Proven effectiveness in treating pediatric acute lymphoblastic leukemia without radiation...a 5-year survival rate of 86.1%
Dec 19, 2024
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The study identified high survival rates and low risk of complications while reducing long-term side effects from systemic irradiation, suggesting new therapeutic possibilities.
Professor Kang Hyung-jin and Professor Hong Kyung-taek of the Department of Pediatrics and Adolescents at Seoul National University Hospital published the results of a prospective phase 2 clinical trial study evaluating the effectiveness and safety of allogeneic hematopoietic stem cell transplantation using only drugs without radiation in 36 high-risk children and adolescents under the age of 21 from February 2014 to August 2021.
Acute lymphoblastic leukemia is the most common blood cancer in children and adolescents, and most of them can be cured with chemotherapy alone, but allogeneic hematopoietic stem cell transplantation is required for recurrent, refractory, and highest-risk patients.
Allogeneic hematopoietic stem cell transplantation is performed through pretreatment therapy, in which a high dose of radiation or anticancer agent is administered to replace the damaged patient's hematopoietic stem cells with healthy donor cells.
The purpose of pretreatment therapy is to remove residual cancer cells from the patient's bone marrow and to help new cells settle in the patient's body. This pretreatment therapy is performed by anticancer drugs, radiation therapy, or a combination of the two, depending on the patient's condition and treatment goal. Systemic irradiation (TBI) has traditionally been used as a standard pretreatment for allogeneic hematopoietic stem cell transplantation, but concerns have been raised in that it can lead to long-term complications such as secondary malignancies and endocrine disorders in growing children and adolescents.
Accordingly, the research team conducted a prospective phase 2 clinical trial on 36 high-risk children under the age of 21 from February 2014 to August 2021 to check whether allogeneic hematopoietic stem cell transplantation can be successfully performed only with anticancer drugs without radiation and to evaluate its effectiveness and safety.
The research team used three anticancer drugs: Busulfan, Fludarabine, and Etoposide to perform customized anticancer drug pretreatment tailored to the patient's condition, and the dose of the attachment plate was adjusted according to the patient's age and blood level to minimize side effects. Afterwards, healthy hematopoietic stem cells collected from donors were injected through the patient's blood vessels so that new blood cells could be produced from the bone marrow. After transplantation, neutrophil implantation took an average of 10 days and platelet implantation took an average of 13 days, so the success rate of implantation was also very high.
As a result of the study, high survival rates and safety were confirmed without the use of radiation. The 5-year survival rate (OS) was 86.1%, and the 5-year event-free survival rate (EFS) was 63.9%. This is superior to the 3-year survival rate (62-79%) of allogeneic hematopoietic stem cell transplantation in children and adolescents reported by the existing International Blood/Myeloid Transplantation Center. In particular, we also recorded better results when compared to the 2-year survival rate (75%) of pretreatment therapy using only drugs reported in the FORUM study.
In addition, the incidence of acute graft versus host disease (GVHD) was 36.1%, severe acute GVHD was 2.8%, chronic GVHD (moderate-severe) was 8.4%, and non-recurring mortality (NRM) was very low at 2.8%.
The research team evaluated that the use of radiation has reduced long-term side effects such as secondary cancer and growth disorders that can be caused by existing systemic radiation, and is significant in that it can provide safer treatments for pediatric and adolescent patients.
Professor Hyung-jin Kang (Department of Pediatrics and Adolescents) said, "This study is significant in that it can provide effective treatment to pediatric and adolescent patients through allogeneic hematopoietic stem cell transplantation without radiation. We expect that it will be combined with immune cell treatments such as CAR-T in the future to further improve treatment outcomes."
Professor Hong Kyung-taek (Department of Pediatrics and Adolescents) "It was confirmed that long-term side effects from radiation can be reduced and survival rates can be improved through patient-tailored treatment."The results of this study will provide new treatment opportunities for pediatric and adolescent patients."
The results of this study have been published in the latest online edition of the European Journal of Hemisphere, a world-renowned magazine in the field of hematology, attracting international attention.
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