Acute lymphoblastic leukemia

Making a diagnosis for a genetic or rare disease can often be challenging. Healthcare professionals typically look at a person’s medical history, symptoms, physical exam, and laboratory test results in order to make a diagnosis. The following resources provide information relating to diagnosis and testing for this condition. If you have questions about getting a diagnosis, you should contact a healthcare professional.

FDA-Approved Treatments

• Nelarabine (Brand name: Arranon®) - Manufactured by GlaxoSmithKline
  FDA-approved indication: Treatment of patients with T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma whose disease has not responded to or has relapsed following treatment with at least two chemotherapy regimens
  National Library of Medicine Drug Information Portal
  Medline Plus Health Information
• Inotuzumab ozogamicin (Brand name: Besponsa) - Manufactured by Pfizer, Inc.
  FDA-approved indication: Treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).
  National Library of Medicine Drug Information Portal
• Blinatumomab (Brand name: Blincyto) - Manufactured by Amgen, Inc.
  FDA-approved indication: March 2018 approval for the treatment of B-cell precursor acute lymphoblastic leukemia (ALL) in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1% in adults and children. Previously approved in December 2014 for treatment of Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).
  National Library of Medicine Drug Information Portal
  Medline Plus Health Information
• Clofarabine (Brand name: Clolar®) - Manufactured by Genzyme Corporation
  FDA-approved indication: Treatment of pediatric patients 1 to 21 years old with relapsed or refractory acute lymphoblastic leukemia after at least two prior regimens
  National Library of Medicine Drug Information Portal
  Medline Plus Health Information
• Buffered intrathecal electrolyte/dextrose injection (Brand name: Elliotts B Solution) - Manufactured by QOL Medical, LLC
  FDA-approved indication: For the intrathecal administration of methotrexate and cytarabine for the prevention or treatment of meningeal leukemia and lymphocytic lymphoma.
  National Library of Medicine Drug Information Portal
• Erwinia L-Asparaginase (Brand name: Erwinase) - Manufactured by EUSA Pharma, Inc.
  FDA-approved indication: Treatment of patients with acute lymphoblastic leukemia (ALL) who have developed hypersensitivity to E. Coli-derived asparaginase.
  National Library of Medicine Drug Information Portal
  Medline Plus Health Information
• Imatinib mesylate (Brand name: Gleevec®)
  FDA-approved indication: Treatment of adult patients with relapsed or refractory Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ALL)
  National Library of Medicine Drug Information Portal
• Ponatinib (Brand name: Iclusig) - Manufactured by ARIAD Pharmaceuticals Inc.
  FDA-approved indication: Treatment of adult patients with chronic phase, accelerated phase, or blast phase chronic myeloid leukemia (CML) that is resistant or intolerant to prior tyrosine kinase inhibitor therapy or Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ALL) that is resistant or intolerant to prior tyrosine kinase inhibitor therapy.
  National Library of Medicine Drug Information Portal
• Vincristine sulfate liposome injection (Brand name: Marqibo) - Manufactured by Talon Therapeutics, Inc.
  FDA-approved indication: Treatment of adult patients with Philadelphia chromosome-negative (Ph-) acute lymphoblastic leukemia (ALL) in second or greater relapse or whose disease has progressed following two or more anti-leukemia therapies.
  National Library of Medicine Drug Information Portal
• Pegaspargase (Brand name: Oncaspar®) - Manufactured by Enzon, Inc.
  FDA-approved indication: Combination chemotherapy for the treatment of patients with acute lymphoblastic leukemia who are hypersensitive to native forms of L-asparaginase.
  National Library of Medicine Drug Information Portal
  Medline Plus Health Information
• mercaptopurine oral solution (Brand name: Purixan) - Manufactured by Nova Laboratories Limited
  FDA-approved indication: Treatment of patients with acute lymphoblastic leukemia as part of a combination regimen.
  National Library of Medicine Drug Information Portal
• Dasatinib (Brand name: Sprycel) - Manufactured by Bristol-Myers Squibb Company
  FDA-approved indication: December 2018, dasatinib (Sprycel) was approved for the treatment of pediatric patients 1 year of age and older with Ph+ CML in chronic phase and newly diagnosed Ph+ ALL in combination with chemotherapy. Previously in June 2013, it was approved for treatment of adults with Philadelphia chromosome-positive acute lymphoblastic leukemia with resistance or intolerance to prior therapy.
  National Library of Medicine Drug Information Portal
  Medline Plus Health Information
• Methotrexate oral solution (Brand name: Xatmep) - Manufactured by Silvergate Pharmaceuticals, Inc.
  FDA-approved indication: April 2017, methotrexate oral solution (Xatmep) was approved for the treatment of pediatric patients with acute lymphoblastic leukemia (ALL) as a component of a combination chemotherapy maintenance regimen.
  National Library of Medicine Drug Information Portal

If you need medical advice, you can look for doctors or other healthcare professionals who have experience with this disease. You may find these specialists through advocacy organizations, clinical trials, or articles published in medical journals. You may also want to contact a university or tertiary medical center in your area, because these centers tend to see more complex cases and have the latest technology and treatments.

If you can’t find a specialist in your local area, try contacting national or international specialists. They may be able to refer you to someone they know through conferences or research efforts. Some specialists may be willing to consult with you or your local doctors over the phone or by email if you can't travel to them for care.

You can find more tips in our guide, How to Find a Disease Specialist. We also encourage you to explore the rest of this page to find resources that can help you find specialists.

Research helps us better understand diseases and can lead to advances in diagnosis and treatment. This section provides resources to help you learn about medical research and ways to get involved.

Support and advocacy groups can help you connect with other patients and families, and they can provide valuable services. Many develop patient-centered information and are the driving force behind research for better treatments and possible cures. They can direct you to research, resources, and services. Many organizations also have experts who serve as medical advisors or provide lists of doctors/clinics. Visit the group’s website or contact them to learn about the services they offer. Inclusion on this list is not an endorsement by GARD.

Living with a genetic or rare disease can impact the daily lives of patients and families. These resources can help families navigate various aspects of living with a rare disease.

These resources provide more information about this condition or associated symptoms. The in-depth resources contain medical and scientific language that may be hard to understand. You may want to review these resources with a medical professional.