Catholic University Catholic Hematology Hospital

In pediatric acute myeloid leukemia (AML), the approach to treatment has evolved from primarily relying on allogeneic hematopoietic stem cell transplantation (HSCT) to now being tailored according to the patient's prognostic group and response to treatment. For those in low-risk groups with a good prognosis, treatment may consist solely of chemotherapy, reserving allogeneic HSCT for cases of relapse.
For groups other than low-risk, HLA typing is conducted during consolidation therapy as remission is achieved, to identify a suitable donor. If a sibling is an HLA match, they are typically the preferred donor. When a sibling match isn’t available, or it’s not possible for them to donate, the search for a donor extends to the National Organ Transplant Management Center (KONOS) in Korea. If no matched donor is found, alternative options such as cord blood transplantation or haploidentical transplantation are considered.

The outcomes of HSCT between HLA-matched siblings and unrelated donors with a matched HLA are similar. The disease-free survival rate post-transplantation can range widely from 10% to 70%, influenced by the patient’s remission status and other risk factors at the time of the transplant. HLA half-matched family member donations have become more common when no suitable related or unrelated full match is available, reducing the instances where HSCT is not an option due to donor availability.

Acute promyelocytic leukemia (APL or M3 subtype) typically leads to HSCT only if there's a relapse. Secondary AML, which arises post-chemotherapy, or AML evolving from myelodysplastic syndrome, often carries a poor prognosis and is usually recommended for allogeneic HSCT during the first remission.

Allogeneic HSCT may be the only curative option for refractory leukemia, characterized by early relapse or resistance to initial treatment post-remission. However, the success rates are not high. Performing HSCT without achieving complete remission after re-induction chemotherapy has a very low cure rate, so it's vital to conduct HSCT as soon after complete remission is induced as possible.

Given the significant side effects and risks associated with HSCT, the decision to proceed with this treatment is made after a thorough consideration of the potential benefits and risks, taking into account the severity of the patient’s disease and the type of donor available.