Gain a deeper understanding of pediatric and young adult Acute Lymphoblastic Leukemia (pALL), its diagnosis, symptoms, treatment options, and how CAR-T cell therapy helps these patients in their cancer journey.

What is Pediatric Acute
Lymphoblastic Leukemia (pALL)?

Pediatric Acute Lymphoblastic Leukemia (pALL) is the most common type of blood cancer diagnosed among children.1 In a child with pALL, the bone marrow makes too many lymphocytes, a type of white blood cell. When this happens, these cells fill up the bone marrow and stop it from making the usual amount of healthy blood cells. Thus, affecting the body’s ability to defend itself and putting the child at increased risk of infection.

“Acute” means that this type of cancer is aggressive in nature and usually gets worse rapidly if it is not treated immediately.


What is B-Cell Pediatric Acute
Lymphoblastic Leukemia (pALL)?

As part of the body’s immune system, B cells are natural defenders of the body. When B cells become cancerous, they can grow out of control and cause a type of blood cancer called B-cell pediatric Acute Lymphoblastic Leukemia (pALL).2

In pediatric Acute Lymphoblastic Leukemia (pALL) patients, abnormal cells in the bone marrow limit the production of red blood cells which carry oxygen, other types of white blood cells, and platelets. As a result, pALL patients may be anemic, more likely to get infections and bruise or bleed easily.3 While many children with pALL recover after their first treatment, some don’t.4

Did you know? According to World Health Organization (WHO), 55,767 cancer cases5 were diagnosed in Southeast Asian children aged 0-14 years old in 2020.


How is Pediatric Acute Lymphoblastic Leukemia (pALL) diagnosed?

To determine if a person has pediatric Acute Lymphoblastic Leukemia (pALL)6, the doctor will ask about the person’s medical history, the family’s medical history, followed by a physical exam to look for possible signs of the disease, such as swollen lymph nodes.

First, a blood test is drawn to check the blood cell counts, liver and kidney function. If the results show a high number of abnormal white blood cells, it could be a sign of pALL. Your care team may then refer you to a hematologist to help guide the next steps of treatment.

To confirm a diagnosis of pALL, a sample from the bone marrow is taken for further testing. Imaging studies, such as X-ray scans, CT scans, MRI, or ultrasound, may also be done for a better understanding of where the leukemia cells have spread.


What are the symptoms of Pediatric Acute Lymphoblastic Leukemia (pALL)?

The lack of healthy blood cells causes most symptoms of pediatric Acute Lymphoblastic Leukemia (pALL). Common Acute Lymphoblastic Leukemia symptoms to look out for include7:

  • Pale skin
  • Feeling tired and breathless
  • Repeated infections over a short time
  • Unusual and frequent bleeding (such as bleeding gums or nosebleeds)
  • High temperature
  • Night sweats
  • Bone and joint pains
  • Swollen lymph nodes
  • Abdominal pain (caused by a swollen liven or spleen)
  • Weight loss
  • Bruises (or small red or purple spots) on the skin

In some cases, the affected cells can spread from the bloodstream into the central nervous system. Thus, causing neurological symptoms (related to the brain and nervous system), including headaches, seizures or fits, blurred vision, and dizziness.


What are the risk factors of Pediatric Acute Lymphoblastic Leukemia (pALL)?

Risk factors refer to anything that increases one’s risk of getting cancer, but they do not determine the diagnosis as Pediatric Acute Lymphoblastic Leukemia (pALL) patients may have few or no known risk factors. According to statistics, the risk for developing pALL is highest in children below 5 years old.19

Factors within your control

  • Exposure to radiation, certain chemotherapy drugs and chemicals (such as benzene)
  • Smoking
  • Certain viral infections
  • Genetic conditions (such as Down syndrome, Klinefelter syndrome, Fanconi Anemia, Bloom syndrome, Ataxia-telangiectasia, Neurofibromatosis, and Li-Fraumeni syndrome)

Factors outside of your control

  • Age
  • Gender
  • Family history

What is refractory or relapsed Pediatric
Acute Lymphoblastic Leukemia (pALL)?

What is refractory or relapsed Pediatric <br/>Acute Lymphoblastic Leukemia (pALL)?

Studies have shown that with the proper treatment, patients with Pediatric Acute Lymphoblastic Leukemia (pALL) have:

  • 98% remission rate after 1 month of therapy9
  • > 80% cure rate9
  • > 90% survival rate (5-year survival rate)10

About 20% of patients with B-cell Pediatric Acute Lymphoblastic Leukemia (pALL) will not have success with initial treatments.11 This means their cancer did not respond to treatment (refractory Acute Lymphoblastic Leukemia).

Some patients who had recovered or seen a decrease in Acute Lymphoblastic Leukemia symptoms later report having a lower number of normal blood cells while also seeing a return of leukemia cells in their bone marrow. This means their cancer has returned (relapsed Acute Lymphoblastic Leukemia).

In the past, the treatment options for patients with relapsed or refractory B-cell Pediatric Acute Lymphoblastic Leukemia (pALL) included chemotherapy, radiation, or stem cell transplant. Since then, scientific advances and medical research have opened doors for new treatments, such as CAR-T cell therapy.

What is refractory or relapsed Pediatric <br/>Acute Lymphoblastic Leukemia (pALL)?

What are the treatment options for Pediatric Acute Lymphoblastic Leukemia (pALL)?

In cancer care, treatment options and recommendations depend on several factors12, including the subtype and classification of Pediatric Acute Lymphoblastic Leukemia (pALL), possible side effects, age, and overall health. It is best to consult with your care team on what is the best journey for your child.



A standard treatment process13 for pALL involves a combination of medication known as R-CHOP. The medicine enters the bloodstream and reaches the whole body, making this treatment helpful for cancers like leukemia.

This is one of the most common ways of treating Pediatric Acute Lymphoblastic Leukemia (pALL), with a recovery rate of 98% for children and 80%-90% for young adults.14 However, it is a long-term treatment option that has intense side effects during the first few months.

  • ~10%-20% of patients experience cancer returning (relapse) after recovering from chemotherapy15, with the treatment becoming less effective over time16


The side effects of chemotherapy depend on the type and dose of drugs given and the length of time they are administered. These may include:

  • Hair loss
  • Mouth sores
  • Loss of appetite
  • Nausea, vomiting, diarrhea
  • Increased risk of infections (from having too few normal white blood cells)
  • Easy bruising or bleeding (from having too few blood platelets)
  • Fatigue (from having too few red blood cells)


Radiation therapy17 uses high-energy radiation to kill cancer cells. While it is not the main treatment used to treat Pediatric Acute Lymphoblastic Leukemia (pALL), it is used to treat leukemia that has spread to the brain and spinal fluid, and is a key step before patients undergo a stem cell transplant.


The possible short-term side effects depend on where the radiation is aimed and can include:

  • Sunburn-like skin changes
  • Hair loss
  • Nausea, vomiting, diarrhea (from radiation to the abdomen)
  • Fatigue
  • Increased risk of infection


Targeted therapy18 uses new drugs that have been developed to target specific parts of cancer cells. They are used instead of or along with chemotherapy.

Targeted drugs such as tyrosine kinase inhibitors (TKIs) help to detect a specific protein, Philadelphia chromosome, that can be found in about 1 out of 4 adult patients with pALL. Thus, targeted therapy with TKI alongside chemo may help some patients achieve complete recovery.


For targeted medication often used to treat pALL, the common side effects include:

  • Diarrhea, nausea, muscle pain, fatigue, and skin rashes
  • Swelling around the eyes, hands, or feet (possibly caused by the drugs’ effects on the heart)
  • Lower red blood cell and platelet counts when treatment starts
  • May slow a child’s growth, especially if used before puberty


Stem cell transplants19 aim to replace damaged bone marrow with healthy cells, which are impaired (due to chemotherapy or radiation) or diseased. The healthy cells replacing the damaged or cancerous ones are obtained from the patient’s bone marrow (autologous transport) or a suitable donor (allogeneic transplant).

There are 2 types of stem cell transplants:

  • Allogeneic transplant, where stem cells come from someone else or a matched donor. This is the preferred type of transplant when treating pALL, and donors are often family members with the same tissue type as you.
  • Autologous transplant, where the stem cells are collected from the patient’s own body and saved.

Both types of transplants help patients create healthy blood cells, red blood cells or platelets, and reduce one’s risk of life-threatening infections, anemia, and bleeding.20 However, a stem cell transplant is an intensive and complex treatment that can cause life-threatening side effects and transplant-related morbidities.


Most short-term side effects21 are from the high doses of chemotherapy or radiation and would go away over time as your child recovers. Common side effects include:

  • Increased risk of infections
  • Low blood cell counts
  • Low blood pressure
  • Shortness of breath, coughing, chest pain, or tightness
  • Fever or chills
  • Hair loss
  • Nausea, vomiting, diarrhea
  • Mouth sores
  • Loss of appetite
  • Fatigue
  • Bleeding

Some side effects may be long-lasting or show up many years later, such as:

  • Growth of another kind of cancer
  • Lung problems
  • Damage to other organs, such as the heart, kidneys, or liver
  • Lack of menstrual periods, which may mean ovary damage and inability to have children (infertility)
  • Vision problems caused by damage to the lens of the eye (cataracts)
  • Damage to joints

Another possible long-term side effect is graft-versus-host disease (GVHD). GVHD can only occur with an allogeneic transplant. It happens when the immune system cells in the donor’s stem cells attack the body. The cells can attack the skin, liver, gastrointestinal (GI) tract, mouth, or other organs and may cause symptoms such as:

  • Skin rashes with itching and blistering
  • Yellowing of the skin and eyes (jaundice)
  • Severe diarrhea and belly cramps
  • Nausea and vomiting
  • Fatigue
  • Muscle aches


Immunotherapy23 enhances one’s immune system to fight cancer. There are different types of immunotherapies that help the immune system in different ways, ranging from immune checkpoint inhibitors that stop attacks on your healthy cells, to helping to find hidden cancer cells and reprogramming immune cells to fight cancer.

Monoclonal antibodies are manufactured antibodies designed to attack specific cells, such as a protein on the surface of leukemia cells.

Both drugs help to treat certain types of B-cell Adolescent and Lymphoblastic Leukemia, typically after chemotherapy has been tried. While some drugs are given as a continuous infusion over 28 days, some are given once a week for 3 or more 4 weeks in a row. Depending on the patient, this process may be repeated for more cycles.

Chimeric Antigen Receptor (CAR) T-cell therapy is a type of adoptive cell immunotherapy that enhances the body’s natural ability to fight cancer with the help of modified T cells.


Before each infusion of monoclonal antibodies, patients will be given medicine to prevent side effects such as:

  • High fever or chills
  • Dizziness or lightheadedness
  • Headache
  • Low blood potassium levels
  • Severe muscle or joint pain
  • Low blood potassium levels
  • Low levels of blood cells (with increased risks of infection, bleeding, and fatigue)
  • Seizures

When it comes to CAR-T cell therapy, all patients need to be monitored carefully after receiving it. CAR-T cell therapy can pose serious side effects ranging from mild to life-threatening. For example, damage to the brain or nervous system (neurological toxicities) resulting in seizures, difficulty speaking and understanding, and loss of balance; and Cytokine Release Syndrome (CRS), a condition that can lead to flu-like symptoms including:

  • Difficulty breathing
  • Fever
  • Chills or shaking chills
  • Severe nausea, vomiting, diarrhea
  • Severe muscle or joint pain
  • Very low blood pressure
  • Dizziness or lightheadedness

How does CAR-T cell therapy work?


CAR-T cell therapy, or Chimeric Antigen Receptor T-cell therapy, is a type of immunotherapy that enhances the body’s natural ability to treat cancer by using modified T-cells.

CAR-T cell therapy involves altering the body’s T-cells, a type of white blood cell found in the immune system, with new receptors. This receptor is called a Chimeric Antigen Receptor, or CAR, and helps to target and stop the spread of cancer cells in the blood.


The CAR protein is called “chimeric” (pronounced ky-MEER-ic) as scientists attach this protein to your T-cells to better detect specific characteristics of cancer cells. In Greek mythology, a “chimera” was an animal with a lion’s head, a goat’s body, and a serpent’s tail. In biology, a chimera is an organism that has a mixture of genetically different cells. Thus, “chimeric” means having parts of various origins.


Antigens are markers on cancer cells that help T-cells recognize them as something to defend against.


The CAR protein, which acts like a cancer-cell tracking device, is added to the modified T-cells. They act as a receptor or a signal because they can now search for the matching antigen on the cancer cells.


These white blood cells are a vital part of the immune system that can find and stop the spread of cells that have been infected or became cancerous. These T-cells are altered to better detect and treat cancer cells.






In order to collect the T cells, the patient's blood is drawn through a process called Leukaphereis. This process takes 3-6 hours in order to extract the T cells from the body.






Patient’s collected T-cells will be reprogrammed into CAR-T cells at a specialized manufacturing facility. The process usually takes 3 to 4 weeks, but timing and manufacturing outcomes can vary.






Before infusion, the physician decides if a short course of chemotherapy is needed to prepare the body. Once the treatment team decides the patient is ready, the patient will receive CAR-T cells through a single infusion that takes less than 30 minutes. At this stage, the increase in CAR- T cells may enhance the patient's immune competence (or their ability to withstand) against cancer cells.






In the short term, regular monitoring to manage side effects is essential. Whether the infusion was received in an inpatient or outpatient setting, it will be necessary to stay close to the treatment center for at least 4 weeks.


In the long term, the treatment team will establish a monitoring plan for ongoing follow-ups. The Food and Drug Administration (FDA) recommends that all patients be followed for 15 years after infusion. The treatment team will offer the patient participation in a long-term registry conducted by the Center for International Blood and Marrow Transplant Research (CIBMTR) for this follow-up. This information is used to help future patients and contributes to understanding the effects of CAR-T cell therapy in the real world outside of clinical trials.

How is CAR-T different<br/> from other therapies?

How is CAR-T different
from other therapies?

CAR-T cell therapy stands out from other cancer therapies because it is an individualized therapy made just for the patient.



Each patient’s infusion is created from the T cells in their own immune system. CAR-T cells may remain active in the body and act as a “living drug” to stop the growth of any new cells that may grow cancerous.20 Long-term data suggests that some patients recovered within months of treatment, meaning all signs and symptoms of their leukemia has disappeared.



Unlike other treatments, CAR-T therapy is designed to be a one-time treatment either with or without hospitalization (and may be done in an in-patient or out-patient setting).In most cases, a short course of chemotherapy is needed to prepare your child’s body for infusion.



CAR-T therapy does not require a donor as it uses one’s own cells for treatment. Thus, it is helpful for patients who may be ineligible for stem cell transplant due to other co-morbidities, while not bearing the risks associated with a transplant. It is considered a widely studied and safe option.

How is CAR-T different<br/> from other therapies?
When should you<br/> consider CAR-T?

When should you
consider CAR-T?

When should you<br/> consider CAR-T?

CAR-T cell therapy might be the next step if the initial treatments have not been working and the cancer has returned. It’s important to seek a second opinion to determine if CAR-T cell therapy is right for your child.

T cell health declines over time and is also affected by additional lines of treatment, such as chemotherapy, which then lowers your child’s overall response rates to CAR-T therapy.

Due to the aggressive nature of the disease, T cells collected in earlier stages can lead to better outcomes and a higher chance for complete recovery.24 T cells can be cryopreserved for up to 2 years.

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  2. A Caregiver’s Guide to Kymriah Therapy, 2019 (11/19, KYM-1223352) – extracted from Novartis resource

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  20. T, J. (2011). Stemcell Transplantation- Types, Risks and Benefits. Journal of Stem Cell Research & Therapy, 01(03). doi:10.4172/2157-7633.1000114

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  23. CAR T Patient & Caregiver Guide, Frankly Speaking About Cancer, Cancer Support Community and Gilda’s Club, Chapter 1 Page 2

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Disclaimer:This is global website for information on CAR-T Cell Therapy and is intended for Patients and Caregivers outside the US. The information on the site is not country specific and may contain information that is outside the approved indication in the country in which you are located.