About KKH Paediatric Blood and Marrow Transplant and Cellular Therapy Programme

The KKH Paediatric Blood and Marrow Transplant and Cellular Therapy (BMTCT) Programme provides specialised care for children with a wide range of malignant and non-malignant conditions that may benefit from blood and marrow transplantation or advanced cellular therapies.We care for patients with blood cancers, bone marrow failure disorders, haemoglobin disorders, primary immunodeficiencies, inherited metabolic disorders and other complex conditions. Supported by a dedicated multidisciplinary team, we work closely with children and their families throughout the treatment journey, from assessment and treatment planning to recovery and long-term follow-up care.

Haematopoietic stem cells (HSC) are multipotent cells found in the bone marrow, peripheral blood, and umbilical cord blood. They form the foundation of your child's blood and immune system. These stem cells have the unique ability to develop into all types of blood cells needed to stay healthy, including red blood cells that carry oxygen, white blood cells that fight infections, and platelets that help blood clot and prevent bleeding.

In healthy children, stem cells continuously produce billions of new blood cells each day, replacing old or damaged cells. When the blood and immune system is diseased or impaired, it cannot function properly. This can lead to serious complications, including anaemia, infections, and bleeding problems.Our programme utilises several sources of these vital stem cells. Bone marrow stem cells are obtained directly from the soft tissue inside bones. Peripheral blood stem cells are collected from the bloodstream after medications mobilise them from the bone marrow. Stem cells can also be sourced from cord blood, which is collected from the umbilical cord after birth and stored in specialized cord blood banks. We also offer advanced cellular therapies such as CAR-T cell therapy, where a patient’s own T cells are modified in the laboratory to better recognise and attack cancer cells before being infused back into the body.

A haematopoietic stem cell transplant (HSCT) may be required when your child’s bone marrow is severely damaged, diseased, or not functioning properly. This can occur in conditions such as leukaemia and other blood cancers, where abnormal cells crowd out healthy blood cells, or in inherited disorders where the bone marrow does not produce adequate healthy cells.

Some children require transplantation because intensive treatments, such as chemotherapy, while necessary to treat cancer, can also damage healthy bone marrow. In these cases, transplantation allows for the restoration of normal blood cell production.

For children with genetic conditions affecting blood or immune function, transplantation offers the potential for a cure by replacing defective cells with healthy ones.

Our programme cares for children with a broad range of conditions affecting the blood, immune system and metabolism.

Blood Cancers: We specialise in treating various types of leukaemia, where abnormal white blood cells multiply uncontrollably, and lymphomas, which are cancers of the lymphatic system. These conditions often require intensive treatment followed by stem cell transplantation to restore healthy blood cell production.

Solid Tumours: Our expertise includes treating certain high-risk solid tumours that require very intensive chemotherapy. In these cases, stem cell transplantation helps rescue and rebuild the bone marrow after high-dose treatment designed to eliminate cancer cells.

Bone Marrow Failure Syndromes: We treat conditions where the bone marrow fails to produce adequate blood cells, whether due to inherited genetic conditions present from birth or acquired disorders that develop over time. These conditions can affect the production of red blood cells, white blood cells, or platelets.

Haemoglobinopathies: Our programme provides transplantation for children with inherited disorders affecting haemoglobin, the protein in red blood cells that carries oxygen. These genetic conditions can cause severe anaemia and other complications that significantly impact quality of life.

Primary Immunodeficiencies: We treat children born with immune systems that cannot function properly, leaving them vulnerable to severe, life-threatening infections. Stem cell transplantation can provide these children with a healthy immune system.

Inborn Errors of Metabolism (IEM): We care for children with inherited metabolic disorders caused by defects in specific enzymes or metabolic pathways. These rare genetic conditions can lead to the accumulation of toxic substances or the inability to produce essential compounds, resulting in serious complications affecting multiple organs and development. For selected IEMs, haematopoietic stem cell transplantation can provide a source of healthy donor cells capable of producing the missing enzyme, helping to slow disease progression, improve long-term outcomes, and enhance quality of life.