This information is provided by the National Institutes of Health (NIH) Genetic and Rare Diseases Information Center (GARD).
Childhood acute lymphoblastic leukemia (ALL) is a type of cancer of the blood and bone marrow, and the most common type of cancer in children. In children with this condition, too many stem cells made by the bone marrow become lymphoblasts, B lymphocytes, or T lymphocytes. These cells do not function normally and have trouble fighting off infections. Signs and symptoms may include fever; easy bruising or bleeding; bone or joint pain; painless lumps in the neck, underarm, stomach, or groin; weakness; fatigue; and/or loss of appetite. Treatment depends on several factors and may include combination chemotherapy, targeted therapy, and/or stem cell transplant.
On August 30 2017, Kymriah became the first gene therapy approved by the FDA. Kymriah is now an option for children and young adults up to 25 years old with B-Cell precursor ALL that has proven resistant to other treatments or has relapsed two or more times.
For more information, visit GARD.