Overview
Acute lymphoblastic leukemia (ALL), also known as acute lymphocytic leukemia or acute lymphoid leukemia, is a cancer of the blood and bone marrow characterized by the uncontrolled proliferation of immature lymphoid cells (lymphoblasts). These abnormal cells accumulate in the bone marrow, crowding out normal blood cell production, and can spread to the blood, lymph nodes, spleen, liver, central nervous system, and other organs. ALL is the most common childhood cancer, though it also occurs in adults. The disease is classified into B-cell ALL (approximately 85% of pediatric cases) and T-cell ALL based on the lineage of the affected lymphoblasts. Key symptoms result from bone marrow failure and organ infiltration. Patients typically present with fatigue, pallor, and weakness due to anemia; recurrent or severe infections due to neutropenia; and easy bruising, petechiae, or bleeding due to thrombocytopenia. Other common features include bone and joint pain, fever, lymphadenopathy (swollen lymph nodes), hepatosplenomegaly (enlarged liver and spleen), and unexplained weight loss. Central nervous system involvement may cause headaches, vomiting, or cranial nerve palsies. The treatment landscape for ALL has evolved significantly, particularly in pediatric patients where cure rates now exceed 85-90%. Treatment typically involves multi-agent chemotherapy administered in phases: induction (to achieve remission), consolidation/intensification (to eliminate residual disease), and maintenance therapy, often spanning two to three years. CNS-directed therapy (intrathecal chemotherapy and/or cranial radiation) is a critical component. For relapsed or refractory disease, options include targeted therapies such as tyrosine kinase inhibitors for Philadelphia chromosome-positive ALL, bispecific T-cell engager antibodies (blinatumomab), antibody-drug conjugates (inotuzumab ozogamicin), and chimeric antigen receptor (CAR) T-cell therapy. Allogeneic hematopoietic stem cell transplantation remains an important option for high-risk or relapsed patients. Adult ALL carries a less favorable prognosis than pediatric ALL, though outcomes have improved with the adoption of pediatric-inspired treatment protocols.
Also known as:
Multifactorial
Caused by a mix of several genes and environmental factors
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
10 eventsPrincess Maxima Center for Pediatric Oncology — PHASE1, PHASE2
Children's Oncology Group — PHASE1
GUSTAVO SALGUERO — NA
Cairo University — NA
Johns Hopkins University — PHASE2, PHASE3
University of Alabama at Birmingham — NA
National Cancer Institute (NCI) — PHASE2
National Cancer Institute (NCI) — NA
University of Washington — PHASE2
Novartis Pharmaceuticals — PHASE2
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
13 availableXatmep
Treatment of pediatric patients with acute lymphoblastic leukemia (ALL) as a component of a combination chemotherapy maintenance regimen
Leukine
For the acceleration of myeloid reconstitution following autologous peripheral blood progenitor cell (PBPC) or bone marrow transplantation in adult and pediatric patients 2 years of age and older with…
For the acceleration of myeloid reconstitution following autologous peripheral blood progenitor cell (PBPC) or bone marrow transplantation in adult and pediatric patients 2 years of age and older with acute lymphoblastic leukemia (ALL)
Methotrexate
treatment of adults and pediatric patients with acute lymphoblastic leukemia (ALL) as part of a combination chemotherapy maintenance regimen
Lipiodol
lymphography in adult and pediatric patients
Novantrone
in combination with other approved drug(s) is indicated in the initial therapy of acute nonlymphocytic leukemia (ANLL) in adults
Aucatzyl
treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL)
Blincyto
treatment of CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (ALL) in the consolidation phase of multiphase chemotherapy in adult and pediatric patients on…
treatment of CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (ALL) in the consolidation phase of multiphase chemotherapy in adult and pediatric patients one month and older
Rylaze
As a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients 1 month or older who h…
As a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients 1 month or older who have developed hypersensitivity to E. coli-derived asparaginase.
Marqibo
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-leuke…
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.
Erwinase
Treatment of patients with acute lymphoblastic leukemia (ALL) who have developed hypersensitivity to E. coli-derived asparaginase.
Arranon
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 r…
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
Clolar
Treatment of pediatric patients 1 to 21 years old with relapsed or refractory acute lymphoblastic leukemia after at least two prior regimens
Oncaspar; Asparlas
Combination chemotherapy for the treatment of patients with acute lymphoblastic leukemia who are hypersensitive to native forms of L-asparaginase.
Rare Disease Specialist
Rare Disease Specialist
Rare Disease Specialist
Treatment Centers
8 centersArkansas Children's Hospital
📍 Little Rock, Arkansas
Children's Hospital of Orange County
📍 Orange, California
👤 Richard Neibeger, MD
Banner University Medical Center - Tucson
📍 Tucson, Arizona
Cedars Sinai Medical Center
📍 Los Angeles, California
👤 Janssen Research & Development, LLC Clinical Trial
UCSF Benioff Children's Hospital Oakland
📍 Oakland, California
👤 Neeta Thakur, MD, MPH
University of California Davis Comprehensive Cancer Center
📍 Sacramento, California
👤 Ann (Annie) W Silk
Harvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
Financial Resources
7 resourcesBesponsa
Pfizer
Acute Lymphoblastic Leukemia
Travel Grants
No travel grants are currently matched to Acute lymphoblastic leukemia.
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Common questions about Acute lymphoblastic leukemia
What is Acute lymphoblastic leukemia?
Acute lymphoblastic leukemia (ALL), also known as acute lymphocytic leukemia or acute lymphoid leukemia, is a cancer of the blood and bone marrow characterized by the uncontrolled proliferation of immature lymphoid cells (lymphoblasts). These abnormal cells accumulate in the bone marrow, crowding out normal blood cell production, and can spread to the blood, lymph nodes, spleen, liver, central nervous system, and other organs. ALL is the most common childhood cancer, though it also occurs in adults. The disease is classified into B-cell ALL (approximately 85% of pediatric cases) and T-cell ALL
How is Acute lymphoblastic leukemia inherited?
Acute lymphoblastic leukemia follows a multifactorial inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
Are there clinical trials for Acute lymphoblastic leukemia?
Yes — 20 recruiting clinical trials are currently listed for Acute lymphoblastic leukemia on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Acute lymphoblastic leukemia?
25 specialists and care centers treating Acute lymphoblastic leukemia are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Acute lymphoblastic leukemia?
13 patient support programs are currently tracked on UniteRare for Acute lymphoblastic leukemia. See the treatments and support programs sections for copay assistance, eligibility, and contact details.