood-brain barrier, thereby increasing the amount of the drug in the CNS. The main risk of giving mannitol is to decrease the cerebral pressure (the pressure inside the skull). This could be prevented by letting the patients lie down for at least 10 hours during and after treatment.

Where is the study run from?
The study was set up at the First Affiliated Hospital of Harbin Medical University (China).

When is the study starting and how long is it expected to run for?
This study started in 1998 and is expected to run until 2018.

Who is funding the study?
China National Natural Science Foundation and China 863 Projects Foundation.

Who is the main contact?
Professor Jin Zhou, jinzhouh85@163.com
Professor Hong Wang, wh557@yahoo.com

Contact information

Dr Jin Zhou
Scientific

First Affiliated Hospital of Harbin Medical University
Department of Hematology
Youzheng Street
Nangang District
Harbin
150001
China

Email	jinzhouh85@163.com

Study information

Study design	Retrospective study of a treatment's long-term outcome
Primary study design	Observational
Secondary study design	Cohort study
Study setting(s)	Hospital
Study type	Treatment
Participant information sheet	Not available in web format, please use the contact details below to request a patient information sheet
Scientific title	Retrospective study on mannitol-assisted prophylaxis and treatment for acute promyelocytic leukemia
Study objectives	It was hypothesized that mannitol could help drugs enter the blood brain barrier (BBB). Thereby it could improve the clinical outcome of acute promyelocytic leukemia (APL) patients during prophylaxis and treatment.
Ethics approval(s)	Harbin Medical University Ethics Committee, 16/10/1997, ref: HM970018
Health condition(s) or problem(s) studied	APL CNS relapse
Intervention	The study involved two groups of APL patients receiving different mannitol-assisted regimens: 1. Patients with CNS relapse received intrathecal chemotherapy plus mannitol-assisted arsenic trioxide (ATO) 2. Patients at risk of CNS relapse received mannitol-assisted ATO prophylaxis
Intervention type	Drug
Pharmaceutical study type(s)
Phase	Not Applicable
Drug / device / biological / vaccine name(s)	Mannitol, arsenic trioxide
Primary outcome measure	1. Disease-free survival 2. Overall survival
Secondary outcome measures	1. Cerebrospinal fluid (CSF) ATO concentrations 2. CSF tests of APL burden 3. Drug side effects evaluation
Overall study start date	01/01/1998
Completion date	31/12/2018

Eligibility

Participant type(s)	Patient
Age group	Other
Sex	Both
Target number of participants	100
Key inclusion criteria	1. Any age APL patients, with either risks of central nervous system (CNS) relapse or already diagnosed with CNS relapse 2. Patients agreed to receive the prophylaxis or treatment
Key exclusion criteria	1. Previous history of severe cardiovascular disease (coronary arterial disease, stroke, etc) 2. Severe chronic disease with poor prognosis (liver disease, kidney disease, etc) 3. Illegal drug use or chronic alcoholism 4. Physical limitations, mental or intellectual disabilities 5. Any condition that may affect the development of this trial
Date of first enrolment	01/01/1998
Date of final enrolment	31/12/2018

Locations

Countries of recruitment

China

Study participating centre

First Affiliated Hospital of Harbin Medical University

Harbin
150001
China

Sponsor information

First Affiliated Hospital of Harbin Medical University (China)
Hospital/treatment centre

c/o Dr Jin Zhou
Department of Hematology
Youzheng Street
Nangang District
Harbin
150001
China

Email	jinzhouh85@163.com
"ROR"	https://ror.org/05vy2sc54

Funders

Funder type

Government

China National Natural Science Foundation (China), No. 81070439

No information available

China 863 Projects Foundation (China), No. 2012AA020903

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
Publication and dissemination plan	Not provided at time of registration
IPD sharing plan

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Results article	results	18/09/2014		Yes	No