This section provides an overview of the primary treatment modalities (types of treatment) for children and young people with cancer.
The primary modalities are used:
- Cytotoxic Chemotherapy
- Surgery
- Radiotherapy (including Proton Radiotherapy)
- Biological Therapies (including targeted therapies and immunotherapy
- Bisphosphonates
- Stem Cell Transplant (HSCT)
Combinations of these modalities are often used in treatment.
A child or young person’s treatment plan may be described as a schedule, regimen, block, course or protocol — all terms for outlining what treatment is planned.
Cytotoxic chemotherapy
‘Cyto’ means cells and ‘toxic’ means harmful/kills therefore cytotoxic chemotherapy is the name given to drugs which destroy rapidly dividing cells including cancer cells, but unfortunately also healthy cells such as hair follicles, blood cells, and gut mucosa.
There are over 100 different chemotherapy medicines which can roughly be grouped into 6 types:
- Alkylating agents
- Antimetabolites
- Topoisomerase inhibitors
- Anti-tumour antibiotics
- Mitotic inhibitors
- Corticosteroids
- Cell cycle specific – act on cells during particular phases of cell cycle
- Cell cycle non-specific – act on cells regardless of phase
- Neo-adjuvant – used before any other modality to shrink cancer
- Adjuvant – used after another modality to “mop up” remaining cancer cells
(Devita, Lawrence and Rosenberg, 2021)
- Pre-administration tests checked prior to start of treatment (baseline) and each cycle
- Full blood count (FBC) check platelets, haemoglobin and neutrophils are at an appropriate level
- Glomerular Filtration rate (GFR) and urea & electrolytes (U&E) indicate renal function as some chemotherapy can be renal toxic
- Liver function tests (LFT) chemotherapy is metabolised in the liver and can lead to damage
- Echocardiogram (ECHO) some chemotherapy can affect the heart
- Audiology some chemotherapy can affect hearing
Any changes from baseline may require dose reductions or omissions, or administration of preventative medications.
- IV Fluids – some chemotherapies can affect organs such as bladder or kidneys and require fluids to flush them out
- Antiemetics - administered before, during and after administration of chemotherapy
- Photobiomodulation (PBM) Therapy
Surgery
Surgery can be used in various ways throughout CYP’s treatment.
If the primary tumour is confined to one area and can be safely removed, surgery may be undertaken with curative intent. The resected specimen is examined to confirm clear margins and ensure complete excision.
Surgery is often coordinated with chemotherapy. For example, in osteosarcoma, patients usually receive chemotherapy prior to surgery, followed by resection and then further chemotherapy.
Neoadjuvant therapy refers to chemotherapy given before surgery to shrink the tumour and improve the chances of complete removal.
Adjuvant therapy refers to chemotherapy and/or radiotherapy given after surgery to reduce the risk of recurrence by treating any remaining microscopic disease.
Reconstructive surgery can be offered of the resection is extensive or disfiguring
Advances in prosthetic design and function mean that limb-sparing surgery is now an option for many patients. Expandable prostheses can also be used in children, allowing adjustment as they grow.
Surgery may also support other treatments. For example, some chemotherapy regimens require insertion of a central venous catheter, which remains in place for the duration of therapy.
In advanced disease, tumours may cause pain or obstruct vital structures. In these cases, surgery can be undertaken to reduce tumour bulk and relieve symptoms.
If a patient has a growth or polyp suspected to be pre-malignant, surgery may be undertaken to remove the lesion along with a margin of surrounding tissue as a preventative measure.
Radiotherapy (Including proton beam)
Radiotherapy uses high-energy radiation to destroy cancer cells and is effective in many childhood and TYA cancers, though it carries short- and long-term risks. It may be given alone or with chemotherapy, before or after surgery, and is also used in palliative and emergency settings to relieve symptoms.
Sometimes CYP will have a balloon inserted into their abdomen to move their bowel and essential organs to the side prior to abdominal radiotherapy to reduce risk of the radiotherapy beam damaging tissue and organs unnecessarily.
Proton beam therapy (PBT) is a form of radiotherapy which can limit some of the late effects of radiotherapy. PBT differs to standard radiotherapy in that it uses proton particles rather than photon x-rays. There is a rapid dose fall off with protons; therefore, the dose is minimised to healthy tissue beyond the tumour.
Biological Therapies (including targeted therapies and immunotherapy)
Immunotherapy treatments use patients’ immune response to recognise and target cancer cells. A therapeutic response is caused by altering the patients’ response to cancer cells (Dobosz & Dzieciątkowski, 2019). They can be used alone or alongside other treatments and work differently to chemotherapy which indiscriminately attacks all fast-growing cells (Shuel, 2022). Immunotherapy is a fine balance between generating a sufficient immune response to attack cancer cells and avoiding severe immune-related adverse reactions.
Types of immunotherapies include:
These are artificial antibodies designed to bind to specific antigens on the surface of the cancer cell. Some block growth signals within the cell to slow or prevent tumour growth, some mark cancer cells so the immune system recognises them as targets, and others can deliver toxic agents directly to cancer cells or stimulate the immune system’s ability to attack cancer cells. Examples of MABs include rituximab used for Burkitt or B-cell non-Hodgkin lymphoma, dinutuximab (anti-GD2) used for neuroblastoma, and blinatumomab used for relapsed/refractory B-cell ALL.
Are a type of antibody-based immunotherapy which redirect patients’ own T-cells to recognise and destroy cancer cells. BiTEs can bind two targets at once: a tumour-associated antigen on the cancer cell, and the CD3 receptor on T-cells. They can trigger T-cell activation and release cytotoxic molecules by bringing T-cells in close proximity with tumour cells, which results in targeted destruction of tumour cells (Dewaele & Fernandes, 2024). BiTEs have shown promising effectiveness in haematological cancers and are now being increasingly explored as potential treatments for solid tumours (Dewaele & Fernandes, 2024).
Are a type of MAB that block inhibitory immune pathways which normally act as ‘brakes’ on the immune system and reactivate T cells to enable them to recognise and destroy cancer cells. Examples of check point inhibitors include nivolumab used in relapsed/refractory Hodgkin lymphoma and durvalumab used in clinical trial for paediatric solid tumours.
These vaccines are designed to stimulate the immune system to recognise tumour cells as harmful. The vaccine introduces tumour antigens and T cells are activated and learn to identify the antigens on tumour cells, which then destroy cancer cells. An example of a cancer vaccine is the human papillomavirus (HPV) vaccine which can prevent numerous cancers including cervical, some head and neck, as well as genital warts. In the UK, it is currently given to children aged 12-13. Research is currently investigating vaccines for neuroblastoma, brain tumours, Wilms, and sarcomas.
Chimeric Antigen Receptor T-cell therapy is used for relapsed or refractory blood cancers (Yang et al. 2023). It uses patient’s own T cells that have been extracted and genetically modified to recognise and destroy cancer cells. Chimeric Antigen Receptor is an artificial receptor created specifically for that cancer (Dobosz & Dzieciątkowski, 2019). Once the T-cells have been modified, they are reinfused to the patient. CAR-T therapy can cause some significant toxicities, including Cytokine release syndrome (CRS), which occurs due to rapid immune activation. Symptoms of CRS include fever, hypotension, and organ dysfunction which highlight the importance of close monitoring and early intervention (Zhang et al., 2023). CAR-T therapy can also cause neurotoxicity with symptoms including confusion, tremors, or seizures. An early warning sign of this is a deterioration in handwriting (Karschnia et al., 2019). Management of toxicities include corticosteroids alongside supportive care.
Are used to stimulate or strengthen the body’s immune system so it can recognise and destroy cancer cells. Examples of cytokine therapies include interleukin which stimulates the growth of T cells and natural killer cells and used in melanoma and renal cell carcinoma treatments, and interferon which can slow tumour growth and was previously used in melanoma and some blood cancers.
This type of immunotherapy uses viruses to infect and destroy cancer cells and can be naturally occurring or genetically modified. Oncolytic virus therapy (OVT) targets specific receptors on cancer cells, causing them to act as hosts for viral replication, before being destroyed (Marayati, Quinn & Beierle, 2019). The benefits of OVT are that it uses a virus’s natural ability to lyse cancer cells and can stimulate a cytotoxic immune response. Also, direct intratumoral injection allows for destruction of local malignant cells while minimising systemic side effects (Marayati, Quinn & Beierle, 2019).
Bisphosphonates
This group of medicines help to strengthen bones and reduces problems caused by cancer or its treatment. They slow down osteoclast resorption and are used to reduce fractures, bone pain, and high calcium levels cause by some cancers or their treatments. Common examples include zoledronic acid and pamidronate, which are usually administered intravenously.
Stem Cell Transplant (HSCT)
Stem cell transplant (also called haematopoietic stem cell transplantation, HSCT) is an intensive but potentially curative treatment used in children and young people with high-risk caners, blood disorders, and immune conditions when standard therapies are insufficient. High-dose chemotherapy is used to destroy diseased bone marrow followed by an infusion of healthy stem cells from the patient’s own extracted cells or from a matched donor. This allows for the bone marrow and immune system to recover. HSCT has been shown to improve survival in paediatric patients with high-risk leukaemias and inherited immune deficiencies, with research reporting high overall survival rates when donor matching and supportive care are optimised (Shyr, Davis & Bertaina, 2023). However, the procedure does carry risks, including infections, graft-versus-host disease, and others, so it is delivered by specialist teams in carefully monitored settings.
References
DeVita, V.T., Lawrence, T.S. and Rosenberg, S.A. (eds.), 2021. Cancer: Principles and Practice of Oncology. 12th ed. Philadelphia: Wolters Kluwer.
Dewaele, L. and Fernandes, R.A. (2024). Bispecific T-cell engagers for the recruitment of T cells in solid tumors: a literature review. Immunotherapy Advances, [online] 5(1). doi:https://doi.org/10.1093/immadv/ltae005.
Dobosz, P. & Dzieciątkowski, T., (2019). The intriguing history of cancer immunotherapy. Frontiers in Immunology, 10(2965). https://doi.org/10.3389/fimmu.2019.02965
Karschnia, P., Jordan, J.T., Forst, D.A., Arrillaga-Romany, I.C., Batchelor, T.T., Baehring, J.M., Clement, N.F., Gonzalez Castro, L.N., Herlopian, A., Maus, M.V., Schwaiblmair, M.H., Soumerai, J.D., Takvorian, R.W., Hochberg, E.P., Barnes, J.A., Abramson, J.S., Frigault, M.J. and Dietrich, J. (2019). Clinical presentation, management, and biomarkers of neurotoxicity after adoptive immunotherapy with CAR T cells. Blood, 133(20), pp.2212–2221. doi:https://doi.org/10.1182/blood-2018-12-893396.
Marayati, R., Quinn, C.H. & Beierle, E.A., (2019). Immunotherapy in pediatric solid tumors — a systematic review. Cancers, 11(12), p.2022. https://doi.org/10.3390/cancers11122022
Shuel, S.L., 2022. Targeted cancer therapies. Canadian Family Physician, 68(7), pp.515–518. https://doi.org/10.46747/cfp.6807515
Shyr, D., Davis, K.L. & Bertaina, A., (2023). Stem cell transplantation for ALL: you've always got a donor, why not always use it? Hematology: The American Society of Hematology Education Program, 2023(1), pp.84–90. Available at: https://pmc.ncbi.nlm.nih.gov/articles/PMC10726989/
Yang, C., Nguyen, J. & Yen, Y., (2023). Complete spectrum of adverse events associated with chimeric antigen receptor (CAR)-T cell therapies. Journal of Biomedical Science, 30(1). https://doi.org/10.1186/s12929-023-00982-8
Zhang, Y., Qin, D., Shou, A.C., Liu, Y., Wang, Y. & Zhou, L., (2023). Exploring CAR-T cell therapy side effects: mechanisms and management strategies. Journal of Clinical Medicine, 12(19), 6124. https://doi.org/10.3390/jcm12196124
Page last reviewed March 2026