Pediatric Bone Marrow Transplant - Children's Bone Marrow Transplant

A bone marrow transplant replaces a child's failing blood system with a healthy one. For many children it is the only treatment that cures the disease.

Pediatric bone marrow transplant, also called children's bone marrow transplant or hematopoietic stem cell transplantation, gives a child new blood-forming stem cells from a donor or from the child's own body. It treats high-risk leukemia, thalassemia, sickle cell disease, aplastic anemia, inherited immune deficiencies and some metabolic diseases. Results in children are strong. In an international survey of 1,000 patients with sickle cell disease, most of them children, 91.4 percent were alive and free of disease five years after a transplant from a matched brother or sister (Gluckman and colleagues, Blood, 2017). This page explains which children need a transplant, donor types, conditioning, the transplant day, isolation, graft versus host disease, infections, life after transplant, and how families from abroad arrange a pediatric bone marrow transplant at Biruni Hospital in Istanbul.

What a bone marrow transplant is

Where the cells come from

When a child needs one

A transplant is recommended when the disease cannot be cured or controlled with standard treatment, or when the risks of living with the disease outweigh the risks of the transplant.

• Leukemia that returned after chemotherapy, or that carries high-risk genetic changes.
• Thalassemia major in a child who depends on regular transfusions every three to four weeks.
• Sickle cell disease with a stroke, an abnormal brain artery scan, repeated pain crises or acute chest syndrome.
• Severe aplastic anemia, especially with a matched sibling donor.
• Severe combined immunodeficiency and other inherited immune disorders.

Diseases we treat

Every referral reaches a multidisciplinary board of pediatric hematologists, oncologists, transplant physicians and intensive care specialists.

Children with a solid tumor

Choosing the donor

• Children can be donors for a brother or sister, and bone marrow collection from a child donor follows strict safety rules.
• A parent donor gives cells from the blood or, under anesthesia, from the hip bone.
• A separate physician checks donor safety.

Families often ask whether a parent is a good donor. A parent always shares half the tissue type with the child, and modern haploidentical methods make parents a strong option when no matched donor exists.

Tissue typing and the donor search

Results in children

Numbers depend on the disease.

In severe combined immunodeficiency, a study of 240 infants transplanted at 25 North American centers between 2000 and 2009 found five-year survival of 94 percent in babies transplanted at three and a half months of age or younger, 90 percent in older infants without infection, and 82 percent in older infants whose infection had cleared before the transplant (Pai and colleagues, 2014). In sickle cell disease, 91.4 percent of patients were alive and free of disease five years after a matched sibling transplant, and overall survival reached 92.9 percent (Gluckman and colleagues, 2017). In high-risk acute lymphoblastic leukemia, the international FORUM trial randomized 417 children and young people aged 4 to 21 years, and two-year survival was 91 percent with total body irradiation and etoposide against 75 percent with chemotherapy-only conditioning, and relapse fell from 33 percent to 12 percent (Peters and colleagues, 2021). Earlier diagnosis, early referral and a healthier child at transplant all improve these results. We review reports fast. The transplant is planned as soon as a donor is ready.

We share our own unit figures on request.

Conditioning

In the FORUM trial, treatment-related deaths at two years were 2 percent after total body irradiation and 9 percent after chemotherapy conditioning (Peters and colleagues, 2021).

Transplant day

Day zero

The donor on the same day

Engraftment and isolation

The child stays in a single room with filtered air in our pediatric transplant unit, and one parent stays in the room day and night.

Graft versus host disease

Children develop severe graft versus host disease less often than adults, and a matched sibling donor gives the lowest risk.

Watching for early signs

Parents learn to report any rash, loose stool, poor appetite or yellow eyes the same day. Early treatment works best.

Infections

• Antibiotics, antifungals and antivirals begin before the counts fall.
• Weekly blood tests track cytomegalovirus, Epstein-Barr virus and adenovirus, so treatment starts before the virus causes illness.
• Any fever is treated within an hour of the first reading.

Long-term follow-up

Late effects

Growth, puberty, fertility, thyroid function, heart, lungs, eyes, kidneys and school performance are checked every year by a long-term follow-up team.

Fertility preservation

Before conditioning, we discuss freezing sperm in adolescent boys and ovarian or testicular tissue in younger children, because high-dose chemotherapy and radiotherapy can cause infertility. The choice belongs to the family.

Family life during treatment

Siblings matter too.

A donor brother or sister needs explanation, attention and praise, and siblings at home feel the absence of a parent and the ill child. Our psychologists and social workers support every family member, prepare children for procedures with age-appropriate stories and play, and help parents plan return to school, which follows six to twelve months after the transplant once the immune system has recovered. Nobody is left out.

For families from abroad

Cost

An unrelated donor search and cell shipment add registry fees, which we list separately in the quote.