This study looks at acute promyelocytic leukemia (APL) in children and adolescents. APL is a blood cancer that starts in very early white blood cells. The purpose of the study is to learn how well treatment works and how safe it is for young people with newly diagnosed APL. The treatment uses arsenic trioxide together with tretinoin, and in some patients with higher-risk disease it also includes gemtuzumab ozogamicin. Other medicines listed in the study materials include cytarabine, methotrexate, and methylprednisolone, which may be used as part of the treatment plan or support care. Some medicines are given by mouth and others are given into a vein or into the fluid around the spine, depending on the treatment step.
The study follows a treatment course made up of several phases. At first, treatment is given to bring the leukemia under control. After that, more treatment is given over time to help keep the disease from returning. Some children may receive only the main drug combination, while others may receive an added medicine based on the type of APL and the level of risk. During the study, doctors watch for side effects and monitor how the disease responds to treatment.
The study is designed for newly diagnosed APL in children and adolescents and compares treatment approaches for different risk groups. The study also examines whether the treatment causes any serious problems and how long children stay in the hospital during therapy.
Who Can Join the Study?
Have a new diagnosis of acute promyelocytic leukemia (APL), confirmed by finding the PML/RARα fusion gene in the leukemia cells. A fusion gene is an abnormal joining of two genes.
Be younger than 18 years old.
Have written informed consent from a parent or legal guardian. Informed consent means permission given after the study has been explained.
If the participant is female and can become pregnant, have a negative pregnancy test using a blood test called beta-HCG.
If the participant can have children, be willing to avoid becoming pregnant or causing a pregnancy during the study and for 3 months after the last dose of the study medicine.
Be willing to work with the study doctor on the most suitable way to prevent pregnancy during the study period.
Who Cannot Join the Study?
If the diagnosis of APL is later found to be incorrect because the PML/RARα rearrangement is not present, the patient cannot stay in the study. This rearrangement is a specific change in the leukemia cells that confirms this type of leukemia.
Significant liver dysfunction, meaning the liver is not working well enough, including:
Bilirubin higher than 3 mg/dL. Bilirubin is a substance made when the body breaks down red blood cells.
ALT or AST higher than 5 times the normal value. These are liver enzymes, which are proteins that can rise when the liver is injured or inflamed.
Kidney function that is too poor, shown by creatinine higher than 2 times the normal value for age. Creatinine is a waste product that helps show how well the kidneys are working.
Heart rhythm problems or other major heart findings, including:
Congenital long QT syndrome, a condition present from birth that affects the heart’s electrical recovery time.
A history of or current ventricular or atrial tachyarrhythmia, which means an abnormally fast heart rhythm from the lower or upper chambers of the heart.
Resting bradycardia lower than 50 beats per minute. Bradycardia means a slow heart rate.
QTc longer than 450 msec on the screening ECG. QTc is a measure of the heart’s electrical timing, and an ECG is a test that records the heart’s activity.
L-FEV lower than 50% or LV-FS lower than 28%. These are heart function measurements that show how well the heart pumps.
Neuropathy, which means nerve damage that can cause pain, numbness, tingling, or weakness.
A concurrent active malignancy, meaning another cancer is present at the same time.
Uncontrolled life-threatening infections, meaning a severe infection that is not being kept under control.
Being pregnant or breastfeeding.
Having already received a different treatment before entering the study, because APL was not suspected at first or because ATRA and/or ATO were not available. ATRA and ATO are study medicines used in this trial.
Aracytin is a brand name for cytarabine. In this trial, it is given by injection into the fluid around the brain and spinal cord to help treat leukemia cells that may be hiding in the central nervous system.
Cytarabine Accord is another brand of cytarabine. It is also given into the spinal fluid and is used to help control leukemia cells in the brain and spinal cord area.
Depo-Medrol is a brand name for methylprednisolone acetate. It is given into the spinal fluid as part of the treatment plan, likely to help reduce inflammation and support the intrathecal therapy used in the study.
Methotrexate is a medicine used to treat cancer. In this trial it is given into the spinal fluid to help prevent or treat leukemia cells in the central nervous system.
Arsenic trioxide is one of the main study medicines. It is given through a vein and helps kill leukemia cells. It is a key part of treatment for acute promyelocytic leukemia in this study.
Vesanoid is a brand name for tretinoin. It is taken by mouth and helps leukemia cells mature into more normal blood cells, which is an important part of treating acute promyelocytic leukemia.
Solu-Medrol is a brand name for methylprednisolone sodium succinate. It is given into the spinal fluid in this trial as part of the treatment approach for leukemia involving the central nervous system.
Mylotarg is a brand name for gemtuzumab ozogamicin. It is given through a vein and is used in the higher-risk group of the study to target leukemia cells more directly.
Acute promyelocytic leukemia – Acute promyelocytic leukemia is a fast-growing type of blood cancer that starts in immature white blood cells called promyelocytes. It develops when these abnormal cells multiply in the bone marrow and crowd out normal blood cells. As the disease progresses, the number of abnormal cells rises and the blood and bone marrow become increasingly filled with these cells. This can lead to worsening bone marrow failure and spread of leukemia cells in the blood. In children and adolescents, it follows the same basic pattern of rapid cell buildup and progression.
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