If pediatric acute leukemia patients receive proper treatment before and after hematopoietic stem cell transplantation (HSCT), treatment outcomes with haploidentical related donors (HRD) can be as good as those with matched unrelated donors (MUD), a study showed.

On Wednesday, the research team, led by Professors Kang Hyoung-jin and Hong Kyung-taek at the Seoul National University Hospital’s Pediatrics Department, released the results of their study.

In the study, the research team provided customized anticancer drugs to patients and compared the treatment outcomes of HRD HSCT with MUD HSCT, who received graft-versus-host disease prophylaxis after transplantation.

HSCT is essential for the treatment of high-risk acute leukemia. However, the patient has to receive HSCT from a matched sibling or a MUD to increase the therapeutic effect. Still, an HSCT candidate has a 25 percent chance of finding a matched sibling and a 50 percent chance of finding a MUD.

HRD HSCT cases have been recently increasing, but almost no studies have compared HRD HSCT with MUD HSCT in pediatric patients.

The research team divided high-risk acute leukemia children who received the first allogeneic HSCT at Seoul National University Children’s Hospital between January 2013 and April 2020 into two groups – one in the HRD group (35 children) and the other in the MUD group (45). Then, the researchers compared their treatment results.

The results showed that HRD HSCT demonstrated outcomes similar to those of MUD HSCT. Leukemia-free survival in the HRD HSCT group was 83.7 percent, and that in the MUD HSCT, 88.6 percent.

In particular, the survival rate of patients with acute myeloid leukemia was 93.8 percent in the HRD group, 8.2 percentage points higher than that of the MUD group, showing excellent treatment results.

The research team said administering the customized busulfan-based anticancer drugs and using cyclophosphamide after transplantation was effective.

“We hope this study can become evidence for patients, finding it difficult to receive HSCT due to lack of a matched donor or whose HSCT is delayed, to receive transplantation from HRD effectively and safely,” Kang said.

The study was published in the latest issue of Transplantation and Cellular Therapy.

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