Conditions and Treatments

Blood disorders in children

Thalassemia is an inherited blood disorder that causes the body to have less haemoglobin than normal. When haemoglobin is insufficient, the red blood cells do not function properly and have a shorter life span. This leads to fewer healthy red blood cells travelling in the bloodstream. The function of red blood cells is to carry oxygen to all parts of the body. When there are insufficient healthy red blood cells to carry out this function, insufficient oxygen is delivered to the body. This may cause a condition called anaemia in which a person may feel lethargic, weak or short of breath. Patients diagnosed with Thalassemia may have mild or severe anemia.

Cancers in children

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The normal human body has mechanisms to control and regulate cell growth and development. Cancer occurs when abnormal cells grow without control and may spread to other parts of the body.

Sometimes abnormal cells may grow to form a mass. A mass of abnormal cells is called a tumour. Not all tumours are cancerous.

Benign tumours are not cancerous. They usually can be removed, and after which they seldom come back. Benign tumours also do not spread to other parts of the body.
Malignant tumours are cancerous. The abnormal cells are more aggressive. They may invade surrounding tissue, and they can spread to other parts of the body. The spread of cancer to other parts of the body is called ‘metastasis’. In this case, the original tumour is called the primary tumour, and the tumour spreads to form secondary (or metastatic) tumours.

Cancer is rare in children and adolescents. Only 1 in every 600 children under the age of 15 years develops a cancer. Of all cancers, 1 in every 100 cancers occurs in a child. In Singapore we see about 100 – 120 new cases of cancer in children each year (data from the Singapore Childhood Cancer Registry, SCCR).

Cancer usually starts when the genetic material (DNA) in the cell is changed or damaged, leading to uncontrolled cell growth. Rarely, doctors may be able to identify predisposing factors in the patient that make the cells in the body more susceptible to DNA damage. However, most of the time, the cause of the cancer is still not known. Cancer is not an infection, so it is not contagious and will not spread to other people. Most cancers are not inherited and it is extremely rare for another child in the family to be similarly affected by the cancer. Very often parents wonder if there is anything they have done or not done that have caused their child’s cancer. There is absolutely no reason for parents to feel guilty or blame themselves for their child’s cancer.

Cancers in children are quite different from cancers in adults. They occur in different parts of the body. The cancer cells have different appearance and characteristics. They also respond differently to treatment. In general, compared to adults, cancers in children develop more quickly, but they also respond better to treatment, and the cure rate for childhood cancers tend to be higher that most adult cancers.

Types of Childhood Cancers

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LEUKAEMIA

Leukaemia is cancer of the white blood cells. Acute leukaemia arises in the bone marrow and can sometimes spread to the central nervous system and/or testes. The leukaemia cells (also known as “blasts”) multiply in an uncontrolled manner, thereby filling up the bone marrow and affect the production and function of the normal blood cells. This results in reduced numbers of normal white blood cells, red blood cells (or haemoglobin, Hb) and platelets. The child appears pale, tired and has bruising or bleeding manifestations, and may become prone to infections. Infiltration of the bone marrow by blasts can also cause bone pain and prolonged or intermittent fever

Acute Lymphoblastic Leukaemia (ALL) is the most common type of childhood cancer. They arise from the lymphocyte lineage of white blood cells, and can be further divided into B-cell or T-cell ALL. The patients are stratified into standard risk, intermediate risk or high risk group based on their disease characteristics and response to treatment. ALL treatment is mainly with multi-agent chemotherapy. With current modern precision diagnostics and chemotherapy regimens, very good cure rates can be achieved, especially for standard (over 90%) and intermediate risk (over 80-90%) patients. Hematopoietic cell transplant (HCT), and/or advanced therapeutics such as immunotherapy and cellular therapy have helped to further increase the cure rates of patients with high risk or relapsed/refractory ALL.
Acute Myeloid Leukaemia (AML) is the most common acute leukaemia affecting adults, but less common in children. AML can arise as a second cancer in patients who previously received chemotherapy. Intensive chemotherapy is used to treat AML. Treatment outcome depends on the subtype of AML and the response to chemotherapy treatment. Hematopoietic cell transplant (HCT) is recommended for certain groups of AML patients who are at a high risk of relapse.
Chronic Myeloid Leukaemia (CML) is also called chronic granulocytic leukaemia. Unlike acute leukaemias, chronic leukaemias such as CML develop slowly, and the patient often remains well for several months. HCT is the only permanent cure to CML. However a class of targeted therapies called tyrosine kinase inhibitors (“TKIs” e.g. imatinib, dasatinib) can now keep CML under control for a long time. CML in “Chronic Phase”, especially when left untreated, can develop into “Accelerated Phase and “Blast Crisis”. When this happens, the disease behaves like acute leukaemia and more intensive chemotherapy is required.

LYMPHOMA

Lymphomas are cancer of the lymphatic system and they arise from lymphoid organs, such as lymph nodes. Lymphoma is broadly classified as Hodgkin Lymphoma or Non-Hodgkin Lymphoma (NHL).

Hodgkin Lymphoma is defined by the presence of a specific type of malignant cell called the Reed-Sternberg cell. The 2 main types of Hodgkin lymphoma are classic Hodgkin lymphoma and nodular lymphocyte-predominant Hodgkin lymphoma. Classic Hodgkin lymphoma is the more common type and is further divided into 4 subtypes – nodular sclerosing, mixed cellularity, lymphocyte-rich and lymphocyte-depleted.
Non-Hodgkin Lymphoma (NHL) in children is broadly divided into 3 types: lymphoblastic lymphoma (LBL), anaplastic large cell lymphoma (ALCL), and aggressive mature B-cell NHL. The aggressive mature B-cell NHL is further classified as Burkitt or Burkitt-like lymphoma, diffuse large B-cell lymphoma (DLBCL), and primary mediastinal B-cell lymphoma (PMBCL). There are several other NHL subtypes which are rare in children.

The most common presentation is enlarged lymph nodes, which can occur in the neck, armpit, groin, chest or abdomen. Large bulky lymph node masses in the chest and neck can cause chest discomfort, difficulty breathing and facial swelling. Large lymph nodes in the abdomen can cause abdominal pain and symptoms of intestinal obstruction. Patients with more advanced disease may also have fever, night sweats or bone pain. The initial evaluation may include laboratory tests, imaging (MRI / CT / PET scans), biopsy of the enlarged lymph node, and evaluation of the bone marrow and cerebrospinal fluid (CSF) to confirm diagnosis and staging. The child would may require intensive supportive care to monitor and manage initial complications such as tumour lysis syndrome and/or large chest mass causing compressive effects on airways and neck vessels.

Lymphoma is treated with subtype-specific chemotherapy. Other treatment modalities may include:

Targeted therapies such as monoclonal antibodies (e.g. rituximab), immunotherapy with checkpoint inhibitors (e.g. pembrolizumab), ALK inhibitors
Hematopoietic cell transplant may be recommended for relapsed / refractory lymphoma
Cellular therapy using CAR T-cells may be recommended for relapsed / refractory aggressive B-cell lymphomas.
Radiotherapy may be needed especially if there is bulky or residual lymphoma disease.

HISTIOCYTOSIS

Histiocytosis is a group of conditions affecting immune cells called histiocytes. There are several types of histiocytosis.

Langerhans Cell Histiocytosis (LCH)

LCH is a histiocytosis more commonly seen in children but can also occur in adults. LCH can affect single (unifocal) or multiple sites (multi-focal), in a single organ system or have multisystem involvement. The skin and the bone are the most commonly affected organ systems. Certain organ involvement (liver, spleen and bone marrow) are associated with a more aggressive disease outcome and hence are called “risk-organs”. A biopsy of the involved area is necessary to ascertain the diagnosis. Further investigations such as blood tests and scans are required for staging. The treatment is dependent on the site and extent of involvement. Low risk single system LCH may only require topical steroids (e.g. skin-only LCH), surgical curettage (unifocal bone LCH), or systemic steroids with/without low-dose chemotherapy. Multifocal and multisystem LCH generally require systemic steroids and chemotherapy. Patients with craniofacial bone involvement are at risk of developing brain involvement, hormonal deficiencies and neurodegenerative late effects, and need to be monitored accordingly. Mutation in the BRAF gene (the BRAF V600E mutation) can be detected in over half of LCH cases. Targeted therapies against BRAF V600E mutation have shown efficacy in relapsed refractory multisystem LCH.

Other Histiocytic Disorders

These are rare conditions and include juvenile xanthogranuloma (JXG), Rosai-Dorfman disease, Erdheim-Chester disease (ECD), hemophagocytic lymphohistiocytosis (HLH) and malignant histiocytosis.

Brain tumours are a heterogeneous group of tumours in the central nervous system, CNS (brain and spinal cord). They can be benign or malignant. In a young infant, while the skull bones are still soft, a brain tumour may cause the head to increase rapidly in size. In an older child, as the brain is confined in the hard bones of the skull, a brain tumour will cause the pressure in the brain (known as “intracranial pressure”, ICP) to increase. Some tumours can obstruct the flow of fluid (cerebrospinal fluid, or CSF) within the brain and cause hydrocephalus, which will further increase ICP. Raised ICP can cause headache, vomiting and drowsiness. Other signs and symptoms depend on the exact location of the brain tumour, and include weakness in limbs or face, unsteadiness or shakiness, seizures or convulsions, squinting of eyes or other visual disturbance, hormonal disturbances, general neurological decline, etc. Many types of brain tumours can also involve or spread to the spinal cord.

After the initial imaging (CT and/or MRI scans), a biopsy is usually needed to get the exact diagnosis. Sometimes the surgeon may be able to remove the entire brain tumour upfront, or a shunt procedure may be needed to relieve the CSF obstruction. Subsequent treatment (chemotherapy and/or radiotherapy) may be needed and will depend on the tumour type. Radiotherapy can cause significant side effects in very young children, so chemotherapy is sometimes used to in an attempt to avoid or delay radiotherapy in these very young children. Types of brain tumours in children include gliomas, embryonal brain tumours, and germ cell tumours.

GLIOMAS

Gliomas are the commonest group of brain tumours. They are a heterogeneous group and have different names depending on the location and cell of origin. Examples of gliomas include astrocytoma, oligodendroglioma etc. Generally, they are also classified as low grade (slow growing and less aggressive, grade 1 or 2) or high grade (fast growing and more aggressive, grade 3 or 4).

Low grade gliomas (LGG) are often curable with surgery alone. Radiotherapy and/or chemotherapy may be required for tumours that cannot be resected completely.
High grade gliomas (HGG) and diffuse midline gliomas are highly aggressive brain tumours and tend to be more challenging to treat.

EPENDYMOMA

Ependymomas are tumours arising from the cells that line the fluid-filled spaces in the brain or spinal cord. The signs and symptoms generally depends on the location of the tumour. Complete surgical resection is the most effective treatment for ependymoma. Radiotherapy may be recommended if the tumour is assessed to have aggressive characteristics or if the tumour cannot be completely removed. Chemotherapy is generally not very effective on ependymoma, but may sometimes be given if surgery and radiotherapy are not able to control the tumour.

EMBRYONAL BRAIN TUMOURS

Medulloblastoma is a type of embryonal brain tumour that arises from the back of the brain (called the “posterior fossa”) and therefore cause symptoms of unbalance, unsteadiness and poor coordination. There may also be headache, vomiting and drowsiness. The initial surgery should aim to achieve gross total resection of the tumour. The subsequent craniospinal radiation therapy and systemic chemotherapy is then tailored based on the molecular subtype and staging.

Other less common embryonal tumours include atypical teratoid rhabdoid tumours (ATRT), embryonal tumours with multi-layered rosettes (ETMR).

CNS GERM CELL TUMOURS

Germ cell tumours are tumours arising from primitive germ cells. They can occur within or outside the central nervous system (CNS), and they commonly occur in the midline. As a result, they can give rise to visual or hormonal problems, or hydrocephalus. They are made up of different cell types and may or may not secrete alpha-foetoprotein (AFP) and/or beta human chorionic gonadotrophin (B-HCG) into the blood or brain fluid (CSF). The cell types include teratoma, germinoma, non-germinoma (of which there are a few subtypes), or mixed. Germinomas tend to respond well to chemotherapy and radiotherapy. If the tumour secretes AFP or □-HCG, these can be used as tumours markers to help monitor the response of the tumour to treatment.

NEUROBLASTOMA

Neuroblastoma is the most common solid cancer occurring outside the brain in childhood. The tumour consists of primitive nerve cells arising usually from the abdomen. It can also arise in the chest, neck or pelvis. Diagnosis and risk stratification are important to guide the management of neuroblastoma. Localised disease is highly curable with surgery, with or without chemotherapy. Selected infants with low risk neuroblastoma may be observed without treatment. High risk neuroblastoma refers to neuroblastoma that has spread to distant sites (bone and/or bone marrow), or has poor biological features (e.g. N-MYC gene amplification). Treatment of high risk neuroblastoma is more challenging and will require intensive multi-modality approaches such as chemotherapy, surgery, radiotherapy, high dose myeloablative therapy (‘MAT”), immunotherapy, targeted therapies (e.g. ALK inhibitors if indicated), differentiating agent and/or experimental approaches.

Immunotherapy in neuroblastoma involves the use of anti-GD2 monoclonal antibodies. GD2 is a molecule that is highly expressed on most neuroblastoma cells. Anti-GD2 monoclonal antibodies are administered into the bloodstream by intravenous infusion. The antibodies will then identify and attach to the GD2 target on neuroblastoma cells, and help trigger an immune response to destroy the cancer cells. Addition of immunotherapy has been shown to improve the survival outcome of children with neuroblastoma, and hence anti-GD2 immunotherapy is now considered part of standard of care for high-risk neuroblastoma. Anti-GD2 immunotherapy given together with chemotherapy (i.e. “chemo-immunotherapy”) is also effective in relapsed or refractory neuroblastoma. Anti-GD2 immunotherapy can cause side effects such as fever, pain, fluid leaking from blood vessels (called “capillary leak” which can lead to puffiness and fluid retention, and if severe can affect heart rate, respiration and blood pressure), allergic reactions, fast heart rate, difficulty breathing, low blood pressure, eye and vision problems, nausea, vomiting, diarrhea. With a trained clinical team and good supportive care in place, these side effects are generally manageable.

RETINOBLASTOMA

Retinoblastoma is a cancer of the eye in childhood. The usual presentation is abnormal red eye reflex of the pupil (“white pupil” seen in photographs) or a squint. Children with very advanced disease may present with pain, redness and bulging of the affected eye.

Retinoblastoma can be hereditary or non-hereditary. Non-hereditary retinoblastomas affect one eye and have a later age of onset. Hereditary retinoblastomas tend to affect both eyes, be multi-focal in nature, and have an earlier age of onset (usually in first year of life). Retinoblastoma is caused by mutations in both copies of the RB1 gene in the cancer cells. RB1 is a tumour suppressor gene, and functions to protect the cell from becoming cancerous. In non-hereditary retinoblastoma, both copies of the RB1 gene are normal in the other cells in the body. In hereditary retinoblastoma, all the other cells in the body have an abnormal or mutated RB1 gene. Patients with hereditary retinoblastoma are therefore also at risk of developing other cancers such as pineoblastoma, osteosarcoma, other sarcomas, and melanoma. Genetic testing and counseling are recommended for all children with retinoblastoma, especially for patients with bilateral disease or early onset disease.

Most children with unilateral disease can be cured with surgical removal of affected eye (“enucleation”) followed by prosthetic eye placement. Management of bilateral disease is more complex as there is a need to try to preserve eye and vision. Focal therapy (laser, “freezing”, intravitreal chemotherapy etc.) and systemic chemotherapy are given. Ophthalmic artery chemotherapy may be considered in suitable patients. Close surveillance by the eye specialist is necessary as new tumours may still develop in the first few years of life. Removal of one or both affected eyes may be necessary if very advanced disease cannot be controlled with focal therapy and chemotherapy. Rarely, radiotherapy may have to be considered for disease with poor response, but this is generally avoided if possible because of the significant side effects of radiation in a young child.

Overall survival rates for retinoblastoma are high in most developed countries as most patients are seen in early stages of disease. Patients with disease spread out of the eye to other parts of the body have a much poorer outcome.

LIVER TUMOURS

Liver tumours are rare in children. The usual presentation is abdominal distension.

Hepatoblastoma is the most common liver cancer in children. This usually occurs in very young children (under age of 3-4 years old). A detailed imaging (usually by MRI or CT scans) is important to evaluate the extent of tumour involvement. The blood alpha-foetoprotein (AFP) is elevated in most cases and can be used as a tumour marker to help monitor the response to treatment. Treatment is by surgical resection and chemotherapy. If the tumour remains unresectable even after chemotherapy, the patient will be referred for liver transplant or for consideration of alternative therapeutic approaches such as interventional radiology procedures (e.g. hepatic arterial chemo-embolisation, radiofrequency ablation) or radiation therapy.

Hepatocellular carcinoma (HCC) is a liver cancer mainly in adults, but may occasionally occur in children. Underlying liver disease is a risk factor for developing HCC, such as hepatitis B or C infection, liver cirrhosis, chronic liver inflammation or chronic liver disease. The tumour marker AFP is also frequently elevated in HCC. Complete surgical resection offers the best chance of cure for HCC. Advanced HCC that is disseminated or not resectable is challenging to treat. Suitable patients may be offered systemic therapies or referred for interventional radiology procedures (e.g. hepatic arterial chemo-embolisation, radiofrequency ablation) or radiation therapy (radioembolisation).

Other liver tumours in children include undifferentiated embryonal sarcoma of the liver (UESL), biliary tract rhabdomyosarcoma, various vascular tumours, mesenchymal hamartoma, focal nodular hyperplasia etc.

KIDNEY TUMOURS

The most common type of malignant kidney tumour in childhood is Wilms tumour (also known as nephroblastoma). Kidney tumours may present as abdominal distension or blood in the urine. After initial imaging (ultrasound, CT or MRI scans), biopsy may be performed to confirm the diagnosis. The patient is then given pre-operative chemotherapy, after which surgery is performed to remove the tumour together with the affected kidney. In rare situations where there is involvement of both kidneys, or if the other kidney is also at risk of developing tumours, a “nephron-sparing” approach will be necessary to preserve sufficient kidney tissue and function. Radiotherapy may be necessary if the tumour shows evidence of spread outside the kidney. After the surgical resection, further post-operative chemotherapy is tailored according to the tumour’s response to the pre-operative chemotherapy. The cure rate depends on the staging and whether there are unfavorable features, namely anaplasia or post-chemotherapy blastemal predominance, seen in the tumour cells. On the whole, over 90% of children with Wilms tumour are cured.

The other types of kidney tumours in childhood are less common and include: clear cell sarcoma of kidney (CCSK), rhabdoid tumour of kidney (RTK, rare), renal cell carcinoma (RCC, rare).

GERM CELL TUMOURS

Germ cells are primitive cells that persist in various parts of the child’s body after birth and may produce tumours. The common sites of occurrence are testes, ovaries, bottom of spine (sacrococcygeal area), chest, abdomen or brain (refer to section on brain tumour). Benign tumours can be cured by surgery alone. Malignant germ cell tumours (“MGCT”) are classified into various subtypes, and may secrete tumour markers alpha-foetoprotein (AFP) and beta-human chorionic gonadotrophin (B-HCG). These are also treated with surgery, but many patients also require chemotherapy and sometimes radiotherapy. On the whole germ cell tumours respond very well to treatment, so cure rates of over 80-90% can be achieved in patients without poor risk factors. Poor prognostic factors for MGCT include advanced stage, older age, and tumour location outside the ovaries or testes.

SOFT TISSUE SARCOMAS

Rhabdomyosarcoma (RMS) is the most common soft tissue tumour in childhood. RMS is an aggressive high-grade soft tissue cancer that can occur anywhere in the body, and may spread to the lungs, bone and bone marrow. The initial evaluation consists of a biopsy to confirm the diagnosis and to ascertain the molecular subtype, followed by staging investigation to determine the extent of tumour involvement and cancer spread. The treatment of RMS consists of chemotherapy and local control by surgery and/or radiation therapy. The exact local control strategy depends on the tumour location, subtype, size and resectability. A multidisciplinary team management is essential to coordinate and provide the complex multimodality care. Survival outcome is better for patients with localized disease, i.e. disease that has not spread to distant sites.

Non-rhabdomyosarcoma soft tissue sarcoma (NRSTS) is a mixed group of tumours of the soft tissue, and can occur anywhere in the body. Compared to RMS, these are in general rarer in childhood but most subtypes become more common with increasing age. Examples of NRSTS include infantile fibrosarcoma, malignant rhabdoid tumour, desmoid tumour, synovial sarcoma, alveolar soft part sarcoma, malignant peripheral nerve sheath tumour etc. Generally, complete surgical removal of the primary tumour should be attempted as this is associated with better outcome. Compared to RMS, many types of NRSTS tend to be less responsive to chemotherapy or radiotherapy. Selected diagnostic subtypes may be amenable to targeted therapies.

BONE TUMOURS

The bone tumours that can occur in children include osteosarcoma and Ewing sarcoma. The usual presentation is swelling, pain or even fracture of the affected bone.

Osteosarcoma (also called osteogenic sarcoma) is the most common malignant bone tumour in children and adolescents, and usually occurs in the fast growing long bones in the arms and legs. After initial scans and biopsy, chemotherapy is used to shrink the primary tumour. The tumour is then resected surgically. The type of surgery (limb salvage or amputation) depends on several factors such as the tumour site and size. After the surgery, more chemotherapy is given to destroy any remaining cancer cells. Survival outcomes are better for patients whose disease have shown good response to chemotherapy and have not spread to distant sites (e.g. lungs).

Ewing Sarcoma can occur in any bone or soft tissue, and can spread to lungs, bone or bone marrow. Treatment is by intensive chemotherapy, surgery and radiation. The exact local control strategy depends on the tumour location and resectability. Like many high-grade sarcomas, the outcomes are better for patients with limited extent of disease e.g. localized tumours or tumours with lung-only spread.

Types of Cancer treatment

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For most brain and solid tumours, the first surgery is a biopsy, in which a small piece of tumour tissue is removed surgically and sent for diagnostic tests. If possible, the surgeon will try to perform tumour resection. However, if the tumour is too big or lies too near important body organs, the surgery may be difficult, dangerous or disfiguring for the child. In these cases and in disseminated cancer a few courses of chemotherapy and/or radiotherapy are given first to shrink the tumour and control the diseminated disease before the surgery.

Another common surgery is for insertion of a port-a-cath or Hickman line. These are devices placed within the child’s body, so as to provide a convenient and less painful access route for the frequent administration of drugs, fluids and blood products into the veins.

More information on surgical oncology can be found here.

Systemic anti-cancer therapy includes chemotherapy, targeted therapy and immunotherapy, which are various medications to treat cancer.

Chemotherapy attacks fast growing cells such as cancer cells.
Targeted therapy attacks cancer cells expressing specific targets.
Immunotherapy acts on the immune system to increase immune killing of cancer cells.

Systemic anti-cancer therapy can consist of a single agent or a combination of these medications, intended to kill or control the growth of cancer cells. The treatment will be given according to the recommended route(s) of administration, dose, schedule and duration. The route of administration can be oral, or via injection into the body (e.g. into a vein or muscle or under the skin, or into the central nervous system via a spinal injection - known as intrathecal or "IT"). If the treatment involves repeated intravenous (IV) administrations, your doctor will advise whether there is a need for a central venous access device, such as a port-a-cath, Hickman line, or peripherally-inserted central catheter (PICC). The treatment may be given in the outpatient ambulatory day therapy unit, or in the inpatient ward setting

This uses radiation to kill cancer cells that lie within the radiation field, and includes a variety of radiation therapy techniques such as conventional external beam radiotherapy, proton beam therapy, brachytherapy (also known as internal radiation therapy). There will usually be a consultation with the radiation oncologist to prepare and plan for the actual radiotherapy sessions. The treatment itself is painless but the child will have to lie still during the session, so young children may need general anaesthesia.

More information on the radiotherapy and proton beam facility can be found here.

Haematopoietic Cell Transplant (HCT)

Haematopoietic stem cells are traditionally obtained from the bone marrow, therefore the transplant procedure is also termed haematopoietic stem cell transplant or bone marrow transplant (BMT). Alternative sources of hematopoietic cells are from the peripheral blood or cord blood. In allogeneic HCT, the hematopoietic cells can be from a matched sibling or family donor, a matched unrelated donor, or a "half-matched" (haploidentical) family donor. In autologous HCT for certain high risk brain and solid tumours, the child’s own stem cells are used.

HCT is mainly used to treat high risk and relapsed leukaemia. HCT may also be used in the treatment of some other malignant (e.g. high-risk neuroblastoma) and non-malignant conditions (e.g. severe combined immunodeficiency, other severe primary immunodeficiency disorders, certain blood or metabolic diseases).

The HCT procedure is carried out in a protective environment, which are single-patient protective isolation rooms with appropriate engineering and air control system (including high efficiency particulate air (HEPA) filter, positive air pressure) to reduce opportunistic infections (particularly fungal) in severely immunocompromised patients. The child typically undergoes a period of conditioning, during which chemotherapy with/without radiotherapy are used to kill malignant cells left in the body and also to prepare the body to receive the donor cells. The donor cells are then infused into the child (recipient). The child (recipient) receives supportive care to reduce risks of transplant-related complications, and is closely monitored in the hospital until the new marrow begins to function sufficiently and complications (if any) are under control.

Haematopoietic Stem Cell Transplantation (HSCT) - A Guide for Patient and Families

Download the HCST Guide here.

Chimeric antigen receptor (CAR) T-cell therapy

CAR T-cell therapy is a cell therapy procedure that involves the infusion of T-cells that have been modified in the laboratory to express chimeric antigen receptors (CARs) on the cell surface.

T-cells, also known as T-lymphocytes, are a type of white blood cells that function as an important part of the body's immune system to recognise and destroy abnormal or infected cells.
Chimeric antigen receptors (CARs) are artificial receptors specially designed to enable T-cells to recognise and kill abnormal cells with greater accuracy and efficacy.
T-cells, collected from the patient (i.e. autologous) or a donor, can be modified or re-programmed in the laboratory to become CAR T-cells and then infused into the patient

The most established CAR T-cell therapy to date is the use of autologous CAR T-cells to treat acute lymphoblastic leukaemia (ALL) or aggressive B-cell lymphomas that have relapsed or are not responding to conventional therapies. Other forms of CAR T-cell therapies and CAR T-cell therapies for other cancers may be available as experimental therapies as part of clinical trials. Your doctor will assess your child's condition and discuss if CAR T-cell therapy is an appropriate treatment option for your child.

Overview of autologous CAR T-cell treatment process:

Supporting the child with cancer

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Full Blood Count (FBC) assesses the number and size of blood components such as red blood cells (RBC), white blood cells (WBC), neutrophils (a subtype of WBC), platelets, blasts (leukaemia cells found in the blood). The FBC is used to evaluate the overall health and has the capability to detect a wide range of abnormalities such as anemia, infection and leukaemia.

What is a bone marrow aspiration (BMA)?

Bone Marrow aspiration (commonly known as BMA) and bone marrow biopsy are procedures used to obtain and examine the bone marrow. The bone marrow can be affected by various disease processes. Bone Marrow Aspiration or biopsy is one of the tests needed to help us diagnose these diseases. Bone Marrow is the soft spongy tissue found within some of our bomes. Bone marrow is the 'factory' of the body's blood cells and can be found/extracted from the back or front of the pelvic bone in adults and older children. For younger children, bone marrow can be found/extracted from the upper shin, just below the knee (Figure 1 below). This is where our body makes red blood cells, white blood cells and platelets. Some of the diseases that can involve the bone marrow includes; Cancers, Bone marrow failures syndrome, infections and other conditions. Your child will be given sedation for the procedure. In addition, a local anaesthetic injection may also be given. Most children do not experience much pain or discomfort after the procedure.

In general, complications are very rare after a bone marrow aspiration or biopsy. However, some possible complications may include; complications arising from sedation, pain, bleeding, infection of the would and injury to surrounding body sutures. After the procedure, the pressure bandage can be removed after 12 to 24 hours if there is no bleeding. The bone marrow samples are sent to various laboratories for appropriate tests such as haematology, pathology or cytogenetics, and lastly, your attending physician will update you on the results once it is available. Please discuss with your doctor if you have any questions about the procedure.

Procedural sedation is a medical technique administered before a procedure to calm a person through ways of pain reduction and anxiety. This medical technique involves giving the patient sedatives or pain pills either through the an intravenous (IV) line in the patient's arm or through swallowing or inhaling them that will ease discomfort, pain and anxiety. Procedural sedation is used only for short and straightforward procedures (i.e., non-surgical procedures) and not recommended for complex surgeries. Not all patients or procedures are suitable for sedation and it is important to exclude patients that are at high risk of failure e.g. extremely anxious patient or the hyperactive child, and procedures that are very painful and prolonged. Your attending physician will prepare the patient, family and environment to contribute towards the success of sedation. Consent for procedural sedation should ensure patient or parent/legal guardians understand the sedation process, risks and benefits and alternatives to sedation.

The ultimate goals of procedural sedation is to minimise the psychological distress and pain associated with the procedures, optimize conditions for the procedure to be completed safely, and ensure that patient undergoes the sedation without any adverse events.

Febrile neutropenia is a medical situation when the patient experiences fever and low neutrophil counts. Many forms of cancer treatment cause suppression of the bone marrow’s ability to produce blood cells. When levels of neutrophils (a type of white blood cells) are reduced, it is called ‘neutropaenia’. When this happens, the body’s ability to fight infections is greatly reduced. ‘Febrile neutropaenia’ means ‘fever during neutropaenia’. This can be a medical emergency, as sometimes the infection can be overwhelming and life-threatening. Therefore, the child ongoing cancer treatment should be brought to the hospital immediately when there is fever. Childhood infections like chickenpox and measles can also be dangerous in a child with cancer. If your child comes into contact with chickenpox or measles, or if suspicious rash develops, you should inform your doctor immediately. You will be given instructions to call the Oncology Nurse and to bring your child to the hospital whenever there is fever or when your child appears unwell.